Surrogate Endpoints in Pivotal Clinical Trials for Drug Approval in Japan Compared to the United States.

ABSTRACT: Regulatory guidance documents exist on surrogate endpoints in the United States. In Japan, there are no established rules or guidance regarding the use of surrogate endpoints, and various aspects remain unclear. The aim of this study was to investigate the use of surrogate endpoints in Japan for drugs approved in Japan from 1999 to 2022, referring to the list established by the Food and Drug Administration. Precisely 2307 drugs were approved in Japan during the 24-year survey period. Of these, 1012 drugs (43.9%) were indicated for diseases for which surrogate endpoints were specified in the Surrogate Endpoint Table established by the Food and Drug Administration. After examining the endpoints used in clinical trials for 1012 drugs, 947 (93.6%) were approved based on the same surrogate endp

SUBMITTER: Yamamoto Y 

PROVIDER: S-EPMC12605959 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature