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

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





FDA In Brief: FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases

The U.S. Food and Drug Administration today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development. This draft guidance is intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and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 ...