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Introduction to Rare Disease Studies: Challenges of Rare Disease From a Statistical, Protocol Development, and Operations Perspective
Session Chair(s)
William B. Smith, MD
CEO, Alliance for Multispecialty Research, LLC , United States
This session is planned to provide a basic overview of studies in rare diseases and how they differ from most other therapeutic areas. Brief overview of the challenges will be presented with special emphasis on statistical challenges, protocol design realities, operational changes required and recruiting perspectives.
Learning Objective : Identify a better understanding of the differences in special population studies and some possible solutions.
Speaker(s)
FDA Perspective
Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, Food and Drug Administration, United States
Industry Perspective
Clinical Pharmacology Group Lead, Shire, United States
Panelist
Chief Medical Officer, Propharma Group, United States
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