中国罕见病药物研发政策及研发现状简析

Analysis on research and development police and status of drug for rare diseases in China

罕见病发病率低、病情复杂、诊断难度大,导致其治疗药物研发面临诸多困难。为满足临床迫切需求,鼓励罕见病药物研发,近年来国家出台一系列政策及技术指导原则。梳理了2015年至今的罕见病治疗药物研发激励政策,通过查阅文献及公开资料,整理2018—2023年批准上市的用于治疗第一批罕见病目录中罕见病治疗的药物信息,分析中国罕见病药物研发现状。建议定期更新罕见病目录,加强罕见病药物研发者权益保护,鼓励优质罕见病药物仿制,以推动罕见病药物研发产业创新发展、满足罕见病患者的用药需求。

The low incidence,complexity and diagnosis difficulty of rare disease lead to the difficulties in the research and development of drugs for rare diseases.To meet the urgent clinical demands and encourage the research of drugs for rare diseases in China,Chinese government has issued a series of policies and technical guidance.The policies for the research and development of drugs for rare diseases is combed from 2015.By consulting the relevant literature and public information,drug approvals for rare diseases listed in the first catalogue from 2018 to 2023 are presented,then the research and development states of orphan drug is analyzed and summarized.It is suggested that the catalogue of rare diseases should be updated regularly,the rights and interests of drug developers for rare diseases should be protected,the high-quality imitation of drugs for rare diseases should be encouraged,so as to promote the development of the pharmaceutical industry for rare diseases and meet the drugs needs of patients with rare diseases.