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Closing Health Gaps through Collaborative ActionW H I T E P A P E RS E P T E M B E R 2 0 2 4A G U I D E T O I M P A C T F U L P L A C E-B A S E D C H A N G EIn collaboration with DeloitteImages:Getty Images 2024 World Economic Forum.All rights reserved.No part of this publication may be reproduced or transmitted in any form or by any means,including photocopying and recording,or by any information storage and retrieval system.Disclaimer This document is published by the World Economic Forum as a contribution to a project,insight area or interaction.The findings,interpretations and conclusions expressed herein are a result of a collaborative process facilitated and endorsed by the World Economic Forum but whose results do not necessarily represent the views of the World Economic Forum,nor the entirety of its Members,Partners or other stakeholders.ContentsForeword 3Executive summary 4Understanding place-based change 51 Community ownership and empowerment 72 Rigorous analytical approach 103 Purposeful and lasting partnerships 134 Sustainable and execution-oriented operating and governance models 17Conclusion 21Contributors 22Endnotes 24Closing Health Gaps through Collaborative Action2Nancy Brown Chief Executive Officer,American Heart InstituteForewordClosing Health Gaps through Collaborative ActionSeptember 2024Fundamentally,we all deserve long,fulfilling and healthy lives regardless of race,ethnicity,gender,sexual orientation,disability,age or location.Over the past century,we have made extraordinary advances towards this goal.Global life expectancy has risen from 46.5 years in 1950 to 71.7 years in 2022,thanks to improved access to services and technological advances.1 However,if we examine this data more closely,stark differences emerge.Health inequities avoidable differences in health between populations exist between and within countries.2 In 2021,Nigerias life expectancy was 30 years lower than Japans.3 These differences are even more striking at a community level.In Boston for instance,average life expectancy shrinks by nearly a quarter of a century between neighbourhoods just two miles apart.4These health inequities are not caused by one single issue,but by a complex mix of environmental,economic and social factors.In fact,up to 80%of individual health outcomes stem from the non-medical drivers of health,many of which are influenced by the place where an individual lives.5 For instance,South Korean women are projected to have a life expectancy exceeding 90 years by 2030,thanks in part to the countrys egalitarian distribution of services that make for healthy living conditions.6 Similar patterns are evident in the“Blue Zones”communities with large numbers of centenarians where the environment and resources allow individuals to live actively,develop social connections,eat nutritious diets and have a positive outlook.7 Unfortunately,many communities are not afforded the same factors.Achieving systemic change requires coordinated action among government,private sector,academia and civil society.This is part of the World Economic Forums core mission.The Global Health Equity Network(GHEN),a Forum initiative,aims to empower all communities to thrive.We believe that every organization is a healthcare organization,playing a crucial role for its workforce,offerings,community and ecosystem.Translating commitments into impact requires localized place-based approaches that understand community needs using existing data sources,perform comprehensive community resource mapping,and focus efforts on programmes that maximize health and well-being.By bringing together individual organizations efforts into a coalition,community-based ecosystems can improve community health outcomes and directly impact peoples lives.Place-based approaches have a long history,but achieving tangible and lasting impact that addresses health disparities remains challenging.Factors such as fragmented engagement,short-term investments and limited community buy-in are significant obstacles.Drawing on insights from cross-sector leaders,this white paper offers practical guidance for organizations committed to improving the health and well-being of communities.The journey towards global health equity is a collective endeavour that demands the concerted effort of individuals,communities and organizations worldwide.Together,we must work towards building a world where we all thrive.Michelle Williams Joan and Julius Jacobson Professor of Epidemiology and Public Health,Harvard T.H.Chan School of Public HealthShyam Bishen Head,Centre for Health and Healthcare;Member of the Executive Committee,World Economic ForumJoe Ucuzoglu Global Chief Executive Officer,DeloitteClosing Health Gaps through Collaborative Action3Executive summaryDespite increasing life expectancy,significant differences in health outcomes exist between and within countries,known as health disparities.Health equity is the pursuit of elimination of these disparities;its the fair and just opportunity for people to fulfil their human potential in the many aspects of health and well-being.8 Achieving health equity requires identifying and addressing the root causes of multifarious inequities:systemic racism and bias,structural flaws in health systems,and inequities in the drivers of health(which go beyond healthcare to social,economic,environmental and commercial factors).9The place where an individual lives is where conditions for healthy outcomes are fostered.Place-based approaches which comprise collaborative,community-led and long-term work in a defined geographic location offer a powerful mechanism to build healthy,inclusive and resilient communities.They bring stakeholders together to fight common challenges and drive sustainable,collective impact.This paper is based on research,interviews and case studies,and outlines four principles that can help promote effective place-based change.Organizations can amplify the impact of place-based strategies through community ownership,a rigorous analytical approach,purposeful partnerships and a sustainable operating model.Community ownership and empowerment Community ownership empowers local organizations and individuals to design,implement and maintain place-based strategies to meet their communitys needs,while being supported by a network of engaged collaborators.Purposeful and lasting partnerships All organizations have a role to play in health equity.They must leverage the differentiated expertise,perspectives and networks of a diverse group of collaborators across the public and private sectors and civil society to create comprehensive and dynamic solutions.Rigorous analytical approach Place-based efforts should stem from a rigorous analytical approach that draws on local and reliable data sources to pinpoint the set of factors that maximize health and close the gaps in long-term outcomes.Sustainable and execution-oriented operating and governance model Efforts should be designed with a strong operational backbone and“the end in mind”to bring about positive and lasting transformation across the healthcare,social,economic and environmental realms.Its a collective societal responsibility to recognize the impact of place on health outcomes.Every organization has a role to play:to help their employees achieve optimal health and well-being,to advance healthy products and services through local community efforts,and through the power of their ecosystems.By embracing place-based approaches,it is possible to create healthier and more resilient communities for all.Closing Health Gaps through Collaborative Action4Understanding place-based change As health disparities persist,place-based change allows for tailored programmes and coordinated efforts to meet multiple community needs.What is place-based change?The importance of place lies in the unique social,economic and environmental factors that influence individuals health and well-being.In fact,up to 80%of individual health outcomes stem from the non-medical drivers of health such as well-paying employment,healthy food and climate.11 Achieving health equity requires identifying and addressing the root causes of inequities within a place:systemic racism and bias,structural flaws in health systems,and inequities in these drivers of health.12Community refers to a group of people sharing common interests,concerns or identities.13 For this paper,its the group of people in a physical location that an effort is serving,all of whom have diverse skills,resources and experiences to contribute.14 The near-ubiquitous adoption of the internet has led to virtual communities;although powerful forums,theyre not considered in this context.Place-based approaches are collaborative,community-led and long-term approaches to building healthy,thriving communities in a defined geographic location.15 They recognize that coordinated action is required to enable systemic change,by bringing together community,government,private sector and academia to help address the root causes of disadvantage and simultaneously address multiple challenges.Approaches often have an anchor institution,a place-based entity rooted in the community and a backbone organization serving as a trusted third-party convener.16 Economic growthJob opportunitiesHousingInternet connectivityFood accessFood securityClimate changeEnvironmental healthNeighbourhoodsPublic safetyPersonal safetySupport systemCommunity relationshipsTransportation and mobilityBasic needsWealth accumulationSecure employmentEmployment benefitsMeaningful workHealth,digital and financial literacyEducational opportunitiesHealth people,communities Sustain well-beingempathyTechologiesCulturesethical mindsetanti-racismStructuresand planetReceive careEconomyWealthEmploymentPhysical environmentFoodInfrastructureEducationConnectionSafetyEconomicEnvironmentalSocialEquityA range of factors impact health equity10FIGURE 1Source:DeloitteHistory of place-based changePlace-based change has evolved over the past century,adapting to the complex dynamics of communities.In the mid-20th century,efforts focused on top-down urban renewal,such as economic regeneration of under-resourced areas.17 By the 1980s and 1990s,with greater understanding of social and environmental factors influence on health,prosperity and economic development,efforts had shifted towards integrated,community-led strategies.18 For instance,initiatives such as the Comprehensive Community Initiatives in the United States,19 URBAN Community Initiatives in Europe20 and the World Health Organizations(WHO)Healthy Cities Movement around the world,21 aimed to foster local development and enhance community health by addressing multiple dimensions of development.As sustainable development gained momentum throughout the 2000s,the importance of local action and community ownership became evident.22 In regions such as Asia and Africa,rapid urbanization increased the popularity of decentralized local development policies,empowering communities to assume greater responsibility for their prosperity and well-being.23 Programmes such as the Kampung Improvement Program(Indonesia)24 and the Millennium Villages Project(Africa)25 emphasized community participation,a strength-based approach and capacity building.Concurrently,the rise of corporate social responsibility encouraged businesses to extend their focus beyond profit,engaging in collaborative development efforts.Since the 2010s,leading academics have put their shoulder to the wheel to advance place-based efforts for health equity.For instance,public health leader Michael Marmot pioneered Marmot Places local communities that adopt a long-term,whole-system approach to address multiple drivers of health.26 Many healthcare systems have also restructured to support place-based,multi-sectoral approaches(e.g.Communities of Care in Singapore).27 These reforms are complemented by government programmes that bring together separate department efforts to focus not only on conditions within the communities,but also on the larger systems that create and sustain them(e.g.the Neighborhood Revitalization Initiative in the United States).28 Evolving methodologies now incorporate advanced analytics,leveraging geospatial data to pinpoint community needs,implement targeted programmes and evaluate outcomes.The COVID-19 pandemic accelerated the adoption of these strategies,underscoring the critical role of local and cross-sector collaboration in managing health crises and advancing health equity.29Principles for effective place-based changeBased on research and consultations with leading cross-sector organizations,this report spotlights four principles to address common challenges and maximize impact on health equity.These principles are not meant to follow a linear path but rather complement and reinforce each other.The following sections outline the best practice and guidance for organizations for each principle.Additional resources are available on the GHEN website,30 including further case studies and tools to support place-based efforts.Principles for effective place-based changeFIGURE 2Common challengesPrinciplesOutcomeLimited community buy-in and capacityMore equitable communities living longer in good healthFocus on factors that seem prevalent but do not result in improved health outcomesSiloed efforts and misaligned objectives across partnersMismatched time horizons and limited coordinating capacityCommunity ownership and empowerment facilitated by resources and support from local cross-sector partners1A rigorous analytical approach to identify the communitys greatest health equity needs through data2Purposeful and lasting partnerships that leverage differentiated capabilities in addressing community health needs3A sustainable operating and execution-oriented operating model that drives measurable outcomes and long-term impact4Source:World Economic ForumCommunity ownership and empowerment1Implementing place-based change often requires shifting established systems,structures,environment,policies and practices.This complex change demands early engagement and co-ownership with the community,otherwise efforts can inadvertently create distrust and resistance as organizations are perceived to serve their own agendas.31 Authentic collaboration means valuing community members as fellow experts and decision-makers.Relationships must be grounded in mutual trust and respect by taking the time to listen,understand needs and empower community voices.Community ownership empowers local organizations and individuals to design,implement and maintain programmes to meet the needs of their communities.Benefits More tailored initiatives:Communities help to set priorities,so programmes are more culturally relevant,context-specific and tailored to unique community needs,increasing the chance of success.Enhanced community leadership:Designing for community ownership fosters buy-in,strengthens social cohesion and enables long-term sustainability.Efficient resource-sharing:Efforts can pool community expertise,networks and assets to address complex challenges collectively,thereby amplifying impact.Trust is a driver of health in its own right.32 Developing trust between traditionally siloed partners and the target community is a monumental challenge.It requires time,resources and a willingness to learn,especially when communities have experienced histories of segregation,oppression and disinvestment.Trust also requires approaches that further reinforce and sustain it over time.1.1 Foster trust with community Guidance Engage authentically:Take time to understand partners goals,strengths and limitations;community engagement is about learning and requires an open and humble mindset.Respect cultural practices,incorporating them into working methods and engagements.Take concrete steps to reduce structural barriers to participation(e.g.location,language and compensation).Acknowledge how past interactions may influence the present-day dynamic and demonstrate what will be different going forward.Be thoughtful about what is being asked of the community and what is being offered in return;the work should be mutually beneficial,additive and meaningfully supportive of shared priorities.Identify trusted voices:Champions are essential for building partnerships,garnering community support and advocating for continued provision of resources.33 They are often trusted community leaders who serve as catalysts for change,such as faith-based organizations(e.g.Channels for Hope for Ebola)34 or community health workers(e.g.NHS CORE20PLUS Ambassadors).35 Map community leaders and recruit equitably across partners,working with them to build trust,gain commitments and mobilize additional champions.Closing Health Gaps through Collaborative Action7Guidance Co-create initiatives:Incorporate diverse perspectives,local knowledge and cultural context through co-creation to boost community adoption.37 Use human-centred design to involve community members and incorporate lived experiences.Community engagement is about learning and people will bring their personal views,experiences and emotions;come with an open mindset and harness this as a creative base for collective action.Allow for modifications as the work evolves to build trust and confidence.Embed an equal decision-making process:Active community participation requires individuals to be valued and heard.The governance model should enable community members to have influence over the initiatives direction,approach and outcomes,aligning with its commitment to equity.Be transparent about decisions,clearly articulating the rationale and process.Facilitate community leadership through broad outreach:Explore participatory methods to identify innovations.38 Crowdsourcing,such as challenge contests or“design-athons”,can generate more creative programmes.Platforms,such as the World Economic Forums UpLink,facilitate challenges to identify start-ups and innovations like YesSF.39 Involve the public at various stages,such as in judging panels or as contributors,to increase community involvement.Guidance Create conditions for sustainment:Invest in expanding the skills,knowledge and resources needed within the community to sustain the initiative.Use community capacity assessments to guide and co-create training initiatives in,for instance,data analysis,quality improvement,leadership or finance,as necessary.Combining these skills with long-standing infrastructure,such as monitoring and evaluation systems,supports communities to meet future needs and promote continuous improvement.Take time for collaboration:When starting out,communities may be at varying levels of readiness for change.Develop a phased approach that begins with relationship building,progresses as trust develops and empowers the community when sustained.Allocate additional time in less receptive areas for communities to gain trust.Learn from like-minded communities:Tap into the wealth of experience from similar communities.Foster peer coaching relationships to share experiences,successes and failures,promoting a culture of innovation and learning.Platforms,such as GHENs Partnership Platform,create space for initiatives to connect with one another.1.2 Co-create solutions and shift power to community1.3 Focus on capacity buildingTo create relevant,effective and sustainable programmes,there needs to be a balance between“top-down”and“bottom-up”perspectives.36 Organizations should be willing to work on equal terms,creating a platform for community voice in design and decision-making.When a backbone organization departs,the community can struggle to sustain initiatives due to lack of resources,time or skills.Place-based efforts should build the capacity and capability of individuals,organizations and the community to lead ongoing health equity work and sustain momentum.Closing Health Gaps through Collaborative Action8BOX 1BOX 2SPSP is a government-backed initiative aiming to disrupt disadvantage and improve well-being for children and families in 10 communities by 2029.40 Each community,supported by a backbone organization,convenes local partners across sectors to enable community-led change.Each partnership embeds the community voice in decision-making processes through local governance bodies.For example,the Far West Community Partnerships(FWCP)board is composed of the five aboriginal community leaders it represents.They use an ethical decision-making framework that combines data from the community and the government.Co-creation and community listening is a fundamental principle.In the city of Logan,some women had limited trust in healthcare providers,reporting language and cultural barriers,and therefore didnt access care during pregnancy.The community-driven,place-based Logan Together initiative facilitated a co-design process involving 500 women from diverse backgrounds and over 20 organizations.Four community“maternity hubs”were established with a spiritually and culturally safe midwife-supported care model.Communities also receive capacity-building funding to develop the skills and infrastructure to implement community action plans.For instance,the Gladstone Region engaging in action Together(GRT)initiative established data infrastructure to understand community needs.After facing increased human service demand in the community,GRT convened a working group with government,corporates and civil society to map current capacity,understand changing demographics and develop local planning solutions.With each partner sharing its data,the Gladstone Region Well-being Data Hub was established as a community-built and-owned data resource to identify and quantify community impacts.41 Early evidence from the 10 communities demonstrates improved well-being among children and families in these communities.For example,Logans maternal hubs led to a 42crease in the number of birth parents receiving nil or inadequate antenatal care,with estimated savings of more than AUD 500,000 million from reduction in birth interventions.42In Zimbabwe,the Chironde community faces high HIV(human immunodeficiency virus)infection rates among adolescents and children,early marriages and low attendance of mothers at antenatal care.The Unki Community Health Programme convenes cross-sector organizations,including a local faith-based organization,a young womens network,a local clinical partner and public sector entities,to promote community health through rigorous data analysis and community ownership.With funding and expertise from Anglo American,community partners collaborate with civil society groups to design,implement and monitor layered services.These include water,sanitation and hygiene(WASH)improvements and behaviour change communication,with targeted support packages for vulnerable groups.Programmes focus on capacity building,such as providing training to build and maintain WASH infrastructure,leadership and monitoring skills,peer mentorship and training for behaviour-change facilitators.As the community is predominantly Christian,trusted traditional authorities and faith-based leaders are engaged to co-create solutions and foster community buy-in,resulting in increased participation and behaviour change.The community-led governance model uses forums such as water point committees to make joint decisions with local expertise empowering the community to lead the changes they want to implement.Local data is used to inform programme focus and alignment by identifying evolving needs,adapting programmes through iterative design and creating long-lasting outcome change.The initiative has boosted community infrastructure,raising adequate sanitation coverage from 22%to 65%(and targeting 80%by 2025),and has improved uptake of clinical services.For instance,enrolment and retention in care for children and adolescents living with HIV has increased,with a 92%viral load suppression rate among those enrolled.Scale-up is planned for 2026,integrating with national and local public-sector frameworks.Stronger Places,Stronger People(SPSP)Australian government SPSP fosters community-led change by empowering local governance bodies,deepening community engagement and establishing adaptable infrastructure to address evolving needs.Unki Community Health Programme Anglo American The Unki Community Health Programme creates long-lasting change by collaborating with trusted local faith-based leaders,investing in capacity building and implementing community-led governance.Closing Health Gaps through Collaborative Action9Rigorous analytical approach2Place-based change should stem from a rigorous analytical approach to pinpoint the factors that maximize health and well-being and close the gap in long-term outcomes.The factors that lead to disparities are complex and,in many cases,established systematically over decades.While there is no simple solution,addressing certain factors may have a greater effect on health outcomes and disparities.To truly maximize impact on investment,organizations should make meaningful use of data analytics to understand the key factors that lead to healthy outcomes,continually monitor impact,and evolve programmes accordingly.Benefits Enhanced outcomes:By understanding which programmes yield the best results,programmes can allocate resources and investments to maximize impact on health outcomes.Continuous improvement:With clear goals and corresponding metrics,the impact of programmes can be monitored and data-informed adjustments made.Clearer communications:An analytical framework provides a common language between partners and a clear rationale for decisions to promote collaboration,alignment and accountability.Often,initiatives focus on more visible or less complex challenges.However,these do not typically improve health equity or health span.By leveraging population health science,theres an opportunity to maximize health impact by focusing on the key drivers of poor health and early death.2.1 Focus on the drivers that maximize longevityGuidance Focus on what matters most:To identify programmes that produce significant health improvements,an examination of the holistic factors and relationships that contribute to health outcomes is required,considering their prevalence and interaction.43 For example,substance exposure of mothers was linked to poor health outcomes for children.44 However,a long-term comparison of childrens cognitive and behavioural outcomes highlighted no difference with maternal substance use,while poverty had a substantial effect.45 This implies that resources should focus on addressing childhood poverty,rather than in utero exposure to substances.In many cases,research demonstrates that early-life and upstream factors are critical drivers of many adult outcomes,such as material deprivation,early childhood education and childhood adversity.This approach requires analytical capability;programmes should work with academic partners(such as universities,think-tanks and research agencies)to design pragmatic research that can inform implementation.Tailor initiatives proportionate to need:Programmes should balance universal population health interventions with targeted actions proportionate to population needs and levels of disadvantage.46 This approach balances equity and fairness,cascading to the most vulnerable,while allowing for diversity and difference.Closing Health Gaps through Collaborative Action10Each community has varying stakeholder perspectives,complex dynamics and differences in data access,integration and quality.Analysis must adapt to what is available,balancing sources and methods to comprehensively understand community needs.It must also leverage opportunities to build disaggregated data infrastructure and use new technologies to enable more effective predictive and evaluative programmes.Communities are dynamic efforts must adapt to their changing needs.Too often,impacts are evaluated at the initiatives end when there is little opportunity for course-correction.Monitoring and evaluation should