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ADVANCING ACCESS&STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU&KARNATAKAEXECUTIVE SUMMARYABOUT THE REPORTABOUT THE EQUIHEALTH ALLIANCEKEY TERMSINTRODUCTIONTHEMATIC FOCUSPrevention,Timely Management andBehaviour ChangeData and SurveillanceFinancingINCLUSION OF LIVED EXPERIENCESCONCLUSIONANNEXURE010305060709213528TABLE OF CONTENTSADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAThis report brings together insights from five convenings held in Tamil Nadu and Karnataka,aimed at advancing equitable healthcare access and strengthening Indias response toclimate-sensitive infectious diseases,with dengue as a focal point.The discussions,convened under the EquiHealth Alliance,united governmentrepresentatives,policymakers,healthcare practitioners,community voices,researchers,andfunders to explore actionable pathways across three critical areas:A)Prevention,Timely Management,and Behaviour Change;B)Data and Surveillance;and C)Financing;the report distils cross-sector perspectives into a forward-looking roadmap fornational and sub-national action.On prevention and behaviour change,stakeholders emphasised the importance ofembedding health education within everyday spacesschools,workplaces,and communitynetworkswhile leveraging frontline workers like ASHAs to lead hyperlocal awareness andcare referral systems.Shifting public perception of dengue as a year-round risk,rather than aseasonal outbreak,emerged as essential to lasting behaviour change.The data and surveillance track called for building an integrated,climate-sensitivesurveillance architectureone that decentralises data collection to local health committeeswhile enabling real-time insights through AI-driven tools and citizen reporting platforms.Creating systems that capture nuanced,patient-level data while facilitating coordinationbetween public and private providers was seen as key to early detection and targetedresponse.EXECUTIVE SUMMARY1ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAFinally,the financing discussions stressed the urgency of moving beyond emergency-drivenfunding toward long-term,sustainable investment.Recommendations included leveragingblended finance,establishing benchmark costs for dengue treatment,and reducing out-of-pocket expenses through insurance and digital vouchers.Strategic use of dormantinfrastructure and catalytic funding for innovation pilots were also identified as high-impactenablers.Throughout,the report centres lived experienceof patients,caregivers,frontline healthworkers,and marginalised communitiesas essential to designing accessible,inclusive,andresilient public health responses.The formation of the EquiHealth Alliance is a directresponse to this need:a tactical platform bridging policy and practice,rooted in equity andsystems change.By bridging diverse perspectives,the report highlights actionable recommendations thatalign with broader national and regional health strategies,emphasising the importance ofmulti-sectoral collaboration,policy integration,and sustainable financing models.Theseinsights aim to inform decision-makers,catalyse partnerships,and drive long-terminvestments in equitable and effective healthcare responses.2ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAABOUT THE REPORTThis report is grounded in the urgent need toaddress climate-sensitive infectious diseases,with dengue as a critical and timely lens.Asrising temperatures,altered rainfall patterns,and unplanned urban expansion increase thevulnerability of populations to vector-bornediseases,there is growing recognition thathealth systems must adaptnot just clinically,but structurally and socially.This report reflectsthat understanding,focusing on the intersectionof access,equity,and systemic resilience inpublic health.The insights captured here are the outcome offive strategic convenings hosted across TamilNadu and Karnataka between December 2024 The process began on 10th December 2024 in Chennai with an introductory roundtable thatserved as a contextual mapping exercise.This open-format discussion helped surface variedperspectives on gaps in infectious disease responseparticularly around fragmentation,datalimitations,and under-recognition of frontline challenges.This convening directly led to theidentification of a need for a multi-stakeholder coalition,and set in motion the formation ofthe EquiHealth Alliance.It also shaped the reports three central thematic tracks:and April 2025.These convenings were intentionally designed as collaborative,cross-sectoralengagements,bringing together government representatives,policymakers,healthcarepractitioners,community voices,researchers,and funders to co-create a roadmap forstrengthening healthcare systems.PREVENTION,TIMELYMANAGEMENT&BEHAVIOUR CHANGEDATA&SURVEILLANCEFINANCING3ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKABuilding on this foundation,subsequent convenings deepened the exploration across thesethemes.On 28th February in Tamil Nadu and 7th March in Karnataka,the dialogues focusedon the broader landscape of infectious diseases,mapping common barriers andopportunities across regions and actors.These engagements reinforced the need forstructural shifts in health financing,decentralised surveillance,and community-rootedpreventive strategies.The final two convenings26th March in Chennai and 2nd April in Bangalorewere morefocused,concentrating specifically on dengue.These sessions brought together governmentrepresentatives,policymakers,healthcare practitioners,community voices,researchers,andfunders to share their domain expertise and discuss solutions that are data-driven,climate-aware,and designed for scale.The lived experiences shared in these forums furtheremphasised the importance of embedding equity and accessibility in every aspect of theresponse.This report is structured thematically,rather than chronologicallyreflecting the threads thatemerged organically through dialogue.It integrates insights,recommendations,and frontlinerealities into a collective vision for strengthening dengue response and public healthresilience more broadly.The result is not just a documentation of events,but a strategicsynthesisa working roadmap shaped by the very actors who will carry it forward.4ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAABOUT THEEQUIHEALTH ALLIANCEThe EquiHealth Alliance is a multi-stakeholder advisory platform committed to advancingequitable access to healthcare for infectious diseases,with an immediate focus on dengue.Born out of a contextual mapping convening in December 2024,the Alliance brings togetherexperts from public health,epidemiology,community engagement,financing,andbehavioural science to co-create evidence-based,scalable solutions.To address the criticalneeds in healthcare,EquiHealth Alliance was initiated as an eco-system building effortcommitted to bridging the gaps in public health responses.With a focus on equitable healthcare access,data-driven decision-making,sustainablefinancing and sustained policy engagement,EquiHealth Alliance stands as a catalyst forchangeensuring that solutions are not just formulated but also effectively implemented atscale.Structured around three core workstreamsPrevention,Timely Management andBehaviour Change;Data and Surveillance;and Financingthe Alliance plays a catalytic role inshaping inclusive,climate-aware healthcare strategies.With Tamil Nadu and Karnataka asinitial focus states,the Alliance aims to inform national policy,support implementation,andmobilise sustainable resources through cross-sector partnerships.Its diverse membership includes government representatives,policymakers,healthcarepractitioners,community voices,researchers,and funders.The members bring their diversestrengths to tackle multiple aspects of this challenge,from research and surveillance tofunding and on-the-ground implementation.The Alliance is housed at AVPN,with ETI servingas technical partner,ensuring both independence and rigor in its approach.5ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKA6Vector-Borne DiseasesIllnesses caused by pathogens transmitted throughvectors such as mosquitoes.Community EngagementStrategies that involve local populations in designingand implementing health interventions.Surveillance SystemsData collection mechanisms to track disease incidenceand inform public health responses.Outcome-Based FinancingFinancial models that link funding to measurablehealth outcomes.Public-Private Partnerships(PPPs)Collaborations between government entities andprivate sector organisations to enhance healthcaredelivery.KEY TERMSADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKADengue remains one of the most pressing public health challenges,with its incidence risingsharply over the years due to rapid urbanisation,climate change,and gaps in vector controlmeasures.India alone witnesses thousands of cases annually,placing a heavy burden onhealthcare systems and communities.Despite significant efforts,dengue prevention andmanagement continue to be hindered by fragmented responses,limited cross-sectorcollaboration,and inadequate community engagement.ADDRESSING DENGUE:A CRITICAL PUBLIC HEALTHCHALLENGEDengue is rapidly emerging as one of the mostsignificant public health threats in India,demandingurgent and coordinated action.India alone accountsfor nearly one-third of the global dengue burden,outof an estimated 100400 million infections annuallyworldwide 1.The trajectory is sharply escalatingfrom 28,066 cases in 2010 to 289,235 reported casesin 2023marking a tenfold increase within just over adecade 2.This alarming rise is compounded by theimpact of climate change,which has intensified thebreeding cycle and infectiousness of dengue vectors.Research shows that rising temperatures acceleratemosquito reproduction,reduce larval developmenttime,and increase bite frequency,while also allowingthe dengue virus to replicate faster within mosquitoes,thereby enhancing transmission rates.Further,theIPCCs Sixth Assessment Report warns of a geographicand altitudinal shift in the dengue burden,withchanging rainfall patterns and rising temperaturesfacilitating the spread of the disease to newer regions,including higher altitudes previously unaffected 3.7ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKATogether,these dynamics underscore the need for a paradigm shift in Indias dengueresponsefrom reactive containment to prevention-focused,community-rooted,andclimate-resilient strategies.Recognising the urgency of this challenge,Tamil Nadu and Karnataka were selected as thestarting points for this initiative based on both the scale of their dengue burden and theopportunity for impactful,state-level action.In Karnataka,15,565 cases of dengue fever werereported in Bengaluru alone in 2024,with an additional 16,156 cases across other districtshighlighting the particular vulnerability of urban populations 4.Meanwhile,Tamil Nadureported 26,740 cases and 13 dengue-related deaths as of December 26,2024,marking thehighest number of recorded cases since 2012,according to the Directorate of Public Healthand Preventive Medicine 5.These numbers are not only alarming in scale but signal deeper systemic challenges indisease surveillance,preventive infrastructure,sustainable funding,and public healthcommunicationmaking both states critical learning grounds for shaping a scalable,multi-stakeholder response.Both Tamil Nadu and Karnataka also offer enabling policy environments,strong public healthinstitutions,and vibrant community networks.These strengths provide fertile ground forpiloting integrated strategies across prevention,surveillance,and financingstrategies thatcan ultimately be replicated and adapted across other Indian states and regions facing similarrisks.We hope that the initiatives and roadmaps developed through these efforts will not onlyenhance dengue response in Tamil Nadu and Karnataka but also serve as scalable models forreplication in other states and at the national level.By fostering collaboration across sectorsand empowering communities with the right tools and knowledge,we aim to drive systemicchange in how dengue and other public health threats are addressed across India.8ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAFINANCINGFOCUSTHEMATIC AREASPREVENTION,TIMELYMANAGEMENT&BEHAVIOURCHANGEDATA&SURVEILLANCEADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKA9PREVENTION,TIMELYMANAGEMENT,&BEHAVIOURCHANGEEmpowering ASHA workers and local community health leaders todrive prevention efforts was widely acknowledged as a successfulstrategy.Lessons from Gorakhpur demonstrated that frontline healthworkers can serve as key influencers in promoting the use of mosquitonets,eliminating breeding sites,and facilitating early symptomrecognition.Many discussions underscored the importance of equipping primaryhealthcare facilities with rapid diagnostic tools.Timely diagnosis notonly improves patient outcomes but also prevents unnecessaryhospitalisations,reducing the burden on tertiary care centres.Shifting community perception of dengue from a seasonal outbreakto a year-round threat is critical.Integrating BCC strategies into schoolcurriculums,workplace policies,and local governance mechanisms wasidentified as an effective approach to drive sustained behaviouralshifts.Prevention remains the most cost-effective and scalable strategy for controllingdengue outbreaks.Discussions under this track emphasised the urgent need tointegrate behaviour change strategies into public health interventions,ensuringcommunities adopt preventative measures in a sustainable manner.KEY INSIGHTSCommunityEngagementEarly Detection&TimelyManagementBehaviour ChangeCommunication(BCC)10ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKANeed forAwarenessPublic awareness campaigns should be strengthened to educatecommunities on dengue prevention,early symptoms,and availablehealthcare services.These initiatives should be multilingual andtailored to different socio-economic groups.Involvement ofKey CommunityGroupsStudents,Resident Welfare Associations(RWAs),and Self-HelpGroups(SHGs)should be actively involved in dengue preventionefforts.Schools and colleges can conduct awareness drives,RWAscan mobilise local action for vector control,and SHGs can supportcommunity-based surveillance.StricterEnforcementPracticesEffective dengue prevention requires a combination of enforcementand incentives.Municipal authorities should ensure strictcompliance with vector control measures through fines,while alsoencouraging participation through incentive programs such ascommunity competitions rewarding dengue-free localities.Workplace&InstitutionalResponsibilityEmployers,universities,and public institutions should integratedengue prevention protocols within their facilities.This includesregular inspections,provision of mosquito repellents,and activeparticipation in awareness initiatives.Effective dengue control demands coordination between healthdepartments,urban planning authorities,sanitation workers,andcommunity organisation.Strengthening multi-sectoralpartnerships will ensure a holistic approach to prevention.Cross-SectoralCollaborationProper SourceIdentificationShifting community perception of dengue from a seasonal outbreakto a year-round threat is critical.Integrating BCC strategies into schoolcurriculums,workplace policies,and local governance mechanismswas identified as an effective approach to drive sustained behaviouralshifts.11ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAPromote ASHA-led initiatives like“DryDay”campaigns for eliminating stagnantwater and support the adoption ofcommunity-friendly innovations such asthe self-degrading Eco Bio Trap(presented in the Bangalore Convening)in collaboration with local authorities.SOURCEREDUCTION&ECO-INNOVATIONSReplace punitive enforcementstrategies with positivemotivators,such as school-levelcompetitions and communitychallenges to foster awarenessand compliance.INCENTIVISEDENGAGEMENTDevelop referral systems thatensure continuity of care afterinitial screening and diagnosis,particularly at the community andprimary care levels.In this system,it is important to connect localrepresentatives,SHGs,RWAs etc.STRENGTHENINGTHE CARECASCADEMs.Mallika Bidappa Tharakan,speaking from her experience working withcommunities through Karnataka Health Promotion Trust(KHPT)added,We talkabout saturating the number of screenings.But the real success of having aninitiative that is community-driven and community-led is in closing the loop of care.Ifwe start increasingly focusing on the multiple roles different stakeholders can play atthe grassroots level in not just screening,but also referring to and having a system inplace where functionaries at the grassroots continue to work with communities toclose the loop of care-that shift in thinking is important to break the cycle.12ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKASUGGESTED ACTIONABLE OUTCOMESShift from individual-centric tofamily-based health messaging,recognising the limitations ofsolely targeting women inhouseholds where they may notbe the final health decision-makers.FAMILY-CENTREDCOMMUNICATIONMODELSAmplify the role of caregiversparticularly women like ASHAsas visible leaders of preventioncampaigns to build credibilityand increase uptake.INCLUSIVELEADERSHIP INCAMPAIGNSEmbed local traditionalknowledge in awareness effortsto prevent top-down impositionof external narratives andensure cultural resonance.COMMUNITY CO-OWNERSHIP OFAWARENESSProvide easy-to-navigatepocketbook-style reference guidesfor clinicians,particularly those inPHCs,to improve risk assessmentand case management.PRACTICALCLINICALGUIDELINESUtilise self-help groups and other grassroots collectives to build aculture of routine health-seeking behaviour,making care accessibleand stigma-free for marginalised groups,including persons withdisabilities and LGBTQ communities.EMBEDDING HEALTH IN LOCAL SOCIAL STRUCTURES13ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKADATA&SURVEILLANCEEffective surveillance mechanisms arefoundational to controlling dengueoutbreaks and ensuring timely responses.The discussions in this track centred onleveraging technology,strengthening data-sharing frameworks,and ensuringtransparency in disease monitoring.The integration of AI-driven surveillance tools and real-time datatracking was highlighted as a game-changer.Pilots in select urbanareas have demonstrated that predictive modelling based on climateconditions,vector breeding patterns,and reported cases cansignificantly enhance early warning systems.Digital HealthInnovationsThe need for seamless coordination between municipal bodies,public health agencies,and private sector players was stronglyemphasised.Data standardisation and interoperability betweendifferent surveillance platforms remain key priorities for future action.Decentralised data collection and reporting at the district level werenoted as crucial for effective dengue response planning.Many expertsadvocated for strengthening municipal capacity to analyse and act uponlocalised disease trends.InterdepartmentalCoordinationRole of LocalGovernments14ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAKEY INSIGHTSCommunity engagement in disease surveillance can be enhancedthrough mobile reporting applications and citizen-driven datacollection.Encouraging the public to report potential breeding sitesand symptoms can help refine outbreak response strategies.Strengthening surveillance requires triangulating data from multiplesources,including hospital admissions,laboratory reports,andcommunity-reported cases.This approach enhances accuracy andallows for better-targeted interventions.To improve household-level surveillance,a structured House Indexcan be developed to assess risk levels based on environmentalconditions,previous case history,and sanitation practices.Thiscan guide targeted vector control measures and communityengagement efforts.Cost Transparency&StandardisedTreatmentA key gap identified in data collection efforts is the lack ofstandardised cost data for dengue treatment across differenthealthcare facilities.Establishing a uniform cost framework canimprove affordability and reduce disparities in care.Public ReportingMechanismsTriangulation ofDataCreation of aHouse Index15ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKALeverage existing mechanisms likeVillage Health Sanitation Committeesand Ward Health Committees fordecentralised data collection andmonsoon-season inspections.REACTIVATELOCALCOMMITTEESMove beyond aggregate casenumbers to incorporate patient-wise variables that allow for betterepidemiological insights andcase tracking.ENHANCEDDATASPECIFICITYDr.Hemant Shewade from ICMR-National Institute of Epidemiology talkedabout the need to have contextual,patient-based variables,along with systems-level understanding of surveillance to make meaningful changes.He opined,Physicians are overburdened.Without health systems intervention,especially forsurveillance mechanisms in place,there can be no meaningful use of numbers anddata.Under the NHM,we have village health nutrition and sanitation committees,and ward committees on health.Only when these committees are empowered,canwe make a sustainable change instead of creating parallel systems.SUGGESTED ACTIONABLE OUTCOMES16ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAEstablish user-friendly platforms suchas hotlines or WhatsApp-basednotifications to engage private healthcareproviders and encourage active reporting.Build in models like the Rapid ResponseTeam(RRT)for follow-up aroundidentified households,as suggested bythe PATH teams work in Uttar Pradesh.PRIVATE SECTORNOTIFICATIONMECHANISMSHarmonise data systems(Integrated Disease SurveillanceProgramme(IDSP),IntegratedHealth Information Platform(IHIP),National Center for Vector BorneDiseases Control(NCVBDC)andbuild district-level analyticalcapacity to clean,verify,andtriangulate surveillance databefore escalation.INTEGRATEDSURVEILLANCEARCHITECTURECreate a trained pool of data collectorsfrom across disease programs.Thislearning was particularly influenced byIndias serosurveillance success duringCOVID-19,which was achieved byutilising the TB surveyors.TASK SHIFTINGFOR EMERGENCYDATA COLLECTIONPromote data transparency byeliminating punitive attitudesamong bureaucrats and enablingfree access to accurate healthdata.DISINCENTIVISINGUNDERREPORTING17ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAFINANCINGSustaining dengue prevention andmanagement efforts requires innovativefinancing models that go beyond short-term,emergency-driven funding.This trackfocused on identifying avenues for long-term investment in public healthinfrastructure and disease controlmeasures.Leveraging public-private partnerships to mobilise resources fordengue response was identified as a critical strategy.The role ofcatalytic capital in scaling up effective interventions was particularlyemphasised.Blended FinanceApproachesExploring microinsurance schemes and integrating dengue coverageinto broader health insurance frameworks emerged as a potentialpathway to improve financial resilience against the disease.Insurance&RiskPoolingMany discussions underscored the importance of shifting from reactive,outbreak-driven funding to proactive,long-term investments in vectorcontrol measures,such as improved urban planning,wastewatermanagement,and innovative mosquito control technologies.SustainableInvestments inVector Control18ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAKEY INSIGHTSMaulik Chokshi from Access Health International remarked as he was summarising thediscussion of the financing group,“Let us get into a standard costing based on the standardtreatment guidelines to determine what the cost of an episode of Dengue is.The morbidity pattern may vary from individual to individual,so let us create a risk adjustment formula which can help in mitigating these differentials.We need to design a package that is not only input-based,but also morbidity-based,as at the moment,it is only infrastructure-based input costing and not morbidity-based costing.This will require doctors,hospitals and patient advocacy groups to reach a standard costing outcome that could help in meeting the funding requirement;if we want to involve the private sector,this can become a financing instrument.”A significant gap highlighted was the absence of a standardised coststructure for dengue treatment across healthcare systems.Establishing such benchmarks would not only promote pricetransparency but also help define fair reimbursement rates underinsurance and public health programs.Reducing Out-of-PocketExpenditureParticipants raised concerns about high out-of-pocket costs incurredby families,especially in private health facilities.Strengthening publicprovisioning,regulating pricing in private settings,and ensuringcoverage for diagnostics and treatment in government schemes weresuggested to mitigate this burden.Need for aStandardTreatment CostFrameworkLong-term dengue control requires sustained investments in trainingand retaining healthcare workers,particularly at the community level.Dedicated funds for capacity building,incentives for field-levelengagement,and upskilling in disease management were emphasisedas critical enablers.UnequalDistribution ofPublic HealthFinancingStakeholders pointed out the mismatch between disease burden andpublic health investment across states.States with high dengueincidence often receive disproportionately low allocations.There is anurgent need to align public financing with epidemiological data toensure equitable resource distribution.Financing HealthWorkforce&Capacity Building19ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAImplement digital voucher systems thatreduce out-of-pocket expenditure whilesimultaneously incentivising privatesector participation,which helped in thecase of tuberculosis.DIGITAL VOUCHERSFOR IMMEDIATESUPPORTAlign intervention needs withappropriate fundersutilisegovernment schemes for large-scale programs and exploreflexible sources like family officesfor non-restrictive initiatives,asthese funders may not be curtailedby reporting timing and overheadslike that of CSR funding.TAILOREDFUNDRAISINGAPPROACHESRepurpose underutilised assets,such asCOVID-era diagnostic machines,for usein dengue testing and surveillance.MAXIMISE USE OFDORMANTINFRASTRUCTUREDevelop training and fellowshipprograms for communityvolunteers,offering small user feesto foster ownership and build apipeline of future health leaders.INCENTIVISINGCOMMUNITYPARTICIPATIONLeverage catalytic philanthropy capitalfunding for early-stage innovation pilots,with an aim for eventual governmentscale-up.Another example given was thatof Pharma industry acting as a seedfunder to generate evidence to be furtherfuel government schemes.PILOT&SCALEINNOVATIONDefine and disseminatebenchmark costs for denguetreatment across public,private,and NGO-run healthcare systemsto increase transparency andaffordability.STANDARDISEDTREATMENTCOSTS20SUGGESTED ACTIONABLE OUTCOMESADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAThe dengue response cannot be complete without centring the lived realities of those mostaffected by the diseasepatients,caregivers,frontline workers,and community leaders.Integrating these voices not only enhances the relevance and acceptability of interventions butalso sheds light on systemic gaps often overlooked in top-down policy design.INCLUSION OF LIVEDEXPERIENCESPATIENT VOICESPatients and caregivers repeatedly emphasised the challenges of accessing timely diagnosisand affordable treatment.Stories from urban and peri-urban communities highlighted theimpact of delayed testing,misinformation,and reliance on informal health providers.Several patients shared that dengue was under-recognised in the initial stages,leading tocomplications and higher treatment costs.In many cases,families had to travel across districts to reach better-equipped hospitals,resulting in lost wages and compounding financial stress.Another common point ofenquiry was the absence of vaccine,wherein multiple patients remarked that the availabilityof a vaccine would have eased their treatment journey.Another subtle implication was that of theunderlying emotional and mental costs of thedisease.Some patients reported that addressingthe anxiety attached to the disease itself was apart of their journey to recovery whereas othersremarked that prolonged exposure to thedisease has significantly affected their mentalstate.21ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAGENDERED IMPACTWomen,especially mothers andfemale caregivers,bore adisproportionate burden duringdengue outbreaks.They often actedas primary caregivers for affectedfamily members while simultaneouslymanaging household responsibilitiesand dealing with inadequate access toformal healthcare.In rural settings,women reportedbeing the last to seek treatment forthemselves,often prioritising childrenand male family members.Menstrualhygiene during hospital stays andvector control activities around waterstorage(traditionally managed bywomen)were cited as overlookedaspects of dengue management.The alliance has unanimously suggested thatthere is a need for a gendered analysis of datato see the difference in disease trends as well asresponsibilities in prevention.The transgender community,who is alsorepresented in the alliance,stressed theimportance of addressing stigma as the startingpoint for widening access to healthcare.Therealities of vulnerable communities that do notencourage routine health-seeking behaviour wasemphasised,suggesting the need for targetedprevention and awareness strategies for differentmarginalised communities.Despite their critical role in preventionmanaging cleanliness,overseeing water use,and recognising early symptomswomen inleadership roles were often absent from planningand feedback mechanisms.22ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAFRONTLINE HEALTH WORKERSAccredited Social Health Activists(ASHAs),Auxiliary Nurse Midwives(ANMs),and community volunteers played a pivotal role in dengue prevention andsurveillance.However,they expressed feeling overburdened,and under-compensated to handle vector-borne diseases.Many described the emotional toll ofworking particularly during outbreaks,and highlighted the challenge of convincinghouseholds to participate in source reduction activities.Despite these challenges,their deep-rooted community presence made them trusted agents of behaviourchange.While recognising the incredible work done by ASHAs,the alliance alsopointed out the need for task-shifting to include student and community volunteers.YOUTH&COMMUNITY LEADERS AND CIVIL SOCIETY ORGANISATIONS(CSO)Local youth volunteers were cited as powerful allies in community clean-up drivesand awareness campaigns.The CSOs working at the grassroots expressed the needfor structured platforms to contribute more meaningfully to public health efforts.Special focus was laid on understanding and incorporating traditional and culturally-sensitive information and methods in creating awareness campaigns,rather thanimposing a one-size-fits-all solution.Engaging with local influencers emerged as acrucial factor in designing culturally responsive interventions.PERSONS WITH DISABILITIESThe members of the alliance emphasised on the need to ensure inclusion at everystep of the way.The Information,Education,and Communication(IEC)materialscreated will have to be in Braille and there needs to be the inclusion of signlanguage interpreters in these awareness campaigns.In addition,it is imperative toensure accessible infrastructure,especially in cases like a vaccination drive,asillustrated through the experience with COVID-19.23ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKA24Dr.Deepa,who has livedexperience of disability and hasdone extensive work on extendingaccess to people with disabilitiesstrongly asserted the need toconsider tools and mechanisms fordisability inclusion as an integralpart of any effort on healthcareaccess enhancement.Sheremarked,“As a practitioner,mostof the hospitals I have seen-Primary Health Centres,CommunityHealth Centres and even districthospitals-most of them areinaccessible.If we are consideringuniversal accessibility,signlanguage interpretation is oneimportant aspect,becauseotherwise people with hearingimpairment cannot convey themessage to the doctor;a braillefacility and physically accessibleinfrastructure is another suchexample.Even if you havevaccination facilities,if it is on thesecond floor with no arrangementsfor people with motor disabilities,the access is effectively curtailed.”Mr.Piyush Kothari,father of Harshil Kothari,a 10-year old boy who battledmultiple organ failure following dengue remarked that his first thought was tosecure a vaccine,which upon enquiry,he understood was not available.Followingup on the need to have health systems prepared for vaccines,he added,“I wantthe government to improve infrastructure in all the hospitals so that all thenecessary facilities are available in-house.It is a disturbing process to keep shiftingfrom place to place amidst such a difficult time.”TAKEAWAYS FOR SYSTEM DESIGNADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAThese lived experiences underscore the need to design dengue responsemechanisms that are not only technically sound but also socially embedded.Incorporating patient feedback loops,gender audits of interventions,and frontlineworker consultations in program design will enhance both uptake and outcomes.Building a responsive system means listening to those who live at the intersectionof health,poverty,and vulnerabilityand allowing their realities to shape how weprepare for and respond to public health crises.25ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKARECOMMENDATIONSPREVENTION,TIMELY MANAGEMENT,&BEHAVIOUR CHANGEInstitutionalise Behaviour ChangeCommunication(BCC):Integrate BCCstrategies across schools,workplaces,and community spaces to promote year-round dengue prevention.Embed Inclusivity in PreventionEfforts:Ensure that preventionstrategies are inclusive and accessible toall communities.This includes promotinginclusive leadership by amplifying therole of women,especially frontlineworkers like ASHAs;creating braillematerials and sign language support tomake IEC efforts accessible for personswith disabilities;and integrating theneeds and voices of sexual and genderminorities,who often face stigma andlimited healthcare access.Support Innovation and Research:Invest in scalable technologies,andlocally adaptable solutions for vectorcontrol,behaviour change tools,andvaccine development.Document and Share Best Practices:Facilitate inter-state learning by disseminating successful case studies and community-driven models,especially thosefrom Tamil Nadu and Karnataka,as knowledge sharing was identified as one of the keyobjectives for Equi Health Alliance.DATA&SURVEILLANCEPromote Multi-Stakeholder Coordination(cross-cutting but grounded insurveillance):Foster sustained partnershipsbetween government departments,civilsociety,and private sector actors to ensure acoordinated dengue response.Strengthen Local SurveillanceSystems:Empower municipalbodies with technology andanalytical tools to collect,triangulate,and act on localiseddata.A call has also been made tointegrate the varying data systemsavailable to ensure that there is acentralised knowledge repositorythat combines data from multiplesources supported by verifiers.Expand Community-Driven Surveillance:Leverage mobile apps and citizen-reportingmechanisms to enhance early warningsystems and encourage public participation.26FINANCINGStandardise Dengue Treatment Costs:Establish and publish benchmark costsfor dengue care to ensure transparencyand affordability,particularly in privatehealthcare settings.Prioritise High-Burden Areas:Allocateresources and interventions strategically,focusing on districts and urban clusterswith recurrent high incidence.Enhance Financing Mechanisms:Adoptblended finance models,expand healthinsurance coverage for dengue,andensure equitable public financing linkedto incidence rates.Invest in Health Workforce Capacity:Allocate dedicated funding for trainingand incentivising community healthworkers to sustain frontline engagementin dengue control.27ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKACONCLUSIONThe convenings in Tamil Nadu and Karnataka have served as critical learning grounds fordeveloping a more effective,inclusive,and sustainable dengue response.They highlightednot only the urgency of addressing systemic gaps across financing,data,and behaviourchange,but also the importance of community engagement and the lived realities of thosemost affected.These discussions underscored the value of coordinated,multi-stakeholderactionbringing together government bodies,health experts,private sector actors,andgrassroots organisationsto co-create scalable and contextually relevant solutions.The formation of the EquiHealth Alliance marks a pivotal step in this journey.As a tacticaladvocacy movement,the Alliance was created to stand in the gaps that often exist betweenpolicy and practice.It aims to bridge siloed efforts,amplify ground-level voices,and ensurethat public health solutions are driven by principles of equity,resilience,and systemictransformation.Looking ahead,we hope that the insights,partnerships,and momentum generated throughthese convenings will catalyse broader national engagement.Our aspiration is for theEquiHealth Alliance to evolve into a national-level tactical advisory bodyinforming policy,enabling innovation,and supporting state and central governments in building a robust,adaptive response to dengue and other public health challenges.28ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAPARLIAMENTARIANS EXPECTED RESULTSPrioritise Dengue:learning from TB Harega Desh Jeetega and the GlobalCoalition Against TB with more than 50 MPs-prioritise dengue as a challengefor India.Parliament-led discussions:Organise seminars and workshops between MPs,technical experts and communities Monitoring and reporting:Essential that the respective MPA or MNA closelymonitored the implementation of dengue prevention measures in theirrespective union councils,regularly visit the designated areas to assess theprogress,identify any gap or issue and ensure that the required actions werepromptly taken.Commitment to solutions like vaccines:The Honourable PM has oftenreiterated commitment to vaccines for all(COVID 19).There is also prioritisationfor vaccines to counter cervical cancer.The parliamentarians can play a crucialrole to encourage last mile delivery through local governments andprioritisation of dengue response at the highest political decision-making levels.Integrate with disaster management strategies:Indias disastermanagement plans should include specific protocols for disease prevention,particularly in post-disaster situations where stagnant water and debris createideal conditions for mosquito breeding.This protocol could include pre-disasteractions like mapping high-risk areas for potential mosquito breeding andstocking emergency supplies.Establishing early warning systems:Another crucial component of mitigatingrisk is the implementation of early warning systems based on climate models.These systems could use rainfall patterns,temperature changes,and humiditylevels to predict potential dengue outbreaks.Thus,they will be able to allowpublic health officials to take proactive measures,such as increasing publicawareness or initiating mosquito control programmes,and guiding resourceallocation.These systems are also useful for other climate-sensitive diseases 7.WHY IS THIS ROLE IMPORTANT?The unprecedented surge in dengue epidemics acrossthe globe in recent decades prompted WHO at the startof 2019 to include the virus in its list of the worlds top10 public health threats-India needs to focus on thesame30ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKADONORS(Ministry of Health andFamily Welfare,Ministry ofJal Shakti and researchbodies like ICMR)EXPECTED RESULTSPPP:Public-private partnerships can beeffectively used to control the high incidenceof dengue.Build and launch a pilot andgovernment authorities collaborating withprivate businesses in the biocontrol sector tocombat mosquito populations in rural andurban water bodies.Clean Up Campaigns:Fund clean upcampaigns in selected areas.Also fund mobilehealth centres could provide rapid diagnostictesting for dengue,distribute insecticide-treated bed nets and water container covers,and educate communities about diseaseprevention.WHY IS THIS ROLE IMPORTANT?Funding will help enhance research,advocacy and helpto design and implement future interventions to reducedengues growing burden and impact on health.As climate changeintensifies,it is reshapingthe epidemiology ofvector-borne diseaseslike dengue,necessitating a proactiveadaptation in Indiaspublic health response.EXPECTED RESULTSFunding for Dengue Control Solutions:Support the development anddeployment of cutting-edge solutions across the entire dengue responsespectrum.This includes vector-level innovations such as the use of Wolbachiabacteria to block virus transmission in mosquitoes;infrastructure improvementsto reduce breeding grounds(e.g.,stormwater drainage and solid wastemanagement);and human-level interventions such as improved diagnostics,drugs,therapeutics,and broader health system strengthening,includingworkforce capacity and last-mile delivery models 8.31ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKA32CIVIL SOCIETYORGANISATIONSEXPECTED RESULTSWHY IS THIS ROLE IMPORTANT?Community engagement is critical to the success andsustainability of dengue response-prevention,treatment and controlCreated by Eucalypfrom the Noun ProjectFrontline heroes:ASHA workers can lead community-based researchinitiatives,engaging local women and youth groups in data collection,environmental monitoring,and mapping mosquito breeding sites.Technology integration:Telemedicine must be better integrated with localhealth systems in rural areas to support rural health workers and patients.ASHA workers can be tasked with using mobile health applications given theclose connection with the community.Research:ASHA workers can lead community-based research initiatives,engaging local women and youth groups in data collection,environmentalmonitoring,and mapping mosquito breeding sites.Championing solutions:Learning from COVID help build a conduciveenvironment for demand for new solutions including therapeutics and vaccinesLived experiences:Build a cohort of champion voices of survivors from allspaces,including most marginalised and help the platform reach decisionmakers and general public 33MEDIAEXPECTED RESULTSWHY IS THIS ROLE IMPORTANT?Sustained and informed reporting by the fourth pillarof Indias democracy has enabled issues to beprioritised with public and decision makers alike.Thedengue response story needs the same impetus.Climate change impact:Climate,specifically temperature,affects the vectorsability to cause and sustain outbreaks,and how the infectiousness of dengue isinfluenced by climatic change.India is a hot bed for this and must be prioritisedas an issue Storytelling:Bringing to forefront the stories of suffering,pain andhelplessness must be presented to the nation and beyondPrevious learnings:Media should become champions of change as was in thetime of polio eradication and other instances-where communication based onscience highlighted key issuesADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAEND NOTES1 https:/ 2 https:/www.orfonline.org/expert-speak/climate-change-and-the-rising-incidence-of-dengue-in-india3 https:/ https:/sansad.in/getFile/loksabhaquestions/annex/183/AU831_Prehwr.pdf?source=pqals 5 https:/ https:/www.who.int/news-room/feature-stories/detail/who-scales-up-response-to-worldwide-surge-in-dengue7 https:/www.orfonline.org/english/expert-speak/climate-change-and-the-rising-incidence-of-dengue-in-india8 https:/www.orfonline.org/english/expert-speak/climate-change-and-the-rising-incidence-of-dengue-in-india34ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAANNEXUREEQUIHEALTHALLIANCE MEMBERS35ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKANAMEDESIGNATIONORGANISATIONDr P SampathAdditional Director-cum-State Program OfficerTamil Nadu Program,NationalCenter for Vector BorneDiseasesDr.Dalbir SinghPresidentGlobal Coalition Against TBDr.Rajni Kant SrivastavaICMR Chair-DiseaseEliminationICMR,New DelhiDr.Girija VaidyanathanIAS(Retd)&Former ChiefSecretaryDr.Nalini KrishnanFounderREACHDr.Usha SriramFounderDIWASSudha SFounderThozhiDr.Hemant ShewandeScientistICMR-National Institute ofEpidemiologyMallika Biddappa-TharakanCore Team Member andLeadKHPTAchintya SrivatsaSr.Program Officer VectorBorne DiseasePATHChirag DuttaLead-Public Affairs andPolicyTakedaVasanth KumarSenior MSL,VaccinesTakedaSujatha NarasimhanLead-Resource MobilisationCatalyst GroupJeyaganesh KState DirectorSwasti,Catalyst GroupTAMIL NADU36ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKANAMEDESIGNATIONORGANISATIONRajeswari DharVice President-Program andImpactSambhav FoundationDr.RamasubramaniamInfectious diseases specialistApollo HospitalsMaulik ChokshiGlobal Director,HealthSystems Research and PolicyAccess Health InternationalDr Mohan KumarSenior Technical OfficerSouth Asia Field Epidemiology andTechnology Network,Inc.(MSSRF)Dr.Alex EapenOfficer in ChargeICMR-National Institute of MalariaResearch(NIMR)-ChennaiDr RamyaAnanthakrishnanDirectorREACHDr.Mohideen AbdulKaderFormerly Chief EntomologistDr.S RaghunanthananProfessor of MedicineMadras Medical College(Retd)Dr.C RajendiranInfectious Diseases SpecialistBillroth HospitalsPrachi SharmaSenior Program ManagerWomen Lift HealthRadhika RameshCOOVillgro FoundationSharun AManagerVillgro FoundationDr.Vidya KrishnaInfectious diseases specialistApollo HospitalsDr Suma ShivakumarConsultantNTEPDr Chethana ThirthahalliTechnical Lead,HealthHCL Foundation37ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKANAMEDESIGNATIONORGANISATIONSukriti ChauhanCEOETI Consulting Pvt LtdStephy StephenResearch AnalystETI Consulting Pvt LtdManasi PrabhakaranResearch AnalystETI Consulting Pvt LtdBhawani MauryaSenior Manager,Health ImpactPlatformAVPNSejal MaheshwariManager,South AsiaAVPN38ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKANAMEDESIGNATIONORGANISATIONDr Naveen ThackerPresidentInternational PediatricAssociationDr Rajnikant SrivastavaICMR-Chair,Disease EliminationICMRDr.Vani H CScientist D&Officer In ChargeICMR-NIMR,Field Unit,BangaloreDr.Shrinivasa BMScientist CICMR-NIMRDr.Dalbir SinghPresidentGlobal Coalition Against TBDr Lokesh NagarajHealth Officer,MahadevapuraZoneBruhat Bengaluru MahanagaraPalike(BBMP),BengaluruVishwanath ReddyPortfolio Manager-StrategicPartnership&ManagementAshraya Hastha TrustPraveen B.Senior Program OfficerWipro FoundationKARNATAKA39ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKAGayana LakshmipathyInvestment AssociateYunus Social BusinessManohar ElavarthiFounderSangama TrustRajesh SrinivasExecutive DirectorSangama TrustVeenaCommunity RepresentativeSangama TrustNitish KumarTeam Lead-Data Science(Evidence for change)Catalyst Management ServicesNAMEDESIGNATIONORGANISATIONShruthiFounder MemberRishika Charitable TrustDr Bhaskar RajakumarProgram DirectorArtparkShankar AGDirector of ProgramsSwastiSabhimanvi DuaProgramme Manager&HealthPractice AssociateSwastiDr.Tanmaye RameneniProgramme AssociateSwastiAkash DeyInfectious Diseases LeadClinton Health Access InitiativeAnshu RainaManager:Healthcare VerticalVillgro Innovations FoundationDr Swaroop NLead for comprehensive primaryhealthcareKHPTDr.Susanta KumarGhoshResource Person(ExternalSupport)AdvisorGovernment of KarnatakaDr.Deepa VAssistant Registrar,NursingSectionRajiv Gandhi University ofHealth Sciences;40ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKADr Taslimarif SaiyedDirector&CEOC-CAMPVishal BharadwajProgramme LeadC-CAMPChoo BengHead of Medical AffairsTakedaAnjana NarainHead,Takeda VaccinesTakedaKomal GuptaSenior ManagerSambhav FoundationNAMEDESIGNATIONORGANISATIONG D GirishaState EntomologistGovt.of KarnatakaAbhijeet WaghmareDirectorPiramal SwasthyaDr Lokesh VHead of Unit,Radiation OncologyKidwai Memorial InstituteKrithika SambasivanLead,Disability ResearchPACTASukriti ChauhanCEOETI Consulting Pvt LtdStephy StephenSenior Research AnalystETI Consulting Pvt LtdManasi PrabhakaranResearch AnalystETI Consulting Pvt LtdMadhavika BajoriaExecutive Director,Health andNutrition PlatformAVPNBhawani MauryaSenior Manager,Health ImpactPlatformAVPN41ADVANCING ACCESS AND STRENGTHENING DENGUE RESPONSE:LESSONS FROM TAMIL NADU AND KARNATAKACompilation&Conceptualisation:Madhavika Bajoria,Executive Director,Health Impact Platform,AVPNBhawani Singh Maurya,Senior Manager,Health Impact Platform,AVPNSukriti Chauhan,CEO,ETI Consulting Pvt.Ltd.Stephy Stephen,Senior Research Analyst,ETI Consulting Pvt.LtdManasi Prabhakaran,Senior Research Analyst,ETI Consulting Pvt.Ltd.infoavpn.asiaavpn.asia171 Tras Street,Union Building,Level 10,Singapore,0790252AVPN Limited is registered in Singaporeas a charity(UEN 201016116M)AVPN is a unique funders network committed to building a vibrant andhigh impact social investment ecosystem across Asia.AVPN is catalysingmore strategic and collaborative social investment from philanthropy toimpact investing,addressing key social challenges facing Asia today andin the future.
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The Animal Health Industry in AustraliaHow veterinary medicines protect and promote the sustainability and prosperity of Australias social,environmental and economic landscapes.Animal Medicines Australia(AMA)is the industry body representing the leading animal health companies in Australia.AMA member companies are the local divisions of global innovators,manufacturers,formulators and registrants of a broad range of veterinary medicines.These companies work at the cutting edge of animal health science to prevent,control and treat illness and injury across the livestock,equine and companion animal sectors.Products from our members account for 80%of all animal health product sales.The livestock sector is forecast to contribute$37.6 billion to Australias agricultural production in 2024-2025.Animal medicines increase farm productivity and deliver significant and sustainable improvements in environmental,safety and health outcomes.Animal medicines underpin the high quality and safety of animal products for local consumption and for export.In the companion animal pet sector,animal medicines facilitate healthier,longer and better-quality partnerships between people and animals.Data from AMAs Pets in Australia:a national survey of pets and people found there are over 28.7 million pets in Australia with 69%of households owning one or more.About Animal Medicines AustraliaAnimal Medicines Australia MembersAMA members medicines increase farm productivity and deliver significant and sustainable improvements in environmental,safety and health outcomes.2Introduction.4Industry snapshot.5What are veterinary medicines and what do they do?.6Sales by product category.7Ensuring quality and safety.8 Australian animal populations over time.10Benefits of animal health.12Innovation.14Table of ContentsAustralias animal health industries provide veterinary medicines and animal health products that are essential to support the health and welfare of our pets,livestock and other animals within our care.Healthy animals provide benefits to all Australians.Healthy animals:Protect human health by preventing transmission of zoonotic diseases Are more productive,providing economic benefits for livestock producers and rural communities Are more sustainable,requiring less land,water and feed resources Provide enduring and rewarding pet ownership experiencesAustralias animal health industries supply high-quality,safe and effective vaccines,parasiticides,antimicrobials,pain relief and other products.Innovative and independently regulated,consumers can have confidence in the quality and safety of veterinary medicines they use.Community confidence is underpinned by an effective and independent regulatory scheme.The Australian Pesticides and Veterinary Medicines Authority rigorously assesses each product prior to registration or sale in Australia.This independent process seeks to ensure that there are no unacceptable risks to users,consumers,animals,the environment or trade.Our industrys products are essential inputs that support Australias$37.6 billion livestock industries and the health and welfare of 28.7 million pets.Improving the health of our animals is often one of the most efficient mechanisms to addressing many of societys current and emerging challenges.Whether it be addressing global food security,the emergence of zoonotic diseases,mitigating and responding to climate change impacts,meeting community expectations on animal health and welfare or facilitating personal health benefits through meaningful pet ownership experiences,animal health industries are working to supply new innovations to help meet these challenges.On behalf of our member companies,Animal Medicines Australia welcomes opportunities to inform policy making that considers the benefits better animal health brings and celebrates the contribution towards more sustainable communities that will subsequently accrue.IntroductionImproving the health of our animals is often one of the most efficient mechanisms to addressing many of societys current and emerging challenges.04The Animal Health Industry in Australia05Responsible pet ownership relies on access to safe,affordable veterinary medicines and animal health products.Australias pet population has grown to 28.7 million pets-with 6.9 million households having a pet.2Veterinary medicines and animal health products include parasiticides,vaccines,antibiotics,nutritional supplements and analgesics.These products are critical inputs to Australias$37.6 billion1 livestock industries.Producers rely on animal health products to prevent and treat animal diseases and to conduct necessary animal husbandry and management practices responsibly and ethically.By optimising animal health,producers improve productivity,profitability,food safety and sustainability.Similarly,animal medicines protect and treat Australias 28.7 million pets from illness,injury and disease.Responsible pet ownership relies on access to safe,affordable veterinary medicines and animal health products.Industry snapshotProducers rely on animal health products to prevent and treat animal diseases and to safely conduct necessary animal husbandry and management practices responsibly and ethically.Essential for Australias$37.6 billionlivestock industries.11 AWE Agricultural forecasts 2025:.au/client/en_AU/search/asset/10361412 AMA Pets in Australia-A National Survey of Pets and People 2022:https:/animalmedicinesaustralia.org.au/report/pets-in-australia-a-national-survey-of-pets-and-people-2/Manufacture and import value3ImportedManufactured in australiaOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedManufactured in AustraliaImported20%Australian sales by sector-2024Production ANimalsCompanion ANimalsOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedCompanion AnimalsProduction Animals$839m$637m57CWhat are veterinary medicines and what do they do?ParasiticidesParasiticides protect and treat animals for internal and external parasites including worms,fleas,flies and ticks.Parasite infections are often entirely preventable but if not treated or managed will result in decreased production or health,impact food safety and can be fatal.Some examples of parasites are heartworm,tapeworm,ticks,lice and fleas.AntibioticsLike humans,animals can sometimes get sick with a bacterial infection.When this occurs,antibiotics are the only effective tool to treat the disease.Bacterial infections that are treated with antibiotics include salmonella,leptospirosis and staphylococcus.Many antibiotic products are unique to animal health.No antibiotic used to treat human diseases is used for growth promotion in animals.AnalgesicsAn analgesic drug is a pain relieving medication given to prevent,relieve and manage pain in animals.Providing pain relief to animals when conducting necessary animal husbandry practices,or to provide comfort to pets suffering painful injuries or illnesses helps contribute to ethical and responsible animal care practices.Nutrition&metabolismRegistered animal health products include vitamin,mineral and nutritional supplements,growth promotants,dietary/therapeutic pet foods,and digestive enzyme supplements.Whether food producing livestock or a family pet,nutrition plays a significant part in animal health and welfare.Immunotherapy(Vaccines)Vaccination is an effective,inexpensive way to prevent deadly disease.Vaccinations also go beyond protecting the health of a single animal,by preventing spread of the disease.Vaccinations prevent a wide spectrum of animal diseases such as canine parvovirus,feline calicivirus,anthrax,tetanus and pulpy kidney and the use of vaccines in animals is an important step in decreasing the need to use antibiotics.Immunotherapy products also include antisera and antivenom,which can be used to counteract poisonous and toxic substances,such as snakebite venom.Other productsOther categories of animal health medicines include treatments targeting various body systems,such as:musculoskeletal therapies(e.g.anti-inflammatories and muscle relaxants);endocrine treatments(including sex hormones,trophic hormones,and insulin);cardiovascular and digestive system therapies;dermatological products(such as antiseptics and diagnostic agents);and preparations for the ears,nose,and throat.What is a registered product?If an animal health product has a defined clinical medical use or a therapeutic effect,it must be registered prior to being sold,whether manufactured locally or imported.The Australian Pesticides and Veterinary Medicines Authority(APVMA)is Australias independent regulator.The APVMA approves the importation,supply and sale of every veterinary medicine and formulated product after a risk assessment of appropriate scientific data has demonstrated the product is efficacious and not unduly hazardous to human or animal health or the environment,and that it will not adversely affect trade.More information is available at www.apvma.gov.au The Animal Health Industry in Australia07Major veterinary medicine categories by sales405001000150020002500300035004000OthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedParasiticidesImmunotherapy(vaccines)Antibiotic&relatedOthersNutrition&metabolism21C%5%6%Total sales:$1.54 billion4 APVMA Annual Sales