我国药品附条件批准程序实施进展:2020—2024年汇总分析

Progress in the implementation of conditional approval process for drugs in China: a pooled analysis from 2020 to 2024

摘要

目的: 对2020—2024年我国附条件批准药物进行系统分析,为完善我国药品附条件批准程序提供建议和参考。方法: 通过国家药品监督管理局公开数据库、国家药品监督管理局药品审评中心公开数据库和药智数据库确定研究期间附条件批准的所有药品,收集并分析其基本信息、上市前临床试验研究和上市后研究要求与进展。结果: 共有143个获批事项经由附条件批准程序上市,其特点包括:研发资源高度集中于抗肿瘤药物、替代终点在上市前关键临床试验中的应用占比较高、附条件批准药品转常规批准的转换率较低。结论: 需加强药品监管等相关部门的导向作用,建立替代终点认定制度,提高附条件批准程序的灵活性。

Abstract

Objective: To conduct a comprehensive analysis of drugs marketed through the conditional approval process in China from 2020 to 2024, along with recommendations for improving this process. Methods: Drugs conditionally approved during the study period were identified through the National Medical Products Administration public data, the Center for Drug Evaluation public data, and the Yaozhi Database. The basic information, pre-market clinical trial studies, and post-marketing study requirements and progress were collected and analyzed. Results: A total of 143 approved matters were marketed through the conditional approval process. Key characteristics included: R&D resources were highly concentrated on antineoplastic drugs, a high percentage of applications in pre-market pivotal clinical trials were based on surrogate endpoints, and the conversion rate of drugs from conditional approval to full approval was low. Conclusion: It is necessary to strengthen the guidance provided by drug regulatory and relevant departments, establish a system for recognizing surrogate endpoints, and improve the flexibility of the conditional approval process.

关键词

附条件批准 / 获批药品 / 基本信息 / 临床试验 / 上市后研究

Key words

conditional approval / approved drug / basic information / clinical trials / post-marketing study

[1] 国家药品监督管理局药品审评中心. 药品附条件批准上市技术指导原则(试行)[EB/OL].(2020-11-19)[2025-05-15].https://www.cde.org.cn/zdyz/opinioninfopage?zdyzIdCODE=3ec9de4b4bfab46c8fc1cbc0211179bf&rddt=1.
[2] 国家市场监督管理总局. 中华人民共和国药品管理法[EB/OL].(2019-08-26)[2025-05-15].https://www.samr.gov.cn/zw/zfxxgk/fdzdgknr/fgs/art/2023/art_b73ec9dabb2d468281 d6c994b4bcdaf8.html.
[3] 国家市场监督管理总局. 药品注册管理办法[EB/OL].(2020-01-22)[2025-05-15].https://www.samr.gov.cn/zw/zfxxgk/fdzdgknr/fgs/art/2023/art_3275cb2a929d4c34ac8c0421b2a9c257.html.
[4] 赵一飞, 耿欣, 徐立华, 等. 对我国附条件批准上市化药药学技术要求的思考[J]. 中国药科大学学报, 2021, 52(5): 636-642.
[5] 国家药品监督管理局. 国家药监局综合司公开征求《药品附条件批准上市申请审评审批工作程序(试行)(修订稿征求意见稿)》意见[EB/OL].(2023-08-25)[2025-05-20].https://www.nmpa.gov.cn/directory/web/nmpa/xxgk/zhqyj/zhqyjyp/20230825104212129.html.
[6] KATO S, ONO S. Accelerated approval for cancer drugs in the United States and the clinical evidence required for conversion to regular approval[J]. J Pharm Innov, 2024, 19(5): 54.
[7] BANZI RT, GERARDI C, BERTELE' V, et al. Approvals of drugs with uncertain benefit-risk profiles in Europe[J]. Eur J Intern Med, 2015, 26(8): 572-584.
[8] 国家药品监督管理局. 2020年度药品审评报告[EB/OL].(2021-06-21)[2025-05-15].https://www.nmpa.gov.cn/xxgk/fgwj/gzwj/gzwjyp/20210621142436183.html.
[9] 国家药品监督管理局. 2021年度药品审评报告[EB/OL].(2022-06-01)[2025-05-15].https://www.nmpa.gov.cn/xxgk/fgwj/gzwj/gzwjyp/20220601110541120.html.
[10] 国家药品监督管理局. 2022年度药品审评报告[EB/OL].(2023-09-06)[2025-05-15].https://www.nmpa.gov.cn/xxgk/fgwj/gzwj/gzwjyp/20230906163722146.html.
[11] 国家药品监督管理局. 2023年度药品审评报告[EB/OL].(2024-02-04)[2025-05-15].https://www.nmpa.gov.cn/xxgk/fgwj/gzwj/gzwjyp/20240204154334141.html.
[12] 国家药品监督管理局药品审评中心. 2024年度药品审评报告[EB/OL].(2025-03-18)[2025-05-15].https://www.cde.org.cn/main/news/viewInfoCommon/54538c67b7e764 fc51666567fc620241.
[13] CHEN S, CHEN X, LIU MS, et al. Regulation of generic drugs in China: progress and effect of the reform of the review and approval system[J]. J Pharm Innov, 2023, 18(2): 340-348.
[14] LUO XX, GUO QX, DU X, et al. Evaluation of clinical trial designs for novel anticancer drugs in China: a cohort study of drugs approved between 2015 and 2021[J]. Drug Discov Today, 2023, 28(6): 103578.
[15] SU L, LIU S, LI GQ, et al. Trends and characteristics of new drug approvals in China, 2011-2021[J]. Ther Innov Regul Sci, 2023, 57(2): 343-351.
[16] LI JL, WANG HY, HUA YZ, et al. Progress and challenges of the new conditional approval process in China: a pooled analysis from 2018 to 2021[J]. Clin Ther, 2023, 45(11): 1111-1118.
[17] 张晓方, 白雪, 孙婉, 等. 2015—2020年美国加速审批与欧洲附条件批准的上市前有效性科学证据特点[J]. 中国新药杂志, 2024, 33(1): 1-9.
[18] 张晓方, 白雪, 孙婉, 等. 2015—2020年美国加速审批与欧洲附条件批准的上市所附条件特点[J]. 中国新药杂志, 2024, 33(8): 737-744.
[19] 朱妍昕, 徐维, 王霞, 等. 基于FAIR原则的循证医学文献本体构建: 以哮喘药物治疗文献本体为例[J]. 情报理论与实践, 2022, 45(1): 187-195.
[20] 中华人民共和国财政部. 关于抗癌药品增值税政策的通知[EB/OL].(2018-04-27)[2025-05-15].https://szs.mof.gov.cn/zhengcefabu/201804/t20180427_2880407.htm.
[21] LUO XX, QIAN F, YANG LN, et al. Assessment of the breakthrough-therapy-designated drugs granted in China: a pooled analysis 2020-2022[J]. Drug Discov Today, 2022, 27(12): 103370.
[22] ACHEAMPONG A, LI LM, ELSHERBINY SM, et al. A crosswalk on the genetic and conventional strategies for enhancing astaxanthin production in Haematococcus pluvialis[J]. Crit Rev Biotechnol, 2024, 44(6): 1018-1039.
[23] 中国抗癌协会. 《中国恶性肿瘤学科发展报告(2023)》——淋巴瘤研究进展篇[EB/OL].(2024-04-30)[2025-05-15].http://www.caca.org.cn/system/2024/04/30/030090651.shtml.
[24] RESSEGUIE EA, PALMER H. Inclusion of histopathology in dose range-finding nonclinical studies for inhaled drug products[J]. Int J Toxicol, 2024, 43(6): 540-548.
[25] ZHAO MM, NIU XW, BAI L, et al. Application of myocardial salvage index as a clinical endpoint: assessment methods and future prospects[J]. J Magn Reson Imaging, 2025, 61(5): 2033-2050.
[26] 朱思睿, 周晓冰, 黄芝瑛, 等. 生物标志物验证与认证现状和科学监管思考[J]. 中国新药杂志, 2020, 29(22): 2570-2575.
[27] 杨莉, 韦彦伊, 吴迪, 等. 美国和欧盟基于替代终点的新药上市审批程序研究及启示[J]. 中国新药杂志, 2022, 31(2): 132-141.
[28] HOEKMAN J, KLAMER TT, MANTEL-TEEUWISSE AK, et al. Characteristics and follow-up of postmarketing studies of conditionally authorized medicines in the EU[J]. Br J Clin Pharmacol, 2016, 82(1): 213-226.
[29] FLEMING TR. Surrogate endpoints and FDA's accelerated approval process[J]. Health Aff (Millwood), 2005, 24(1): 67-78.
[30] 张烁, 金思羽, 杨莉. 美国抗肿瘤药加速批准程序运行效果分析及对我国的启示[J]. 中国新药杂志, 2024, 33(6): 521-530.