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The Rare Disease Clinical Outcome Assessment Consortium: Aiming to Fulfill Unmet Drug Development Endpoint Measurement Needs
Session Chair(s)
Lindsey Murray, PhD, MPH
Executive Director, Rare Disease COA Consortium
Critical Path Institute, United States
Appropriate endpoint selection for rare disease trials is challenging. The Rare Disease Clinical Outcome Assessment (COA) Consortium’s aim is the creation of a resource of publicly available COAs deemed fit-for-purpose as trial endpoint measures.
Learning Objective : Explain a definition of “rare disease” and describe the global burden of rare diseases and the challenges to drug development; Describe the unmet need that the Rare Disease Clinical Outcome Assessment Consortium is intended to address. •Discuss why a pre-competitive, multi-stakeholder collaboration is the optimal setting for achieving its aim of creating a database of publicly available COAs.
Speaker(s)
Panelist
Michelle Campbell, PhD
FDA, United States
Associate Director, Stakeholder Engagement and Clinical Outcomes, ON, OND, CDER
Panelist
Stephen Joel Coons, PhD
Critical Path Institute, United States
Senior Advisor
Panelist
Vanessa Boulanger, MSc
National Organization for Rare Disorders (NORD), United States
Director of Research Programs
Panelist
Dylan Trundell, MSc
Roche Products Ltd., United Kingdom
Senior Principal PCOR Scientist
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