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Exploring Bayesian Approaches Applied to New Treatments for Rare Diseases
Session Chair(s)
Freda Cooner, PHD
Statistician, FDA, United States
Drug trials for rare diseases often have small sample sizes, making it difficult to show statistically significant treatment differences. Bayesian approaches based on the “borrowing of strength” from other studies may potentially inform regulatory decisions. This session will discuss the potential of applying a Bayesian framework in rare disease drug development and assess anticipated difficulties in doing so. Some case studies will be used to illustrate the general concept.
Learning Objective : Describe the basics of Bayesian methodology; Discuss the potential of applying Bayesian framework to the orphan drug clinical development program for certain types of rare diseases.
Speaker(s)
John Troiani, MD, PHD
Acting Lead Medical Advisor, Office of Compliance-Immediate Office, CDER, FDA, United States
Bayesian Methods in Related Diseases
Scott Berry, PHD
President and Senior Statistical Scientist, Berry Consultants LLC, United States
Bayesian Design and Modeling for Rare Diseases
Ram Tiwari, PHD, MS
Head of Statistical Methodology, Bristol Myres Squibb , United States
Panelist
Karen Lynn Price, PHD
Senior Research Fellow, Statistical Innovation Center/Design Hub, Eli Lilly and Company, United States
Panelist
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