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

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%

SUBMITTER: Zhu T 

PROVIDER: S-EPMC11997453 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature