Statistical Considerations for Small-Sized Clinical Trials Utilizing a Platform Setting

• Activity Number: 203 - Recent Advances in Bayesian Adaptive Designs, from Early Phase to Confirmatory Trials
• Type: Topic-Contributed
• Date/Time: Tuesday, August 10, 2021 : 1:30 PM to 3:20 PM
• Sponsor: Caucus for Women in Statistics
• Author(s): Feiran Jiao* and Yeh-Fong Chen
• Companies: FDA and FDA
• Keywords: small size clinical trial; historical borrowing; platform setting

Abstract

One major challenge in conducting rare disease and pediatric clinical trials is recruitment. Platform trials may reduce costs and increase trial efficiency. Even in the platform trial setting, borrowing information from external control data sources may be needed for small trials. In this presentation, we will discuss the use of platform trials that take into account external ‘historical’ information, including using natural history data from outside the platform as a control. We will use simulation studies to assess previously proposed approaches to borrow historical information such as test-then-pool, power priors, and hierarchical modeling.