Platform Trial Designs for Sequential Treatment Evaluation

April 17, 2024 11:30am - April 17, 2024 12pm

Speaker: Alex Kaizer (University of Colorado Anschutz Medical Campus)


The potential strengths of platform trials have been elucidated for oncology, infectious diseases, and other settings. In this talk we first present the lessons learned from a sequential platform trial used in the West Africa Ebola virus disease outbreak and how these may translate to the context of oncology research. For example, one shortcoming of the original design was that supplemental information from controls in previous trial segments was not utilized. We address this limitation by proposing an adaptive design methodology that facilitates information sharing within the trial. The design also allows the use of multisource adaptive randomization to target information balance within a trial segment if other segments of data are incorporated. Compared to the standard design, we demonstrate that MEMs with adaptive randomization can improve power with limited type-I error inflation. We conclude by discussing some of the future directions and potential applications of newer methodologies and applications for platform trial designs that both include and extend the lessons learned previously.