Cancer Drugs Approved Based on Surrogate Endpoint: A Retrospective Observational Study in the United States and China

IF 2.9 2区医学 Q2 ONCOLOGY

Cancer Medicine Pub Date : 2025-04-15 DOI:10.1002/cam4.70864

Ting Zhu, Jinjia Zhong, Yafang Huang

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Abstract

Background

Hundreds of cancer drugs are approved globally based on surrogate endpoint response rate (RR). However, the characteristics of RR-based approvals remain unknown.

Methods

In this retrospective study, all cancer drug-indication pairs approved based on RR in the United States and China up to December 2023 were analyzed.

Results

A total of 249 RR-supported drug-indication pairs were identified in the United States and 98 in China. In the United States, 98 of the 249 (39.4%) indications were granted regular approval (RA), whereas in China, only 21 of 98 (21.4%) approvals followed this regulatory pathway, with the remainder receiving accelerated approval (AA). The conversion rate from AA to RA was significantly lower in China compared to the United States (13.3% vs. 28.1%, p < 0.001). The proportion of AA withdrawals was significantly lower in China compared to the United States (1.0% vs. 10.4%, p < 0.001). Among all indications, the median RR in China was 60.9% (IQR, 35.8%–75.0%), which was significantly higher than the 45.0% (IQR, 29.0%–61.0%) in the United States (p < 0.001). In China, 18 of the 98 (18.4%) had an RR less than 30%. In contrast, in the United States, 26.9% of the 249 had an RR less than 30%.

Conclusions

Compared to the United States, RR-supported approvals in China are characterized by higher RR values and a stricter RA pathway. Regulatory authorities in both countries may need to consider both the quantity and quality during cancer drug development based on surrogate endpoints.

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基于替代终点批准的抗癌药物：美国和中国的回顾性观察研究

基于替代终点反应率（RR），全球有数百种癌症药物获得批准。然而，基于rr的审批的特点仍然未知。方法回顾性分析截至2023年12月美国和中国基于RR批准的所有癌症药物适应症对。结果美国共鉴定出249个rr支持的药物指征对，中国鉴定出98个。在美国，249个适应症中有98个（39.4%）获得了常规批准（RA），而在中国，98个适应症中只有21个（21.4%）获得了常规批准（RA），其余的都获得了加速批准（AA）。从AA到RA的转化率在中国明显低于美国（13.3%比28.1%,p < 0.001）。与美国相比，中国的AA撤资比例明显较低（1.0%对10.4%,p < 0.001）。在所有适应症中，中国的中位RR为60.9% (IQR, 35.8% ~ 75.0%)，显著高于美国的45.0% (IQR, 29.0% ~ 61.0%) （p < 0.001）。在中国，98家银行中有18家（18.4%）的资本金比率低于30%。相比之下，在美国，249人中有26.9%的人的风险比低于30%。与美国相比，中国的RR支持审批具有更高的RR值和更严格的RA途径的特点。在基于替代终点的癌症药物开发过程中，两国的监管机构可能需要同时考虑数量和质量。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Cancer Medicine ONCOLOGY-

CiteScore

5.50

自引率

2.50%

发文量

907

审稿时长

19 weeks

期刊介绍： Cancer Medicine is a peer-reviewed, open access, interdisciplinary journal providing rapid publication of research from global biomedical researchers across the cancer sciences. The journal will consider submissions from all oncologic specialties, including, but not limited to, the following areas: Clinical Cancer Research Translational research ∙ clinical trials ∙ chemotherapy ∙ radiation therapy ∙ surgical therapy ∙ clinical observations ∙ clinical guidelines ∙ genetic consultation ∙ ethical considerations Cancer Biology: Molecular biology ∙ cellular biology ∙ molecular genetics ∙ genomics ∙ immunology ∙ epigenetics ∙ metabolic studies ∙ proteomics ∙ cytopathology ∙ carcinogenesis ∙ drug discovery and delivery. Cancer Prevention: Behavioral science ∙ psychosocial studies ∙ screening ∙ nutrition ∙ epidemiology and prevention ∙ community outreach. Bioinformatics: Gene expressions profiles ∙ gene regulation networks ∙ genome bioinformatics ∙ pathwayanalysis ∙ prognostic biomarkers. Cancer Medicine publishes original research articles, systematic reviews, meta-analyses, and research methods papers, along with invited editorials and commentaries. Original research papers must report well-conducted research with conclusions supported by the data presented in the paper.