Advantages and Challenges of Adaptive CNS Trials: Real World Lessons

Co-Chairs: Ron Marcus, MD; Judith Kando, PharmD

The Adaptive Design Working Group (ADWG) hosted a scientific session during the 2015 annual meeting that focused primarily on dose response adaptation in Phase 2 clinical studies.  The current Adaptive Design Scientific session will start off with an update on the expansion of adaptive design methodology in neuroscience clinical trials and then focus on different topics. Session will begin with a presentation of a comparative effectiveness study in status epilepticus followed by a discussion of an adaptation of dose and/or endpoint in a range of age-related disease characteristics in a Phase 2 proof-of-concept rare disease pediatric epilepsy study. An adaptation of the primary endpoint in a Phase 2 cognitive impairment associated with schizophrenia study will be presented, and lastly, attendees will hear an exploration of the multiple layers of considerations, that one may encounter, in the development of a confirmatory, adaptive trial having multiple endpoints and multiple adaptive features (e.g., sample size adaptation and treatment allocation adaptation).  An FDA statistician will comment on the presentations as well as participate in the panel discussion. During the discussion, panelists will provide answers to questions that emerge during the session as well as answer questions raised during the 2016 webinar series.