Natural history study« Back to Glossary Index
A natural history study is a preplanned observational study intended to track the course of the disease. Its purpose is to identify demographic, genetic, environmental, and other variables (e.g., treatment modalities, concomitant medications) that correlate with the disease’s development and outcomes. Natural history studies are likely to include patients receiving the current standard of care and/or emergent care, which may alter some manifestations of the disease.
The natural history of a disease is traditionally defined as the course a disease takes in the absence of intervention in individuals with the disease, from the disease’s onset until either the disease’s resolution or the individual’s death.
U.S. Food and Drug Administration (FDA) Guidance Rare Diseases: Natural History Studies for Drug Development
NCATS Toolkit for Patient-Focused Therapy Development: Natural History Study