There has been increasing interest in the use of innovative statistical methods and clinical trial designs, including Bayesian methods and designs, in the regulatory evaluation of rare disease products. Several recent regulatory workshops and white papers have discussed opportunities to use Bayesian approaches to make sometimes desperately needed safe and effective treatments available to patients with rare diseases faster. Congress, under the 21st Century Cures Act and the sixth Prescription Drug User Fee Act, has also asked FDA to focus on this area and hold public workshops and develop related guidances for industry.
In this talk, I will review the landscape of U.S. regulation of rare disease products and discuss how Bayesian methods have fit into this area in the past and how they might spur product development even further in the future. I will review specific Bayesian trial designs and approaches, discuss potential pitfalls and areas where further methodological or practical development is needed, and present rare disease case studies from the FDA's Center for Biologics Evaluation and Research.