Meeting 2: Accelerating Rare Diseases Research and Orphan Product Development
- When:
- November 23, 2009 (8:00 AM Eastern)
- Where:
- National Academy of Sciences Building • 2101 Constitution Avenue, NW, Washington, DC 20006 Map
- Topics:
- Biomedical and Health Research, Diseases, Quality and Patient Safety
- Activity:
- Accelerating Rare Diseases Research and Orphan Product Development
- Board:
- Board on Health Sciences Policy
8:20 Welcome and introductions
Thomas Boat, M.D., Committee Chair
8:30 Panel 1
National Organization for Rare Disorders
Wayne L. Pines
Genetic Alliance
Sharon F. Terry, President and CEO
Discussion
9:10 Panel 2
Cystic Fibrosis Foundation Therapeutics, Inc.
Diana R. Wetmore, Ph.D., Vice President of Alliance Management
Multiple Myeloma Research Foundation and Multiple Myeloma Research Consortium
Susan Kelley, M.D., Chief Medical Officer
Friedreich’s Ataxia Research Alliance
Jennifer Farmer, M.S., Executive Director
Fanconi Anemia Research Fund (by phone)
Dave Frohnmayer, J.D., Co-founder and Board Vice President;
Lynn Frohnmayer, M.S.W., Co-founder and Advisor to the Board
10:10 Break
10:30 Discussion before, during, and after break
11:00 Panel 3
Pharmaceutical Research and Manufacturers Association (PhRMA)
Alan Goldhammer, Ph.D., Vice President Scientific and Regulatory Affairs
Advanced Medical Technology Association (AdvaMed)
Susan Alpert, Ph.D., M.D., Senior Vice President, Global Regulatory Affairs, Medtronic, Inc.)
Biotechnology Industry Organization (BIO)
Sara Radcliffe, M.P.H., Acting Executive Vice President, Health and Vice President for Science and Regulatory Affairs
Discussion
Noon Working lunch for committee members and speakers
1:00 Panel 4
Mark L. Batshaw, M.D.
Principal Investigator, Urea Cycle Disorders Consortium (NIH Rare Diseases Clinical Research Network); Chief Academic Officer, Children's National Medical Center; Chairman of Pediatrics and Associate Dean for Academic Affairs, the George Washington University School of Medicine and Health Sciences
Frederick Kaplan, M.D.
Isaac & Rose Nassau Professor of Orthopaedic Molecular Medicine; Director, Center for Research in FOP & Related Disorders, University of Pennsylvania School of Medicine
Alan K. Percy, M.D.
Principal Investigator, Angelman, Rett, and Prader-Willi Syndromes Consortium (NIH Rare Diseases Clinical Research Network); Medical Director, Civitan International Research Center; Professor of Pediatric Neurology, University of Alabama at Birmingham
Discussion
2:20 Open discussion
2:45 Adjourn open session