Data:https:/www.apvma.gov.au/about/accountability-and-reporting/annual-product-sales-data/publication-annu-al-product-sales-data-2023-24Total$value of registered products4By financial yearTotal value of products:$1.54 billion0500000000100000000015000000002000000000AntibioticsImmunotherapyNutOtherPar2023-242022-232021-222020-212019-20-20192020-202020212022-2023-20232024-20212022$2b$1.5b$1.0b$0.5bTotal number of registered products4By financial year-20192020-20202021-202120222022-2023-202320240Total number of products:3,616350030002500200015001000500050010001500200025003000350040002013-20142014-20152015-20162016-20172017-20182018-20192019-20202020-20212021-20222022-2023050010001500200025003000350040002023-20242014-20152015-20162016-20172017-20182018-20192019-20202020-20212021-20222022-20232023-202405001000150020002500300035004000AntibioticsImmunotherapyNutOtherPar2023-242022-232021-222020-212019-20ParasiticidesOthersNutrition&metabolismVaccinesAntibiotics08Veterinary medicines and animal health products are highly regulated.They are subject to rigorous,independent regulatory systems.These systems work to minimise risks to users,animals,consumers and the environment.RegistrationBefore any product can be marketed for sale,it must first be registered for use.The Australian Pesticides and Veterinary Medicines Authority(APVMA)is responsible for conducting independent,science-based risk assessments prior to registering veterinary medicines.The APVMA assesses risks for:yUsers:to determine whether the proposed use will present any unacceptable risks to users(such as veterinarians,farmers or pet owners)when using the product,yAnimals:to ensure that a product is safe to use on a treated animal,yConsumers:to determine whether the use will result in any unacceptable residues in animal products,such as meat,dairy or poultry,yThe environment:to consider any potential risk to the environment,including non-target plants,animals and ecosystems,yTrade:to determine whether potential products may risk access to export markets for animal products.The APVMA will only register a product if it is satisfied that any identified risks can be managed.This includes approving the label instructions and precautions necessary to inform responsible use practices.Post-registration control of useAustralias state and territory governments are responsible for controlling how veterinary medicines are used.This includes ensuring that only appropriately trained and qualified people can access and use certain medicines(especially potentially hazardous products)and establishing licensing and other control systems.Good manufacturing practiceAll manufacturers of registered veterinary medicines need to comply with a rigorous manufacturing licencing scheme.This is similar to the manufacturing licensing scheme for human medicines.The scheme ensures the highest manufacturing standards are applied.To obtain and maintain a licence,manufacturers are regularly audited by independent inspectors who examine every aspect of the manufacturing process,including the source and quality of all ingredients,manufacturing equipment and packaging materials.Ensuring Quality and SafetyThe Animal Health Industry in Australia09PharmacovigilanceAnimal health companies operate detailed pharmacovigilance systems that identify and track examples where a veterinary medicine may appear to be ineffective or related to an adverse reaction.These systems allow a rapid investigation of any product quality or safety issue.Similarly,APVMAs Adverse Experience Reporting Program collects reports from users on reactions or poor outcomes from products.Adverse Experience Reporting informs whether product recalls are necessary and can indicate if additional controls are required.Monitoring,compliance and enforcementAll Commonwealth,state and territory government regulatory schemes involve significant reporting and compliance obligations.Animal health companies take great care to ensure that all regulatory obligations are met,recognising that significant penalties can be imposed for failing to meet legislated standards.Other regulatory schemesIn addition to APVMA and state and territory dedicated control systems,multiple other regulatory systems also apply to veterinary medicines.These include:Gene technology regulation Biosecurity and import regulations Poisons scheduling Maximum residue limits(domestic and export destinations)Withholding periods and export slaughter intervals Dangerous Goods transport(air,land and sea)and Dangerous Goods storage Chemical diversion illicit drugs and chemicals of security concern Retail storage Work Health&Safety Trade Measurement Waste managementUsers of veterinary medicine products can be confident that extensive,effective and rigorous regulatory systems are in place to guarantee that registered veterinary medicines,when used in accordance with label instructions,or under veterinary supervision,are safe and effective.10Australias livestock populations are influenced by multiple factors including climate,commodity prices,market conditions and regulatory controls.Veterinary medicines and animal health products are critical inputs supporting these industries.Livestock(2018 to 2024/2025)5Australian animal populations over time5 MLA Industry Projections-Australian cattle:http:/.au/prices-markets/Trends-analysis/cattle-projections/MLA Industry Projections-Australian sheep https:/.au/prices-markets/Trends-analysis/sheep-projections/ABS:https:/www.abs.gov.au/statistics/industry/agriculture/australian-agriculture-livestock/2022-23ACMF Australian Industry Facts&Figures-Chicken Production:Australian Pork-Market Report:https:/.au/market-reports2018202020222024Manufacture and import value32018ChickenOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedAnimal populations over time-Chicken2018 2020 2022 2024Source:https:/chicken.org.au/our-product/facts-and-figures/0100200300400500600700800202020222024/25675.3701707MillionMillionMillionMillionManufacture and import value3BeefOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedAnimal populations over time-BEEF2020 2022 2024 2025Source:https:/.au/globalassets/mla-corporate/prices-markets/documents/trends-analysis/cattle-projections/september-2024-update_mla-australian-cattle-industry-projections_020924.pdf0510152025303520202022202524.629.330.6Manufacture and import value32018DairyOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedAnimal populations over time-DAIRY2018 2020 2022 2024Source:https:/www.abs.gov.au/statistics/industry/agriculture/agricultural-commodities-australia/latest-release#livestock0.00.51.01.52.02.52020202220242.32.42.1Manufacture and import value3SheepOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedAnimal populations over time-SHEEP2020 2022 2024 2025Source:NEW=https:/.au/contentassets/65e47c3c775c43f38007fd894daf2095/revised_march-2025_mla-australian-sheep-industry-projections_130325.pdfOLD=https:/.au/globalassets/mla-corporate/prices-markets/documents/trends-analysis/sheep-projections/september-2024-update_mla-australian-sheep-industry-proj0102030405060708073.263.77679Manufacture and import value32018PorkOthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedAnimal populations over time-PORK2018 2020 2022 2024Source:https:/.au/market-reports01234562020202220245.35.65.8Million202020222024The Animal Health Industry in Australia30.1202420255.787562.111Manufacture and import value3Total households with a pet*OthersParasiticidesNutrition andmetabolismImmunotherapyAntibiotic andrelatedTotal households with a pet2013 2016 2019 2021 2023Source:Pet report0123456785.75.96.86.92016201920212022Million020406080100Manufacture and import value3%of households with a pet*63 13201620192021Percent62ai%6 AMA Pets in Australia-A National Survey of Pets and People 2022:https:/animalmedicinesaustralia.org.au/report/pets-in-australia-a-national-survey-of-pets-and-people-2/024681012Total pets in Australia by type6DogsCatsFishBirdsSmall mammalsReptilesOther20162019202120224.75.16.36.43.83.74.95.311.111.311.211.35.45.45.53.90.50.61.50.90.40.30.60.51.51.80.40.4MillionIn 2022 there were 28.7 million pets in Australia6202269Food SafetySome veterinary medicines and animal heath products are used to ensure animal products are safe to be consumed by humans.This is particularly important for protecting against potentially harmful zoonotic diseases.For example,each year in Australia more than 10,000 people are infected by salmonella bacteria that cause gastroenteritis.Controlling salmonella in poultry production significantly reduces these infections and benefits human health.Benefits of animal healthAnimal health,welfare and social licenceVeterinary medicines are essential to protecting the health and welfare of our pets and livestock.Indeed,good animal health is a precondition to good animal welfare.Animal illness,injury and disease can cause pain,distress and discomfort.By maintaining animal health,we can improve animal welfare of our animals.Animal health products also help meet community expectations around necessary animal husbandry practices.New innovations in animal health and pain relief can mean that necessary husbandry procedures can be done in a way that minimises or avoids pain and distress.Productivity and sustainabilityHealthier animals need fewer land,feed and water resources,increasing productivity for producers while minimising environmental impacts.According to the UN FAO,improved animal health,genetics and husbandry could reduce global livestock emissions by up to 55%.Improving animal health offers a cost-effective and sustainable opportunity for livestock industries to reduce emissions and manage climate risks.The Animal Health Industry in Australia13Human healthAround 70%of new and emerging diseases are zoonotic,meaning that they can spread between humans and animals.Protecting the health of our animals through vaccination,preventative treatments and improved biosecurity measures can prevent zoonotic diseases being transferred to humans.Animal health industries are constantly innovating to provide new products and services that treat new and emerging diseases.Benefits for pet ownersOur pets play multiple,varied and important roles in our lives.They are companions for relaxation,for exercise and for teaching children responsibility.They provide humour,fun and a sense of purpose.Overall,pet ownership helps build happier,healthier and more connected communities across Australia.Animal health products and veterinary medicines are a core component of responsible pet ownership.Responsible and judicious use of these products ensure owners meet the needs of their pets,and can enjoy long,rewarding and beneficial ownership experiences.TradeResponsible use of veterinary medicines and animal health products can ensure that animal sourced food and fibre meets standards required by export markets,facilitating and supporting productivity and sustainability of agricultural industries which are critical to Australias economy.14A new medicine or product can take anywhere from five to 15 years7 and tens of millions of dollars($USD)in research and development from first discovery until it is available to treat an animal.This requires significant investment by companies to identify,research and develop new products.This includes conducting tests and trials required to generate data to demonstrate to a regulator that a product is safe and efficacious.Animal health companies continue to innovate in different ways to provide new products and services for animal owners.Innovation can mean using existing products in new ways,developing new application and use tools to enhance safety,addressing sustainability and waste issues through improved packaging and recycling or by using genetics,data monitoring and surveillance systems to address animal health issues before clinical indications of illness are present.Whatever the form a new innovation takes,it will be supported by extensive research,development and testing.Animal health companies support extensive Australian research activities that ensure quality data supporting Australian use patterns can be assessed by regulators.Future innovations,such as the digital revolution in predictive,monitoring and veterinarian diagnostics technology,will reduce health threats and continue to provide safer,more effective and more sustainable products to meet an ever-widening range of animal health challenges.Innovation7AHE Global Benchmarking Survey 2020:https:/animalhealtheurope.eu/resources/publications/global-benchmarking-survey-2020-im-pact-on-competitiveness-of-the-animal-health-industry/A new medicine or product can take anywhere from five to 15 years and tens of millions of dollars in research and development from first discovery until it is available to treat an animal.The Animal Health Industry in Australia15Copyright All copyright subsisting in the contents of this publication is owned by Animal Medicines Australia and may not be reproduced without the prior consent of Animal Medicines Australia Pty Ltd.Where permission is granted,it will be subject to the requirement that Animal Medicines Australia Pty Ltd is acknowledged on any reproduced or quoted material,whether in whole or in part.All rights reserved.ISBN:978-0-646-71795-1Leaders in animal health16Animal Medicines Australia Pty LtdLevel 2/15 National CircuitBarton ACT 2600AustraliaE enquiriesanimalmedicines.org.auT 61 2 6257 9022W animalmedicinesaustralia.org.auanimalmedicinesaustraliaAnimalMedOzAnimalMedicinesAustralia
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AACR CANCER PROGRESS REPORT 2024AACR.org|CancerProgressReport.org#CancerProgressReportCANCER PROGRESS REPORT 2024Inspiring Science.Fueling Progress.Revolutionizing Care.Spotlight on CANCER IN CHILDREN,ADOLESCENTS AND YOUNG ADULTSPage intentionally left blank.AACR.org|CancerProgressReport.org#CancerProgressReportCANCER PROGRESS REPORT 2024Inspiring Science.Fueling Progress.Revolutionizing Care.PLEASE CITE THIS REPORT AS:American Association for Cancer Research.AACR Cancer Progress Report Year.Accessed on:Month,Year.Available at:cancerprogressreport.orgISBN:979-8-9857852-7-2Spotlight on CANCER IN CHILDREN,ADOLESCENTS AND YOUNG ADULTSTable of ContentsMessage From AACR.1Executive Summary.2Snapshot of a Year of Progress.9Cancer in 2024.11Research:Driving Progress Against Cancer.11Cancer in Children,Adolescents,and Young Adults(AYA).14Cancer:An Ongoing Challenge.16Inequities in the Burden of Cancer in the United States.17Variable Progress Against Different Types of Cancer and Stages of Diagnosis.20The Growing Population Burden of Cancer.22The Global Burden of Cancer.23Funding Cancer Research:A Vital Investment.24Understanding the Path to Cancer Development.26Cancer Development:Generating Knowledge.26Basic Research:Vital for Making Progress Against Cancer.28Cancer Development:Interpreting Knowledge.29Changes That Contribute to Cancer Initiation.31Genetic Alterations.31RNA Variations.31Protein Modifications.33Epigenetic Changes.34Systems That Enable Cancer Progression.34The Blood System.34The Lymphatic System.34The Immune System.35The Microbiome.36Processes That Promote Cancer Growth and Metastasis.36Tumor Evolution and Heterogeneity.36Epithelial-to-mesenchymal Transition.37Tumor Microenvironment.37Understanding the Biology of Childhood Cancers.37Chromosomal Rearrangements in Childhood Cancers.38The Promise of Precision Medicine for Childhood Cancers.39Cancer Development:Integrating Knowledge.41Reducing the Risk of Cancer Development.43Eliminate Tobacco Use.44Maintain a Healthy Weight,Eat a Healthy Diet,and Stay Active.47Reduce Alcohol Consumption.52Protect Skin From UV Exposure.52Prevent and Eliminate Infection From Cancer-causing Pathogens.54Limit Exposure to Environmental Risk Factors.55Be Cognizant of Hormonal Factors.58Pregnancy and Breastfeeding.58Hormone Replacement Therapy.58Increasing Cancer Risk Among Children and AYAs.59Tobacco Use in Children and AYAs.59Body Weight,Diet,and Physical Activity in Children and AYAs.59Alcohol Exposure in Children and AYAs.61Human Papillomavirus and AYA Populations.61Environmental Risk Factors in Children and AYAs.61Screening for Early Detection.63Importance of Cancer Screening and Follow-up Care.63Guidelines for Cancer Screening.66Eligibility Criteria for Cancer Screening.66Recommendations for Cancer Screening.67Tests for Cancer Screening.69Genetic Testing and Surveillance in Children With Cancer Predisposition.69Suboptimal Uptake of Cancer Screening.71Progress Toward Increasing Adherence to Cancer Screening Guidelines.71Using Electronic Health Records.72Reducing Structural Barriers.73Implementing Culturally Tailored Strategies Through Community Engagement.74Emerging Technologies for Early Detection of Cancer.74Inspiring Science.Fueling Progress.Revolutionizing Care.79Clinical Research .79Progress Across the Clinical Cancer Care Continuum.85Advances in Cancer Treatment With Surgery.88Performing Less Invasive Cancer Surgery.88Visualizing Breast Cancer Cells More Precisely During Surgery .90Advances in Radiation-based Approaches to Cancer Care.91Advances in Treatment With Cytotoxic Chemotherapy.93Advances in Treatment With Molecularly Targeted Therapeutics.94Spolight on Cancer in Children,Adolescents and Young AdultsAACR Cancer Progress Report 2024II Expanding Precision Treatments Against Common Cancer Types .95Personalizing Treatment for Patients With a Rare Solid Tumor .101Adding Precision to the Treatment of Blood Cancers.104Advances in Treatment With Immunotherapeutics.106Releasing the Brakes on the Immune System.107Enhancing Immune Cell Function.111Boosting the Cancer-killing Power of Immune Cells.112Directing the Immune System to Cancer Cells.114Research-driven Progress Against Childhood and AYA Cancers.115Advances in the Treatment of Leukemia.120Advances in the Treatment of Brain Tumors.121Advances in the Treatment of Solid Tumors Outside the Brain.122Advances in Biomarker-based Treatments .122Supporting Cancer Patients and Survivors.129Challenges Faced by Survivors.130Physical Challenges.130Psychosocial Challenges.132Financial Challenges.132Unique Challenges Faced by Older Adults.132Cancer Survivorship in Childhood and AYAs.133Challenges Faced by Childhood and AYA Cancer Survivors.133Promoting Health in Childhood and AYA Cancer Survivors.138Improving Health-related Quality of Life and Outcomes.139Participating in Physical Activity.140Eating a Healthy Diet and Maintaining a Healthy Weight.140Eliminating Alcohol and Tobacco Use.140Integrating Palliative Care.141Improving Mental Health.141Delivering Care to Cancer Survivors.142Coordinating Care.142Leveraging Patient Reported Outcomes.143Supporting Caregivers.144Envisioning the Future of Cancer Science and Medicine.146Cancer Engineering:An Interdisciplinary Approach to Drive Progress Against Cancer.146A New Age of Radiation Therapy.150Advances in Intraventricular Compartmental Radioimmunotherapy.150Emergence of Radiotheranostics.151A New Wave of Imaging Technologies.151Visualizing Tumor Metabolism Better.151Monitoring Treatment Response Effectively.153Advancing Cancer Research and Patient Care Through Evidence-based Policies.154Investments in Research for a Healthier Future.154A Diverse Cancer Research and Care Workforce Drives Innovation.157Programs to Expand and Diversify the Scientific Research Workforce.157Programs to Strengthen and Expand the Health Care Workforce.158Ensuring Safe and Effective Cancer Therapies Through Regulatory Science.160Diversifying and Decentralizing Trials.160Rapidly Delivering Safe and Effective Therapies to Patients.161Addressing Cancer Drug Shortages.161Advancing Policies to Strengthen Cancer Prevention and Screening Programs.162CDC Screening Programs.162EPA Cancer Moonshot Programs .162Leveraging Policy to Reduce Tobacco-related Illness.163Accelerating Progress Against Childhood Cancer.163Addressing Cancer Disparities and Improving Patient Outcomes.164Inflation Reduction Act.165Environmental Racism and Environmental Justice.166Improving the Use of Digital Information in Cancer Treatment and Management.166Conclusion.167AACR Call to Action.168References.170Glossary.193Appendix.200Index.201AACR Initiatives Accelerating Cancer Research.204AACR Cancer Progress Report 2024IIIList of Report GraphicsFiguresFigure 1:Research Driving Progress Against Lung Cancer,p.13Figure 2:Progress Against Cancer in Children and Adolescents,p.15Figure 3:Why Do US Cancer Disparities Exist?,p.21Figure 4:Hallmarks of Cancer Cells,p.27Figure 5:The Medical Research Cycle Driving Progress Against Cancer,p.28Figure 6:Inherited Cancer Risk,p.33Figure 7:Generation of Fusion Proteins Through Chromosomal Rearrangements,p.39Figure 8:Precision Medicine,p.42Figure 9:Modifiable Cancer Risks,p.44Figure 10:Beyond the Lungs:Cancers Caused by Smoking Tobacco,p.45Figure 11:Reasons to Maintain a Healthy Weight and Stay Active,p.48Figure 12:Alcohol and Cancer Risk,p.52Figure 13:What Can Cancer Screening Find and What Can Be Done?,p.64Figure 14:Cancer Screening Continuum,p.65Figure 15:Biomarker-guided Clinical Trial Design,p.82Figure 16:The Pillars of Cancer Treatment,p.85Figure 17:Targeting Tumors Blood Supply,p.97Figure 18:Decades of Research Breakthroughs Along the Way to Developing Immune Checkpoint Inhibitors,p.109Figure 19:Expanding Scope of Immune Checkpoint Inhibitors,p.110Figure 20:Research Milestones on the Road to Developing TRK-targeted Therapeutics,p.128Figure 21:NIH Funding and the Importance of Continuing Robust Increases,p.157SidebarsSidebar 1:The Medical Research Community:Driving Progress Together,p.12Sidebar 2:US Population Groups That Experience Cancer Disparities,p.18Sidebar 3:Cancer Inequities in the United States,p.19Sidebar 4:Commonly Used Models in Cancer Research,p.29Sidebar 5:How Are Cancers and Tumors Characterized?,p.30Sidebar 6:What Are Genetic Alterations?,p.32Sidebar 7:Cancer Growth:Local and Systemic Influences,p.35Sidebar 8:Key Differences in Hallmarks of Cancer Cells Between Childhood and Adult Cancers,p.38Sidebar 9:The National Cancer Institutes Precision Medicine Initiatives,p.41Sidebar 10:Making Healthy Food Choices:Nutrition Labels,p.50Sidebar 11:Physical Activity Guidelines,p.51Sidebar 12:Guidelines for Alcohol Consumption,p.53Sidebar 13:Ways to Protect Your Skin,p.54Sidebar 14:Ways to Reduce Cancer Risk From Pathogens,p.56Sidebar 15:What Is Causing the Rise in Early-onset Cancers?,p.60Sidebar 16:HPV Vaccination Recommendations,p.62Sidebar 17:Benefits and Potential Harms of Cancer Screening,p.66Sidebar 18:USPSTF Guidelines for Cancer Screening,p.68Sidebar 19:USPSTF-recommended Tests to Screen for Cancer,p.70Sidebar 20:The Cancer Screening Research Network,p.75Sidebar 21:Artificial Intelligence and Liquid Biopsy:New Frontiers in Early Detection of Cancer,p.76AACR Cancer Progress Report 2024IV Sidebar 22:FDA-approved Minimally Invasive Tests for Early Cancer Detection or Risk Reduction,p.77Sidebar 23:Therapeutic Development,p.80Sidebar 24:Types of Clinical Trials,p.81Sidebar 25:Disparities in Clinical Trial Participation,p.84Sidebar 26:Disparities in Cancer Treatment,p.87Sidebar 27:Using Surgery for Cancer Treatment,p.89Sidebar 28:Commonly Used Terms and Benchmarks in Clinical Studies,p.90Sidebar 29:Using Radiation in Cancer Treatment,p.92Sidebar 30:The Increasing Precision of Molecularly Targeted Therapeutics,p.94Sidebar 31:The Challenge of Treatment Resistance,p.95Sidebar 32:Companion Diagnostics,p.96Sidebar 33:Targeting the Undruggable KRAS,p.101Sidebar 34:The Challenges Posed by Rare Cancers,p.104Sidebar 35:Recent Advances Against Blood Cancers,p.105Sidebar 36:Key Cells of the Immune System,p.107Sidebar 37:How Immunotherapeutics Work,p.108Sidebar 38:T-Cell Based Adoptive Cell Therapy,p.112Sidebar 39:CAR T-cell Therapies Approved by the US Food and Drug Administration,p.113Sidebar 40:Phases of Cancer Survivorship,p.131Sidebar 41:Fertility Preservation After a Diagnosis of Cancer,p.134Sidebar 42:Support for Childhood and AYA Cancer Patients and Survivors,p.139Sidebar 43:What Is Palliative Care?,p.141Sidebar 44:Cancer Survivorship Experience and Personal Growth,p.142Sidebar 45:Patient Navigation for Cancer Survivors,p.143Sidebar 46:Patient Reported Outcomes,p.144Sidebar 47:Technological Innovations Emerging From Interdisciplinary Approaches,p.147Sidebar 48:National Cancer Institute(NCI)Programs Promoting a Diverse Scientific Workforce,p.159Sidebar 49:CMS Proposed Policies to Address Drug Shortages,p.162Sidebar 50:CDC Programs to Promote Cancer Health Equity,p.165TablesTable 1:Estimated Burden of Common Types of Cancer in the United States in 2024,p.17 Table 2:Most Common Chromosomal Rearrangements in Childhood Cancers,p.40Table 3:Cancer Types and Cancer Cases Caused by Pathogens Globally,p.55Table 4:Genetic Testing and Surveillance of Cancer Predisposition Syndromes in Children,p.71Table 5:Percentage of Eligible Individuals Up to Date With USPSTF Screening Guidelines in United States in 2021,p.72Table 6:A Selected List of AI-assisted Medical Devices and Software for Early Cancer Detection Approved by FDA During July 2023June 2024,p.77Table 7:Newly FDA-approved Anticancer Agents:July 2023June 2024,p.86Table 8:A Selected List of Radiotheranostic Pairs Currently Being Tested in Clinical Trials to Treat Different Types of Cancer,p.152Supplementary MaterialTable 1:Newly FDA-approved Anticancer Agents:July 2023June 2024,p.200 Scan the QR code to download report graphicsAll report graphics are free to download.Please include the following copyright language for public use(e.g.,presentations):2024 American Association for Cancer Research.AACR Cancer Progress Report 2024.AACR Cancer Progress Report 2024VSurvivor SpotlightsJulia K.Levine Venice,California.9899Diagnosis:Breast Cancer“Do as much evidence-based research as possible,go to conferences,meet other advocates,and dont be afraid to talk to scientists.Most importantly,advocate for yourself.”Dr.Humberto M.Guiot Guaynabo,Puerto Rico .102103Diagnosis:Pancreatic Cancer“My life is an example of how much clinical trials can help patients.A few months ago,I thought that everything was over.And if it were not because of clinical trials,I would not be here right now.”Jennifer Ficko Pooler,Georgia.116117Diagnosis:Melanoma“Many cancers are on the rise.We need research so that we can find cures,or perhaps prevent cancers from occurring in the first place.This can only be accomplished through funding for cancer research.We must have congressional support.”Vicki W.Jones Spokane,Washington.118119Diagnosis:Multiple Myeloma“There are two words I use all the time.One is progress;the other is progression.I am lucky that progress in cancer research has been faster than the progression of my disease.”Michael Methner East Brunswick,New Jersey.124125Diagnosis:Glioma“Cancer doesnt have boundaries.It affects everyone irrespective of religion,political views,or orientation.We must have better treatments to help everybody,and that requires funding.”Mike and Emily Methner,Michaels parentsParker Shaw Lakeside,California.126127Diagnosis:Neuroblastoma“More investment in cutting-edge,less toxic treatments is crucialnot just for Parker,but for the future of all children battling cancer.”Dave and Crystal Shaw,Parkers parentsLourdes Monje Philadelphia,Pennsylvania.136137Diagnosis:Breast Cancer“There are a lot of really specific challenges as a young adult diagnosed with cancer,specifically metastatic breast cancerlike the prospect of having children,the prospect of having a career.”AACR Cancer Progress Report 2024VI Steering CommitteePatricia M.LoRusso,DO,PhD(hc),FAACRChairAACR President 2024-2025Professor of MedicineChief of the Early Phase Clinical Trials ProgramYale UniversityAssociate Center Director of Experimental TherapeuticsYale Cancer CenterNew Haven,ConnecticutCathy J.Bradley,PhDPaul A.Bunn,Jr.Endowed Chair in Cancer ResearchProfessor and Dean,Colorado School of Public HealthDeputy Director,University of Colorado Cancer CenterDenver,ColoradoJohn L.Cleveland,PhDCenter Director and Executive Vice PresidentMoffitt Cancer Center and Research InstituteProfessor and George V.Cortner and Theodore J.Couch Endowed Chair USF Health Morsani College of MedicineTampa,FloridaMaximilian Diehn,MD,PhDJack,Lulu,and Sam Willson ProfessorVice Chair of ResearchDivision Chief of Radiation and Cancer BiologyDepartment of Radiation OncologyStanford University School of MedicineStanford,CaliforniaJane C.Figueiredo,PhDProfessor,Departments of Medicine and Computational BiomedicineDirector,Community and Population Health ResearchSamuel Oschin Comprehensive Cancer InstituteMember,Cedars-Sinai Cancer Cedars-Sinai Medical CenterLos Angeles,CaliforniaChristopher Flowers,MD,MSDivision Head,Division of Cancer MedicineChair,Professor,Department of Lymphoma/MyelomaJohn Brooks Williams and Elizabeth Williams Distinguished University Chair in Cancer MedicineMD Anderson Cancer CenterHouston,TexasMargaret Foti,PhD,MD(hc)Chief Executive OfficerAmerican Association for Cancer ResearchPhiladelphia,PennsylvaniaHedvig Hricak,MD,PhD,Dr(hc)(m)Carroll and Milton Petrie Endowed Chair of RadiologyProfessor Gerstner Sloan Kettering Graduate School of Biomedical SciencesMemorial Sloan Kettering Cancer CenterNew York,New YorkAdana A.M.Llanos,PhD,MPHAssociate Professor Department of Epidemiology,Mailman School of Public Health Co-Leader,Cancer Population Science,Herbert Irving Comprehensive Cancer CenterColumbia University Irving Medical CenterNew York,New YorkMichael Pignone,MD,MPHRebecca and John Kirkland Distinguished Professor of MedicineVice Chair for Quality and Innovation,Duke Department of MedicineACS Clinical Research Professor Duke Cancer InstituteDuke UniversityDurham,North CarolinaKimberly Stegmaier,MDVice Chair of Pediatric Oncology ResearchCo-Director,Pediatric Hematologic Malignancy Program(Basic and Translational Research)Ted Williams ChairProfessor of PediatricsDana-Farber Cancer Institute and Boston Childrens HospitalBoston,MassachusettsKiran Turaga,MD,MPHProfessor of Surgery(Oncology)Division Chief,Surgical Oncology,SurgeryAssistant Medical Director,Clinical Trials OfficeYale School of MedicineNew Haven,ConnecticutMarcel R.M.van den Brink,MD,PhDPresident,City of Hope Los Angeles and City of Hope National Medical CenterDeana and Steve Campbell Chief Physician Executive Distinguished ChairChief Physician ExecutiveCity of Hope National Medical CenterLos Angeles,CaliforniaAACR Cancer Progress Report 2024VIIAACR StaffRajarshi Sengupta,PhDSenior Director,Scientific Research Analysis and DisseminationSayyed Kaleem Zaidi,PhDDeputy Director,Scientific Research Analysis and DisseminationPatrick A.Williams,PhDSenior Scientific Research Analyst,Scientific Research Analysis and DisseminationHeather M.ClarkSenior Print/Web Designer,Marketing,Creative Services,and Multimedia ProductionJoshua F.GoldsteinSenior Director,Brand Strategy CommunicationsJenna M.BachenSenior Creative Director,Marketing,Creative Services,and Multimedia ProductionMichael BeveridgeSenior Director,CommunicationsRichard G.BuckChief Communications Officer and Vice PresidentPaul J.Driscoll,Jr.Chief Marketing Officer and Vice PresidentMatt GontarchickAssociate Director,Government AffairsBenjamin Krinsky,PhDDirector,Federal Government AffairsJon G.Retzlaff,MBA,MPAChief Policy Officer and Vice PresidentBlake RostineManager,Congressional AffairsCarrie Treadwell,MBADirector,Strategic Patient Advocacy and EngagementRukiya Umoja,PharmD,RPhAssociate Director,Regulatory Science and PolicyNicholas Warren,PhDAssociate Director,Science and Regulatory PolicyAbout the American Association for Cancer ResearchFounded in 1907,the American Association for Cancer Research(AACR)is the worlds first and largest professional organization dedicated to advancing cancer research and its mission to prevent and cure cancer.AACR membership includes more than 58,000 laboratory,translational,and clinical researchers;population scientists;other health care professionals;and patient advocates residing in 142 countries and territories around the world.Presently,32%of members live outside the United States and 22%of AACRs international members are located in countries with emerging economies.The AACR marshals the full spectrum of expertise of the cancer community to accelerate progress in the prevention,diagnosis,and treatment of cancer by annually convening more than 30 conferences and educational workshops,the largest of which is the AACR Annual Meeting.The AACR publishes 10 prestigious,peer-reviewed scientific journals.Other AACR publications include Cancer Today,a magazine for cancer patients and caregivers;the annual AACR Cancer Progress Report;AACR Cancer Disparities Progress Report;AACR Annual Impact Report;Leading Discoveries,the AACRs awareness and donor magazine;and the blog,Cancer Research Catalyst.In addition,the AACR funds meritorious research directly as well as in cooperation with numerous cancer organizations.As the Scientific Partner of Stand Up To Cancer,the AACR provides expert peer review,grants administration,and scientific oversight of team science and individual investigator grants in cancer research that have the potential for near-term patient benefit.The AACR actively communicates with legislators and other policymakers about the value of cancer research and related biomedical science in saving lives from cancer.For more information about the AACR,visit AACR.org.We acknowledge Joanne Boehme for copy editing the document.AACR Cancer Progress Report 2024VIII Message From AACRWe are living in a transformative era for cancer research.In the United States,the overall cancer death rate is declining steadily,and the number of cancer survivors has reached an unprecedented level.This remarkable progress is driven by breakthroughs across all areas of cancer science and medicine,even tackling once intractable diseases such as advanced lung cancer and metastatic melanoma.Fueled by trailblazing scientific discoveries and technological innovations,we are now positioned to achieve even greater advances that will save countless more lives.The AACR Cancer Progress Report 2024 highlights the significant strides made possible through medical research,much of which is supported by federal investments in the National Institutes of Health(NIH),National Cancer Institute(NCI),US Food and Drug Administration(FDA),and Centers for Disease Control and Prevention(CDC).These investments have catalyzed a wave of scientific breakthroughs that are deepening our understanding of the biological complexities of cancer and accelerating the development of more effective tools for prevention,detection,diagnosis,and treatment.Noteworthy advancements between July 1,2023,and June 30,2024,the 12 months covered in this report,include the approval by FDA of 15 new anticancer therapeutics and the expansion of 15 previously approved therapeutics for the treatment of additional cancer types.During the same period,FDA also approved a new imaging agent to aid breast cancer surgery,several artificial intelligence(AI)-based tools to improve early detection and diagnosis of cancers,and two minimally invasive tests for assessing inherited cancer risk or for early detection of cancer.Among the newly approved treatments highlighted in this report are the first tumor-infiltrating lymphocyte therapy,a pioneering immunotherapy strategy,for advanced melanoma;a new bispecific antibody against a novel target for patients with small cell lung cancer,a particularly intractable disease;and several new molecularly targeted therapeutics and immunotherapeutics for the treatment of an array of blood cancers.Advances in personalized treatment for cancers driven by mutated KRAS,one of the most frequently altered genes in cancer and long assumed to be“undruggable,”continue unabated with the approval of a KRAS-targeted therapeutic to treat colorectal cancer.Spectacular progress has also been made against childhood cancers,with groundbreaking clinical advances that are transforming outcomes for patients.While cancer remains the leading cause of death by a disease among children,the landscape of childhood cancer care is evolving rapidly,driven by innovative research,new treatments,and a deeper understanding of the unique biology of childhood cancers.Just in the 12 months covered in this report,FDA approved three molecularly targeted therapeutics for the treatment of common childhood cancers,such as glioma and neuroblastoma,as well as exceedingly rare pediatric cancers driven by alteration in the NTRK gene.Despite these achievements,in 2024,it is estimated that more than two million new cases of cancer will be diagnosed in the United States and more than 611,000 people will die from the disease.Globally,there were an estimated 20 million new cancer cases and over 9.7 million deaths from cancer in 2022.Unfortunately,cancer continues to present numerous complex challenges.Incidence rates for some cancers are increasing.Of particular concern among public health experts are the rising cases of breast,colorectal,gastric,and certain blood cancers in adults younger than 50,reasons for which are not fully understood.Additionally,as we detail in the recently published AACR Cancer Disparities Progress Report 2024,health inequities persist,with racial and ethnic minorities and other medically underserved populations bearing a disproportionate burden of cancer.Looking to the future,we strongly believe that we have never been in a better position to bring lifesaving cancer science from the laboratory to the clinic.Integration of emerging technologies,such as sophisticated tumor profiling,liquid biopsies,AI,and novel drug delivery systems,promises to open new frontiers in cancer medicine and revolutionize patient care.And while we continue to push the boundaries of science,our goal is clear:it aims to provide every cancer patient with the best possible chance for a cure and a long,healthy life,even those who have been diagnosed with metastatic disease.The bipartisan support for NIH and NCI funding has been instrumental in our progress against cancer,and continued investment will ensure that we sustain this momentum.It is concerning that after almost a decade of growing federal budgets for medical research,Congress cut NIH funding in fiscal year(FY)2024.This budget reduction threatens to curtail the progress seen in recent years and stymie future strides against cancer.Therefore,AACR urges Congress to uphold robust funding increases for these critical institutions,as well as for FDA and CDC,to drive forward the next wave of breakthroughs in cancer science and medicine.Together,with sustained commitment and investment,we can continue to push the boundaries of what is possible in cancer research and patient care and move closer to the goal of preventing and curing all cancers for all populations.Patricia M.LoRusso,DO,PhD(hc),FAACRPresident,AACRMargaret Foti,PhD,MD(hc)Chief Executive Officer,AACRAACR Cancer Progress Report 20241Executive SummaryWe are witnessing tremendous progress against the collection of diseases we call cancer.The rapid pace of these advances is attributable to research discoveries in basic,translational,clinical,and population science as well as technological innovations that are continually being translated to improvements in cancer prevention,detection,diagnosis,and treatment.As the first and largest professional organization in the world whose mission is to prevent and cure all cancers,the American Association for Cancer Research(AACR)is dedicated to increasing public understanding of cancer and the importance of medical research for saving lives.It is also committed to advocating for increased federal funding to government entities that fuel progress against cancer,in particular,the National Institutes of Health(NIH),National Cancer Institute(NCI),United States(US)Food and Drug Administration(FDA),and Centers for Disease Control and Prevention(CDC).The annual AACR Cancer Progress Report to Congress and the American public is a cornerstone of AACRs educational and advocacy efforts.This fourteenth edition of the report highlights how medical research continues to extend and improve lives,like the lives of the courageous individuals featured in this report who have shared their experiences with cancer.It also underscores how federal funding for NIH,NCI,FDA,and CDC is vital if we are to maintain the momentum of progress against cancer for the benefit of all patients.Cancer in 2024The spectacular progress being made against cancer is resulting in a steady decline in cancer death rates,and a consistent rise in the number of people who live longer and fuller lives after a cancer diagnosis.In fact,the overall cancer death rate in the United States has fallen by 33 percent between 1991 and 2021,a reduction that translates into averting more than 4.1 million deaths from cancer.The drop in overall cancer mortality is attributable to reductions in smoking,as well as improvements in early detection and treatment of certain cancers.The steady decline in death rates for colorectal cancer and female breast cancer since the 1990s has helped drive down overall US cancer mortality.In addition,the decrease in US lung cancer death rate,the pace of which has accelerated in recent years,has contributed significantly to reducing the overall US cancer death rate in the past decade.Further contributing to the progress are the downward trends in death rates for leukemia,melanoma,and kidney cancer,attributable to breakthroughs in precision medicine.Thanks to the research-driven advances,more than 18 million individuals with a history of cancer were alive in the United States as of January 1,2022,and the number is projected to rise to 26 million by 2040.Even though significant progress has been made,cancer continues to be an ongoing public health challenge in the United States and around the world.In the United States alone,it is estimated that more than two million new cancer cases will be diagnosed in 2024.Among the challenges we face is that the advances have not been uniform for all types and stages of cancer.As one example,while the overall cancer incidence in the United States has stabilized in recent years,cases of certain cancer types,such as pancreatic cancer,uterine cancer,and human papillomavirus(HPV)-associated oral cancers,are increasing.Furthermore,the age-and sex-specific incidence of certain cancers is on the rise.For instance,a growing concern among public health experts is the rising incidence of early-onset colorectal cancercancer in adults younger than 50 years.Another significant challenge we face is the disproportionately higher burden of cancer in US racial and ethnic minority groups and other medically underserved populations.Cancer disparities are driven largely by complex and interrelated structural and social factors.Increased collaboration among all stakeholders working toward the bold vision of health equity is vital if we are to ensure that research-driven advances against cancer benefit all patients,regardless of their race,ethnicity,age,sexual orientation,gender identity,socioeconomic status,or geographic location.ESW119892021Reduction In Breast Cancer Death Rate42I0K FEWER CANCER DEATHSAACR Cancer Progress Report 20242 The burden of cancer and its economic toll,both on individuals and on the US health care system,are expected to rise in the coming decades,underscoring the urgent need for more research in medicine and public health to accelerate the pace of progress against cancer.The progress highlighted in this report was made as a direct result of the cumulative efforts of individuals working across the spectrum of medical research and the support from the federal government.Importantly,public sector funding from NIH and NCI directly benefits patients through the development of lifesaving anticancer therapeutics and preventive interventions.Continued federal investments in NIH,NCI,FDA,and CDC will help the medical research community maintain the momentum of scientific and technological innovation and ensure that we meet the Presidents goal of reducing US cancer death rates by half by 2047.The Landscape of Childhood,Adolescent and Young Adult(AYA)CancersThis edition of the AACR Cancer Progress Report highlights the state of cancer in children and AYAs across the cancer continuum.A dedicated spotlight is included in all the relevant sections throughout the report.Cancers are rare in children(14 years and younger),adolescents(15 to 19 years),and young adults(20 to 39 years).In the United States in 2024,approximately 9,620 children and 5,290 adolescents will be diagnosed with cancer.Leukemia and cancers of the nervous system,including brain tumors,are the most common cancers in children.Among adolescents,brain and nervous system tumors are the leading causes of new cancer cases,followed by lymphoma and leukemia.In contrast,young adults are most commonly diagnosed with solid tumors,including thyroid cancer,melanoma,and breast cancer.Research over the past two decades has uncovered key biological differences between childhood and adult cancers.For example,emerging evidence indicates that genetic mutations inherited from parents play an important role in the development of childhood cancers.Another difference is the greater prevalence of structural DNA alterations,such as chromosomal rearrangements,in childhood cancers.Recent findings have identified nearly 300 fusion genes arising from chromosomal rearrangements that are associated with childhood cancers.The knowledge that inherited genetic mutations play an important role in the development of childhood cancers is also helping researchers to develop surveillance strategies for monitoring and managing the risk of childhood cancers.For example,researchers have developed specific genetic tests,as well as surveillance recommendations,to monitor children who have LiFraumeni syndrome,a collection of diseases that is caused by inherited mutations in the TP53 gene and predisposes children to a wide range of early-onset cancers,including soft tissue sarcomas,osteosarcomas,breast cancer,brain tumors,and leukemia.Modifiable risk factors play a far less critical role in the development of childhood cancers compared to cancers in adults.Regardless,there is some evidence that exposure to certain modifiable factors can increase the risk of cancer among children.AYA individuals diagnosed with cancer can be exposed to similar modifiable cancer risk factors as adults,and for a greater length of time compared to children.When combined with genetic predispositions,e.g.,Lynch syndrome,such exposures can further increase the risk of cancer development.According to NCI,an estimated 84,100 AYA individuals will be diagnosed with cancer in the United States in 2024,which is 4.2 percent of all cancer diagnoses.Although this is a small percentage compared to adults ESW2DevelopmentRisk ReductionEarly DetectionTreatmentSurvivorshipDriversof HealthAdverse Health OutcomesCANCER RESEARCH AND CARE TRAINEES AND WORKFORCEStructuralInequitiesSocietalInjusticesRacismDiscriminationSegregationDisparities inCANCER CARE CONTINUUMEducatorsCancer ResearchersPhysiciansPhysician-Scientists Health CareProfessionalsSPOTLIGHTExecutive SummaryAACR Cancer Progress Report 20243who receive a diagnosis of cancer,there has been a rise in certain types of early-onset cancers that are caused by a combination of factors including genetic predisposition,diet,microbiome,excess body weight,and environmental exposures.Infection with certain pathogens can also increase the risk of cancer in this group.The unprecedented progress in the treatment of childhood and AYA cancers is reflected in the greater than 85 percent 5-year relative survival rates for all cancers combined among childhood and AYA patients.Many of the initial advances in the treatment of childhood cancers were made through intensification of cytotoxic chemotherapeutics,which while effective were associated with significant toxicities,including late and long-term adverse effects.With greater understanding of the biology of childhood and AYA cancers and innovations in technology,there is an increasing focus on utilizing personalized approaches to target cancers more precisely as well as on reducing treatment intensities among patients who have a favorable prognosis,to improve their quality of life.The National Cancer Institute is leading the efforts to harness the knowledge gleaned from genomic analyses of childhood cancers to develop strategies for precision or personalized medicine.In recent years,FDA has approved a broad array of precision therapeutics to treat a variety of childhood cancers.Just in the 12 months covered by this report(July 1,2023June 30,2024),FDA approved a new molecularly targeted therapy,tovorafenib(Ojemda),for the treatment of children with certain brain tumor types.During the same period,FDA also expanded the use of a molecularly targeted therapeutic,eflornithine(Iwilfin),previously approved for certain infectious and hormonal diseases,as the first treatment to reduce the risk of relapse in children with high-risk neuroblastoma.In addition,FDA expanded the use of a molecularly targeted therapeutic,repotrectinib(Augtyro)previously approved for patients with certain type of lung cancer for the treatment of children with any solid tumor that has certain genetic alterations.Furthermore,several molecularly targeted therapeutics and immunotherapeutics that were already approved by FDA for use in adults with certain cancers received expanded approvals for the treatment of children with the same types of cancer.The promise of these therapeutics is illustrated through the personal experiences of Michael Methner(see p.125)and Parker Shaw(see p.127).Despite major progress,some cancers in children and AYA,such as sarcomas or certain brain tumors,have been difficult to treat and continue to have poor survival.Childhood and AYA cancer survivors such as Lourdes Monje(see p.137)also face unique challenges compared to their peers who have never been diagnosed with cancer.These challenges include long and late-term side effects from cancer and its treatments,financial toxicity,difficulty finding work,lower levels of educational attainment,psychosocial issues,and others.Because of the unique challenges faced by this population,researchers and care providers must ensure that the needs of this population are adequately addressed across the spectrum of cancer care.Understanding the Path to Cancer DevelopmentSeminal contributions from decades of research in basic,clinical,translational,and population science have established cancer as a collection of diseases that are characterized by uncontrolled cell division.A key insight from this knowledge is that different cancer types share many characteristics or hallmarks,including the ability of cancer cells to acquire changes that make their genome unstable,divide limitlessly,grow uncontrollably,escape cell death,spread to other tissues in the body,evade destruction by the immune system,and increase nutrients and oxygen supply to tumors.Research has shown that hallmarks of cancer are primarily acquired through mutations in the genetic material of normal cells.There are two types of genetic mutations associated with 5-year Relative Survival Rate(ALL CANCERS COMBINED)ESW320122019mid-1970s86hX%Children(Ages 014)Adolescents(Ages 1519)Executive SummaryAACR Cancer Progress Report 20244 cancer:inherited and somatic.Inherited mutations are passed on from parents to children and contribute to about 10 percent of all cancer cases.The remaining 90 percent of all cancer cases stem from somatic mutations,which are acquired throughout a persons lifetime and can arise in multiple ways,such as from errors made during cell division,or due to exposure to modifiable risk factors including smoking,certain viral infections,and UV radiation and/or cancer-causing chemicals.Cancer initiation,development,and progression are all multistep processes that are further influenced by changes inside and outside the cell.As the disease progresses,cancer cells acquire additional characteristics that enable them to establish mutually beneficial interactions with their surroundings,known as the tumor microenvironment.Research has shown that the tumor microenvironment affects the growth of cancer cells and cancer cells influence the tumor microenvironment to promote their survival.Technological advances in understanding cancer at the levels of single cells and molecules have demonstrated that each patients cancer is unique.This important insight is the basis for precision medicine,or personalized medicine,which is broadly defined as treating patients based on molecular characteristics that distinguish them from other individuals with the same disease.Rapid developments in precision medicine are yielding new and effective anticancer therapeutics to treat cancer types for which there were no effective treatment options just two decades ago.Reducing the Risk of Cancer DevelopmentResearch in basic,translational,and population sciences has broadened our understanding of the factors that increase an individuals risk of developing cancer.It is estimated that 40 percent of all cancer cases in the United States are attributable to preventable causes.Many of these risk factors are modifiable,such as reducing tobacco use,avoiding an unhealthy diet,staying physically active,limiting exposure to UV radiation,reducing or eliminating alcohol consumption,and preventing and treating cancer-causing pathogenic infections.Between 1991 and 2021 the overall cancer mortality in the United States declined by 33 percent,in part due to the implementation of public health campaigns and policy initiatives that helped reduce smoking and increase early detection of cancers.Although smoking rates have declined,the increasing prevalence of other risk factors,including obesity among US children and adults,are cause for public health concern.Additionally,there is a lack of widespread utilization in the United States of preventive interventions,such as vaccination against cancer-causing pathogens,including HPV,which is the primary cause of cervical cancer.Environmental risk factors,such as air pollution,water contamination,and naturally occurring radon gas,also increase a persons risk for certain types of cancers,such as lung cancer.There is also an increasing recognition that endocrine-disrupting chemicals,such as those found in hair straightening products,food packaging,and many other consumer products,can increase the risk of certain diseases,including cancers of the breast and thyroid.Exposure to elevated levels of carcinogens in certain occupations,such as firefighting or welding,can increase the risk of certain types of cancer.Furthermore,occupations that involve night shift work,which can disrupt the bodys natural sleep patterns,as well as the lack of sleep due to overwork,can increase an individuals risk of developing cancer.Finally,hormonal factors that result from normal physiology,such as pregnancy and breastfeeding can also increase or decrease the risk of developing certain types of cancer.As we learn more about cancer risk factors and identify segments of the US population who are exposed to elevated levels of these factors,new and equitable policies must be developed and implemented to reduce cancer risk and improve the health of all populations,including those exposed to environmental and occupational cancer risk factors.Hallmarks of Cancer CellsESW4Spread to other parts of the bodyMultiply limitlesslyIncrease blood vessel formation toward tumorEvade the immune systemIncrease nutrient and oxygen supply to the tumorEscape cell deathGrow uncontrollablyAccumulate changes in the genetic materialExecutive SummaryAACR Cancer Progress Report 20245Screening for Early DetectionCancer screening refers to checking for cancer,or for abnormal cells that may become cancerous,in people who do not have any signs or symptoms of the disease.Cancer screening can help detect cancer at the earliest possible stage when it can be treated successfully,with a higher likelihood of cure.Accruing evidence shows that cancer screening saves lives and reduces the burden of the disease at a population level.In the United States,the US Preventive Services Task Force,a panel of experts in preventive medicine,periodically issues evidence-based screening recommendations for cancers of breast,cervix,colon and rectum,lung and bronchus,and prostate.Key considerations that determine who should receive screening and for which cancer include biological sex and age of the individual,as well as genetic,environmental,behavioral,and social influences.Cancer screening is a multistep process that includes receiving the recommended test,as well as follow-up care if the initial test shows abnormal findings.Unfortunately,disadvantaged segments of the US population experience inequities in receiving the recommended cancer screening and follow-up care.There are several reasons for low rates of cancer screening,including social and structural barriers;bias and discrimination against marginalized populations in the health care system;mistrust of health care professionals among minoritized populations;lack of access to quality health insurance;low health literacy;and miscommunication between patients and providers.Researchers have identified evidence-based interventions that are proving effective in increasing adherence to recommended screening guidelines and follow-up care.These interventions include using electronic health records to educate and inform patients and providers about routine cancer screening;reducing structural barriers so that it is easier for people to take the routine cancer screening test;and implementing culturally tailored strategies to build trust between patients and providers.Researchers are also cautiously optimistic about the potential of recent technological advances,such as implementation of artificial intelligence(AI)and minimally invasive screening tests,in improving early detection of cancers.In recent years,FDA has approved several AI-assisted medical devices to aid clinicians in cancer diagnosis.During the 12 months covered by this report(July 1,2023June 30,2024),FDA also approved two minimally invasive tests for inherited risk prediction or early detection of cancer.These approvals underscore the potential of AI and minimally invasive screening tests to detect cancers early.However,large prospective studies are required to establish that these approaches will improve early detection of cancers without increasing harm for individuals and/or further exacerbating existing inequities in the receipt of cancer screening and follow-up care.Inspiring