国内外罕见病药物临床试验概况分析与思考

Analysis ofclinical trials of rare disease drugsin China and abroad

目的分析比较国内外罕见病药物临床试验的现状,为进一步推动我国罕见病药物临床试验发展提供参考。方法通过提取我国“药物临床试验登记与信息公示平台”中罕见病药物临床试验数据,从项目注册情况、试验类型和分期、涉及的疾病、方案设计等多个层面分析我国罕见病药物临床试验的特点,并与国外罕见病药物临床试验的注册数据进行比较。结果截至2022年3月31日,我国“药物临床试验登记与信息公示平台”共登记罕见病药物临床试验337项,涉及37个病种、125种试验药物。其中,生物等效性试验127项,占37.6%;国内项目278项,占82.4%;含儿童受试者的项目103项,占比约30%。与国外相比,我国罕见病药物临床试验具有如下特点:(1)项目数量呈明显增长趋势;(2)创新药物临床试验少,仿制药生物等效性试验多;(3)有儿童受试者参与的试验项目比例较高。结论我国罕见病创新药临床研究依然是薄弱环节,有待在完善立法、加大财政支持力度、人才培养、创新研究路径和方法等方面持续发力。

Objective To analyze the status quo of clinical trials of rare disease drugs in China and abroad,to provide reference for further promoting the development of clinical research of rare disease drugs in China.Methods By extracting the data of clinical trials of rare disease drugs in China's"drug clinical trial registration and information publicity platform",weanalyzedthe characteristics of clinical trials of rare disease drugs in China from the aspects of project registration,test type and stage,involved diseases,and scheme design,and comparedthem with the registration data of foreign clinical trials of rare disease drugs.Results As of March 31,2022,there were a total of 337 clinical trials of rare disease drugs in China's"drug clinical trial registration and information publicity platform",involving 37 diseases and 125 experimental drugs.Among them,there were 127 bioequivalence tests,accountingfor 37.6%;278 domestic projects,accountingfor 82.4%;and 103 projects involving children,accountingfor about 37.0%.Compared with those in other countries,clinical trials of rare disease drugs in China had the following characteristics:(1)the number of clinical trialsshowedan obviously increasing trend;(2)there were few clinical trials of innovative drugs but many bioequivalence tests of generic drugs;and(3)the proportion of trials involving children was high.Conclusion The clinical research on innovative drugs for rare diseases is still a weak link in China,and we need to improve legislation,increase financial support,enhance personnel training,and improve innovative research paths and methods.