be a continuous process to evolve programmes with community needs.2.2 Balance data collection methods and understand limitations2.3 Monitor outcomes and evolve programmes accordingly Guidance Employ quantitative and qualitative methods:Create data circles that combine both quantitative and qualitative data to generate actionable insights through disaggregation and triangulation of data,maintaining a comprehensive data matrix throughout.Utilize quantitative data,such as health and demographic statistics,to quantify the problem and disparities.Augment data,test hypotheses and understand lived experiences through community listening and ethnographic research,ensuring that representative and diverse voices foster deeper understanding.Recognize limitations:Inaccuracies,incomplete collection,inherent biases and reliance on anecdotal evidence are examples of factors that can compromise data reliability.47 There may be data gaps and limited interoperability across organizations.Recognizing and documenting these limitations helps prevent misinformed decisions or inaccurate conclusions.Scrutinize sources and validate findings using trusted leaders where possible.When embarking on a new programme,consider whether to strengthen current data systems or integrate a new sustainable system,rather than creating a parallel stream.Utilize AI tools:By analysing large datasets,AI algorithms and predictive modelling techniques can identify mortality patterns,forecast problems for high-risk disadvantaged populations,predict health behaviour and simulate targeted programmes.Be cautious of how the algorithm is trained and use ethical systems that have been developed using responsible AI principles.48 Guidance Develop a strategic framework for learning:Use a logic model to assess progress,outlining indicators for output and both short-term and long-term outcomes.This should balance measurable indicators(e.g.changes to infrastructure)with complex structural challenges affected by factors that are not always attributable to the work(e.g.health outcomes).Implementing a shared-measurement system for collective impact helps facilitate alignment and mutual accountability.Involve all partners,including community members,to define learning objectives.Create a culture that celebrates successes and encourages learning from failures to continuously improve and make progress.49 Measure the health and economic impacts of investments:A financial impact metric demonstrates how health or other economic indicators(e.g.economic activities and worker productivity)improve for each dollar spent on a course of action.50 This understanding of how programmes enable long-term community health equity can help shape strategies and be used for communications.Contextualize targets:Health inequities are affected by complex political and economic factors,making it difficult to measure their impact on health due to attribution,timescale,complexity and external factors.51 Understand programme limitations and embed these into the strategic framework to understand relative success.Closing Health Gaps through Collaborative Action11BOX 3BOX 4The life expectancy for New York City(NYC)residents dropped dramatically from 82.6 years(2019)to 78 years(2020)due to the pandemic,representing the biggest and fastest drop in lifespan in a century.It has yet to recover.Racial and ethnic inequities persist,with Black New Yorkers having the lowest life expectancy,at 76.1 years.HealthyNYC is a campaign convening cross-sector citywide stakeholders to increase life expectancy to 83 years by 2030,which would be the longest life expectancy ever recorded in NYC.52 To reach this goal,the city has defined specific mortality reduction targets for the seven leading causes(or“drivers”)of death in NYC,expected to avert an estimated 7,300 preventable deaths.These achievable,forward-planning goals were developed and modelled by epidemiologists and population health experts,who analysed local vital statistics data across race,place and time.HealthyNYC Strategy Maps are logic models aimed at reducing mortality rates by highlighting the evidence-based activities linked to the seven drivers of mortality for maximum impact.These maps emphasize the role of prevention in reaching each goal and consider cross-cutting factors such as access to care,climate change,mental health and social needs.Strategy Maps were created by NYC Health Department experts and informed by the agencys rich history of community and stakeholder engagement,and by neighbourhood-level surveillance data,programme evaluation data and current literature on the comparative mortality impact of specific programmes.Progress on the defined goals will be monitored and reported annually.Research and experience suggest two-thirds of KPs nearly 13 million members have at least one social factor impacting their health.54 Through cross-functional,multidisciplinary collaboration,the Kaiser Permanente Community Support Hub provides end-to-end social health support for members,identifying social needs and referring members to existing social health programmes offered by a robust network of thousands of community-based organizations and government assistance programmes.Interventions are personalized,proactive and targeted to improve health outcomes.Members social needs are understood through standardized screening surveys and by mining medical records using natural language processing(an artificial intelligence subfield).A social risk score is calculated from multiple personal,clinical and community-level attributes to identify individuals with social health needs and to inform targeted outreach.For example,KP is partnering with a national grocery delivery service provider to assess the effectiveness of free deliveries of healthy groceries to low-income members at high risk of diet-related diseases.55 Additionally,interventions are developed in response to specific community needs,identified through a robust tri-annual analytical process and dialogue with local partners.56 For instance,during the 2021 heat wave in Oregon,clinical data was used to identify members whose health conditions put them at heat-related health risk.KP collaborated with a local community partner to provide hundreds of air conditioners at no cost to these members the following year.57Real-world evidence and rigorous research measure the effectiveness of interventions.After a referral,follow-ups are performed to understand whether the service was utilized,to what extent the members needs were met and how effective the intervention was.Cohort studies are conducted for interventions and adjusted for confounding factors,ensuring that the improvements observed are statistically significant and attributable.This approach leverages the knowledge and experience of experts to create effective solutions for social needs and to support continuous learning.More than 2.6 million members were screened for social needs in 2023 and 350,000 members were connected to vital programmes that provided immediate and longer-term relief.58 The hub focuses on supporting low-to rising-risk members,while care teams continue to focus on helping members with more complex needs.HealthyNYC New York City governmentHealthyNYC aims to increase New Yorkers life expectancy to 83 years by 2030 by targeting the seven leading causes of death with specific,evidence-based activities,and reporting progress annually.Community Support Hub-Kaiser Permanente(KP)53The KP Community Support Hub uses a data-driven approach to deeply understand members needs,personalize interventions and measure effectiveness through robust real-world research and evidence.Closing Health Gaps through Collaborative Action12Purposeful and lasting partnerships3Place-based change should engage a diverse group of partners to leverage their differentiated expertise,perspectives and networks to create comprehensive solutions.Often,there will be several initiatives operating independently in a community.Lasting change cannot be achieved alone;it requires coordinated action across partners to address complex challenges.Aggregating siloed efforts into a place-based coalition requires a common understanding,shared commitment and unified approach.Benefits Amplified impact:Collaboration allows pooling of resources,expertise and influence for a more coordinated approach and scaling to larger systems.Greater innovation:Diverse perspectives spark innovative solutions,leading to more creative and effective strategies.Shared accountability:Each partners vested interest fosters commitment and sustained efforts.A whole-of-society approach involves all sectors government,business,academia,non-profits and community organizations.59 It acknowledges that health is influenced by multiple sectors and healthcare alone cannot address these matters.60 3.1 Take a whole-of-society approachGuidance Identify decision-makers and influencers:Perform ecosystem mapping to identify key initiatives,individuals and their interconnections.Prioritize key decision-makers,distinguishing between those with true decision rights and those who need to be informed.Exercise caution to avoid potential corruption risks and understand individuals motivations.Involve systemic and local actors:Addressing deep-rooted challenges requires multilayered interventions;convene partners from different levels to bridge grassroots efforts with broader systems.Early engagement with government aligns initiatives with policies and fosters shared commitment.Universities or other thought-leading institutions provide diverse perspectives from varied experience.In Philadelphia,for example,a diverse set of 40 organizations including local community groups,academia,businesses and the city and state governments convene quarterly to collectively address the Black maternal health gap.61 Identify a trusted backbone organization:Effective partnership requires navigating complex governance structures and diverse stakeholder priorities.Identify a trusted and respected convener to bring people together,challenge existing ways of working and deeply understand community needs.They should remain impartial and unbiased,with no conflict of interest between investments and overall health equity impact.Closing Health Gaps through Collaborative Action13Guidance Understand the ecosystem:Map the strengths and weaknesses of existing projects,organizations and people while undertaking ecosystem mapping.Understanding available and untapped resources helps gauge capacity,enable resource-sharing and inform investment strategies.Build on existing initiatives:Rather than starting from scratch,look for opportunities to scale existing community initiatives aligned to the vision.Catalyse partners influence as employers in the community to create a healthy work environment.65 Define roles based on strengths:Identify roles that leverage partners capabilities,aligning to organizational goals,their level of operations and their influence.Clearly communicate expectations and the level of expertise.Be realistic on whats possible,allowing for flexibility.Coordinating diverse stakeholder missions,priorities and efforts can be complex.Place-based efforts require participants to work towards a shared vision through unified approaches to tackle issues jointly.Successful place-based approaches take time to understand existing community dynamics.A strengths-based approach builds off existing assets,resources and capabilities to strengthen community ownership and maximize potential.64 Clearly defining roles and responsibilities of each partner,while aligning with interests and feasibility,fosters effective collaboration.3.2 Articulate a common place and purpose3.3 Leverage partners strengths and define rolesGuidance Set clear parameters on place:Clearly define a geographical focus area to align stakeholders.62 Balance breadth and focus to capture community nuance,while influencing the wider system.Aligning with local government boundaries can provide a well-established and replicable system.Focus on shared outcomes:Articulate an outcome-focused purpose that unifies partners for long-term goals.Build on the aims of current initiatives and define a common problem partners will probably have slightly different definitions.Utilize research,data and contextual understanding to develop a compelling story;it can be a useful tool to align stakeholders and mobilize resources,e.g.the Regional Blueprint for Inclusive Growth for the Greater Washington Partnership.63 Ground the coalition in shared values:Develop guiding principles that reflect collaborative ways of working and current approaches for engagement,and create a sense of collective responsibility.This should guide democratic and transparent practices,rooted in accountability,trust and co-ownership among partners.Closing Health Gaps through Collaborative Action14The supersetting approachFIGURE 3PrinciplesPartnersSupersettingKindergartenSchoolSupermarketLibraryMuseumSports clubIntegrationEmpowermentContextKnowledgeParticipationPublic sectorPrivate sectorPoliticiansAcademiaMediaCivil societySource:Cities for Better Health68BOX 5Tingbjerg is a public housing area located about eight kilometres north of Copenhagen city centre,characterized by high ethnic diversity and featuring signs of social vulnerability.Prevalence rates of chronic diseases are high compared to other parts of Copenhagen with almost 10%of adult residents diagnosed with Type 2 diabetes compared to an average of 4%for Copenhagen.Tingbjerg Changing Diabetes is a long-term community-based initiative that applies the“supersetting”approach the coordinated engagement of multiple stakeholders in multiple settings to attain amplified impacts from comprehensive interventions.67TCD has partnerships across the public sector,private sector,civil society and academia and spans health promotion,social development,housing,education,employment,environment,day care,culture and other sectors.Local stakeholders and untapped community resources are identified by mapping physical infrastructure,settings,organizations and social networks to inform the coalition and its programmes.In accordance with the supersetting approach,TCD partnerships rest on a foundation of mutual trust,shared values and principles.The partnerships therefore require less steering and are maintained through dynamic and loose governance structures.This helps legitimize individual partners to contribute whatever resources they possess when and where they can,and enables these to be used in the best way possible for the common good.The initiative started among a small“coalition of the willing”,and is gradually expanding to numerous partners over time.Unpublished findings from analysing national health register data indicate that Type 2 diabetes incidence rates among adult(18 )residents of Tingbjerg have stabilized,while still increasing in Denmark as a whole.The supersetting approach has proved effective in creating trust among stakeholders and community members and aims to be scaled to other neighbourhoods across Novos 40 “Cities for Better Health”.Tingbjerg Changing Diabetes(TCD)Novo Nordisk66 TCD is a dynamic,cross-sector partnership that mobilizes local resources and strengthens networks for collective community action to achieve positive and measurable changes in health outcomes.Closing Health Gaps through Collaborative Action15BOX 6A maternal death occurs almost every two minutes globally,with 95%occurring in low-and lower-middle income countries.69 MSD for Mothers aims to create a world where women do not die while giving birth,and operates in over 70 countries through several place-based initiatives.Each place-based initiative convenes diverse partners and key decision-makers to prevent childbirth-related deaths.In Nigeria,for example,MSD collaborates with a consortium of cross-sector stakeholders,harnessing their strengths to implement multiple interventions in quality maternity care and contraception.For instance,IntegratE is an intervention that expands contraceptive access through local,private drug stores.Co-funded by MSD and the Bill&Melinda Gates Foundation,it is delivered by five partners with unique roles in research,advocacy,training and digital infrastructure.70 Another effort,Saving Mothers Giving Life(SMGL),is co-funded by USAID and MSD.It has expanded access to high-quality,comprehensive maternity care in areas with high rates of maternal mortality,working closely with local government authorities,community-based organizations and an implementation partner,Pathfinder International.Critical to success is grounding the coalition in shared values and goals.For example,the MSD for Mothers Safer Childbirth Cities in the United States initiative aligns coalitions across 20 cities to make these cities safer,more equitable places to give birth.71 Each coalition adapts the objectives,partnerships and interventions to the local context.The national Community of Practice,led by the Association for Maternal and Child Health Programs,facilitates mutual learning and strengthens capabilities in coalition-building,stakeholder engagement and sustainability efforts.To date,MSD for Mothers has reached more than 30 million women worldwide,as part of MSDs goal to reach more than 50 million women by 2025.MSD for Mothers MSDMSD takes a holistic approach to improving maternal health through strong public-private partnerships,harnessing the strengths of each collaborator towards a shared goal.Closing Health Gaps through Collaborative Action16Sustainable and execution-oriented operating and governance models4Efforts should be designed with a strong operational backbone and“the end in mind”to bring about positive and lasting transformation across social,economic and environmental realms.Traditionally,many projects are time-limited and rely on short-term funding,with investors and donors expecting results within a few years.However,addressing complex social challenges often takes decades to show significant health improvements.Sustaining momentum beyond initial implementation requires ongoing commitment,ample resources and a robust operational backbone with a clear long-term goal.Benefits Increased likelihood of success:A shared commitment to the longer term increases the chances of achieving desired outcomes and making sustained,impactful change.Economic and social resilience:Establishing infrastructure enables communities to adapt to evolving needs more effectively and overcome adversity.Deeper commitment from partners:Setting upfront long-term expectations demonstrates commitment,defines an end goal for sustainment and encourages greater buy-in from partners.Creating and implementing place-based efforts requires coordination,time and effort to align the individual pieces.Often these efforts are under-estimated and under-resourced.72 Robust accountability systems and a coordinating team are essential for making progress and keeping the work a priority.4.1 Create a system of shared accountability and implementation rigourGuidance Create a team and supporting mechanisms:The expectation that collaboration can occur without supporting infrastructure is a frequent reason for initiatives faltering.73 A coalition requires a separate,dedicated backbone team to plan,manage and support the initiative,including facilitation,communications,data collection,reporting and administrative tasks.Closing Health Gaps through Collaborative Action17Guidance Plan for measurable“quick wins”:Balance long-term goals with shorter-term projects to demonstrate change is possible and align with investors shorter time horizons.A strategic learning framework is a useful tool to demonstrate how inputs drive to the desired outcomes.Establish proportionate governance structures:Effective governance structures help guide the initiatives direction,operationalize the vision and support accountability,without being overbearing.Tailor the governance framework to the collaboratives needs,culture,norms and available funding.Clearly document the purpose of each committee,the level of authority granted and whether it is time-bound or established to exist as long as the project.Align to project management best practice:Implement structures to facilitate alignment and track progress,including a comprehensive project plan,reporting processes and communication mechanisms.The project plan should align with the strategic learning framework,be co-developed with partners based on what is feasible and incorporate reflection periods for adaption.Establish a reporting structure to monitor goals and collective impact,enhancing internal and external storytelling.Pockets of excellence exist within our communities,but often these models remain unknown and struggle to continue when scaling to other communities.Often,those that succeed,plan for the desired legacy at the start and develop a model that is adaptable to other communities.Building trust and achieving meaningful impact requires time,yet organizations often face pressure to demonstrate immediate results.A phased approach can secure funder commitment through“quick wins”,while aligning the longer-term goal with broader system priorities enables coordinated efforts.4.2 Design the model with“the end in mind”4.3 Have a long-term plan,linking to broader system prioritiesGuidance Invest in underlying infrastructure:Develop scalable infrastructure that can grow with demand and evolve with needs,such as modular facilities and adaptable service delivery models.Customizable and replicable technology solutions are particularly effective for cost-effective scale up,community empowerment and providing quality data to inform programmes.Design with sustainable resourcing:Ensure consistent staffing and sufficient funding for sustainment and long-term planning.Resourcing should be designed to match workload and demand,so that the model can be effectively continued.This model should align with a phased approach for capacity-building,using initial resources to demonstrate impact and empower the community over the long term.Balance highly-skilled roles with opportunities for those with less time and experience,creating a more inclusive and scalable model.Identify opportunities for scaling during design:Identify similar communities where your intervention model can be replicated early but remain adaptable in the shorter term.This flexibility engenders relevance and responsiveness to changing dynamics,and empowers the community to adjust the model based on their evolving needs.Closing Health Gaps through Collaborative Action18 Align with health system priorities:Position the initiative to complement local priorities,such as government policies,to work collaboratively towards the same goals.Engage with local and regional governments during design or after initial impact to maintain alignment and discuss scaling possibilities.Identify funding streams early:Traditional models with finite funding linked to the financial year may not suit long-term collective action.Funding needs to be flexible to respond to changing community context.When designing the model,assess the long-term sustainability requirements and be creative with resourcing for example,by leveraging volunteers.For resources requiring continued investment,have a plan for funding considering different streams.Pursuing multi-year agreements promotes long-term sustainability,mitigating challenges stemming from disparate collaborators.The C/Can timelineFIGURE 4Pre-city selectionYear 1-3Year 3-5 Year 5-10 City submits applicationEstablishment of project structure and priorities,including stakeholder and needs assessment,solutions planning and project developmentCollaboration with C/Can local sustainability partners to execute projectProviding support to local and national authorities to embed change and measure impactSource:City Cancer Challenge Foundation75BOX 7Seven out of 10 cancer deaths worldwide occur in low-and middle-income countries(LMIC).74 Despite many efforts in cancer prevention,few LMICs have a cohesive long-term plan across the patient journey.C/Can leverages cities as catalysts for sustainable change,supporting local stakeholders across sectors to collectively design,plan and implement cancer solutions.With a multi-year growth plan,this approach prioritizes local ownership and sustainability,with the objective to scale city solutions to the regional and national levels.The model is underpinned by a collaboration between Roche and C/Can,driving shared accountability and implementation rigour.The framework includes several areas:Co-creation of a shared vision and goals,with clear objectives,areas of collaboration and engagement principles.An operating model for engagement at the global and city levels,with regular communication around planning and reporting.A monitoring and evaluation framework,including key performance indicators(KPIs),tools and a reporting structure.An external communications and engagement plan for events,speaking opportunities and external reports,as well as internal communications.A clear structure for onboarding,collaboration and support,with a designated point of contact at the city and global levels.A governance structure,outlining the accountabilities and decision-making power of the people involved in the partnerships,and limiting escalation by design.C/Can and its partners,including Roche,have reported impacting the lives of 67.5 million people,actively supporting more than 4,200 health workers to drive transformative change,and pioneering the development of more than 100 unique cancer-care solutions.City Cancer Challenge(C/Can)C/Can,Roche and other partnersC/Can has established an effective operating and governance structure to work cohesively across cities,following a plan for scaling and long-term funding through multiple partners.Closing Health Gaps through Collaborative Action19BOX 8In 2019,70%of deaths in Bangladesh were attributed to NCDs.76 A partnership between the Bangladesh Ministry of Health and Family Welfare(MOH&FW),Medtronic LABS and BRAC addresses healthcare inequity for NCDs,focusing on diabetes and hypertension in underserved areas.The replicable model was designed for scaling with:Screening and follow-up provided by BRACs extensive team of community health workers(CHWs).The governments investment in NCD management infrastructure for diagnosis and treatment.Support through Medtronic LABS adaptable modular digital platform,SPICE.These elements support cost-effective expansion,empowering communities and encouraging quality care within existing primary healthcare structures.The initiative leverages digitized care pathways,community-based screening,risk-based management and data-driven decisions to provide prevention-focused care for NCDs.By integrating technology into existing workflows,it enhances current systems without introducing new ones.Efficient implementation is enabled through clearly defined roles,mobilization of community resources and digital tools.The initiative aims for countrywide expansion with gradual geographic spread,collaborating with additional partners,and with initial funding from the health ministry,Medtronic LABS and BRAC.This allows for tangible results and complete technology integration while funding for broader implementation.The project had expanded across 175 facilities in two districts by 2023,reaching more than 126,000 people,with 92,000 screened,33,000 referred for diagnosis and 15,000 enrolled.360-Degree NCD Care Model BRAC,Medtronic LABS and Bangladesh Ministry of Health The partnership has designed and implemented a scalable,technology-based model to address healthcare inequality for non-communicable diseases(NCDs),with a long-term plan for expansion and funding.Closing Health Gaps through Collaborative Action20ConclusionLocal needs are constantly changing with ever-evolving challenges.While some areas benefit from substantial investment,others remain underserved.In the prevailing resource-constrained environment,addressing these challenges can be a