Natural history (NH) studies are an important tool for understanding the etiology, range of manifestations, and progression of rare diseases. Well conducted NH studies can yield information on biomarkers and other correlates of clinical outcome. Obtaining maximum value to support drug development programs depends on conducting these NH studies early, long before potential therapeutic agents are identified for development. Comprehensive, good quality NH studies designed with an eye toward supporting drug development programs can avoid some of the common problems that lead to stalled, slow, or inefficient drug development for rare diseases.
When: May 16–17, 2012
Where: Natcher Conference Center | NIH Campus | Bethesda, Maryland
Visit the Workshop's website to view the agenda and register.