Science.Fueling Progress.Revolutionizing Care.The dedicated efforts of researchers working across the continuum of cancer science and medicine power breakthroughs in clinical care that are improving survival and quality of life for patients in the United States and around the world.Clinical trials are a vital part of medical research because they establish whether new cancer treatments are safe and effective.Therefore,it is imperative that participants in clinical trials represent the full spectrum of the patient population who may use these treatments if they are approved.Unfortunately,participation in cancer clinical trials is low,and there is a significant lack of sociodemographic diversity among those who do participate.It is imperative that researchers and policymakers work together to address the many barriers to clinical trial participation.Enhancing the availability of clinical studies,particularly in community settings,can be transformative for patients,as reflected in the personal story of Dr.Humberto M.Guiot(see p.103).Surgery,radiotherapy,and cytotoxic chemotherapy constitute three of the five main pillars of cancer treatment.However,these therapies can have long-term adverse effects on patients.Through ongoing clinical studies,researchers are evaluating whether less aggressive surgery,radiotherapy,and cytotoxic chemotherapy can be appropriate for some patients with cancer,allowing these patients to experience improved quality of life.Among the advances made between July 1,2023,and June 30,2024,are the 15 new anticancer therapeutics approved for use by FDA.During the same period,FDA also approved a new imaging agent to aid breast cancer surgery and expanded the use of 15 previously approved anticancer therapeutics to treat additional cancer types.Included in the FDA approvals are the first tumor-infiltrating lymphocyte-based cellular immunotherapy that is benefiting patients with advanced melanoma such as Jennifer Ficko(see p.117),a new T cellengaging ESW525%OF THE REDUCTIONROUTINE MAMMOGRAPHY.IN BREAST CANCER MORTALITYIN THE UNITED STATES FROM 1975 TO 2019IS ATTRIBUTABLE TOExecutive SummaryAACR Cancer Progress Report 20246 bispecific antibody against a novel target for patients with small cell lung cancer,the first AKT-targeted therapeutic for patients with breast cancer such as Julia K.Levine(see p.99),the first KRAS-targeted therapy for certain patients with colorectal cancer,and several new molecularly targeted therapeutics and immunotherapeutics for the treatment of patients with an array of blood cancers,such as Vicki W.Jones(see p.119)who is receiving a new molecularly targeted therapeutic to treat her multiple myeloma.While these exciting new advances have the potential to transform clinical care,much work is needed to ensure equitable access to these treatments for all patient populations.Supporting Cancer Patients and SurvivorsAccording to NCI,a person is considered a cancer survivor from the time of cancer diagnosis through the balance of their life.As of January 2022,the most recent year for which such data are available,there were more than 18 million people living in the United States with a history of a cancer diagnosis,which equates to about 5 percent of the US population.This is a significant improvement from 50 years ago when cancer survivors constituted only 1.4 percent of the US population.Understanding and addressing the short-and long-term challenges faced by cancer survivors,supporting their quality of life,and ensuring that care is accessible and equitable are important priorities in cancer survivorship research.Each person diagnosed with cancer has a unique experience ranging from successful treatment and living cancer free for the remainder of life to living a high-quality life through successful management of metastatic cancer to experiencing varying degrees of side effects to a subsequent cancer diagnosis with the same or a different type of cancer.Survivors often face physical,psychosocial,and financial challenges,both during and after the conclusion of treatment.Cancer survivors should adhere to a healthy diet,engage in physical activity,reduce or eliminate the consumption of alcohol,and stop smoking,all of which help mitigate the physical challenges associated with a diagnosis of cancer.Researchers are also using other evidence-based strategies,including palliative care,pyscho-oncology,patient-reported outcomes,and patient navigation,to help reduce the adverse impact of a cancer diagnosis on the physical,mental,and financial health of cancer survivors.Understanding the survivorship challenges,as well as how to reduce or eliminate them,is an active area of research that is continually evolving as new therapies are introduced in the clinic.Challenges experienced by patients and survivors of cancer also extend to friends and family members who often act as informal caregivers.There are an estimated four million caregivers who are caring for an adult cancer patient in the United States.These caregivers support cancer survivors in multiple ways,such as by arranging transportation for clinical appointments,helping with day-to-day activities,assisting in medical care or other clinical tasks,coordinating care,and providing emotional support.Caregiving often leads to burnout,which negatively impacts caregivers psychological and emotional well-being.More evidence of the challenges faced by caregivers is emerging through ongoing research,which also highlights the many opportunities to assist this vulnerable population.Envisioning the Future of Cancer Science and MedicineBreakthrough discoveries and technological advances across the fields of medicine have substantially increased the understanding of cancer initiation and progression,providing the foundational knowledge for better strategies to reduce the risk of developing cancer,detect cancer at the earliest possible stage,and treat cancer effectively and more precisely.As a result,cancer deaths are declining,and survivors are living longer and fuller lives.Researchers,including AACR president,2024-2025,Patricia M.LoRusso,DO,PhD(hc),FAACR(see p.149),firmly believe that the fast-paced trajectory of progress against cancer can be further accelerated through sustained and predictable funding for cancer research.Radiotherapy,one of the pillars of cancer treatment,has experienced a wave of innovation in the past decade,including delivering radiation precisely to tumors,thus minimizing harm to the surrounding normal tissues.Radiotheranostics is another promising technique for detecting and treating cancer using radioisotopes that has shown remarkable success“The future of cancer science and medicine is promising.Cancer diagnostics are becoming more sophisticated.New technologies,such as spatial transcriptomics,are helping us study tumors at a cellular level.Artificial intelligencebased approaches are beginning to transform cancer detection,diagnosis,treatment decision making and response monitoring.”Patricia M.LoRusso,DO,PhD(hc),FAACR AACR President,20242025Executive SummaryAACR Cancer Progress Report 20247against multiple cancer types,marking a significant advance in radiation-based cancer treatment.Advances in non-invasive cancer imaging are revolutionizing visualization of tumor metabolism and assessment and monitoring of treatment response inside the body,thus enabling clinicians to make informed treatment decisions in a timely manner.Another exciting advance is the emergence of cancer engineering,a powerful interdisciplinary approach that combines principles from engineering,biology,and medicine for understanding the complexities of cancer development to improve health outcomes.Advancing Cancer Research and Patient Care Through Evidence-based PoliciesSustained investments in medical research are critical for progress against cancer,including risk reduction,early detection,and treatment.As the largest public source of funding for medical research,NIH supports a vast array of scientific and educational programs that enable breakthroughs,which benefit human health and train the next generation of researchers.Within NIH,NCI leads the National Cancer Program and is the worlds largest single supporter of cancer research and training.Federal support is also needed to ensure that the benefits of medical research are shared by all populations.Achieving health equity requires further investments in NIH and NCI on cancer disparities research and in education and training programs to ensure that the cancer research and care workforce is broadly representative of society.Other key investments include FDA programs for improving access to and diversity of cancer clinical trials;CDC initiatives for building a robust public health infrastructure and programs to improve cancer screening and reduce the use of tobacco;and US Environmental Protection Agency(EPA)actions and environmental health policies for reducing environmental exposures to carcinogens.In recent decades,cancer mortality rates have declined for many childhood cancers.However,further policy solutions are needed to continue to expand research on cancer in children and adolescents,improve data collection,and expand and increase access to clinical trials for children and AYA with cancer.Congress has begun considering several pieces of legislation to address these issues,but more work is needed to speed progress against childhood,AYA,and other rare cancers.AACR Call to ActionFrom fiscal year FY 2016 to FY 2023,Congress increased NIH funding for eight consecutive years.These funding increases for medical research accelerated the pace of scientific progress and contributed to the longer-term decline in cancer mortality in the United States.Unfortunately,after years of growing federal budgets for medical research,Congress cut NIH funding in FY 2024.This budget reduction threatens to curtail the progress seen in recent years and stymie future advancements.AACR urges Congress to continue to support robust,sustained,and predictable funding growth for the medical research and health programs that are vital to the fight against cancer.We call on Congress to:Appropriate at least$51.3 billion in FY 2025 for the base budget of NIH and at least$7.934 billion for NCI.Provide$3.6 billion in dedicated funding for Cancer Moonshot activities through FY 2026 in addition to other funding,consistent with the Presidents FY 2025 budget.Appropriate at least$472.4 million in FY 2025 for the CDC Division of Cancer Prevention to support comprehensive cancer control,central cancer registries,and screening and awareness programs for specific cancers.Allocate$55 million in funding for the Oncology Center of Excellence at FDA in FY 2025 to provide regulators with the staff and tools necessary to conduct expedited review of cancer-related medical products.By following these recommendations,Congress will help speed the rate of discovery and create vital pathways for young scientists to contribute to future advances in cancer research.This investment will improve our nations health,including the lives of the millions of people who have been affected by cancer.Scan the QR code to watch a video summary of the report.Executive SummaryAACR Cancer Progress Report 20248 Snapshot of a Year of ProgressResearch continues to advance immunotherapy,leading to:The first tumor-infiltrating lymphocyte-based cellular immunotherapy that is benefiting patients with advanced melanoma(see Jennifer Fickos story,p.117).The first approval of an immune checkpoint inhibitor for patients with a rare tumor of the head and neck which originates in the nasopharynx.Three new T cell-engaging bispecific antibodies for patients with multiple myeloma(see Vicki W.Joness story,p.119)and lung cancer.Research continues to harness the power of molecularly targeted therapy,leading to:A first-in-class AKT targeted therapeutic for patients with breast cancer,(see Julia K.Levines story,p.99).First approval of a KRAS targeted therapeutic for patients with metastatic colon cancer;the treatment may also benefit patients with advanced pancreatic cancer(see Dr.Humberto M.Guiots story,p.103).A new molecularly targeted therapeutic for desmoid tumors,an extremely rare and potentially debilitating condition.Between July 1,2023,And June 30,2024,FDA Approved:New anticancer therapeutics,which are now benefiting patients with various types of cancerPreviously approved anticancer therapeutics for treating new types of cancerNew imaging agentMinimally invasive tests for assessing inherited cancer risk or for early detection of cancerSeveral artificial intelligence(AI)-based tools to improve early detection and diagnosis of cancers19912021Reduction In Overall Cancer Death Rate33%4.1 MILLION LIVES SAVEDof all US cancer cases are attributable to preventable risk factors 40%CigaretteSmokingExcess Body WeightAlcoholConsumptionUV RadiationExposurePoor DietPathogenicInfectionsPhysicalInactivityUV19.3%7.6%5.4%4.6%4.2%3.4%3.1NCER RISK FACTORS151512AIAACR Cancer Progress Report 20249Call to ActionFor the Fiscal Year 2025,AACR urges Congress to continue to support robust,sustained,and predictable funding for the federal medical research and health programs vital to progress against cancer:FDA approved several anticancer drugs for children with cancer(July 2023June 2024),including:Molecularly targeted therapeutics to:Treat certain types of brain tumor(see Michael Methners story,p.125).Reduce the risk of high-risk neuroblastoma relapse(see Parker Shaws story,p.127).Treat a wide array of cancer types that have a specific genetic alteration known as NTRK gene fusion.Several molecularly targeted therapeutics and immunotherapeutics that were already approved for use in adults for the treatment of children with the same cancer types.AYA cancer survivors(see Lourdes Monjes story,p.137)face long term side effects;40 percent experience multiple chronic health conditions(e.g.,hearing loss,stroke,diabetes.)NIHNCICDCsDivision of Cancer Prevention and ControlFDAsOncology Center of Excellence$51.3 BILLION$7.934 BILLION$472.4 MILLION$55 MILLIONCancer in Children,Adolescents and Young Adults(AYA)in the USAdvances in Genomics Accelerating Progress Against Childhood CancersESTIMATED CANCER CASES(2024)OVERALL CANCER DEATH RATES63p702021Children(Ages 014)Adolescents(Ages 1519)Children(Ages 014)AYA(Ages 1539)9,62084,100Assessing cancer riskGenetic testing for surveillanceTreatment decisionsNCIChildrens Oncology Group Pediatric Molecular Analysis for Therapy Choice Trial*Understanding biologyFusion Oncoproteins in Childhood Cancers Consortium*TUMOR CHARACTERIZATIONCHILDREN,ADOLESCENTS ANDYOUNG ADULTS WITH CANCERChildhood Cancer Data Initiative*Molecular Characterization Initiatives*NCI initiative.AACR Cancer Progress Report 202410 Cancer in 2024IN THIS SECTION,YOU WILL LEARN:In the United States(US),the overall cancer death rate has been steadily declining since the 1990s,with the reductions between 1991 and 2021 translating into more than 4.1 million cancer deaths avoided.The decline in overall US cancer death rate is attributable to reduction in smoking rates,as well as improvements in treatment and early detection of certain cancers.More than 18 million cancer survivors were living in the United States as of January 1,2022.Progress has not been even against all cancer types or all stages of a given cancer type.Many segments of the US population experience stark inequities in the cancer burden;these inequities are largely driven by structural and social factors.It is imperative that all stakeholders work together to implement evidence-based interventions including public policies that guarantee equitable access to quality health care for all patients,regardless of their race,ethnicity,age,sexual orientation,gender identity,socioeconomic status,or geographic location.The economic burden of cancer on individuals and the US health care system is expected to rise in the coming decades,highlighting the urgent need for more research and increased federal support for medical science and public health to accelerate the pace of progress against cancer.Research:Driving Progress Against CancerResearch is the foundation of progress against the collection of diseases we call cancer.It improves survival and quality of life for people around the world because it is the driving force behind every clinical breakthrough and every public policy designed to improve human health.Discoveries across the major areas of cancer research,including basic,clinical,translational,and population science,provide the foundation for advances in cancer prevention,early detection,diagnosis,treatment,and survivorship.Every clinical advance and every policy that spurs progress against cancer is the culmination of a complex process that requires collaboration over the course of many years among numerous stakeholders(see Sidebar 1,p.12).AACR Cancer Progress Report 202411The remarkable advances made against cancerin particular,improvements in early detection,diagnosis,treatment,and risk reductionare resulting in a steady decline in US cancer death rates year after year.In fact,the age-adjusted overall cancer death rate has fallen by 33 percent between 1991 and 2021,a reduction that translates into an estimated 4.1 million fewer deaths from cancer(2).The reduction in overall US cancer mortality rate can be attributed to significant reduction in smoking rates,as well as improvements in treatment and early detection of certain cancers.The Medical Research Community:Driving Progress TogetherProgress against cancer can be accelerated when all stakeholders who are dedicated to fundamentally changing the burden of cancer work together.Further increasing collaborations will amplify future breakthroughs.The key stakeholders in medical research include:Source:(1).SavingLivesTogetherBiopharmaceutical,diagnostic,and medical device company research teamsIndividuals diagnosed with cancer,their caregivers,family members,friends,and colleaguesHealth care systems and clinical teamsAcademic and government researchers from a diverse array of specialtiesCommunity partners,suchas religious organizations,local health clinics,food banks,transportation services,and other community-basedorganizationsIndividual community scientists,patient navigators,patient advocates,other cancer advocates,and members of advocacy groupsHealth insurance companiesFederal funding agenciesPolicymakers and regulatorsPhilanthropic organizations,including nonprofit sponsors and individual donors,cancer-focused professional organizations,and cancer-focused foundationsSIDEBAR 1Cancer in 2024AACR Cancer Progress Report 202412 Reduction in death rates for breast cancer among females and colorectal cancer among those over age 50 since the 1990s contributed to the progress in reducing overall US cancer mortality(2).According to a recent analysis,US breast cancer mortality declined by 42 percent,averting greater than 490,000 deaths between 1989 and 2021,because of advances in screening mammography and treatment(2).The death rate for colorectal cancer,overall,has declined by 39 percent between 2000 and 2022(3).However,mortality has been rising among those diagnosed before the age of 50(see Cancer in Children,Adolescents,and Young Adults(AYA),p.14).The accelerated decline in overall cancer mortality in the past decade has been driven largely by rapid decreases in US lung Research Driving Progress Against Lung CancerThanks to research-driven clinical breakthroughs and steep reduction in US smoking rate,lung cancer mortality is declining rapidly.In fact,the decrease in lung cancer mortality per year accelerated from 2 percent between 2005 and 2013 to 4 percent between 2013 and 2021(4).Basic research discoveries have identified numerous cellular pathways that are associated with lung cancer development.Key components of these pathways include proteins such as KRAS,EGFR,FGFR,ALK,ROS1,RET,MET,NTRK,HER2,and DLL3.Research has also shown that cancer cells evade destruction by the immune system because they have high levels of proteins that can attach to and trigger brakes on immune cells,stopping them from attacking cancer cells.Collectively,this knowledge has laid the foundation for personalized treatments for patients with lung cancer,in particular,molecularly targeted therapeutics and immunotherapeutics,which have resulted in remarkable lasting responses.Indicated on the timeline are important milestones in lung cancer precision medicine,including first US Food and Drug Administration(FDA)approvals for molecularly targeted therapeutics or immunotherapeutics that have distinct mechanism of action.While not included in the figure,large scale clinical studies such as the National Lung Screening Trial and NederlandsLeuvens Longkanker Screenings Onderzoek have demonstrated that early detection using low-dose computed tomography(LDCT)screening can lower lung cancer mortality(5,6).Population-level implementation of LDCT use(current uptake of which is extremely low)among eligible individuals can further reduce the burden of lung cancer in the United States.FIRST FDA APPROVALS OF THERAPEUTICS WITH DISTINCT MECHANISMS OF ACTION1978Single agent therapy(cisplatin)2003EGFR inhibitor(gefitinib)2011ALK/ROS-1 inhibitor(crizotinib)2006Angiogenesis inhibitor(bevacizumab)2018Targeted therapy based on tumor-agnostic biomarker(larotrectinib for NTRK gene fusions)2015Mutant EGFRT790M inhibitor(osimertinib)Immune checkpoint inhibitor(pembrolizumab)2017Immunotherapy based on tumor-agnostic biomarker(pembrolizumab for MSIh-dMMR status)MEK/RAF inhibitor(dabrafenib&trametinib)2021Bispecific antibody(amivantamab-vmjw)KRAS G12C inhibitor(sotorasib)2016cobas EGFR Mutation test(cobas liquid biopsy CDx)2020Immune checkpoint inhibitors(nivolumab&ipilimumab)Liquid biopsy NGS test(Guardant360 CDx)RET inhibitor(selpercatinib)MET inhibitor(capmatinib)2022HER-2 inhibitor(fam-trastuzumab deruxtecan-nxki)Companion DiagnosticChemotherapyMolecularly Targeted TherapyImmunotherapy2024T cell-engaging bispecific antibody(tarlatamab-dlle)FIGURE 1Cancer in 2024AACR Cancer Progress Report 202413cancer death rates in both men and women,attributable to public health interventions to reduce smoking as well as advances in treatment(see Figure 1,p.13)and early detection(3).Research-driven advances in treatment have resulted in a steady decline in death rates despite increasing incidence for leukemia,melanoma,and kidney cancer(2).For example,groundbreaking basic research in the 1960s through 1980s that identified the mechanistic underpinnings of chronic myeloid leukemia(CML),a cancer of the blood and bone marrow,propelled the development of a cascade of new treatments that have drastically improved outcomes for patients(7).Advances in the treatment of kidney cancer,in particular,with molecularly targeted therapeutics and immunotherapeutics have transformed clinical care for these patients.In fact,in a recent study,an immunotherapeutic,pembrolizumab(Keytruda),was shown to be the first postsurgical treatment that helps patients with early-stage kidney cancer live longer(8).Among the major advances made across the clinical cancer care continuum from July 1,2023,to June 30,2024,are 15 new anticancer therapeutics that were approved for use by the US Food and Drug Administration(FDA)(see Progress Across the Clinical Cancer Care Continuum,p.85).During this period,FDA also approved new uses for 15 previously approved anticancer therapeutics,a new imaging agent to help visualize cancerous cells during surgery,and several artificial intelligence(AI)based tools to improve early detection and diagnosis of cancers.Collectively,advances such as these and those described in past editions of this annual report are helping to increase the number of children and adults who live longer and fuller lives after a cancer diagnosis.Indeed,the 5-year relative survival rate for all cancers combined has increased from 49 percent for those diagnosed in the mid-1970s to 69 percent among those diagnosed during 2013 to 2019(4).As of January 1,2022,more than 18 million individuals with a history of cancer were alive in the United States,and the number is projected to grow to 26 million by 2040(9).Additionally,because of improved treatments,increasing numbers of individuals are now living longer despite being diagnosed with metastatic disease(10).Continued research to address the survivorship needs of the growing number of individuals living with cancer must be a priority for US medicine and public health(see Supporting Cancer Patients and Survivors,p.129).Cancer in Children,Adolescents,and Young Adults(AYA)Compared to cancers in adults,cancers are rare in children,adolescents and young adults(AYA).In the United States in 2024,approximately 9,620 children(14 years and younger)and 5,290 adolescents(15 to 19 years)will be diagnosed with cancer(4).Leukemia and cancers of the nervous system,including brain tumors,are the most common cancers in children.Among adolescents,brain and nervous system tumors are the leading sites of new cancer cases,followed by lymphoma and leukemia.In contrast,young adults ages 20 to 39 years are most commonly diagnosed with solid tumors,including thyroid cancer,melanoma,and breast cancer.Decades of research-driven advances in cancer science and medicine,including the identification and therapeutic targeting of cellular and molecular drivers of cancer(see Research-driven Progress Against Childhood and AYA Cancers,p.115),along with progress in surgical techniques and optimization of radiotherapy and chemotherapy have led to a steady decline in cancer death rates for children and Increasing Treatment Options for Melanoma2013trametinibdabrafenib2014combination oftrametinib and dabrafenibpembrolizumabnivolumab2018combination ofencorafenib and binimetinib 2011ipilimumabvemurafenib 2015combination ofipilimumaband nivolumab combination ofcobimetinib and vemurafenib2020combination ofatezolizumab and cobimetinib and vemurafenib2022combination of relatlimab-rmbw and nivolumab 1975Dacarbazine1995Interferon alfa-2bChemotherapyImmunotherapyMolecularly Targeted Therapy1992IL22024lifileucelW1SPOTLIGHTCancer in 2024AACR Cancer Progress Report 202414 adolescents.Among US children(14 years and younger)and adolescents(15 to 19 years),overall cancer death rates have declined by 70 percent and 63 percent,respectively,between 1970 and 2021(4).Just in the past two decades,the overall cancer death rate for children and adolescents declined by 24 percent(11).The 5-year relative survival rate for all cancers combined has improved for US children from 58 percent during the mid-1970s to 85 percent for those diagnosed between 2013 and 2019(4).However,there are significant differences in survival rates between different cancer types(see Figure 2,p.15).Cancer survival has also improved for AYAs.Based on a recent study that evaluated survival trends across 33 common AYA cancers,those diagnosed between 2010 and 2018 had a 5-year relative survival of 86 percent(12).Of the 33 cancer types,25 had significant improvement in 5-year Progress Against Cancer in Children and AdolescentsFive-year relative survival rates for the US children and adolescents(ages 0 to 19)who were diagnosed with cancer from 2013 to 2019 were substantially higher compared to those diagnosed from 1975 to 1979.Childhood cancers are classified using the International Classification of Childhood Cancer(ICCC).Improvement in the 5-year relative survival rate was seen for all cancers combined as well as for several individual cancer types.Source:(3).FIVE-YEAR SURVIVAL RATES(%)FOR CERTAIN CHILDHOOD AND ADOLESCENT(019 YRS)CANCERS204006080100Thyroid carcinomaRhabdomyosarcomaRetinoblastomaNon-Hodgkin lymphomaNeuroblastoma and ganglioneuroblastomaMelanomaLeukemiaHodgkin lymphomaHepatic tumorsBrain and central nervoussystem neoplasmsBone tumorsAll ICCC sites combined1975197920132019Year of DiagnosisFIGURE 2AGE GROUPSCANCER INCIDENCE RATE(PER 100,000)Children (ages 15)17.1Adolescents and young adults(AYA)(ages 1539)74.9Adults (ages 4064)529Older adults (ages 6574)1,753Source:(3).W2Cancer in 2024AACR Cancer Progress Report 202415relative survival since 2000.However,AYAs had a much lower 5-year relative survival than children for four cancers,including acute lymphocytic leukemia(ALL)and Ewing sarcoma(12).Despite the progress,cancer is the leading cause of disease-related death among US children,with around 1,040 children expected to die from the disease this year(4).Additionally,there are disparities in the burden of childhood and AYA cancers for racial and ethnic minority groups and other medically underserved populations in the United States.As one example,while the overall cancer death rate for White children and adolescents declined by 12 percent between 2011 and 2021,the rates did not change significantly for Black and Hispanic children and adolescents(11).Recent data also show that non-Hispanic Black AYAs experience worse survival for many cancers compared to other racial and ethnic groups(12).Additionally,there are disparities based on socioeconomic status.For instance,children with cancer living in Alabama counties with persistent poverty during 20002016 were 30 percent more likely to die within 5 years of cancer diagnosis,compared to those not living in Alabama counties with persistent poverty(13).Addressing the barriers that drive survival disparities in childhood and AYA cancers,such as lack of clinical trial enrollment,access to guideline-adherent treatments,and long-term survivorship care,as well as identifying biological features of these cancers is vital for continued progress.While overall cancer incidence in the United States has stabilized in recent years,a rising concern among public health experts is the steadily increasing incidence of certain cancer types among individuals younger than 50 years,a phenomenon referred to as early-onset cancer.According to a recent report,the incidence of early-onset cancers,particularly among individuals aged 30 to 39 years,increased significantly during 2010 to 2019(15).In 2019,most early-onset cancers were diagnosed in the breast,thyroid,colon,and rectum(15).Between 2010 and 2019 the greatest increase in early-onset cancer occurred for those arising in the gastrointestinal system.In fact,many studies have reported an increase in the incidence of early-onset colorectal cancer(16,17).According to a recent report,between 2011 and 2019,colorectal cancer incidence rates increased by 1.9 percent per year in people younger than 50 years(16,18).Another cancer for which the incidence rate has been rising in US young adults is cervical cancer(19,20).Specifically,a recent analysis revealed that cervical cancer incidence among women ages 30 to 34 years increased by 2.5 percent per year between 2012 and 2019(20).Considering that cervical cancers are largely preventablemost cases are caused by infection with human papillomavirus(HPV),and HPV vaccination(see Prevent and Eliminate Infection From Cancer-causing Pathogens,p.54)and cervical cancer screening are extremely effective in reducing the burden of cervical cancerthese data emphasize the critical importance of public health measures to boost cervical cancer prevention and early detection in the United States(see Screening for Early Detection,p.63).In this regard,research has shown that in young women who were most likely to have received the HPV vaccines,cervical cancer incidence is declining rapidly.As one example,in women aged 20 to 24 years,invasive cervical cancer incidence decreased by 65 percent from 2012 to 2019 compared to only by 24 percent from 2005 to 2012(2).Cancer:An Ongoing ChallengeAlthough incredible progress has been made against cancer,it continues to be an enormous public health challenge in the United States and around the world.In the United States,an estimated 2,001,140 new cases of cancer will be diagnosed in 2024 and 611,720 people will die from the disease(see Table 1,p.17).Men have a higher incidence of many cancer types,including bladder,colon,and brain cancer,compared to women,and ongoing research is evaluating the role of a range of biological factors including genetics,epigenetics,metabolism,and immunity in mediating the sex differences in cancer burden(21-23).In addition,many population groups in the United States experience disproportionately high rates of cancer incidence and death that are attributable largely to structural and socioeconomic Often referred to as central nervous system tumors,these cancers are the second leading cause of cancer-related death in AYAs and the leading cause of death for those between 15 and 24 years old(14).AN ESTIMATED 208,620US ADOLESCENTS AND YOUNG ADULTS(AYA)ARE LIVING WITH A BRAIN TUMOR DIAGNOSIS IN 2024.W3Cancer in 2024AACR Cancer Progress Report 202416 disadvantages.It should also be noted that current estimates of the cancer burden do not reflect the adverse impact of COVID-19,which caused declines in screening,early detection,new cancer diagnoses,and delays or discontinuations in cancer treatment,especially for medically underserved populations(24-27).Ongoing monitoring of cancer-related data at a population level is warranted to assess the long-term consequences of COVID-19 for cancer burden in the United States.Inequities in the Burden of Cancer in the United StatesWhile we are making unprecedented advances against cancer,these advances have not benefited everyone equally.Because of a long history of structural inequities and systemic injustices in the United States,certain segments of the population continue to shoulder a disproportionate burden of adverse health conditions,including cancer.Cancer disparities are one of the most pressing public health challenges in the United States.The National Cancer Institute(NCI)defines cancer disparities as adverse differences in cancer-related measures,such as number of new cases,number of deaths,cancer-related health complications,survivorship and quality of life after cancer treatment,screening rates,and cancer stage at diagnosis,that exist among certain population groups(see Sidebar 2,p.18).As detailed in the AACR Cancer Disparities Progress Report 2024(29),US racial and ethnic minority groups and other medically underserved populations shoulder a disproportionately higher burden of cancer(see Sidebar 3,p.19).As one example,during 20172021,the incidence rate for all cancers combined was higher among American Indian and Alaska Native(AI/AN)people compared to non-Hispanic(NH)White people(3).During the same time,overall cancer mortality rates were higher among Black and AI/AN individuals compared to NH White individuals.Additionally,during 20142020,patients with cancer from all racial and ethnic minority groups had a lower 5year relative survival compared to NH White people(3).There has been progress in reducing cancer disparities in recent years.As one example,the gap in overall cancer death rates between Black and White populations has narrowed by more than 50 percent over the past two decades(3).However,Black individuals still had a 9 percent higher overall cancer death rate compared to White individuals,and the highest death rate from cancer among all US racial or ethnic groups,in 2022(3).Researchers studying the science of cancer disparities are increasingly recognizing the heterogeneity in the cancer burden within each of the major racial or ethnic minority groups.As one example,striking disparities in cancer burden Estimated Burden of Common Types of Cancer in the United States in 2024NEW CASESDEATHSAll Cancers Combined2,001,140611,720Breast 313,51042,780Prostate299,01035,250Lung and Bronchus234,580125,070Colorectal152,81053,010Melanoma(Skin)100,6408,290Bladder83,19016,840Kidney and Renal Pelvis81,61014,390Non-Hodgkin Lymphoma80,62020,140Uterine67,88013,250Pancreatic66,44051,750Thyroid 44,0202,170Liver and Intrahepatic bile duct 41,63029,840Myeloma 35,78012,540Source:(3).TABLE 1COVID-19 caused significant disruption of cancer careThese adversities were particularly severe among racial and ethnic minority groups and other medically underserved patients.Source:(28).W42020201920192020Early-stage diagnosisUnder-diagnosesLate-stage diagnosisCancer in 2024AACR Cancer Progress Report 202417have been identified within Asian subpopulations and between Native Hawaiian or other Pacific Islander(NHOPI)and Asian individuals(29).Notably,the US Asian population has ancestry in numerous countries of origin,and the NHOPI population comprises diverse subgroups with distinct variations in historical backgrounds,languages,and cultural traditions.However,Asian and NHOPI populations continue to be grouped together in cancer epidemiologic data.Stark differences in cancer incidence and outcomes have also been observed within the AI/AN populations when cancer data are disaggregated by geographic location.Research has shown that among AI/AN individuals,Indigenous Alaskans had the highest incidence of colorectal cancer between 2014 and 2018,compared to any other US racial population(36).The colorectal cancer incidence among Indigenous Alaskans was in fact the highest in the world in 2018(36).These findings indicate that collection of disaggregated data is a vital step to US Population Groups That Experience Cancer DisparitiesAccording to the National Cancer Institute(NCI),cancer disparities are adverse differences in cancer-related measures,such as number of new cases and deaths,cancer-related health complications,quality of life after cancer treatment,financial burden,screening rates,and stage at diagnosis that are shouldered by certain population groups including:Individuals belonging to certain ancestry,racial or ethnic minority populationsIndividuals of low socioeconomic status(SES),including low educational attainmentIndividuals who lack or have inadequate health insurance coverageIndividuals belonging to sexual and gender minority communitiesIndividuals with disabilitiesAdolescents and young adults(AYA)(Ages 1539)Individuals who are incarceratedImmigrants,refugees,or asylum seekersOlder adults(Ages 65 )Citizens of sovereign Native NationsResidents in certain geographic locations,including rural areas,or of certain types of neighborhoods,such as those with low access to resourcesSIDEBAR 2PASSPORTPASSPORTDeaths from all cancers combined are higher among Chinese,Korean,and Vietnamese subgroups and lower among Japanese and Indian subgroups compared to an aggregated Asian American reference group.Source:(37).W5Asian American22&%ChineseVietnameseKoreanJapaneseIndian19ncer in 2024AACR Cancer Progress Report 202418 fully understanding cancer disparities and developing effective strategies for achieving health equity.In addition to racial and ethnic minority groups,many segments of the US population shoulder a disproportionate burden of cancer.These include residents in rural areas that lack access to cutting-edge cancer treatments and/or state-of-the-art health care facilities,sexual and gender minorities(SGM)who experience bias and discrimination in health care settings,and low-income households in counties with persistent poverty and limited access to healthy food and/or the needed health care.In addition,older adults,veterans,undocumented immigrants and refugees,individuals with disabilities,individuals who are incarcerated,adolescents,and young adults all are medically underserved to varying degrees and face unique challenges in the burden of cancer.Cancer Inequities in the United StatesMany segments of the US population shoulder a disproportionate burden of cancer.Selected examples of disparate cancer incidence and outcomes from recent studies are provided here.Disparities in other aspects of cancer care are highlighted in relevant sections throughout the report.An in-depth discussion of cancer disparities and recent progress in addressing these inequities is detailed in AACR Cancer Disparities Progress Report 2024(29).40%more likelyBREAST CANCER MORTALITYBlack women with breast cancer are nearly 40 percent more likely to die from it compared to White women with breast cancer(3).16%and 9%less likely5-YEAR SURVIVALBlack and Latino individuals with lung cancer are 16 percent and 9 percent less likely,respectively,to survive 5 years after their diagnoses compared to White individuals(30).79%and 98%higherKIDNEY CANCER INCIDENCEThe incidence of kidney cancer is 79 percent higher among non-Hispanic American Indian/Alaska Native(AI/AN)men and 98 percent higher among non-Hispanic AI/AN women compared to non-Hispanic White men and women(31).12%and 13%higherTOBACCO-ASSOCIATED AND HPV-ASSOCIATED CANCER INCIDENCEThe incidence of tobacco-associated cancers and human papillomavirus(HPV)-associated cancers are 12 percent and 13 percent higher,respectively,among rural populations compared to their urban counterparts(32).1.73X and 2.26X more likelyCANCER IN SEXUAL AND GENDER MINORITY POPULATIONSGay men are 1.73 times more likely to be diagnosed with cancer than heterosexual men,while lesbian women are 2.26 times more likely to be diagnosed with cancer than heterosexual women(33).18-21%and 64-69%higherPATIENTS WITH DISABILITIESThe likelihood of dying from gastric cancer is 18 percent to 21 percent higher in patients with disabilities and 64 percent to 69 percent higher in patients with severe disabilities,compared to individuals without disabilities(34).7.1%higherPERSISTENT POVERTYDuring 20142018,deaths from all cancers combined were 7.1 percent higher in counties experiencing persistent poverty compared to counties not experiencing persistent poverty(35).SIDEBAR 3Cancer in 2024AACR Cancer Progress Report 202419It should be noted that patients with intersectional identities,for instance,racial or ethnic minority patients from SGM communities,often experience multilevel barriers to cancer care that adversely impact screening,diagnosis,treatment,and survivorship.As another example,older adults(age 65 or older)with cancer often experience multilevel barriers to cancer care and those living in rural areas have an even greater burdenmore likely to die within 1 year of cancer diagnosiscompared to those living in urban areas(38).Cancer disparities are driven by complex and interrelated factors.Systemic inequities resulting from a long history of racism and contemporary injustices in the United States continue to have lasting,multigenerational adverse effects on marginalized populations in all aspects of life,including on health outcomes.Researchers use various frameworks to understand and address the influences that affect health outcomes and contribute to health disparities,including cancer disparities.These frameworks integrate influences from structural factors and include the interplay of biological factors,mental health,and modifiable risk factors(e.g.,smoking and diet)with nonclinical factors called social drivers of health(SDOH)(39,40).According to NCI,SDOH,sometimes also called social determinants of health,are the social,economic,and physical conditions in the places where people are born and where they live,learn,work,play,and get older that can affect their health,well-being,and quality of life.Social drivers of health include factors such as socioeconomic status;housing;transportation;and access to healthy food,clean air and water,and health care services(see Figure 3,p.21).A major social driver of cancer disparities is inadequate access to quality health care.Health insurance is a key determinant of whether individuals receive the needed health care.In 2021,nearly 27 percent of US adults ages 18 to 64 who were uninsured delayed or did not receive needed medical care,compared to a little over 7 percent of those who had either public or private insurance(41).A substantial proportion of racial and ethnic minorities and medically underserved populations in the United States lack health care access(29).Individuals lacking health insurance are less likely to be up to date with recommended cancer screening and are more likely to be diagnosed with cancer at an advanced stage(29).Uninsured patients are also less likely to receive needed treatments and more likely to experience worse cancer outcomes compared to privately insured patients(42-45).Considering that a significant proportion of the US population is affected by cancer disparities,it is important that public health experts intensify efforts designed to improve the understanding and mitigation of these inequities.Only with new insights obtained through innovative and inclusive science,such as basic research assessing the effects of chronic stress and using biospecimens from diverse populations,clinical studies involving participants from all sociodemographic backgrounds,and health care delivery and implementation research that is representative of every community,will we be able to achieve health equity for all populations.Variable Progress Against Different Types of Cancer and Stages of DiagnosisA significant challenge in cancer science and medicine is the uneven progress against different cancer types and different stages of a given cancer type.This challenge is illustrated by the fact that the 5-year relative survival rates for US patients vary widely depending on both the type of cancer diagnosed and the stage at diagnosis(3).For example,the overall 5-year relative survival rates of 91 percent for patients with breast cancer and nearly 98 percent for patients with prostate cancer stand in stark contrast to the overall 5-year relative survival rates of 13 percent for those with pancreatic cancer or 8 percent for those with glioblastoma multiforme(GBM),an aggressive form of brain cancer(3).In addition,among women with breast cancer and men with prostate cancer,those with early-stage disease,i.e.,whose cancer is confined to the breast,or to the prostate,have 5-year relative survival rates of almost 100 percent,while those whose cancer has spread to other organs the 5-year relative survival rates are 32 percent and 37 percent,respectively(3).Notably,the greater 5-year survival among individuals whose cancers were caught early through screening can be partly attributed to lead time bias,a phenomenon where early diagnosis falsely makes it appear that people are surviving longer(see Screening for Early Detection,p.63).Variable progress against different cancer types can be accredited in part to disparities in lifesaving therapeutic options that are available for different cancer types.As an example,just in the past decade,FDA has approved 14 molecularly targeted therapeutics and two immunotherapeutics for the treatment of patients with breast cancer.As a result,patients have a deep selection of therapeutics to choose from and breast cancer mortality has been declining steadily;between 2013 and 2022 the breast cancer death rate fell by an average of 1.2 percent per year(3).In contrast,progress has been slow for patients with GBM.Since the approval of the chemotherapeutic temozolomide nearly 25 years ago,no new anticancer agents have shown promise in improving overall survival.Consequently,the 5-year relative survival rate for patients with GBM remains at a dismal 8 percent(3).Developing new and effective tests for the early detection of more cancer types(see Screening for Early Detection,Cancer in 2024AACR Cancer Progress Report 202420 Why Do US Cancer Disparities Exist?Complex and interrelated structural and social factors,stemming from a long history of racism and discrimination,drive cancer disparities.These factors include social drivers of health(SDOH)as well as biological factors,mental health,and modifiable risk factors.The National Cancer Institute defines SDOH,sometimes also known as social determinants of health,as the social,economic,and physical conditions in the places where people are born and where they live,learn,work,play,and get older that can affect their health,well-being,and quality of life.Social drivers of health have a major influence on peoples physical and mental health,well-being,and quality of life.In the United States,historical racism and contemporary injustices have perpetuated and exacerbated systemic inequities,resulting in adverse differences in SDOH for racial and ethnic minorities and medically underserved populations.The circle in the figure depicts the complex and interconnected key drivers of health and how they influence both at societal and community levels and at the individual level.Selected examples of the multilevel factors that make up drivers of health are highlighted.Collectively,these factors impact every stage of the cancer continuum,leading to worse health outcomes for racial and ethnic minorities and other underserved populations(shown at the bottom).FIGURE 3DEVELOPMENTRISK REDUCTIONEARLY DETECTIONTREATMENTSURVIVORSHIPCANCER RESEARCH AND CARE TRAINEES AND WORKFORCEEDUCATORS CANCER RESEARCHERS PHYSICIANS PHYSICIAN-SCIENTISTS HEALTH CARE PROFESSIONALSCANCER CARE CONTINUUMRacism Discrimination SegregationStructural Inequities Societal InjusticesDisparities InAdverse Health OutcomesDriversof HealthBEHAVIORALDiet,Tobacco use,Excess body weight,Physical inactivityENVIRONMENTALAir/water quality,Community safetyHousing,Transportation,CLINICALHealth care qualityHealth care access,PSYCHOSOCIAL IsolationStress,Mental health,BIOLOGICALGenetic,Epigenetic,Tumor microenvironmentSOCIOECONOMICEducation,Income,EmploymentCULTURALHealth beliefs,Health-related attitudesCancer in 2024AACR Cancer Progress Report 202421p.63)could also help address the challenge of variable progress between types of cancer because the likelihood of a cure is much higher when cancer is diagnosed at an early stage when it is confined to its original location and has not spread to distant sites.Additionally,intensive research to uncover currently unknown biological drivers of cancer initiation and progression is needed to identify novel targets and improve therapeutic options for hard-to-treat cancers.The Growing Population Burden of CancerThe public health challenges posed by cancer are predicted to grow considerably in the coming decades unless we develop and implement more effective strategies for cancer prevention,early detection,and treatment.In the United States alone,the number of new cancer cases diagnosed each year is expected to surpass 2.5 million by 2050(46).This is because cancer is primarily a disease of aging;57 percent of diagnoses occur among those 65 and older(2),and this segment of the US population is expected to grow from 57.8 million in 2022 to more than 82 million in 2050(47).Also contributing to the projected increase in the number of US cancer cases are the high rates of excess body weight,physical inactivity,and alcohol consumption(48)and the continued use of cigarettes by 11.5 percent of adults(49)(see Reducing the Risk of Cancer Development,p.43).Furthermore,a significant proportion of lung cancers(16 percent in women and 10 percent in men)are diagnosed in individuals without a history of smoking(50)and there is an urgent need for more research to understand the increasing trends of lung cancer incidence among those without a history of smoking(51).In this regard,a recent study showed that in Asian and Pacific Islander adults who never smoked,lung cancer incidence increased by an average of 2 percent per year between 2007 and 2018,while rates were stable in other US racial and ethnic groups(52).Identification of risk factors,a deep characterization of their disease,and the development of evidence-based early detection and treatments are critical needs to lower the burden of lung cancer in this population of patients who do not have a history of smoking.While overall cancer incidence in the United States has stabilized in recent years,the incidence of certain cancer types as well as the age-and sex-specific incidence of certain cancers is increasing.For example,the incidence of pancreatic cancer,uterine cancer,and HPV-associated oral cancers has Source:(46).W61.83MILLION20222.53MILLION20500.60MILLION20220.97MILLION2050Cancer CasesCancer DeathsESTIMATEDESTIMATEDBurden of Colorectal Cancer in the United States(20172021)Source:(3).W7Age 503.2%increaseevery yearAge 65 2.0creaseevery year Age 30Attributable to Selected Factors01020515UVReducing the Risk of Cancer DevelopmentAACR Cancer Progress Report 202444 from 42.4 percent in 1965 to 11.5 percent in 2021(49).However,even in 2021,the most recent year for which such data are available,an estimated 46 million US adults reported using any tobacco product(e.g.,cigarettes,cigars,pipes)(49).In the United States there are geographical differences in smoking rates,which has led to certain regions with high smoking prevalence being associated with higher rates of lung cancer mortality(183).There are striking sociodemographic disparities in the use of tobacco products as well as in exposure to secondhand smoke,also a cancer risk factor,as highlighted in the AACR Cancer Disparities Progress Report 2024.Overall tobacco use is higher among US residents who live in rural areas and in the Midwest,those with lower levels of household income and educational attainment,those who are uninsured or insured by Medicaid,those experiencing psychological distress,and those who have a disability(49,184).Furthermore,US adults who identify as belonging to the sexual and gender minority(SGM)population have higher rates of using tobacco products.Beyond the Lungs:Cancers Caused by Smoking TobaccoSmoking tobacco increases an individuals risk of developing not only lung cancer,but also 17 other types of cancer.No level of exposure to tobacco smoke is safe,including exposure to secondhand smoke.Use of smokeless tobacco(such as chewing tobacco and snuff)can cause oral,esophageal,and pancreatic cancer.Source:(1,48).FIGURE 10Nasal CavityNasopharynxOral CavityOropharynxHypopharynxHEAD AND NECKLarynxLUNG AND BRONCHUSHEMATOPOIETIC SYSTEMAcute MyeloidLeukemiaUROGENITAL SYSTEMKidneyUreterBladderOvary*Uterine Cervix*Certain subtypes of ovarian cancerDIGESTIVE SYSTEMEsophagusStomachPancreasLiverColonSmoking Prevalence by US RegionSource:(49).W13WestMidwestSouthNortheast12.4%8.9.4%Reducing the Risk of Cancer DevelopmentAACR Cancer Progress Report 202445Similar to the trend in adults,tobacco use among US youth such as middle and high school students is also declining,with 6.6 percent of middle and 12.6 percent of high school students reporting current use of a tobacco product in 2023(185).The number of high school students with current use of any tobacco
