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Innovative and Efficient Trial Designs and Statistical Approaches in Small Patient Populations: Rare Diseases and Pediatrics
Session Chair(s)
Patrick Frey
Senior Director, Regulatory Policy, Neurocrine Biosciences, United States
Progress has been made to advance drug development in rare disease and pediatric populations, but challenges remain. Stakeholders will convene to discuss barriers to studying small patient populations and innovative approaches to overcome them. Join the DIA Patient Engagement Community for a follow up round table discussion (session #330.1 RT L) on Wednesday, June 30, 1:00PM - 2:00PM EDT.
Learning Objective : Discuss current experiences in the use and acceptance of novel trial designs; Discuss barriers and approaches to address barriers to drug development in small affected patient populations (eg, Bayesian strategies to increase statistical efficiency), with a focus on rare disease and pediatric patient populations; Identify opportunities to further advance the use of novel trial designs.
Speaker(s)
Kathleen M. Donohue, MD, MSC
Director of the Division of Rare Diseases & Medical Genetics, OND, CDER , FDA, United States
Panelist
Leman Yel, MD
Vice President, Head of Clinical Medicine, Plasma Derived Therapies Business Uni, Takeda Pharmaceutical Company Limited, United States
Panelist
Yan Wang, PHD
Statistical Team Leader, Division of Biometrics IV, Office of Biostatistics, OTS, FDA, United States
Panelist
Edward Neilan, MD, PHD
Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD), United States
Panelist
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