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Natural History Studies for Rare Diseases and Orphan Conditions
Program Code:
346
Date:
Wednesday, June 27, 2012
Time:
10:00 AM to 11:30 AM
EST
CHAIR
:
Dr. Stemhagen has more than 30 years of research experience, 20 yrs in safety surveillance. She has expertise in design, conduct, and analysis of peri-approval registries, LSS, actual use studies, including >35 regulatory-mandated safety studies. She has developed REMS for more than 80 products.
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PRESENTER
(S):
Dr. Stemhagen has more than 30 years of research experience, 20 yrs in safety surveillance. She has expertise in design, conduct, and analysis of peri-approval registries, LSS, actual use studies, including >35 regulatory-mandated safety studies. She has developed REMS for more than 80 products.
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Marshall Summar, Childrens National Medical Center, United States
Anne Pariser is the Associate Director for Rare Diseases in OND/CDER/FDA. She has 10 years of experience at FDA, and during this time has worked almost exclusively on therapeutics development for Orphan diseases and to address unmet medical needs in rare disease populations.
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Description
This session presents an overview of the design, conduct, and benefits of natural history studies of rare diseases and orphan conditions from varied perspectives of researchers, patients and a regulatory agency.
Learning Objectives:
Discuss the need for natural history studies in drug development
Describe the types of natural history studies and their strengths and weaknesses
Define the key elements of an orphan disease natural history study Discuss FDA initiatives regarding rare diseases.