RAPS

FDA officials stress natural history studies in supporting rare disease drug development

To promote the development of more treatments for rare diseases, sponsors should leverage data from natural history studies when it is not possible to use data from two adequate and well-controlled ...

Food and Drug Administration (FDA): What It Is and Does

The Food and Drug Administration is a government agency that regulates certain food, drugs, cosmetics, and medical products.
RAPS

Industry Seeks More From FDA on Natural History Study Guidance

Merck, Lundbeck and Ionis Pharmaceuticals are seeking more details from the US Food and Drug Administration’s (FDA) draft guidance on natural history studies, including discussions of using ...