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AACR CANCER PROGRESS REPORT 2025AACR.org CancerProgressReport.org#CancerProgressReportFORUNIFYINGA CONTINUUM OFINNOVATIONIMPACTANDCANCERSCIENCEMEDICINE:Spotlight on ADVANCES IN BLOOD CANCERAACR CANCER PROGRESS REPORT 2025ISBN:979-8-9857852-8-9AACR CANCER PROGRESS REPORT 2025AACR.org CancerProgressReport.org#CancerProgressReportPLEASE CITE THIS REPORT AS:American Association for Cancer Research.AACR Cancer Progress Report 2025.Accessed:Month Day,Year.Available from:LinkTable of ContentsMessage From AACR .1Executive Summary .3Snapshot of a Year of Progress .12Cancer in 2025 .14Research:Driving Progress Against Cancer .14Cancer:An Ongoing Challenge .18Inequities in the Burden of Cancer in the United States.19Variable Progress Against Different Types of Cancer and Stages of Diagnosis.21The Growing Population Burden of Cancer.23The Global Burden of Cancer.24Funding Cancer Research:A Vital Investment .26Understanding the Path to Cancer Development .29Cancer Development:Generating Knowledge Through Basic Research .29Basic Research Decoding Cancers Complexities.31Cancer Development:Translating Knowledge to Understand the Biology of Cancer .34Influences Inside the Cell.34Genetic Alterations .34RNA Variations .37Protein Modifications .38Epigenetic Changes .40Influences Outside the Cell.40Circulatory and Immune Systems .41The Nervous System .43Microbiome .44Tumor Microenvironment .45Cancer Development:Integrating Knowledge to Advance Precision Medicine .46Technological Innovations Fueling Precision Medicine.46Reducing the Risk of Cancer Development .51Eliminate Tobacco Use .53Maintain a Healthy Weight,Eat a Healthy Diet,and Stay Active .56Reduce Alcohol Consumption .61Protect Skin From UV Exposure .62Prevent and Eliminate Infection From Cancer-causing Pathogens .64Limit Exposure to Environmental Risk Factors .67Where You Live Matters.69Where You Work Matters.69Be Cognizant of Hormonal Factors .69Family Planning,Pregnancy,and Breastfeeding.69Hormone Replacement Therapy.70Cancer Screening for Early Detection .71Routine Cancer Screening Saves Lives.71Evidence Guides Cancer Screening Recommendations .73Eligibility Criteria for Cancer Screening.73Recommendations for Cancer Screening.73Tests for Cancer Screening.76Research Improves the Science of Screening .76Effective Strategies Increase Screening Uptake .78Reducing Structural Barriers.80Integrating Patient Navigation.81Engaging Communities.82Technological Innovations Advance Cancer Screening for Early Detection .82Advances in Artificial Intelligence(AI).82Advances in Minimally Invasive Screening Approaches.84Advances in Imaging.85Genetic Insights Advancing Cancer Risk Reduction and Interception.85Unifying Cancer Science and Medicine:A Continuum of Innovation for Impact .88Medical Research .88Clinical Research .89Innovations in Cancer Clinical Trials.90Progress Across the Clinical Cancer Care Continuum .96Advances in Cancer Treatment With Surgery.99Minimizing the Use of Invasive Cancer Surgery .100Advances in Radiation-based Approaches to Cancer Care.102Applying Precision to Radiation Therapy .105Advances in Treatment With Chemotherapy .105Advances in Treatment With Molecularly Targeted Therapeutics.106Advancing Targeted Approaches in Breast Cancer Care .108AACR Cancer Progress Report 2025II Matching Therapies to the Molecular Drivers of Lung Cancer .110Delivering Cytotoxic Agents Precisely to Cancer Cells .113Innovating Ovarian Cancer Treatment Through a Novel Combination .117Personalizing Treatments for Patients With Rare Cancers .117Bringing the Promise of Precision Medicine to Children,Adolescents,and Young Adults With Cancer .123Advances in Treatment With Immunotherapeutics.128Releasing the Brakes on the Immune System .128Boosting the Cancer-killing Power of Immune Cells .134Directing the Immune System to Cancer Cells .137A Decade of Progress:Transformative Advances in Blood Cancer .144The State of Blood Cancers in 2025 .144Leukemias.145Lymphomas.149Multiple Myeloma.149Blood Cancers:An Ongoing Challenge .150Global Burden of Blood Cancers.150Disparities in the Burden of Blood Cancers in the United States.151Unique Biology of Blood Cancers .151Precision Medicine Driving Advances Against Blood Cancers .154Diagnosing Blood Cancers With Increasing Precision.156A Revolution in Molecularly Targeted Therapies.156Breakthroughs in Treating Leukemias .156Milestones in Managing Lymphomas .159Progress in Targeting Multiple Myeloma .163Transforming Blood Cancer Treatment With Immunotherapy.164Harnessing CAR T Cells to Treat Blood Cancers .164Unleashing the Immune System Against Blood Cancers .167Redirecting T Cells to Eradicate Blood Cancers .167Flagging Blood Cancer Cells for Destruction by the Immune System.169Supporting Survivorship .170New Horizons in Blood Cancer Science and Medicine .171Intercepting Precancerous Conditions in Blood Cancers.171Precancerous Conditions That May Become Myeloid Malignancies .172Precancerous Conditions That May Become Lymphoid Malignancies .173Precancerous Conditions That May Become Multiple Myeloma .173Modernizing Clinical Trial Design for Blood Cancers.174Supporting Cancer Patients and Survivors .177Addressing the Challenges Faced by Survivors .178Physical Challenges.178Psychosocial Challenges.180Financial Challenges.181Addressing the Unique Challenges Faced by Vulnerable Populations .181Challenges Faced by Children.181Challenges Faced by Adolescents and Young Adults.182Challenges Faced by Older Adults.184Improving Quality of Life and Health Outcomes .185Participating in Physical Activity.185Eating a Healthy Diet and Maintaining a Healthy Weight.186Eliminating Alcohol and Tobacco Use.186Integrating Palliative Care.187Improving Mental Health.188Delivering Care to Cancer Survivors.190Coordinating Care.190Leveraging Patient Reported Outcomes.190Supporting Caregivers .192Envisioning the Future of Cancer Science and Medicine .194A New Wave of Technologies .194New Advances in Artificial Intelligence.194Accelerating Basic Research Discoveries .195Improving Detection and Diagnosis .195Advancing New Treatments .199Enhancing Clinical Decision-Making .200Fairness and Oversight.201AACR Cancer Progress Report 2025IIICRISPR in Cancer Research and Drug Development.201Basic Research Discoveries .202Drug Development .202Next-Generation Treatments .202Liquid Biopsy in Cancer Care.203Diagnosing Cancer and Predicting Metastases .203Monitoring Treatment Response and Predicting Resistance .203Detecting Minimal Residual Disease and Recurrence .203New Frontiers in Cancer Treatment .205Cancer Vaccines.205Radiopharmaceutical Therapy.207Advancing Cancer Science and Medical Research Through Evidence-based Policies .209Investing in Research to Achieve a Healthier Future .209A Diverse Cancer Research and Care Workforce Drives Innovation .214Applying Regulatory Science to Ensure Safe and Effective Cancer Therapies .214Increasing Access to and Decentralizing Trials .216Rapidly Delivering Safe and Effective Therapies to Patients .216Regulatory Collaboration and Global Harmonization .217Advancing Policies to Strengthen Cancer Prevention and Screening Programs .218Mitigating Preventable Risk Factors .218Improving Screening Guidelines and Risk-based Approaches.220Cancer Prevention and Screening Programs.220Leveraging Policy to Reduce Tobacco-related Illness .221Flavored Tobacco Products.222Funding Federal Regulatory,Enforcement,and Control Activities.223Addressing Cancer Disparities and Improving Patient Outcomes .224Environmental Justice and Cancer.224Patient-centered Care and Outcomes and Survivorship.224Improving the Use of Digital Information in Cancer Treatment and Management .226AACR Call to Action .227References .229Glossary .255Appendix.263Index .266AACR Initiatives Accelerating Cancer Research .267AACR Cancer Progress Report 2025IV List of Report GraphicsFiguresFigure 1:Increasing Innovative Treatment Options for Advanced Melanoma,p.16Figure 2:Progress Against Cancer in Children and Adolescents,p.17Figure 3:Why Do US Cancer Disparities Exist?,p.22Figure 4:Hallmarks of Cancer Cells,p.30Figure 5:The Medical Research Cycle Driving Progress Against Cancer,p.32Figure 6:Inherited Cancer Risk,p.36Figure 7:Precision Medicine,p.47Figure 8:Modifiable Cancer Risks,p.52Figure 9:Cancers Caused by Smoking Tobacco,p.54Figure 10:Reasons to Maintain a Healthy Weight and Stay Active,p.56Figure 11:Alcohol and Cancer Risk,p.62Figure 12:What Can Cancer Screening Find and What Can Be Done?,p.72Figure 13:Cancer Screening Is a Continuum,p.76Figure 14:USPSTF Recommendation for Breast Cancer Genetic Testing,p.86Figure 15:Timeline of Tissue-agnostic FDA Approvals of Cancer Treatments,p.92Figure 16:The Pillars of Cancer Treatment,p.97Figure 17:Precision Medicine Driving Progress Against Lung Cancer,p.111Figure 18:Timeline of First FDA Approvals of Immune Checkpoint Inhibitors for Different Cancer Types,p.131Figure 19:Expanding Scope of Immune Checkpoint Inhibitors,p.132Figure 20:Blood Formation in Adults,p.145Figure 21:The Pathway to Progress Against Chronic Myelogenous Leukemia,p.148Figure 22:Generation of Fusion Proteins Through Chromosomal Rearrangements,p.154Figure 23:Molecularly Targeting Vulnerabilities of Cancer Cells in Acute Myeloid Leukemia,p.158Figure 24:Intercepting Cancer,p.172Figure 25:NIH Funding and the Importance of Continuing Robust Increases,p.211Figure 26:Federal Programs Contribute to Decreasing E-cigarette Use Among Youth,p.222SidebarsSidebar 1:The Medical Research Community:Driving Progress Together,p.15Sidebar 2:US Population Groups That Experience Cancer Disparities,p.19Sidebar 3:Cancer Inequities in the United States,p.20Sidebar 4:What Is Causing the Rise in Early-onset Cancers?,p.25Sidebar 5:Funding Medical Research.Accelerating Discoveries.Saving Lives.,p.27Sidebar 6:Commonly Used Models in Cancer Research,p.31Sidebar 7:Basic Research Driving Clinical Advances Against Cancer,p.33Sidebar 8:What Are Genetic Alterations?,p.35Sidebar 9:Extrachromosomal DNA in Cancer,p.37Sidebar 10:Technologies Accelerating Discovery in Cancer Development,p.39Sidebar 11:Cancer Growth:Local and Systemic Influences,p.41AACR Cancer Progress Report 2025VSidebar 12:The Therapeutic Impact of Understanding Cancer Biology,p.46Sidebar 13:The Human Tumor Atlas Network,p.48Sidebar 14:Cancer Grand Challenges,p.49Sidebar 15:Emerging Role of Stress in Cancer Risk,p.53Sidebar 16:Ways to Reduce Cancer Risk,p.57Sidebar 17:Therapeutic Interventions to Reduce Obesity,p.59Sidebar 18:Physical Activity Guidelines,p.61Sidebar 19:Guidelines for Alcohol Consumption,p.63Sidebar 20:Ways to Protect Your Skin,p.64Sidebar 21:Ways to Reduce Cancer Risk From Pathogens,p.66Sidebar 22:Per-and Poly-fluoroalkyl Substances(PFAS)The Forever Chemical,p.68Sidebar 23:Differences Between Routine Screening,Genetic Testing,and Diagnostic Testing,p.74Sidebar 24:USPSTF Guidelines for Cancer Screening,p.75Sidebar 25:USPSTF-recommended Tests to Screen for Cancer,p.77Sidebar 26:Disparities in Adherence to Routine Cancer Screening and Follow-up Care,p.80Sidebar 27:Therapeutic Development,p.90Sidebar 28:A New Age of Cancer Clinical Trials,p.91Sidebar 29:Disparities in Clinical Trial Participation,p.94Sidebar 30:Disparities in Cancer Treatment,p.99Sidebar 31:Using Surgery for Cancer Treatment,p.100Sidebar 32:Commonly Used Terms in Clinical Studies,p.101Sidebar 33:Using Radiation in Cancer Treatment,p.104Sidebar 34:The Increasing Precision of Molecularly Targeted Therapeutics,p.107Sidebar 35:Companion Diagnostics,p.108Sidebar 36:Targeting the Undruggable KRAS,p.109Sidebar 37:The Challenge of Treatment Resistance,p.110Sidebar 38:The Challenges Posed by Rare Cancers,p.122Sidebar 39:Key Cells of the Immune System,p.129Sidebar 40:How Immunotherapeutics Work,p.130Sidebar 41:T-cellbased Adoptive Cell Therapy,p.135Sidebar 42:Targeting Brain Tumors With CAR T Cells,p.137Sidebar 43:Types of Blood Cancers,p.146Sidebar 44:Disparities Across the Continuum of Blood Cancer Research and Care,p.152Sidebar 45:Key Differences Between Blood Cancers and Solid Tumors,p.153Sidebar 46:CAR T-cell Therapies Approved by the US Food and Drug Administration(FDA),p.165Sidebar 47:MyeloMATCH Precision Medicine Trials:A New Era in Blood Cancer Treatment,p.175Sidebar 48:Phases of Cancer Survivorship,p.179Sidebar 49:Fertility Preservation After a Diagnosis of Cancer,p.183Sidebar 50:What Is Palliative Care?,p.187Sidebar 51:Cancer Survivorship Experience and Resilience,p.189Sidebar 52:Patient Navigation for Cancer Survivors,p.191Sidebar 53:Patient Reported Outcomes,p.192Sidebar 54:Understanding Artificial Intelligence and Its Impact on Cancer Care,p.198Sidebar 55:Liquid Biopsy,p.204AACR Cancer Progress Report 2025VI Sidebar 56:Next-generation KRAS Therapies,p.206Sidebar 57:Radioligand Therapy,p.207Sidebar 58:FDA Milestones in Cancer Drug Development(20112025):15 Years of Progress in Advancing Oncology Innovation,p.215Sidebar 59:DCPC Public Health Campaigns and Screening Tools,p.221TablesTable 1:Estimated Burden of Common Types of Cancer in the United States in 2025,p.18 Table 2:Cancer Types and Cancer Cases Caused by Pathogens Globally,p.65Table 3:Percentage of Eligible Individuals Up to Date With USPSTF Screening Guidelines in the United States,p.79Table 4:A Selected List of FDA-approved AI-assisted Medical Devices and Software in Cancer Early Detection (July 1,2024June 30,2025),p.83Table 5:FDA-approved Minimally Invasive Tests and Devices for Cancer Screening for Early Detection (July 1,2024June 30,2025),p.84Table 6:Newly FDA-approved Anticancer Agents(July 1,2024June 30,2025),p.98Table 7:Most Common Blood Cancers:Estimated Number of New Cases and Deaths in the United States and Globally in 2025,p.147Table 8:Survival Rates of the Most Common Subtypes of non-Hodgkin Lymphoma,p.149Table 9:A Selected List of Repurposed FDA-approved Precision Medicine Drugs,p.155Table 10:FDA-approved New Molecularly Targeted Therapies Against Leukemias(20152025),p.157Table 11:FDA-approved New Molecularly Targeted Therapies Against Lymphomas(20152025),p.162Table 12:FDA-approved New Molecularly Targeted Therapies Against Multiple Myeloma(20152025),p.163Table 13:FDA-approved New Molecularly Targeted Therapies to Treat Other Hematologic Malignancies(20152025),p.164Table 14:Selected Clinical Trials Evaluating Interception of High-risk Smoldering Multiple Myeloma Using T-celldirecting Therapies,p.174Supplementary MaterialSupplementary Table 1:Carcinogenic Toxins Released by Wildfires,p.263Supplementary Table 2:Surgeries for the Prevention of Cancer,p.264 Supplementary Table 3:Newly FDA-approved Anticancer Agents(July 1,2024June 30,2025),p.265Scan the QR code to download report graphicsAll report graphics are free to download.Please include the following copyright language for public use(e.g.,presentations):2025 American Association for Cancer Research.AACR Cancer Progress Report 2025.AACR Cancer Progress Report 2025VIIRichard Schlueter .212Diagnosis:Head and Neck Cancer(Oropharyngeal Cancer)“When science is disrupted,hope is delayedand for patients like me,time is everything.”Bob Fortin .114 Diagnosis:Pancreatic Cancer“Weve got to beat this disease.And the only way to do that is by funding research.”Survivor SpotlightsMichelle Anderson-Benjamin .118Diagnosis:Triple-Negative Breast Cancer“Give us the science.Give us the trials.Give us the choice.”Dawn Varrati .124Diagnosis:Bile Duct Cancer(Cholangiocarcinoma)“You want to change the cancer story for millions of people?Fund the science.Fund the trials.”Alex Hepner .126Diagnosis:Brain Cancer(Glioma)“Im a walking,talking example of the difference cancer research can make.”Quinn Johnsen .138Diagnosis:Synovial Sarcoma“Afami-cel was a silver bulletbuilt from decades of research.We need to fund more of them.”John C.(Jack)Moorman .160Diagnosis:Acute Myeloid Leukemia“Cancer research saved my fathers life.Now it has saved mine.”Greg Myers .142 Diagnosis:Stomach Cancer(Gastric Cancer)“Im still here.Im still fighting.And Ill keep going as long as theres another option to try.”Mary Catherine Riley.120 Diagnosis:Ovarian Cancer“For my type of cancer,there was no treatment.Now there is,because of research.”AACR Cancer Progress Report 2025VIII Steering CommitteeLillian L.Siu,MD,FAACR,FRCPCSteering Committee ChairAACR President 20252026Professor,University of TorontoMedical Oncologist,Princess Margaret Cancer CentreDirector,Phase I Clinical Trials ProgramBMO Chair in Precision Cancer GenomicsPrincess Margaret Cancer Centre,University Health NetworkToronto,Ontario,CanadaPeter J.Allen,MDDavid C.Sabiston,Jr.Distinguished Professor of SurgeryChair,Department of SurgeryChief,Division of Surgical OncologyHealth System Vice President for Cancer ServicesMember of the Duke Cancer InstituteDuke University School of MedicineDurham,North CarolinaKenneth C.Anderson,MD,PhD(hc)Kraft Family Professor of Medicine at Harvard Medical SchoolDirector,LeBow Institute for Myeloma Therapeutics and Jerome Lipper Multiple Myeloma CenterDana-Farber Cancer InstituteDoris Duke Distinguished Clinical Research Scientist American Cancer Society Clinical Research ProfessorBoston,MassachusettsMichael A.Carducci,MD,FACP,FASCOAEGON Professor in Prostate Cancer ResearchVice Chair for Solid Tumor OncologyMember,Miller-Coulson Academy of Clinical ExcellenceSidney Kimmel Comprehensive Cancer Center at Johns Hopkins Baltimore,MarylandAndrew E.Chapman DO,FACP,FCPPDirector,Sidney Kimmel Comprehensive Cancer CenterEVP Cancer Research and Oncology Services,JeffersonProfessor of Medical OncologyClinical Professor,Jefferson College of NursingCo-Director,Jefferson Senior Adult Oncology CenterPhiladelphia,PennsylvaniaSteven J.Chmura,MD,PhDProfessor of Radiation and Cellular OncologyProfessor of MedicineProfessor of Neurological SurgeryMedical Director,Comprehensive Cancer CenterDirector,Clinical and Translational Research for Radiation OncologyUniversity of Chicago MedicineChicago,IllinoisMargaret Foti,PhD,MD(hc)Chief Executive OfficerAmerican Association for Cancer ResearchPhiladelphia,PennsylvaniaSophia H.L.George,PhDProfessor,Department of Ob,Gyn and Reproductive SciencesDivision of Gynecologic OncologyAssociate Director,Sylvester Comprehensive Cancer CenterUniversity of Miami Miller School of MedicineMiami,FloridaLucy A.Godley,MD,PhDJeffrey and Marianne Silver Family Professor of OncologyProfessor,Medicine(Hematology and Oncology)Northwestern University Feinberg School of MedicineChicago,IllinoisAna P.Gomes,PhDPrincipal InvestigatorAssociate Member,Department of Molecular OncologyMoffitt Cancer CenterTampa,FloridaCarmen E.Guerra,MD,MSCE,FACPRuth C.and Raymond G.Perelman Professor and Vice Chair,MedicineAssociate Director of Community Outreach and Engagement,Abramson Cancer CenterPerelman School of Medicine,University of PennsylvaniaPhiladelphia,PennsylvaniaTrey Ideker,PhDProfessor,Department of Medicine Adjunct Professor,Departments of Bioengineering and Computer Science Member,Moores Cancer CenterDirector,ARPA-H ADAPT,Dynamic Digital Tumors for Precision Oncology Director,Bridge2AI Cell Maps for AI Co-Director,Cancer Cell Map Initiative University of California San Diego La Jolla,CaliforniaMeredith S.Irwin,MDChief of Paediatrics,Oncologist and Senior ScientistThe Hospital for Sick Children(SickKids)Professor and Chair,Department of Paediatrics,Temerty Faculty of MedicineUniversity of TorontoToronto,Ontario,CanadaMarcela V.Maus,MD,PhDPaula J.OKeeffe Endowed Chair of the Massachusetts General Hospital Cancer CenterDirector,Cellular Immunotherapy ProgramProfessor,Hematology and Oncology,Massachusetts General Hospital and Harvard Medical SchoolBoston,MassachusettsSandra S.McAllister,PhDAssociate Professor of Medicine,Harvard Medical SchoolCo-Director,Aging and Cancer Research Labs,Division of Hematology,Brigham&Womens HospitalMember,Breast Cancer Program,Dana-Farber/Harvard Cancer CenterMember,Mass General Brigham Cancer InstituteAssociate Member,Broad InstituteBoston,MassachusettsJason A.Mendoza,MD,MPHProfessor and Associate Program Head,Cancer Prevention Program,Fred Hutchinson Cancer CenterAssociate Director,Community Outreach and Engagement,Fred Hutch/UW/Seattle Childrens Cancer ConsortiumProfessor,University of Washington,Seattle Childrens Research InstituteSeattle,WashingtonJohn M.Salsman,PhDDirector of Clinical Research in Adolescent and Young Adult(AYA)Oncology and Co-Leader,Cancer Prevention and Control Program,Atrium Health Wake Forest Baptist Comprehensive Cancer CenterProfessor,Social Sciences and Health Policy&Associate Director,Public Health Sciences,Wake Forest University School of MedicineWinston-Salem,North CarolinaThe AACR would like to acknowledge Electra D.Paskett,PhD,FACE,FAACR,Marion N.Rowley Professor of Cancer Research,James Cancer Hospital,The Ohio State University,for her help with the development of this report.AACR Cancer Progress Report 2025IxAACR StaffRajarshi Sengupta,PhDSenior Director,Scientific Research Analysis and Dissemination Sayyed Kaleem Zaidi,PhDDeputy Director,Scientific Research Analysis and DisseminationElizabeth Blackman,MPH,PhDScientific Research Analyst,Scientific Research Analysis and DisseminationKristin Primm,MPH,PhDScientific Research Analyst,Scientific Research Analysis and DisseminationBrett M.Sansbury,PhDSenior Scientific Research Analyst,Scientific Research Analysis and DisseminationHeather M.ClarkSenior Web/Print Designer,Marketing,Creative Services,and Multimedia ProductionJoshua F.GoldsteinSenior Director,Brand Strategy Communications,Communications and Public RelationsJenna M.BachenSenior Creative Director,Marketing,Creative Services,and Multimedia ProductionRichard G.BuckChief Communications Officer and Vice President,Communications and Public RelationsBrad Davidson,PhD Regulatory Science and Policy Analyst,Office of Science Policy and Government AffairsPaul J.Driscoll,Jr.Chief Marketing Officer and Vice President,Marketing,Creative Services,and Multimedia ProductionAraceli EstradaSenior Manager,Patient Advocacy,Office of Science Policy and Government AffairsMatt GontarchickAssociate Director,Government Affairs,Office of Science Policy and Government AffairsAndrew MatthiusSenior Writer and Editor,Communications and Public RelationsCarly McCallieDirector,Government Affairs,Office of Science Policy and Government AffairsJon G.Retzlaff,MBA,MPAChief Policy Officer and Vice President,Office of Science Policy and Government AffairsBlake RostineSenior Manager,Government Affairs,Office of Science Policy and Government AffairsCarrie Treadwell,MBASenior Director,Strategic Patient Advocacy and Engagement,Office of Science Policy and Government AffairsRukiya Umoja,PharmD,RPhAssociate Director,Regulatory Science and Policy,Office of Science Policy and Government AffairsDavid Zahavi,PhD Senior Science Policy Analyst,Office of Science Policy and Government AffairsAbout the American Association for Cancer ResearchFounded in 1907,the American Association for Cancer Research(AACR)is the worlds first and largest professional organization dedicated to advancing cancer research and its mission to prevent and cure cancer.AACR membership includes more than 58,000 laboratory,translational,and clinical researchers;population scientists;other health care professionals;and patient advocates residing in 141 countries and territories around the world.Presently,34%of members live outside the United States and 20%of AACRs international members are located in countries building cancer research capacity.The AACR marshals the full spectrum of expertise of the cancer community to accelerate progress in the prevention,diagnosis,and treatment of cancer by annually convening more than 30 conferences and educational workshops,the largest of which is the AACR Annual Meeting.The AACR publishes 10 prestigious,peer-reviewed scientific journals.Other AACR publications include Cancer Today,a magazine for cancer patients and caregivers;the annual AACR Cancer Progress Report;AACR Cancer Disparities Progress Report;AACR Annual Impact Report;Leading Discoveries,the AACRs awareness and donor magazine;and the blog,Cancer Research Catalyst.In addition,the AACR funds meritorious research directly as well as in cooperation with numerous cancer organizations.As the Scientific Partner of Stand Up To Cancer,the AACR provides expert peer review,grants administration,and scientific oversight of team science and individual investigator grants in cancer research that have the potential for near-term patient benefit.The AACR actively communicates with legislators and other policymakers about the value of cancer research and related biomedical science in saving lives from cancer.For more information about the AACR,visit AACR.org.We acknowledge Joanne Boehme for copy editing the document;Tanya Bondar,PhD,for help with the A Decade of Progress:Transformative Advances in Blood Cancer section;and Elizabeth Hubbard for project coordination and administrative support.AACR Cancer Progress Report 2025x Launched in 2011 to commemorate the 40th anniversary of the landmark National Cancer Act of 1971,which galvanized national efforts to conquer cancer,the AACR Cancer Progress Report series celebrates its 15th year in 2025.This milestone is a testament to the extraordinary progress against cancer that has been catalyzed by decades of transformative medical research and technological innovation.Discoveries across basic,translational,clinical,and population sciences are driving breakthroughs in cancer medicine,leading to a steady decline in overall US cancer death rate and allowing more people to live longer,fuller lives after a cancer diagnosis.Improvements in cancer prevention and early detection have averted an estimated 4.75 million deaths from five major cancer types over the past 45 years,underscoring the pivotal role of public health measures alongside medical innovation.As of January 1,2025,the number of cancer survivors in the United States reached 18.6 million,a significant increase from the 12 million survivors reported in our inaugural report,and this number is projected to surpass 22 million by 2035.Among the most critical drivers of the progress against cancer are precision medicine and cancer immunotherapy,which are revolutionizing how cancer is diagnosed and treated.When the first report was published in 2011,there were no cellular immunotherapies,and only one immune checkpoint inhibitor had been approved by the US Food and Drug Administration(FDA).As of June 30,2025,nine adoptive cell therapies have been approved,including the most recent groundbreaking T-cell receptor T-cell therapy for synovial sarcoma,and 15 immune checkpoint inhibitors are available for treating more than 20 cancer types.Innovations in molecularly targeted therapeutics continue to fuel precision medicine,such as the approval of the first menin inhibitor for acute myeloid leukemia;promising advances for patients with rare cancers,including the first IDH1/2 inhibitor for low-grade glioma;and the first-ever HER3-targeted therapy for NRG1 fusionpositive lung and pancreatic tumors.This year we also report FDA approvals of two new antibodydrug conjugates,a rapidly growing class of therapeutics,for the treatment of breast and lung cancers.These remarkable advances are a direct result of sustained federal investments in medical research and regulatory science,particularly through National Institutes of Health(NIH),National Cancer Institute(NCI),Centers for Disease Control and Prevention(CDC),and FDA.However,this progress is now at risk.In fiscal year(FY)2024,Congress reduced NIH funding for the first time in nearly a decade,and in 2025a year marked by grant delays,canceled clinical trials,mass layoffs in the research workforce,and political interferencethe cancer research and care community is facing its most serious funding crisis in a generation.Past funding reductions,such as those caused by the 2013 sequestration process,forced laboratories to shutter,some of which never resumed work,and delayed the development of high-potential therapies among other setbacks.Without robust,sustained,and predictable annual increases in funding,we risk reversing hard-won progress against cancer.In this regard,it is encouraging to see a bipartisan Senate effort not only to maintain current funding levels,but also to increase support for medical research,reaffirming the federal commitment to saving lives.The need for robust support is underscored by the fact that cancer still exacts a heavy human and economic toll across the nation and around the world.More than two million people in the United States are expected to be diagnosed with cancer in 2025,and over 600,000 will die from the disease.Around the globe,the incidence of certain cancersincluding colorectal,breast,gastric,and othersis rising among adults under 50.Stark disparities remain,with medically underserved communities bearing a disproportionate cancer burden.At the same time,the future of the scientific workforce is at risk.The declining NIH payline leaves many early-career and early-stage investigators unable to launch independent research careers,particularly in underfunded areas such as pediatric and rare cancers.Young investigators are the backbone of future scientific innovation,driving fresh ideas and bold discoveries,and the nation must do more to support them.To help address the current medical research funding crisis,AACR has launched its largest-ever grant program to provide$15 million in support of early-stage and mid-career cancer researchers,ensuring that talented scientists at critical points in their careers can continue advancing lifesaving discoveries.Despite serious challenges,the resilience of cancer researchers and the momentum of scientific discovery have never been stronger.Emerging technologies are opening new frontiers in how we understand,detect,and treat cancer.Artificial intelligence is poised to transform every aspect of cancer research and patient care,from drug discovery and diagnostics to clinical decision-making and survivorship care.Innovative tools such as liquid biopsies and multi-cancer early detection tests promise to identify cancer at earlier,more treatable stages.Exciting Message From AACRAACR Cancer Progress Report 20251advances in cancer vaccines,including those leveraging mRNA platforms,hold the potential to both prevent and treat intractable diseases,such as pancreatic cancer and kidney cancer.Meanwhile,next-generation molecularly targeted therapies,such as protein degraders,molecular glues and mutant-specific inhibitors,are expanding our ability to tackle some of the most elusive cancer targets.These and other breakthroughs on the horizon reaffirm that with sustained investment and scientific ingenuity,we can dramatically accelerate the pace of progress for the benefit of cancer patients.Realizing this promise will depend on scientific innovation and on our ability to foster collaborations across disciplines,sectors,and borders.Initiatives such as AACR Project GENIE and other pioneering efforts led by AACR and other organizations exemplify the power of global cooperation to drive scientific breakthroughs and ensure that patients around the world benefit from the latest advances.The AACR Cancer Progress Report 2025 is both a celebration of what cancer science and medicine have delivered and a Call to Action to safeguard its future.The evidence is clear:federal investments in cancer research yield extraordinary returns in lives saved,suffering reduced,and hope restored.To continue advancing toward a future where cancer is preventable,treatable,and curable for all,Congress must act to ensure robust,sustained,and predictable funding increases for the NIH,NCI,FDA,and CDC,and invest in the next generation of cancer researchers and physician-scientists.Together,with bold vision,bipartisan leadership,and unwavering commitment,we can build on 15 years of progress against cancer and continue transforming cancer care for our patients everywhere.Lillian L.Siu,MD,FAACR,FRCPCPresident,AACRMargaret Foti,PhD,MD(hc)Chief Executive Officer,AACRAACR Cancer Progress Report 20252 We are making remarkable strides against the collection of diseases known as cancer.Breakthroughs across basic,translational,clinical,and population sciencescombined with rapid technological innovationare transforming how we prevent,detect,diagnose,and treat cancer.As the first and largest professional organization in the world dedicated to preventing and curing all cancers,the American Association for Cancer Research(AACR)remains committed to enhancing public understanding of cancer and advocating for robust,sustained,and predictable investment in medical researchinvestment that is essential to saving lives.This includes steadfast support for the US federal agencies that fuel progress against cancer,particularly the National Institutes of Health(NIH),National Cancer Institute(NCI),Food and Drug Administration(FDA),and Centers for Disease Control and Prevention(CDC).However,the significant gains made in cancer science and medicine are under threat.Recent disruptions to the federal workforce,along with growing uncertainty about the future of medical research funding,pose a grave risk to the scientific enterprise.Any erosion of support for NIH would have a catastrophic impact on future discoveries and delay lifesaving advances for patients and families.It is therefore encouraging to see a bipartisan effort in the Senate not only to maintain current funding levels,but also to increase support for medical research and continue the federal investment in saving lives.Now in its 15th edition,the AACR Cancer Progress Report series to Congress and the American public is a major cornerstone of our educational and advocacy efforts.Over the past decade and a half,these reports have showcased the extraordinary momentum in cancer research and how sustained federal investment has led to improvements in treatment options,survival,and quality of life for cancer patients.This years report continues that traditionhighlighting how medical research can transform the lives of cancer patients,including those of the nine courageous individuals featured in this report.It also makes clear that continued progress hinges on unwavering support for NIH,NCI,FDA,and CDC and on a stable research infrastructure that can deliver advances for the benefit of all patients affected by this dreadful disease.Cancer in 2025Significant progress against cancer has led to a steady decline in cancer death rates and a consistent increase in the number of individuals living longer,fuller lives after a diagnosis.In the United States,the overall cancer death rate decreased by 34 percent between 1991 and 2023,a reduction that translates into averting more than 4.5 million deaths from cancer.This reduction in cancer mortality is largely attributable to national public health efforts,including prevention initiatives,screening programs,and advancements in treatment for certain cancers.The steady progress in reducing cancer mortality is largely driven by reduced smoking rates and subsequent decline in lung cancer deaths,a trend that has accelerated in recent years due to advances in treatment and early detection.Steady declines in death rates for colorectal cancer and female breast cancer since the 1990s have also played a key role in reducing the overall cancer mortality.More recently,advances in precision medicine have led to reductions in death rates for leukemia,melanoma,and kidney cancer,further contributing to overall progress.Thanks to the research-driven advances across the cancer care continuum,approximately 18.6 million individuals with a history of cancer were living in the United Executive SummaryAverage Annual Decline in US Cancer Death Rates(20112023)ESW1Per Year1.81%Per Year1.33%Per Year1.54%MENWOMENOVERALLAACR Cancer Progress Report 20253States as of January 1,2025,and this number is projected to exceed 22 million by 2035.While remarkable progress has been made,cancer remains a major public health challenge in the United States and globally.In the United States,more than two million new cases are projected for 2025 alone,underscoring the urgent need for continued investment in research,prevention,early detection,and equitable access to care.One of the key challenges we face is the uneven progress across cancer types.For example,the overall 5-year relative survival rate remains just 13 percent for pancreatic cancer and 6 percent for glioblastoma multiforme,an aggressive form of brain cancer,compared to 92 percent for breast cancer and 98 percent for prostate cancer.Additionally,the burden of cancer continues to disproportionately affect racial and ethnic minority groups and other medically underserved populations in the United States.These disparities are driven by a long history of structural inequities and systemic injustices and are driven by complex and interrelated structural and social factors.The burden of cancer and its economic toll,both on individuals and on US health care systems,are expected to rise in the coming decades,underscoring the urgent need for more research in medicine and public health to accelerate the pace of progress against cancer.The progress highlighted in this report was made as a direct result of the cumulative efforts of individuals working across the spectrum of medical research supported by federal funding.Importantly,public sector funding from NIH and NCI directly benefits patients through the development of lifesaving anticancer therapeutics and preventive interventions.Recent funding cuts and proposed budget reductions will stall progress,disrupt the research enterprise,and jeopardize future discoveries.Without strong federal support for medical research,the United States risks falling behind in scientific innovation and losing momentum in efforts to prevent,treat,and ultimately cure cancer.Continued federal investments in NIH,NCI,FDA,and CDC are essential to preserve US leadership in medical research,support the next generation of cancer researchers,and ensure that future breakthroughs translate into longer,healthier lives for everyone.Understanding the Path to Cancer DevelopmentDecades of basic research have established cancer as a collection of diseases characterized by the uncontrolled growth of cells.Cancer development is driven by disruptions of the molecular and cellular functions that regulate how cells grow,divide,and survive.Many of these changes are often just the first step in a complex,multistep process that is influenced by changes both inside and outside the cell.Research has shown that different cancer types share certain characteristics or hallmarks,including the ability of cancer cells to acquire changes that make their genome unstable,divide limitlessly,grow uncontrollably,escape cell death,spread to other tissues in the body,evade destruction by the immune system,and increase nutrients and oxygen supply to tumors.One of the hallmarks of cancer cells is alterations in the DNA sequence,also called genetic mutations.There are two types of genetic mutations associated with cancer:germline and somatic.Germline mutations are inherited and passed down from parents to children and contribute to about 10 percent of all cancer cases.The remaining 90 percent of all cancer cases stem from somatic mutations,which are acquired throughout a persons lifetime and can arise in multiple ways,such as due to errors made during cell division,or in response to environmental exposures,lifestyle factors,or because of chronic health conditions.In addition to DNA mutations,Hallmarks of Cancer Cells ESW2Spread to other parts of the bodyMultiply limitlesslyIncrease blood vessel formation toward tumorEvade the immune systemIncrease nutrient and oxygen supply to the tumorEscape cell deathGrow uncontrollablyAccumulate changes in the genetic materialAACR Cancer Progress Report 20254 other changes inside the cellincluding RNA variations,protein modifications,and epigenetic alterationsoften work together to drive cancer initiation and progression.Influences outside the cell also play a critical role in cancer development.These external factors include the circulatory and immune systems,the nervous system,and the microbiome.As the disease progresses,cancer cells acquire additional characteristics that allow them to shape and respond to their surroundings,creating the tumor microenvironment(TME).Research has shown that the TME affects the growth of cancer cells,and cancer cells influence the TME to promote their survival.Technological advances have made it possible to study cancer at the levels of single cells and molecules,revealing that each patients cancer is unique.This foundational insight is the basis for precision medicine,or personalized medicine,which is broadly defined as treating patients based on molecular characteristics that distinguish them from other individuals with the same disease.Rapid developments in precision medicine are yielding new and effective ways to make a diagnosis,plan treatment,and predict and monitor treatment response.Reducing the Risk of Cancer DevelopmentAdvances in basic,translational,and clinical research and population sciences have broadened our understanding of factors that contribute to an individuals risk of developing cancer.Approximately 40 percent of all cancers in the United States are attributable to modifiable causes.These risk factors include tobacco use,excess body weight,physical inactivity,excess exposure to ultraviolet(UV)radiation,alcohol consumption,environmental factors,and cancer-causing pathogenic infections.Between 1975 and 2020,approximately 5.94 million cancer deaths were averted due to public health interventions focused on tobacco control,national screening programs,and vaccination initiatives.While smoking rates have declined significantly,the rising prevalence of other risk factors,particularly obesity among children and adults as well as alcohol consumption,remains a significant public health concern.If left unaddressed,these trends can jeopardize the progress made against cancer over the last 50 years.In the United States,the prevalence of obesity among adults was 40.3 percent between 2021 and 2023.There is emerging evidence that weight loss interventions,such as bariatric surgery and glucagon-like peptide-1(GLP-1)medications(e.g.,Wegovy,Ozempic),reduce the risk of developing obesity-related cancers.When combined with a healthy diet and regular physical activity,these interventions can significantly promote weight loss in individuals with obesity,subsequently lowering the risk of cancers such as liver,colon,pancreatic,esophageal,and gallbladder.While addressing modifiable risk factors is crucial in reducing the risk of developing certain cancers,psychosocial and systemic factors cannot be overlooked.Psychosocial factors,such as stress,have been linked to increased cancer risk.Stress may directly contribute to cancer development by promoting Cigarette Use Among US Adults and Youth(20112024)ESW3PERCENT CIGARETTE USEAdults 18 yearsYouth 18 years2011201220132014201520162017201820192020202120222023202405101520AACR Cancer Progress Report 20255inflammation,disrupting hormone balance,and impairing immune function.It may also indirectly raise cancer risk by encouraging unhealthy behaviors such as smoking,excessive alcohol consumption,and poor dietary habits.Environmental risk factors,which are often unavoidable,are present in the air,water,and food,and can contribute to cancer development.The rising incidence of lung cancer among individuals who have never smoked is increasingly linked to exposure to carcinogens found in air pollution.Another class of environmental carcinogens that is of increasing concern is per-and poly-fluoroalkyl substances(PFAS),which are a group of endocrine-disrupting chemicals used in household products,food packaging,textiles,and flame retardants.PFAS persist in the environment,increasing long-term exposure to these harmful substances.Exposure to PFAS has been linked to an increased risk for cancers of the kidney,liver,and thyroid.In recent years,wildfires in the Western United States and Canada have grown more frequent and severe due to climate change.Individuals at greatest risk of exposures to the carcinogenic byproducts from wildfire suppression are those living near affected areas as well as firefighters or first responders involved in containment efforts.Exposure to these carcinogens increases the risk for multiple cancers including lung cancer and gliomas.As our understanding of cancer risk factors evolve,it is essential to invest in collaborative and translational research that builds upon existing knowledge and accelerates the pace of progress in cancer prevention and control.Additionally,lawmakers should prioritize developing policies that protect all segments of the US population,especially those unable to avoid risk factors due to their geographic location or occupation.Cancer Screening for Early DetectionCancer screening means checking for signs of cancer or for cells that might become cancerous in people who have no symptoms of the disease.The goal is to find cancer early,when it is most treatable and the chances of better outcomes are the highest.A growing body of evidence shows that screening not only saves lives,but it also helps reduce the overall burden of cancer across the population.In the United States,experts on the US Preventive Services Task Force(USPSTF)develop guidelines for individuals at an average risk of developing cancers of the breast,cervix,colon and rectum,and prostate,as well as for individuals at a higher risk of developing lung cancer.These recommendations are based on several factors,including a persons age,sex,family history,lifestyle,and environmental exposures.The USPSTF regularly reviews new scientific evidence and,if needed,revises these screening guidelines.Screening is a process that also involves follow-up care if something abnormal is detected.Unfortunately,many people,especially those in medically underserved communities,face barriers that prevent them from getting screened or receiving timely follow-up care.These barriers include lack of insurance,limited access to quality care,mistrust of the medical system,experiences of discrimination,and poor communication with providers.Researchers have identified proven strategies that are helping more people get screened,such as incorporating patient navigation,using electronic health records to send reminders,making it easier to access testing,and building trust through approaches that are tailored to the needs of different communities.Early detection of cancer is a very active area of research,with growing excitement around new technologies.During the 12 months covered by this report,FDA has approved several devices and software that use artificial intelligence(AI)to detect cancer earlier and more accurately.Another area of rapid progress is the development of minimally invasive screening approaches,with FDA approvals of a blood-based and a stool-based test for colorectal cancer screening,and of a self-collection device that enables at-home sample collection for cervical cancer screening.Innovations in imaging,as well as a better understanding of genetic alterations that increase a persons cancer risk,are also improving early detection of cancer.These advances hold promise,but experts emphasize the need for long-term studies to ensure these tools improve outcomes without causing harm or widening existing cancer disparities.Proportion of Total Cancer Deaths Averted Due to Screening and Removal of Precancerous Lesions(19752020)ESW4020406010080CervicalColorectalProstateBreast100yV%CR Cancer Progress Report 20256 Unifying Cancer Science and Medicine:A Continuum of Innovation for ImpactThe dedicated efforts of researchers across the spectrum of cancer science and medicine are driving breakthroughs in clinical care that are improving survival and enhancing quality of life for patients in the United States and worldwide.Clinical trials are a cornerstone of medical research,providing the evidence needed to determine whether new cancer treatments are safe and effective.Advances in our understanding of the genetic drivers of cancer have led to innovative approaches to clinical trial design and execution.For patients with cancer,participating in clinical studies can be life-changing,as reflected in the personal stories of Michelle Anderson-Benjamin(see p.118),and Bob Fortin(see p.114).It is essential that clinical trial participants reflect the diversity of the US population that may use and ultimately benefit from these treatments.Yet,participation in cancer clinical trials remains low,with significant sociodemographic disparities among those enrolled.Addressing these challenges requires collaboration among researchers,clinicians,and policymakers to remove barriers and promote equitable access to research opportunities.Surgery,radiotherapy,and chemotherapy are three of the five foundational pillars of cancer treatment.While effective,these therapies can have long-term side effects.Ongoing clinical studies are exploring whether less intensive use of these modalities can provide comparable outcomes for some patients while improving their quality of life.In parallel,more refined uses,such as molecularly targeted radiotherapeutics and optimized chemotherapeutic formulations,are being integrated into standard care.Fueled by research,innovative personalized cancer treatments are rapidly moving from the laboratory to the clinic,offering patients safer and more effective options than ever before.Between July 1,2024 and June 30,2025,FDA approved 20 new therapeutics for treating various cancer types,one new device for treating lung cancer,and expanded indications for eight previously approved therapies to include new cancer types.These advances include several groundbreaking immunotherapies.For example,afamitresgene autoleucel,a first-in-class adoptive cell therapy,known as T-cell receptor(TCR)T-cell therapy,is offering new hope to patients with synovial sarcoma,such as Quinn Johnsen(see p.138).A new therapeutic antibody,zolbetuximab-clzb(Vyloy),targeting a novel protein on stomach cancer cells is now benefiting individuals with gastroesophageal cancer,such as Greg Myers(see p.142).These approvals reflect the growing potential of immunotherapy to tackle rare and intractable cancers.The dramatic rise in the use of immune checkpoint inhibitorsimmunotherapies that help restore the immune systems ability to recognize and attack cancer cellsfurther underscores this potential.Since the first AACR Cancer Progress Report was released in 2011,the number of FDA-approved ICIs has grown from just one for melanoma to 15 therapies now used to treat more than 20 cancer types.Equally impressive are the innovative newly approved molecularly targeted therapies that are reshaping and improving cancer care.A new combination regimen comprising avutometinib and defactinib is now providing effective treatment for certain types of ovarian cancer,as seen in the experience of Mary Catherine Riley(see p.120).Patients with low-grade glioma,such as Alex Hepner(see p.126),are benefiting from vorasidenib(Voranigo),the first targeted therapy that inhibits mutated forms of the proteins IDH1 and IDH2 in brain tumors.For patients such as Dawn Varrati(see p.124),diagnosed with the aggressive bile duct cancer cholangiocarcinoma,the approval of zanidatamab-hrii(Ziihera),a new bispecific antibody,has opened new treatment possibilities.These cutting-edge therapies exemplify how deeper biological insights are translating into more precise and effective treatments for patients with difficult-to-treat cancers.While these developments mark significant progress against cancer,much work remains to ensure that all patient populations have access to the latest innovations in cancer treatment.Immunotherapy has advanced rapidly,offering patients powerful,personalized treatment optionsESW5115090920112025FDA-APPROVED IMMUNE CHECKPOINTINHIBITORSFDA-APPROVED ADOPTIVE T-CELL THERAPIESFDA-APPROVED BISPECIFIC T-CELL ENGAGERSAACR Cancer Progress Report 20257A Decade of Progress:Transformative Advances in Blood CancerBlood cancers,also known as hematologic malignancies,are a group of cancers that begin in blood-forming tissues of the body,such as the bone marrow.These cancers are broadly categorized as leukemia,lymphoma,and multiple myeloma,and pose a significant public health challenge in the United States and globally.In 2025,an estimated 192,070 new cases will be diagnosed,and 56,110 deaths will occur in the United States from the three most common blood cancer types(leukemia,lymphoma,and multiple myeloma);1,393,600 new cases and 745,000 deaths are estimated globally.Thanks to research-driven scientific breakthroughs and technological innovations,death rates from blood cancers have declined significantly,and many people diagnosed today are living longer and fuller lives.As a result,once considered among the most difficult cancers to treat,blood cancers are increasingly becoming manageable or even curable for many patients.As an example,mortality rates for non-Hodgkin lymphoma(NHL)the most common type of blood cancer in the United Stateshave declined by 47 percent between 1997 and 2023.As of January 1,2025,over 1.67 million survivors of blood cancers were living in the United States.Deeper understanding of molecular underpinnings of blood cancers,propelled by significant federal investments in discovery science,led to the development and approval of one of the first molecularly targeted therapeutics,imatinib(Gleevec),for the treatment of chronic myelogenous leukemia(CML).Imatinib not only transformed clinical care of patients with CML,but it also catalyzed a revolution in precision medicine that has significantly improved health outcomes for millions of patients with cancer.A remarkable example of progress in blood cancer precision medicine is the recent approval of revumenib,the first menin-targeted therapy,offering a new treatment option for patients with acute myeloid leukemia(AML),such as John C.(Jack)Moorman(see p.160).Progress against blood cancers has also paved the way for breakthroughs in other areas of medicine.Many treatments originally developed for leukemia or lymphoma are now being evaluated,and in some cases successfully used,for solid tumors.Some discoveries in blood cancers have even had applications in treating autoimmune diseases and rare genetic disorders.Despite rapid strides against blood cancers over the past decade,advances in precision medicine are not benefiting everyone equally.In the United States,certain populations,such as residents of rural areas and medically underserved populations,still face significant barriers to accessing the latest treatments.Similarly,in many low-and middle-income countries,people with blood cancers often go undiagnosed,untreated,or they receive care that is outdated due to lack of resources and infrastructure.Although challenges such as access to cutting-edge treatments remain,the pace of progress against blood cancers has never been more rapid.Researchers are innovating clinical trial design to inform clinical decisions,such as whether to monitor or intercept blood cancer and whether a patient would benefit from stopping the treatment.These innovations are leading the way to develop similar approaches for solid tumors.Continued federal investment in research and greater collaboration across all sectors are vital to ensure access to quality care and secure a brighter future for all patients with blood cancers.Supporting Cancer Patients and SurvivorsAccording to NCI,a person is considered a cancer survivor from the time of cancer diagnosis through the balance of their life.Unprecedented advances in cancer treatments over the past decade have led to more patients living longer and fuller lives after a cancer diagnosis.As of January 1,2025,more than 18.6 million adults and children with a history of cancer were living in the United States,which equates to about 5.5 percent of the population.This is a substantial improvement from 50 years ago,when cancer survivors constituted only 1.4 percent of the US population.The number of cancer survivors is projected to grow to over 22 million by 2035.Depending on the type and stage of cancer,as well as the age at which an individual is diagnosed,cancer survivorship can encompass a wide range of unique experiences.For example,some people may live cancer free for the remainder of life following successful treatment,while others may manage cancer as a chronic illness requiring ongoing treatment and monitoring,experience persistent side effects,or face a recurrence or a new cancer diagnosis.Understanding and addressing the physical,psychosocial,and financial challenges FDA-approved Treatments for Blood Cancer(20152025)New molecularly targeted therapies New immunotherapies2921ESW6AACR Cancer Progress Report 20258 faced by cancer survivors,supporting their quality of life,and ensuring that care is accessible and equitable are important priorities in cancer survivorship research.Research has shown that maintaining a healthy diet,staying physically active,limiting alcohol intake,and quitting smoking can help mitigate the physical challenges associated with a cancer diagnosis.Researchers are also using other evidence-based strategies,including palliative care,behavioral health interventions,patient-reported outcomes,and patient navigation,to help reduce the adverse impact of a cancer diagnosis on the physical and emotional health of cancer survivors.Understanding the challenges experienced by cancer survivors,as well as how to reduce or eliminate them,is an active and continually evolving area of research.Challenges experienced by patients and survivors of cancer also extend to friends and family members who are often informal caregivers.It is estimated that over four million caregivers are caring for an adult cancer patient in the United States.These caregivers support cancer survivors in various ways,such as arranging transportation for medical appointments,helping with day-to-day activities,coordinating care,and providing emotional support.While fulfilling the caregiving role,this can be physically,emotionally,and financially demanding.Caregivers are at an increased risk for psychological distress,and many face disruptions to their employment,education,or personal well-being.As the number of cancer survivors continues to grow,advancing survivorship and caregiver support through research,policy,and practice is essential to improving outcomes for all those affected by cancer.Envisioning the Future of Cancer Science and MedicineBreakthrough discoveries and technological advances across the fields of medicine have substantially increased our understanding of cancer initiation and progression.This foundational knowledge is driving better strategies to reduce the risk of developing cancer,detect cancer at the earliest possible stage,and treat cancer effectively and more preciselywith fewer long-term side effects.As a result,cancer deaths are declining,and cancer survivors are living longer and fuller lives.Technological and therapeutic advances against cancer and their impact on saving and improving lives of patients are a source of great optimism for cancer researchers,including AACR President,20252026,Lillian L.Siu,MD,FAACR,FRCPC(see p.196).Emerging technologies are driving progress in cancer research by enabling researchers to investigate tumors with greater precision and gain deeper biological insights.AI is transforming cancer research and patient care by enabling faster,more accurate,and personalized approaches across the cancer continuum.AI is now being applied to analyze histopathology images,identify genomic alterations,discover new drug candidates,improve early detection,streamline drug development,and support clinical decision-making.CRISPR gene editing tools are advancing cancer research and drug development by enabling disease modeling,high-ESW718.6 MILLIONSURVIVORS(5.5%OF THE POPULATION)3 MILLIONSURVIVORS(1.4%OF THE POPULATION)20251971“I think the field of cancer science and medicine is moving incredibly fast,and we must be at the cutting edge to ensure benefit for our patients.Artificial intelligence AI will help us learn from massive amounts of data to recognize patterns more efficiently.Liquid biopsy is a game changer,allowing us to detect cancer at a molecular level and intervene before it returns.And with more targeted,less toxic therapies,we are entering an era where even the most difficult cancers may become manageable.”Lillian L.Siu,MD,FAACR,FRCPC AACR President,20252026AACR Cancer Progress Report 20259throughput drug screening,and the development of new therapeutic strategies.CRISPR-based systems are being used to uncover which mutations drive tumor development,identify vulnerabilities in cancer cells,and design personalized treatment approaches.Another rapidly advancing technology is liquid biopsy,the analysis of blood or other body fluids for tumor-derived material routinely shed during cancer development and treatment.Liquid biopsy offers a minimally invasive alternative to the traditional tissue biopsy and is being integrated into efforts to enhance early detection,improve diagnosis,inform treatment selection,and monitor disease progression or recurrence.Emerging therapies,many of which are still the focus of ongoing research,are advancing the frontiers of precision oncology and are poised to transform the future of cancer care.One exciting area is the development of cancer vaccines that either prevent cancer in high-risk individuals or treat existing disease by training the immune system to recognize and attack tumor-specific targets.Preventive cancer vaccines that target the molecular characteristics of cancer hold great promise to reduce cancer risk,lessen the burden of ongoing surveillance,and reshape how we approach cancer prevention.Therapeutic cancer vaccines are designed to activate a patients immune system after cancer has developed,often by targeting unique proteins found on cancer cells.Another promising class of therapies is radiopharmaceuticals,including radioligand therapies(RLTs),which deliver cytotoxic radiation directly to cancer cells.RLTs combine a radioactive compound with a targeting agent that binds to specific proteins expressed on the surface of cancer cells,enabling the delivery of high-energy radiation to tumors while sparing surrounding healthy tissues and offering a highly localized,tumor-specific radiotherapy approach.Advancing Cancer Science and Medicine Through Evidence-based PoliciesFederal government policies play a critical role in advancing cancer research,developing safe and effective new treatments,improving patient outcomes,and supporting the next generation of researchers and physician-scientists.Sustained investments over decades in vitally important agencies such as NIH,NCI,FDA,and CDC have led to significant progress across the cancer care continuumfrom improvements in prevention and early detection to breakthroughs in treatment and survivorship.These agencies support the discovery of lifesaving therapies,strengthen the clinical trial infrastructure,train the scientific workforce,and expand access to cancer screening and care.Federal funding of medical research also spurs our nations economy.Every$1 in NIH funding returns$2.56 in economic activity.The proposed$17.97 billion reduction to NIH funding in the administrations FY 2026 budget would severely undermine this progress and jeopardize the nations leadership in medical research.Such drastic reductions would stall scientific discovery,limit access to promising new therapies for patients such as Richard Schlueter(see p.212),and discourage the next generation of researchers at a critical time.Therefore,AACR was thrilled that the Senate Appropriations Committee voted in a bipartisan manner on July 31,2025,to advance the FY 2026 Labor,Health and Human Services,and Education(Labor-HHS-Ed)Appropriations Subcommittee bill and provide$47.2 billion for NIH and$7.374 billion for NCI,reflecting funding increases in these agencies of$400 million and$150 million,respectively.According to a recent poll,77 percent of Americans oppose reducing federal funding for medical research.By rejecting the administrations proposed cuts to NIH,the US Senate aligned with the publics priorities,recognizing the vital role of NIH in accelerating progress against cancer and countless other diseases affecting millions of Americans.These bipartisan actions are essential to protecting our nations scientific enterprise,preserving public trust in science,and sustaining the lifesaving research on which patients and families depend.AACR Call to ActionFor more than 50 years,bipartisan investment in medical research has driven historic progress against cancer,delivering earlier detection,better treatments,and more time for millions of patients and families.That progress is now in jeopardy.Prolonged funding uncertainty and administrative and political interference are weakening cancer research,undermining scientific integrity,and eroding the infrastructure that turns discovery into patient care.Promising signs of bipartisan resolve in Congress show that this damage can be stopped and progress restored.The fight against cancer is at a decisive moment,and lawmakers have the power to ensure that promising science moves forward,discoveries reach patients,and hope for a future without cancer is not lost.AACR urges Congress to take immediate action to:Restart clinical trials and restore canceled research grants to ensure that patients are not turned away from lifesaving studies and that promising science is not lost at a critical stage.AACR Cancer Progress Report 202510 Support the federal research infrastructure to repair the damage caused by mass reductions in workforce,frozen contracts,and suspended peer review.Discovery has stalled,and scientific capacity is breaking down.Protect public health programs that prevent cancer to avoid losing ground on screening,HPV vaccination,tobacco cessation,and early interventions.These efforts save lives.Ensure that new treatments reach patients without delay to prevent promising therapies from being trapped in bureaucratic limbo while families wait for help that may come too late.Foster early-career and early-stage scientists and stabilize research careers to stop the exodus of postdoctoral researchers and junior investigators who are abandoning science or being recruited overseas.When they leave,they take future cures with them.Defend the independence and integrity of science by reversing the August executive order that politicizes federal grantmaking,thus restoring safeguards that keep research free from political interference and ensuring that grantmaking,peer review,and public policies are guided by scientific evidence rather than ideology.Reassert Americas global leadership in medical innovation to preserve decades of progress and prevent other nations from overtaking the United States in the race for the next generation of cures.Provide no less than$51.303 billion for NIH and$7.934 billion for NCI in fiscal year 2026 to sustain the scientific workforce,power new breakthroughs against cancer and other human diseases,and uphold a national commitment to the patients and families who are relying on lifesaving progress.Cancer touches every family,every community,and every generation.At this defining moment,Congress owes it to every patient,every survivor,and every family to protect the progress we have made and deliver on the promise of a future without cancer.AACR Cancer Progress Report 202511Research continues to advance immunotherapy,leading to:The first T-cell receptor(TCR)T-cell therapy that is benefiting patients with synovial sarcoma,such as Quinn Johnsen(see p.138).A new therapeutic antibody targeting a novel protein on stomach cancer cells that is benefiting individuals with gastroesophageal cancer,such as Greg Myers(see p.142).The first approval of an immune checkpoint inhibitor for the treatment of patients with anal cancer.Research continues to power precision medicine,leading to:A new combination regimen that is providing new hope to patients with ovarian cancer,such as Mary Catherine Riley(see p.120).The first approval of an IDH-targeted therapy for brain tumors that is bringing new hope to young adult patients,such as Alex Hepner(see p.126),who are commonly affected by this mutation.A new antibodydrug conjugate that is benefiting patients with breast cancer,such as Michelle Anderson-Benjamin(see p.118),as well as those with lung cancer.Modifiable risk factors such as:Between July 1,2024,and June 30,2025,the US Food and Drug Administration(FDA)approved:New anticancer therapeutics,which are now benefiting patients with various types of cancerPreviously approved anticancer therapeutics for treating new types of cancerNew devices,one for treating lung cancer and one for cervical cancer screeningMinimally invasive tests for colorectal cancer screeningSeveral artificial intelligence(AI)based tools to improve early detection and diagnosis of cancersSnapshot of a Year of Progress19912023Reduction In Overall Cancer Death Rate34%4.5 MILLION LIVES SAVED20822AIOf all cancer cases in the USARE ATTRIBUTABLE TO:Of all cancer cases and deaths globally Cigarette Smoking Excess Body Weight Alcohol Consumption UV Radiation Exposure Poor Diet Pathogenic Infections Physical Inactivity40ACR Cancer Progress Report 202512 In just the 12 months covered in this report,FDA approved:The first menin-targeted therapy for patients with acute myeloid leukemia(AML)such as John C.