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
urrent Status and Characteristics of Novel Cancer Drugs Approved in China with Clinical Response Rate as a Surrogate Endpoint
背景 癌症为我国主要死因，占疾病死亡23.1%。为满足严重威胁生命的疾病或亟须患者，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。并分析比较不同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高于60%，16个（16.8%）适应证的临床缓解率低于30%［有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。
BackgroundCancer is a leading cause of death in Chinaaccounting for 23.1% of disease-related deaths. To address the urgent needs of patients with severely life-threatening diseasesthe National Medical Products AdministrationNMPA allows the use of clinical response ratesRRs as a surrogate endpoint to support conditional approval of new drugs. Objective To analysis of the current situation and characteristics of new anticancer drugs approved in China based on clinical RRs as a surrogate endpoint. MethodsThis study systematically collected data on anticancer drugs approved by the NMPA from 2017 to 2023focusing on anticancer drugs and their indications that were approved based on RRs as surrogate endpoint. The study analyzed the initial approval types of these drugsincluding regular approval and conditional approval the types of efficacy endpoints used for subsequent conversion to regular approvalclinical treatment linestrial designs for initial drug approval and post-marketing confirmationdrug mechanism classificationsand cancer types. Additionallythe study compared the differences in RRs among anticancer drugs with different NMPA approval types. ResultsFrom 2017 to 2023NMPA approved 95 indications for 68 new anticancer drugs based on RRs as surrogate endpoint for advanced or metastaticcancers. Among these2122.1% were granted regular approvalwhile 7477.9% were conditionally approved. Of the 74 conditionally approved indications1317.6% were subsequently converted to regular approval. The median RRs for all 95 indications was 59.0%35.8%75.8%. Specifically4749.5% indications had RRs higher than 60%while 1616.8% had RRs lower than 30% with 1593.8% of these being for second-line or higher treatment. There was no statistically significant difference in RRs between conditionally approved and regularly approved anticancer drugsP=0.076. ConclusionThe standards for conditional approval of novel anticancer drugs in China based on RRs are strict and show no significant difference compared to regular approval. The RRs of the majority of novel anticancer drugs exceed internationally recommended standards.
10.12114/j.issn.1007-9572.2024.0475
医药卫生
抗癌药适应证临床缓解率完全缓解率附条件批准
.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[EB/OL].(2025-01-22)[2025-02-05].https://chinaxiv.org/abs/202501.00213.点此复制
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