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DIA 2021 Global Annual Meeting
The Rare Disease Clinical Outcome Assessment Consortium: Collaboration Aimed at Accelerating Rare Disease Drug Development
Lindsey Murray, PhD, MPH
- Associate Director, PRO 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.
Tori Brooks, MPH
- Research Associate II
- Mapi Research Trust, United States
Kiera Berggren, MA, MS
- Research Speech-Language Pathologist, Department of Neurology
- Virginia Commonwealth University, United States
Timothy Andrew Howell, MA
- Research Associate III
- Evidera, United States
Naomi Knoble, PhD
- Reviewer, Division of Clinical Outcome Assessment, OND, CDER
- FDA, United States
- Senior Research Program Manager
- National Organization for Rare Disorders (NORD), United States