struggle.Place-based change is an acknowledgement that all change is local and offers hope that more can be achieved that individuals can live longer,more fulfilled lives.Through collaboration across government,private sector,academia and the community,coalitions should work towards a common purpose,supported by adequate resources,dedicated leadership and shared power and decision-making.By enabling community ownership,taking a rigorous analytical approach,building purposeful partnerships and designing operating and government models to last,initiatives can take a holistic approach that maximizes impact.A growing number of effective,multi-sector collaboratives are emerging as promising examples.The time is right to build momentum.Past efforts and a growing body of knowledge on effective approaches are available to inform impactful initiatives.Public and private funding is increasingly supporting community collaboration.Globally,there is growing commitment to change and a renewed opportunity to include the communities left behind.Ultimately,it is the collective opportunity of the global community to recognize the impact of place on health outcomes and work towards equitable solutions.Embracing place-based approaches can create healthier and more resilient communities.It is time to seize this opportunity to drive impactful change,so that people can thrive in their own unique places.Closing Health Gaps through Collaborative Action21ContributorsAuthors Hannah Blackman-MackManager,Deloitte;Project Fellow,Global Health Equity Network,World Economic Forum Ty GreeneLead,Health Equity,World Economic ForumDavid RabinowitzPrincipal,DeloitteSarah ShierSenior Manager,Deloitte Report advisers Jay BhattDeloitte Centre for Health Solutions,DeloitteShyam BishenHead,Centre for Health and Healthcare,World Economic ForumCyril EngmannSenior Director,Integrated Program Quality&Impact,PATH;Neonatologist&Professor,Department of Pediatrics&Global Health,University of WashingtonMary-Ann Etiebet Executive Director,Merck for Mothers,MSDJudith MooreHead,Healthcare Access and Outcomes,World Economic ForumBola OwolabiDirector,National Healthcare Inequalities Improvement Programme,NHS;Vice-President,Royal Society for Public HealthAlexandra PlowrightHead of Community Health and Well-being,Anglo AmericanGreg RehPrincipal,DeloitteClosing Health Gaps through Collaborative Action22Acknowledgements This white paper is the culmination of a comprehensive and inclusive stakeholder consultation process and would not be possible without the support of key partners.The authors thank the following members of the World Economic Forums Global Health Equity Network for their insightful contributions and feedback:American Heart Association:Gerald Johnson,Eduardo SanchezBoston University:Sandro GaleaBP:April ClarkBRAC:Imran Ahmed Chowdhury,Chris LyneC/Can:Harold Cottin,Stephanie ShahiniCity of New York:Georgia Kinsley,Jenna Mandal-RicciDeloitte:Josh LeeIndependent adviser:Cristina Stefen Kaiser Permanente:Pamela Schwartz,Anand Shah,Vivian Tan,John VuLwala Community Alliance:Katie Gray,Lauren Marlar,Ash RogersMedtronic LABS:Garima Sahai,Megha Kumar,Ruchika Singhal,Anne StakeMSD:Temitayo ErogbogboNovo Nordisk:Paul Bloch,Louise Hesseldal,Jo Jowell,Lykke SchmidtNational University of Singapore:Rayner TanTakeda:Bob Imberman,Linn ParrishUnited Way:Nicole CooperUS Deloitte Health Equity Institute:Nicole KelmWatsi:Kimmy Coseteng,Mackinnon Engen,Emily Michael,Jane Ngige-MuturiProductionMichela Liberale Dorbol Designer,World Economic ForumMadhur Singh Editor,Astra 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Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of PolicyW H I T E P A P E RS E P T E M B E R 2 0 2 4Images:Getty Images 2024 World Economic Forum.All rights reserved.No part of this publication may be reproduced or transmitted in any form or by any means,including photocopying and recording,or by any information storage and retrieval system.Disclaimer This document is published by the World Economic Forum as a contribution to a project,insight area or interaction.The findings,interpretations and conclusions expressed herein are a result of a collaborative process facilitated and endorsed by the World Economic Forum but whose results do not necessarily represent the views of the World Economic Forum,nor the entirety of its Members,Partners or other stakeholders.ContentsForewordExecutive summaryIntroduction1 Streamlining regulation:The role of policy in unlocking the promise of genomics and human health2 Scaling sustainable alternatives:The role of policy in delivering sustainable alternatives for hard-to-abate industries3 Commercialization and adoption:The role of policy in shaping sustainable food and agricultural systemsConclusionContributorsEndnotes34561015202122Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 2ForewordThe blending of the digital,physical and biological worlds is reshaping society.Policy is at the forefront:for decades,policy has been structuring the responsible transition of biology into a commercial technology.As tools evolve to more reliably harness biology,the interplay between policy and innovation has never been more important to safeguard the human-centred transition to a resilient,bio-based economy.Although not always recognized,this interplay is not new:effective policies have propelled groundbreaking technologies from the laboratory to widespread societal use for centuries.Without the foundational support of well-crafted policies,even the most promising innovations risk stagnation or failing to reach those who would benefit the most.As the journey to a bio-based economy advances,policy will be key in fostering resilient and equitable economies through the commercial application of bio-innovation.At the core of this transition,the recipe for success,is biology.Bio-innovation has provided us with rapid responses to human health crises and solutions to reverse diminishing agricultural productivity due to climate change.Even Ozempic,the diabetes and weight-loss drug,is a product of biomanufacturing.Beyond human health and agricultural applications,bio-based approaches have also provided early-stage alternatives for many daily necessities historically associated with polluting or high-emissions production.In addition to technological progress,policy is the key driver that will define the magnitude of the impact that bio-innovation is capable of achieving.As we build a future where bio-innovation increasingly delivers better commercial alternatives,without increased policy intervention,bio-solutions will be incapable of delivering upon their ultimate potential.The goal is clear:to ensure that the innovations not only of today but of tomorrow as well can effectively address the pressing challenges facing humanity and the planet.By examining past successes where timely and supportive policies have saved lives and improved environmental outcomes,it becomes clear that evolving these frameworks and delivering them efficiently is crucial.Bio-based approaches will continue to provide the tools and means necessary to deliver innovative solutions to human health,industrial practices that shrink our reliance on extractive processes,and the ability to reimagine sustainable food systems.In a world fast approaching climate tipping points where society requires practical human-centric alternatives,what role will policy play in amplifying bio-solutions for better planetary and human outcomes?This paper spotlights pivotal policy interventions in three key areas for biotech application:health;energy and chemicals;and food and agricultural systems.Jelena Bojovi Director,Centre for the Fourth Industrial Revolution SerbiaAccelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy September 2024Brynne Stanton Lead,Bioeconomy,World Economic ForumDidier Toubia Chief Executive Officer,Aleph FarmsAccelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 3Executive summary For centuries,innovation has fuelled economies and given rise to new markets and industries,bridging gaps in education,healthcare and human prosperity.For example,the digital transformation has permeated every aspect of daily life,redefining how people communicate,work,connect,build communities and engage with the world.The internet as it exists today was not only a paradigm-shifting innovation,but also constituted a major political achievement owing to visionary policy-makers who created the legal and policy frameworks that enabled its exponential growth.1Well recognized for establishing rules and regulations for the betterment of society,policy sets forth guidance and best practices across organizations,governments and institutions.Bio-solutions represent one area where the digital,physical and biological worlds are coalescing to introduce bio-based options into everyday life,with policy playing a central role in making this all possible.2 Due to rapid technological progress and historical investment in incumbent processes and products,enabling policies for bio-solutions have,at times,struggled to keep pace with innovation.The catalytic role of supportive policies around bio-solutions can take many forms,but increased attention is necessary to ensure that innovation can efficiently deliver societal interventions,especially in those cases where there is no Plan B(e.g.diseases without viable treatments,or commercial alternatives that operate outside of planetary boundaries).This paper highlights the opportunities for,and the potential impact of,enabling policies for biotechnology.It focuses on the supportive policies that have provided significant societal and environmental benefits.It explores how:streamlined regulatory approval saved millions of lives during the pandemic.regional policies are opening up opportunities to move beyond petroleum-derived chemicals.the powerful synergy between policy and innovation is helping biotech crops restore the planet and reduce emissions.Furthermore,it spotlights areas where policy development could serve as a multiplier for impact,along with the key obstacles that must be addressed to enable biology to provide transformative solutions more effectively for humanity and the planet.Policy is a fundamental enabler of the tech-driven bioeconomy.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 4IntroductionPolicy:Bio-innovations fundamental enabler.The capacity to read,write,edit and,increasingly,functionalize DNA is expanding the reach of application areas for the tech-driven bioeconomy.3 Up to 60%of physical inputs to the global economy could be produced with biology.4 Typically taking the form of a law,regulation,procedure,administrative action,incentive or voluntary practice of government and other institutions,the role of policy in amplifying innovation is often underemphasized and undervalued.This paper shines a spotlight on the importance of policy in amplifying bio-solutions,as well as areas that can benefit from enhanced policy focus,to ultimately realize the full potential of a bio-based economy for society and the environment.Agility in policy-making is crucial to promote a responsible transition:it is imperative to enable the unforeseen potential of emerging technologies,and to ensure policies can better co-evolve with the technology itself while paving the way for responsible,human-centred adoption.The following sections highlight examples of the key bio-innovation areas underpinned by existing and/or emerging policy frameworks.These policies have been instrumental in enabling bio-solutions to save millions of lives,boost agricultural productivity and deliver sustainable energy alternatives.These cases bring to the forefront the significant societal benefits that can result from the harmonization of policy and innovation,while highlighting further areas of development for transformative outcomes.At a time when the capabilities of programming biology are rapidly evolving,bringing policy to the forefront of the discussion has never been more crucial:it is through policy that a responsible,human-centred transition will take place to deliver upon all that bio-innovation has to offer.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 5Streamlining regulation:The role of policy in unlocking the promise of genomics and human health1Did you know?Emergency regulatory approval of the COVID-19 mRNA vaccine saved nearly 20 million lives in the first year of use.And what else?Policies supporting the sharing of data on human health can help facilitate better patient outcomes through genomics.?Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 6In December 2019,COVID-19 triggered a human health crisis and impacted lives in ways that changed how people worked,lived and socialized,and claimed over 3 million lives in 2020 alone.5 Given the nature and scale of the crisis,the need for innovation to deliver immediate action across the globe was felt like never before.An innovative solution had been decades in the making:the ability to de novo synthesize RNA(ribonucleic acid,a molecule essential for most bodily functions),as both public and private biotech research and development(R&D)were pushing the boundaries of how mRNA(messenger RNA,which carries instructions for cells)could be deployed in vaccines(Figure 1).Spurred by data-sharing portals such as the COVID-19 data portal,6 and owing to decades of foundational R&D,mRNA vaccines for COVID-19 were produced in record time of less than a year.7 The unprecedented development of mRNA vaccines and the vaccination of millions of people was an innovation triumph that was instrumental in controlling the COVID-19 outbreak.mRNA vaccine commercial productionFIGURE 1Yet,the scientific innovation behind mRNA vaccines alone would have been insufficient to address a human health crisis of this magnitude.On 18 December 2020,the Food and Drug Administration of the United States(US)issued an emergency-use authorization for the Moderna COVID-19 vaccine.8 Policy,and in particular emergency-use approval,was the real triumph that permitted vaccines to address the pandemic,the groundwork for which was being laid for decades before.9 In that first year alone,vaccinations are estimated to have saved nearly 20 million lives.Since their introduction in 2020,COVID-19 vaccines have reduced deaths due to the pandemic by at least 57%.10 Beyond fast-track approvals,long-term enabling policy frameworks including accelerated approvals,priority reviews,as well as breakthrough therapies are encouraging an influx of innovative bio-solutions for patients in need.11 Such an approach has enabled a fundamental shift away from a single,emergency-focused policy response to a more comprehensive,ongoing policy framework capable of addressing a multitude of healthcare challenges.After designing in silico based on the intended target,mRNA vaccines are produced using enzymes in large-scale reactions.The resulting mRNA is then stabilized,packaged and purified through a series of downstream steps to create the final mRNA vaccine that is delivered to patients.Design1TranscriptionmRNAPurification,stabilization and packagingmRNA vaccine234567Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 7And while public views on vaccines still vary and vaccine distribution remains inequitable,12 without prompt policy intervention,the outcomes and the number of lives saved during this critical moment in global history would have been drastically different.Beyond emergency regulatory approvals,policy has also played an instrumental role in data sharing to drive forward scientific innovation.As technology advances,data sharing remains central to scientific and medical progress.Advances such as the Human Genome Project,CRISPR-Cas9 and AlphaFold illustrate how sharing data across borders,institutions and organizations drives breakthroughs in biotechnology for the betterment of humankind.For decades,human genomic data has promised to revolutionize disease diagnosis,treatment and prevention through personalized healthcare(Figure 2).One area holding tremendous potential is the application of genomics to treat cancer and rare diseases.Cancer continues to be the foremost cause of mortality globally,responsible for nearly 10 million deaths in 2022,equating to approximately one-fifth of all global deaths.13 At the same time,7,000 types of rare diseases affect 300 million people around the world.14Benefits of sharing health and genetic dataFIGURE 2Secure sharing of health and genetic data provides benefits for citizens by advancing the healthcare system.At the same time,trustworthy secondary use of health and genetic data for research creates opportunities for new discoveries.Empower citizens to have control over their health and genetic dataBetter diagnostics and treatment lead to improved patient safety,continuity of care and improved healthcare efficiencyBetter health and genetic data policy provides greater opportunities for research and innovationAssist policy-makers and regulators in accessing relevant health and genetic dataFacilitate access to health and genetic data for innovators in the industryGrant access to health and genetic data to researchersEnable healthcare professionals to have access to relevant health and genetic dataHealth data from medical devicesHealth and genetic dataElectronic health recordsAccelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 8Despite its promise,leveraging human genomic data for better patient outcomes faces significant obstacles,including high costs,storage and computational requirements,as well as the challenge of integrating genetic data with other clinical data,to name a few.The government of the Republic of Serbia is laying the foundation for a successful integration of genetic and health data.15 Legislation enacted in October 2023 supports the creation of a biomedical data registry and a centralized national electronic health record system.16 Other consortia,such as the Global Alliance for Genomics and Health,17 are laying the foundation for responsible use of genomic data by setting standards for responsibly collecting,storing,analysing and sharing genomic data.Safeguarding responsible use of genomic data for healthcare and research is of paramount importance in ensuring that genetic data can be used ethically and optimally.Other efforts,such as Together for CHANGE(Changing Healthcare for People of African Ancestry through an InterNational Genomics&Equity initiative),are aimed at addressing inequities in genomics research.18 With the goal of establishing the most extensive genomics research database of African ancestry to date,the project uses de-identified genomic and phenotypic data from up to 500,000 volunteers.Such coalitions underscore the importance of creating more streamlined frameworks for collaboration to ultimately accelerate the pace of bio-innovation and expand their beneficiary base with targeted and tailored therapies.Other efforts aimed at addressing the barriers associated with cross-border data sharing are also being implemented.For instance,China recently introduced new regulations aimed at unlocking the value of data for cross-border transfers.19 As countries and institutions continue to evolve policies and practices for cross-border data management and flow,data sharing will continue to play a pivotal role in delivering scientific breakthroughs for better,more equitable outcomes for patients.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 9Scaling sustainable alternatives:The role of policy in delivering sustainable alternatives for hard-to-abate industries2Did you know?Policies in support of biological production of everyday chemicals and energy are gaining steam and reducing greenhouse gas(GHG)emissions by up to 90%.And what else?While biology is projected to deliver significant emissions reductions at scale,without more policies to level the playing field with incumbent technologies,many innovative bio-solutions will fall short in delivering upon the extent of their planetary impact.?Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 10Beyond improving human health outcomes,biology can be applied in the production of everyday products to deliver more environmentally conscious alternatives.20 Owing to the inherent programmability of the genetic code,biology has long been applied as commercial technology in the production of many of the goods used on a daily basis.21 One area where biology has delivered myriad alternatives at scale is in the production of common everyday chemicals(Figure 3).22 For instance,commercial bio-production of organic acids has been shown to reduce GHG emissions by up to 90%as compared to conventional chemical synthesis and fossil-based processes.23 Enzymes also play a significant role in commercial chemical production,and enzyme-produced bio-ingredients have been estimated to reduce carbon dioxide(CO2)emissions by as much as 23%.24Bio-based production of chemicalsFIGURE 3Source:Gabrielli,P.,Rosa,L.,Gazzani,M.,Meys,R.,Bardow,A.,Mazzotti,M.,&Sansavini,G.(2023).Net-zero emissions chemical industry in a world of limited resources.One Earth,6(6),682-704.https:/doi.org/10.1016/j.oneear.2023.05.006Bio-based processSugar,bio-waste and/or side streamsEngineered microbesFermentationDownstream processingBio-based productDrillingDistillationRefiningChemical conversionPetrochemical productFossil fuel-derived/traditional processBio-based chemicals can be produced from sugar or other waste,and other side streams.Commercial-scale fermentation with optimized microbes is followed by downstream processing to deliver the desired bio-based products.Traditional alternatives make use of drilling to extract fossil-fuel substrates,which are then distilled,refined and chemically converted to render the final product.The chemical industry is currently responsible for 6%of global GHG emissions.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 11Historically and still today,the commercial synthesis of chemicals has relied on petroleum-derived feedstocks.25 Commercial production of chemicals requires high temperature and pressure,consumes fossil-derived energy,and generates significant quantities of hazardous waste.Amid growing environmental concerns,regional policies now support the adoption and scaling of bio-based commercial practices as a sustainable alternative.26 While bio-based processes have been running at scale for decades,commercial adoption remains relatively niche as compared to incumbents.This is one area where policy can play a significant and influential role,and policy-makers are responding.With a call for supportive regulatory frameworks and more European-based financing opportunities,the European Commission has set forth greater efforts in expanding European biotechnology and biomanufacturing.27 Governments have also taken notice and implemented procurement policies,such as the European Green Public Procurement(GPP)criteria,which prioritize bio-based products and stimulate market demand to support industry growth.28Yet despite these and other policies aimed at supporting commercialization of bio-solutions,reaching commercial scale still requires significant capital expenditure and production capacity remains in high demand.29 The role of policy in accelerating the transition away from extractive technologies to a bio-based economy cannot be overstated.Support tools such as the Pilots4U open database,a network of open-access pilot and multipurpose demo-infrastructures in Europe,aims to help bio-based processes reach commercial scale by highlighting available infrastructure assets.30 Given the lack of cost parity and the significant costs associated with commercializing bio-based technologies,as well as the sheer scale of manufacturing required,additional policies in the form of financial incentives such as tax credits and grants encouraging investment in bio-based industries are still needed.These and other incentives will go a long way in offsetting the capital-intensive costs associated with R&D,technology adoption and infrastructure development,as well as commercial deployment and scaling.Biologys ability to deliver game-changing production alternatives isnt limited to chemicals,as bio-innovation has long been applied to meet energy needs across industries.One area receiving renewed enthusiasm is the application of biotechnology to sustainably produce aviation fuel.The global aviation industry is estimated to be responsible for human-induced CO2 emissions totalling 2.5%,31 and air travel shows no signs of slowing:flights are expected to surpass 16 million by the year 2050,representing an increase of 44%from 2019.32 And while the aviation industry targets net-zero by 2050,drastic advances in sustainable aviation technologies will be required to decouple the industrys growth from carbon emissions.33 Sustainable aviation fuel(SAF)represents a promising path to reduce emissions and consists of bio-based and synthetic fuels that serve as“drop-in”replacements for jet fuel.34 Biology can be employed in the production of SAF,which can be produced from various biological inputs and production methods(Figure 4A).35 Current estimates suggest that SAF can reduce the industrys emissions by 80%as compared to conventional jet fuel.36 However,SAF usage made up only 0.2%of aviation fuel consumption in 2023.37 Formidable obstacles must be overcome in order to realize the industrys short-and longer-term emissions goals with SAF.38 Increasing supply and adoption as well as excessive costs remain at the forefront of these challenges,as SAF costs are currently 120-700%higher than those of conventional jet fuel.39 Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 12Powering aviation and energy needs with bio-solutionsFIGURE 4APolicy will continue to influence commercialization and adoption of SAF(see box below).In addition to grants that support early-stage R&D,40 mandates aimed at increasing demand have also started to take shape in Europe41 and beyond.42 Further subsidies and direct financial support aimed at lowering production costs and facilitating infrastructure development(investments totalling$1.45 trillion will be required to deliver the quantities needed),43 as well as sustainability mandates for the aviation industry,will all be necessary to scale SAF production to meaningfully decarbonize aviation.The role of policy is paramount in ensuring that SAF is both affordable and widely adopted across the industry.Policy spotlightReFuelEU Aviation promotes the increased use of SAF as the single most powerful tool to decrease aviation CO2 emissions.The measure is part of the“Fit for 55”package aimed at achieving a 55%emissions reduction by 2030.44 It mandates that aviation fuel suppliers progressively increase the proportion of SAF blended with conventional aviation fuel at airports in the European Union.45 Policy is also being applied to enable biology to meet energy demands in areas extending beyond aviation.Energy and heating represent another area that has long relied on fossil inputs:in 2019,energy and heating generated 34%of global GHG emissions.46 Disruptions to natural gas supply chains have only further extended the need to identify alternative sources.By capturing methane from waste streams,biogas represents an energy alternative that holds the potential to reduce GHG emissions by up to 90%to generate electricity and heat (Figure 4B).47 TapressureThis is a schematic representation of various sustainable aviation fuel(SAF)production processes employing bio-innovation.Biological inputs and processes are deployed across a variety of SAF production methods.Raw materialsVegetable wastesMunicipal organic wastesEnzymatic cocktailCO2CO2 sunlightSAFSAFSAFSAFSyngasSugarsMicroorganismsAcetogenic bacteriaAlgaeProcessesBio-innovation in sustainable aviation fuelsNote:Ta:Tankage pressure Source:Segura,A.,Jimnez,L.,&Molina,L.