我国2020-2024年突破性治疗药物程序使用情况分析

Analysis of the use of breakthrough therapy program in China between 2020-2024

关键词:  突破性治疗程序  药品加速审评程序  药物研发

Key words:  breakthrough therapy  expedited review program  drug R&D

摘要:

目的 通过对2020-2024年纳入突破性治疗药物程序的品种进行特征分析,了解突破性治疗药物程序的使用情况,为完善我国突破性治疗药物审评工作程序提供参考。方法 检索国家药品审评中心发布的2020年1月至2024年7月纳入突破性治疗药物品种名单,进一步通过药物临床试验登记与信息公示平台查找品种临床试验信息,分析试验特征。结果 在296个纳入突破性治疗药物程序的品种中,39.4%的品种使用了非随机化试验,35.8%的品种使用单臂设计,69.1%的品种采取开放试验,60.6%的品种使用替代终点。然而,47.5%的品种临床试验处于Ⅲ期,35.1%的品种在临床试验开始后才获得突破性治疗药物资格。结论 我国突破性治疗药物程序为具有显著临床价值的药物提供了灵活的临床试验设计,有助于加速药品研发上市；但仍然存在程序在研发中介入较晚、申请人申报程序不及时等问题。

Abstract:

Objective: To gain insight into the utilisation of the breakthrough therapy program established by the Chinese drug agency, by examining the attributes of the drugs included in the program from 2020 to 2024. This will serve as a foundation for the enhancement of the breakthrough therapy program in China. Methods: The list of drugs included in the breakthrough therapy program from Jan 2020 to Jul 2024, released by the National Drug Evaluation Centre, was retrieved; and the information of the drugs" clinical trials was further found. The characteristics of the trials were then analysed through the platform of drug clinical trial registration and information publicity. Results: Of the 296 drugs included in the breakthrough therapy program, 39.4% employed non-randomised trials, 35.8% utilised single-arm designs, 69.1% conducted open-label trials, and 60.6% employed surrogate endpoints. Nevertheless, 47.5% of the drugs were in phase III clinical trials, and 35.1% were designated as breakthrough therapy only after the commencement of clinical trials. Conclusion: the Chinese breakthrough therapy program offers flexibility in clinical trial design for drugs with significant clinical value, thereby facilitating the acceleration of the development and launch of medicines. However, there are still concerns that require attention, including the program"s delayed involvement in research and development and the applicant"s untimely requests for this program.