Clinical Outcomes Assessments (COA) Endpoints for Use in Rare and Ultra-Rare Disease Clinical Trials
Andrew E. Mulberg, MD
Vice President, Global Regulatory Affairs
Amicus Therapeutics , United States
This session will explore the challenges and opportunities related to patient-focused clinical outcome assessment (COA) endpoint selection, development and implementation in rare and ultra-rare disease drug development. Panelists from industry, the FDA and instrument development experts will provide insight into the identification and implementation of fit-for-purpose COAs in rare disease trials, including challenges specific to endpoint strategy development and clinical trial design. Relevant case studies will be highlighted and special considerations discussed.
Learning Objective : Describe opportunities and challenges in rare disease drug development; Identify how challenges in rare disease clinical trials may be addressed and overcome; Determine how innovators and patient advocates can work with FDA to promote innovation in this space.
Ebony N. Dashiell-Aje, PhD
Clinical Outcome Assessments Reviewer, COA Staff, OND, CDER
FDA, United States
Chad Gwaltney, PhD
Gwaltney Consulting, United States
Director, Regulatory Patient Engagement and Outcomes Research
BioMarin Pharmaceutical Inc., United States