publication date: Mar. 4, 2016

FDA Providing $2 Million for Natural History Studies in Rare Diseases


FDA will provide $2 million in two to five research grants for the study of the natural history of rare diseases. The objective of the grants is to expedite the development of products for these conditions.

The Feb. 29 announcement marks the first time FDA will provide funding through its Orphan Products Grants to collect data on the progression of specific rare diseases in individuals over time.

Rare diseases, as defined by the Orphan Drug Act, are diseases that have a prevalence of less than 200,000 persons in the U.S. Altogether, about 7,000 known rare diseases affect approximately 30 million Americans.

The studies will focus on characterizing the natural history of the diseases, identifying subpopulations, developing and showing the validity of clinical outcome measures, biomarkers and companion diagnostics.

Information on the progression of many rare diseases is often unavailable. Natural history is the course a disease takes from the time immediately prior to its inception, progressing through a pre-symptomatic phase and different clinical stages, to a final outcome in the absence of treatment.

“Rare diseases are often poorly understood,” Gayatri Rao, director of the FDA Office of Orphan Products Development, within the Office of Special Medical Programs, said in a statement. “Not understanding how a rare disease progresses is often a major obstacle in the development of life-saving medical products. Information about a disease’s natural history can aid in clinical trial design, … Continue reading 42-09 FDA Providing $2 Million for Natural History Studies in Rare Diseases

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