我国先进治疗药品的范围及分类研究和建议  

Research and Suggestions on the Scope and Classification of Advanced Therapy Medicinal Product in China

作　　者：卢加琪 刘丹 寇雅真 王雪 陈昊 王文波 尹华静 王晶 孙涛[1] 韦薇[1] 鲁爽[1] 王庆利[1] 何伍[1] 王涛[1] LU Jia-qi;LIU Dan;KOU Ya-zhen;WANG Xue;CHEN Hao;WANG Wen-bo;YIN Hua-jing;WANG Jing;SUN Tao;WEI Wei;LU Shuang;WANG Qing-li;HE Wu;WANG Tao(Center for Drug Evaluation,NMPA)

机构地区：[1]国家药品监督管理局药品审评中心

出　　处：《中国食品药品监管》2024年第5期10-25,共16页China Food & Drug Administration Magazine

摘　　要：近年来先进治疗药品研发申报呈爆发式增长,引领生物医药的第三次产业革命。全球范围内嵌合抗原受体T细胞(CAR-T)治疗产品、腺相关病毒载体基因治疗产品等不同类型的细胞和基因治疗产品相继批准上市,为复发难治疾病提供了有效的治疗手段。先进治疗药品通常采用细胞或组织经基因修饰和(或)体外操作制备,种类包括基因修饰细胞、核酸、病毒载体和人工组织等。由于这类产品类型复杂、多样,明确其定义和分类,有利于优化产品注册申报路径,加速各类产品技术指南的发布,推动产品快速研发上市,助力我国药品监管与国际接轨。本文通过调研美国食品药品监督管理局(FDA)、欧洲药品管理局(EMA)、日本药品医疗器械综合机构(PMDA)等药品监管机构在法规层面对先进治疗药品的定义及监管分类情况,结合我国产品申报现状和审评积累,提出了对先进治疗药品分类和描述的建议,为我国相关监管政策制定提供参考。In recent years,the development and registration of advanced therapy medicinal product(ATMP)have shown explosive growth,leading the third industrial revolution of biomedicine.Different types of cell and gene therapy products,such as CAR-T cell therapy products and AAV gene therapy products,have been approved worldwide,offering effective treatments for relapsed and refractory diseases.ATMP are typically genetically modified and/or manufactured via in vitro manipulation of cells or tissues,including genetically modified cells,nucleic acids,viral vectors,and tissue engineering products.Due to the complexity and diversity of ATMP,clarifying their definition and classification is conducive to optimizing the drug registration path,accelerating the release of technical guidelines for various products,promoting the rapid development and marketing of ATMP,and fostering international regulatory convergence.This article investigates the definition and regulatory classification of ATMP in FDA,EMA,and PMDA.It proposes a description and classification of ATMP for drug regulation in China based on the current landscape of ATMP applications and review experiences,which could serve as a reference for formulating relevant policies and regulations.

关 键 词：先进治疗药品 药品监管 法律法规 分类 细胞治疗药品 基因治疗药品 

分 类 号：R95[医药卫生—药学]