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The Rare Disease Clinical Outcome Assessment Consortium: Collaboration Aimed at Accelerating Rare Disease Drug Development
Session Chair(s)
Lindsey Murray, PhD, MPH
Executive Director, Rare Disease COA Consortium, Critical Path Institute, United States
A key aim of the Rare Disease Clinical Outcome Assessment (COA) Consortium is the creation of a resource of publicly available COAs suitable as trial endpoint measures. Methodological challenges in rare disease trial settings will also be addressed.
Learning Objective : Describe the unmet need that the Rare Disease Clinical Outcome Assessment Consortium seeks to address; Describe the Rare Disease COA Resource, including the purpose, process for development, domains included, and progress to-date; Describe a literature review to identify approaches to evaluate clinical benefit in populations with heterogeneity; Describe COVID-19 mitigation strategies in pediatric rare disease clinical trials.
Speaker(s)
Tori Brooks, MPH
Research Associate II, Mapi Research Trust, United States
Academic Update
Kiera Berggren, MA, MS
Research Speech-Language Pathologist, Department of Neurology, Virginia Commonwealth University, United States
Academic Update
Timothy Andrew Howell, MA
Research Associate III, Evidera, United States
Industry Update
Naomi Knoble, PhD
Reviewer, Division of Clinical Outcome Assessment, OND, CDER, FDA, United States
FDA Update
Allison Seebald
Senior Research Program Manager, National Organization for Rare Disorders (NORD), United States
Patient Update
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