(2022).Can microbiology help to make aviation more sustainable?Microbial Biotechnology,16(2),190194.https:/doi.org/10.1111/1751-7915.14191Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 13Powering aviation and energy needs with bio-solutionsFIGURE 4BHow does it work?Processing of agricultural waste to break down organic matter results in the production of biogas,which is primarily composed of methane(CH4)and carbon dioxide(CO2).Refined from these products,biomethane represents a concentrated,storable and versatile energy source with promising GHG mitigation potential.The chemical composition of biomethane is very similar to methane,and biomethane can be seamlessly integrated into existing gas infrastructure.However,before biogas can meet growing electricity demand and heat homes,costs must be reduced significantly.Limited financial incentives and subsidies hinder cost competitiveness against fossil fuels,impeding broader market adoption.The Biomethane Decree of Denmark represents a supportive policy aimed at boosting production of biomethane by 2030 and replacing 100%of natural gas imports with biomethane while also allocating incentives to support its production.48 Policies in other countries crafted in a similar vein will be essential to achieving biogas cost parity with extractive production methods,and to establish biogas as the sustainable energy solution of the future.Akin to the other bio-innovations that have yet to make their way into the commercial mainstream,a combination of strategic deployment,supportive policies and technological advancements is needed to maximize the environmental and economic benefits of biogas.Inputs such as livestock waste,energy crops,waste water and food waste can be used in the production of biogas.By making use of an anaerobic digestor,anaerobic microbes process these inputs into biogas,which provides a sustainable and alternative source of heat and electricity.Livestock wasteCropsHeatElectricityBiogasAnaerobic digesterDigestateWaste waterFood waste Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 14Commercialization and adoption:The role of policy in shaping sustainable food and agricultural systems3Did you know?Application of biotech crops has delivered the equivalent of removing 15.6 million cars off the road for a year(49%of registered cars in the United Kingdom(UK).And what else?While bio-based production of food protein can reduce emissions by up to 90%,in the absence of more policies that favour adoption and decrease production costs,bio-based protein production faces an uphill battle in making its way onto more consumer plates.?Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 15For decades,biotechnology has been applied to food and agricultural systems to decrease the reliance on fossil inputs across many fronts,and to produce options that are healthier and/or have lesser environmental impact.Alongside myriad policies to responsibly deploy game-changing innovations across the food system,much ground remains to be covered before GHG emissions associated with agriculture and food production can be meaningfully reduced.Even so,policies put into place across regions have ensured that these innovations are already making progress in sustainably feeding the population and providing healthier choices through innovative,bio-based approaches.Delivering healthier,bio-based optionsThe European Commission has agreed on regulations that limit the amount of trans-fat in foods on the European market.49 Bio-innovation provides a safe and environmentally-friendly solution for food producers to meet this demand by using enzymes that render oil free of trans-fats.50 Historically produced through industrial chemistry,the biological process eliminates the need for chemicals and post-bleaching,reducing the generation of wastewater.In agriculture alone,bio-innovation has provided crops with many important self-sustaining properties(Figure 5A);biotechnology continues to improve sustainability of global agricultural practices in ways that are not always appreciated by the consumer.For example,emissions reductions from decreased production of small-molecule chemistries(think pesticides and herbicides)and the reduced number of field operations required when applying biotechnology to agriculture,continue to reduce the fossil reliance of existing agricultural practices and have delivered considerable benefits to the planet.Application of biotechnology in agricultureFIGURE 5AThe gene providing resistance is isolated from the hostThe resistance gene is optimized for activity in the target crop The optimized resistance gene is inserted into the crop genomeThe resulting crops demonstrate increased resistance to pestsAccelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 16How does it work?Commonly derived from petroleum,the production of herbicides and pesticides requires fossil fuel inputs.Biotechnology has enabled crops to become more self-sustaining.The results include reduced pesticide and water usage as well as improved harvests and yields in those regions where policy supports responsible adoption.Supportive policies in regions such as Africa,India,China,US and Brazil(Figure 5B)have delivered reductions in pesticide use ranging from 50%to 70%for specific crops,e.g.cotton containing a gene providing insect resistance(Figure 5B).These approaches are estimated to have achieved a significant reduction in the release of GHG emissions over the last two decades,equivalent to removing 15.6 million cars from the road for a year(49%of registered cars in the UK).51The reduction in pesticide use due to biotech crops has been significant,contributing to more sustainable agricultural practices globally.52 Between 1996 and 2016,the use of genetically-modified(GM)crops reduced pesticide spraying by nearly 671,400 tonnes,equating to an 8.2%reduction.This has decreased the environmental impact associated with crop protection practices by 19%as measured by the Environmental Impact Quotient(EIQ).53 However,these achievements have only been made possible by corresponding policy frameworks that facilitate their commercial use.54 Policies in support of agricultural biotechnology worldwideFIGURE 5BChina has approved the commercial planting of new biotech crop varieties,including corn and soybeans,to address domestic agricultural challenges and reduce dependence on imports.This strategic move aligns with Chinas goal of achieving food security while optimizing agricultural practices and has been particularly successful in regions with high pest pressure.As the leading producer of biotech crops such as soybean and cotton,Brazil has developed a range of policies to support the adoption of biotech crops,balancing innovation with safety to ensure that these crops are safely integrated into the agriculture sector.As a result,growers have benefited from increased agricultural productivity and efficiency.Brazil is also experiencing an increase in the number of applications for registration of biopesticides,forcing regulatory authorities to adapt procedures and legislation to meet the unique requirements of products.As a result,the number of registered biopesticides has skyrocketed,with an overall increase of 404%in recent years.Kenya has lifted a ban on biotech crops,allowing for their cultivation and importation.This policy change aims to enhance food security and bolster agricultural resilience in the face of climate change and other challenges.In the US,federal agencies like the US Department of Agriculture consistently fund research in agricultural biotechnology to develop crops with new capabilities.These efforts focus on addressing pressing challenges such as climate change impacts,pest resistance and nutritional deficiencies through innovative biotechnological solutions.With an eye on creating an enabling environment for research and innovation in identifying solutions to sustainably feed the population,the European Commission has adopted a proposal for new regulations on plants produced through novel genomic techniques,part of the broader legislative efforts to support the EUs farm-to-fork and biodiversity strategies.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 17As climate conditions escalate,so too do the challenges associated with preserving and maintaining food and agricultural systems.Agricultural biotechnology plays a pivotal role not only in ensuring adequate food supply,but also in addressing the growing risk of crop extinctions worldwide due to climate change-related factors.For instance,biotechnological tools offer effective interventions to preserve crop cultivars and combat biodiversity loss.Addressing biodiversity lossAbout two plant species vanish each year on average,a rate that is 500 times faster than the natural rate of extinction.59 Species critical to human food sources,such as bananas,papayas and certain cocoa varieties,are also facing heightened threats.Inbreeding depression60 among endangered species further diminishes genetic diversity,hastening extinction.Biotechnology,particularly through gene editing,provides rapid solutions to mitigate biodiversity loss.61 While regulatory frameworks have extended the ability of biotechnology to provide meaningful intervention in combatting biodiversity loss,policy-makers continue to grapple with related crucial questions such as the conservation status of modified species and the appropriate regulatory bodies.Harmonization of policies across regions is essential in preserving biodiversity and responsibly safeguarding food security for the growing population.At the same time,the stress on the food system to provide enough nourishment for the growing human population remains one of the biggest challenges facing humankind.Similar to agriculture,life-science breakthroughs can also be applied to the way that food is produced beyond the cultivation of crops,such as cultured post-harvest protein.Despite global efforts,over 9%of the worlds population experienced chronic hunger in 2022 alone.62 A fundamental shift in trajectory is essential to achieve the United Nations Sustainable Development Goal of Zero Hunger by 2030.63 Adding to the growing climate challenges,food and agriculture contribute to over one-third of global GHG emissions,64 with animal agriculture alone responsible for 15-20%.65 Emissions from livestock production have reached an all-time high,and biotechnology is increasingly being deployed as a source of protein innovation for consumers.Furthermore,the demand for protein is set to double by 2050.66 As with other bio-innovations,coordinated policy solutions are required to transform current food systems and responsibly deliver such innovations to the masses.Such tools are being applied to expand the portfolio of offerings and reduce environmental impact;67 food protein production through fermentation has shown much promise,and in some instances is associated with reducing GHG emissions by up to 90%,68 and reducing land and water usage overall.69 Dairy proteins,egg proteins,meat proteins and collagen are just a few now being produced commercially in an animal-free fashion.The production of animal-sourced foods from cell culture represents another tool to help meet the growing demand for sustainable and ethical food sources(Figure 6).Produced through cellular agriculture,the carbon footprint of cultivated meat is 92%lower than that of conventional beef,44%lower than pork and equivalent to chicken when produced using renewable energy sources.70 Cultivated meatFIGURE 61234Cultivated meat is typically produced through harm-free harvesting from the animal.The resulting animal cells are grown in a lab environment and further processed to produce cultivated meat products for the consumer.Cell-based products Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 18The environmental benefits extend well beyond carbon emissions,as lab-grown protein production requires significantly less land and water than conventional production,making it a sustainable alternative for addressing global food security and environmental challenges.Important policy milestones Singapore has established regulatory frameworks for the approval of cultured meat for the market,a global first that highlights the urgent need for clear guidelines;71 other regions are investing in infrastructure for production.72 The US Department of Agriculture(USDA)and Food and Drug Administration(FDA)are safely bringing cell-based meat products to market faster through a series of efforts to proactively address the safety of cultivated meat.73 Israel and other countries have also been proactive in developing comprehensive strategies to regulate novel foods,aiming to foster a robust innovation ecosystem,attract capital investments and maintain a leadership position in food technology.74 In an important step,the EUs“farm to fork”strategy has recognized the role of bio-based products in improving sustainability of food systems through innovation,and the European Food Safety Authority(EFSA)has released a scientific opinion requested by the European Commission:“New Developments in Biotechnology applied to microorganisms”which could have far-reaching implications for more sustainable ingredients and food systems generally.75Policy has played a powerful role in responsibly bringing more sustainable food options to the masses through standardization of safety protocols to prevent health risks,facilitating international trade by harmonizing standards,and promoting consumer confidence through rigorous testing and transparency.However,the implementation of further supportive policy frameworks around regulation,commercial production and fiscal incentives is urgently required to decrease costs,extend the benefits of lab-grown protein and to bring these innovative solutions to the plates of more consumers.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 19ConclusionSupportive policies are recognized for their ability to increase adoption,drive cost reductions,provide benefits to the environment,spur economic growth and so much more.The rapid adoption of solar panels represents one prominent example of the power of policy:due to the confluence of technological advancements,economic factors,increasing environmental awareness,and above all else,supportive policies,the adoption of solar panels has skyrocketed.76 The world stands on the brink of a revolution through the convergence of the biological,digital and physical worlds.What role will policy play in bringing tech-driven bioeconomy innovations into the mainstream and enabling a better bio-future that is accessible to all?Regardless of what has been delivered,as well as the benefits that innovation is on the cusp of providing,one thing remains clear:without technological interventions and supportive policies,the impacts of global crises like COVID-19 would have been much worse,and far fewer viable options for addressing climate change would exist.While bio-innovations to address planetary human challenges continue to gain commercial traction,it is the corresponding policies that hold the keys to the timeliness of response and the efficient delivery of outcomes for better human and planetary outcomes.This paper has highlighted the impact of biotechnology policy in key areas health,energy and chemicals,food and agriculture that offer significant societal and environmental benefits.It also makes the case for policy to extend its reach and outlines key obstacles that must be overcome to enable biotechnology to deliver transformative solutions more efficiently.Overcoming these challenges through coordinated public-private partnership,and seizing such opportunities is essential to unlocking biotechnologys full potential to drive innovation and address the urgent,unforeseen global issues of this era.Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 20ContributorsAuthorsJelena BojoviDirector,Centre for the Fourth Industrial Revolution SerbiaValeria DAmicoStrategic Integration Specialist,Bioeconomy,World Economic ForumDanijela ukiProject Manager,Centre for the Fourth Industrial Revolution SerbiaBranislava GemoviProject Manager,Centre for the Fourth Industrial Revolution SerbiaEstelle GoujonProject Fellow,World Economic ForumBrynne StantonThematic Lead,Bioeconomy,World Economic ForumWe thank the following for their contributions to this reportAlok Medikepura AnilFounder and Managing Director,Next Big Innovation Labs,IndiaYair BezmanFood Tech Evaluator&Content Expert,Israel Innovation AuthorityKasper Bruun KnudsenGlobal Public Affairs Manager,NovonesisPatrick CaiChair in Synthetic Genomics,Chemical Biology and Biological Chemistry University of ManchesterGiovanni ColomboDirector,Public Affairs,dsm-firmenichEmma CurbishleyHead of International Markets Procurement,Regeneron PharmaceuticalsSantanu DasguptaSenior Vice-President,Reliance Industries,IndiaShani DayanProject and Partnership Manager,Israeli Centre for the Fourth Industrial Revolution,Israel Innovation AuthorityMatthew EverettChief Procurement Officer,Regeneron PharmaceuticalsClaus Crone FuglsangChief Scientific Officer,NovonesisHelene HuangSenior Research Associate,Centre for Trustworthy TechnologyPlamena MarkovaChief of International Relations,European Molecular Biology Laboratory,(EMBL)Keiichi MochidaBioproductivity Informatics Research Team,RIKEN Center for Sustainable Resource ScienceReint-Jan Groot NeulendDirector,Public Affairs and Sustainability,dsm-firmenichDaniella PartemHead,Israeli Centre for the Fourth Industrial Revolution,Israel Innovation AuthorityShade SabituDirector,Regulatory Science and Operations,Pivot BioBeth ShapiroChief Scientific Officer,Colossal BiosciencesPav SidhuManager,Global Procurement,International Markets(UK and France),Regeneron PharmaceuticalsDidier ToubiaCo-Founder and Chief Executive Officer,Aleph FarmsCarmel WallisInternational Relations Officer,European Molecular Biology Laboratory,(EMBL)Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 21Endnotes1.Marshall,W.(2014,January 18).How Policymakers Enabled The Internet.Forbes.Retrieved from:https:/ Economic Forum.(2024).Accelerating the tech-driven bioeconomy.Geneva:World Economic Forum.Retrieved from:https:/www.weforum.org/publications/accelerating-the-tech-driven-bioeconomy/3.Speight,R.,&Stanton,B.(2024,May 28).How we can deliver a better tomorrow through generative biology.Retrieved from:https:/www.weforum.org/agenda/2024/05/overcome-barriers-to-generative-biology/4.McKinsey.The Bio Revolution:Innovations transforming economies,societies,and our lives.(n.d.)Retrieved from:https:/ 5.World Health 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from:www.food- Economic Forum(2024).Creating a Vibrant Food Innovation Ecosystem:How Israel Is Advancing Alternative Proteins Across Sectors.Retrieved from:https:/www3.weforum.org/docs/WEF_Protein_Diversification_2024.pdf 75.European Food Safety Authority.(2024,July 22).New developments in biotechnology applied to microorganisms.Retrieved from:https:/www.efsa.europa.eu/en/efsajournal/pub/8895 76.Nijsse,F.J.,Mercure,J.-F.,Ameli,N.,Larosa,F.,Kothari,S.,Rickman,J.,&Pollitt,P.V.(2023,October 17).The momentum of the solar energy transition.Nature Communications,14.doi:https:/doi.org/10.1038/s41467-023-41971-7 Accelerating the Global Transition to a Bio-based Economy:The Strategic Role of Policy 24World Economic Forum9193 route de la CapiteCH-1223 Cologny/GenevaSwitzerland Tel.: 41(0)22 869 1212Fax: 41(0)22 786 2744contactweforum.orgwww.weforum.orgThe World Economic Forum,committed to improving the state of the world,is the International Organization for Public-Private Cooperation.The Forum 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THE WORLD LEADER IN SERVING SCIENCE1Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.THE PULSE 2023Global R&D Insights in Pharmaceuticals Methodology&Profile 3Executive Summary 8Industry Trends&Challenges 16Hot Topics 24Outsourcing 28Appendix 32THE WORLD LEADER IN SERVING SCIENCE2Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.About The PulseThe future for drug developers is defined by how effectively they adopt innovative strategies and new technologies,while navigating industry challenges and complexities.Thats why the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders at biotech and pharmaceutical organizations around the globe to assess trends in drug discovery and development.Respondents shared the therapeutic areas in their pipelines,barriers to bringing drugs to market,innovations that are driving transformation,and attitudes toward key topics such as outsourcing,patient recruitment,diversity,decentralized trials,and more.In our second annual report,youll go beyond the data to learn what these insights mean for drug developers across the globe,and how you can prepare to successfully navigate the evolving drug development landscape.Discover what industry leaders are facing today,and how their outlook on pharmaceutical research and development is pushing the industry forward.Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.This report,including the information contained herein and commentary associated herewith(“materials”),is provided as a service of PPD.TheseMaterials,basedon publicly available information,market research conducted on behalf of PPD,as well asthe knowledge and experience of PPDsemployees,have been prepared solely for informational and educational purposes and should notbe relied upon for professional advice.Anyfurther use of these Materials requires the express written consent of PPD.THE WORLD LEADER IN SERVING SCIENCE3Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.3METHODOLOGY&PROFILETHE WORLD LEADER IN SERVING SCIENCE4Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.150 participants were surveyed in March/April 2023The online survey was conducted on behalf of the PPD clinical research business of Thermo Fisher Scientific by Life Science Strategy Group(LSSG)using its proprietary panel of more than 70,000 life science stakeholders and biopharma/biotech industry outsourcing decision makers and its affiliated APAC partners respondent panel.Participants were provided an honorarium for their time.Methodology&ProfileParticipants were screened to ensure they met the following criteria:Industry:Pharmaceutical,biopharmaceutical,or biotechnology companyLevel:Director level or higher with drug development decision-making responsibilityRole:Work in a role related to drug development Company:Have at least one compound in developmentGeography:Asia,Europe,US/CanadaTHE WORLD LEADER IN SERVING SCIENCE5Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.SegmentationRESPONDENTS WERE CLASSIFIED INTO THE FOLLOWING SEGMENTS.Statistical DifferencesThroughout the report,letters are used to indicate statistically significant differences between segments at the 90%confidence level.GeographyUS/Canada(n=65)Europe(n=48)Asia/Pacific(n=37)Company SizeSmall/Mid-Sized Companies:Annual R&D spend$1 billion*(n=85)Large Companies:Annual R&D spend$1 billion*(n=65)*Ranges in China were adjusted to reflect market conditions Small/Mid=annual R&D spend Greater use of RWD/RWE Patient diversity Small/Mid-Sized Companies Top 2:Innovative trial design New approaches to investment and fundingTop 5 Key Initiatives:Large CompaniesTop 5 Key Initiatives:Small/Mid-Sized CompaniesTHE WORLD LEADER IN SERVING SCIENCE11Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Top 5 Strategies for Removing Barriers to Patient ParticipationPatient RecruitmentKEY TAKEAWAYS: Optimism around recruiting qualified patients has increased from where it stood in 2022;in 2023,38el more positive about their ability to recruit patients versus just 26%who felt this way in 2022.Negativity around recruiting qualified patients has dropped from 42%to 28%. Top patient recruitment/participation strategies include establishing or improving relationships with patient advocacy groups,building inclusion criteria into protocol designs,and patient assistance resources.THE WORLD LEADER IN SERVING SCIENCE12Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Decentralized TrialsPast,Current,and Expected Future Use of Decentralized Clinical Trial ElementsKEY TAKEAWAYS: The prevalence of decentralized trial elements continues to grow with about 40%of trials currently using DCT. DCT growth is expected to continue,and in two years over 90%of participants expect to be using DCT in at least some of their trials.THE WORLD LEADER IN SERVING SCIENCE13Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.OutsourcingKEY TAKEAWAYS: More participants have upped their use of FSP outsourcing(41%)than say they have increased their use of full-service outsourcing(27%). Full-service outsourcing makes up about 40%of clinical work that is outsourced,with FSP and Hybrid models each used for about 25%,and the remainder(13%)going to insourcing or temporary staffing. Patient recruitment and clinical laboratory&diagnostic services are the top drug development activities likely to be outsourced.Top 5 Activities Likely to OutsourceTHE WORLD LEADER IN SERVING SCIENCE14Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Large Small/Mid Segment:Key HighlightsCategoryLarge Company ResponsesSmall/Mid-Sized Company ResponsesLeading Therapeutic Areas Oncology Immunology/Rheumatology Metabolic/Endocrine Rare Diseases Cardiovascular Oncology Immunology/Rheumatology Rare Diseases Neurology Infectious DiseasesClinical Development Timelines Reduced timelines Increased timelinesChallenges Patient diversity FundingTransformational Trends Use of RWD Leveraging new drug development technologies(e.g.,mRNA,CRISPR,gene-editing)Innovative trial designTop Strategies Pursued Patient diversity Personalized/precision medicine Genetherapy Innovative trial design Creative approaches to funding/investmentPatient Recruitment More optimistic about recruiting qualified patients vs.2 years ago Less success in affecting patient diversity vs.2 years ago More likely to utilize patient advocacy groups and patient education to increase diverse enrollment Less optimistic about recruiting qualified patients No change in ability to affect patient diversity vs.2 years ago Using inclusion criteria in protocol designs and patient assistance resources to increase diversityOutsourcing Increasing FSP use,decreasing full-service use Increasing FSP use,maintaining full-service useTHE WORLD LEADER IN SERVING SCIENCE15Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Geographic Regions:Notable Differences*Meaningful,statistically significant differences between regions at the 90%confidence level.CategoryUS/CanadaEuropeAsiaLeading Therapeutic Areas(Drug development in TAs higher than other regions)Infectious diseases HepatologyClinical Development Timelines Increased timelines Increased timelines Reduced timelinesChallenges Increased trial complexity Talent/staff shortages Increased trial complexity Keeping up with technology innovationTransformational Trends Innovative trial design Greater use of RWD/E Innovative trial design Personalized/precision medicineTop Strategies Pursued Innovative trial design Innovative trial design Personalized/precision medicinePatient Recruitment More say it is too soon to see impact from patient diversity efforts compared to Asia More say it is too soon to see impact from patient diversity efforts compared to Asia Lags other regions in DCT use More optimistic about recruiting qualified patients More say their ability to affect patient diversity has worsened over last 2 yearsOutsourcing Prefer full-service and hybrid More likely to outsource lab/diagnostic services compared to Asia Prefer full-service More likely to outsource lab/diagnostic services compared to Asia Prefer and most often use FSP More likely to outsource for post-approval support,product registrationTHE WORLD LEADER IN SERVING SCIENCE16Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.1616DETAILED FINDINGS:INDUSTRY TRENDS&CHALLENGES Leading therapeutic areas for drug development Clinical trial timelines Challenges Transformational trends Strategies pursuedTHE WORLD LEADER IN SERVING