欧美罕见病药物研发的监管机制分析及启示  

Analysis of regulatory mechanisms of rare disease drug development in Europe and United States

作　　者：张皖晋 卓阳 孙搏 ZHANG Wan-jin;ZHUO Yang;SUN Bo(Shanghai Center for Drug Evaluation and Inspection,SHANGHAI 201203,China)

机构地区：[1]上海药品审评核查中心,上海201203

出　　处：《中国新药与临床杂志》2023年第2期85-90,共6页Chinese Journal of New Drugs and Clinical Remedies

摘　　要：罕见病药物的临床研发需密切结合疾病特征,在严谨科学的基础上采用灵活的设计方法,相关监管机制和审评政策在很大程度上影响罕见病药物研发进程。本文梳理了美国和欧盟关于罕见病药物定义、资格认定程序、研发中采取的监管策略、审评机制和相关激励措施等,结合我国罕见病药物研发现状和审评情况,从罕见病药物的管理和资格认定、审评审批优惠政策和罕见病药物研发策略等方面为我国加快罕见病药物研发和监管策略的实施提供参考思路,以提高我国罕见病防治能力建设。The clinical development of drugs for rare diseases needs to be closely integrated with disease characteristics and adopt a flexible design approach based on science,and the related regulatory mechanisms and review policies have a significant impact on the process of drug development for rare diseases.This paper introduces the definition of rare disease drugs,qualification determination procedures,regulatory strategies,review mechanisms and related incentives adopted by the United States and the European Union in rare disease drug development in recent years,from the qualification determination of rare disease drugs,the review and approval of preferential policies and rare disease drug R&D strategies to provide reference ideas to accelerate the implementation of rare disease drug development and regulatory strategies in China to improve the capacity building of rare disease prevention and treatment,taking into account the current situation of rare disease drug development and review.

关 键 词：罕见病 罕见病药物 药物批准 审评机制 

分 类 号：R951[医药卫生—药学]