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DIA 2021 Global Annual Meeting

The Rare Disease Clinical Outcome Assessment Consortium: Collaboration Aimed at Accelerating Rare Disease Drug Development

Session Chair(s)

Lindsey Murray, PhD, MPH

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.

Speaker(s)

Tori Brooks, MPH

Academic Update

Tori Brooks, MPH

  • Research Associate II
  • Mapi Research Trust, United States
Kiera Berggren, MA, MS

Academic Update

Kiera Berggren, MA, MS

  • Research Speech-Language Pathologist, Department of Neurology
  • Virginia Commonwealth University, United States
Timothy Andrew Howell, MA

Industry Update

Timothy Andrew Howell, MA

  • Research Associate III
  • Evidera, United States
Naomi Knoble, PhD

FDA Update

Naomi Knoble, PhD

  • Reviewer, Division of Clinical Outcome Assessment, OND, CDER
  • FDA, United States
Allison Seebald

Patient Update

Allison Seebald

  • Senior Research Program Manager
  • National Organization for Rare Disorders (NORD), United States