SCIENCE17Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Therapeutic AreasOncology significantly outpaces all other therapeutic areas for drug development,particularly among large companies. Immunology/Rheumatology and Rare Diseases are the next most common development areas. In addition to Oncology,large companies are also more likely than small/mid-sized companies to be developing therapeutics for Metabolic/Endocrine,Cardiovascular,and Dermatology.Leading Therapeutic Areas for Drug Development(TAs selected by 10%or more*)YOY Top 10 TAs in 2023 are the same as in 2022.REGIONAL NOTES Infectious Disease drug development is highest in US/Canada.Asia outpaces other regions for Hepatology.Letters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q2.Which therapeutic areas are leading your organizations drug development pipeline today?Please choose up to 5.THE WORLD LEADER IN SERVING SCIENCE18Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Clinical Development Timeline Total MarketAcross the industry,somewhat more participants indicate that clinical development timelines are extending. For those whose timelines have increased,most say they have lengthened by less than a year,but 2 out of 5 indicate their timelines have stretched out by more than 12 months. Those who have experienced shorter development timelines say the reduction has primarily been in the range of 1 to 11 months.Change in Timeline to Produce a Drug Compared to 2 Years Ago(from first-in-human trials through regulatory submission)YOY The lengthening timeline trend may have plateaued;only 41%say timelines are longer vs.52%in 2022.Base:All respondents excluding“dont know,”n=140Q6.Compared to two years ago,how has the average timeline to produce a drug(from first-in-human trials through regulatory submission)changed at your organization?THE WORLD LEADER IN SERVING SCIENCE19Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Clinical Development Timeline by Company SizeChange in Timeline to Produce a Drug Compared to 2 Years Ago(from first-in-human trials through regulatory submission)Letters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents excluding“dont know,”Large:n=62;Small/Mid:n=78Q6.Compared to two years ago,how has the average timeline to produce a drug(from first-in-human trials through regulatory submission)changed at your organization?The longer clinical development timelines for the industry are driven by small/mid-sized companies. Half of participants from small/mid-sized companies indicate their timelines are longer than they were two years ago;however,the situation is nearly reversed in the large company segment,where almost 50%say timelines are shorter.THE WORLD LEADER IN SERVING SCIENCE20Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Clinical Development Timeline by RegionLetters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents excluding“dont know,”US/Canada n=62;Europe n=41;Asia n=37Q6.Compared to two years ago,how has the average timeline to produce a drug(from first-in-human trials through regulatory submission)changed at your organization?Participants in US/Canada and Europe report their trial timelines have tended to lengthen over the last two years,while the majority in Asia have seen theirs shorten.Change in Timeline to Produce a Drug Compared to 2 Years Ago(from first-in-human trials through regulatory submission)THE WORLD LEADER IN SERVING SCIENCE21Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Biggest ChallengesLetters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q3.What are the biggest challenges your organization is currently facing?Please select your top 5 biggest challenges.Q3b.Listed below are the top challenges you indicated your organization is facing.Please rank these top challenges your organization is facing,with Rank#1=Biggest challenge.As in 2022,patient recruitment and trial complexity continue to be the largest organizational hurdles. A notable proportion are also quite concerned with maximizing ROI. Respondents at small/mid-sized companies are especially challenged by a lack of funding and talent,while addressing patient diversity and keeping up with technology are of more concern for those at large companies.YOY Supply chain disruptions(17%)are less problematic in 2023,down from 32%in 2022.REGIONAL NOTES Talent/staff shortages are a bigger challenge in US/Canada than elsewhere.Keeping up with technology&innovation is the biggest challenge in Asia.Pharmaceutical Companies Biggest Challenges(Challenges selected by 25%or more*)*See appendix for additional demographic detailTHE WORLD LEADER IN SERVING SCIENCE22Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Top Transformational TrendsSee appendix for full trend descriptions provided in the survey.2023 wording:“Innovative trial design(e.g.,adaptive,synthetic arms,umbrella,etc.)”2022 wording:“Adaptive trial design”the broader context provided in 2023 may have led to the jump from 2022.Letters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q4.What are the top five trends that are driving transformation in clinical trials?Please select your top 5 trends.Q4b.Listed below are the top trends you indicated are driving transformation in clinical trials.Please rank these top trends that are driving transformation in clinical trials,with Rank#1=Most impactful trend.While innovation in trial design tops the list of clinical development trends(especially among small/mid-sized companies),there is little consensus within the industry about which trends are most impactful.YOY Strengthening in 2023:Innovative trial design Artificial intelligenceSoftening in 2023:New technologies in drug developmentREGIONAL NOTES US/Canada&Europe:Innovative trial design is#1.Asia:Personalized/precision medicine is#1.US/Canada more apt to name RWD as a top trend than other regions.Trends Driving Transformation in Clinical Trials (Trends selected by 30%or more*)*See appendix for additional demographic detailTHE WORLD LEADER IN SERVING SCIENCE23Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Key InitiativesSee appendix for full trend descriptions provided in the survey.Letters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q5.Which specific innovations,strategies,and/or technologies are your organization currently pursuing?Please select all that apply.Reflecting the dispersion of opinions about leading transformational trends,a wide variety of strategies,technologies and innovations are in use across the industry chief among them overall are innovative trial design,RWD/RWE,patient-centricity,and big data. Many in large companies are also focusing on patient diversity,personalized medicine,cell and gene therapy,decentralization,and digitalization. Given that respondents from small/mid-sized companies cite lack of funding as a top challenge,it is not surprising that implementing new investment and funding approaches is a key strategy for them,second only to innovative trial design. As with innovative trial design this year,adaptive trial design was the top strategy pursued in 2022.YOY Digitalization has slipped down the list compared to other strategies/technologies.REGIONAL NOTES Participants in Asia are significantly less likely to be pursing innovative trial design than those in US/Canada or Europe.Innovations,Strategies,and Technologies Employed by Pharmaceutical Companies(Pursuits selected by 40%or more*)*See appendix for additional demographic detailTHE WORLD LEADER IN SERVING SCIENCE24Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.DETAILED FINDINGS:HOT TOPICS24 Patient recruitment Patient diversity strategies DecentralizationTHE WORLD LEADER IN SERVING SCIENCE25Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Patient Recruitment Almost all participants are tracking patient diversity,and there is more positivity than negativity about progress being made in this area among those who have been monitoring long enough to see results,but for about 1 in 5 it is still too early to tell.Large companies have a higher proportion than small/mid-sized companies who say their ability to affect patient diversity has worsened over the last two years.In total,compared to how they felt two years ago,participants are slightly more optimistic than pessimistic about their ability to recruit qualified patients however,this is driven by those at large firms;for those in small/mid-sized companies,it is roughly an even split.Ability to Recruit Qualified Patients Compared to 2 Years AgoAbility to Affect Diverse Patient Enrollment Compared to 2 Years AgoLetters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q17.How would you describe your ability to recruit qualified patients for your studies compared to two years ago?Q18.What type of change,if any,has your organization seen in its ability to affect diverse patient enrollment into your studies compared to two years ago?(Excludes dont know;Total:n=143;Large n=62;Small/Mid:n=81)YOY Optimism is up from 2022 when only 26%indicated they were more optimistic.REGIONAL NOTES Optimism about patient recruitment in Asia exceeds levels in US/Canada&Europe,but they believe their ability to impact patient diversity has worsened.THE WORLD LEADER IN SERVING SCIENCE26Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Patient Participation StrategiesMultiple strategies are used to enhance patient participation and meet diversity goals. Large Companies:Building relationships with patient advocacy groups is the leading strategy,followed by remote monitoring and patient education. Small/Mid-Sized Companies:Protocol designs that incorporate inclusion criteria and patient assistance resources are the top two strategies,perhaps because they are less resource-intensive than other strategies.Top Strategies Used by Sponsors to Remove Barriers to Patient Participation and Meet Diversity Targets(Strategies selected by 40%or more*)Letters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q19.What strategies to remove barriers to patient participation in clinical trials is your organization currently employing to meet diversity targets?(Select all that apply.)THE WORLD LEADER IN SERVING SCIENCE27Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Decentralized Trial ElementsParticipants indicate they have increased their use of decentralized clinical trial(DCT)elements from a year ago,and this trend is expected to continue,with about half of clinical trials estimated to be using DCT in 2025. The proportion of those using DCT in more than 50%of their trials is similar among large and small/mid-sized companies.Past,Current,and Expected Future Use of Decentralized Clinical Trial ElementsAverage%of Clinical Trials Conducted with DCT ElementsProportion of Clinical Trials Conducted with DCT ElementsLetters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,excluding“NA/Dont know,”n=138,138,141;Large:n=64,64,64,Small/Mid:n=74,74,77Q7.One year ago,what percentage of your companys clinical trials would you estimate were conducted with decentralized elements?Q8.What percentage of your companys current clinical trials would you estimate are being conducted with decentralized elements?Q9.Finally,what percentage of your companys clinical trials would you estimate will be conducted using decentralized elements in two years(2025)?YOY Participants are more bullish about DCT than they were in 2022,when they predicted that 36%of trials would be using DCT in two years(2024).REGIONAL NOTES DCT usage is growing in all regions,but its adoption in Europe significantly lags other regions.THE WORLD LEADER IN SERVING SCIENCE28Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.DETAILED FINDINGS:OUTSOURCING28 Outsourcing model:usage and trends Drug development activities likely to outsourceTHE WORLD LEADER IN SERVING SCIENCE29Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Outsourcing ModelsThe full-service model is used for the greatest share of current clinical development work,and it is most preferred. FSP and Hybrid models also account for a substantial share of clinical development work and are preferred by many.Outsourcing Models:Share of Current Clinical Development WorkMost Preferred Outsourcing Modelfor Clinical DevelopmentLetters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85 Q12.For the areas in your company with which you are familiar,what percent of current clinical development work that is outsourced is accomplished via the following outsourcing models?(question modified in 2023)Q13.Which outsourcing model for clinical development work do you most prefer?YOY The general pattern of outsourcing model usage in 2023 is consistent with 2022.REGIONAL NOTES US/Canada&Europe:Full-service is the most common model.Asia:FSP is most common and most preferred model.THE WORLD LEADER IN SERVING SCIENCE30Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Outsourcing TrendsFSP outsourcing is growing faster than full-service outsourcing,and this is the case across the industry,regardless of company size.Change in Use of Full-Service and FSP Models Compared to 2 Years AgoLetters indicate statistically significant difference between groups at the 90%confidence level.Base:All respondents,n=150;Large:n=65,Small/Mid:n=85Q14.Over the past two years,how has your company changed its full-service clinical trial outsourcing behavior?Q15.Over the past two years,how has your company changed its functional service provider(FSP)outsourcing behavior?REGIONAL NOTES FSP outsourcing is increasing faster than full-service outsourcing in all regions surveyed.THE WORLD LEADER IN SERVING SCIENCE31Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Outsourcing of Drug Development ActivitiesOutsourcing is expected to encompass a wide variety of drug development activities in the next two years;at the top across both large and small/mid-sized segments are patient recruitment,clinical lab and diagnostic services,and clinical trial management. Not surprisingly,those in small/mid-sized companies expect to use outsourcing more extensively than their large company counterparts,particularly for RWD,biostatistical analysis,post-approval support,data management,and safety analysis.Likelihood to Outsource Drug Development Activities in the Next 2 YearsLetters indicate statistically significant difference between groups at the 90%confidence level.Base:Excludes“NA/dont know,”varies by activity statement.All respondents:n=143-150;Large:n=62-65,Small/Mid:n=79-85Q16.Using the scale provided,please indicate how likely your company is to outsource each of the below drug development activities in the next 2 years.5-point scale:Not at all likely to Extremely likely.(question modified in 2023)YOY Increase in 2023:Post-approval support Study designDecrease in 2023:Data managementREGIONAL NOTES Asia is less likely to outsource clinical lab&diagnostic services,but more apt to use it for product registration and post-approval support.THE WORLD LEADER IN SERVING SCIENCE32Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.APPENDIX32THE WORLD LEADER IN SERVING SCIENCE33Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Survey participant screening criteria Currently work for:Biopharmaceutical,biotechnology or pharmaceutical company Geography:Asia Australia Europe Middle East/India US/Canada Drug development phases:Decision-making responsibility in at least one of the following:Drug discovery Preclinical Phase I Phase II Phase III Phase IV Pipeline:Company has at least one(1)unique molecule/compound in development pipeline Job level:Director or higher Decision Maker:Highly or somewhat involved in outsourcing services to vendors/CROs(e.g.,deciding to keep activities in-house vs.outsource,vendor selection,vendor management,etc.)in support of clinical trials Primary functional area:Pre-trial/preclinical development(preclinical/toxicology,translational medicine)Clinical development(clinical development,clinical operations,clinical data management,clinical research,feasibility,patient recruitment,biostatistics/statistical programming,safety/pharmacovigilance(PV),other R&D)Peri-/post-approval/registries/HEOR(medical affairs,market access,HEOR)Quality and regulatory(regulatory affairs,quality assurance/control(QA/QC)and compliance)Business services(alliance management/partnering,finance,procurement/vendor management,purchasing)Executive management/C-level Commercial/Marketing Compensation:Participants were compensated according to their agreement to participate in the Life Science Strategy Group(LSSG)panel.THE WORLD LEADER IN SERVING SCIENCE34Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Participant Demographics(1 of 3)Company Size*Ranges in China were adjusted to reflect market conditions.to USD Conversion at time of data collection:Under 350M=Under$50M,350M to 700M=$50M to$100M,700M to 3.5B=$100M to$500M,Over 3.5B=Over$500MLetters indicate statistically significant difference between groups at the 90%confidence level.S10.Which of the below ranges most closely represents your companys annual R&D spend?S9.What is the size of the organization you work for in terms of employees?Your best estimate is fine.S8.How many unique molecules/compounds are in your companys development pipeline?THE WORLD LEADER IN SERVING SCIENCE35Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Participant Demographics(2 of 3)Location/Job Level/Primary FunctionLetters indicate statistically significant difference between groups at the 90%confidence level.S3.In which of the below regions is your company headquarters located?S2.In which of the below regions is your office located?S4.What is your job level?S5.Which of the following best describes your current,primary functional area?THE WORLD LEADER IN SERVING SCIENCE36Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Participant Demographics(3 of 3)Drug DevelopmentLetters indicate statistically significant difference between groups at the 90%confidence level.Q1.In which categories is your organization/company developing or commercializing products?Please select all that apply.S6.In which development phase(s)do you have decision-making responsibility?Please select all that apply.S7.Over the past 2 years,what is your level of involvement with outsourcing services to vendors/CROs(e.g.,deciding to keep activities in-house vs.outsource,vendor selection,vendor management,etc.)in support of your clinical trials?THE WORLD LEADER IN SERVING SCIENCE37Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Transformational Trends Accelerated development/approvals in rare/orphan disease Artificial intelligence(e.g.,to enable recruitment,advanced analytics,RWD) Big data and analytics(data science) Data collection through wearables/connected health devices Digital and decentralized trials Digitalization(e.g.,cloud computing,APIs,digital platforms) Expansion of development to emerging markets(e.g.,China,Latin America) Greater use of RWD/RWE to complement data from clinical trials Increasing focus on patient diversity Innovative trial design(e.g.,adaptive,synthetic arms,umbrella,etc.) Leveraging new technologies in drug development(e.g.,mRNA,CRISPR,gene-editing technologies) Patient-centricity(e.g.,patient participation in protocol design,partnering with patient advocacy groups,etc.) Personalized/precision medicine(e.g.,companion diagnostics) Reducing research site burden Risk Based Quality Management(RBQM)programs with technologies that address surveillance,identification and reportingQ4.What are the top five trends that are driving transformation in clinical trials?Please select your top 5 trends.Q4b.Listed below are the top trends you indicated are driving transformation in clinical trials.Please rank these top trends that are driving transformation in clinical trials,with Rank#1=Most impactful trend.FULL DESCRIPTIONS PROVIDED IN THE SURVEYTHE WORLD LEADER IN SERVING SCIENCE38Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Innovations,Strategies&Techniques Artificial intelligence(e.g.,to enable recruitment,advanced analytics,RWD) Big data and analytics(data science) Cell and gene therapy Data collection through wearables/connected health devices Decentralized,digital and remote trials Digitalization(e.g.,cloud computing,APIs,digital platforms) Greater use of RWD/RWE Innovative trial design(e.g.,adaptive,synthetic arms,umbrella,etc.) New approaches to investment and funding Patient diversity Patient-centricity Personalized/precision medicine(e.g.,companion diagnostics)FULL DESCRIPTIONS PROVIDED IN THE SURVEYQ5.Which specific innovations,strategies,and/or technologies are your organization currently pursuing?Please select all that apply.THE WORLD LEADER IN SERVING SCIENCE39Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Leading Therapeutic Areas for Drug DevelopmentLetters indicate statistically significant difference between groups at the 90%confidence level.Q2.Which therapeutic areas are leading your organizations drug development pipeline today?Please choose up to 5.THE WORLD LEADER IN SERVING SCIENCE40Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Biggest ChallengesLetters indicate statistically significant difference between groups at the 90%confidence level.Q3.What are the biggest challenges your organization is currently facing?Please select your top 5 biggest challenges.Q3b.Listed below are the top challenges you indicated your organization is facing.Please rank these top challenges your organization is facing,with Rank#1=Biggest challenge.THE WORLD LEADER IN SERVING SCIENCE41Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Transformational TrendsLetters indicate statistically significant difference between groups at the 90%confidence level.Q4.What are the top five trends that are driving transformation in clinical trials?Please select your top 5 trends.Q4b.Listed below are the top trends you indicated are driving transformation in clinical trials.Please rank these top trends that are driving transformation in clinical trials,with Rank#1=Most impactful trend.THE WORLD LEADER IN SERVING SCIENCE42Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Innovations,Strategies&Techniques Currently PursuingLetters indicate statistically significant difference between groups at the 90%confidence level.Q5.Which specific innovations,strategies,and/or technologies are your organization currently pursuing?Please select all that apply.THE WORLD LEADER IN SERVING SCIENCE43Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Patient Participation StrategiesLetters indicate statistically significant difference between groups at the 90%confidence level.Q19.What strategies to remove barriers to patient participation in clinical trials is your organization currently employing to meet diversity targets?Copyright 2023 by Pharmaceutical Product Development(“PPD”),part of Thermo Fisher Scientific.All rights reserved.Go deeper at ahead of the curve with our experts takes on how these insights will influence the future of the drug development industry.
2024-10-15
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The Pulse 2024Global R&D insights in pharmaceuticals Thought leadershipppdMethodology 4 Executive summary 9Detailed findings:Industry trends and challenges 16Detailed findings:Hot topics 27Detailed findings:Outsourcing 32Appendix 37Contents2The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.About The PulseThe future for drug developers is defined by how effectively they adopt innovative strategies and new technologies,while navigating industry challenges and complexities.Thats why the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders at biotech and pharmaceutical organizations around the globe to assess trends in drug discovery and development.Respondents shared the therapeutic areas in their pipelines,barriers to bringing drugs to market,innovations that are driving transformation,and attitudes toward key topics such as outsourcing,patient recruitment,diversity,decentralized trials,and more.In our third annual report,youll go beyond the data to learn what these insights mean for drug developers across the globe,and how you can prepare to successfully navigate the evolving drug development landscape.Discover what industry leaders are facing today,and how their outlook on pharmaceutical research and development is pushing the industry forward.3The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Methodology4The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Methodology Participants were screened to ensure they met the following criteria:Industry:Pharmaceutical,biopharmaceutical,or biotechnology company Level:Director level or higher with drug development decision-making responsibility Role:Work in a role related to drug development Company:Have at least one compound in development Geography:Asia-Pacific,Europe,US-CanadaMethodology150 participants were surveyed in Q2 2024The online survey was conducted on behalf of the PPD clinical research business of Thermo Fisher Scientific by Life Science Strategy Group(LSSG)using its proprietary panel of more than 70,000 life science stakeholders and biopharma/biotech industry outsourcing decision makers and its affiliated APAC partners respondent panel.Participants were provided an honorarium for their time.5The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Respondents were classified into the following segmentsSegments*Ranges in China were adjusted to reflect market conditions Small/Mid=annual R&D spend 700 million Large=annual R&D spend 700 millionStatistical DifferencesStatistically significant differences between segments at a 90%confidence level are indicated with letters throughout the report.Company Size Small/Mid-size Biopharma Companies:annual R&D spend$1 billion*(n=86)Large Biopharma Companies:annual R&D spend$1 billion*(n=64)6The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Sample profile overview:TotalSample Profile Overview Total1Primary Functional Area37# %7P)%5%3%US/CanadaEuropeAsiaMiddle East/IndiaAustraliaNET:Asia:21%Headquarters LocationDrug Development Categories62$%Job Level/RoleDirectorVice PresidentPresident/C-Level43W%Organization Typen=64n=86Large=annual R&D spend of$1 billion or moreSmall/Mid=annual R&D spend less than$1 billion60U880# %3%BiologicsNovel small molecule drugsGene therapiesCell therapiesVaccinesGeneric small molecule drugsBiosimilarsNucleic acid therapiesDiagnosticsOther(Please specify)Average number categories:3.0Clinical DevelopmentExecutive Management/C-levelPeri-/post-approval/Registries/HEORQuality and RegulatoryPre-trial/Preclinical Development*See Appendix for additional demographic detailBase:All respondents(n=150).Charts may not total 100%due to rounding.S5.Which of the following best describes your current,primary functional area?(See Appendix for complete descriptions of functional areas)S4.What is your job level?S10.Which of the below ranges most closely represents your companys annual R&D spend?S3.In which of the below regions is your company headquarters located?Q1.In which categories is your organization/company developing or commercializing products?Please select all that apply.7The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Sample profile overview:Customer segmentsSample Profile Overview Customer Segments2Primary Functional Area44%53)%9%ClinicaldevelopmentExec mgmt/C-levelPeri-/post-approval/Registries/HEORQuality and regulatoryPre-trial/preclinicaldevelopmentLarge(A)Small/Mid(B)Job Level/RoleHeadquarters Location42V3%6%5%Large(A)Small/Mid(B)AustraliaMiddle East/IndiaAsiaEuropeU.S./CanadaDrug Development Categories73fPE80(%2PH)3$%3%BiologicsNovel smallmoleculedrugsGenetherapiesCelltherapiesVaccinesGeneric smallmoleculedrugsBiosimilarsNucleic acidtherapiesDiagnosticsOther77Q %Large(A)Small/Mid(B)President/C-levelVice PresidentDirectorSmall/Mid(B)Large(A)Avg.#Categories3.72.525ABBBBBBABLetters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents.Large(annual R&D spend of$1 billion or more):n=64;Small/Mid(annual R&D spend less than$1 billion):n=86S5.Which of the following best describes your current,primary functional area?