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Wednesday, June 27 • 2:00pm - 3:15pm
#370: Clinical Outcome Assessments (COA) Endpoints for Use in Rare and Ultra-Rare Disease Clinical Trials

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Component Type: Forum
Level: Intermediate
CE: ACPE 1.25 Application UAN: 0286-0000-18-667-L04-P; CME 1.25; IACET 1.25; RN 1.25

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 Objectives

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.


Adora Ndu


Sarrit Kovacs

Chad Gwaltney, PhD

Kate Delaney


Kate Delaney

Director, Regulatory Patient Engagement and Outcomes Research, BioMarin Pharmaceutical Inc.
Kate Delaney has worked in pediatric rare disease research for nearly 20 years and is currently Director, Patient Engagement and Outcomes Research, BioMarin Pharmaceutical. Publications include neurocognitive and PRO assessment work and regulatory patient engagement. Kate's career... Read More →
avatar for Chad Gwaltney

Chad Gwaltney

President, Gwaltney Consulting
Dr. Gwaltney’s work focuses on the development of innovative methods to measure patient-centered outcomes in clinical trials. He has published numerous articles and book chapters addressing how the patient’s perspective can be examined to better understand product efficacy and... Read More →
avatar for Sarrit Kovacs

Sarrit Kovacs

Clinical Outcome Assessments (COA) Staff, OND, CDER, FDA
Sarrit Kovacs, PhD is a reviewer with the Clinical Outcome Assessments (COA) Staff in the Office of New Drugs (OND) in the Center for Drug Evaluation and Research (CDER) at FDA. Sarrit has nearly 20 years of social science research experience and is a consultant to CDER and other... Read More →
avatar for Adora Ndu

Adora Ndu

Chief Regulatory Affairs Officer, BridgeBio Pharma, United States
Adora Ndu, PharmD, JD, is Chief Regulatory Affairs Officer at Bridgebio Pharma. Prior to joining Bridgebio, Adora spent several years at BioMarin Pharmaceutical in various roles including as Group Vice President, and Head of Worldwide Research and Development Strategy, Scientific... Read More →

Wednesday June 27, 2018 2:00pm - 3:15pm EDT
Room 204AB Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  09: Regulatory, Forum