(Jack)Moorman(see p.160),with cancers harboring specific genetic alterations.A novel cytotoxic fusion protein for the treatment of cutaneous T-cell lymphoma,a rare form of non-Hodgkin lymphoma.A new CAR T-cell therapy for patients with acute lymphocytic leukemia(ALL),expanding options for this aggressive cancer.Over the past decade,FDA has approved numerous innovative therapies for the treatment of blood cancers,including:New molecularly targeted therapies,including 4 antibodydrug conjugates(ADCs),one of the most exciting and rapidly advancing classes of agents driving precision medicineNew immunotherapeutics,including 7 CAR T-cell therapies and 8 bispecific T-cell engagers(BiTEs),two of the most promising frontiers in cancer immunotherapyMortality Reduction in the United States for Selected Blood Cancers(19912023)AACR urges Congress to take immediate action to:RESTART clinical trials and restore canceled research grants SUPPORT the federal research infrastructure PROTECT public health programs that prevent cancer ENSURE that new treatments reach patients without delay FOSTER early-career and early-stage scientists and stabilize research careers DEFEND the independence and integrity of science REASSERT Americas global leadership in medical innovation PROVIDE no less than$51.303 billion for NIH and$7.934 billion for NCI in fiscal year 2026 Chronic myeloid leukemia 71C1%Non-Hodgkin lymphomaMultiple myeloma 2921AACR Cancer Progress Report 202513IN THIS SECTION,YOU WILL LEARN:Research:Driving Progress Against CancerResearch is the cornerstone of progress against the collection of diseases we call cancer.It drives every basic science discovery,accelerates every clinical breakthrough,and informs public policies designed to improve health outcomes,ultimately leading to better survival and quality of life.Advances across basic,clinical,translational,and population sciences,along with technological innovations such as artificial intelligence and machine learning(see Envisioning the Future of Cancer Science and Medicine,p.194),are fueling new strategies for cancer prevention,early detection,diagnosis,treatment,and survivorship care.Every scientific innovation and effective policy that drives progress against cancer represents the culmination of a complex process shaped by years of coordinated effort and multidisciplinary collaboration among diverse stakeholders(see Sidebar 1,p.15).Remarkable progress against cancerin particular,improvements in early detection,diagnosis,treatment,and risk reductionhas led to a steady decline in US cancer death rates over the past several decades.Between 1991 and 2023,the age-adjusted overall cancer mortality rate declined by 34 percent,a reduction that translates into averting more than 4.5 million deaths from cancer(2).The steady progress in reducing cancer mortality is largely attributable to reduced smoking rates and subsequent declines in lung cancer mortality,declines that have accelerated in recent years due to advances in treatment and early detection(see Matching Therapies to the Molecular Drivers of Lung Cancer,p.110)(2).Declines in death rates for colorectal cancer and female breast cancer have also contributed to the progress in reducing overall Cancer in 2025 In the United States(US),the overall cancer death rate has been steadily declining since the 1990s,translating into more than 4.5 million cancer deaths avoided between 1991 and 2023.The decline in overall US cancer death rate is attributable to reduction in smoking rates,as well as improvements in treatment and early detection of certain cancers.Approximately 18.6 million cancer survivors were living in the US as of January 1,2025.Progress has not been equal against all cancer types or all stages of a given cancer type.Many segments of the US population experience stark inequities in the cancer burden;these inequities are largely driven by structural and social factors.The economic burden of cancer on individuals and the US health care systems is expected to rise in the coming decades,highlighting the urgent need for more research and increased federal support for medical science and public health.It is imperative that all stakeholders work together to implement evidence-based interventions,including public policies that guarantee equitable access to quality health care for all patients.AACR Cancer Progress Report 202514 US cancer mortality(2).According to a recent analysis,US breast cancer mortality declined by 44 percent,averting more than 517,000 deaths between 1989 and 2022,owing to advances in screening mammography and personalized treatments(see Cancer Screening for Early Detection,p.71)(2,3).Similarly,the death rate for colorectal cancer declined by 49 percent between 1990 and 2023(4).However,this progress has been limited to older adults,as colorectal cancer death rates among The Medical Research Community:Driving Progress TogetherProgress against cancer can be accelerated when all stakeholders who are dedicated to fundamentally changing the burden of cancer work together.Further increasing collaborations will amplify future breakthroughs.The key stakeholders in medical research include:Adapted from(1).SIDEBAR 1Biopharmaceutical,diagnostic,and medical device company research teamsIndividuals diagnosed with cancer,their caregivers,family members,friends,and colleaguesHealth care systems and clinical teamsAcademic and government researchers from a diverse array of specialtiesCommunity partners,suchas religious organizations,local health clinics,food banks,and travel servicesIndividual community scientists,patient navigators,patient advocates,other cancer advocates,and members of advocacy groupsHealth insurance companiesFederal funding organizationsPolicymakers and regulatorsPhilanthropic organizations,including nonprofit sponsors and individual donors,cancer-focused professional organizations,and cancer-focused foundationsSavingLivesTogetherCancer in 2025AACR Cancer Progress Report 202515those diagnosed before age 50 increased by approximately 14 percent between 1990 and 2023(4).Breakthroughs across the spectrum of cancer science and medicine have led to unprecedented progress against aggressive cancers once considered intractable.Research-driven advances in treatment have resulted in a steady decline in death rates for leukemia,melanoma,and kidney cancer,despite increasing incidence of these cancers(2).For example,groundbreaking basic research in the 1960s through 1980s that identified the mechanistic underpinnings of chronic myeloid leukemia(CML),a cancer of the blood and bone marrow,propelled the development of a cascade of new treatments for CML that have drastically improved outcomes for these patients(see Leukemias,p.145)(5).As a result,the 5-year relative survival rate for CML has more than tripled,from 22 percent in the mid-1970s to 70 percent among individuals diagnosed between 2015 and 2021(4,6).More recently,advances in molecularly targeted therapies and immunotherapies have transformed clinical care for several previously difficult-to-treat cancers,including metastatic melanoma(see Figure 1,p.16),lung cancer(see Matching Average Annual Decline in US Death Rates for Selected Cancers(20112023)SITE PERCENT CHANGE Lung3.9%Colorectal1.5male Breast 1.2%Prostate0.7%Source:(4).W1Increasing Innovative Treatment Options for Advanced MelanomaSince 2011,the FDA approved numerous immunotherapies and molecularly targeted therapies for advanced melanoma,reflecting major breakthroughs in cancer research.These advances have revolutionized the therapeutic landscape for advanced melanoma,leading to dramatic improvements in survival and fewer melanoma-related deaths.This timeline focuses on systemic treatments for metastatic melanoma of the skin;other therapeutics have been approved for the treatment of localized lesions or for other rarer forms of melanomas.For example,in January 2022,FDA approved tebentafusp(Kimmtrak)for the treatment of certain patients with uveal melanoma,an aggressive cancer of the eye.Data from:NCI Surveillance,Epidemiology,and End Results Program.trametinibdabrafenibtrametinib dabrafenibpembrolizumabnivolumabencorafenib binimetinib ipilimumabvemurafenib ipilimumab nivolumab cobimetinib vemurafenibatezolizumab cobimetinib vemurafenibrelatlimab-rmbw nivolumab ImmunotherapyMolecularly Targeted Therapylifileucel17 11201320142011Most Recent Data20152018202020222024356%INCREASE IN 5-YEAR RELATIVE SURVIVAL*2.7%1.97CREASE IN MORTALITY*5-year survival estimates based on cases diagnosed from 2005 to 2011 versus 2015 to 2021.Death rate per 100,000 people.FIGURE 1Cancer in 2025AACR Cancer Progress Report 202516 Therapies to the Molecular Drivers of Lung Cancer,p.110),kidney cancer,and certain types of breast cancer.For example,the immunotherapy pembrolizumab(Keytruda)was recently shown to be the first postsurgical treatment to extend survival in patients with early-stage kidney cancer(7).Among the major advances made across the clinical cancer care continuum from July 1,2024,to June 30,2025,are 20 new anticancer therapeutics that were approved for use by the US Food and Drug Administration(FDA).During this period,FDA also approved new uses for eight previously approved anticancer therapeutics and one cancer treatment device;two new early detection screening tests,including the first liquid biopsy test and a next-generation multitarget stool DNA test for colorectal cancer screening;a device for at-home sample collection for cervical cancer screening;and a wearable device that uses low-intensity electrical fields to slow the growth of lung cancer cells.FDA also approved a number of artificial intelligence-powered devices and software for aiding cancer risk prediction,diagnosis,and early detection.Collectively,advances such as theseas well as those highlighted in past 14 editions of this annual reportare helping more children and adults live longer,fuller lives following a cancer diagnosis,with fewer treatment-related side effects.The 5-year relative survival rate for all cancers combined has increased from 49 percent for those diagnosed between 1975 and 1977 to 70 percent among those diagnosed between 2015 and 2021(2,4).Similar progress has been made among US children(ages 0 to 14)and adolescents(ages 15 to 19),diagnosed with cancer whose 5-year relative survival rates increased from 58 percent to 85 percent in children and from 68 percent to 87 percent in adolescents over the same time period(see Figure 2,p.17)(2,8).In 2011,when the inaugural AACR Cancer Progress Report was released,there were an estimated 12 million cancer survivors living in the United States.As of January 1,2025,the number of Progress Against Cancer in Children and AdolescentsFive-year relative survival rates for the US children and adolescents(ages 0 to 19)who were diagnosed with cancer from 2014 to 2020 were substantially higher compared to those diagnosed from 1975 to 1979.Childhood cancers are classified using the International Classification of Childhood Cancer(ICCC).Improvement in the 5-year relative survival rate was observed for all cancers combined as well as for several individual cancer types.FIVE-YEAR SURVIVAL RATES(%)FOR CERTAIN CHILDHOOD AND ADOLESCENT(019 YRS)CANCERS204006080100Thyroid carcinomaRhabdomyosarcomaRetinoblastomaNon-Hodgkin lymphomaNeuroblastoma and ganglioneuroblastomaMelanomaLeukemiaHodgkin lymphomaHepatic tumorsBrain and central nervoussystem neoplasmsBone tumorsAll ICCC sites combined1975197920142020Year of DiagnosisFIGURE 2Cancer in 2025AACR Cancer Progress Report 202517people living with a history of cancer had grown to 18.6 million,representing approximately 5.5 percent of the US population(9).This reflects a nearly four-fold increase compared to 50 years ago,when cancer survivors made up only 1.4 percent of the US population.The number of cancer survivors is projected to grow to over 22 million by 2035(9).Thanks to remarkable advances in cancer research,cancer survivors are now living years,even decades,beyond their initial diagnosis.As of 2025,nearly 50 percent of US cancer survivors have lived 10 years or more since their cancer diagnosis,and 22 percent have lived 20 years or more(9).Moreover,nearly 80 percent of US cancer survivors are age 60 or older,and this proportion is expected to increase with continued advances in treatment and the aging of the population(9).As the population of cancer survivors grows,prioritizing research that addresses their evolving needs must remain a critical focus for US medicine and public health(see Supporting Cancer Patients and Survivors,p.177).Cancer:An Ongoing ChallengeWhile substantial progress has been made against cancer,the disease continues to be an enormous public health challenge in the United States and around the world.In 2025,an estimated 2,041,910 new cancer cases will be diagnosed and 618,120 people will die from the disease in the United States(see Table 1,p.18).In addition,many population groups in the United States experience disproportionately high rates of cancer incidence and mortality.Estimated Burden of Common Types of Cancer in the United States in 2025New CasesDeathsALL CANCERS COMBINED2,041,910618,120Breast 319,75042,680Prostate313,78035,770Lung and bronchus226,650124,730Colorectal154,27052,900Melanoma of the skin104,9608,430Bladder84,87017,420Kidney and renal pelvis80,98014,510Non-Hodgkin lymphoma80,35019,390Uterine67,88013,250Pancreatic67,44051,980Thyroid44,0202,290Liver and intrahepatic bile duct42,24030,090Myeloma 36,11012,030Sources:(2,4).TABLE 1The COVID pandemic caused a marked disruption in cancer diagnoses,disproportionately affecting racial and ethnic minorities and other medically underserved populations,exacerbating existing disparities in timely cancer detection.Source:(11).W32020201920192020Early-stage diagnosisUnder-diagnosesLate-stage diagnosisSources:(9,10).W210.4 MILLION6.2 MILLION20402025Number of cancer survivors living 15 years after diagnosisCancer in 2025AACR Cancer Progress Report 202518 Inequities in the Burden of Cancer in the United StatesDespite overall progress against cancer,significant disparities persist among certain population groups in the United States,across the cancer continuum,linked to structural and socioeconomic disadvantages.Because of a long history of structural inequities and systemic injustices,certain segments of the US population continue to shoulder a disproportionate burden of adverse health conditions,including cancer.These unequal burdens have led researchers and public health experts to closely study cancer disparities.Cancer disparities are one of the most pressing public health challenges in the United States.The National Cancer Institute(NCI)defines cancer disparities as adverse differences in cancer-related measures,such as number of new cases,number of deaths,cancer-related health complications,survivorship and quality of life after cancer treatment,screening rates,and cancer stage at diagnosis,that exist among certain population groups(see Sidebar 2,p.19).The AACR Cancer Disparities Progress Report 2024(12)highlights the disproportionate burden of cancer among US racial and ethnic minority groups and other medically underserved populations(see Sidebar 3,p.20).As one example,while non-Hispanic(NH)White individuals have the highest overall cancer incidence rate,NH Black individuals had the highest overall cancer mortality rate,followed by American Indian/Alaska Natives(AI/AN).Further,between 2015 and 2021,patients with cancer from all racial and ethnic minority groups had lower 5year relative survival rates(61.7 to 68.4 percent)than NH White individuals(70.8 percent)(4).US Population Groups That Experience Cancer DisparitiesAccording to the National Cancer Institute,cancer disparities are adverse differences in cancer-related measures,such as number of new cases and deaths,cancer-related health complications,quality of life after cancer treatment,financial burden,screening rates,and stage at diagnosis that are shouldered by certain population groups including:Individuals belonging to certain ancestry,racial or ethnic minority populationsIndividuals of low socioeconomic status,including low educational attainmentIndividuals who lack or have inadequate health insurance coverageIndividuals belonging to sexual and/or gender minority communitiesIndividuals with disabilitiesAdolescents and young adultsIndividuals who are incarceratedImmigrants,refugees,or asylum seekersOlder adultsCitizens of American Indian/Alaska Native Tribal NationsResidents in certain geographic locations,including rural areas,or of certain types of neighborhoods,such as those with low access to resourcesSIDEBAR 2PASSPORTPASSPORTCancer in 2025AACR Cancer Progress Report 202519Research on the science of cancer disparities is revealing significant differences in incidence and mortality across subpopulations within racial and ethnic groups,underscoring the need to disaggregate data within these populations.Striking disparities in cancer burden have been identified within Asian subpopulations and between Native Hawaiian or Other Pacific Islander(NHOPI)and Asian individuals(12).As an example,the risk of death from pancreatic ductal adenocarcinoma was 10 percent and 22 percent higher among East Asians and Pacific Islanders,respectively,compared to South Asians(20).Notably,the US Asian population has ancestry in numerous countries of origin,and the NHOPI population comprises diverse subgroups with distinct variations in historical backgrounds,languages,and cultural traditions.Still,Asian and NHOPI populations continue to be grouped together in cancer epidemiologic data.Cancer disparities are caused by many overlapping factors at the individual,community,and societal levels.These include aspects like biology,personal behaviors,access to healthy environments,cultural influences,and differences in quality of health care systems,factors that can affect people throughout their lives(22).To help researchers understand and address these influences,the National Institute on Minority Health and Health Disparities developed a framework to guide the study of contributors Cancer Inequities in the United StatesMany segments of the US population shoulder a disproportionate burden of cancer.Selected examples of disparate cancer incidence and outcomes from recent studies are provided here.Disparities in other aspects of cancer care are highlighted in relevant sections throughout the report.An in-depth discussion of cancer disparities and recent progress in addressing these inequities,as well as a call to action,is detailed in AACR Cancer Disparities Progress Report 2024(12).16%and 10%higher,9%lowerBetween 2018 and 2022,cancer death rates were 16 percent higher among Black men compared to White men.Black women experienced 10 percent higher cancer mortality compared to White women,despite having a 9 percent lower incidence rate(13).28%increased riskBlack pediatric cancer patients have a 28 percent increased risk of death compared to White pediatric cancer patients(14).1.10-,1.34-,and 1.62-fold higherAmong patients diagnosed with non-Hodgkin lymphoma,the risk of death was 1.10-,1.34-,and 1.62-fold higher in East Asian,Southeast Asian,and Native Hawaiian or Other Pacific Islander patients,respectively,compared to non-Hispanic White patients(15).19%vs.32%From 2014 to 2018,5-year survival for stomach cancer was 19 percent and 32 percent among American Indian/Alaska Native and non-Hispanic White individuals,respectively(16).2-fold higherLesbians have a nearly 2-fold higher incidence rate of thyroid cancer,basal cell carcinoma,and non-Hodgkin lymphoma compared to straight women(17).2X belowIn the United States,geographic areas with higher proportions of families living 2 times below the federal poverty level have a higher burden of cervical cancer(18).5.6%and 12.8%higherFrom 2017 to 2021,residents of the Appalachian region of the United States had 5.6 percent and 12.8 percent higher incidence and mortality rates,respectively,from all cancers combined than non-Appalachian residents(19).SIDEBAR 3Cancer in 2025AACR Cancer Progress Report 202520 to adverse health outcomes and health disparities,including those related to cancer(22).This framework emphasizes the importance of addressing both biological drivers and social drivers of health(SDOH)simultaneously,rather than focusing on any one driver in isolation.NCI defines SDOH,sometimes also called social determinants of health,as the social,economic,and physical conditions in the places where people are born and where they live,learn,work,play,and get older that can affect their health,well-being,and quality of life.SDOH include factors such as socioeconomic status;housing;transportation;and access to healthy food,clean air and water,and health care services(see Figure 3,p.22).It is important to recognize that patients with intersectional identitiessuch as individuals from racial and ethnic minority populations living in disadvantaged rural or urban areasoften face multilevel barriers to cancer care that negatively impact screening,diagnosis,treatment,and survivorship.As an example,Black women living in more socially disadvantaged neighborhoods had significantly worse breast cancer survival compared to those living in the least disadvantaged neighborhoods,even after accounting for clinical and individual socioeconomic factors(23).Neighborhood disadvantage is measured using a weighted composite score based on neighborhood-level characteristics,including the proportion of Black residents,individuals living below the federal poverty level,those receiving public assistance,living in female-headed households,unemployed,or under 18 years of age.Higher scores reflect greater levels of disadvantage.Inadequate access to quality health care is a major social driver of cancer disparities.A recent review of disparities across the lung cancer continuum revealed that individuals who were younger and not eligible for Medicare had lower screening rates;uninsured patients or those with Medicaid were less likely to receive curative surgery;and individuals with Medicaid or no insurance were less likely to utilize novel therapies and enroll in clinical trials(24).Similar patterns have been observed in breast cancer care,in which women with private insurance were significantly more likely to receive molecularly targeted therapy or immunotherapy,treatments associated with better outcomes(25,26).Cancer disparities affect multiple segments of the US population,making disparities a significant public health concern that requires tailored efforts to better understand and address these inequities(see Addressing Cancer Disparities and Improving Patient Outcomes,p.224).Health equity for all populations can only be achieved through new insights gained from innovative,inclusive,and translational science.This includes basic research using biospecimens from diverse populations;clinical studies with participants from all sociodemographic backgrounds;and health care delivery and implementation research that reflects every community.Variable Progress Against Different Types of Cancer and Stages of DiagnosisA significant challenge in cancer science and medicine is the uneven progress against different cancer types and different stages of a given cancer type.This challenge is reflected in the wide variation in 5-year relative survival rates among US cancer patients,depending on both the type of cancer diagnosed and the stage at diagnosis(4).For example,the overall 5-year relative survival rate is just 13 percent for pancreatic cancer and 6 percent for glioblastoma multiforme(GBM),an aggressive form of brain cancer,in stark contrast with 92 percent for breast cancer and 98 percent for prostate cancer(4).However,when examining stage-specific 5-year survival rates for breast and prostate cancers,striking differences emerge.Individuals diagnosed with localized diseasecancer that is confined to the breast or prostate tissueshave a 5-year relative survival rate of 100 percent,while individuals diagnosed with metastatic diseasedisease that has spread to other organshave a 33 and 38 percent 5-year relative survival rate,respectively.Between 2015 and 2019,cervical cancer incidence and mortality were 25%and 42%higher,respectively,in rural counties compared to urban counties.Source:(21).W4Breast Cancer Death Rate in the United States(20142022)Source:(4).W5AVERAGE1.2creaseevery year Cancer in 2025AACR Cancer Progress Report 202521Why Do US Cancer Disparities Exist?Complex and interrelated structural and social factors,stemming from a long history of racism and discrimination,drive cancer disparities.These factors include social drivers of health(SDOH)as well as biological factors,mental health,and modifiable risk factors.The National Cancer Institute defines SDOH,sometimes also called social determinants of health,as the social,economic,and physical conditions in the places where people are born and where they live,learn,work,play,and get older that can affect their health,well-being,and quality of life.SDOH have a major influence on peoples physical and mental health,well-being,and quality of life.In the United States,historical racism and contemporary injustices have perpetuated and exacerbated systemic inequities,resulting in adverse differences in SDOH for racial and ethnic minorities and medically underserved populations.The circle in the figure depicts key drivers of health and how they interconnect both at societal and community levels and at the individual level.Selected examples of the multilevel factors that make up drivers of health are highlighted.Collectively,these factors impact every stage of the cancer continuum,leading to worse health outcomes for racial and ethnic minorities and other underserved populations(shown at the bottom).DEVELOPMENTRISK REDUCTIONEARLY DETECTIONTREATMENTSURVIVORSHIPCANCER RESEARCH AND CARE TRAINEES AND WORKFORCEEDUCATORS CANCER RESEARCHERS PHYSICIANS PHYSICIAN-SCIENTISTS HEALTH CARE PROFESSIONALSCANCER CARE CONTINUUMRacism Discrimination SegregationStructural Inequities Societal InjusticesDisparities InAdverse Health OutcomesDriversof HealthBEHAVIORALDiet,Tobacco use,Excess body weight,Physical inactivityENVIRONMENTALAir/water quality,Community safetyHousing,Transportation,CLINICALHealth care qualityHealth care access,PSYCHOSOCIAL IsolationStress,Mental health,BIOLOGICALGenetic,Epigenetic,Tumor microenvironmentSOCIOECONOMICEducation,Income,EmploymentCULTURALHealth beliefs,Health-related attitudesINDIVIDUAL INTERPERSONAL COMMUNITY SOCIETALINDIVIDUAL INTERPERSONAL COMMUNITY SOCIETALFIGURE 3Cancer in 2025AACR Cancer Progress Report 202522 Variable progress against different cancer types can be explained in part by disparities in lifesaving therapeutic options that are available for different cancer types.For example,since the release of the first edition of this report in 2011,FDA has approved 20 new molecularly targeted therapeutics,excluding endocrine therapies,and two immunotherapeutics for treating patients with breast cancer.As a result,patients have a deeper selection of therapeutics for the treatment of their disease and breast cancer mortality has been declining steadily.In contrast,treatment options for patients with GBM remain extremely limited.Since the approval of the chemotherapeutic temozolomide nearly 25 years ago,no new anticancer agents have shown promise in improving overall survival.This in part explains why the 5-year relative survival rate for patients with GBM remains at a dismal 6 percent(4).Developing new and effective tests for the early detection of more cancer types(see Cancer Screening for Early Detection,p.71),in addition to expanding the eligibility for established screening recommendations(27),could also help address the challenges of variable progress between different types of cancer,since the likelihood of a better outcome is much higher when cancer is diagnosed at an early stage,when it is confined to its original location and not spread to distant sites(see Cancer Screening for Early Detection,p.71 and Figure 12,p.72).Additionally,improving and expanding therapeutic options for difficult-to-treat cancers will require intensive research that builds on existing knowledge to uncover the biological drivers of these diseases and inform the development of more effective treatments.The Growing Population Burden of CancerThe public health challenge posed by cancer is expected to increase even further in the coming decades unless more effective strategies for prevention,early detection,and treatment are implemented.In the United States,the number of new cancer cases(including non-melanoma skin cancer)diagnosed annually is projected to exceed 3.5 million by 2050a nearly 40 percent increase from 2025 estimates(28).A major driver of this rise is the growing proportion of older adults in the population,as cancer risk increases significantly with increasing age.In fact,in 2022,59 percent of all cancer cases occurred in individuals age 65 and older.By 2050,this age group is projected to exceed 82 million people,representing about 23 percent of the total US population.That is a 50 percent increase from 2022,when there were about 58 million older adults,making up roughly 17 percent of the population(29).Notably,cancer cases in the age 65 and older population have remained steady over time,from 61 percent in 1995 to 59 percent in 2021,which could result from the decline in smoking-related cancers in this age group(2).Also contributing to the projected increase in the number of US cancer cases is the high prevalence of excess body weight,physical inactivity,alcohol consumption,environmental factors,and continued tobacco use(see Reducing the Risk of Cancer Development,p.51)(31,32).For example,although smoking rates have declined precipitously since the 1970s,11 percent of adults still smoke.However,lung cancer is on the rise in non-smokers(33)and makes up about 16 percent and 10 perce
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Global health emergency corps frameworkGlobal health emergency corps frameworkGlobal health emergency corps frameworkISBN 978-92-4-010944-5(electronic version)ISBN 978-92-4-010945-2(print version)World Health Organization 2025Some rights reserved.This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0 IGO licence(CC BY-NC-SA 3.0 IGO;https:/creativecommons.org/licenses/by-nc-sa/3.0/igo).Under the terms of this licence,you may copy,redistribute and adapt the work for non-commercial purposes,provided the work is appropriately cited,as indicated below.In any use of this work,there should be no suggestion that WHO endorses any specific organization,products or services.The use of the WHO logo is not permitted.If you adapt the work,then you must license your work under the same or equivalent Creative Commons licence.If you create a translation of this work,you should add the following disclaimer along with the suggested citation:“This translation was not 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whether permission is needed for that reuse and to obtain permission from the copyright holder.The risk of claims resulting from infringement ofany third-party-owned component in the work rests solely with the user.General disclaimers.The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of WHO concerning the legal status of any country,territory,city or area or of its authorities,or concerning the delimitation of its frontiers or boundaries.Dotted and dashed lines on maps represent approximate border lines for which there may notyet be full agreement.The mention of specific companies or of certain manufacturers products does not imply that they are endorsed or recommended by WHO in preference to others of a similar nature that are not mentioned.Errors and omissions excepted,the names of proprietary products are distinguished by initial capital letters.All reasonable precautions have been taken by WHO to verify the information contained in this publication.However,the published material is being distributed without warranty of any kind,either expressed or implied.The responsibility for the interpretation and use of the material lies with the reader.In no event shall WHO be liable for damages arising from its use.iiiForeword ivAcknowledgements vAbbreviations viExecutive summary vii1.Introduction 12.Background 4Summary of relevant post-COVID-19 recommendations 4Actions in response to the post-COVID-19 recommendations 63.Purpose of this document 94.Design process and approach 105.Vision 116.Objectives 127.Guiding principles and values 148.Strategy 15Globally and regionally coordinated 179.GHEC at the national level 199.1.The national health emergency workforce 209.2.National surge capacities 239.3.Connected leaders atthenational level 269.4.Guiding questions on GHEC at national level 2910.GHEC,a connected ecosystem 3010.1.Initial implementation of the GHEC approach 3110.2.Health emergency networks for surge and coordination in the GHEC ecosystem 3310.3.Leaders networks in the GHEC ecosystem 4311.Conclusion 47Annex 1.References 48Annex 2.Definitions 50ContentsGlobal health emergency corps frameworkivThe COVID-19 pandemic has exposed our collective vulnerability to health emergencies.No country has been spared its toll on lives and livelihoods.We havewitnessed unprecedented scientific achievements and acts of solidarity,but also have endured tragic inequities,delayed responses,and preventable loss of life.Theselessons compel us to redesign our approach to future crises.The Global Health Emergency Corps(GHEC)is a transformative approach to the most important element in any emergency response its people.Launched at the World Health Assembly in 2023,GHEC is more than a workforce framework;itis a commitment to global solidarity,prioritizing sovereignty and equity while also fostering preparedness and response capacities.Anchored in the WHO Health Emergency Prevention,Preparedness,Response,and Resilience(HEPR)framework,it encourages countries to strengthen their health emergency workforces,surge capacities,andnetworked leaders for decisive action.This first iteration of the GHEC framework provides practical guidance for adopting and adapting the GHEC national workforce pyramids and their collective ecosystem.This ecosystem offers a foundation for collaboration across countries,regions,and organizations,bringing together the best of existing networks in a more standardized,interoperable,and effective approach.As you read and use this document,I encourage you to reflect on our commitment to sovereignty,equity,andsolidarity.Together we can transform global preparedness and safeguard future generations.Dr Michael RyanExecutive Director,WHO Health Emergencies Programme and Deputy Director-GeneralForewordvThe GHEC framework was developed by the World Health Organizations Health Emergencies Programme.The guidance was developed with the support of the members of the GHEC Design Group and in close collaboration with other WHO departments,regional and country offices.Special gratitude is expressed to the members of the Design Group tasked with elaborating a process for operationalizing the GHEC and producing this document to be used as reference at national,regional and global levels for adaptation and adoption of the GHEC approach.Design Group member gathered in a workshop held in Montreux,Switzerland from 30 October to 1 November 2024 to review and finalize the GHEC framework.WHO appreciates the input of the following experts:Amanda McClelland,Andrew Lee,Carita Davis,David Aponte Jurado,Didier Koumavi Ekouvi,Duncan Selbie,Edson Rwagasore,Eduardo Samo Gudo,Gail Carson,Guoqing Shi,James Chan,Johanna Hanefeld,Leonard Notaras,Leonardo Gomes Menezes,Melkamu Abte,Meredith Neilson,Mohannad Al-Nsour,Nikki Romanik,Sadaf Lynes,Soha Shawqi A Albayat,Thebeyame Macheke,Valerie Nkamgang Bemo,Waqo Boru.WHO also wishes to acknowledge the contributions to the GHEC concept and drafting of the framework by colleagues from various WHO regional and country offices including Abdou Salam Gueye,Alexander Rosewell,Alfred Rutagengwa,Ara Johannes,Babatunde Olowokure,CiroUgarte,Edwin Salvador,Gina Samaan,Gundo Weiler,Ishata Nannie Conteh,Jerry-jonas Mbasha,Kentse Moakofhi,Leonardo Hernandez,Luis De La Fuente,Nilesh Buddh,Oleg Storozhenko,Rick Brennan,Samar Al-Mutawakel,Sean Casey,Sharon Salmon,Sourabh Sinha,Sugi Wagawatta Perera,Wasiq Khan.Invaluable contributions to the GHEC concept and drafting of the framework were also provided by colleagues from the WHO headquarters particularly from the following:Abdi Mahamud,Armand Bejtullahu,Awa Ndir,Camila Philbert Lajolo,Catherine Smallwood,Chikwe Ihekweazu,Elizabeth de Guia Tecson,Emma Fitzpatrick,Flavio Salio,Huan Xu,Ian Clarke,Ibrahima Soce Fall,Jian Li,Linda Doull,Maria Popovich,Patrick Anthony Drury,Marie-Amlie Degail,Marion Muehlen,Michel Yao,Michael Ryan,Nedret Emiroglu,Nicoleta Dumitru,Renee Christensen,Scott Pendergast,Siobhan Fitzpatrick,Sohel Saikat,and Tapas Nair.WHO also acknowledges the contributions in the drafting of the framework from consultants Ebele Mogo and Gillian Dacey.The development of the GHEC framework and coordination of the drafting process was led by Scott Dowell and Christophe Schmachtel(WHO Health Emergencies Programme).WHO also wishes to acknowledge the financial contribution of the Gates Foundation which supported thedevelopment of this document.AcknowledgementsGlobal health emergency corps frameworkviACoDDAFENET Corps of Disease DetectivesAFENETAfrican Field Epidemiology NetworkAUSMATAustralian Medical Assistance TeamAVoHC-SURGEAfrican Volunteer Health Corps Strengthening and Utilizing Response Groups for EmergenciesCOVID-19Coronavirus Disease 2019ECDCEuropean Centre for Disease Prevention and Control EOC-NETPublic Health Emergency Operations Centre NetworkEMTEmergency Medical TeamEPHFEssential Public Health FunctionsEEAEuropean Economic AreaFETPField Epidemiology Training ProgramsGHCGlobal Health ClusterGHECGlobal Health Emergency CorpsGOARNGlobal Outbreak Alert and Response NetworkHEPRWHO Framework on Strengthening Health Emergency Preparedness,Response,and ResilienceIANPHIInternational Association of National Public Health InstitutesIHRInternational Health RegulationsNPHANational Public Health AgencyNPHINational Public Health InstitutePHEOCPublic Health Emergency Operations CentreRRTRapid Response TeamSARSSevere Acute Respiratory SyndromeSARS-CoV-2Severe Acute Respiratory Syndrome Coronavirus 2TEPHINET Training Programs in Epidemiology and Public Health Interventions NetworkWHOWorld Health OrganizationAbbreviationsviiExecutive summaryThe COVID-19 pandemic exposed significant vulnerabilities within the global public health system,ascountries faced overwhelming challenges in responding to a rapidly evolving crisis.Existing systems and institutions were inadequately prepared for the scale of the pandemic,leading to fragmented responses,and exacerbating global inequities.Ashighlighted by reviewing authorities such as the Independent Panel on Pandemic Preparedness and Response(1)and the Global Pandemic Monitoring Board(2),there is a critical need for leadership at the highest levels and for collaborative,equitable action in preparing for future global health emergencies.The draft Pandemic Agreement specifically calls for countries to invest in a skilled workforce to respond tohealth emergencies and to establish response teams in coordination with WHO and others(3).The Global Health Emergency Corps(GHEC)is the body of experts in ministries and agencies in every country who work on health emergencies and the global ecosystem through which they coordinate.GHECwas launched in 2023 in direct response to these calls for proactive change.GHEC aims to strengthen and connect health emergency response capacities across countries,regions and globally through a predictable,scalable,and structured approach to health emergency workforce set up and coordination.GHEC is anchored within the broader WHO Health Emergency Prevention,Preparedness,Response and Resilience(HEPR)framework.It begins by prioritizing sovereignty,equity,and solidarity in addressing health emergencies.By adopting and adapting the GHEC framework,countries enhance their access to an ecosystem of emergency leaders,interoperable surge capacities,and human resources for strengthened health emergency systems and responses.The three levels of the GHEC workforce form afoundational framework,which countries are encouraged to adopt and adapt to their unique contexts,including variations in system types,resourcecapacities,and workforce structures.Thelevels represent(i)a strengthened and structured national emergency health workforce capable of rapidly scaling up to meet the demands of a health crisis,(ii)deployable and interoperable surge capacities that canbe rapidly mobilized in response to emerging health threats,and(iii)connected leadership at all levels to ensure swift and effective decision-making,with the collective expertise needed for a strong,unifiedresponse.This is the first version of the GHEC framework and is intended to be updated as experience is gained with its implementation and adaptation.This document outlines the guiding principles that form GHECs foundation for ensuring coherence and efficiency in national,regional,and global preparedness and response capabilities.It includes guiding questions to help countries assess and structure their national health emergency workforces to ensure more effective and interoperable responses.Additionally,it highlights the institutions and networks that form the GHEC ecosystem,detailing the resources they provide for enhanced emergency preparedness and responses.Furthermore,it provides examples of existing deployments and regional and global leadersnetworks,illustrating components of the GHECecosystem inaction.Future iterations will build on this foundation,incorporating lessons and best practices from further GHEC simulations,activations,and work in countries,continuously deepening the core capabilities of this ecosystem for a more coherent and efficient response to future health threats.viii11.IntroductionWhen faced with the common threat of Severe Acute Respiratory Syndrome Coronavirus 2(SARS-CoV-2),countries and institutions across the world were rapidly overwhelmed by the monumental task of responding to a fast-moving threat,needing to invent ad hoc structures and alliances to scale response efforts.AstheIndependent Panel on Pandemic Preparednessand Response stated:“Current institutions,public and private,failed to protect people from a devastating pandemic.Without change,they will not prevent a future one.That is why the Panel is recommending a fundamental transformation designed to ensure commitment at the highest level to a new system that is coordinated,connected,fast-moving,accountable,just,and equitable in other words,a complete pandemic preparedness and response system on which citizens can rely to keep them safe and healthy.”Rather than proactively coordinating their respective response efforts,many countries took very different approaches,leading to the accelerated spread of COVID-19.Some,led by those that had most directly experienced Severe Acute Respiratory Syndrome(SARS)in 2003,mounted aggressive surveillance,contact tracing,and public health suppression measures that proved remarkably effective at containing the virus over weeks to months.Others took a much less proactive approach.The results ranged from near-complete containment to massive outbreaks with eventual spillover to all other countries.More than 16 million lives were lost in the first 2 years of the pandemic(4),and estimates of the economic impact ranged from$10 trillion to$15 trillion(5),equating to approximately 10%of global Gross Domestic Product(6).Interventions that result in even modest improvements in these enormous numbers would rank among the most consequential global public health interventions in history.However,response systems within individual countries often proved inadequate to the challenges of COVID-19.Existing health emergency structures were overwhelmed by a pandemic that led authorities to take drastic decisions such as closing schools and borders and declaring lockdowns,therewith disrupting global supply chains,and damaging entire economies,causing socio-economic problems for a large part of the population worldwide.Most countries needed to establish new leadership structures ad-hoc,often led by higher level officials with limited previous experience managing health emergencies.Moreover,the systems required for surge responses for clinical care,contact tracing,risk communications,community engagement,and eventually,scaled up vaccination campaigns were quickly overwhelmed.This necessitated solutions to upscale response capacities amidst limited possibilities for deployment between countries.The inevitable result was an ineffective response characterized by a lack of global solidarity,and eventually,massive global inequity.The pandemic was a stark reminder that“our well-being depends on the well-being of others”(7).Equity is therefore at the heart of efforts to strengthen the global health architecture.The Global Health Emergency Corps(GHEC)is the body of experts in ministries and agencies in every country who work on health emergencies and the global ecosystem through which they coordinate.Assuch,it offers a more uniform yet adaptable approach to strengthening the health emergency workforce within and among countries that can provide coherence to the global system that proved ill-equipped during the pandemic,whilst building on the many good examples of collaborations to strengthen the health emergency workforce over the years.GHEC builds upon the principle of national sovereignty and the recognition that decisions on response,whether to a pandemic or other health emergency,will always rest with national authorities.It is a core component of the WHO Framework on Strengthening Health Emergency Preparedness,Response,and Resilience(HEPR)(8).Global health emergency corps framework2Countries are encouraged to adopt and adapt the GHEC framework by identifying and investing in their standing and on-call emergency workforce capacity to manage ongoing health emergency preparedness and response work,complemented by surge teams and experts that can be deployed as needed either from within or outside the country,and health emergency leaders who are well embedded within the highest level of multisectoral health security coordination within the government.Together,these three levels can be visualized as a GHEC workforce pyramid that is part ofthe countrys public health workforce(Fig.1).The three levels of the GHEC workforce pyramid are present in varying degrees in all countries,yet many times inconsistently recognized and structured.In line with the HEPR framework,GHEC embraces an approach to collaboration and coordination that identifies the need to connect workforce capacities and capabilities across the core health emergency sub-systems of collaborative surveillance,community protection,safeand scalable care,access to countermeasures,andemergency coordination(the 5Cs).GHEC is not to be understood as a separate entity ornetwork but as the structured workforce(thebody of experts)at country level working on health emergencies and as a global ecosystem in which countries and health emergency actors collaborate andcoordinate more efficiently through interconnected health emergency networks at all levels of the GHECpyramid.Pandemics are rare but other health emergencies are not.Fortunately,preparing systematically for a pandemic also synergistically prepares countries and regions for other types of emergency responses.Across a broad range of health emergencies,investing in a well-organized emergency workforce,interoperable surge capacities,and connected leaders has proven to be invaluable.Exercising these capacities through regular responses to a range of health emergencies further strengthens them,laying a necessary foundation for responding to large epidemics and future pandemics.This GHEC document has been developed with the purpose of setting guiding principles and actions tofacilitate a systems approach to the individual andcollective capacity of countries and institutions in preparing for and responding to disease outbreaks,pandemics and health consequences of emergencies or disasters.Entities may be international,regional,or national.The common shared activity is for these entities to play a role in the prevention,preparedness,and response to emergencies where there is an impact on human health and well-being.GHEC embraces a systems approach that integrates professionals and their resources,an approach to collaboration and coordination,and the need to identify and connect collaboration platforms.The challenge that this document aims to address is reduction of duplication,wasted resources and missed opportunities to build and strengthen capacities and emergency response capabilities while providing a coordinated approach formuch needed expertise and response capacity intimes of need.Public health workforceConnected leadersSurge capacitiesEmergency workforceFig.1.The GHEC workforce pyramid atcountrylevel31.IntroductionThis framework was developed with contributions from a wide range of stakeholders,including those from large high-income countries with complex health emergency infrastructure,and from small and resource-limited countries with only the basic elements ofa health emergency workforce.Itreflects contributions from leaders of high functioning global health emergency networks,regional health emergency leaders,as well as global health organizations.The document spotlights examples from various settings in textboxes to illustrate variations in institutional structures,national and regional organizations.These examples show components of the health emergency corps in every country,or case studies on the functioning of the GHEC ecosystem.The GHEC approach is intended to support countries in assessing and structuring their current health emergency workforce and related coordination and collaboration structures,including their participation in regional and global health emergency collaboration structures and networks.This includes identifying elements that are missing or require strengthening to ensure a robust response to health emergencies in coordination with neighbouring countries,regionally,andglobally when necessary.For regional and global networks and collaboration structures,the GHEC approach is intended to encourage better connection and coherence to avoid duplication in support ofcapacities at country level.Box 1Vignette How the Global Health Emergency Corps could have changed the COVID response Had the Global Health Emergency Corps been in place in January 2020 the course of the COVID-19 pandemic might have played out differently.Corps members in public health leadership positions in countries around the world,having exercised scenarios such as the SARS-1 outbreak,would have recognized this threat for what it was by the early days of January the spread of an efficiently transmitted human coronavirus.In close communication and acting in unison on a modified SARS-1 playbook,countries across the globe could have put measures in place to contain the threat,as did many places with SARS-1 experience,such as Singapore,Viet Nam,Canada,Thailand,Australia,and China(12).SARS-CoV-2 was a more challenging pathogen,with pre-symptomatic transmission and biologically significant mutations,and the sociopolitical environment and inequitable distribution of resources impeded control.Still,consistently applied measures,such as travel restrictions,mask mandates,and rigorous testing,contact tracing,isolation,and quarantine could have limited spread(12).The result could have been like the early COVID-19 results in those countries driving the effective reproductive number(Re)below 1 and substantially delaying exponential growth of the pandemic.Fewer places would