(See Appendix for complete descriptions of functional areas)S4.What is your job level?S3.In which of the below regions is your company headquarters located?Q1.In which categories is your organization/company developing or commercializing products?Please select all that apply.8The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Executive summary9The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Drug developmentKey takeaways:Oncology(64%)is the leading therapeutic area for drug development,followed by Immunology/Rheumatology(41%)and Rare Diseases(31%).The rising cost of clinical trials is the top challenge followed by patient recruitment and the increasing complexity of clinical trials.Increasingly complex protocol designs and difficult patient recruitment are top challenges increasing the cost of clinical trials.Enrollment of hard-to-find populations and complex regulatory requirements are top challenges increasing the complexity of clinical trials.Innovative trial design,personalized/precision medicine and AI are the top trends driving transformation in clinical trials.Adaptive trial design is the top element considered innovative,followed by novel endpoints,biomarker-driven approaches,and multiple trial arms.Experience with clinical development timelines is mixed.While 30%of participants report shorter timelines and one-quarter indicate no change,45%say producing a drug takes longer now than it did two years ago.Drug Development9Key Takeaways:Top 5 Challenges21!%7I9930%Rising cost of clinical trialsPatient recruitment in clinical trialsIncreasing complexity of clinical trialsMaximizing asset value/ROIFeasibility and site selection%Ranked Top 2%Selected Top 5Drug Development9Key Takeaways:Top 5 ChallengesTop 5 Opportunities22)RQPGC%Innovative trial designPersonalized/precision medicineArtificial intelligenceLeveraging new technologiesPatient-centricity%Ranked Top 2%Selected Top 510The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Drug developmentKey takeaways:Drug development leaders use a variety of technologies and innovations;the most common overall are innovative trial design,artificial intelligence(AI),and personalized/precision medicine.Biopharma companies also are pursuing multiple strategic initiatives,with maximizing asset values the most common overall,but the two segments differ somewhat on other top strategies.Other leading strategies for large biopharma:Patient-centricity Greater use of RWD/RWE Patient diversity Other leading strategies for small/mid biopharma:New approaches to investment and funding Vendor rationalization Greater use of RWD/RWEDrug Development10Key Takeaways:Drug development leaders use a variety of technologies and innovations;the most common overall are innovative trial design,artificial intelligence(AI),and personalized/precision medicine.Biopharma companies also are pursuing multiple strategic initiatives,with maximizing asset values the most common overall,but the two segments differ somewhat on other top strategies:Other Leading Strategies:Large Biopharma:Patient-centricityGreater use of RWD/RWEPatient diversityOther Leading Strategies:Small/Mid Biopharma:New approaches to investment and fundingVendor rationalizationGreater use of RWD/RWETop 5 Technologies Pursuing:Large Biopharma66YVUR%Artificial Intelligence(AI)Personalized/precision medicineDigitalizationBig data and analytics(data science)Innovative trial designDrug Development10Key Takeaways:Drug development leaders use a variety of technologies and innovations;the most common overall are innovative trial design,artificial intelligence(AI),and personalized/precision medicine.Biopharma companies also are pursuing multiple strategic initiatives,with maximizing asset values the most common overall,but the two segments differ somewhat on other top strategies:Other Leading Strategies:Large Biopharma:Patient-centricityGreater use of RWD/RWEPatient diversityOther Leading Strategies:Small/Mid Biopharma:New approaches to investment and fundingVendor rationalizationGreater use of RWD/RWETop 5 Technologies Pursuing:Large Biopharma66YVUR%Artificial Intelligence(AI)Personalized/precision medicineDigitalizationBig data and analytics(data science)Innovative trial designTop 5 Technologies Pursuing:Small/Mid-size Biopharma64EDC6%Innovative trial designPersonalized/precision medicineArtificial Intelligence(AI)Big data and analytics(data science)Digitalization11The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Patient recruitmentKey takeaways:Large biopharma participants express more positivity than their Small/Mid-size counterparts about recruiting qualified patients and affecting diverse patient enrollment.Top patient recruitment/participation strategies include establishing or improving relationships with patient advocacy groups,using more sites for each clinical study,and conducting research in more countries.The primary goals of patient-focused strategies are to remove barriers to patient participation and increase overall enrollment.Patient Recruitment11Large biopharma participants express more positivity than their Small/Mid-size counterparts about recruiting qualified patients and affecting diverse patient enrollment.Top patient recruitment/participation strategies include establishing or improving relationships with patient advocacy groups,using more sites for each clinical study,and conducting research in more countries.The primary goals of patient-focused strategies are to remove barriers to patient participation and increase overall enrollment.Key Takeaways:Ability to Recruit Qualified Patients Compared to 2 Years Ago%More OptimisticAbility to Affect Diverse Patient Enrollment Compared to 2 Years Ago%Improved0 0P%LargeBiopharmaSmall/Mid-sizeBiopharma0 0P%LargeBiopharmaSmall/Mid-sizeBiopharmaTop 5 Strategies for Removing Barriers to Patient Participation0 0P%Establishing or improving relationships with patient advocacy groupsUsing more sites for each clinical studyConducting research in more countriesPatient assistance resourcesBuilding more inclusive entry criteria into protocolsPatient Recruitment11Large biopharma participants express more positivity than their Small/Mid-size counterparts about recruiting qualified patients and affecting diverse patient enrollment.Top patient recruitment/participation strategies include establishing or improving relationships with patient advocacy groups,using more sites for each clinical study,and conducting research in more countries.The primary goals of patient-focused strategies are to remove barriers to patient participation and increase overall enrollment.Key Takeaways:Ability to Recruit Qualified Patients Compared to 2 Years Ago%More OptimisticAbility to Affect Diverse Patient Enrollment Compared to 2 Years Ago%Improved0 0P%LargeBiopharmaSmall/Mid-sizeBiopharma0 0P%LargeBiopharmaSmall/Mid-sizeBiopharmaTop 5 Strategies for Removing Barriers to Patient Participation 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.0 0P%Establishing or improving relationships with patient advocacy groupsUsing more sites for each clinical studyConducting research in more countriesPatient assistance resourcesBuilding more inclusive entry criteria into protocols12The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Decentralized trialsKey takeaways:The prevalence of decentralized trial elements continues to grow with 38%of trials currently using DCT.DCT growth is expected to continue,and in two years,over 90%expect to be using DCT in at least some of their trials,similar to 2023 estimates.Decentralized Trials12Key Takeaways:The prevalence of decentralized trial elements continues to grow with 38%of trials currently using DCT.DCT growth is expected to continue,and in two years,over 90%expect to be using DCT in at least some of their trials,similar to 2023 estimates.Past,Current,and Expected Future Use of Decentralized Clinical Trial Elements328H%0 0%1 Yr AgoCurrentIn 2 YrsAverage%of clinical trials conducted with DCT elementsOver 50%of trials11%to 50%of trials10%of trials or lessProportion of trials using DCT:13The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.OutsourcingKey takeaways:FSP outsourcing is growing faster than full-service outsourcing 35%of participants say they have increased FSP outsourcing versus just 29%who have upped their use of full-service outsourcing.However,full-service outsourcing still makes up 34%of clinical work that is currently outsourced,with FSP and Hybrid models each comprising about 25%,and the remainder(13%)going to insourcing or temporary staffing.Patient recruitment and clinical laboratory&diagnostic services are the top drug development activities likely to be outsourced,similar to 2023.Outsourcing13Key Takeaways:FSP outsourcing is growing faster than full-service outsourcing 35%of participants say they have increased FSP outsourcing versus just 29%who have upped their use of full-service outsourcing.However,full-service outsourcing still makes up 34%of clinical work that is currently outsourced,with FSP and Hybrid models each comprising about 25%,and the remainder(13%)going to insourcing or temporary staffing.Patient recruitment and clinical laboratory&diagnostic services are the top drug development activities likely to be outsourced,similar to 2023.Top 5 Activities Likely to Outsource43E16(ssdcc%Patient recruitmentClinical laboratory and diagnostic servicesClinical data managementClinical trial management(end-to-end)RWD(i.e.,access to data)%Extremely Likely%Extremely likely/Very likely14The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Large small/mid biopharma comparisonCategoryLarge Biopharma ResponsesSmall/Mid Biopharma ResponsesLeading Therapeutic Areas Oncology Immunology/Rheumatology Cardiovascular Neurology Hematology Rare Diseases Infectious Diseases Oncology Immunology/Rheumatology Rare Diseases Neurology Infectious Diseases CardiovascularClinical Development Timelines Increased timelines Increased timelinesChallenges Rising cost of clinical trials Increasing complexity of clinical trials Patient recruitment in clinical trials Maximizing asset value/ROI Rising cost of clinical trials Patient recruitment in clinical trials Increasing complexity of clinical trials Lack of fundingClinical Trial Cost Drivers Increasingly complex protocol designs Patient recruitment is more difficult Increasingly complex protocol designs Patient recruitment is more difficultClinical Trial Complexity Drivers Enrollment of hard-to-find patient populations Innovative therapies that require complex protocols Enrollment of hard-to-find patient populations Compliance with complex regulatory requirements Pressure to shorten trial timelinesTransformational Trends Personalized/precision medicine Artificial intelligence New drug development technologies RWD/RWE to complement clinical trial data Decentralized/hybrid trial elements Innovative trial design Artificial intelligence Personalized/precision medicine Digitalization New drug development technologiesTop Innovations Pursued Artificial intelligence Personalized/precision medicine Digitalization Innovative trial design Personalized/precision medicine Artificial intelligenceTop Strategies Pursued Patient-centricity Greater use of RWD/RWE Maximizing asset value Patient diversity Sustainability efforts Maximizing asset value New approaches to investment and funding Vendor rationalizationPatient Recruitment More optimistic about recruiting qualified patients More success in affecting patient diversity vs.2 years ago Utilizing patient advocacy groups and data/technology to target patients Primary goal of patient-focused strategies:Increase overall enrollment Mixed perceptions about ability to recruit qualified patients For many,too early to know if patient diversity efforts are working Utilizing patient advocacy groups,more sites,and more countries Primary goal of patient-focused strategies:Remove barriers to patient participationTop Areas for Outsourcing Patient recruitment Clinical laboratory and diagnostic services Clinical monitoring/operations management Clinical trial management Patient recruitment Clinical laboratory and diagnostic services Clinical data management Clinical trial management RWD(i.e.,access to data)Bulleted items in bold text indicate a variance between the large and small/mid biopharma subgroups.15The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Detailed findings:Industry trends and challenges16The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Therapeutic areasOncology significantly outpaces all other therapeutic areas for drug development as indicated by participants from both large and small/mid-size biopharma companies.Immunology/Rheumatology,Rare Diseases,Cardiology,and Neurology are the next most prevalent therapeutic areas.Large company participants are more likely than those at Small/Mid-size companies to be developing therapeutics for Cardiovascular and Hematology.OncologyImmunology/RheumatologyRare DiseasesCardiovascularNeurologyInfectious DiseasesHematologyGastroenterologyMetabolic/EndocrineDermatologyRespiratory/AllergyAverage number TAs:3.5 3.8 3.3 71A5 (!%9pC1)!iG%B0 %9%Small/Mid(B)Large(A)TotalTherapeutic Areas17Leading Therapeutic Areas for Drug Development(TAs selected by 10%or more*)BBBLetters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86Q2.Which therapeutic areas are leading your organizations drug development pipeline today?Please choose up to 5.*See Appendix for complete detailYOYThe same TAs made the top 10 in 2024 as in 2022 and 2023.17The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Clinical development timeline total marketYOYLike 2023,fewer than half state their development timelines have extended,indicating the trend of lengthening timelines may have plateaued.Across the industry,somewhat more sponsors indicate that clinical development timelines are extending.For those whose timelines have increased,the split is fairly even between those who say timelines have lengthened by less than a year and those indicating timelines have increased by more than a year.Sponsors who have experienced shorter development timelines say the reduction has primarily been in the range of 1 to 11 months.LONGERSHORTERClinical Development Timeline Total Market183#&#%6E0%Note:3%indicated not applicable/dont knowChange in Timeline to Produce a Drug Compared to 2 Years Ago(from first in-human trials through regulatory submission)24 months12 to 23 months1 to 11 monthsNo change1 to 11 months12 to 23 months24 monthsLengthened by:Shortened by:Base:All respondents excluding dont know;Total:n=145Q7.Compared to two years ago,how has the average timeline to produce a drug(from first-in-human trials through regulatory submission)changed at your organization?18The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Clinical development timeline by market segmentParticipants from both biopharma segments indicate somewhat longer clinical development timelines compared to what they experienced two years ago.While similar proportions of participants from large and small/mid companies indicate increased timelines,this issue is more pronounced in the Small/Mid-size company segment,with 27%reporting increased timelines of over one year as opposed to just 16%among their counterparts at Large companies.No statistically significant difference between groups at the 90%confidence levelBase:All respondents excluding dont know;Large:n=63,Small/Mid:n=82Q7.Compared to two years ago,how has the average timeline to produce a drug(from first-in-human trials through regulatory submission)changed at your organization?YOYFewer large company participants report shortening timelines in 2024(33%)than did in 2023(48%),and more are reporting longer timelines,43%in 2024 vs.29%in 2023.In 2024,the pattern for large company participants is more similar to the small/mid segment than it was in 2023.Clinical Development Timeline by Market SegmentParticipants from both biopharma segments indicate somewhat longer clinical development timelines compared to what they experienced two years ago.19Change in Timeline to Produce a Drug Compared to 2 Years Ago(from first in-human trials through regulatory submission)LONGERSHORTER2$%LONGERSHORTER4# #%4%Small/Mid(B)Large(A)43F3$ months12 to 23 months1 to 11 monthsNo change1 to 11 months12 to 23 months24 monthsLengthened by:Shortened by:27The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Biggest challengesYOYTalent/staff shortages are less of an issue for those at Small/Mid companies in 2024(19%)than in 2023(35%).As in 2022 and 2023,patient recruitment and trial complexity continue to be large organizational hurdles,following the rising cost of clinical trials as the largest hurdle(new challenge added in 2024).Participants from Small/Mid-size companies are more challenged by a lack of funding(similar to 2023),while the lack of internal expertise to utilize RWD is more problematic for those at Large biopharma companies.Letters indicate statistically significant difference between groups at the 90%confidence levelRising cost of clinical trialsPatient recruitment in clinical trialsIncreasing complexity of clinical trialsMaximizing asset value/ROIFeasibility and site selectionLack of fundingFinding vendors w/scientific/therapeutic expertiseElongated study startup timeLack of or limited relevant RWD/RWELack internal resources/experts for RWD/RWENavigating changing regulatory landscapeTalent/staff shortagesBiggest Challenges20Pharmaceutical Companies Biggest Challenges(Challenges selected by 20%or more*)*See Appendix for complete detail21!%7%9%9%5I9930(#!#%5%9%5D493(%#% #%8#%8%7%8%6SB83151( %ASmall/Mid(B)Large(A)Total%Ranked Top 2%Selected Top 5Key:BALetters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86 Q3.What are the biggest challenges your organization is currently facing?Please select your top 5 biggest challenges.Q3b.Please rank these top challenges that your organization is facing,with Rank#1=Biggest challenge.20The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Deeper dive:Increasing cost of clinical trialsMultiple factors drive the cost of clinical trials,including increasingly complex protocol designs,difficulty with patient recruitment,increasing length of clinical trials,inflation and other macroeconomic factors,development of innovative therapies that require complex protocols,and compliance with regulatory requirements.Participants from both segments are similar in their views about the factors that are most responsible for increasing clinical research costs.No statistically significant differences between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86 Q23.(NEW)What are the top 3 factors most responsible for the increasing cost of clinical trials?Please select up your top 3.21Deeper Dive:Increasing Cost of Clinical TrialsFactors Responsible for Increasing Cost of Clinical Trials0 0P%Increasinglycomplex protocoldesignsPatientrecruitment ismore difficultIncreasing lengthof clinical trialsInflation andothermacroeconomicfactorsDevelopment ofinnovativetherapies thatrequire complexprotocolsCompliance withregulatoryrequirementsPressure toshorten trialtimelinesNeed to capturemore data andinsights fromclinical trialsIncreasingamount of trialdata collectedper studyAdoption of newtechnologiesGreateremphasis on sitemonitoringTotalLarge(A)Small/Mid(B)12312321The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Deeper dive:Patient recruitment problems/difficultiesTop challenges for patient recruitment include identifying the right patients and competition with other trials or sites,especially given low patient numbers for rare diseases and diversity requirements.Participants at Large companies are particularly impacted by competition with other trials or sites,while those at Small/Mid-size biopharma companies struggle with identifying the right patients.Base:Respondents who selected“Patient recruitment in clinical trials”as a top 5 challenge(Q3a);Total:n=58,Large:n=22,Small/Mid:n=36 Q26(NEW):Earlier you indicated that“patient recruitment in clinical trials”is currently a challenge for your organization.What,specifically,is especially problematic or difficult about patient recruitment or managing patient recruitment?(free text/open-end response).Deeper Dive:Patient Recruitment Problems/Difficulties22Patient Recruitment Difficulties(number of mentions)Total(n=58)Large(n=22)Small/Mid(n=36)Identifying the right patients27720Competition with other trials or sites20128Patient willingness and retention927Identification of sites734Diversity requirements633Lack of funding/resources523Timelines for recruitment514“Patient recruitment is often slower than estimated,sometimes due to competition over the“best”sites.”-Respondent from a large biopharma“Additional competitors within rare disease therapeutic areas limits the overall population of clinical trial amenable patients.Additionally,with new therapies that are novel and not approved globally inclusion and exclusion criteria are increasingly complex thus limiting amenable patients for long term clinical trials.Success within rare diseases increases difficulty for any future therapy.”-Respondent from a small/mid biopharma22The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Deeper dive:Increasing cost of clinical trialsEnrollment of hard-to-find patient populations,compliance with complex regulatory requirements,and innovative therapies requiring complex protocols are the top factors most responsible for the increasing complexity of clinical trials.The pressure to shorten trial timelines(particularly among those from Small/Mid-size companies)and the need to capture more data and insights from clinical trials are other key factors.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86 Q22.(NEW)What are the top 3 factors most responsible for the increasing complexity of clinical trials?Please select your top 3.23Deeper Dive:Increasing Complexity of Clinical TrialsFactors Responsible for Increasing Complexity of Clinical Trials0 0P%Enrollment ofhard-to-findpatientpopulationsCompliancewith complexregulatoryrequirementsDevelopment ofinnovativetherapies thatrequirecomplexprotocolsPressure toshorten trialtimelinesNeed tocapture moredata andinsights fromclinical trialsStudyprioritizationamong sitesManagingmultiplevendorsAdoption ofnewtechnologiesLack of or needto achievealignment ontrial strategy/designManagementof multiplestudyplatforms/technologiesIncreasinguse ofdecentralizedtrialsInability toachieve theright balance ofFSO and FSPoutsourcingmodelsTotalLarge(A)Small/Mid(B)B123123=notably higher than the other segment23The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Top transformational trendsYOYStrengthening in 2024:Personalized/precision medicine Leveraging new technologies Increasing focus on patient diversity(especially with Small/Mid)AI(especially with Large)While there is little consensus about which trends are most impactful,trial design innovation(especially for those in Small/Mid-size organizations),personalized medicine,and AI top the list.Those at Small/Mid-size companies also consider digitalization to be a top trend,while their counterparts at Large organizations put more emphasis on greater use of RWD/RWE.Innovative trial designPersonalized/precision medicine(e.g.,companion diagnostics)Artificial intelligenceLeveraging new tech dev(e.g.,mRNA,CRISPR,gene-editing)Patient-centricityIncreasing focus on patient diversityDecentralized/hybrid trial elementsAccelerated development/approvals in rare/orphan diseaseGreater use of RWD/RWE to complement data from clinical trialsBig data and analytics(data science)Digitalization(e.g.,cloud computing,APIs,digital platforms)Sustainability effortsTop Transformational Trends24Trends Driving Transformation in Clinical Trials22)%5RQPGCCBAA96%3 %9%9%9%8%9BSPPDBE8H8#% YIPDCCC5E%8%Small/Mid(B)Large(A)Total%Ranked Top 2%Selected Top 5Key:ABBABABSee Appendix for full trend descriptions provided in the survey.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=65,Small/Mid:n=85Q4.What are the top five trends that are driving transformation in clinical trials?Please select your top 5 trends.Q4b.Please rank these top trends,with Rank#1=Most impactful trend.24The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Key initiativesReflecting the dispersion of opinions about leading transformational trends,sponsors are pursuing a wide variety of technologies and innovations chief among them is innovative trial design,followed by AI,personalized medicine,big data,and digitalization.Among Large company participants,AI,personalized medicine,and digitalization are the top innovations and technologies.Innovative trial design is the key pursuit among those at Small/Mid-size companies,and similar to their Large company counterparts,AI and personalized medicine are receiving a lot of attention along with big data.Innovations and Technologies Employed by Pharmaceutical Companies0 0Pp%Innovative trial designArtificial intelligencePersonalized/precisionmedicineBig data and analyticsDigitalizationLeveraging newtechnologies in drugdevelopmentDigital/hybridtrial elementsTotalLarge(A)Small/Mid(B)Average number innovations/technologies:3.03.73.3Key Initiatives25BBB123123BLetters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86Q5.(REVISED innovations/technologies separated from strategies in Q6)Which specific innovations and/or technologies are being pursued currently by your organization?Please select all that apply.YOYSponsors are engaging nearly all of these innovations and technologies more in 2024,especially AI.The only exception is digital/hybrid trial elements.25The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Current strategiesYOYLarge company participants are more apt to be looking for new approaches to investment and funding in 2024(33%)than they were in 2023(14%).Multiple strategies are also currently pursued,with top strategies overall including efforts to maximize asset value,greater use of RWD/RWE,and patient-centricity.RWD/RWE and patient-centricity strategies are strategies that a majority of Large company participants are engaged in.Maximizing asset value and finding new avenues for funding are top strategies among those from Small/Mid-size companies.S St tr ra at te eg giie es s P Pu ur rs su ue ed d b by y P Ph ha ar rmma ac ce eu ut tiic ca all C Co ommp pa an niie es s0 0Ppforts tomaximizeasset valueGreater use ofRWD/RWEPatient-centricityVendorrationalizationPatient diversityStrategiesto reduce siteburdenNew approaches toinvestment andfundingMergers andacquisitionsRisk-basedcontracts withvendorsSustainabilityeffortsTotalLarge(A)Small/Mid(B)A Av ve er ra ag ge e n nu ummb be er r s st tr ra at te eg giie es s:3 3.8 84 4.4 44 4.1 1BBB1 12 23 31 12 23 3BLetters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86Q6.(REVISED strategies separated from innovations/technologies in Q5)Which specific strategies are being pursued currently by your organization?Please select all that apply.26The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Detailed findings:Hot topics27The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Patient recruitmentExperience with patient recruitment is mixed and varies significantly by organization size almost half of those from Large companies are more optimistic about their ability to recruit qualified patients,but this drops to only 30%among Small/Mid-size participants.Tracking patient diversity is very widespread,and there is more positivity than negativity about progress being made in this area among those who have been monitoring long enough to see results,but for about 1 in 5 it is still too early to tell.Large company participants are significantly more likely to claim improvement in their ability to enroll diverse patients compared to their counterparts in the Small/Mid-size segment,where a higher proportion indicate it is too early to tell,similar to their responses in 2023.