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DIA 2018 Global Annual Meeting

Clinical Outcomes Assessments (COA) Endpoints for Use in Rare and Ultra-Rare Disease Clinical Trials

    Session Chair(s)
      Andrew E. Mulberg, MD

      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


      Ebony N. Dashiell-Aje, PhD

      • Clinical Outcome Assessments Reviewer, COA Staff, OND, CDER
      • FDA, United States
      Chad  Gwaltney, PhD


      Chad Gwaltney, PhD

      • President
      • Gwaltney Consulting, United States
      Kate  Delaney


      Kate Delaney

      • Director, Regulatory Patient Engagement and Outcomes Research
      • BioMarin Pharmaceutical Inc., United States