Posted by Michael Wonder on 23 Mar 2019
FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases
22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development.
This draft guidance is intended to help inform the design and implementation of natural history studies that can be used to support the development of safe and effective drugs and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 a natural history study.
In April, the FDA is holding a public meeting to obtain patients’ and caregivers’ perspectives on the impacts of rare diseases on daily life and to assess their common experiences.
