DEMO CON 2026 - NeuCitePress Biomedical Research Conference

Background: Conventional RCTs infeasible for rare pediatric neurological disorders.

Objective: Develop Bayesian adaptive framework for Dravet syndrome gene therapy trials.

Methods: Response-adaptive randomization, Bayesian hierarchical models, external control borrowing. 10,000 simulations per scenario.

Results: Sample size reduced 38%, type I error controlled at 2.5%. Power 87% with 60...