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, often 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.
This workshop brought together thought leaders in the design, conduct, and evaluation of natural history studies to discuss the role of these studies in the development of therapeutic candidates.