Is There a Recipe for Successful Implementation of Registries for Rare Diseases?

Track:
Rare/Orphan Diseases

Day & Time:
June 25, 1:45PM - 3:15PM

Session Number:
260

Room Number:
153C

Level:
Intermediate

Type:
Session

Title:
Is There a Recipe for Successful Implementation of Registries for Rare Diseases?

Chair(s):
Martine Zimmermann, PharmD
Executive Director, Global Regulatory Affairs
Alexion Pharma International Sàrl, Switzerland

Description:
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(s):
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.

Presentation(s) & Speaker(s):
Public-private Partnerships for Registries: Challenges and Solutions
Samantha Parker, MBA
Director of External Affairs and Rare Disease Partnerships
Orphan Europe, France

Urea Cycle Disorders as a Model of Rare Disease Registries
Marshall Summar, MD
Chief, Genetics and Metabolism
Children's National Medical Center, United States

FDA Perspective
Larissa Lapteva
Medical Officer, Rare Diseases Program, Office of New Drugs, CDER
FDA, United States