RAPS

Natural History Studies for Rare Diseases: FDA Drafts Guidance

The US Food and Drug Administration (FDA) on Friday published new draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of ...
The American Journal of Managed Care

FDA Releases Draft Guidance Document on Natural History Studies for Rare Diseases

Earlier this week, the FDA released a draft guidance document titled, "Rare Diseases: Natural History Studies for Drug Development” with the intention of informing both the design and implementation ...
Pharm Exec

Real-World Data Brings New Insights to Natural History of Disease Studies

Natural history studies guide clinically meaningful endpoint selection, trial duration, and population enrichment, and can supply historical/external controls when randomized internal controls are ...
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 ...