Online Program

Study Design and Statistical Considerations in Rare Disease Clinical Trials: Reviewers’ Perspective (302486)

Wonyul Lee, US Food and Drug Administration 
*Yan Wang, US Food and Drug Administration 

Keywords: adaptive design, Bayesian borrowing, global tests, multiple-domain responder index

Major challenges in study design and statistical considerations in rare disease clinical trials include small patient populations, limited data of natural history, and heterogeneous clinical manifestations. This talk will present some strategies to address these challenges, including adaptive design for treatment duration and efficacy endpoints, global tests for multiple endpoints, multiple-domain responder index and Bayesian borrowing methods.

Keywords: adaptive design, Bayesian borrowing, global tests, multiple-domain responder index