省级药监局视角下CAR-T治疗药品生产监管探讨

Discussion on Production Supervision of CAR-T Therapeutic Drugs from the Perspective of Provincial Medical Products Administrations

目的:为省级药监局建立和完善已上市CAR-T治疗药品生产监管制度提供参考。方法:分析CAR-T治疗药品的产品、药学研究、临床研究、生产、临床使用等方面特点,介绍WHO、PIC/S、美国、欧盟等监管机构的监管政策,梳理我国目前的监管体系。结果与结论:从省级药监局的职责出发,提出已上市CAR-T治疗药品生产监管应重点关注的内容,包括变更管理关注可比性研究、GMP符合性检查关注6个关注点和延伸检查、GVP符合性检查关注上市后监测、日常监督检查关注风险信号和企业对医疗机构的管理策略。

Objective:Aims to provide references for provincial medical products administration to establish and improve regulatory systems for the production supervision of post-marketing CAR-T therapeutic drugs.Methods:The analysis encompasses the distinctive characteristics of CAR-T therapeutic drugs,including product features,pharmaceutical and clinical research,production,and clinical use.Policies from regulatory agencies such as WHO,PIC/S,FDA,and EMA are introduced,and the existing regulatory system in China is outlined.Results and Conclusion:From the perspective of provincial medical products administration,key areas for attention in the production supervision of post-marketing CAR-T therapeutic drugs are identified.These include a focus on comparability research in change management,six specific points in GMP compliance inspections,extended inspections,post-marketing monitoring in GVP compliance inspections,and daily supervision inspections that focuses on risk signals and enterprise management strategies for medical institutions.