摘要
随着嵌合抗原受体T细胞(CAR-T)产品获批上市数量的增加,其监管要求日益规范化。美国食品药品监督管理局(FDA)和中国国家药品监督管理局(NMPA)等监管机构针对CAR-T疗法的长期安全性和有效性提出了严格的监管要求,并强调了真实世界数据(RWD)和真实世界证据(RWE)的重要性。本文总结了国内外CAR-T产品的上市概况和相关监管要求的演变趋势,分析了一个国产CAR-T产品典型案例的真实世界研究(RWS)数据,即阿基仑赛注射液的真实世界疗效和安全性。同时,提出了关于推动CAR-T疗法RWS的思考,强调了提高重视程度、建立长期监测机制和反馈机制、规范操作流程以及加强患者支持的重要性,旨在为相关监管人员、研究人员、临床医师和企业人员等提供参考,促进CAR-T疗法的规范、安全、有效应用。
With the growing number of chimeric antigen receptor T-cell(CAR-T)products approved for market,regulatory requirements are becoming more standardized.Authorities such as the U.S.Food and Drug Administration(FDA)and China's National Medical Products Administration(NMPA)have established rigorous standards for the long-term safety and efficacy of CAR-T therapies,highlighting the significance of real-world data(RWD)and real-world evidence(RWE).This paper reviews the global and domestic regulatory landscape and evolving trends in CAR-T oversight,and examines real-world study(Rws)data from a representative domestic case:the effectiveness and safety of Axicabtagene Ciloleucel injection.The paper also offers reflections on promoting Rws for CAR-T therapies in China,emphasizing the importance of raising awareness,establishing long-term monitoring and feedback mechanisms,standardizing procedures,and enhancing patient support.The aim is to provide guidance for regulators,researchers,clinicians,and industry stakeholders to support the safe and effective use of CAR-T therapies.
作者
赵婧华
ZHAO Jing-hua(Shanghai Fosun Kairos Biotechnology Co.,Ltd.)
出处
《中国食品药品监管》
2025年第6期42-51,共10页
China Food & Drug Administration Magazine
关键词
嵌合抗原受体T细胞疗法
监管要求
真实世界研究
中国人群
案例分析
chimeric antigen receptor T cell(CAR-T)therapy
regulatory requirements
real-world study
Chinese population
case analysis