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86.Q18.How would you describe your ability to recruit qualified patients for your studies compared to two years ago?Base:All respondents excluding dont know;Total:n=145;Large n=63;Small/Mid:n=82.Q21.What type of change,if any,has your organization seen in its ability to affect diverse patient enrollment into your studies compared to two years ago?YOYOptimism remains unchanged since 2023,though ability to affect diverse patient enrollment has improved,with 42eling improvement compared to just 29%in 2023.This change is largely driven by Large company participants,with 59%saying it has improved compared to only 32%saying so in 2023.28Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86.Q18.How would you describe your ability to recruit qualified patients for your studies compared to two years ago?Base:All respondents excluding dont know;Total:n=145;Large n=63;Small/Mid:n=82.Q21.What type of change,if any,has your organization seen in its ability to affect diverse patient enrollment into your studies compared to two years ago?Ability to Recruit Qualified Patients Compared to 2 Years AgoMore optimisticNo changeLess optimistic384(H%00%Small/Mid(B)Large(A)TotalBABA28Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86.Q18.How would you describe your ability to recruit qualified patients for your studies compared to two years ago?Base:All respondents excluding dont know;Total:n=145;Large n=63;Small/Mid:n=82.Q21.What type of change,if any,has your organization seen in its ability to affect diverse patient enrollment into your studies compared to two years ago?Ability to Recruit Qualified Patients Compared to 2 Years AgoMore optimisticNo changeLess optimistic384(H%00%Small/Mid(B)Large(A)TotalAbility to Affect Diverse Patient Enrollment Compared to 2 Years Ago422%4%3Y0%2)3%6%5%Small/Mid(B)Large(A)TotalImprovedNo changeWorsenedToo early to knowDo not track patient diversityBA28The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Patient strategiesYOYNotable changes vs 2023TotalPAG relationships More sites Remote monitoring Telehealth Home visits Large BiopharmaMore countries Patient assistance Remote monitoring Patient education Telehealth Home visits Small/Mid-size BiopharmaPAG relationships More sites More countries Inclusive protocols Telehealth While building patient advocacy group relationships is a key strategy used by participants from both large and small/mid-size biopharma companies,sponsors continue to employ multiple strategies to encourage patient participation.As the pandemic recedes into the past,the use of remote monitoring,telehealth visits,and home visits have declined substantially as methods for removing barriers to patient participation.Added in 2024Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86Q19.(REVISED)What patient-focused or recruitment strategies are being used currently by your organization?Select all that apply.Average number strategies:4.84.94.7Relationships with patient advocacy organizationsUsing more sites for each clinical studyConducting research in more countriesPatient assistance resourcesBuilding inclusive entry criteria into protocolsLeveraging data/tech to identify target patientsPatient-centric platforms/appsPatient educationRemote monitoring51RQE84438VFEA99755c864AEA91%Small/Mid(B)Large(A)TotalPatient Strategies29Top Patient-focused/Recruitment Strategies Currently Used(Strategies selected by 30%or more*)=draws attention*See Appendix for complete detailAA29The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Patient strategy goalsTop goals of patient-focused strategies include removing barriers to patient participation and increasing overall enrollment.Removing barriers to patient participation is driven primarily by Small/Mid-size biopharma participants.Those from Large organizations focus on increasing overall enrollment,followed by strengthening relationships with physicians who serve more diverse patients.Increasing patient diversity is more of a goal for participants at Large companies than it is for those in the Small/Mid-size segment.Letters indicate statistically significant difference between groups at the 90%confidence levelBase=Using one or more patient-focused or recruitment strategies(Q19);Total:n=148;Large:n=64;Small/Mid:n=84Q20.(NEW)Which of the following are the top intended goal(s)of the patient-focused strategies your organization is using currently?Rank up to 3.Remove barriers to patient participationIncrease overall enrollmentStrengthen relationships with physicianswho serve more diverse patientsImprove patient retention ratesIncrease patient-centricityIncrease patient diversityStrengthen connection to the community15&%9TRHE630%5ASGABE10%5%6dQII2$)0Patient Strategy GoalsGoals of Patient-Focused StrategiesSmall/Mid(B)Large(A)TotalBA=draws attention%Ranked#1%Ranked Top 3Key:B30The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Clinical trial decentralizationParticipants indicate they have increased their use of decentralized clinical trial(DCT)elements from a year ago,and this trend is expected to continue,with about half of clinical trials estimated to be using DCT in 2026.While participants from both large and small/mid-size participants reported similar DCT use and expectations in 2023,this year the Small/Mid segment indicates somewhat greater use of DCT and higher expected use by 2026.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents excluding not applicable/dont know;Total:n=131,137,143;Large:n=61,63,63,Small/Mid:n=70,74,80Q8.One year ago,what percentage of your companys clinical trials would you estimate were conducted with decentralized elements?Q9.What percentage of your companys current clinical trials would you estimate are being conducted with decentralized elements?Q10.Finally,what percentage of your companys clinical trials would you estimate will be conducted using decentralized elements in two years(2026)?YOYCompared to 2023,DCT usage has softened among participants in the Large segment,averaging use in 35%of current trials versus 43%reported in 2023.6%8%5 !9C6%TotalLarge(A)Small/Mid(B)19 C9$!%7%8%6%9%9%TotalLarge(A)Small/Mid(B)154(%0%8%TotalLarge(A)Small/Mid(B)0.00.00 .000.00.00P.00.00%1 Year AgoCurrentIn 2 yearsTotalLarge(A)Small/Mid(B)Past,Current,and Expected Future Use of Decentralized Clinical Trial(DCT)ElementsAverage%of Clinical Trials Conducted with DCT ElementsProportion of Clinical Trials Conducted with DCT ElementsOver 70%of trials51%to 70%of trials31%to 50%of trials11%to 30%of trials0%to 10%of trialsIn 2 Years(%of respondents)Current(%of respondents)1 Year Ago(%of respondents)A31The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Detailed findings:Outsourcing32The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Outsourcing modelsThe full-service model is used for the greatest share of current clinical development work,and it is most preferred.FSP and hybrid models also account for a substantial share of clinical development work and are preferred by many,with insourcing as the least used and preferred.Participants in Large biopharma use FSP more than their counterparts in the small/mid-size segment.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86 Q13.For the areas in your company with which you are familiar,what percent of current clinical development work is accomplished via the following outsourcing models?Q14.Which outsourcing model for clinical development work do you most prefer?YOYThe general pattern of outsourcing model usage in 2024 is consistent with 2023 and 2022.33Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86 Q13.For the areas in your company with which you are familiar,what percent of current clinical development work is accomplished via the following outsourcing models?Q14.Which outsourcing model for clinical development work do you most prefer?Outsourcing ModelsShare of Current Clinical Development Work34063#&!)%TotalLarge(A)Small/Mid(B)Insourcing/Temp StaffHybridFSPFull-ServiceB33Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86 Q13.For the areas in your company with which you are familiar,what percent of current clinical development work is accomplished via the following outsourcing models?Q14.Which outsourcing model for clinical development work do you most prefer?Outsourcing ModelsShare of Current Clinical Development Work34063#&!)%TotalLarge(A)Small/Mid(B)35381%No preferenceInsourcing/Temp StaffHybridFSPFull-ServiceMost Preferred Outsourcing Modelfor Clinical DevelopmentSmall/Mid(B)Large(A)Total334%B 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.33The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Outsourcing trendsFSP outsourcing is growing faster than full-service outsourcing(FSO),and this is the case for participants in both large and small/mid-size biopharma segments.Participants in both segments appear to be switching away from full-service outsourcing to some degree,with over 15%in each segment decreasing their use of FSO.Letters indicate statistically significant difference between groups at the 90%confidence level Base:All respondents;Total:n=150;Large:n=64,Small/Mid:n=86Q15.Over the past two years,how has your company changed its full-service(FSO)clinical trial outsourcing behavior?Q16.Over the past two years,how has your company changed its functional service provider(FSP)outsourcing behavior?YOYWhile still growing at a faster rate than FSO,the growth rate of FSP may be starting to slow,with 35%indicating increased usage of FSP in 2024 compared to 41%in 2023.34Change in Use of Full-Service and FSP Models Compared to 2 Years AgoIncreased Use of Outsourcing ModelDecreased Use of Outsourcing ModelTotal295%Full-ServiceFSP17%Small/Mid(B)297%Full-ServiceFSP19%Large(A)303%Full-ServiceFSP16%9%Indicating:34The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Outsourcing of drug development activitiesOutsourcing will comprise a wide variety of drug development activities in the next two years,particularly patient recruitment and clinical lab and diagnostic services.Not surprisingly,those at Small/Mid-size companies expect to use outsourcing more extensively than their counterparts in the Large biopharma segment.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents excluding dont know;varies by activity statement.Total:n=143-150;Large:n=63-64,Small/Mid:n=80-86Q17.Using the scale provided,please indicate how likely your company is to outsource each of the following drug development activities in the next 2 years.5-point scale:Not at all likely to Extremely likely.YOYSignificant increases in 2024:Data management Preclinical evaluations Regulatory consulting Patient recruitmentClinical laboratory and diagnostic servicesClinical data managementClinical trial managementRWD(i.e.,access to data)Clinical monitoring/operations managementClinical supply managementRWE(i.e.,generation of insights)Pharmacovigilance solutionsMarket access/Value demonstrationPre-and post-approval safety analysisBiostatistical servicesRegulatory consultingMedical writing35Likelihood to Outsource Drug Development Activities in the Next 2 Years(Activities selected by 55%or more*)43E16(55&0$&!&ssdcccYXXVVVU#1 #dcHRHVPFCDD9HBXUG6ED5A1364vruggpihfibd%Small/Mid(B)Large(A)Total%Extremely likelyExtremely/Very likelyAAAAAAAAAAAAAAAAAAAAAAAAAAA=draws attention*See Appendix for complete detailAAA35The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Sponsor Vendor Relationship Models36Most Preferred Sponsor Vendor Relationship Model328(E6%6 %7%5%5%6%TotalLarge(A)Small/Mid(B)Transactional study-basedInformal preferred partnerStrategic partnerFormal preferred partnerTransactional compound/program-basedSponsor Vendor Relationship ModelsShare of Current Clinical Development Work283$!(!%TotalLarge(A)Small/Mid(B)Transactional study-basedInformal preferred partnerStrategic partnerFormal preferred partnerTransactional compound/program-basedBBAANot sure/No preference 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Sponsor Vendor Relationship Models36Most Preferred Sponsor Vendor Relationship Model328(E6%6 %7%5%5%6%TotalLarge(A)Small/Mid(B)Transactional study-basedInformal preferred partnerStrategic partnerFormal preferred partnerTransactional compound/program-basedANot sure/No preferenceSponsor vendor relationship modelsFormal and strategic partnerships are used more than other types of relationship models and are the most preferred,however,a substantial proportion of clinical development work uses informal partners or is transactional in nature.Participants from Large companies currently use significantly more strategic and formal preferred partners and prefer to use these models as well.Almost half prefer the strategic partner model a change from 2023 when more than half preferred the formal partner model.Although a mix of models is used by those at Small/Mid-size companies,and they are significantly more likely to use the study-based transactional model,many would prefer a strategic partnership.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents;n=150;Large:n=64,Small/Mid:n=86 Q11.For the areas in your company with which you are familiar,what percentage of current clinical development work is accomplished via the following sponsor-vendor relationship models?Q12.Which sponsor-vendor relationship model do you most prefer?36The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Appendix37The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Survey participant screening criteriaCurrently work for:Pharmaceutical,biopharma,or biotech company Geography:Asia Australia Europe Middle East/India US/CanadaDrug development phases:Decision-making responsibility in at least at least one of the following Drug discovery Preclinical Phase I Phase II Phase III Phase IV/Lage stage/RegistriesNote:those who selected only“Drug discovery”were excluded from the survey.Pipeline:Company has at least one(1)unique molecule/compound in development pipelineJob level:Director or higherDecision Maker:Highly or somewhat involved in outsourcing services to vendors/CROs(e.g.,deciding to keep activities in-house vs.outsource,vendor selection,vendor management,etc.)in support of clinical trialsPrimary functional area:Pre-trial/Preclinical Development(preclinical/toxicology,translational medicine)Clinical Development(clinical development,clinical operations,clinical data management,clinical research,feasibility,patient recruitment,biostatistics/statistical programming,safety/pharmacovigilance(PV),other R&D)Peri-/post-approval/Registries/HEOR(medical affairs,market access,HEOR)Quality and Regulatory(regulatory affairs,quality assurance/control(QA/QC)and compliance)Executive Management/C-level Participants were compensated according to their agreement to participate in the Life Science Strategy Group(LSSG)panel.38The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Participant demographics(1 of 3)Company size*Ranges in China were adjusted to reflect market conditions.to USD Conversion:Under 350M=Under$50M,350M to 700M=$50M to$100M,700M to 3.5B=$100M to$500M,Over 3.5B=Over$500MLetters indicate statistically significant difference between groups at the 90%confidence levelS10.Which of the below ranges most closely represents your companys annual R&D spend?S9.What is the size of the organization you work for in terms of employees?Your best estimate is fine.S8.How many unique molecules/compounds are in your companys development pipeline?Demographics-Company SizeTotalLargeSmall/MidUS/CanadaEuropeAsia(A)(B)Base:All respondents1506486734631Annual R&D SpendChina to USD ConversionUnder$100 million/Under 350 million*29%0P35%(Under$50M USD)$100 million to$999 million/350 million to 700 million*29%0P05%($50M to$100M USD)Small/Mid-size Biopharma Sub-Total57%00cRR%$1 billion to$2 billion/700 million to 3.5 billion*17A%0)%($100M to$500M USD)Over$2 billion/3.5 billion*25Y%0!7%(Over$500M USD)Large Biopharma Sub-Total430%07HH%Number of Employees1-49 employees11%0 !%0%6P-199 employees23%65(2 0-999 employees22)$2%1,000-9,999 employees19%,000 or more employees25V%1&3%Number of Unique Molecules/Compunds in Pipeline1 molecule/compound2%0%3%3%0%3%2 to 3 molecules/compounds22%91%)%4 to 5 molecules/compounds233 2%6 to 7 molecules/compounds17 %8 to 9 molecules/compounds9%3%5 ore more molecules/compounds27P%923%6BBBAAABAAABA39The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Participant demographics(2 of 3)Location/Job level/Primary functionLetters indicate statistically significant difference between groups at the 90%confidence levelS3.In which of the below regions is your company headquarters located?S2.In which of the below regions is your office located?S4.What is your job level?S5.Which of the following best describes your current,primary functional area?Demographics-Company SizeTotalLargeSmall/MidUS/CanadaEuropeAsia(A)(B)(C)(D)(E)Base:All respondents1506486734631S10Annual R&D SpendChina to USD ConversionUnder$100 million/Under 350 million*29%0P35%(Under$50M USD)$100 million to$999 million/350 million to 700 million*29%0P05%($50M to$100M USD)Small/Mid-size Biopharma Sub-Total57%00cRR%$1 billion to$2 billion/700 million to 3.5 billion*17A%0)%($100M to$500M USD)Over$2 billion/3.5 billion*25Y%0!7%(Over$500M USD)Large Biopharma Sub-Total430%07HH%S9Number of Employees1-49 employees11%0 !%0%6P-199 employees23%65(2 0-999 employees22)$2%1,000-9,999 employees19%,000 or more employees25V%1&3%S8Number of Unique Molecules/Compunds in Pipeline1 molecule/compound2%0%3%3%0%3%2 to 3 molecules/compounds22%91%)%4 to 5 molecules/compounds233 2%6 to 7 molecules/compounds17 %8 to 9 molecules/compounds9%3%5 ore more molecules/compounds27P%923%6mographics-Geography/Job Level/Primary Function TotalLargeSmall/Mid(A)(B)Base:All respondents1506486734631Company Headquarters LocationUS/Canada50BV%Europe293%5%3%Asia/Australia/Middle East/India Sub-Total21%5%0%Asia13%4%0R%Australia3%5%1%1%0%Middle East/India5%6%5%0%0&%Office LocationUS/Canada49BS0%0%0%Europe314(%00%0%Asia/Australia/Middle East/India Sub-Total21#%0%00%Asia12%0%0X%Australia3%5%1%0%0%Middle East/India6%6%6%0%0)%Job LevelDirector62wQXpa%Vice President24 !5%President1%0%1%1%0%0%C-level13%3!%9%3%Primary Functional ResponsibilityPre-trial/Preclinical Development7%5%9%8%9%3%Clinical Development37D3D7#%Peri-/Post-approval/Registries/HEOR205%Quality and Regulatory13%8%72%Executive Management/C-level23)&)BA40The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Participant demographics(3 of 3)Drug developmentLetters indicate statistically significant difference between groups at the 90%confidence levelQ1.In which categories is your organization/company developing or commercializing products?Please select all that apply.S6.In which development phase(s)do you have decision-making responsibility?Please select all that apply.S7.Over the past 2 years,what is your level of involvement with outsourcing services to vendors/CROs(e.g.,deciding to keep activities in-house vs.outsource,vendor selection,vendor management,etc.)in support of your clinical trials?Demographics-Company SizeTotalLargeSmall/MidUS/CanadaEuropeAsia(A)(B)(C)(D)(E)Base:All respondents1506486734631S10Annual R&D SpendChina to USD ConversionUnder$100 million/Under 350 million*29%0P35%(Under$50M USD)$100 million to$999 million/350 million to 700 million*29%0P05%($50M to$100M USD)Small/Mid-size Biopharma Sub-Total57%00cRR%$1 billion to$2 billion/700 million to 3.5 billion*17A%0)%($100M to$500M USD)Over$2 billion/3.5 billion*25Y%0!7%(Over$500M USD)Large Biopharma Sub-Total430%07HH%S9Number of Employees1-49 employees11%0 !%0%6P-199 employees23%65(2 0-999 employees22)$2%1,000-9,999 employees19%,000 or more employees25V%1&3%S8Number of Unique Molecules/Compunds in Pipeline1 molecule/compound2%0%3%3%0%3%2 to 3 molecules/compounds22%91%)%4 to 5 molecules/compounds233 2%6 to 7 molecules/compounds17 %8 to 9 molecules/compounds9%3%5 ore more molecules/compounds27P%923%6mographics-Geography/Job Level/Primary Function TotalLargeSmall/MidUS/CanadaEuropeAsia(A)(B)(C)(D)(E)Base:All respondents1506486734631S3Company Headquarters LocationUS/Canada50BV%Europe293%5%3%Asia/Australia/Middle East/India Sub-Total21%5%0%Asia13%4%0R%Australia3%5%1%1%0%Middle East/India5%6%5%0%0&%S2Office LocationUS/Canada49BS0%0%0%Europe314(%00%0%Asia/Australia/Middle East/India Sub-Total21#%0%00%Asia12%0%0X%Australia3%5%1%0%0%Middle East/India6%6%6%0%0)%S4Job LevelDirector62wQXpa%Vice President24 !5%President1%0%1%1%0%0%C-level13%3!%9%3%S5Primary Functional ResponsibilityPre-trial/Preclinical Development7%5%9%8%9%3%Clinical Development37D3D7#%Peri-/Post-approval/Registries/HEOR205%Quality and Regulatory13%8%72%Executive Management/C-level23)&)mographics-Drug DevelopmentTotalLargeSmall/Mid(A)(B)Base:All respondents1506486734631Categories Engaged in for Drug Development/CommercializationNovel small molecule drugs55fHfT2%Generic small molecule drugs2307)%Biologics60sPYeU%Biosimilars20(ll therapies38E30R5%Gene therapies38P)3H5%Nucleic acid therapies180%Vaccines308$%9)%Diagnostics14&%Other3%2%3%3%4%0%Average number of categegories3.03.72.52.73.62.7Clinical Development Phases Where Respondent is Responsible for Making DecisionsDrug discovery34#B73)%Preclinical539dbP9%Phase I77fxxq%Phase II80ue%Phase III70riwgX%Phase IV/Late stage/Registries47PESAB%Level of Involvement in Outsourcing Clinical Trial ActivitiesHighly involved91%Somewhat involved9%7%4BBBAAA41The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Transformational trendsFull descriptions provided in the survey Accelerated development/approvals in rare/orphan disease Artificial intelligence(e.g.,to enable recruitment,advanced analytics,RWD/RWE)Big data and analytics(data science)Decentralized/hybrid trial elements Digitalization(e.g.,cloud computing,APIs,digital platforms)Greater use of RWD/RWE to complement data from clinical trials Increasing focus on patient diversity Innovative trial design Leveraging new technologies in drug development(e.g.,mRNA,CRISPR,gene-editing technologies)Patient-centricity(e.g.,patient participation in protocol design,partnering with patient advocacy groups,etc.)Personalized/precision medicine (e.g.,companion diagnostics)Sustainability effortsQ5.(REVISED)Which specific innovations,strategies,and/or technologies are being pursued currently by your organization?Please select all that apply.Q6.(NEW)Which specific strategies are being pursued currently by your organization?Please select all that apply.42The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Innovations/technologies and strategiesFull descriptions provided in the survey Artificial intelligence(e.g.,to enable recruitment,advanced analytics,RWD/RWE)Big data and analytics(data science)Digital/hybrid trial elements Digitalization(e.g.,cloud computing,APIs,digital platforms)Innovative trial design Leveraging new technologies in drug development (e.g.,mRNA,CRISPR,gene-editing technologies)Personalized/precision medicine (e.g.,companion diagnostics)Efforts to maximize asset value Greater use of RWD/RWE Mergers and acquisitions New approaches to investment and funding Patient-centricity Patient-diversity Risk-based contracts with vendors Strategies to reduce site burden Sustainability efforts Vendor rationalization(i.e.,reduce the number of vendors used)Q5.(REVISED)Which specific innovations and/or technologies are being pursued currently by your organization?Please select all that apply.Q6.(NEW)Which specific strategies are being pursued currently by your organization?Please select all that apply.43The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Leading therapeutic areas for drug developmentLetters indicate statistically significant difference between groups at the 90%confidence levelQ2.Which therapeutic areas are leading your organizations drug development pipeline today?Please choose up to 5.TotalLargeSmall/Mid(A)(B)Base:All respondents1506486Oncology70iq%Immunology/Rheumatology43GA%Rare Diseases31%5rdiovascular29B %Neurology290(%Infectious Diseases21 !%Hematology19%Gastroenterology15%Metabolic/Endocrine14rmatology13%9%Respiratory/Allergy11%9%Pediatrics9%8%9%Ophthalmology8%5%Orthopedics/Rheumatology7%6%8%Analgesic/Pain Management7%6%7%Womens Health6%6%6%Urology5%8%3%Hepatology5%9%1%Nephrology/Renal Diseases4%5%3%Critical Care3%2%3%Other1%2%0%Average number of TAs3.53.83.3BBB44The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Biggest challengesLetters indicate statistically significant difference between groups at the 90%confidence levelQ3.What are the biggest challenges your organization is currently facing?Please select your top 5 biggest challenges.Q3b.Listed below are the top challenges you indicated your organization is facing.Please rank these top challenges that your organization is facing,with Rank#1=Biggest challenge.Base:All respondentsSelected Top 5Ranked Top 2Selected Top 5Ranked Top 2Selected Top 5Ranked Top 2Rising cost of clinical trials49!D#S %Patient recruitment in clinical trials39!4B#%Increasing complexity of clinical trials3998%Maximizing asset value/ROI3333asibility and site selection30%7(%51%8%Lack of funding285#%Finding appropriate scientific/therapeutic expertise in vendors271%Elongated study startup time27%(%Lack of or limited relevant RWD/RWE23%9%9%8%Lack of internal resources/expertise to use RWD/RWE21%7%Navigating the changing regulatory landscape21%9# %8%Talent/staf shortages21%5%5%6%Diversity of patients enrolled19%3(%5%2%Patient retention in clinical trials19%3 %6%1%Logistics issues17%7%5%9%Research site burden17%6%5!%7%Using artificial intelligence(AI)in clinical development15%4%3%5ta integration15%5 %6%5ta management15%5%5%5%Incorporating decentralized/hybrid trial elements in trial designs13%3%2%3%Business continuity planning9%5%5%7%506486(A)(B)TotalLargeSmall/MidBBAAB45The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Patient participation strategiesOutsourcing of drug development activitiesLetters indicate statistically significant difference between groups at the 90%confidence levelQ19.(REVISED)Which patient-focused or recruitment strategies are being used currently by your organization?Select all that apply.Letters indicate statistically significant difference between groups at the 90%confidence levelBase:All respondents excluding dont know;varies by activity statement.Q17.Using the scale provided,please indicate how likely your company is to outsource each of the following drug development activities in the next 2 years.5-point scale:Not at all likely to Extremely likely.TotalLargeSmall/MidUS/CanadaEuropeAsia(A)(B)(C)(D)(E)Base:All respondents1506585654837Establishing or improving relationships with patient advocacy groups/organizations56cQ%Using more sites for each clinical study468R%Conducting research in more countries456Q%Patient assistance resources(e.g.,patient concierge,travel reimbursement,etc.)414E%Building more inclusive entry criteria into protocol designs39A8%Leveraging data and technology to identify target patients39E4%Patient-centric platforms/apps37A4%Patient education3593%Remote monitoring3518%Improving the diversity of clinical trial and ofce staf274%Increasing patient compensation27(%Virtual/telehealth visits20#%Home visits17%Mobile clinics11%7%None of the above1%0%2%3%0%0%0%0%0%0%0%0pppppp%TotalBiopharmaBiotechUS/CanadaEuropeAsiaQ19.Patient Participation StrategiesTotalBiopharmaBiotechUS/CanadaEuropeAsia1Establishing or improving relationships with patient advocacy groups/organizations56cQXRX%2 Using more sites for each clinical study468RUC)onducting research in more countries456QIR#%4 Patient assistance resources(e.g.,patient concierge,travel reimbursement,etc.)414EG0Building more inclusive entry criteria into protocol designs39A8A5%6 Leveraging data and technology to identify target patients39E46F5%7 Patient-centric platforms/apps37A46C)%8 Patient education35932&X%9 Remote monitoring35188F Improving the diversity of clinical trial and office staff274&3# Increasing patient compensation27(&B Virtual/telehealth visits20#%Home visits17 Mobile clinics11%7%6 None of the above1%0%2%3%0%0%Sample Size1506486734631AABBTotalLargeSmall/MidUS/CanadaEuropeAsia(A)(B)(C)(D)(E)Base:All respondents(excluding NA/dont know;varies by activity)143-15061-6480-8668-7344-4629-31Patient recruitment73d%Clinical laboratory and diagnostic services73c%Clinical data management64Hv%Clinical trial management-full-service/end-to-end trial support63Rr%RWD(i.e.,access to data)63Hu%Clinical monitoring/Clinical operations management63Vg%Clinical supply management60Pg%RWE(i.e.,generation of insights)59Fp%Pharmacovigilance solutions58Ci%Market access/Value demonstration58Dh%Pre-and post-approval safety analysis56Df%Biostatistical services569i%Regulatory consulting56Hb%Medical writing55Bd%Post-approval support53Ba%Site/KOL identification52DX%Quality and compliance services49V%Preclinical evaluations49FQ%Product registration48AS%Study design373&9X%0%0%0%0%0%0000000%Q17.OutsourcingTotalBiopharmaBiotechUS/CanadaEuropeAsiaAlpha OrdSorte Q17.