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Virtual Event

Jun 14, 2020 9:00 AM - Jun 18, 2020 5:00 PM

(Eastern Standard Time)

Fort Washington, PA 19034

DIA 2020 Global Annual Meeting

The Rare Disease Clinical Outcome Assessment Consortium: Aiming to Fulfill Unmet Drug Development Endpoint Measurement Needs

Session Chair(s)

Lindsey  Murray, PhD, MPH

Lindsey Murray, PhD, MPH

Executive Director, Rare Disease COA Consortium

Critical Path Institute, United States

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 Objective : 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.

Speaker(s)

Michelle  Campbell, PhD

Panelist

Michelle Campbell, PhD

FDA, United States

Associate Director, Stakeholder Engagement and Clinical Outcomes, ON, OND, CDER

Stephen Joel  Coons, PhD

Panelist

Stephen Joel Coons, PhD

Critical Path Institute, United States

Senior Advisor

Vanessa  Boulanger, MSc

Panelist

Vanessa Boulanger, MSc

National Organization for Rare Disorders (NORD), United States

Director of Research Programs

Dylan  Trundell, MSc

Panelist

Dylan Trundell, MSc

Roche Products Ltd., United Kingdom

Senior Principal PCOR Scientist

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