Speaker
Wei Chen
(Peking University Health Science Center, Beijing)
Description
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 patients. Completion time reduced 14 months.
Conclusion: Bayesian adaptive designs improve efficiency for rare pediatric disorder trials.
Author
Wei Chen
(Peking University Health Science Center, Beijing)