A Natural History is the understanding of a disease progression throughout the lifespan of a patient. The collection of data from a significant number of patients affected by diverse phenotypes inside the same condition contribute to describe the history of a disease. It is a major issue in Rare Diseases research.View this resource Bookmark this resource
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. Although the focus of this guidance is rare diseases; the recommendations in the guidance may be applicable to drug development for nonrare diseases.