中美附条件批准药品上市后确证性研究分析  

Confirmatory Studies on Post-Marketing of Conditional Appoval of Drug in China and the United States

作　　者：魏欣悦 董玲 Wei Xinyue;Dong Ling(School of Chinese Materia Medica.BUCM,Beijing 100029,China;School of Life Sciences,Beijing University of Chinese Medicine,Beijing 100029,China)

机构地区：[1]北京中医药大学中药学院,北京100029 [2]北京中医药大学生命科学学院,北京100029

出　　处：《中国药事》2025年第3期253-265,共13页Chinese Pharmaceutical Affairs

摘　　要：目的:旨在探寻中美附条件批准药品上市后确证性研究政策的完善方向,以及研发企业在制定上市后确证性研究研发策略时的考量点,以供业界参考。方法:通过对比中美两国附条件批准药品上市后确证性研究的相关法规政策要求,以及中美两国在附条件批准上市后确证性研究上的实践情况,结合实际案例,对两国上市后确证性研究设计方案进行分类汇总,进而深入探讨中美两国在政策及实施上的优化方向。结果与结论:中美两国对附条件批准上市后确证性研究的启动和完成时间、研究结果解读的要求在同步收紧,标准也愈发清晰,但确证性研究开展进度缓慢仍然是两国监管部门面临的挑战。虽然随机对照试验被美国食品药品管理局(Food and Drug Administration,FDA)和国家药品监督管理局药品审评中心(Center for Drug Evaluation,CDE)推荐用于确证性研究设计,但研发企业在单臂试验的使用上仍有可尝试和突破的空间。Objective:To explore the improvement direction of the regulations and policies on confirmatory studies on post-marketing of conditional approval of drug in China and the United States,as well as the considerations of research and development(R&D)enterprises in building development strategies of confirmatory studies,and for the industry’s reference.Methods:By comparing the requirements of relevant regulations and policies of confirmatory studies on post-marketing of conditional approval of drug in China and the United States,and the practice of the studies in the two countries,with the analyzing of real cases,the design schemes of post-marketing confirmatory studies in China and the United States were classified and summarized,and then the optimization direction of the policy and implementation in China and the United States was further discussed.Results and Conclusion:The requirements for the initiation time,completion time and the interpretation of research results of post-marketing confirmatory studies in China and the United States have been tightened simultaneously,and the criteria have become clearer,however,the slow progress of confirmatory studies is still a challenge faced by the regulatory authorities in both countries.Although randomized controlled trials are recommended by the Food and Drug Administration(FDA)and Center for Drug Evaluation(CDE)of NMPA for confirmatory study design,there is still room for experimentation and breakthrough in the use of single-arm trials for R&D enterprises.

关 键 词：附条件批准 加速批准 确证性研究 药品监管 审评审批 

分 类 号：R95[医药卫生—药学]