have experienced explosive outbreaks,and more would have had experiences like Australia,New Zealand,and others with substantial delays in exponential spread.This flattening of the epidemic curve could have led in many places to a result like the first SARS containment of the pandemic threat.For places where the epidemic did manage to take hold,Corps members and their institutions,coordinated globally by WHO might have concentrated efforts,sharing information,deploying diagnostics,antiviral treatments,and mRNA vaccines.The pandemic could have been significantly curtailed in early 2020,saving millions of lives and trillions of dollars,or at least itsexponential spread could have been substantially delayed.Global health emergency corps framework4Summary of relevant post-COVID-19 recommendationsThe COVID-19 pandemic exposed critical gaps in global health workforce preparedness,highlighting the need for resilient systems and better coordinated responses.Various expert committees have since reviewed and drawn lessons from these shortcomings,including the Global Preparedness Monitoring Board,the Independent Panel for Pandemic Preparedness and Response,the International Health Regulations Review Committee,andthe Independent Oversight and Advisory Committee for the WHO Health Emergencies Programme.National public health workforceThe public health workforce includes all people who contribute to the delivery of at least one of 12essential public health functions(EPHFs),as part ofintegrated health system services and functions(10).This workforce comprises people working in diverse occupations,from health and non-health sectors,and can be conceptually framed as three overlapping groups:core public health personnel who work exclusively on the EPHFs;health and care workers who spend some of their time delivering the EPHFs as part of their clinical or social care roles;and personnel from occupations allied to health who play critical roles inaddressing the determinants of health.The Global Preparedness Monitoring Board emphasizes the need for national public health systems to invest in the public health workforce and core capacities for surveillance,early detection,and resource allocation tomitigate health threats(9).It is critical to define EPHFs,including emergency preparedness and response,and enhance the capacities required to deliver these functions(10).The Global Preparedness Monitoring Board emphasizes that such investments must be equitable,as inequities not only undermine national capacities but also erode the trust and international collaboration essential for preventing outbreak amplification(2).Emergency workforceThe health emergency workforce draws from the public health workforce,with a reduced proportion of the public health workforce fully dedicated to or on-call towork on emergency preparedness and response.The emergency workforce must be well-trained and comprise a range of emergency-specific skills and expertise to ensure the EPHFs related to emergency preparedness and response are fulfilled.The capabilities need to cover the main health emergency preparedness and response capability areas of collaborative surveillance,community protection,safe and scalable care,access tocountermeasures,and emergency coordination(8).National governments must ensure they have agile health emergency systems that can equitably address emergencies through better information sharing,science-based action,research,and development(9).Support to these teams must leverage global coordination and resources,such as making more robust use of WHO collaborating centres worldwide,as well as expert networks,such as technical advisory bodies and public health institutes(11).2.Background52.BackgroundSurge capacitiesAccording to the Independent Panel,the efficiency of national responses depended to a large degree on each countrys ability and agility to manage surge health workforce demands(12).The Panel emphasizes that in our interconnected world,global health security is only as strong as its weakest link,making national public health systems the first line of defence against the nextpandemic(11).National health systems must thus ensure they have the ability to deploy surge capacities including experts and multi-disciplinary teams for risk assessment and investigations,community,clinical,and supportive services,without compromising existing core needs.Itmay also include the capacity to receive surge capacities from abroad,when required.On the other side,countries and organizations preparing their experts and teams for surge deployments to reinforce country capacities in emergency situations should do so based on existing quality standards and through the relevant coordination platforms.Note:the term surge capacities is used interchangeably with the term rapid response capacities in this document.Connected leadersConnected leaders,who communicate through trusted informal and formal channels,enable the rapid sharing of information and knowledge,and the coordination of responses before,during and after emergencies occur.The Global Preparedness Monitoring Board concluded that“the first year of the COVID-19 pandemic was defined by a collective failure to take preparedness seriously and act rapidly on the basis of science”,and it characterized the second year as“marked by profound inequalities and a failure of leaders to understand our interconnectedness and act accordingly”(9).The Board noted that improved collaboration between countries serves as a critical shield against the amplification of outbreaks(2).TheIndependent Panel underscored the decisive role of leadership and competence as even more important than financial resources in the pandemic response(12).According to the panel,previously assessed levels of preparedness had not accounted for the impact of political leadership,trust in government institutions,and countries abilities to mount fast and adaptable responses.The Panel noted the need for champion leaders at the highest political levels,who can help to close the gaps in the international system and activate responses during crises(11).In fact,the need for stronger leadership and better coordination was a prominent theme in virtually all the post-COVID-19 recommendations(12).Panels highlighted the need for improved leadership and coordination at national,regional,and international levels(13,14),including through stronger networking between experts and existing health emergency networksand platforms(15).Global health emergency corps framework6Actions in response to the post-COVID-19 recommendationsThe summary above synthesizes over 300 recommendations identified in numerous expert reviews on the successes and shortcomings of the global response to the COVID-19 pandemic as they related to the coordination,workforce capacity,networking,and health emergency leadership(Fig.2).WHOhas been working with Member States and partners to translate these into concrete and concerted action to save lives and reduce morbidity,presenting them in theWHO Framework for Health Emergency Preparedness,Response,and Resilience(the HEPR framework)(8).The recommendations are categorized into the three main themes of global governance,financing andHEPRsystems.Published in 2023,the report from the WHO Director-General,Dr Tedros Ghebreyesus,titled“Strengthening health emergency prevention,preparedness,response and resilience”(HEPR)lays out the“five Cs”as the five core health emergency capability areas:Collaborative surveillance,Community protection,safe and scalable Care,access to Countermeasures,and emergency Coordination(Fig.3).Fig.2.Reviews,reports,and processes that have contributed to the framework for a strengthened global architecture for health emergency prevention,preparedness,response,and resiliencePandemic AccordGovernanceSystemsFinancingEquityInclusivityCoherenceIndependent Panel for Pandemic Preparedness and Response report GPMB and IOAC reportsOther reportsIHR Review Committee on the Functioning of the International Health Regulations(2005)during the COVID-19 ResponsePan-European Commission on Health and Sustainable Development High Level Independent Panel on Financing the Global Commons for Pandemic Preparedness and Response reportINB and WGPR processesG20 and G7 processesOther processesGPMB:Global Preparedness Monitoring Board;Intergovernmental Negotiating Body to draft and negotiate a WHO convention,agreement or other international instrument on pandemic prevention,preparedness and response;IOAC:Independent Oversight and Advisory Committee for the WHO Health Emergencies Programme;WGPR:Member States Working Group on Strengthening WHO Preparedness and Response to Health Emergencies.Source:World Health Organization.Strengthening the global architecture for health emergency prevention,preparedness,response and resilience.Geneva;2023More than 300 recommendations72.BackgroundFig.3.The five Cs of health emergency prevention,preparedness,response,and resilienceSafe and scalable careScalable clinical care during emergenciesProtection of health workers and patientsMaintenance of essential health servicesCommunity protectionCommunity engagement,risk communication and infodemic managementPopulation and environmental public health interventionsMultisectoral action for social and economic protectionEmergency coordinationStrengthened workforce capacity for health emergenciesStrengthening health emergency preparedness,readiness,and resilienceHealth emergency alert and response coordinationCollaborative surveillanceStrong national integrated disease,threat,andvulnerability surveillanceEffective diagnostics and laboratory capacity for pathogen and genomic surveillanceCollaborative approaches for event detection,risk assessment,and response monitoringAccess to countermeasuresFast tracked R&DScalable manufacturing platformsCoordinated supply chains&emergency distributionAccess to countermeasuresEmergency coordinationCommunityprotectionSafe andscalable careCollaborative surveillanceGlobal health emergency corps framework8The Global Health Emergency Corps,launched in 2023,is anchored within the emergency coordination component of the five Cs to reflect the central and cross-cutting nature of the health emergency workforce for all five capability areas.It emphasizes the need for a coordinated workforce capacity for health emergencies,by enhancing coordinated leadership,deployable and interoperable surge capacities,and a well-practiced emergency workforce present in all the five subsystems.In practice,the deployment planning and organization of the emergency workforce is a core function of emergency coordination but necessarily involves engaging professionals from the other 4 Cs.Depending on the emergency,the workforce engaged in community protection or countermeasures may take precedence,or those working on collaborative surveillance or care may become more prominent,while the others continue with their regular duties untilan emergency requiring their skills emerges.The GHEC builds on a series of longstanding calls for strengthening the worlds health emergency and pandemic responders.Highlighted as a priority by Dr Tedros Adhanom Ghebreyesus in his earliest days as theWHO Director-General and characterized as a“Global Epidemic Response and Mobilization(GERM)team”by BillGates in his book“How to Stop the Next Pandemic”(16),thoughtful and influential leaders have repeatedly called for a dedicated cadre of health emergency professionals.Launched at the World Health Assembly in May 2023,the GHEC is the logical outgrowth of the post-COVID-19 recommendations.It reflects the need for a strengthened collaborative approach within and among countries and health emergency networks,for connected health emergency leaders,surge capacities and an established group of dedicated and on-call professionals in every country to lead anew era of emergency coordination.GHEC is well integrated with the HEPR framework,the International Health Regulations(IHR)concept of National Health Authorities,and a reinvigorated approach to pandemic financing.Box 2Why a“Corps”?Corps A body of persons having a common activity oroccupation,e.g.the press corps Merriam-Webster dictionaryHealth Emergency Corps A body of experts in ministries and agencies in every country who work on health emergencies and the global ecosystem through which they coordinate to:Strengthen responses to all health emergencies,andStop the next pandemic93.Purpose of this documentThe purpose of this document is twofold.1 To set guiding principles and actions to strengthen the individual and collective capacity of countries and institutions in preparing for and responding to disease outbreaks,pandemics and health impacts ofemergencies or disasters.2 To address the challenge of connecting regional and global response mechanisms in support of countries,reducing duplication,and maximizing opportunities in building and strengthening capacities and capabilities whilst providing a coordinated approach for much needed expertise and response capacity attimes of need.The GHEC frames the complex health emergency workforce ecosystem with the aim of bringing coherence and efficiency to the web of actors andrelationships from the local to the global level.Global health emergency corps framework10The broad concepts of the GHEC framework arose from the numerous reviews of the COVID-19 response cited above and were articulated by WHO Director-General,DrTedros Adhanom Ghebreyesus and other WHO leaders culminating in the 2023 launch of GHEC in the margins of the 2023 World Health Assembly.Inthe months following the launch further feedback from countries,networks,and regions emphasized the importance of country leadership and national sovereignty,the value of existing health emergency networks,and the growing role of regional entities in responding to epidemics and other health emergencies.The development of the GHEC framework originated in the publication of the HEPR framework(8).Theframework expands on the Emergency Coordination section,5.1 on“Strengthened workforce capacities for health emergencies”,and a concept note on strengthening health emergency workforce capacities that had been developed through an extensive consultation process throughout 2022 and2023 as part of the broad process to develop theHEPR framework.In 2024,WHO convened a GHEC Design Group of experts from 15 countries and leading health organizations and networks with the specific task of developing the GHEC framework.Identification of design team members sought balance in terms of regional representation,large and small countries,gender and content expertise.The development process included six online consultations over a period of approximately 6 months and a face-to-face workshop,held from 30 October to1November 2024 in Montreux,Switzerland.An initial draft outline was prepared by the WHO GHEC Secretariat for the first consultation,then revised based on feedback by the group members.Members of the Design Group contributed text to different sections ofthe document which was consolidated by the GHEC Secretariat.At the end of that period the group was convened in a workshop that used a scenario-based exercise to test the GHEC framework and proposed modifications and improvements.Final revisions and clearances of the framework document involved all WHO regional offices,design team members,and network representatives,as well as internal WHO clearances.4.Design process and approach11The vision of the GHEC is a well-coordinated health emergency workforce centred in countries comprised of coordinated leadership,interoperable surge capacities,and a well-practiced emergency workforce able to rapidly detect and respond to disease outbreaks,pandemics,and humanitarian emergencies.5.VisionGlobal health emergency corps framework12The objectives of the GHEC are twofold:to strengthen the readiness and response to health emergencies,and to stop the next pandemic.The two objectives are interrelated and mutually reinforcing.Strengthening theresponse to all health emergencies depends primarily on a robust,well-skilled health emergency workforce in all countries,on interoperable surge capacities,and on leadership and coordination at national,regional,and global levels.These are critical elements that contribute to stopping the next pandemic most efficiently.Success also depends on the earliest possible detection,a decisive and coordinated global response,and universally accessible medical countermeasures.However,the most critical factor remains a well-coordinated workforce.The notion of a“corps”describes a body of persons having a common activity or occupation(Box 2).Applied to the GHEC context,this concept translates into the need for a Health Emergency Corps at the country level,a structured and coordinated approach toits health emergency workforce capacity.By leveraging the collective resources of all countries inawell-coordinated way,the GHEC ecosystem allows formore effective and timely response that can be scaled up as needed to deliver better coordinated regional and global responses.While collaboration and coordination platforms remain specific to each region,maintaining a similar structure enables globalcoordination when needed.6.ObjectivesTable 1.Ten benefits for countries adopting and adapting the GHEC approachAt the leadership level1 Equip your emergency leaders with quick access to evidence sourced from trusted counterparts.2 Connect your emergency leaders with their trusted counterparts in the region and globally.3 Help your country be a prominent voice in coordinating regional and global response efforts.At the surge capacity level4 Faster on-ground deployment of national surge capacities,due to established plug-and-play models.5 Access to a trusted backup of surge capacities that can be activated quickly when your country needs extra support on-the-ground.6 Create interoperable capacities within your national surge system that allow you to support your region in responding to health emergencies faster.7 Improve connectedness between your government and other governments and institutions regionally and globally,through the networks channels.At the emergency workforce level8 Provide coordinated access to enhancing the expertise and skills of your emergency workforce across multiple specialities through expert networks and institutions.9 Provide your surge teams,experts,and emergency workforce with platforms to exchange,jointly train and conduct simulation exercises with their peers across different countries andspecialties.10 Enable access to experienced human resources who can share learnings on outbreak preparedness and responses that may help inform the way your country strengthens itsownhealth systems.136.ObjectivesTable 2.Six asks of countriesAsk 1Adopt and adapt the workforce pyramid as your framework for strengthening and globally connecting your national health emergency corps.Ask 2Identify your senior health emergency leaders who can be connected to regional and global networks of trusted colleagues.Ask 3Take steps to build interoperable surge capacities from your trained emergency workforce.Ask 4Commit to deploying your surge capacities as part of coordinated international emergency efforts.Ask 5Allocate resources to build the right size,expertise,and skills of the health emergency workforce for timely detection,alert,and response to all health threats.Ask 6Map the participation of your countrys institutions in health emergency networks and ensure that network focal points are well-connected with your national health emergency leadership and coordination structure.Global health emergency corps framework14The GHEC is based on three guiding principles:Sovereignty:It is owned by countries and their national institutions competent in health emergency leadership,surge capacities,and emergency workforce coordination Equity:It is committed to providing timely and equal access to information and support to every country and population Solidarity:It is supported by countries,regional and global networks,and institutions committed to collaboration to achieve the common vision of GHEC Furthermore,committing to the GHEC entails voluntary adherence to a set of core values which include:Quality and professionalism:Enhancing the quality and professionalism of pandemic and health emergency response and coordination,including through joint training and exercises Predictability and efficiency:Functioning based on standard operating procedures supported by thenecessary tools and management support Trust and acceptance:Continuously building ontrust and acceptance amongst members 7.Guiding principles and values158.StrategyEffectively leveraging the GHEC begins with the recognition of national sovereignty,assuming that in a pandemic or other health emergency every country will prioritize the needs of its citizens and direct the actions of its emergency responders.Yet,each country will also depend on the coordinated actions of other countries if a regional epidemic or pandemic is to be stopped.Similarly,some health emergencies may overwhelm the capacities of affected countries where international cooperation may be required.These apparently competing observations can be more easily reconciled if the health emergency workforce pyramids in all countries are similarly structured.A structured,yet adapted approach to strengthening the health emergency workforce in every country(Fig.4),with interoperable surge capacities to ensure that no area or country is overwhelmed,and an interconnected group of leaders coordinating their responses at national,regional,and global levels,provide the basis for a more effective response to health emergencies including pandemics.The boundaries and names shown and the designations used on this map do not imply the expression of any opinion whatsoever on the part of the World Health Organization concerning the legal status of any country,territory,city or area or of its authorities,or concerning the delimitation of its frontiers or boundaries.Dotted and dashed lines on maps represent approximate border lines for which there may not yet be full agreement.WHO 2025.All rights reserved.Not applicableA health emergency workforce centered in countriesConnected leaders Connect senior national health emergency leaders inatrusted network.Surge capacities Standardize quality and enhance interoperability between national,regional and global rapid response capacities.Emergency workforce Strengthen local and national health emergency preparedness andresponse workforce.There is no global health security without local and national health security.Dr Tedros Adhanom GhebreyesusWHO Director-GeneralEvery country has an emergency corps.Public health workforceConnected leadersSurge capacitiesEmergency workforceFig.4.Consistently organized health emergency workforces in every country Global health emergency corps framework16The elements of a national workforce corresponding to the GHEC are summarized in the upper three levels of Fig.5,hereinafter referred to as health emergency corps.The bottom and largest level depicts the entirety of the national public health workforce to deliver on the essential public health functions and requires the definition at country level of the essential public health functions and subfunctions,development of competency-based education,and mapping and measurement of occupations.This work lays a solid foundation for the national Health Emergency Corps.Establishing the emergency-specific capacities at the leadership,surge and emergency workforce levels requires a recognition of the specificities inherent in health emergency management,whilst also recognizing that these capacities are embedded within the national public health workforce.It will also lead to creating robust,sustainable,and attractive career pathways forhealth emergency workers.As the GHEC framework is adopted and adapted more explicitly in countries and regions,career recognition and opportunities may arise for a range of public health professionals who may have previously engaged in emergency response only as an occasional responsibility.Clear career pathways will likely include entry-level positions for recent graduates such as emergency response coordinators,surge deployers,or field officers.Mid-level regional managers,technical advisors,surge coordinators and team leaders are suitable positions for those with several years of experience;while global health directors,policy advisors,and connected leaders are best suited to seasoned professionals who have worked on health emergencies at national and sub-national levels for years and demonstrated their leadership skills.International secondments,including potential WHO-supported initiatives,could provide seasoned professionals with opportunities to share their expertise across countries and on the global stage,fostering cross-border learning and collaboration.Fig.5.A framework for strengthening national health emergency leadership,surge capacities,andemergency workforce capacityConnected leadersPredictable and institutionalized coordination between senior-level strategic and technical health emergency leaders during preparedness and responseSurge capacitiesEnhancing the quality,predictability and interoperability of national,regional and global surge capacities by strengthening country rapid response capacities and leverage existing networks and mechanisms on the basis of common quality standards and coordination protocolsEmergency workforceStrengthening national emergency workforce for alert,response and preparedness coordination&implementation(e.g.professionalization of occupations,where relevant)National public health workforceImplementing the WHO Roadmap to increase national workforce capacity to deliver the essential public health functions(EPHFs),including a focus on emergency preparedness and response178.StrategyFostering such robust workforces requires countries toadopt a comprehensive approach to training programs including a foundation of focused training in such fields as epidemiology,emergency management,emergency logistics,and laboratory science,continuing education including advanced certifications,and periodic simulation exercises to ensure that skills remain up to date.Mentorship,peer support,and ongoing professional development seminars and workshops are helpful,recognizing that global mobility and flexibility inwork arrangements make health emergency response work attractive to many talented young professionals.Partnerships between government agencies,non-governmental organizations,and academic institutions provide added career flexibility and incentives for those entering the field and for retaining talented individuals who have been in health emergency work for many years.In the event of a multinational health emergency orregional epidemic,these national workforce pyramids need to be aligned and coordinated for the most effective response(Fig.6).Strategically,for GHEC,such alignment leans heavily on effective existing emergency response networks.Such longstanding networks already coordinate the response to transnational health emergencies in the areas of humanitarian,disaster response,and public health emergency responses.Networks and coordination mechanisms such as,but not limited to,the Global Outbreak Alert and Response Network(GOARN),the Emergency Medical Teams(EMT)Network,Public Health Emergency Operations Center Network(EOC-NET),and the Global Health Cluster(GHC)already provide well-recognized global leadership andcoordination in their respective spheres.The boundaries and names shown and the designations used on this map do not imply the expression of any opinion whatsoever on the part of the World Health Organization concerning the legal status of any country,territory,city or area or of its authorities,or concerning the delimitation of its frontiers or boundaries.Dotted and dashed lines on maps represent approximate border lines for which there may not yet be full agreement.WHO 2025.All rights reserved.?Globally and regionally coordinatedRegional networksRegional institutionsEOC-NETFig.6.Global and regional coordination of health emergency corps,building on existing networksGlobal health emergency corps framework18The GHEC adds value to these networks by providing acommon framework for plugging into and supporting the national health emergency corps and facilitating collaboration and coordination between health emergency networks and mechanisms,thus aligning the respective contributions,reducing duplication,and maximizing opportunities in strengthening capacities and capabilities in health emergency preparedness and response.Such collaboration facilitates the sharing of resources,expertise,and best practices across networks to avoid duplication of efforts and optimize resource allocation.The primary responsibility for coordinating the emergency response within a country rests with the national authorities.Complementary to this and in line with the GHEC approach,global and regional coordination(Fig.6)will only happen as needed.Therefore,for emergencies affecting a few neighbouring countries,sub-regional coordination may be employed,while regional coordination may be required for more widely dispersed or fast-moving epidemics,and in rare circumstances global coordination may be required fordiffuse emergencies or pandemics.The subsequent section provides a more detailed description of the recommended functions and capability requirements for the health emergency corps at country level,followed by a section describing the GHEC ecosystem in which countries and health emergency actors collaborate through connected health emergency networks.19At country level,the GHEC represents the body of persons in their respective ministries and organizations working on health emergencies,covering all emergency preparedness and response capability areas identified in the HEPR framework(the 5Cs),i.e.,emergency coordination,collaborative surveillance,community protection,safe and scalable care,and access to countermeasures.What makes the Health Emergency Corps an active player in the GHEC ecosystem(section9)are the connections established and regularly put into practice through different health emergency networks and mechanisms.Connections occur and are encouraged at all levels of the GHEC pyramid,including health emergency workforce,surge capacities and leadership.The GHEC approach encourages countries to map these connections and ensure that country focal points of regional and global health emergency networks are well-connected with the health emergency leadership and coordination structure of the country.Taken globally,at least 70%of the GHEC workforce is projected to be professionals working at the country level,with smaller proportions at the regional level(upto 20%)and even less at the global level(10%).Each country will determine the specific composition of its Health Emergency Corps,in alignment with the common parameters framed in this document,adapted as needed to fit its unique national governance structure,its institutional mandates and public health laws,and its local cultures and practices.Thestructure of the Corps is recommended to include three distinct yet interlinked and complementary components,derived from the broader national public health workforce:the staff of the emergency workforce,thesurge capacities,and the connected leaders.9.GHEC at the national levelBox 3The Health Emergency Corps and its positioning within the national workforce capacity to implement the essential public health functions(EPHF)Achieving and sustaining progress towards global health goals such as universal health coverage and health security requires a health and care workforce that can deliver the full range of essential public health functions,including dedicated personnel charged with emergency preparedness and response functions.In light of the lessons learned from the COVID-19 pandemic and other pressing public health challenges,and after a comprehensive review of existing lists of essential public health functions and related concepts,WHO proposed a unified list of 12 high-level essential public health functions.Unsurprisingly,public health emergency management:managing public health emergencies for international and national health security is among these functions.Recognizing that the implementation of each essential public health functions is interconnected,the concept of the Global Health Emergency Corps is based on the learning that a dedicated and specially trained and equipped workforce is required in every country to manage public health emergencies.The GHEC framework therefore provides a consistent approach to implementing this component of the EPHFs,recognizing the need for coordinated leadership,deployable and interoperable surge capacities,andacompetent health emergency workforce.For more information on strengthening national workforce capacity to undertake essential public health functions,please consult:https:/www.who.int/teams/health-workforce/pheworkforceGlobal health emergency corps framework209.1.The national health emergency workforceThe health emergency workforce,comprised of full-time and on-call persons,forms the base of the Health Emergency Corps in every country.Each country has a unique set of institutions and capacities that comprise their health emergency workforce,but there are common elements that can contribute to a more coherent and effective cross-country collaboration inthe GHEC ecosystem.Fully dedicated emergency staff form a small but important part of the national health emergency workforce,and these are likely to be distributed across several EPHF functions including public health emergency management,surveillance,and health protection.Whilst in some countries it may be seen asa hard-to-achieve objective,the COVID-19 pandemic has shown that the rapidly changing dynamic of an infectious threat that transcends boundaries of countries across the world requires well-trained emergency coordination and response structures.Ataminimum,this would translate into having a standing health emergency monitoring and coordination structure(13)such as a public health emergency operations centre.For large-scale or high-impact emergencies,much larger numbers of technical experts and other responders may be temporarily reassigned from their full-time duties to the response for a time-limited period.These additional,on-call emergency workforce members should be drawn from across the national public health workforce to support all five health emergency preparedness and response capability areas:collaborative surveillance,community protection,safe and scalable care,access to countermeasures,andemergency coordination.The sources for these on-call responders vary by country and by emergency,including governmental institutions in human health,animal health,and environmental health,as well as non-governmental organizations,universities,civil society organizations,and the private sector.These varied sources of expertise make the clear identification of institutional responsibilities(17),leadership and coordination structures,and dedicated health emergency staff evenmore important.All countries should have an agile cadre of emergency professionals and experts across a range of disciplines(18).These professionals should be able to activate rapidly,exchange data and information predictably to enable decision making and related action,and reach emergency zones fast with the right skills,equipment,and operational support(19).In countries with ongoing humanitarian emergencies,member organizations of the health cluster or similar health emergency coordination mechanism are key contributors to the countrys health emergency workforce.Specific coordination structures,such as the cluster approach itself,exist in these instances and their role is important to consider in reaching the most vulnerable populations.The emergency workforce is to be activated and coordinated by the relevant competent authority.Depending on the national governance systems for emergency preparedness and response,national or sub-national activation may be undertaken by the Ministry of Health,or the national(or subnational)public health authority or agency.During emergencies,members work within well-defined emergency management systems(such as incident management systems)to pull together the required multi-sectoral and interdisciplinary capacities.Often,their work is organized and coordinated from a Public Health Emergency Operations Center(PHEOC)through anincident management team.219.GHEC at the national levelTo function well,the cadre of health emergency professionals at the national level require the following capabilities:Clarified institutional accountabilities for health emergency leadership,coordination,and response,including surge capacities Dedicated personnel with the right numbers,expertise,and skills toenable timely detection,alert,and response to new events as well as preparedness,prevention and readiness assessment,planning,resource mobilization and implementation.This includes emergency trained professionals incore health emergency capability areas of collaborative surveillance,community protection,safe and scalable care,access to countermeasures,and emergency coordination Sufficient operational support capacity through adequate supplies,space,systems,and financing Continuous specialized and interdisciplinary learning through regular trainings and simulations based on local hazard profiles Box 4Workforce requirements for a Public Health Emergency Operations Center(PHEOC)PHEOCs can be large and complex structures when fully activated,or small and minimally resourced when on watch mode.PHEOCs normally function using an incident command system that is designed for consistency and interoperability to promote the most efficient rapid activation and response to health emergencies.A PHEOC serves as a hub for coordinating the preparedness for,response to,and recovery from public health emergencies.The preparedness includes planning,such as risk and resource mapping,development of plans and procedures,and training and exercising.Theresponse includes all activities related to investigation,response,and recovery.The PHEOC also serves as a hub for coordinating resources and information to support response actions during a public health emergency and enhances communication and collaboration among relevant stakeholders(WHO Regional Office for Africa,2021).Typically,a PHEOC has two types of staff:permanent and surge staff.The permanent staff is responsible for the day-to-day operation of the PHEOC.These include PHEOC manager,leaders of the key functional areas and staff under each area.The PHEOC manager reports to the leadership under which the PHEOC is placed in the ministrys organizational structure and the PHEOC staff report to the PHEOC manager.An EOC should also be able activate rosters of multi-disciplinary and multisectoral experts who can be mobilized and staff the PHEOC,in accordance with the needs identified in the Incident management structure required for the operation.Surge staff can also be activated from partners in which case the procedures need to be clear for requesting this type of support.For further information,refer to the Framework for a Public Health Emergency Operations Center(WHO,2015),currently being updated,or the Handbook for Public Health Emergency Operations Center Operations and Management(WHO Regional Office for Africa,2021)Global health emergency corps framework22Box 5Mozambiques health emergency workforce drawing from multiple sectorsThe Instituto Nacional de Saude is Mozambiques National Public Health Institute,responsible for many of the essential public health functions.Health emergency coordination is often the responsibility of INS,with the emergency response coordination function played by the Public Health EOC,located within the National Directorate of Public Health in theMinistry of Health.In Mozambique,some important components of the emergency workforce are under provincial and district health authorities.In addition,Mozambique has important community health systems,often supported by non-governmental organizations(NGOs).NGOs recruit talented staff and can play important roles in health emergencies.Universities also are a source fortalented technical staff.Although valuable staff may not be within the government coming from civil society or outside the national health system the government sometimes struggles to get them on board in a timely manner when they are needed.Hence,the GHEC emergency workforce in Mozambique must provide the framework that allows for the inclusion of all these elements as needed in an emergency.Box 6China organizing a health emergency workforce in a large complex country Health emergency responses in China are coordinated at the national level from the Chinese Center for Disease Control and Prevention(China CDC)Public Health Emergency Center,under the direction of the National Disease Control and Prevention Administration and the National Health Commission.With some 30 full-time emergency staff,the Center pulls in and coordinates the contributions of technical experts from other institutes nutritional,occupational,infectious,and others.The Center also conducts annual exercises with involved groups to practice surge and coordination.These structures are replicated at varying scales in each of the Provincial CDCs,and at the county and city CDCs across mainland China.Most of the health emergency workforce is pulled in as needed on an occasional basis from their full-time technical work.A particularly valuable component of the coordinated health emergency responses in China is the use of the Public Health Emergency Management System,which was established following SARS 2003.This system places a heavy emphasis on internet-based reporting and advanced disease surveillance systems,facilitating nationwide information flow and coordination for apopulation of more than 1.4 billion.239.GHEC at the national level9.2.National surge capacities In addition to the dedicated body of persons comprising the health emergency workforce,with its dedicated and on-call components,countries should have workforce mechanisms in place to ensure overwhelmed areas are identified and timely supported.This can typically occur in the event of a geographically concentrated health emergency,such as that engendered by an earthquake,but also through a fast-evolving outbreak that overwhelms the local health systems capacities.In such cases,having surge capacity that can be drawn from the temporary repurposing of staff,hiring of additional staff or the deployment of on-call,pre-trained and equipped teams and experts is valuable(20).In implementation of the IHR(2005),every country should develop and test a national multisectoral workforce surge strategic plan that is based on a gap analysis of required surge health workforce for emergencies based on the countrys risk profile(21).Thesurge strategy and plan should include the ability to deploy and receive multidisciplinary surge teams and experts for public health rapid response,clinical care,humanitarian,or disaster response anywhere within its own borders.These teams should include emergency medical teams(including specialized care teams),and public health rapid response teams,both potentially including laboratory capacities,and community-based,volunteer and health teams.Countries are encouraged to establish and maintain their own surge teams based on their capacities and risk profile,and where possible,based on international standards adapted to the country context.For situations that require collaboration and external expertise,countries should also be capable of receiving surge teams from neighbouring countries,other countries,or regional and international organizations,as needed(19).While different approaches exist to developing such deployable surge capacities,many focus on training as the primary activity to build such response capacities,which does not represent the full scope of activities required to achieve sustained improvements in rapid response.The GHEC approach encourages countries to develop sustainable and interoperable systems for surge,where possible enhancing efficiencies by integrating common functions,trainings,and exercises.In this context major global emergency response networks and regional entities have committed to supporting countries in developing strong and scalable rapid response capacities based on the development and planned implementation of joint guidance(22).At the national level,countries can enhance the quality,predictability,and interoperability of their rapid response capacities by developing the following capabilities:Developing and using national minimum standards for rapid response capacities aligned to international standards.Investing in the sustainability of surge capacity development by covering all domains identified in Fig.7(note:more detailed guidance is being developed by a GHEC Technical Working Group onRapid Response Capacities)Establishing integrated and coherent activation,coordination and information exchange protocols,tools,and platforms across surge deployment mechanisms Setting up quality assurance processes and mechanisms for surge capacities Carrying out regular training,workshops and simulations for joint and interdisciplinary learning,cooperation and experience sharing,including with regional and international partners.Global health emergency corps framework24Fig.7.Common domains for the development of sustainable rapid response capacitiesAccountability QualityTraining&simulation exercisesHR&team managementTechnical expertiseData&reportingOperational capacity&capabilityActivation plans&coordinationDeployment history&learningTeam health&welfareTechnical standardsOperational requirementsGuiding principlesLegislationCore standardsCoordination mechanismTypologySystem for surge Relatability Flexibility AgilityBox 7Emergency medical teams as part of Ethiopias surge capacities Ethiopia has different types of workforces who can be deployed within 24hrs for any emergency.These include the rapid response teams(RRTs),AVoHC SURGE responders,European FETP,and EMTs,the latter being a particularly active portion of Ethiopias public health emergency workforce.The initial training of EMTs is the responsibility of EMT programme managers who also oversee continuous education ofEMT personnel.Regular simulation exercises are held to test team coordination and response procedures.Medical countermeasure stockpiles are also managed.In a response,EMTs composed of doctors,nurses,paramedics,and support workers provide on-site clinical management and medical services.EMT clinicians and field epidemiologists may also conduct disease surveillance asper national guidelines on integrated disease surveillance and response,and public health emergency management.This includes collecting epidemiological data from patients.Operations staff include drivers and maintenance crews supporting the logistics of transporting EMTs,equipment and medical supplies to response sites via ambulances and other vehicles.Warehouses are used to pre-position response assets.All of this requires coordination,using incident managers for activation,communication,and operations management when teams are deployed for emergency response.Ambulance dispatch isalso coordinated,and cross-border agreements allow for potential international surge support fromneighbouring country EMTs under the Ministry ofHealth.259.GHEC at the national levelBox 8 The role of research in building rapid response team capacities inPapua New Guinea Research plays a critical role in informing an evidence-based approach to building,training,and sustaining RRTs.In 2024,the Papua New Guinea National Department of Health with financial support from the WHO Regional Office for the Western Pacific and technical support from the University of Newcastle,conducted research on the barriers and enablers to the timely activation and effectiveness of provincial rapid response teams responding to public health alerts inPapua New Guinea.Research findings highlighted that training alone is insufficient for achieving rapid team mobilization andcontainment of public health threats.Sustainable structures supported by clear plans,guidelines,and standard operating procedures are necessary to secure timely funding,maintain human resources,and ensure logistical readiness.Key barriers such as funding delays,financial system rigidity,and the countrys challenging geography hinder response effectiveness.With over 80%of the countrys population living in isolated areas accessible only by helicopter,boat,or on foot addressing these issues requires enhanced communication and structured coordination mechanisms.Providing training to district-level personnel will equip local responders with the skills needed for effective emergency response,in addition,designated focal points for RRT coordination within each provincial health authority would streamline communication and logistics during emergency responses.This will enhance early detection,improve response capabilities,and strengthen resilience inremote communities,enabling a more proactive approach to emergencies.RRTs need more than just training.Challenges andenablers associated with operationalization should be identified and a culture of continual reflection,improvement,and adaptability tochangingneeds created.Box 9 Australias national emergency medical team national and international clinical deployments The Australian Governments Medical Assistance Team(AUSMAT)has the capability of deploying a multidisciplinary emergency medical team to respond to requests in both national and international health emergencies.The National Critical Care and Trauma Response Centre(NCCTRC)is the headquarters and operational base for AUSMAT.AUSMAT consists of multidisciplinary clinicians including nurses,doctors,allied health professionals,medical equipment technicians,biomedical scientists,and logistics officers.AUSMAT can rapidly deploy at short notice to support and assist populations when their health care system is overwhelmed by all hazards disasters.AUSMAT is WHO verified as an EMT Type 1 Mobile and Fixed,and EMT Type 2 Surgical Field Hospital.To ensure teams are prepared,the NCCTRC conducts initial training,continuous education,and simulation exercises for AUSMAT personnel.Anonline resource hub for AUSMAT members and the broader EMT community supports ongoing education and resourcing.The NCCTRC maintains a cache of equipment and supplies to ensure that AUSMAT teams have the capacity and resources available to be fully self-sufficient in the event of a deployment.Both national and international responses are possible,tailored to the request of the host country.Such deployments may include an EMT configuration,specialized care teams,public health rapid response teams,or a bespoke team matrix.AUSMAT mostly deploys internationally however itfirst deployed nationally in 2019 in response to theBlack Summer bushfires in Victoria and southern New South Wales.In response to the COVID-19 pandemic,domestic deployments expanded to include establishing the Howard Springs International Quarantine Facility for repatriated Australians,supporting a Tasmania Emergency Department following an outbreak of COVID-19 amongst hospital staff,and supporting the Victorian Aged Care Responseto COVID-19.Global health emergency corps framework269.3.Connected leaders atthenational levelEvery country has its unique health governance structures in place during emergencies.Health leaders in countries tend to sit within a similar subset of institutions at national or state levels and occupy similar positions including the health minister,chief medical officer/director-general for health,head of national public health authority or agency,director of the national reference laboratory,PHEOC manager,and others.Public health emergencies that involve many countries require strong political and technical leadership and acoordinated approach between many institutions andlevels of government for effective control(9).Strong national leadership is an essential ingredient to provide“unity of purpose and strategy as well as coordination with other countries for resources and coordinated response”(13).The need for multi-country coordination arises in instances of regional health emergencies such as a drought-induced famine and especially true in case of a widespread epidemic such as COVID-19 where ineffective control in one country can quickly impact neighbouring and distant countries(24).Close communication and coordination among the top technical leaders in each country is essential in such circumstances for the most effective control(25).Health emergency coordination and leadership structures are different in every country,but similarities intheir composition exist many times“consisting of Head of Government,Health Minister,Chief Medical Officer/Lead Public Health Officer,Epidemiologists and Virologists as well as civil defense/military representatives”(13).Box 10Thailands public health rapid response teams wide community-level response Established in 2004 in the wake of the first SARS crisis,Thailands surveillance and rapid response teams have expanded to encompass approximately 1,000 teams distributed across the country of 71 million people(23).A key feature of the teams is the use of a multidisciplinary approach with medical doctors,veterinarians,pharmacists,and nurses participating.The RRTs played a key early role in the response to avian influenza in 2004,MERS in 2015,and COVID in 2020,when Thailand identified the first case outside ofChina but had early success in containing its spread.The RRTs supported the COVID response by conducting surveillance and extensive contact tracing,supplemented by the activities of one million existing village health volunteers who were recruited and provided with public health training specific to theCOVID-19 situation.279.GHEC at the national levelThe GHEC approach encourages every country to clearly identify their top technical emergency leaders including those with the relevant experience who might be outside of the government or national health system,who will play influential roles in a health emergency and will be influential in enabling policy-level decisions that are likely toprove decisive in the early stages of a pandemic(26).These leaders are likely those embedded in the senior health emergency coordination structure at the national level.To enable these leaders to have access to quick and trusted information from their peers in other countries,it is recommended that they be networked with their counterparts asneeded,whether amongst neighbouring countries,at the sub-regional level,or regionally and globally in the event of a widespread emergency or pandemic.The characteristics and proposed responsibilities for the connected national leaders include maintaining awareness ofpotential epidemic threats,establishing the authority to direct or influence national epidemic response policies,and leading national epidemic response and pandemic preparatory work,as summarized in Table 3.Table 3.Connected national leaders proposed responsibilitiesResponsibilities of connected leadersNational level activities1 Maintain ongoing awareness of potential transnational epidemic threats Oversee or maintain awareness of the full range of national surveillance targeting potential transnational epidemic threats Work to ensure the analysis and response to potential threats aligns with global norms such as the IHR and response metrics like 7-1-7 metric 2 Establish and maintain authority to direct or influence national epidemic response policy Establish personal authority through formal(directives or statutes)orinformal(precedent and norms)means to direct or influence national epidemic response policy decisions Maintain national policy decision influence through regular exercising ofsuch,trust building,networking with other national authorities 3 Engage regularly in routine naturally occurring outbreak and epidemic responses Lead or influence national level responses to transnational and potential transnational epidemics on a routine basis Align national activation and coordination protocols with common protocols at regional level in collaboration with respective regional emergency directors4 Conduct periodic drills to exercise and test aspects of transnational responses unlikely to be seen in routine epidemics Lead or facilitate simulation exercises at the national level against a range of pandemic threats unlikely to be seen in routine practice Participate in joint reviews,exercises,and training at national,regional,and global levels Share learnings through regional information exchange platforms incollaboration with respective regional emergency directors5 Establish standard operating procedures for a range of potential transnational epidemics Work with relevant sectors within government to develop and gain approval for a range of standard operating procedures addressing varied scenarios of transnational epidemic threatsGlobal health emergency corps framework28Identification of appropriate leaders will be straightforward in some situations and complicated in others.Many countries have a National Public Health Agency with a director and a lead for Health Emergencies,that responsibility may lie with the Ministry of Health,or the lead may depend on the type of health emergency and its origins.All countries have or soon will identify a National Health Authority broadly responsible for IHR implementation in response to the 2024 revision of the IHR,and many countries established multi-sectoral committees or other mechanisms to oversee the COVID-19 response.Once identified,these leaders can be regularly networked with their counterparts elsewhere,as highlighted by the Mycoplasma and the avian influenza A(H5N1)examples(Box 20).Such networking helps to ensure the leaders are empowered with the best possible information about new threats.Also,it is envisioned that the network conduct drills and exercises in addition to emergency responses to strengthen the network for both expected and rare emergencies.A well-constituted set of connected health emergency leaders at the national level entails several elements.It begins with trusted and supported health emergency leadership embedded within the competent national structure that is responsible for crisis control such as the Ministry of Health,National Public Health Agency(NPHA)or the equivalent.These leaders must be well connected,respected,and influential with national policy makers who are likely to make the most critical decisions early in a pandemic,such as instituting travel restrictions,specific public health,and social measures,or launching vaccination campaigns.There should be established and predictable triggers and mechanisms for gathering these leaders across countries and regions to enable common situational awareness and collective decision-making.Regular networking,simulation exercises,and experience sharing amongst health emergency leaders across countries and regions will strengthen the network and improve its effectiveness(See more in section 9.3 Leaders networks in the GHEC ecosystem).Box 11Examples of National Technical Emergency LeadersThe identification of top technical leaders to participate in the GHEC has varied from country tocountry.For example:In China,the director of the Public Health Emergency Operations Center in the Chinese Center for Disease Control and Prevention(China CDC)is identified as a primary point of contact for the Global Health Emergency Corps.China CDC is the lead technical public health agency under the National Disease Control and Prevention Administration(NDCPA)and the National Health Commission,and the IHRNational Focal Point rests with NDCPA.In Qatar,the director of the Health Emergency Department is the primary lead for GHEC as well as being responsible for implementation of all IHR mandates and leading the Public Health Emergency Operations Center.There is also a high level multisectoral committee,a proposed one health committee at ministers/assistant minister level,andthere was a COVID-era strategic committee,along with a multisectoral national committee ledbythe Prime Minister.In Mozambique the primary technical leader for GHEC is the director of the National Public Health Institute.The Instituto Nacional de Saude is an institution subordinated to the Ministry of Health and dedicated to the generation of technical and scientific information in health for Mozambique.In Germany,the acting Vice President and director of the Centre for International Health Protection at the Robert Koch Institute(Germanys NPHA)is the primary technical lead for GHEC.299.GHEC at the national level9.4.Guiding questions on GHEC at national level Connected leaders What is the highest multisectoral health emergency coordination structure in your country?Who are the technical health emergency leaders represented in this coordination structure?How are they linked to the operational and tactical response coordination(i.e.,EOC,PHEOC,etc.)