美国食品药品监督管理局接受不确定性:治疗多重耐药的人类免疫缺陷病毒感染的单克隆抗体加速获批  

U.S.Food and Drug Administration accepting uncertainty:expedited approval for a monoclonal antibody for the treatment of multi-drug resistant human immunodeficiency virus infection

作　　者：李冠乔 卢希[2] 孟醒 陈晓媛 张绮 王涛 张林琦[2] LI Guan-qiao;LU Xi;MENG Xing;CHEN Xiao-yuan;ZHANG Qi;WANG Tao;ZHANG Lin-qi(Tsinghua Clinical Research Institute,School of Medicine,Tsinghua University,Beijing 100084,China;School of Medicine and Vanke School of Public Health,Tsinghua University,Beijing 100084,China;Center of Drug Evaluation,National Medical Products Administration,Beijing 100022,China)

机构地区：[1]清华大学医学院清华临床试验中心,北京100084 [2]清华大学医学院清华大学万科公共卫生与健康学院,北京100084 [3]国家药品监督管理局药品审评中心,北京100022

出　　处：《中国临床药理学杂志》2021年第1期89-93,96,共6页The Chinese Journal of Clinical Pharmacology

摘　　要：未满足的临床需求影响着临床试验的科学设计和药品审批的监管决策。美国食品药品监督管理局(FDA)于2018年批准了全球首个抗艾滋病的单克隆抗体艾巴利珠单抗(ibalizumab)用于治疗多重耐药的人类免疫缺陷病毒(HIV)感染。该药物的上市批准主要基于1项纳入40例患者的Ⅲ期单臂的简化临床试验。这体现了面对抗HIV多重耐药的未满足的医疗需求,FDA所做的风险和获益的权衡,以及采取的科学、灵活的审评态度。本文以此药为例,讨论了简化临床试验设计和FDA在审评中的监管考量,以期对我国药品审评的制度改革有所裨益。Unmet medical needs influence the scientific design of clinical trials and regulatory decisions for drug approval.In 2018,the U.S.Food and Drug Administration(FDA)granted the marketing approval of the world’s first anti-human immunodeficiency virus(HIV)monoclonal antibody,ibalizumab,for the treatment of multi-drug resistant(MDR)HIV infection,based primarily on a single-arm phase III streamlined clinical trial,involving 40 MDR HIV patients.This underscores the FDA’s consideration of benefit-risk balance and flexibility on drug review when addressing unmet medical needs.In this paper,taking this as an example,we discussed the streamlined clinical trial design and regulatory considerations by the FDA,which might have implications on regulatory reform in China.

关 键 词：艾滋病 人类免疫缺陷病毒感染 多重耐药 药品审评 简化临床试验设计 

分 类 号：R95[医药卫生—药学]