摘要
Abstract
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.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Key words
chimeric antigen receptor T cell(CAR-T)therapy/regulatory requirements/real-world study/Chinese population/case analysis分类
医药卫生
