Bayesian and Adaptive Trial Designs for Ultra-Rare Populations

Bayesian and adaptive approaches offer alternative ways to design and analyze studies when patient populations are extremely small. This session will frame how these methods differ from traditional trial designs and why they are increasingly considered in ultra-rare disease research, including their potential to support learning over time, reduce patient burden, and make more efficient use of limited data.

Moderator: Edward Neilan, MD, PhD, Chief Medical and Scientific Officer, NORD 

Panelists:

• Yuan Ji, PhD, Professor of Biostatistics, University of Chicago
• Kelley Kidwell, PhD, Professor of Biostatistics, Interim Associate Dean of Faculty Affairs, University of Michigan School of Public Health, a NORD Rare Disease Center of Excellence 
• Olivia Morgan, PhD, Statistician, FDA

View PDF Presentation from this Session
View Slideshow Presentation from this Session