Room: Marriott Balcony B
Rare disease, or Orphan disease, for drugs/biologics is defined as disease with a prevalence of less than 200,000 in the United States at any given time, or as disease with a prevalence of less than 5 per 10,000 in Europe. In this roundtable discussion, we will focus on discussion about the use of historical data or natural history of disease in each stage of rare disease drug development. Additionally, we will also discuss about how data from natural history studies can be applied in rare disease to support a drug’s marketing approval. The discussion will also be based on the FDA guidance about “Rare Diseases: Natural History Studies for Drug Development”. Case studies may be provided to facilitate the discussion.