?How do they engage with regional or global networks of peers to share information on health emergencies,including pandemic response strategies and measures?What kinds of multi-sectoral exercises,or trainings are conducted to prepare senior health emergency leaders for informed and quick decision-making during emergencies?Surge capacities What are the required surge capacities identified in your national multisectoral workforce surge strategic plan(as per IHR)?Which surge capacities does your country have on stand-by mode,ready for deployment within 72hours?What standards guide your national surge capacities,and how closely aligned are they withinternational standards?Which regional and global mechanisms or networks for building,deploying,and receiving surgecapacities is your country a part of?What are the protocols for deploying your surge capacities or receiving international surge capacities when required?What type of exercises or trainings conducted to prepare your surge capacities for activation andcollaboration during emergencies?Emergency workforce What is the composition of your emergency workforce dealing with the core capability areas ofemergency coordination,collaborative surveillance,community protection,safe and scalable care,and access to countermeasures?What are the identified gaps?What logistical and financial resources are available to your emergency workforce to perform theirtasks(such as office space,information technology,vehicles,funding)?What are the identified gaps?What is the operational and tactical health emergency coordination platform to activate andmobilize the necessary workforce for health emergencies,including partners(PHEOC,orequivalent)?How does the health emergency coordination platform link up with the multi-sectoral governance and coordination mechanisms when required?What academic and professional education pathways,including continuous and interdisciplinary learning pathways are available for the workforce involved in health emergencies?How active is your country in regional and global health emergency networks?Global health emergency corps framework30At the regional and global levels,GHEC represents the ecosystem in which countries and health emergency actors collaborate more efficiently through strengthened connections among health emergency networks and collaborations across national technical leaders.Recognizing the complexity of a constantly evolving global health emergency landscape,the interacting components are best defined through a systems approach that identifies the principal actors and their relationships.The GHEC ecosystem is defined by the health emergency response actors involved in regional and global collaboration on health emergencies through connected health emergency networks.A defining feature of GHEC is its guiding principles andvalues(see section 6).Therefore,health emergency response actors that are considered part of the GHEC ecosystem are those who recognize and commit to the guiding principles of sovereignty,equity,and solidarity as well as the core values of(i)quality and professionalism,(ii)predictability and efficiency,and(iii)trust and acceptance in health emergency management.Being part of the GHEC therefore implies for any regional and global health emergency actor and network a commitment to enhance the coherence and efficiency of collaboration and coordination of the health emergency workforce across the five Cs(HEPR core capability areas).Globally,the following health emergency networks have demonstrated their alignment with and commitment to the Global Health Emergency Corps as they strengthen workforce capacities and facilitate coordination between countries and health emergency actors at alllevels of the GHEC pyramid:Global Outbreak Alert and Response Network(GOARN)Emergency Medical Teams(EMT)Initiative Public Health Emergency Operations Centres Network(EOC-NET)Global Health Cluster Stand by Partnership programme International Association of National Public Health Institutes(IANPHI)Training Programs in Epidemiology and Public Health Interventions Network(TEPHINET)Many of these networks have regional chapters which add to regional mechanisms and networks dedicated to supporting countries in their health emergency workforce capacities.A selection of specific regional mechanisms and networks includes:The Pan American Health Organizations Regional Response Team The WHO-Africa Centres for Disease Control and Prevention(Africa CDC)joint initiative of African Volunteer Health Corps&Strengthening the Utilization of Response Groups for Emergencies(AVoHC-SURGE)The African health emergency preparedness andresponse leaders network The African Field Epidemiology Network(AFENET)Corps of Disease Detectives(ACoDD)The European Union Health Task Force See below for a more detailed description of each of the networks,the types of collaboration modalities they support,and how they can be activated in response to emergencies in support of the response at country level.The GHEC approach is designed to provide coherence,consistency,and efficiency by structuring collaborations across the three levels of the GHEC pyramid(emergency workforce,surge capacities,connected leaders).Many networks situate themselves in the surge capacity and emergency workforce levels,demonstrating the need forcross-network connections to ensure consistency andavoid duplication.The GHEC ecosystem also aims to strengthen and expand networks of technical leaders.The COVID-19 pandemic demonstrated a lack of connection between the most senior technical leaders who work within each countrys highest level of health emergency coordination structure to influence key decision making.Such networks exist either in ad hoc forms(Box 12)or at sub-regional levels,with increasing levels of networking also needed among senior technical leaders from countries indifferent regions.10.GHEC,a connected ecosystem 31Box 12Sub-regional Leaders Network the Regional Ebola Task Force of2022As an outbreak of haemorrhagic fever caused by Sudan ebolavirus unfolded in Uganda in September of 2022,a sub-regional leaders network was rapidly convened and activated.This regional task force,convened by the WHO Regional Office for Africa and Africa CDC,was comprised of a top technical leader from each of 9 surrounding countries at highest risk from disease spread.Eligible leaders included the directors of the National Public Health Institute,the head of the national Public Health Emergency Operations Center,or other designated senior officials.The leaders rapidly agreed to principles and guidelines and monthly virtual meetings facilitated both information sharing as well as cross-border cooperation.The group disbanded after resolution of the outbreak but laid the groundwork for other such leaders networks in response to future epidemics.WHO is currently working with pathfinder countries to adapt the GHEC framework at country level and identify gaps for further investment.This includes supporting countries in adopting a systematic approach to building different types of rapid response capacities,including emergency medical teams and public health rapid response teams,as well as exploring the utility of generative artificial intelligence in scaling the roll outofthe GHEC framework.10.1.2.Investing in sustainability and interoperability of rapid response capacities In implementing the GHEC approach,WHO established a global level Technical Working Group with national experts from across health emergency networks bringing together expertise from different settings and regions and tasked with producing a common guidance and benchmarks for establishing national rapid response capacities whether these are public health rapid response teams,emergency medical teams or other types of deployable capacities.This guidance is expected to assist countries and partners supporting them in establishing their rapid response capacities in aconsistent and predictable manner.10.1.3.Enhancing timely information sharing and coordinating by establishing connected leaders networks WHO and partners have conducted a broad consultation process to understand,map and support existing or create new health emergency leaders networks at regional or sub-regional levels.The process has demonstrated that networking amongst senior technical leaders is very much valued and that networks of national technical health emergency leaders already exist or are in development at sub-regional and regional levels.See examples in boxes 12 and 19.Valuable as these networks are,they have mostly emerged or were created on an ad hoc basis,often not institutionally supported over time,therefore short-lived.10.1.Initial implementation oftheGHEC approach 10.1.1.Advocating for and supporting health emergency corps capacities at country level In line with its vision and guiding principles,the primary focus of the GHEC is to advocate for country ownership and support countries in the adaptation and adoption of the GHEC framework.With the Pandemic Fund having recognized the investments in the health emergency workforce as a core priority,WHO and partners have encouraged countries to use the GHEC framework in identifying their health emergency workforce and coordination capacities that require further investments.Dedicated assistance has been provided through the proposal development stage and will continue in the implementation phase,including by leveraging resources from partners best placed to support project implementation.10.GHEC,a connected ecosystem Global health emergency corps framework32GHEC leaders networks are envisioned to have the explicitly aim of building trusted working relationships among the participants over time,with deliberately planned convenings and procedures for connecting in times of emergencies.A key role globally is to develop connections between regional leaders networks.10.1.4.Fostering trust for more predictable collaboration through exercising The efficiency of collaboration and coordination is dependent on the level of trust between the different actors.Simulation exercises provide an opportunity to enhance this trust by interacting,practicing,and validating operational processes and ways of working in a safe environment.Countries are therefore encouraged to practice and validate the collaboration and coordination of their national health emergency corps on a regular basis.WHO is also working with interested countries and partners to run regional or global simulation exercises to practice the cross-country collaboration and coordination through the GHEC ecosystem.A first global exercise was held in April 2025.10.1.5.Enhancing coherence and connectedness of the GHEC ecosystem Recognizing the need for connecting regional and global response mechanisms and networks to maximize collaboration in building and strengthening capacities,WHO has established a global GHEC working group bringing together secretariats of the global networks,regional office focal points,and the relevant technical teams to facilitate information exchange,identify common priorities and agree on common activities in support of strengthening health emergency corps capacities at country level.This working group is set to evolve over time and,as needed,support regional equivalents.10.1.6.Activating the GHEC approach inresponse operationsThe GHEC approach should translate into increased efficiency in the information sharing and external surge support mechanisms on which countries can rely during health emergencies,including pandemics.Achieving this requires a broad awareness and understanding of the regional and global health emergency(leadership and surge)mechanisms and networks that exist and how they relate to and complement each other.In emergency response operations,these are brought together through the partnership pillar of the WHO-led Incident Management Support Teams.Box 13Activating the GHEC ecosystem formpox 2024An initial activation of this approach was implemented in the 2024 mpox response where WHO worked on three layers:(i)with IANPHI to understand the workforce gaps at national level through the National Public Health Institutes,(ii)with GOARN to map the different surge deployments to selected countries,and(iii)convened a call between technical leaders from affected countries and other countries to discuss the most effective control measures,share best practices and coordinate their efforts to halt theoutbreak.3310.GHEC,a connected ecosystem 10.2.Health emergency networks for surge and coordination in the GHEC ecosystem 10.2.1.Global Outbreak Alert and Response Network(GOARN)Description GOARN(26)is a network of more than 310 technical institutions and networks globally,supporting preparedness,operational readiness,and response.This includes capacity strengthening foroutbreak response,rapid information sharing and the deployment of staff and resources to affected countries.Coordinated by an Operational Support Team based at the WHO headquarters in Geneva and governed by a Steering committee,GOARN delivers rapid and effective support to prevent and control infectious disease outbreaks and public health emergencies when requested.Collaborations supported and role inthe GHEC ecosystemGOARN has strengthened collaborations across national outbreak response institutions since its inception in 2000.By facilitating strategic institutional groupings,formal and informal twinning of institutions,global and regional partner meetings,and collaborative projects,GOARN has helped to enhance the quality and disseminate standards for outbreak alert and response across key institutions from many countries.Box 14Building global surge deployment capacity through GOARN trainings Equipping responders with tools for deployment:In June 2024,WHO,GOARN and the Chinese Center for Disease Control and Prevention(China CDC)along with four other GOARN partners Guangdong Provincial Center for Disease Control and Prevention;National Institute for Infectious Diseases,Japan;Ministry of Health Labor andWelfare,Japan;and the Robert Koch Institute,Germany jointly delivered GOARNs Orientation to International Outbreak Response training module.This training,attended by 78 participants from 10 countries,focused on strengthening emergency response capacity.The goal was to equip participants with the tools needed for effective deployment in emergency settings,ensuring they are well-prepared torespond swiftly and efficiently.“I hope that more public health experts will have more opportunities to participate in GOARN trainings,so that we can be fully prepared to respond to any international public health events in the future.”Dr Yan Li Deputy Director,Guangdong Provincial Center for Disease Control and Prevention.“This partnership fosters a sense of global solidarity and shared responsibility in addressing challenges faced when responding to an outbreak in the field,helping to build trust among partners and facilitating smoother and more effective joint efforts inthe future.”Enhancing womens leadership skills:In September 2024,WHO and GOARN held their first leadership training for women,aimed atenhancing the emergency response leadership skills of women in outbreak response.Supported by the Australian government and hosted by the National Critical Care Trauma and Response Centr
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Guidance for best practices for clinical trialsGuidance for best practices for clinical trialsGuidance for best practices for clinical trialsISBN 978-92-4-009771-1(electronic version)ISBN 978-92-4-009772-8(print version)World Health Organization 2024Some rights reserved.This work is available under the Creative Commons Attribution-NonCommercial-ShareAlike 3.0IGO licence(CCBY-NC-SA3.0IGO;https:/creativecommons.org/licenses/by-nc-sa/3.0/igo).Under the terms of this licence,you may copy,redistribute and adapt the work for non-commercial purposes,provided the work is appropriately cited,as indicated below.In any use of this work,there should be no suggestion that WHO endorses any specific organization,products or services.The use of the WHO logo is not permitted.If you adapt the work,then you must license your work under the same or equivalent Creative Commons licence.If you create a translation of this work,you should add the following disclaimer along with the suggested citation:“This translation was not created by the World Health Organization(WHO).WHO is not responsible for the content or accuracy of this translation.The original English edition shall be the binding and authentic edition”.Any mediation relating to disputes arising under the licence shall be conducted in accordance with the mediation rules of the World Intellectual Property Organization(http:/www.wipo.int/amc/en/mediation/rules/).Suggested citation.Guidance for best practices for clinical trials.Geneva:World Health Organization;2024.Licence:CCBY-NC-SA3.0IGO.Cataloguing-in-Publication(CIP)data.CIP data are available at https:/iris.who.int/.Sales,rights and licensing.To purchase WHO publications,see https:/www.who.int/publications/book-orders.To submit requests for commercial use and queries on rights and licensing,see https:/www.who.int/copyright.Third-party materials.If you wish to reuse material from this work that is attributed to a third party,such as tables,figures or images,it is your responsibility to determine whether permission is needed for that reuse and to obtain permission from the copyright holder.The risk of claims resulting from infringement of any third-party-owned component in the work rests solely with the user.General disclaimers.The designations employed and the presentation of the material in this publication do not imply the expression of any opinion whatsoever on the part of WHO concerning the legal status of any country,territory,city or area or of its authorities,or concerning the delimitation of its frontiers or boundaries.Dotted and dashed lines on maps represent approximate border lines for which there may not yet be full agreement.The mention of specific companies or of certain manufacturers products does not imply that they are endorsed or recommended by WHO in preference to others of a similar nature that are not mentioned.Errors and omissions excepted,the names of proprietary products are distinguished by initial capital letters.All reasonable precautions have been taken by WHO to verify the information contained in this publication.However,the published material is being distributed without warranty of any kind,either expressed or implied.The responsibility for the interpretation and use of the material lies with the reader.In no event shall WHO be liable for damages arising from its use.iiiContents Foreword ivAcknowledgements viAbbreviations viiiExecutive summary x1.Introduction 11.1 Clinical research:importance and types 21.2 Ascertainment of treatment effects:observational studies vs clinical trials 31.3 The clinical trials environment:an evolving landscape 41.4 Persistent challenges to clinical trial enablement 51.5 Steps required to improve evidence generation 82.Key scientific and ethical considerations for clinical trials 112.1 Good clinical trials are designed to produce scientifically sound answers to relevant questions 122.2 Good clinical trials respect the rights and well-being of participants 202.3 Good clinical trials are collaborative and transparent 232.4 Good clinical trials are designed to be feasible for their context 242.5 Good clinical trials manage quality effectively and efficiently 253.Guidance on strengthening the clinical trial ecosystem 273.1 Clinical trial ecosystem pillars 283.2 Clinical trial ecosystem cross-cutting themes 374.Conclusion 41Annexes 43Annex 1.Provisions for rapid funding and approval of good randomized evidence generation in emergencies 44Annex 2.Recommendations for Member States,research funders and researchers 46References 54ivForewordA core role of World Health Organization(WHO)is to support strengthening of national capabilities in the health sector.WHO considers that strengthening of country-led research and development(R&D)ecosystems to advance health science and facilitate faster and more equitable access to safe and effective health interventions is of the utmost importance to a countrys population health and economic well-being.Clinical trials are an essential component of a strong country-driven R&D ecosystem.Unnecessary bureaucracy,uncoordinated approval processes and the lack of an enabling environment are currently barriers in some countries and as a result slow down and prevent equitable access for people to health innovation that can save and transform lives.In 2022 the World Health Assembly adopted resolution(WHA 75.8)“Strengthening clinical trials to improve high quality evidence on health interventions and to improve research quality and coordination”,which called on WHO to develop this guidance.Throughout the guidance a major focus is to address public health priorities through clinical and public health research,and in particular to address the health needs of developing countries in an equitable manner.Importantly,enhancing clinical trial capacity is essential for all countries with many efficiency gains possible in high-income countries as well as middle-and low-income countries.Therefore,the reforms called for can have a major impact worldwide.This guidance has a number of recommendations.Firstly,patient,participant and community engagement are placed centrally in the trial planning and implementation phases to ensure the research meets public needs and maintains trust.Secondly,major new recommendations are included on reforms that enable trials in underrepresented populations such as children,pregnant women and older adults.Thirdly,the guidance lays out how to focus trial design and oversight on the key scientific and ethical considerations that determine whether trials are ethical,efficient and informative.Here risk based and proportionate approaches are advocated so that we move away from one size fits all oversight or audit,to those that are tailored to risk.For the first time in WHO guidance,recommendations are provided that can practically assist national health authorities,regulatory authorities,funders and others in how best they can facilitate clinical trials and research to enable evidence generation on health interventions.Sustained domestic support and resources are the only way to finance this transformation.Longstanding recommendations,also referred to in previous World Health Assembly resolutions,on a minimum spending of 2%of health budgets on Science and R&D,and 5%of health-related development assistance on research have not been met by many countries,and in other countries resources are needed for reform rather than supporting uncoordinated processes.Countries that choose to prioritize and resource a reformed clinical research ecosystem,enabling the work of their clinical researchers with input from the public,private sector and local communities,will gain major benefits including:Improved trust between the public and the health research community Better locally derived evidence for clinical practitioners and public health decisions Improved health outcomes,faster and more equitable access to innovation and medical products that are better tailored to individual patient needs,and hence progress towards health-related sustainable development goals(SDGs)vForeword National resilience and security including a more robust clinical trial ecosystem ensuring faster response to health crisis Healthier populations and more productive economies Economic benefits from a thriving science and innovation ecosystem that provides opportunities for jobs,spin outs into new small and medium sized companies along with private sector investmentIn coordination and collaboration with its partners,WHO is committed to provide support to countries that wish to apply this guidance to reform,improve and streamline their oversight and approval processes and hence strengthen their clinical research system.Jeremy FarrarChief ScientistWorld Health OrganizationviAcknowledgementsThe WHO Secretariat thanks all Member States for their guidance and direction and gratefully acknowledges the valuable inputs of many individuals and partners throughout the development of this document.The Secretariat conducted stakeholder consultations on the most relevant existing guidance documents for best practices regarding clinical trials.Permission from Council of International Organizations of Medical Sciences(CIOMS)(1)and the Good Clinical Trials Collaborative(GCTC)(2)for incorporation or adaptation of their guidance is gratefully acknowledged.The development of the guidance in the WHO secretariat was overseen by Jeremy Farrar,Chief Scientist,and John Reeder,Director of the Department of Research for Health,Science Division.Vasee Moorthy,Senior Advisor in the Department of Research for Health,led the overall development process with invaluable support from Wei Zhang,Technical Officer,Department of Research for Health.The Technical Advisory Group(TAG)for Development of Best Practices for Clinical Trials was constituted through a public call for nominations.The TAG members are Akbar Fotouhi(Tehran University of Medical Sciences,Iran(Islamic Republic of),Evelyn Gitau(African Population and Health Research Center,Ethiopia),Herman Goossens(University of Antwerp,Belgium),Marian Knight(University of Oxford,United Kingdom of Great Britain and Northern Ireland),Roli Mathur(Indian Council of Medical Research,India),Ann Meeker-OConnell(Independent expert,United States of America),Sharon Nachman(Stony Brook Childrens Hospital,United States of America),John Norrie(University of Edinburgh,United Kingdom of Great Britain and Northern Ireland),Thomas Nyirenda(European and Developing Countries Clinical Trials Partnership,South Africa),CS Pramesh(Tata Memorial Centre,India),Fiona Russell(University of Melbourne,Australia),Sofia P.Salas(Universidad del Desarrollo,Chile),Karla Soares-Weiser(Cochrane,United Kingdom of Great Britain and Northern Ireland),Fergus Sweeney(Independent expert,Ireland),Huixia Yang(Peking University First Hospital,China),and Nonhlanhla Yende-Zuma(Centre for the AIDS Programme of Research,South Africa).They provided essential technical advice throughout.Advice was also received from An-Wen Chan,Chair of the International Clinical Trials Registry Platform(ICTRP)advisory group.WHO acknowledges the significant contribution of Christina Reith,Associate Professor at the Nuffield Department of Population Health,University of Oxford,United Kingdom,for her extensive senior technical writing support during the drafting and consolidation stages,which played a pivotal role in the guidance development.WHO also recognizes the valuable technical input and critical review provided by colleagues from regional offices and headquarters,including WHO Regional Office for Africa:Joseph Chukwudi Okeibunor;WHO Regional Office for Americas:Luis Gabriel Cuervo Amore,Ludovic Reveiz,Carla Saenz;WHO Regional Office for the Eastern Mediterranean:Arshad Altaf and Arash Rashidian;WHO Regional Office for Europe:Marge Reinap;WHO Regional Office for South-East Asia:Manju Rani;WHO Regional Office for Western Pacific:Mengji Chen and Kidong Park;WHO Health Emergencies Programme:Janet Diaz,Nina Gobat,Ana Maria Henao Restrepo,Jamie Rylance and Steven Mcgloughlin;Digital Health and Innovation:Alain Labrique;Gender,Rights and Equity-Diversity,Equity and Inclusion:Shirin Heidari;Global HIV,Hepatitis and STIs Programmes:Nathan Ford;Global Malaria Programme:Lindsey Wu;Global TB Programme:Francesca Conradie,Fuad Mirzayey,Samuel Schumacher and Matteo Zignol;Health Emergency Intelligence and Surveillance Systems:Chikwe Ihekweazu;Immunization,Vaccines and Biologicals:Joachim Maria Hombach;Maternal,Newborn,Child and Adolescent Health and Ageing:Nigel Rollins;Mental Health and Substance Use:Rodrigo Cataldi;Quality Assurance of Norms and Standards:Lisa Askie;Regulation and Prequalification:Samvel Azatyan,Marion Laumonier,Hiiti Sillo and Marie Valentin;Research for Health:Tanja Kuchenmueller,Katherine Littler,Ghassan Karam,Martina Penazzato,viiAcknowledgementsAndreas Reis and Anna Laura Ross;Sexual and Reproductive Health and Research:Avni Amin,Mercedes Bonet Semenas and Mariana Widmer;Special Programme for Research&Training in Tropical Diseases:Garry Aslanyan,Anna Thorson and Mahnaz Vahedi.WHO express gratitude to the GCTC and the CIOMS for close collaboration during the writing of this document,as well as to the WHO Collaborating Centre for research information sharing,e-learning,and capacity development,based in the Centre for Tropical Medicine and Global Health,Nuffield Department of Clinical Medicine,University of Oxford,United Kingdom of Great Britain and Northern Ireland,for their technical support in conducting the online global stakeholder survey on barriers and priority actions for strengthening clinical trials ecosystems as part of the guidance development.Thanks are also extended to the many hundreds of participants who contributed their valuable expertise and insights through the online public consultation and in-person regional and global consultations throughout 2023 and early 2024.Although it is not possible to name all here,without their support and expertise this guidance would not have been possible.Inputs were received from leading research funders,national health authorities,national ethics authorities,national regulatory authorities and civil society and nongovernmental organizations.The development of this guidance was made possible through funding from the EDCTP2 programme,supported by the EU(European Union(grant number CSA2023WHO-3454-WHORCT),with funding from the National Institute for Health and Care Research(NIHR)in the United Kingdom(where NIHR is funded by the Department of Health and Social Care).The NIHR Global Health Research portfolio supports high-quality applied health research for the direct and primary benefit of people in low-and middle-income countries,using international development funding aid from the Government of the United Kingdom,to support global health research.The views expressed in this publication are not necessarily those of EDCTP,NIHR or the Department of Health and Social Care in the United Kingdom.viiiAbbreviationsAIArtificial IntelligenceAIDS Acquired Immune Deficiency SyndromeAVAREF African Vaccine Regulatory ForumCDC Centers for Disease Control and PreventionCDISC Clinical Data Interchange Standards ConsortiumCIOMSCouncil for International Organizations of Medical SciencesCOMETCore Outcome Measures in Effectiveness TrialsCONSORTConsolidated Standards of Reporting TrialsCOVIDcoronavirus disease CTTI Clinical Trials Transformation InitiativeCTUclinical trial unitDMCdata management committeeECethics committeeEDCTPEuropean&Developing Countries Clinical Trials PartnershipEEAEuropean Economic AreaEMAEuropean Medicines AgencyESSENCEEnhancing Support for Strengthening the Effectiveness of National Capacity EffortsFDAFood and Drug AdministrationGCTCGood Clinical Trials CollaborativeGLOPIDGlobal Research Collaboration for Infectious Disease PreparednessHIChigh-income countriesHIV human immunodeficiency virus ICHInternational Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human UseICHOMInternational Consortium for Health Outcomes Measurement ICTRPInternational Clinical Trial Registration PlatformIRASIntegrated Research Application SystemixList of acronyms IRBInstitutional Review BoardJPIAMRJoint Programming Initiative on Antimicrobial ResistanceLMIClow-and middle-income countriesMHRAMedicines and Healthcare products Regulatory AgencyNCANational Competent AuthorityNITAGNational Immunization Technical Advisory Groups NetworkNRANational Regulatory AuthorityPAHOPan American Health OrganizationPRISMAPreferred Reporting Items for Systematic Reviews and Meta-AnalysesPROMsPatient Reported Outcomes MeasuresRCTrandomized clinical trial R&Dresearch and developmentRECresearch ethics committeeSAGERSex and Gender Equity in ResearchSCTIStandardized Data Collection for Cardiovascular Trials InitiativeSDTM Study Data Tabulation ModelSPIRITStandard Protocol Items:Recommendations for Interventional TrialsTBtuberculosisTDRUNICEF/UNDP/World Bank/WHO Special Programme for Research and Training in Tropical DiseasesUKCDRUK Collaborative on Development ResearchWHO World Health Organization xExecutive summaryObjectiveThis document responds to requests by the World Health Assembly to the Director-General in resolution WHA75.8(2022)on strengthening clinical trials to provide high-quality evidence on health interventions and to improve research quality and coordination(3)to identify and propose best practices and other measures to strengthen the global clinical trial ecosystem and to review existing guidance and develop new guidance as needed on best practices for clinical trials.This guidance updates and adapts the previous work of the World Health Organization(WHO)on research capacity(4)for the context of well-designed and well-implemented clinical trials as framed in resolution WHA75.8(2022).It aims to enhance clinical research efficiency,minimize research waste and provide guidance on sustained clinical trials that are always functional and active for endemic conditions and can pivot in time of emergency or pandemics.Section 1 provides an introduction.For key scientific and ethical considerations for well-designed and well-implemented trials the reader should refer directly to Section 2.For guidance on strengthening the clinical trial ecosystem,including capacity development and addressing inefficiencies,refer to Section 3 and for recommendations to Member States,research funders and researchers refer to Annex 2.ScopeThis document is intended to provide guidance to WHOs Member States and any staff members of non-State actor organizations whose work is related to clinical trials in any way,including the planning,conduct,analysis,oversight,interpretation and funding of all clinical trials to assess the effects of any health intervention for any purpose in any setting.Such staff members include those involved in educating others about clinical trials.The remit includes:any design for a clinical trial:but with a focus on randomized clinical trials,including comparisons of two or more interventions,whether blinded or not,and whether parallel,cluster,crossover,factorial,adaptive platform,decentralized or other design;any health intervention:including(but not limited to)administration of pharmaceutical medicines,cells and other biological products,and vaccines;surgical or radiological procedures;diagnostics;use of medical devices,nutritional measures;cognitive,behavioural and psychological interventions;supportive or preventive care,including process-of-care changes;physical therapy interventions;digital and public health approaches;traditional or herbal measures;and screening processes.The interventions may be novel or pre-existing but being used in a different way(for example,repurposed or optimized)or to gain further knowledge about current practices;any purpose:including(but not limited to)evidence for guideline development;recommendations for clinical practice or public health strategies;and health technology assessments;xiExecutive summary any setting:any geographical,economic or societal context,and any context including clinical trials based in hospital,primary care or community settings;or where the intervention is delivered directly to a participant;any role:including researchers and clinicians,patient and public groups(including trial participants),regulators and other national health authorities,ethics committees and institutional review boards,research funders,and all trial sponsors(academic,government,nonprofit and commercial).There will often be important local,national or regional contextual factors or regulations that are crucial to consider,and national bodies working with local patient groups and affected communities are best placed to ensure appropriate local adaptation of this guidance and compliance with universal scientific and ethical standards.This document aims to complement other guidance in order to support implementation of universal ethical and scientific standards in the context of clinical trials,with a focus on under-represented populations;it does not represent a legal standard and does not supersede any existing guidance.In particular,this guidance shares many common concepts and principles with guidance produced by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use(ICH)(5),especially the ICH E8(R1)General Considerations for Clinical Studies guideline(6),(the draft ICH E6(R3)Good Clinical Practice guideline(7),and the ICH E9 statistical principles guideline(8)and its associated addendum(9).In addition,it shares attributes with two further recent guidance documents that were highlighted through WHOs public consultation process in 2022:those of the Council for International Organizations of Medical Sciences(CIOMS)on clinical research in resource-limited settings(10)and the Good Clinical Trials Collaborative(GCTC)(11).Both the CIOMS and GCTC guidance have served as sources,with adaptations as needed,for this document.Additional sources highlighted through the consultation include the World Medical Associations(WMA)Declaration of Helsinki(12)on medical research involving human subjects,the WMA Declaration of Taipei on Ethical Considerations regarding Health Databases and Biobanks(13)and CIOMS International Ethical Guidelines on Health-related Research involving Humans(2016)(14).For clinical trials designed to support submission to regulatory authorities concerned with medicinal products,trial sponsors should also refer to the ICH guidelines,in particular ICH E8(R1)(6)and ICH E6(R3)(7)and other relevant ICH guidelines,along with any relevant guidance issued by the authorities to which they plan to submit.As noted above,the scope of this WHO guidance is restricted to neither medicinal products nor clinical trials conducted in support of regulatory approval.Approach to developmentIn March 2023,under the guidance of the WHO Technical Advisory Group(TAG)for the Development of Best Practices for Clinical Trials,the initial guidance was drafted by Vasee Moorthy and Christina Reith,drawing from existing CIOMS and GCTC guidance.The TAG provided feedback on this draft in writing and during a teleconference in May 2023.This feedback was incorporated into a revised draft,which was published on the WHO website for public consultation from July to September 2023.The WHO Secretariat disseminated the public consultation for the draft guidance to regional offices,relevant headquarters technical programs,professional networks,non-state actors in official relations with the WHO,and other key stakeholders in clinical research.A total of 179 responses from 48 countries were received,with approximately 30%from academic stakeholders,followed by non-governmental organizations and national health or regulatory authorities.Additionally,the WHO Secretariat organized a consultation with private sector Guidance for best practices for clinical trialsxiiGuidance for best practices for clinical trialsrepresentatives at a side event during the 76th World Health Assembly and held an information session to gather feedback from Member States in September 2023.A global stakeholder survey was launched in August 2023,in collaboration with the WHO Collaborating Centre for research information sharing,e-learning,and capacity development,to identify barriers in conducting clinical trials and propose priority actions.Nearly 3000 participants worldwide responded to the survey.Outcomes from the global stakeholder survey were further discussed in in-person consultations held in Brasilia,Brazil;Lusaka,Zambia;Delhi,India;Cairo,Egypt;Kuala Lumpur,Malaysia;and Geneva,Switzerland,attended by about 300 experts and stakeholders.These consultations provided additional input for the draft guidance on strengthening the clinical trial ecosystem.The final guidance,prepared by Vasee Moorthy and Christina Reith,integrated all received feedback.In April 2023,a TAG meeting was held to review the final draft before its submission for executive clearance and publication.Declarations of interest were collected from all members of the WHO Technical Advisory Group(TAG)that oversaw the development of the guidance and any relevant interests are publicly disclosed on the website for the TAG.1.IntroductionCommunity health services for parents and children in Gyabankrom,Central Region,Ghana.WHO/Fanjan Combrink21.1 Clinical research:importance and typesClinical research is indispensable for resolving public health challenges.Clinical research studies can be thought of as spanning five generic areas of activity:measuring the magnitude and distribution of the health problem;understanding the diverse causes or the determinants of the problem,whether they are due to biological,behavioural,social or environmental factors;developing solutions or interventions that will help to prevent,mitigate or cure the problem;implementing or delivering solutions through policies and programmes;and evaluating the impact of these solutions on the level and distribution of the problem.Clinical studies broadly fall into two groups:non-interventional and interventional.Non-interventional studies are observational in nature(hence sometimes known as observational studies),in which health outcomes are typically compared between individuals who received or were exposed to a certain factor and those who did not,and in which the allocation to treatment or exposure is not predetermined by a study protocol.In contrast,interventional clinical studies(known as clinical trials)evaluate the effects of prospectively assigning subjects to one or more interventions on health outcomes.For comparisons of two or more interventions,a key aspect of such prospective assignment is the process of randomization to help 1.Introductionto ensure that the efficacy and safety of treatments are assessed reliably;the key importance of why this matters is discussed in Section 1.2 and Section2.Such clinical trials are known as randomized clinical trials(RCTs),with the intervention to which a participant is allocated sometimes referred to as an“arm”of a clinical trial.RCTs may involve prospective allocation of individuals to interventions or prospective allocation of a group of people(for example,in a particular community,school or region),and are sometimes known as cluster RCTs.There are,however,some circumstances where prospective assignment necessarily does not support randomization,such as clinical trials very early in an interventions development,or in some oncological,rare disease and diagnostics trials whereby only one intervention is tested(that is,“single arm”trials).Clinical trial interventions may include(but are not limited to)administration of pharmaceutical medicines,cells and other biological products,and vaccines;surgical or radiological procedures;diagnostics;use of medical devices,nutritional measures;cognitive,behavioural and psychological interventions;supportive or preventive care,including process-of-care changes;physical therapy interventions;digital and public health approaches;traditional or herbal measures;and screening processes.The interventions may be novel or pre-existing but being used in a different way(for example,repurposed or optimized)or to gain further knowledge about current practice.In RCTs,interventions may include placebo or another comparator(sometimes known as a control)and may provide no additional active intervention beyond usual practice or standard care.Clinical trials may be carried out at any level of the health system,from home,community or primary level care through to secondary,tertiary or intensive care settings.31.IntroductionIn addition to traditional parallel-group RCTs,a range of further trial designs exists,including(but not limited to)crossover,factorial,adaptive and platform trials.In addition,the variety of options for performing any of these trial designs is wide,depending on the nature of the trial.These can include decentralized trials,point of care trials and more traditional investigator location-based trials,or,more commonly,combinations of these elements in a single trial.Platform,basket and umbrella trials all use master protocols(1518)which allow simultaneous evaluation of multiple interventions within the same overall trial structure.Platform trials are designed to study multiple interventions among people with one or more closely related diseases(for example,cancers due to genomic subtypes)or health conditions(for example,pneumonia).They may use a common control group(for example,treatment A vs treatment B vs a common control)or,more efficiently,a factorial design that involves more than one randomized comparison(for example,treatment A vs placebo A and treatment B vs placebo B)such that some participants may get more than one active treatment while a minority receives a placebo.They are efficient and flexible,allowing for modification of the ongoing trial in the light of accumulating trial data,with new research questions being introduced as amendments rather than as new trials.For example,arms can be added to test new interventions once initial questions have been addressed,while existing arms may be discontinued if it becomes apparent that an intervention is ineffective or harmful based on predefined decision algorithms.Such platform trials can be open-ended and with intervention arms added at different points in time.In particular,the emergence of large adaptive platform trials with pragmatic features embedded into health systems was pivotal in generating evidence for use of therapeutics in coronavirus disease(COVID-19).Increasingly,trials also use streamlined“decentralized”approaches(19)(where some aspects are delivered in or close to peoples homes)or point-of-care designs(where a trial is conducted in clinical practice settings).Such trials can address critical questions in clinical care settings rather than in specialized research environments.(20)All clinical trials should help to resolve important uncertainties about the effects of health interventions.Depending on the context,the results may be needed to determine whether to proceed with development,further evaluation of the intervention or inform regulatory licensing,clinical guidelines and/or health policy.In each case,any uncertainties applying to the specific question(s)that remain at the end of the clinical trial should be sufficiently small to allow meaningful decisions to be made.1.2 Ascertainment of treatment effects:observational studies vs clinical trialsObservational studies and clinical trials are both highly valuable in clinical research and may be complementary.However,they must be designed and analysed appropriately,and used in the right context(2124).Robust observational studies can be extremely useful for identifying associations of risk factors with disease(with good examples being smoking with lung cancer,and blood pressure and cholesterol with cardiovascular disease),but their value for the assessment of the effects of treatment is more limited.Observational studies may also have an important role in the identification of large effects(adverse or beneficial)of an intervention on rare health outcomes that would not normally be expected to occur,particularly those that are not likely to be related to the indications for(or contraindications to)the intervention of interest.A major limitation of observational studies is their inherent potential biases.One of the most important of these is confounding,in which a factor is associated with an exposure of interest(but is not a direct consequence of it)and,independently,influences the risk of the outcome of interest.For example,“confounding by indication(or contraindication)”may occur when a treatment tends to be provided more(or less)frequently to individuals with conditions associated with increased or decreased risks of the outcome of interest.This type of bias can produce misleading estimates not just of the size but also of the direction of treatment effects,and these can remain even after statistical adjustment for observed differences between different groups Guidance for best practices for clinical trials4Guidance for best practices for clinical trialsof individuals.Equally,biases can arise owing to differences in ascertainment or detection of an outcome.In addition,the reliability of recall of treatment exposure can differ between those who develop a certain outcome and those who do not.These potential biases mean that observational studies can be unreliable for determining the effects of health interventions,especially when(as can often be the case)the effects of the treatment of interest are only moderate or null(25).Discussion of design considerations and methods to avoid bias and confounding in observational studies is beyond the scope of this guideline.However,this limitation is highly relevant because most interventions for most common serious conditions have only modest effects on health and disease,even if they have a large effect on intermediate features(for example,physiological or laboratory tests).However,even modest improvements in health can be important,particularly if the intervention can be used widely for a common condition or if multiple interventions with moderate effects can be used in combination,provided any benefits are not substantially offset by detrimental effects.Therefore,it is vital that these modest effects are detected reliably.This requires clinical trials that ensure stringent control over systematic errors such as biases and confounding factors(which,in general,requires proper methods in randomization,blinding and masking,as well as appropriate statistical analysis)and strict control of random error(which necessitates appropriate sample sizes).RCTs therefore have a central role in generating the evidence needed to inform the development and implementation of health interventions,because they can reliably determine whether a health intervention is safe and effective by ensuring that any biases or random errors inherent in the study design are small with respect to the expected treatment effect.The results of such RCTs and their associated meta-analyses(whereby data from multiple clinical trials addressing a similar research question are statistically combined)(21)have been transformative in advancing global public health.1.3 The clinical trials environment:an evolving landscapeThe clinical trial environment has evolved substantially since the concepts of clinical trials were introduced,with important changes having also taken place in the social,ethical and regulatory environment globally.There is now a broader recognition of the very large health,social and economic returns on investments in research.Clinical trials and the development of interventions are being supported by industry,non-industry parties(such as academic institutions),government agencies and publicprivate partnerships,sometimes with support from external partners in translational research.In terms of ethical principles there have been revisions of the Declaration of Helsinki(12)and development of guidance on clinical trials,notably the CIOMS international ethical guidelines(14).In terms of regulatory guidelines,ICH clinical trial guidelines are available,in particular ICH E8(R1)(6)and ICH E6(R3)(7).There is also recent guidance from the Good Clinical Trials Collaborative(11),as well as the creation of new or updated guidance or pathways being developed by regulators.Also,interest has grown in trials methodology,including increasing use of flexible and practical approaches to trial design,with a growing recognition of how routinely collected data,sometimes known as real world data,can add value and drive efficiency of clinical trials.For example,data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources(such as electronic health records,medical claims data,data from product or disease registries,and data gathered through digital health technologies)can be used to help with the enrolment and outcome ascertainment in trials(2629).Patient organizations and advocacy groups have come to the fore globally in recent years,advocating patient,community and public involvement in clinical trials as a cornerstone in the design and conduct of clinical trials.However,more remains to be done to ensure that trial design and implementation include adequate engagement with patients,communities and the public.This guidance includes elements on 51.Introductionsuch engagement that will not only help to ensure that clinical trials are relevant to the populations they are intended to serve,but also raise awareness of the role of clinical research in public health and the quality of life.Guidance and initiatives for patient involvement and good participatory practice in clinical trials have been developed,by WHO and others,available across a range of interventions and settings(3038).The way in which information is shared and communicated is also rapidly evolving.This provides valuable opportunities for more efficient,collaborative and transparent trial processes,but also presents potential risks in terms of a growing potential for global propagation of misinformation or“fake news”which is detrimental to public health.Well-designed RCTs and the maintenance and promotion of clear,valid sources for reliable information on their design and results are a strong defence against misinformation.In areas of medicine where clinical trials are common,such as oncology,cardiovascular disease and some infectious diseases,patient outcomes have markedly improved as interventions and service delivery have been iteratively enhanced.While the focus of this guidance is on later-stage RCTs that evaluate safety and effectiveness of interventions,earlier-stage translational research is another valuable area that has emerged as central to advancing health outcomes,by acting as the bridge between basic science and later-stage evaluations.Applications of artificial intelligence(AI)in the clinical trials arena were advancing rapidly at the time of finalizing this guidance,including but not restricted to drug and vaccine discovery and molecule design,AI-enhanced diagnostic approaches,predictive modelling of trial outcome intended to improve trial design,participant recruitment and retention and digitization.All these factors have significantly changed the environment for clinical trials.However,the research landscape must continue to evolve to fulfil its potential.1.4 Persistent challenges to clinical trial enablementThere is an urgent need to avoid wasteful procedures and make clinical trials more efficient so that they can be done on an adequate scale to produce reliable evidence at reasonable costs.This is because,despite the widely recognized importance of clinical trials,in many areas of health the evidence base remains weak,with decision-making processes lacking results from enough well-designed and well-conducted clinical trials.This problem is global,affecting high-,middle-and low-income countries.The result can be failure to identify and use effective and safe interventions or the continuing use of ineffective or hazardous interventions;for example,millions of doses of ineffective treatments were used during the COVID-19 pandemic.As a consequence,resources are wasted through both direct immediate costs and indirect downstream costs,unnecessary harm or suffering may be caused,and trust is reduced in those who develop or use health interventions.The need to reduce research waste is a long-recognized global issue affecting clinical trials across a spectrum of settings,with urgency to address this problem having been the focus of much discussion.However,it was particularly highlighted by the research response to the pandemic of COVID-19:more than 22000 COVID-19-related clinical trials were registered,of which the vast majority are thought to have contributed little to the evidence base.A small proportion of such clinical