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DIA 2018 Global Annual Meeting

The DIA 2018 Global Annual Meeting brings together thousands of innovators from around the globe.

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

    Session Chair(s)
      Adora  Ndu, JD, PharmD

      Adora Ndu, JD, PharmD

      • Executive Director, Global Regulatory Policy, Research and Engagement
      • BioMarin Pharmaceutical Inc., 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