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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)
Academic Update
Tori Brooks, MPH
Mapi Research Trust, United States
Research Associate II
Academic Update
Kiera Berggren, MA, MS
Virginia Commonwealth University, United States
Research Speech-Language Pathologist, Department of Neurology
Industry Update
Timothy Andrew Howell, MA
Evidera, United States
Research Associate III
FDA Update
Naomi Knoble, PhD
FDA, United States
Reviewer, Division of Clinical Outcome Assessment, OND, CDER
Patient Update
Allison Seebald
National Organization for Rare Disorders (NORD), United States
Senior Research Program Manager
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