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Exploring Bayesian Approaches Applied to New Treatments for Rare Diseases

Track:
Statistical Science and Quantitative Thinking

Day & Time:
June 18, 1:30PM - 3:00PM (Pacific Standard Time)

Session Number:
372

Room Number:
7A

Level:
Intermediate

Type:
Session

Title:
Exploring Bayesian Approaches Applied to New Treatments for Rare Diseases

Chair(s):
Freda W. Cooner, PhD
Lead Mathematical Statistician, Div. of Biometrics III, OB, OTS, CDER
FDA, United States

Description:
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(s):
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.

Presentation(s) & Speaker(s):
Bayesian Methods in Related Diseases
John Troiani, MD, PhD
Acting Lead Medical Advisor, Office of Compliance-Immediate Office, CDER
FDA, United States

Bayesian Design and Modeling for Rare Diseases
Scott M Berry, PhD
President and Senior Statistical Scientist
Berry Consultants LLC, United States

Panelist
Ram Tiwari, PhD
Associate Director, Office of Biostatistics, OTS, CDER
FDA, United States

Panelist
Karen Lynn Price, PhD, MA
Research Advisor
Eli Lilly and Company, United States