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Tuesday, June 29 • 4:00pm - 5:00pm
#256 SL: Innovative and Efficient Trial Designs and Statistical Approaches in Small Patient Populations: Rare Diseases and Pediatrics

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-21-587-L04-P; CME 1.00; IACET 1.00; RN 1.00

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 Objectives

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.


Patrick Frey


Kathleen M. Donohue, MD, MSc

Leman Yel, MD

Yan Wang, PhD

Edward Neilan, MD, PhD

avatar for Patrick Frey

Patrick Frey

Director, Global Regulatory and R&D Policy, Amgen, United States
Patrick Frey is a Director of Regulatory Policy at Amgen. He leads a small team focused on US regulatory issues within the Global Regulatory and R&D Policy organization. He specific focus areas are accelerated regulatory pathways, decentralized trials, and innovative trial designs... Read More →

Kathleen Donohue

Director of the Division of Rare Diseases & Medical Genetics, OND, CDER, FDA, United States
Dr. Katie Donohue graduated from medical school at Virginia Commonwealth University. She completed her residency in Internal Medicine and fellowships in Allergy & Immunology at Columbia University - New York Presbyterian Hospital. When she finished her clinical training, she joined... Read More →
avatar for Edward Neilan

Edward Neilan

Chief Medical and Scientific Officer, National Organization for Rare Disorders (NORD), United States
Dr. Neilan is a physician-scientist and rare disease expert with experience in both academic medicine and the pharmaceutical industry. His expertise spans laboratory research, medical practice, as well as clinical trial design and execution across all phases of development. After... Read More →

Yan Wang

Statistical Team Leader, Division of Biometrics IV, Office of Biostatistics, OTS, FDA, United States
Dr. Yan Wang has over 10 years research experiences in the general area of pharmaceutical sciences, with special expertise in complex drug delivery systems, such as polymeric microspheres/implants. She is currently the Scientific Lead for long acting drug products in the Office of... Read More →
avatar for Leman Yel

Leman Yel

Vice President, Head of Clinical Medicine, Plasma Derived Therapies Business Uni, Takeda Pharmaceutical Company Limited, United States
Dr. Leman Yel is the VP, Head of Clinical Medicine at Takeda Plasma Derived Therapies Business Unit (PDT BU). She is experienced in early to late phase product development in industry (Baxter, Baxalta, Shire and Takeda) following an accomplished academic medicine career in immunology... Read More →

Tuesday June 29, 2021 4:00pm - 5:00pm EDT
TBD Virtual Event Horsham, PA 19044
  02: ClinTrials -ClinOps, Forum |   11: Statistics, Forum |   09: Regulatory, Forum