Pediatric Cholestatic Disease Program

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