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Monday, June 15 • 3:30pm - 4:30pm
#129 SL: The Rare Disease Clinical Outcome Assessment Consortium: Aiming to Fulfill Unmet Drug Development Endpoint Measurement Needs

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Component Type: Forum
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-20-636-L04-P; CME 1.00; IACET 1.00; RN 1.00

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 Objectives

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.


Lindsey Murray, PhD, MPH


Michelle Campbell, PhD

Stephen Joel Coons, PhD

Vanessa Boulanger, MSc

Dylan Trundell, MSc

avatar for Lindsey Murray

Lindsey Murray

Associate Director, PRO Consortium, Critical Path Institute, United States
Lindsey Murray, PhD, is the Associate Director for Rare Diseases at the Critical Path Institute’s (C-Path) Patient-Reported Outcome (PRO) Consortium. Dr. Murray has more than 14 years of experience in clinical and health outcomes research, specializing in quantifying the patient’s... Read More →
avatar for Vanessa Boulanger

Vanessa Boulanger

Director of Research Programs, National Organization for Rare Disorders (NORD), United States
Vanessa Boulanger is the Director of Research at the National Organization for Rare Disorders (NORD). In this role, Vanessa oversees the management, growth, and implementation of NORD’s research and scientific activities. In 2018, Vanessa was appointed as a member of the Patient-Centered... Read More →
avatar for Michelle Campbell

Michelle Campbell

Associate Director, Stakeholder Engagement and Clinical Outcomes, ON, OND, CDER, FDA, United States
Michelle Campbell is the Associate Director for Stakeholder Engagement and Clinical Outcomes in the Office of Neuroscience, Office of New Drugs (OND), Center for Drug Evaluation and Research (CDER), U.S. Food and Drug Administration (FDA). Dr. Campbell joined FDA in 2014 and was previously... Read More →
avatar for Stephen Joel Coons

Stephen Joel Coons

Executive Director, PRO Consortium, Critical Path Institute
Stephen Joel Coons is Executive Director of the Patient-Reported Outcome (PRO) Consortium at Critical Path Institute (C-Path). C-Path, an independent nonprofit organization, established the PRO Consortium in cooperation with the U.S. Food and Drug Administration and the pharmaceutical... Read More →

Dylan Trundell

Senior Principal PCOR Scientist, Roche Products Ltd.
Dylan Trundell is a health outcomes researcher with 10+ years experience. He has led the development of clinical trial measurement strategies, including the development of novel COAs, across several disease areas, including multiple rare diseases.

Monday June 15, 2020 3:30pm - 4:30pm EDT
TBD Virtual Event Horsham, PA 19044
  02: ClinTrials -ClinOps, Forum