Abstract:
The 13
th five-year period is remarkable regarding the construction of pharmaceutical innovation ecosystems in China and the vigorous advances in clinical research and development that have led to many anticancer drugs being successfully approved in China. The scientific design and rational selection of primary endpoints in confirmatory trials have fundamental impacts on the reliable evaluation of clinical benefits and whether new drugs could be marketed successfully. Such endpoints include the choice between single-endpoint design and multi-endpoint design, overall survival, and alternative endpoints. This study first summarized published regulatory guidelines related to clinical trial endpoints in China and beyond, especially for cancer trials. Then, classification, advantages, and disadvantages of commonly used endpoints of anticancer new drugs were described along with key considerations for the selection of primary endpoints. Based on January 1st, 2018 to December 31st, 2019 clinical trials published on the China Food and Drug Administration Registration and Information Disclosure Platform, characteristics of primary endpoint design and usage of common endpoints of new anticancer drugs in phase Ⅲ trials were analyzed and described. Meanwhile, key suggestions for the design of primary endpoints in confirmatory trials of anticancer drugs were recommended, to provide support and reference for related stakeholders and designers of key trials in new anticancer drugs.