我国基于临床缓解率替代终点上市抗癌新药的现状及特征研究  

Current Status and Characteristics of Novel Cancer Drugs Approved in China with Clinical Response Rate as a Surrogate Endpoint

作　　者：朱婷 钟锦佳 吴昊 黄亚芳 ZHU Ting;ZHONG Jinjia;WU Hao;HUANG Yafang(School of General Practice and Continuing Education,Capital Medical University,Beijing 100069,China)

机构地区：[1]首都医科大学全科医学与继续教育学院,北京市100069

出　　处：《中国全科医学》2025年第20期2523-2529,共7页Chinese General Practice

基　　金：国家自然科学基金资助项目(82104133)。

摘　　要：背景癌症为我国居民的主要死因,占疾病死亡人数的23.1%。为满足严重威胁生命的疾病或亟需患者要求,国家药品监督管理局(NMPA)允许使用临床缓解率作为替代终点支持新药附条件批准。目的分析我国基于临床缓解率替代终点上市抗癌新药的现状及特征。方法系统采集NMPA 2017—2023年批准的抗癌新药,以基于临床缓解率替代终点上市抗癌新药及适应证为研究对象。分析其首次获批类型(常规批准、附条件批准)、后续转为常规批准所用疗效终点类型、临床治疗线、用于初始药物批准和上市后确认的试验设计、药物机制分类、癌症类型等。并比较不同NMPA批准类型间抗癌新药临床缓解率的差异。结果2017—2023年,针对晚期或转移性癌症,NMPA基于临床缓解率替代终点批准了68种抗癌新药的95个适应证,其中21个(22.1%)为常规批准,74个(77.9%)为附条件批准。74个附条件批准中,13个(17.6%)转为常规批准。95个适应证临床缓解率中位数为59.0%(35.8%,75.8%),其中47个(49.5%)适应证的临床缓解率≥60%,16个(16.8%)适应证的临床缓解率<30%[15个(93.8%)为二线或更高线治疗]。附条件批准抗癌新药与常规批准抗癌新药临床缓解率比较,差异无统计学意义(P=0.076)。结论我国抗癌新药基于临床缓解率通过的附条件批准上市的标准严格,其与常规批准无明显差异。绝大多数的抗癌新药临床缓解率高于国际建议标准。Background Cancer is a leading cause of death among residents in China,accounting for 23.1%of all disease-related deaths.To address the urgent needs of patients with severely life-threatening diseases,the National Medical Products Administration(NMPA)allows the use of clinical response rates(RRs)as a surrogate endpoint to support conditional approval of new drugs.Objective To analyze the current situation and characteristics of new anticancer drugs approved in China based on clinical RRs as a surrogate endpoint.Methods This study systematically collected data on anticancer drugs approved by the NMPA from 2017 to 2023,focusing on anticancer drugs and their indications that were approved based on RRs as surrogate endpoint.The study analyzed the initial approval types of these drugs(including regular approval and conditional approval),the types of efficacy endpoints used for subsequent conversion to regular approval,clinical treatment lines,trial designs for initial drug approval and post-marketing confirmation,drug mechanism classifications,and cancer types.Additionally,the study compared the differences in RRs among anticancer drugs with different NMPA approval types.Results From 2017 to 2023,NMPA approved 95 indications for 68 new anticancer drugs based on RRs as surrogate endpoint for advanced or metastatic cancers. Among these,21(22.1%) were granted regular approval,while 74(77.9%) were conditionally approved. Of the 74 conditionally approved indications,13(17.6%) were subsequently converted to regular approval. The median RRs for all 95 indications was 59.0%(35.8%,75.8%). Specifically,47(49.5%) indications had RRs higher than or equal to 60%,while 16(16.8%) had RRs lower than 30% [with 15(93.8%) of these being for second-line or higher treatment]. There was no statistically significant difference in RRs between conditionally approved and regularly approved anticancer drugs(P=0.076). Conclusion The standards for conditional approval of novel anticancer drugs in China based on RRs are strict and show no signific

关 键 词：抗癌药 适应证 临床缓解率 完全缓解率 附条件批准 

分 类 号：R979.1[医药卫生—药品]