Is There a Recipe for Successful Implementation of Registries for Rare Diseases?
Track
:
Track 17: Rare/Orphan Diseases
Program Code:
260
Date:
Tuesday, June 25, 2013
Time:
1:45 PM to 3:15 PM
EST
Location:
153C
CHAIR
:
Martine Zimmermann, PharmD (SPKNON), Vice President, Global Regulatory Affairs, Alexion Pharma International Sŕrl, Switzerland
20 years of experience in scientific research, clinical development and global regulatory affairs. Spent 4 years in basic research in TOKYO. Working in Global Reg Affairs since 2000. Currenly ex director at Alexion Pharma a biopharma company specialized in the dev of drugs for ultra orphan disease
SPEAKER
(S):
Samantha Parker, MBA (SPKNON), Director of External Affairs and Rare Disease Partnerships, Orphan Europe, France
Samantha’s role, in Orphan Europe, includes collaboration with rare disease expert networks and registries. She currently focuses on partnerships between academic disease registries and those run by the industry for PMS and RMPs. She is a member of the EUCERD and the IRDiRC
Marshall Summar, MD (SPKSUP), Chief, Genetics and Metabolism, Children's National Medical Center, United States
Chief: Division of Genetics and Metabolism, Children’s National Medical Center / Director: Clinical Research Center / Vice Chair Special Projects and External Opportunities Margaret O’Malley Chair of Molecular Genetics. He is an international expert in biochemical genetics and the urea cycle.
Larissa Lapteva, (SPKAGY), Medical Officer, Rare Diseases Program, Office of New Drugs, CDER, FDA, United States
Dr. Larissa Lapteva represents the Rare Disease Program of the Office of New Drugs, CDER, FDA.
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.