美国 FDA 生物制品评估与研究中心 （CBER） 举措的最新情况.pdf

1、2024 CMC Strategy Forum JapanTokyo Marriott Hotel,JapanDecember 9-10,2024Update on the initiatives at the Center for Biologics Evaluation and Research(CBER),U.S.FDAIngrid Markovic,Ph.D.Senior Science Advisor for CMCFDA/CBERCBER ICH Quality Lead&Regulatory Chair for ICH Q6 EWG12CBER Products Gene the

2、rapies Human tissues and cellular products Xenotransplantation products Allergenics Live biotherapeutic products Vaccines(preventative and therapeutic)Whole blood,plasma,and blood products Devices related to biologicswww.fda.gov3Presentation Outline Lifecycle Management Tools CP/PACMP Application of

3、 platform technology provision Efforts to facilitate CMC Readiness Pilot(CDRP)Program Exploring concurrent submission and product review with other regulatory authorities for Cell&Gene Therapieswww.fda.govLifecycle Management Tools-CP/PACMP-Past,Present&Future5Some examples include:Scale-up/scale-ou

4、t,new manufacturing site,new analytical assayReacting to manufacturing problem or contaminationReagent or material is no longer available or in short supplyImprove product quality based on new scientific or clinical informationProcess improvements(e.g.,more efficient or streamlined process,better im

5、purity profile,etc.)Cost effectivenessCell bank has expired or been exhaustedChanges can occur at any point in the product lifecycle,but one needs to ensure that the change does not negatively impact product quality as it relates to S&EManufacturing Changes Are Inevitable6Tools:ICH Q12 Established C

6、onditions(EC)Elements(e.g.parameters,attributes,controls,specifications,etc)necessary to assure product quality that require a submission if changed Post-approval Change Management Protocols(PACMP)Aligned with US FDAs comparability protocol(CP)Predictability regarding timing of implementation of PA