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Tuesday, June 20 • 10:30am - 11:45am
#223: Introduction to Rare Disease Studies: Challenges of Rare Disease From a Statistical, Protocol Development, and Operations Perspective

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Component Type: Session
Level: Intermediate
CE: CME 1.25; IACET 1.25; RN 1.25

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 Objectives

Identify a better understanding of the differences in special population studies and some possible solutions.


William B. Smith, MD


FDA Perspective
Yeh-Fong Chen, PhD

Industry Perspective
Robert P. Homolka, MBA, MS

Gil Price, MD

avatar for William Smith

William Smith

CEO, Alliance for Multispecialty Research/NOCCR
William B. Smith, MD, FACC, is a Professor of Medicine at the University of Tennessee Medical Center in Knoxville, Tennessee. Dr. Smith is board certified in Nephrology, Internal Medicine, Cardiology and Critical Care. Dr. Smith is the President of New Orleans Center for Clinical... Read More →

avatar for Yeh-Fong Chen

Yeh-Fong Chen

Mathematical Statistician (Team Lead), Office of Translational Sciences, CDER, FDA
Dr. Chen is the Team Leader of the Division of Biometrics IX within CDER of FDA, supervising reviewers for the Division of Non-Malignant Hematology Products. She joined FDA in 2000 after receiving her Ph.D. from the University of Iowa. She has had years' experience in reviewing drug... Read More →

Robert Homolka

Clinical Pharmacology Group Lead, Shire
Mr. Robert Homolka is the Director of Clinical Pharmacology Operations at Shire. For the last 20 years, Mr. Homolka has worked at AstraZeneca followed by Shire in Clinical Pharmacology. He has participated in, managed, and/ or overseen more than 200 studies while part of Clinical... Read More →
avatar for Gil Price

Gil Price

Chief Medical Officer, Propharma Group
Over the past 25 years, Dr. Price has worked in various capacities including professional communications, clinical development, and pharmacovigilance in differing therapeutic areas including vaccines, oncology, and infectious disease. He is considered by many to be a KOL in pharm... Read More →

Tuesday June 20, 2017 10:30am - 11:45am CDT
S503ab McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616
  07: SpecialPops, Session