trials(whether publicly or non-publicly funded),probably less than 10%,were well-designed and well-implemented(with a widespread problem being that many such clinical trials were not randomized and/or sufficiently large to answer their intended question)and contributed meaningfully to policy recommendations by WHO and other bodies.This waste in clinical trials results from various factors.These include clinical trials never being done or completed,failure to articulate clear research questions,duplication of previous research,use of inefficient trial processes,failure to produce scientifically robust and clinically relevant answers,or results never being published.The lost opportunity cost of trials that are poorly designed or not completed and reported is significant and results in draining of available resources which Guidance for best practices for clinical trials6Guidance for best practices for clinical trialsare not then available to conduct and complete well-designed trials.Additionally,a prevailing risk-averse mentality hampers innovation and the adoption of new perspectives,leading to disproportionately burdensome trial processes and data collection practices.The absence of efficient and coordinated procedures for approving clinical trials poses a significant challenge,characterized by vast intercountry and inter-regional heterogeneity in approval processes by regulatory and ethics authorities.Some countries have mature systems,but these may still have significant inefficiencies and over-utilise risk-averse approaches.If approval processes are unduly lengthy,enthusiasm and the ability to enrol large numbers in local populations may wane.Such delays can lead to a reduction in evidence generated for exactly the types of people such authorities are trying to benefit.Many countries also lack the necessary resources for a robust infrastructure or have not yet achieved adequate efficiency.The multitude of applications with various processes and lengthy timelines results in delayed initiation of trials and may lead to a loss of motivation to engage in clinical research.This issue intensifies for multiregional or international trials,important for achieving both statistical power and broad representativeness.Intermittent use of clinical trial infrastructure also specifically needs to be addressed to avoid periodic fallow or“cold”periods in clinical trial activity,primarily driven by project-based research and intermittent clinical trial funding.This problem results in inefficiencies,skill loss and neglect of key areas in the clinical trial ecosystem.A lack of adequate funding for clinical trials remains a major issue globally with ongoing disparities in investment and access to clinical trial infrastructure especially when taken in context of global disease burden(39).The result is inequity and a lack of justice in fairly accessing affordable,safe and efficacious interventions,the consequences of which were particularly highlighted during the COVID-19 pandemic.This state of affairs is particularly prominent in resource-limited settings and areas where the traditional model of development of interventions does not provide incentives for research and development(R&D),with trials undertaken in high-income countries(HICs)historically dominating,focusing on diseases prevalent in those settings.The result has been an increasingly conducive clinical trials environment,infrastructure and capacity to address the health priorities of HICs and commercial market interests.In contrast,the limited health care and research capacity and/or commercial viability in low-and middle-income countries(LMICs)1 means that clinical research in these regions has often focused on observational or implementation studies conducted after the registration or approval of an intervention in HICs.However,populations in(LMICs)bear the highest burden of preventable disease globally,facing several specific challenges.First,they continue to face a high level of communicable diseases,such as neonatal sepsis,malaria,tuberculosis,chronic hepatitis B and C,HIV infection/AIDS,diarrhoeal diseases and neglected tropical diseases,and in some areas are being seriously impacted by epidemic outbreaks of diseases,which affect different regions in different ways.In 2021,children up to 14 years of age accounted for 25%of the global population and 42%of the population in low-income countries(40).Secondly,neonatal,maternal and nutritional diseases are prevalent,and neonatal,under-5 and maternal mortality is high in LMICs,although declining.In addition,LMICs have similar rates of noncommunicable diseases to those in upper-middle-and high-income countries but lack adequate clinical trial focus outside such more affluent settings.Thirdly,although the disease burden in LMICs has decreased since 1990,with communicable diseases projected to fall further over time,their burden from noncommunicable diseases will become proportionally higher.Although trials in LMICs do take place,they tend to be funded by international donors from HICs,or by industry,as opposed to being able to be resourced by LMICs themselves.There is therefore a pressing need to promote and advance efficient and sustained well-designed and well-implemented clinical trials that address local health needs across all stages of clinical research in LMICs and other resource-limited settings(39),encompassing both communicable and noncommunicable diseases in 1 The World Banks bands of income levels are commonly used to classify countries in terms of resources.In this document the term LMICs refers to the World Bank country classifications,whereas resource-limited settings refer to locales that may be common in low-income countries but may also exist in middle-and high-income countries,for example in remote and/or deprived communities.Moreover,a setting can change over time and may no longer be considered low-resource or newly become low-resource.71.Introductionorder to address the morbidity and mortality risks affecting people in those settings.If this does not occur,entire populations could miss out on the vaccines,diagnostics and other interventions that are needed as part of sustainable development globally.Moreover,conducting research in LMICs can foster capacity-building.By investing in training programmes and constructing laboratory facilities that meet international standards,research funders can contribute to the development of the necessary infrastructure and resources to execute high-quality research in LMICs.This,in turn,can lead to functional international networks and a sustained and equitable global health research landscape.Inequities in post-trial access to interventions tested in clinical trials also remain a major concern,especially in relation to LMICs.Indeed,there have been examples of trials where the disease burden in LMICs led to them being targeted for inclusion in clinical trials,yet these data were then used to file for marketing authorization in HICs or high-resource settings,often leading to availability of interventions in the latter but not the former.Similarly,trials of diagnostics that took place in LMICs or low-resource settings have sometimes failed to provide any post-diagnostic support for those with the diagnosed condition.These are examples of exploitation and a clear breach of ethical principles,and there must be a more systematic end-to-end approach for ensuring that new interventions are globally affordable and accessible,from discovery through to development and distribution.Inequities in leadership in clinical trials is an unresolved barrier.Local researchers,funders,communities and organizations should share an equal leadership role in prioritization,design,implementation and reporting of clinical trials.A further major challenge is that clinical trial cohorts have often lacked diversity,with under-representation of certain populations,resulting in them being underserved by clinical trials,including(but not limited to):Groups by demographic factors age extremes:neonates,infants,children and adolescents(despite this group representing a large proportion of the population in some settings)and older people(with elderly patients often being excluded from clinical trials on account of their being more comorbid and hence thought likely,but incorrectly,to obscure potential effects of an intervention even when there is a comparator group;this is an issue since they often carry a significant burden of disease and therefore represent a population in whom absolute effects of an intervention may be particularly large)women of child-bearing age pregnant and lactating women different ethnic minority groups male/female sex(depending on trial context,although women were often previously under-represented);Groups by social and economic factors people living in remote areas socioeconomically disadvantaged socially marginalized people stigmatized populations including LGBTQI people people in alternative residential circumstances(for example,migrants,asylum seekers,refugees,occupants of care homes,prison populations,traveller communities,the homeless and those of no fixed abode)religious minorities people who do not attend regular medical appointments people who face language barriers and digital exclusion/disadvantage carers military veterans;Guidance for best practices for clinical trials8Guidance for best practices for clinical trialsGroups by health status multimorbidity people who lack the capacity to give consent for themselves cognitive impairment learning disability people with neglected tropical diseases people with addictions people with multiple health conditions or those who are severely ill people with physical disabilities or who are visually/hearing impaired people with rare diseases and genetic disease subtypes.Such lack of clinical trial inclusivity and diversity can lead to trial results being less generalizable to groups who would potentially benefit from the findings,despite them often being groups with the highest burden from a particular disease or condition.This weakness has impeded the quality of available evidence for decision-making,leaving huge uncertainties related to care and inequitable access to interventions.It can also reduce the willingness of people in those under-represented groups to accept treatment recommendations based on a trials findings.Finally,although patient and community engagement in clinical trials has improved,there remains a lack of broadly applicable disease-agnostic standard practice for such involvement.This inadequacy can lead to inappropriate trial design and implementation,mistrust in research,and failure of clinical trials to start,reach completion or produce results meaningful to the populations they are intended to serve.1.5 Steps required to improve evidence generationThe above issues urgently need to be addressed to advance global public health.This requires identification of relevant research questions,proportionate design and conduct of clinical trials and strengthening of the global clinical trials environment,or“ecosystem”.1.5.1 IdentificationofarelevantresearchquestionA prerequisite for conducting a good clinical trial is identification of an important and relevant research question,the answers to which will fill gaps in evidence to inform research priority setting.Clinical trials should principally focus on public health and disease areas of national and global priority and address questions that are clinically pertinent to the communities and populations affected by them;at the same time,they should consider epidemiological trends to address potential(and future)health threats.It is vital not only to identify a relevant question but also to ascertain if it has already been robustly answered.This can be facilitated by conducting and reporting systematic reviews(for example,according to the guidelines on Preferred Reporting Items for Systematic Reviews and Meta-Analyses(PRISMA)(41)as part of clinical trial planning.Such reviews comprehensively evaluate and synthesize available evidence,and as such can consolidate existing knowledge and improve future clinical trials by providing insights into the strengths and limitations of prior studies,as well as guide the selection of interventions and outcome measures.By conducting a systematic review researchers can prevent unnecessary duplication and minimize research waste(42,43),and such reviews should be promoted by funders and seen as complementary to clinical trials.However,it should also be borne in mind that systematic reviews may be subject to bias,either because trials with more promising results are more likely to be published and known about than those with less promising results or because such reviews may under-represent certain populations if the existing evidence is not relevant to them(for example,trials in LMICs may not be prominent owing to research having historically been done in HICs).91.IntroductionTherefore,additional measures(such as policy gap analysis)may also need to be considered as part of the process of identification of a relevant research question.Such reviews should include both available evidence from clinical trial results and review of ongoing research available in clinical trial registries such as the primary registries from International Clinical Trials Registration Platform(ICTRP)(44)and ClinicalTrials.gov(45).1.5.2 Efficientandrisk-proportionatedesign and conduct of clinical trialsClinical trial quality can be defined as fitness for purpose,where the purpose is to provide reliable results of sufficient robustness to enable informed decision-making based on the trial outcomes.Attributes of clinical trial quality include good trial design,conduct and appropriate analysis.One area identified as a potential barrier to clinical trials has been over-interpretation of existing regulations and guidance for clinical trials.A frequent consequence has been excessive bureaucracy,which has also resulted in unnecessarily onerous and disproportionate trial procedures,with,for example,even minor trial processes or trial staff changes(which do not materially affect the reliability or safety of a trial)often requiring extensive documentation for no benefit.This lack of proportionality has sometimes had the adverse consequence of reducing rather than improving the number of reliably informative trials across a range of settings.Instead,trial“quality”should focus on good design and processes that assure the absence of errors that matter to decision-making that is,errors that have a meaningful impact on the safety of trial participants or credibility of the results(and thereby the care of future patients)and not be confused with the volume of paperwork(including collecting and filing documents),the length of clinical trial protocols and other documentation(46).Crucially,trial processes should be proportionate to their context and any associated risks,with efficient implementation.Streamlining and quality are not opposed.Enabling such an approach need not compromise the robustness of the data generated to answer relevant scientific questions;rather it can substantially enhance available evidence from high-quality clinical trials and hence population health worldwide.Section 2 of this document provides high-level guidance for clinical trial design and conduct with a focus on the key scientific and ethical features that should be universal to all clinical trials in order to enable them to produce reliably informative,high-quality evidence relevant for informing national and international guidelines and decision-making,regardless of context.A proportionate approach focusing on the main considerations and what really matters can enable more good quality trials to be conducted.1.5.3 Strengthening of the global clinical trial ecosystemFor clinical trials to achieve their intended aims,measures must be taken to enhance the capabilities to conduct relevant trials globally.This requires:action by not only those designing and conducting clinical trials but also all parties involved in prioritizing,funding,approving and overseeing clinical trials;investment in and availability of clinical trial infrastructure globally;and efficient communication between all those involved.Crucially,patients and communities should be seen as a key part of the clinical trials ecosystem and involved at all stages of the lifecycle of a clinical trial.Section 3 of this document provides high-level guidance and recommendations on best practices for the strengthening of the ecosystem for clinical trials,including enabling actions.2.Keyscientificandethicalconsiderations for clinical trials Patients at the Radboudumc Amalia Childrens Hospital use VR headsets for relaxation,entertainment,meditation,and hypnosis.WHO/Marie Oleinik12Reliably informative,ethical and efficient clinical trials(”good”trials)need to address the following five key points which capture the necessary qualities of a well-planned,well-run and clinically relevant trial.They should:be designed to produce scientifically-sound answers to relevant questions respect the rights and well-being of participants be collaborative and transparent be designed to be feasible for their context manage trial quality effectively and efficiently.The methods and approaches needed to apply these qualities will differ in small or large ways from trial to trial,but their validity is universal.2.1 Good clinical trials are designed to produce scientificallysoundanswerstorelevant questions Clinical trials should help to resolve important uncertainties about effects of health interventions.Depending on the context,the results may be needed to determine whether to proceed with development or further evaluation of the intervention or to inform regulatory licensing,clinical guidelines and/or health policy.In each case,relevant uncertainties applying to the specific question(s)that remain at the end of the trial should be sufficiently small to allow meaningful decisions to be made.As indicated in Section 1.2,most health interventions only have moderate effects,and clinical trials intended to reliably ascertain such effects 2.Keyscientificandethicalconsiderations for clinical trialstypically require clinical trials with the following features:randomization without foreknowledge of intervention allocation so as to ensure that any differences in health outcomes observed between the randomized groups are due to either the effect of the study intervention or the play of chance(that is,control for systematic errors),as well as blinding/masking of allocated trial intervention(where feasible)to further minimize bias.2.1.1 Robust intervention allocationKey message.Randomization requires generation of an unpredictable allocation schedule with concealment of the intervention to which a particular participant has been allocated until after the point of randomization.It should be impossible to predict in advance to which study intervention an individual trial participant or individual cluster(for instance,hospital or city in a cluster clinical trial)is likely to be allocated,so that investigators,health care providers and other staff involved and potential participants are not aware of which intervention to which they will be assigned.Why this is important.Randomization allows for like-with-like comparisons so that subsequent differences in health outcomes between the groups(beneficial or adverse)are due either to the play of chance or causally to differences in the study intervention.The absence of adequate concealment of allocation before randomization can lead to selection bias(that is,the decision to enter a particular participant in a trial can be influenced by knowledge of which intervention they are likely to be assigned to).2.1.2 Blinding/maskingofallocatedtrialintervention(wherefeasible)Key message.Knowledge of the allocated trial intervention may influence the behaviour of participants,those who care for them,and those 132.Key scientific and ethical considerations for clinical trialsassessing study outcomes(particularly if these are subjective in nature).These problems can be avoided through use of placebo medications or dummy interventions and by ensuring that those individuals or systems responsible for assessing participant outcomes,as well as all who are responsible for care of participants,are unaware of the intervention allocation.Why this is important.In many clinical trials,knowledge of the allocated intervention can influence the nature and intensity of clinical management,the reporting of symptoms or the assessment of functional status or clinical outcomes,introducing bias.Where feasible,masking(or blinding)participants,investigators,health care providers,and those assessing outcomes to the assigned intervention through use of placebo medications or dummy interventions can help to prevent such issues,as can the use of information that is recorded separately from the clinical trial(for instance,in routine clinical databases and disease registries).These considerations are important for the assessment of both the efficacy and the safety of the intervention,including processes relating to adjudication of outcomes and considerations of whether an individual health event is believed to have been caused by the intervention.If blinding of an allocated trial intervention is not feasible(for example in trials of different types of patient management or surgical procedures),blinded or masked outcome assessment should be pursued for objectively determined outcomes,for example through use of a prospective randomized open-label blinded endpoint(PROBE)design(see also Section 2.1.9 ascertainment of outcomes).All good clinical trials should include the features set out in sections 2.1.32.1.12.2.1.3 Appropriate trial population Clinical trials often exclude populations that the intervention may well benefit,sometimes precluding access to certain interventions for the populations excluded from the trials.Key messages.The eligibility criteria should be tailored to the question that the trial sets out to answer.Inclusion criteria should not be unnecessarily restrictive.Efforts should be made to include a broad and varied population(for example,with appropriate balance of sex/gender,age,race/ethnic and socioeconomic diversity),unless there is a good medical or scientific justification for doing otherwise.Exclusion criteria should be focused on identifying individuals for whom participation would place them at undue risk by comparison with any potential benefits(for example,based on their medical history or concomitant medication),for whom the benefits have already been reliably demonstrated,or for whom the intervention is not relevant.Why this is important.Inclusive eligibility criteria increase the relevance and generalizability of the findings.They may sometimes allow assessment of whether there is good evidence of material differences in the effects(beneficial or adverse)and/or acceptability of an intervention or its delivery in any particular subgroup(for example,based on specific genetic,demographic or health characteristics),even though statistical power to detect whether such differences exist may be limited.Populations should not be excluded based solely on an argument of potentially insufficient statistical power to detect subgroup specific effects.Guidance has been developed to improve inclusion of underrepresented groups(4750).Decentralized or point-of-care trials may help to increase the diversity of clinical trial enrolment by increasing trial accessibility.In addition,diversification of trial staff can help to improve community engagement and diversification of recruited clinical trial populations(51).Specific examples of populations that have typically been excluded from clinical trials(either explicitly or by implicit exclusion)include pregnant and lactating women,infants and children,and older adults.This practice has been hugely detrimental and such people should be eligible for trial enrolment unless a valid justification is provided for their exclusion(for example,if there is a serious safety concern or a contraindication to a certain intervention or if they are at very low risk of the health issue the trial seeks to address).Some ways to foster enablement of clinical trials in two specific groups are outlined below in subsections(a)and(b).Recommendations Guidance for best practices for clinical trials14Guidance for best practices for clinical trialsrelated to inclusion of older adults,another important frequently-excluded group,are contained in Annex2.(a)Enablingclinicaltrialsinpregnantandlactatingwomenandwomenofchild-bearing ageDemand for facilitation of inclusion of pregnant and lactating women(52)and women of child-bearing age in clinical trials is growing.Their participation requires consideration of the specific barriers they may face to enrolment,owing to not only the incorrect perception that this is a default ethical or scientific position but also practical issues.Some of these potential barriers,such as limited literacy to provide informed consent and legal restrictions(for example,third party consent requirements),are common to many populations,whereas others are more prevalent in women,such as the need for child-care provision and,particularly in some regions,mobility restrictions.Many clinical trials could enable recruitment of pregnant and lactating women by assessing pre-existing evidence for the safe use of the same or similar intervention in this population,such as its use for a different clinical indication.Due consideration should be given to the severity of the condition for which the intervention is intended in this population,and the potential for improved outcomes in both the recipients and their offspring.For trials of novel interventions for maternal disease where there is no pre-existing evidence of use of the same or similar interventions in this population,preclinical reproductive toxicology studies should be reviewed to guide decision-making.Where maternal disease is severe,maternal and pregnancy outcomes are expected to be poor and reproductive toxicology studies are reassuring,benefits to women of inclusion in a trial are likely to outweigh any potential risks.For diseases with high fatality for which no alternative interventions are available,reproductive-toxicity studies should be expedited,and pregnant individuals included as far as possible in clinical trials of new interventions.In several therapeutic areas,practical ways to accelerate investigation of new interventions in pregnant and lactating women have been developed,with calls to action involving multiple stakeholders.There is now ethical guidance for the inclusion of pregnant women in the development of vaccines against emerging pathogens,and subsequent vaccination programmes as well as for advancing research into HIV and coinfections in pregnant women(53,54).An ICH guideline(E21)is also currently in development for inclusion of pregnant and lactating women in clinical trials(55).Excretion of a drug or its metabolites into human milk should be examined where applicable and feasible,and offspring of breastfeeding women enrolled into clinical trials monitored for any effects of an intervention(6).(b)EnablingpaediatricclinicaltrialsChildren should not be an afterthought in clinical development programmes but considered from the outset(56),with inclusion as early as possible in clinical trials of interventions of potential benefit to them.Wherever possible,extrapolation of adult efficacy and safety data to children should be considered.Use of age-agnostic trial enrolment and standardized weight-band dosing for children,with parallel enrolment of all children across all weight bands,should also be considered,potentially coupled with enrolment by development stage.Such an approach could mitigate against enrolment of children being done in an age-stratified way(that is,starting with older children),which disadvantages younger patients.Development of appropriate paediatric interventions should be prioritized with attention given to factors such as palatability of formulations,flexibility for weight-based dosing and stability for use in a wide range of geographical locations.Use of pharmacometric modelling and simulation techniques(such as creation of synthetic control arms)may help to improve the design of some paediatric trials.Several initiatives are underway to enable paediatric clinical trials.In particular,in 2016,the World Health Assembly adopted resolution WHA69.20 on promoting innovation and access to quality,safe,efficacious and affordable medicines for children(57).WHO and other stakeholders have joined forces to accelerate access to effective paediatric diagnostics and medicines,notably for HIV infection and tuberculosis(5860),and the Global Accelerator for Paediatric Formulations Network(GAP-f)(61)was created to build on and formalize the model developed within the HIV community to provide a sustainable mechanism to ensure that safer,more effective and more durable paediatric formulations are developed and made available to children against an accelerated timeline.Measures such as the FDA Paediatric Research Equity Act(62)and EU Paediatric Regulation(63)have also enabled trials in this population.152.Key scientific and ethical considerations for clinical trialsThe use of global networks may further enable clinical trial participation in such populations.For example,the Global Network for Womens and Childrens Health Research(64)is a partnership dedicated to improving maternal and child health outcomes and building health research capacity in resource-poor settings by testing cost-effective,sustainable interventions that provide guidance for the practice of evidence-based medicine.2.1.4 Adequate size Key message.A clinical trial should be sufficiently sized and statistically powered to provide a robust answer to the question it sets out to address.Why this is important.For the effects of health care interventions to be reliably detected or reliably refuted,random errors must be small by comparison with the expected size of the effect of the intervention.The best way to minimize the impact of random errors is to study sufficiently large numbers of participants who will develop the health outcome that the intervention is intended to prevent or treat(noting that clinical trials assessing impact on discrete health outcomes such as mortality will require more participants than those assessing impact on continuous measures such as laboratory results as is often the case in early-phase trials)(65).Trials in early stages of drug development(for example,first-in-human trials)have a specific objective for which a smaller size is typically appropriate.In some scenarios it is challenging to enrol large numbers of participants,such as trials assessing interventions for rare diseases.For such trials,it may be helpful to contribute to a broader collaboration to conduct them,through large,multicentre clinical trials,or to select a clinically relevant outcome for which the effect size is expected to be larger(for example,a clinically validated physiological or imaging biomarker).It may be possible to reduce the impact of random errors through statistical analyses or by making assessments at a time when the effects of the intervention are expected to be greatest.Use of alternative study designs to facilitate recruitment(such as point-of-care and decentralized trials)may help to ensure adequate trial size.Meta-analysis may be particularly helpful when the effects of an intervention on an outcome are likely to be moderate and too few cases have occurred in any individual trial to assess the effects sufficiently reliably or to assess whether there any important differences in treatment response between different patient groups(2123).Use of core outcomes(see Section 2.1.7)may facilitate prospective meta-analyses.2.1.5 Adherence to allocated trial intervention Key message.Efforts should be made to facilitate and encourage adherence to the allocated intervention(s)where appropriate and feasible.Why this is important.Although there may be instances in which it is appropriate for trial participants to stop their allocated intervention(for example,in the case of a major intolerance),the potential ability for the trial to accurately determine and quantify the impact of the intervention(whether beneficial or harmful)should be carefully considered.For RCTs,if trial participants allocated to an active intervention do not receive it as planned or if those allocated to the control group(for example,placebo or usual care)start to receive the active intervention,then the contrast between the two study groups is lower.Consequently,the ability to assess any difference in outcome between the arms of the trial is reduced(and a false conclusion that there is no meaningful difference between the interventions when in fact there is one is more likely).Adherence to allocated trial intervention may be facilitated through for example,pre-randomization“run-in”phases(on placebo or even active intervention)and supporting trial participants to continue(for example,where feasible,options for remote follow-up rather than in-person clinic visits).2.1.6 Completenessoffollow-upKey message.Participant outcomes should be ascertained for the full duration of the clinical trial,regardless of whether a participant continues to receive the allocated intervention or ceases to do so(because,for instance,of perceived or real adverse effects of the intervention),with every effort made to proactively minimize the loss of data.In some cases,it may also be appropriate to continue follow-up for many years after the main analyses have been reported.Guidance for best practices for clinical trials16Guidance for best practices for clinical trialsWhy this is important.For RCTs,continued follow-up of all randomized participants(even if some stop their assigned intervention)maintains the like-with-like comparison produced by the randomization process.Premature cessation of follow-up or post-randomization exclusion of participants should therefore be avoided as it may introduce systematic bias,particularly as the type of people excluded from one intervention group may differ from those excluded from another.Incomplete follow-up may reduce the statistical power of a clinical trial(that is,the ability to distinguish any differences in outcome between the interventions)and underestimate the true effects(benefits or hazards)of the intervention.Extended follow-up can allow for detection of beneficial or harmful effects of the study intervention that may persist or emerge months or years after the initial randomized comparison.2.1.7 Relevant measures of outcomes,as simple as possibleKey message.The outcomes that are assessed in a clinical trial need to be relevant to the question being addressed and should be as simple as possible.When trials are intended to achieve marketing authorization or change policy,it is often helpful to discuss the choice of trial outcomes with regulators and/or policy-makers.Use of standardized core outcome sets(that is,the minimum outcomes that should be measured and reported in all clinical trials of a specific condition,reflecting outcomes relevant to decision-makers and patients)should be considered for all trials,to enable the results of studies being compared,contrasted and combined(for example,in later meta-analyses)as appropriate.Outcomes may include physiological measures,symptom scores,participant-reported outcomes(PROMs)(66)(that is,measurement tools that patients use to provide information on aspects of their health status that are relevant to their quality of life,including symptoms,functionality,and physical,mental and social health),functional status,clinical events or use of health care services.The way in which these are assessed should be sufficiently robust and interpretable(for example,clinically validated in a relevant context,particularly for surrogate outcomes given their potential limitations(67).Why this is important.The ways by which the consequences of the intervention are measured should be sensitive to the expected effects of the intervention and appropriate to the study question,and in general should be applicable and clinically or scientifically meaningful for the relevant population.The choice of outcomes may vary depending on the extent of prior knowledge of the effects of the intervention(for instance,early trials may assess the effects on imaging and laboratory markers whereas later trials study the effects on clinical outcomes)or change over time according to the changing epidemiology of a condition(for example on account of pathogen mutation and associated impact on clinical effects).It is rarely possible or desirable to assess the full range of potential outcomes in a single trial.Instead,there should be a focus on providing a robust answer to the specific,well-formulated question that can have impact on patients and policy.Use of core outcomes can both enhance the ability to undertake meaningful comparisons as part of evidence synthesis and decrease research waste.Several initiatives for the use of core outcomes already exist for both disease-agnostic and disease-specific outcomes,including the International Consortium for Health Outcomes Measurement(68),Core Outcome Measures in Effectiveness Trials(69),Standardized Data Collection for Cardiovascular Trials Initiative(70),Standardised Outcomes in Nephrology(71),and the International Alliance of Mental Health Research Funders Common Measures in Mental Health Science Initiative(72).2.1.8 Proportionate,efficientandreliable capture of dataKey messages.Data collection should focus on the key aspects needed for assessment and interpretation of the trial results as specified in the protocol and should not be excessive.The extent to which information(for example,on participant characteristics,concomitant treatments,clinical events and laboratory markers)is detected and recorded,and the means and level of detail to which this is done,should be tailored to each clinical trial.In general,data collection should be kept to the simplest level possible to answer the question,and collecting non-essential variables should be avoided.Tools and methods for data collection,storage,exchange and access should enable the trial to be conducted as designed,support privacy and security,and enable reliable and consistent analyses.Digital 172.Key scientific and ethical considerations for clinical trialstechnology and routine collection of health care data can provide alternative or complementary means to record information about participants and their health at study entry,during the intervention and follow-up period,and for many years beyond,where available and appropriate.Why this is important.The volume,nature and level of detail of data collection should be balanced against its potential value.Disproportionate data collection wastes time and resources.It places an unnecessary burden on trial participants and staff,distracts attention from those aspects of the trial that have greatest consequence for the participants,and reduces the scale(number of participants and duration of follow-up)of what is achievable with available resources.In some trials,it may be appropriate to measure some features(for example,intermediate biomarkers)in a subset of participants,chosen on the basis of baseline characteristics or random selection,or at a limited number of timepoints.The choice of method used for data collection can have an important bearing on trial reliability and feasibility.Use of data standards(for example,the Clinical Data Interchange Standards Consortium Study Data Tabulation Model CDISC SDTM(73)can help to ensure data quality and data integrity,as well as to facilitate potential meta-analysis and data sharing.Use of digital technology and routinely-collected health care data can improve the relevance and completeness of information collected(for instance,by reducing loss to follow-up)as well as reduce the burden on those conducting the trial and its participants,provided that the data are used appropriately.2.1.9 Ascertainment of outcomes Key message.Processes for ascertaining study outcomes should adopt an approach that is not influenced by the intervention trial participants or randomized groups receive.These measures include the frequency and intensity of assessments.For RCTs,particular care should be taken to ensure that the people assessing,clarifying and adjudicating study outcomes are not influenced by knowledge of the allocated intervention(that is,the outcome assessment is blinded or masked).Equally,the methods for acquiring,processing and combining sources of information(in order,for example,to define participant characteristics or clinical outcomes)should be designed and operated without access to information about the intervention allocation for individual participants or knowledge of the unblinded trial results.Why this is important.If the methods used to assess,clarify or classify outcomes differ between the assigned interventions,the results may be biased in one direction or other leading to inappropriate conclusions about the true effect of the intervention.Therefore,the approach used to assess what happens to participants should be the same regardless of the assigned intervention,and those making judgements about the occurrence or nature of these outcomes should be unaware of the assigned intervention(or features,such as symptoms or laboratory assays,that would make it easier to guess the assignment)for each participant.2.1.10 Statistical analysis Key messages.The trial should be designed to robustly answer a clearly articulated key question on which the primary analysis should focus.It is not good practice to seek to answer multiple questions through secondary analyses,which can often be misleading.Trial results should be analysed in accordance with the protocol and statistical analysis plan,with the latter being developed and clearly specified when the protocol is written,and finalized at the latest before the study results become known(that is,before conduct of any unblinded analyses on study outcomes).Any analyses conducted after the initial results are known should be clearly identified as such(8).For RCTs,the main analyses should follow the intention-to-treat principle,meaning that outcomes should be compared according to the intervention arm to which the participants were originally allocated at randomization,regardless of whether some of those participants subsequently received some or none of the intended intervention,and regardless of the extent to which the post-randomization follow-up procedures were completed.Subgroup analyses should be interpreted cautiously,with due consideration given to prior understanding of disease mechanism,especially if they are not prespecified or are multiple in number(whether Guidance for best practices for clinical trials18Guidance for best practices for clinical trialsprespecified or not).In general,any prognostic features that are to be used in analyses of intervention effects in clinical trials should be irreversibly recorded or identified before randomization.Reporting on data disaggregated by sex(consistent with the Sex and Gender Equity in Research SAGER guidelines)(74)can be valuable.Any findings should be interpreted with respect to other existing evidence and clinical context.Why this is important.A statistical analysis plan should be specified before any knowledge of the trial results(for example,unblinding of the treatment allocation in a RCT)in order to avoid the possibility that choices about the analysis approach may be biased(8).For RCTs,a particular strength is the existence of a randomized control group with which to compare the incidence of all health events.Consequently,it is possible to distinguish those events that are causally impacted by allocation to the intervention from those that are part of the background health of the participants.Analysing all participants according to the intervention to which they were originally allocated(“intention-to-treat”analysis)is important because,even in a properly randomized trial,bias can be inadvertently introduced by the post-randomization removal of certain individuals from analyses(such as those who are found later not to meet the eligibility criteria,who do not adhere to their allocated study treatment or who commence an active intervention having been allocated to a control group)if the reason for removal might have been influenced by the treatment allocation.Additional analyses can also be reported;for example,when the frequency of a specific side-effect is being described,it may be justifiable to analyse its incidence only among those who received the active intervention,because randomized comparisons may not be needed to assess large effects.However,in assessing moderate effects of the treatment,“on-treatment”or“per protocol”analyses can be misleading,and intention-to-treat analyses are generally more trustworthy for assessing whether there is any real difference between the allocated trial interventions in their effects.One of the most important sources of bias in the analysis is undue concentration on just part of the evidence(such as selective emphasis on the result in one subgroup or subsidiary outcome out of many that is defined after consideration of the data).Apparent differences between the therapeutic effects in different subgroups of study participants can often be produced solely by the play of chance.Subgroups therefore need to be relevant,prespecified and limited in number.Analysis of results in subgroups determined by characteristics observed after randomization should be avoided because,if the recorded value of some feature is(or could be)affected by the trial intervention,then comparisons within subgroups that are defined by that factor might be biased.It is important to interpret results in specific subgroups(for example men and women)cautiously and consider whether they are consistent with the overall result.Failure to do so can lead to people in those subgroups being treated inappropriately(given an intervention that is ineffective or harmful)or untreated inappropriately(not being given an intervention that would benefit them)when there is no good evidence that the effect varies between them.Although a sound statistical approach is critical in clinical research,it is equally important to focus on the clinical magnitude and relevance of any effect size rather than solely its statistical significance(7578),as well as any new findings in the context of previous findings(for example,using the Grading of Recommendations Assessment,Development and Evaluation GRADE approach(79).2.1.11 Assessingbeneficialandharmfuleffects of the interventionKey messages.Data generated during the course of conducting a clinical trial may reveal new information about the effects of the intervention which is sufficiently clear that it necessitates alteration of the ways in which the trial is conducted and participants are cared for or which is sufficiently compelling as to warrant a change in the use of the intervention both within and outside the trial.Potential harms of the intervention should be considered alongside potential benefits and in the wider clinical and health contexts.Why this is important.Not every health event that happens in a trial is caused by one of the interventions;individuals involved in a trial may suffer health events that have nothing to do with the trial or the interventions being studied.(The less 192.Key scientific and ethical considerations for clinical trialshealthy the participants in the trial,the more likely that any health event is related to factors other than the intervention.)Assessing whether signals(for example,rates of clinical events or laboratory abnormalities)seen among those allocated to receive a health intervention are significantly more or less frequent than in a control group(where applicable)provides a reliable assessment of the impact of the intervention.It provides a fair assessment of which events are causally impacted by allocation to the intervention compared with those that are part of the background health of the participants.In an ongoing trial,such unblinded comparisons should be conducted by a group(such as a data monitoring committee,also known as a data and safety monitoring board)that is independent(or protected by a firewall)from the trial team to avoid prematurely unblinding the emerging results to those involved in running the trial.By contrast,reports of individual events that are believed(for instance,by the participant or a doctor)to be caused by the intervention are much less informative,owing to the lack of a comparison with the incidence of the event in any control group and the inherently imprecise judgement of causality.The exceptions are events that are rare in the types of people involved in the trial but known to be potentially strongly associated with particular interventions(for example,anaphylaxis or bone marrow failure in association with medicines),which can be viewed as events of special interest for reporting and analysis.Depending on the type of trial,the degree of knowledge of the intervention(s)and the population in which they are being studied,the protocol may specify certain events that require,or indeed do not require,to be collected and recorded.Equally it may specify which recorded events might be expected to occur in the population of interest and hence which may not require reporting in an expedited manner.Effects of health interventions may differ(they may be harmful or beneficial)and follow different time courses,and may occur at different frequencies and in particular groups of individuals.Some interventions(such as surgery or chemotherapy)may be associated with little or even a hazardous effect in the short-term but provide longer-term benefit.It should also be recognized that for many interventions the benefits may not be apparent on an individual basis,such as where a detrimental outcome has been prevented(for example,a stroke or infection).2.1.12 Monitoring emerging information onbenefitsandharmsKey messages.An independent data monitoring committee provides a robust means to evaluate safety and efficacy data from an ongoing trial,including for RCTs unblinded comparisons of the frequency of particular events,without prematurely unblinding any others involved in the design,conduct or governance of the trial.For many clinical trials,particularly in early-phase trials,the functions of a data monitoring committee could be provided internally from the entity running the trial,but those involved should nonetheless be rendered independent by being adequately protected by a firewall from the trial team to ensure that awareness of results does not introduce bias(or the perception of bias).Use of a charter that details the structure and organization of the data monitoring committee can promote transparency and facilitate such committees to operate more effectively.Some trials may not require a data monitoring committee(for example,if the trial is short-term and would not be modified regardless of interim data),although they may still benefit from some form of independent oversight.A data management committee(DMC)should include members with relevant skills to understand and interpret the emerging safety and efficacy data,and where appropriate take into consideration patient and public perspectives.A DMC should review analyses of the emerging data,unblinded to any randomized intervention group so as to be able to make informed decisions given knowledge about the potential adverse effects of a specific treatment(which would not be possible if they were not unblinded).The DMC should advise the trial organizers when there is clear evidence to suggest a change in the protocol or procedures,including cessation of one or more aspects of the trial.Such changes may be due to evidence of benefit or harm or futility(where continuing the trial is unlikely to provide any meaningful new information).In making such recommendations,a DMC should take account of both the unblinded analyses of the trial results and Guidance for best practices for clinical trials20Guidance for best practices for clinical trialsinformation available from other sources(including publications from other trials).Why this is important.All those involved in the design,conduct and oversight of an ongoing trial should remain unaware of the interim results until after the conclusion of the study so as not to introduce bias into the results(as in the case,for example,of stopping the trial early when the results happen by chance to look favourable or adverse).The requirement for,and timing and nature of,any interim analyses should be carefully considered so as not to risk premature decision-making based on limited data.2.2 Good clinical trials respect therightsandwell-beingofparticipantsEthical clinical trials(1214)combine the search for answers to important questions with scientific validity and appropriate protection and respect for all involved,particularly participants.Independent review of proposals for new research,through an institutional review board(IRB),research ethics committee(REC)or equivalent is an important governance tool and can help to ensure appropriate steps are taken to protect the rights and welfare of participants.2.2.1 AppropriatecommunicationwithparticipantsKey message.At all stages of a clinical trial(before,during and after),relevant,easily-understandable information should be shared with trial participants(or,where applicable,their legal representatives),with a careful balance of the duty to inform against the risk of information saturation and account being taken of the clinical context.Information should be provided in a clear manner and in suitable languages and formats for the intended audiences.Co-developing and piloting such clinical trial information with target populations is valuable.The most important information for participants decision-making should be clearly highlighted and excessive length of information materials and consent forms should be avoided.Why this is important.Providing timely and relevant information to participants during a trial facilitates ethical research with benefits to both the participants and the quality of the trial results.It is essential that potential or recruited trial participants are appropriately informed,but presenting excessive or exhaustive detail can work against this objective by overwhelming,confusing or disconcerting potential participants.Care should be taken to communicate effectively and enable relevant discussion,taking into account accessibility(for example,to those who are illiterate).In some circumstances it can be helpful to provide information in visual,audio,animated or interactive computer-based formats.The exact approach may be influenced by the context of the research,including clinical,cultural or other issues.At the end of the trial,the key results should be made available to participants in a form that is accessible and understandable.As specified in Section 2.3.1,the development of clinical trial information in partnership with patients,the public and communities can facilitate the inclusion of diverse populations.2.2.2 Relevant consent Key messages.The trial consent process should clearly explain to potential trial participants(or,where applicable,their legal representatives)the reasons why the trial is being done,the questions it is seeking to answer,what is involved for them,and the potential benefits and risks of participation(12).Where appropriate,this should include an assent process for those lacking capacity to give full consent such as children and minors(56).The extent,nature and timing of information provided before and during the informed consent process should be guided by the level of additional risks and commitment that participation in the trial would involve in the context of the usual clinical care or circumstances that the same individuals would normally receive.The information provided should prioritize the needs and expectations of the prospective participant rather than those of the organization or individuals conducting the trial.Consent information should be widely accessible and readily understandable(for example,with respect to readability),avoid legalistic or other technical language,and be as succinct as possible.Approaches to obtaining and maintaining ongoing 212.Key scientific and ethical considerations for clinical trialsconsent and communication should be relevant to the trial it relates to,with due consideration given to cultural and community contexts.Where appropriate,electronic consent mechanisms may be used(80).The consent process should facilitate optimal use of data where possible through inclusion of wording that allows for appropriate and relevant future application of data or for use of biological samples in research(13).Where co-enrolment in another clinical trial is not possible this should be scientifically justified.Where payment for clinical trial participation is offered,this should also be explained and justified.Conversely reimbursement for costs incurred by participants is a broadly accepted practice in many contexts and should be distinguished from payments to participate.Why this is important.Consent is valid if it is informed,voluntary and competently given before entry into a trial.There are some situations in which an individual cannot give informed consent(for example,for infants,minors or individuals lacking decision-making capacity,in which case consent should be obtained from the participants guardian or legally authorized representative)or it is not practical to do so because of the urgency of the medical situation(for example,in cases of trauma or medical emergencies,in which case consent should be sought later in the trial if and whenever the participant recovers the capacity to consent or from their guardian or legally authorized representative once they are available).For some trials and in some individual situations,explicit consent may be modified or waived(14).In such cases,there should be minimal additional risks and burdens to participation in comparison to the usual care a prospective participant might receive outside the trial.Waivers or modifications of informed consent may also be necessary in some clinical trials in which the intervention is directed at an entire community(cluster randomized trials),making it impossible to avoid the intervention.S
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