%Extremely/Very Likely to Outsource(Top 2 Box)91 Patient recruitment73dxet2 Clinical laboratory and diagnostic services73cqh# Clinical data management64Hvdahd Clinical trial management-full-service/end-to-end trial support63Rrbaq5 RWD(i.e.,access to data)63HuYpbF Clinical monitoring/Clinical operations management63VgYrXW Clinical supply management60PgaWa8 RWE(i.e.,generation of insights)59FpItb9 Pharmacovigilance solutions58CiQRq0 Market access/Value demonstration58DhSYh11 Pre-and post-approval safety analysis56DfFge2 Biostatistical services569iSX13 Regulatory consulting56HbIca4 Medical writing55BdRYU15 Post-approval support53BaFaW16 Site/KOL identification52DX9gX17 Quality and compliance services49V7Tq18 Preclinical evaluations49FQFHX19 Product registration48AS2Yp 20 Study design373&9X%Alpha OrdSorte Q17.%Extremely Likely to Outsource91 Patient recruitment43#XHF)2 Clinical laboratory and diagnostic services451UGCB# Clinical data management31)09d Clinical trial management-full-service/end-to-end trial support36G6925 RWD(i.e.,access to data)286$(8F Clinical monitoring/Clinical operations management35 E87#W Clinical supply management35#D2A28 RWE(i.e.,generation of insights)265C9 Pharmacovigilance solutions30A)(2q0 Market access/Value demonstration241$&11 Pre-and post-approval safety analysis273#52 Biostatistical services266%313 Regulatory consulting2124 Medical writing264&315 Post-approval support244$ 16 Site/KOL identification23&5217 Quality and compliance services20%$#18 Preclinical evaluations23 %$#19 Product registration18%8% 20 Study design14%7&%Alpha OrdSorte Q17.%Very Likely to Outsource91 Patient recruitment30A0 E2 Clinical laboratory and diagnostic services281&$7&# Clinical data management330662)d Clinical trial management-full-service/end-to-end trial support270&95 RWD(i.e.,access to data)35095A$F Clinical monitoring/Clinical operations management286!55W Clinical supply management25$)8 RWE(i.e.,generation of insights)3425)0H9 Pharmacovigilance solutions28)($Hq0 Market access/Value demonstration34(8)5B11 Pre-and post-approval safety analysis30%4$)2 Biostatistical services303)913 Regulatory consulting35551F)4 Medical writing290&915 Post-approval support2914716 Site/KOL identification29%2(3&17 Quality and compliance services29%2 0H18 Preclinical evaluations26&!%919 Product registration303(7S 20 Study design23 $2%Alpha OrdSorte Q17.Sample Sizer per Statement(excl NA/dont know)91 Patient recruitment150648673463132 Clinical laboratory and diagnostic services149648572463123 Clinical data management148648473443164 Clinical trial management-full-service/end-to-end trial support1506486734631175 RWD(i.e.,access to data)146638371462946 Clinical monitoring/Clinical operations management150648673463157 Clinical supply management1496485724631188 RWE(i.e.,generation of insights)1436380684629109 Pharmacovigilance solutions1496386724631710 Market access/Value demonstration14964857246311211 Pre-and post-approval safety analysis1476483714531112 Biostatistical services14964857345311613 Regulatory consulting1496386724631814 Medical writing15064867346311115 Post-approval support14664827244301916 Site/KOL identification14964857246311517 Quality and compliance services14863857146311318 Preclinical evaluations1456184704431AAAAAAAAAAAAAAAA%extremely/very like to outsource46The Pulse 2024 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.Copyright,2024 by Pharmaceutical Product Development(PPD),the clinical research business of Thermo Fisher Scientific.All rights reserved.This report,including the information contained herein and commentary associated herewith(“materials”),is provided as a service of PPD.These Materials,based on publicly available information,market research conducted on behalf of PPD,as well as the knowledge and experience of PPDs employees,have been prepared solely for informational and educational purposes and should not be relied upon for professional advice.Any further use of these Materials requires the express written consent of PPD.Stay ahead of the curve with our experts takes on how these insights will influence the future of the drug development industry.Go deeper at
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Identifying clients needs and challenges in rare disease clinical developmentClinical researchppdIntroduction 3 Demographics 5 Outsourcing with a Contract Research Organization(CRO)12Patient Resources&Drug Development 18Clinical Trial Details 25 Contents2 Rare diseases survey 1INTRODUCTIONRARE DISEASES SURVEY3 Rare diseases survey by PPD,part of Thermo Fisher ScientificThis survey was conducted in April 2023 among 100 respondents who work in some capacity within the rare disease sector and who currently outsource(or plan to outsource within the next 2 years)some of their research needs to a contract research organization(CRO).The goal of the study was to identify clients needs and the challenges they are facing in terms of rare disease clinicaldevelopment.4 Rare diseases survey study was designed to be global but with a strong bias toward North America-based respondents,who comprise 70%of the audience;20%of the audience are from Europe,while the remaining 10%are in the Asia Pacific region.The bulk of the audience(72%)works at a biopharmaceutical company,while 18%are employed by a small molecule pharmaceutical company;the remaining 10%are at R&D laboratories(6%)or academic institutions/universities(4%).91%of the audience noted that they have at least 10 years of experience within the life science sector,and 87%are in a director-level position or higher,representing an approach that supports high relevance and strong data integrity.By design,only respondents whose companies or organizations are working within rare disease,and whose companies or organizations are either currently partnering with a CRO or plan to do so within the next 24 months,were able to complete the survey.RESPONDENT EXPERIENCEOf respondents have 10 years life sciences experiences91%RESPONDENT SENIORITYOf respondents are director level or above87%5 Rare diseases survey asked if they are currently partnering with a CRO as part of their rare disease drug development plan,86%of respondents noted that they were:55%of that group of respondents said they were working with at least 3 contract partners.44%of these respondents expect their outsourcing activity to increase over the upcoming 24 months,while 47lt that their partnerships would likely remain at the current level.The decision drivers that were identified as being the most important when selecting a CRO to partner with on rare disease studies were“Therapeutic expertise”,“Direct experience with cell&gene therapies”,“Reliability”,“Agility/flexibility”,and“End-to-end service”.47%of respondents reported that their companies conducted an oncology-focused rare disease clinical trial within the most recent 24 months;Genetic Disorders was the second highest therapy area at 42%,with Neurology in third(33%).The most frequently selected challenge faced by the audience when conducting rare disease clinical trials was“Recruiting patients for the trial”,which was chosen by 52%of the participants.“Complexity of the clinical trial requirements”was a distant second at 16%.Decentralized clinical trials within the rare disease space were used to varying degrees by respondents companies:11%reported that a fully decentralized model was used for at least one of their rare disease clinical trials over the most recent 12 months,while 57%reported that at least one“hybrid”clinical trial,involving both traditional and decentralized aspects,was conducted over the same period.ECRF and eConsent software,at 44%and 42%,respectively,were the most used decentralized elements.Respondents reported that“Coordination of visits or assessments over time”(64%)and“Transportation to&from clinical trial sites”(51%)were the top needs for patients during their in-person clinical trial visits.Of respondents expect their outsourcing activity to increase over the next 24 months44%Of respondents whose companies are currently outsourcing work for rare disease clinical trials are using 3 CROs55%Of respondents said that their companies are currently utilizing CROs as part of their rare disease drug development efforts86%6 Rare diseases survey section of the survey was dedicated to answering an assortment of questions around topics such as patient advocacy groups,natural history studies,patient&disease registries,tokenization,and drug development.40%of respondents noted that relationship building between their companies and patient advocacy groups are handled by an internal unit;37%said that a mix of internal and external resources are utilized to create and maintain these relationships.Among the study participants who said that their companies utilized natural history studies(79%of the audience),“Development of clinical outcome assessments”(57%)and“Identifying patient population”(56%)were named as the top two uses for the resource.21%of respondents claimed that they were neither using nor considering using a natural history study to build their clinical development program.Patient/disease registries appear to be a useful tool among the study audience,as only 9%of respondents said that they are not considering the use of a registry.The most frequently given reason for utilizing a patient/disease registry was“Clinical trial design/identify key sub-groups”(66%);the next highest reason,“Uncover unmet needs and market potential”,was selected by 40%of respondents.Of the novel approaches to drug development that were included as part of the study,“External/Synthetic control arms”drew the most interest at 46%;“Basket/bucket trials”were second with 44%.With none of the given options breaking the 50%mark with participants,this might represent an opportunity to build awareness and knowledge of the various tools.Of respondents feel that patient recruitment is the most challenging aspect of rare disease clinical trials52%Of respondents reported that their companies have conducted hybrid clinical trials that utilize some decentralized elements over the most recent 12 months.57%Of respondents companies conducted an Oncology-focused rare disease clinical trial in the past 24 months.47%7 Rare diseases survey 2DEMOGRAPHICSRARE DISEASES SURVEY8 Rare diseases survey or Other Academic Institution4%2.1 Survey Participants Company/Organization TypeResearch and Development(R&D)Laboratory6r%Biopharmaceutical CompanySmall Molecule Pharmaceutical Company2.2 Survey Participants Regional Location70%North America20%Europe10%Asia Pacific9 Rare diseases survey Manager1%2.3 Survey Participants Years of Life Science Experience2.5 Survey Participants Department/Work Area2.4 Survey Participants Job Title54%Research and Development (R&D)15%Business Development5%Drug Production/Manufacturing17%Clinical Operations/Management2%Quality Assurance/Quality Control2%Purchasing/Procurement2%External Manufacturing1%Supply Chain1%Regulatory Affairs38I%Director/Senior Director91%More than 10 yearsExecutive(VP,President,CEO,CIO,CTO,COO,etc.)Manager/Senior Manager12tween 3 and 5 years1tween 5 and 10 years8 Rare diseases survey respondents said they act as a point-of-contact between the contractor and their company21%of respondents said they are part of a team that establishes the criteria for the contractor selection process70%of respondents said they are part of the decision-making group that conducts research into/interviews/selects a potential partnership opportunityIs your company currently developing and/or commercializing a drug ortherapy that is used in the treatment of patients with a rare disease?2.6 Survey Participants Companys Involvement with Rare Disease2.7 Survey Participants Role When Deciding on/Working with Outsourcing Partners100%Yes70%9! Rare diseases survey 3OUTSOURCING WITH A CONTRACT RESEARCH ORGANIZATION(CRO)RARE DISEASES SURVEY12 Rare diseases survey company has multiple therapeutics for rare disease(s)across various stages of development.My company has multiple therapeutics for rare disease(s)in the early stages of development.My company has a single therapeutic for rare disease(s)in the early stages of development.My company has multiple therapeutics for rare disease(s)in the late stages ofdevelopment.My company has a single therapeutic for rare disease(s)in the late stages of development.3.1 Survey Participants Companys Size3.2 Survey Participants Companys Rare Disease Portfolio Status32!%74%6B6%Small company(Up to 500 employees)Mid-sized company(501 to 2,500 employees)Large company(2,500 employees)13 Rare diseases survey Partnering with a Contract Research Organization(CRO)Is your company currently utilizing any Contract Research Organizations(CROs)as part of your rare disease drug(s)development?3.3a Partnering with a CRO Number of Contract PartnersWith how many CROs are you currently working?NOTE:Total number of respondents is 86;the respondents who answered“Yes”to Question 3.3 were prompted to answer this.86%YesNo22%1 CRO23%2 CROs34%3-4 CROs21%5 CROsNOTE:Total number of respondents is 14;the respondents who answered“No”to Question 3.3 were prompted to answer this.3.3b Partnering with a CRO Number of Contract PartnersWith how many CROs does your company expect to work when it does begin to outsource projects?29%1 CRO43%2 CROs21%3-4 CROs5 CROs7 Rare diseases survey Current Outsourcing Activity(CROs):Research NeedsApproximately what percentage of your research needs is your company currently outsourcing?NOTE:Total number of respondents is 86;the respondents who answered“Yes”to Question 3.3 were prompted to answer this.Up to 20!%to 40(A%to 60$a%to 80$%to 100%Research NeedsRespondents3.3d Possible Outsourcing Activity(CROs):Research NeedsApproximately what percentage of your research needs do you expect your company to outsource?NOTE:Total number of respondents is 14;the respondents who answered“No”to Question 3.3 were prompted to answer this.Up to 20%7!%to 40!A%to 60)a%to 80!%to 100!%Research NeedsRespondents15 Rare diseases survey will decrease8%I am not sure1%3.3e Future Outsourcing Behavior(CROs)What do you believe your companys outsourcing activity with CROs will be like 24 months from now compared to its current level?It will increase44G%It will stay at the same levelNOTE:Total number of respondents is 86;the respondents who answered“Yes”to Question 3.3 were prompted to answer this.Small-sized CRO5%3.4a Preference in CRO SizeWhat size CRO do you prefer to work with?NOTE:Total number of respondents is 55;the respondents who answered“Yes”to Question 3.4 were prompted to answer this.49E%Large-sized CROMedium-sized CRONot Sure6%3.4 Decision Driver:Size of the Contract Research Organization(CRO)When considering a partnership with a CRO,do you have a preference in the size of the contract organization?559%YesNo16 Rare diseases survey Decision Drivers:Choosing a CRO Partner for Rare Disease StudiesWhat factors appeal to you when making your choice for a CRO partner for Rare Disease studies?(Respondents were asked to rank their top 5 decision drivers from the 11 possible responses,with choice#1 being the driver they felt was most important.The lower the number,the more important respondents felt the factor was.)2.831.962.69Direct experience with cell and gene therapiesReliability3.073.263.25End-to-end serviceRelationships with clinical trial sitesCost3.84Digital solutions/technical capabilities3.94Communication3.94Relationships with patient advocacy groups3.97AccessibilityTherapeutic expertisev2.94Agility/flexibility17 Rare diseases survey 4PATIENT RESOURCES&DRUG DEVELOPMENTRARE DISEASES SURVEY18 Rare diseases survey Establishing Relationships with Patient Advocacy GroupsDoes your company build and maintain its own relationships with patient advocacy groups,or does it rely on third parties to establish connections?4.1a Benefits of Relationships with Patient Advocacy GroupsWhat benefits have you experienced through your connection with patient advocacy groups?40%of respondents said their company has an internal unit that establishes relationships with patient advocacy groups.23%of respondents said their company looks to other organizations to build connections with patient advocacygroups.37%of respondents said their company uses both internal and external resources to create and maintain relationships with patient advocacy groups.10%5%83%2 %9%3%6$%2%Collaborative opportunitiesSupport and empowermentBetter communicationFirst-hand experience and feedback from patientsInnovation/technologiesAccess to patients for clinical trialsAccess to grant fundingIncrease in enrollment rateTransparency/agility/faster responsesAwareness and education about rare diseasesTeam motivationPatient recruitmentNOTE:Total number of respondents is 100;multiple answers were allowed.40#7T%A better understanding of patients needs19 Rare diseases survey Using Natural History StudiesWhich of the following uses of natural history studies are you considering to inform your clinical developmentprogram?(Multiple answers were allowed)4.3 Using Disease or Patient RegistriesIn which of the following areas are you considering the use of a disease or patient registry?(Multiple answers were allowed)Development of clinical outcome assessmentsIdentifying patient populationIdentifying or developing biomarkersDesigning external control studiesWe are not considering a natural history study5675!Wf%9%1554%Clinical trial design/identify key sub-groupsWe are not considering a registryOtherUncover unmet needs and market potentialSupport regulatory and HTA submissionsPre-clinical portfolio prioritization/investment decisionsSupport go-to-market activities4.4 Novel Approaches to Drug DevelopmentWhich of the following novel approaches to drug development are of interest to your company for use in clinicaltrials?(Multiple answers were allowed)4.5 Clinical Trial Site LocationsIs your company open to conducting clinical trials in less-conventional geographic regions at sites that are(a)relatively new to clinical trials but(b)appropriately supported for success?External/Synthetic control armsBasket/bucket trialsPlatform studiesUmbrella trialsMaster observational trials(MOTs)Other4475 %2Fc %YesNoNot Sure21 Rare diseases survey Challenges Facing the Rare Disease Drug Development SpaceWhat do you consider to be the biggest challenges facing rare disease drug development in the upcoming 10-year period?(Multiple answers were allowed)59%Concerns with costs54S%Increasing complexity in clinical trial designHeterogeneity of patient population with certain rare diseases41%807%2!%Expanded regulatory requirementsDetermining responsibility in clinical trial governanceLack of understanding of rare diseases natural historyLow prevalence ofconditionOtherFinding qualified staff to handle increased technological demandsAccess to appropriate patient pools62 Rare diseases survey 5CLINICAL TRIAL DETAILSRARE DISEASES SURVEY23 Rare diseases survey Rare Disease Clinical Trials by Therapy AreaAcross which therapeutic areas has your company run rare disease clinical trials over the most recent 24 months?(Multiple answers were allowed)47B%OncologyGenetic disorders331%8%NeurologyMusculoskeletal disordersImmunologyOphthalmology21%4%6%7%2%4%HematologyToxicologyOtherHormonal disordersInfectious diseaseRespiratory diseaseNon-malignant disorders24 Rare diseases survey Pediatric Patients within Rare Disease Clinical TrialsApproximately what percentage of the rare disease clinical trials that you have conducted over the most recent 24 months included pediatric patients?5.3 Challenges Faced when Conducting Rare Disease Clinical TrialsFrom the following list,what do you feel is the most difficult or challenging aspect of rare disease clinical trials?16%1%9%1R%Recruiting patients for the trialComplexity of the clinical trial requirementsDevelopment and access to patient registriesRegulatory barriersEducating patients and their family/caregivers5%8%2%6%Patient retentionIdentifying a diverse patient populationCollection and management of dataLength of the trial startup process0)%Up to 203!%to 40 A%to 60%6a%to 80%6%to 100%6%Rare Disease Clinical TrialsRespondents25 Rare diseases survey How CRO Partners Can Help Address Challenges with Rare Disease Clinical TrialsWhat actions would you like to see from a CRO partner to help navigate through these challenges?(Multiple answers were allowed)24%Assistance with patient recruitment/enrollmentPatient identification/maintain a patient database16%Relationships with/access to regional clinical trial sitesDemonstrate overall expertise/experience,guidance,andcommitment10%9%7%1%5%9%8%5%8%4%Greater/improved communication and listeningAssistance with patient retention andcomplianceGreater adaptability/flexibility andefficiencyAbility to conduct decentralized clinical trialsRegulatory supportEngagement with patient advocacy groups and KOLsKnowledge of/access within therapeutic areas of interestImproved technology and technical expertiseOtherHelp in improving clinical trial design26 Rare diseases survey fully decentralized clinical trial(DCT)that leverages digital tools and supporting services,such as eConsent,eCOA,home healthcare,etc.to enable the patient to complete some or all scheduled visits from their home5.4 Types of Clinical Trials Conducted for Rare Disease TherapiesWhat types of clinical trials has your company conducted for rare disease therapies during the most recent 12-month period?(Multiple answers were allowed)5.5 Utilizing Decentralized Elements in Rare Disease Clinical TrialsApproximately what percentage of the rare disease clinical trials that were conducted during this period utilized remote or decentralized elements?A traditional clinical trial that is fully conducted at a designatedsite48%A“hybrid”clinical trial that incorporated at least some decentralized clinical trialelements57%0%Up to 201!%to 402A%to 60a%to 80%3%to 100%3%Rare Disease Clinical TrialsRespondents27 Rare diseases survey Types of Decentralized Elements Used in Decentralized Rare Disease Clinical Trials Which decentralized elements did you utilize when conducting a hybrid or fully remote rare disease clinical trial?(Multiple answers were allowed)42D%Electronic informed consent/eConsent softwareElectronic case report forms/eCRF software371%Electronic clinical outcomes assessment(eCOA)Telemedicine services26#%Electronic patient-reported outcomes/ePro softwareIn-home nurse services/health visits98%9%Pharmacy-based clinicsRemote patient monitoringMobile clinics16%Remote or satellite clinical trial sitesWearable technologies28 Rare diseases survey Future Plans for Reusing Decentralized ElementsWhich of the following decentralized elements that were utilized when conducting a hybrid or fully remote rare disease clinical trial would you be interested in using again?(Multiple answers were allowed)5.5c Future Plans for Trying Previously Unused Decentralized ElementsWhich of the following decentralized elements that were not utilized when conducting a hybrid or fully remote rare disease clinical trial would you be interested in using in future clinical trials?(Multiple answers were allowed)Electronic clinical outcomes assessment(eCOA)32%Electronic clinical outcomes assessment(eCOA)8%Other0%Other2%Mobile clinics4%Mobile clinicsWearable technologies11%Wearable technologies29%Telemedicine services23%Telemedicine services14%In-home nurse services/health visits17%In-home nurse services/health visits161%Pharmacy-based clinics2%Pharmacy-based clinicsRemote or satellite clinical trial sites11%Remote or satellite clinical trial sites15%Remote patient monitoring26%Remote patient monitoring23%Electronic informed consent/eConsent software12%Electronic informed consent/eConsent softwareElectronic patient-reported outcomes/ePro software17%Electronic patient-reported outcomes/ePro softwareElectronic case report forms/eCRF software31%Electronic case report forms/eCRF software14) Rare diseases survey Patient Requirements for In-Person Visits During Clinical TrialsDo patients in your rare disease clinical trials require any of the following services for their in-person clinic visits?(Multiple answers were allowed)5.7 Additional Resources Used to Increase/Share Knowledge about Rare DiseaseWhich of the following resources does your company use to network and share knowledge about rare diseases?(Multiple answers were allowed)Conferences/eventsSeminars/webinarsOnline forums or community sitesSocial media67UPtd%Coordination of visits or assessments over time31()(vice/software training51%Transportation to/from clinical trial sitesMobility assistanceAssistance with medical equipmentAssistance with reimbursement needs35%Medication reminders30 Rare diseases survey Additional Resources Used to Increase/Share Knowledge Online ForumsWhich online forums or community sites do you find most beneficial?(Multiple answers were allowed)NOTE:Total number of respondents is 55;respondents who chose“Online forums or community sites”in Question 5.7 were prompted to answer this.There is a total of 83 mentions in this question;the table above highlights the Top 10 answers from that list.Rare disease forumsTwitterOnline forumsLinkedInPatient advocacy sitesDisease specific groupsCompany websiteRare Disease Clinical Research Network(RDCRN)FacebookConferences16%7%7%4%4%4%41 Rare diseases survey Additional Resources Used to Increase/Share Knowledge Conferences&EventsWhich conferences/events do you find most beneficial?(Multiple answers were allowed)NOTE:Total number of respondents is 74;respondents who chose“Conferences/events”in Question 5.7 were prompted to answer this.There is a total of 119 items mentioned in this question;the table above highlights the Top 17 answers from that list.Other9%Orphan Drug and Rare Disease conferenceDisease specific conferences9%World conferencesWorld Congress on Rare DiseasesAmerican Thoracic SocietyAmerican Society of Hematology(ASH)American Association of Cancer Research(AACR)American Society of Clinical Oncology(ASCO)European Conference on Rare DiseasesNational Organization for Rare Disorders(NORD)American Society of Gene&Cell Therapy(ASGCT)European Society of Gene&Cell Therapy(ESGCT)Patient organizationLarge scale multinational eventsRare Disease Day ForumEuropean Society for Medical Oncology(ESMO)7%5%3%7%7%4%4%4%3%9%3%8%5%32 Rare diseases survey Additional Resources Used to Increase/Share Knowledge Social MediaWhich social media platforms do you find most beneficial?(Multiple answers were allowed)NOTE:Total number of respondents is 50;respondents who answered“Social media”in Question 5.7.were prompted to answer this.There is a total of 74 items mentioned in this question;the table above highlights the Top 13 answers from that list.FacebookShare4RarePatient advocacyLinkedInTikTokSearch advertisingPatient groupsTwitterLinks to patient groupsPharmaceutical publicationInstagramMedical conference,symposium,and publications44%4%20%2%2%2%2%6H%4%2 24 Thermo Fisher Scientific Inc.All rights reserved.All trademarks are the property of Thermo Fisher Scientific and its subsidiaries unless otherwise specified.08.2024 Learn more at
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