Friday, March 19, 2021
Materials
Agenda
Participant List
Presentations
Welcome & Introduction
Jessica Weber, Forum for Collaborative Research
Stakeholder Perspectives On Demonstrating Efficacy And Safety In Clinical Trials:
Academic Perspective
Saul Karpen, Emory University School of Medicine/ Children’s Healthcare of Atlanta
Henkjan Verkade, University of Groningen
Patient Advocacy Perspective
Roberta Smith, Alagille Syndrome Alliance
Pediatric Drug Development: FDA Perspective
Ruby Mehta, U.S. Food and Drug Administration
Overview of Current Paediatric Investigation Plans: EMA Perspective
Chrissi Pallidis, European Medicines Agency
Industry Perspective
Pam Vig, Mirum Pharma
