罕见病药物的临床审评：挑战与思考

Clinical evaluation of drugs for rare diseases:challenges and considerations

罕见病药物一直是药物研发中的薄弱环节,许多国家对罕见病药物的研发、上市均给予积极鼓励的政策,促进了罕见病药物的发展。近年来,为缓解中国罕见病药物缺乏现状,满足临床迫切需求,国家出台了一系列政策鼓励罕见病药物的研发。目前有大量的罕见病药物在中国申请进行临床试验或提出上市申请,对临床审评带来了新的挑战。本文对罕见病药物审评工作中遇到的问题进行梳理,并参考美国罕见病药物研发的技术标准对相关问题进行探讨,以期为中国罕见病药物研发、评价提供新的思路。

Drugs for rare diseases have always been the weakpoints in drug research.The governments of many countries have actively encouraged with the policy support the development of drugs for rare diseases,which has greatly promoted the development of related drugs.To solve the drug shortage for rare diseases and meet the urgent clinical demands in China,Chinese government has issued a series of policies to encourage the research of drugs for rare diseases and there are a large number of drugs being applied for clinical trials or new drug approvals for rare diseases now in China.This has brought new challenges to the clinical evaluation.This paper summarizes the problems encountered in the review of drugs for rare diseases,and discusses these issues referring to the related American technical standards in order to provide referential ideas for the research and evaluation of the drugs for rare diseases in China.