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

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.