In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development, addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack of natural history data to guide the design of successful clinical trials.
The natural history of a disease is the course it takes in the absence of investigational intervention. To gain insight into this, investigators conduct observational studies designed to follow the course of the disease. The objective of these studies is to identify demographic, genetic, environmental, and other factors that correlate with the development and outcomes of the disease.
Read more to learn about the important role natural history studies play in drug development and their benefits.