Group-Sequential Designs With an Externally-Driven Change of Primary Endpoint.

ABSTRACT: Clinical trials conducted during the COVID-19 pandemic demonstrated the value of adaptive design methods in emerging disease settings, when there can be considerable uncertainty around disease natural history, anticipated endpoint effect sizes and population size. In such settings, there may also be uncertainty regarding the most appropriate primary endpoint. This might lead to an externally-driven decision to change the primary endpoint during the course of an adaptive trial. If information on the new primary endpoint is already being collected, initially as a secondary endpoint, the trial could continue with a new primary endpoint. In this case it is unclear how statistical inference on the final primary endpoint should be adjusted for interim analyses monitoring the initial primary endp

SUBMITTER: Yarahmadi A 

PROVIDER: S-EPMC12694703 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature