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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
Yeh-Fong Chen, PHD
Food and Drug Administration, United States
Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER
Industry Perspective
Robert P. Homolka, MBA, MS
Shire, United States
Clinical Pharmacology Group Lead
Panelist
Gil Price, MD
Propharma Group, United States
Chief Medical Officer