科学表征细胞体内过程,推动细胞治疗产品高质量发展  

Scientifically Characterizing the Cellular Behavior in vivo to Promote High-Quality Development of Cell Therapy Medicinal Products

作　　者：魏春敏 潘鹏玉 高丽丽 吴婷婷 王玉珠[1] WEI Chun-min;PAN Peng-yu;GAO Li-li;WU Ting-ting;WANG Yu-zhu(Center for Drug Evaluation,National Medical Products Administration;National Institutes for Food and Drug Control)

机构地区：[1]国家药品监督管理局药品审评中心 [2]中国食品药品检定研究院

出　　处：《中国食品药品监管》2024年第11期32-43,共12页China Food & Drug Administration Magazine

基　　金：药品监管科学全国重点实验室课题“先进治疗产品体内过程评价方法”(2024SKLDRS0231)。

摘　　要：细胞治疗技术的突飞猛进引领了复杂难治性疾病治疗范式的重要转变,以CAR-T为代表的细胞治疗产品陆续获批上市,标志着药物治疗史上一个新时代的到来。与临床治疗新技术不同,用于上市申请的细胞治疗产品应遵循药品注册相关法规要求,开展必要的临床药理学研究,科学表征其体内过程。为推动我国细胞治疗产品高质量发展,国家药品监督管理局药品审评中心组织起草了《细胞治疗产品临床药理学研究技术指导原则(试行)》,针对细胞治疗产品体内过程特点,重点阐述开展必要的细胞动力学、药效学及影响因素研究,探索细胞治疗产品安全有效性可控关键要素的相关考虑。The rapid development of cell therapy technology has led to an important paradigm shift in the treatment of complex and refractory diseases,marking the arrival of a new therapeutic drug era represented by CAR-Ts approved in succession.Unlike that of new clinical technologies,necessary clinical pharmacology studies of cell therapy medicinal products should be carried out to make the cellular behavior in vivo clear and meet the regulatory requirements of new drug registration.The Center for Drug Evaluation of the National Medical Products Administration compiled“Technical Guidance for Clinical Pharmacology Research of Cell Therapy Medicinal Products”,focusing on the characteristics of cellular behavior in patierts,elaborating on the relevant considerations of necessary studies on cellular kinetics and pharmacodynamics,exploring the key factors involving the safety and efficacy,in order to promote the high-quality development of cell therapy medicinal products in China.

关 键 词：细胞治疗产品 嵌合抗原受体修饰T细胞 肿瘤浸润淋巴细胞 T细胞受体工程化修饰T细胞 

分 类 号：R95[医药卫生—药学]