戻る Agenda
Is There a Recipe for Successful Implementation of Registries for Rare Diseases?
Session Chair(s)
Martine Zimmermann, PHARMD
Senior Vice President, Head of Global Regulatory Affairs
Ipsen , France
This session will describe how registries can support development of new drug for rare diseases, how they can contribute to regulatory approval, and decisions on access to orphan medicinal products.
Learning Objective : Discuss how registries have been identifies as high priorities by governing bodies; Explain how this translates into increased collaborating to foster development and access to new medicine for rare diseases.
Speaker(s)
Public-private Partnerships for Registries: Challenges and Solutions
Samantha Parker, MBA
Orphan Europe, France
Director of External Affairs and Rare Disease Partnerships
Urea Cycle Disorders as a Model of Rare Disease Registries
Marshall Lynn Summar, MD
Children's National Medical Center, United States
Division Chief, Genetics and Metabolism; Director, Rare Disease Institute
FDA Perspective
Larissa Lapteva
FDA, United States
Associate Director, Division of Clinical Evaluation, OTAT, CBER