All Times EDT
Keywords: innovative; non-inferiority; external data; Bayesian
In this talk, I will discuss a submission to the FDA Complex Innovative Trial Design (CID) Pilot Meeting Program for a randomized, double-blind, active-controlled, group sequential, non-inferiority trial to evaluate the safety and effectiveness of a drug to treat pediatric multiple sclerosis (MS) patients. Notable aspects of the proposed design included a primary analysis based on a non-inferiority comparison against an active control, information borrowing from historical data in both the adult and pediatric MS populations, and a Bayesian inferential framework. I will provide an overview of the proposed trial, describe the rationale for an active-controlled, non-inferiority design in this setting, and highlight important iterations of the design, including the selection of an appropriate non-inferiority margin, the choice and role of historical data for information borrowing, and the Bayesian approach.