Rare Diseases: Natural History Studies for Drug Development Guidance for Industry

Rare Diseases: Natural History Studies for Drug Development Guidance for Industry

Today the U.S. Food and Drug Administration (FDA) is making available a draft guidance titled Rare Diseases: Natural History Studies for Drug Development, Guidance for Industry. This guidance is intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and biological products for rare diseases. Specifically, this guidance describes the broad potential uses of a natural history study in all phases of drug development for rare diseases. It covers the strengths and weaknesses of various types of natural history study designs, common data elements and research plans, and a practical framework for the conduct of a natural history study.  Most rare diseases have no approved therapies. Overall, this presents a significant unmet public health need, making this area a priority for the FDA. FDA looks forward to receiving comments on this draft guidance from stakeholders via the associated docket (FDA-2019-D-0481). More information on FDA’s efforts pertaining to rare diseases is available on FDA’s Office of Orphan Products Development and Center for Drug Evaluation and Research’s Rare Diseases Program websites.