FDA makes $2 million available to fund natural history studies in rare diseases

The US Food and Drug Administration (FDA) has announced the availability of $2 million in research grants to fund natural history studies in rare diseases.

The aim is to collect data on how specific rare diseases progress in individuals over time so that knowledge can inform and support product development and approval. This will be the first time the FDA will provide funding through its Orphan Products Grants to conduct these types of studies for rare diseases.

Natural history is the course a disease takes in affected individuals 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. This type of information is often not available, or incomplete, for many rare diseases.

“Rare diseases are often poorly understood,” said Gayatri Rao, M.D., J.D., director of the FDA’s Office of Orphan Products Development. “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, identify study end points and lead to faster, better trials – hopefully leading to new and effective diagnostics and treatments.”

Goal to help characterise the natural history of rare diseases

The goal of the programme is to help characterise the natural history of rare diseases, identify subpopulations, and develop and/or validate clinical outcome measures, biomarkers and companion diagnostics.

FDA is using the funds for this new Orphan Products Natural History Grants Programme as well as the existing Orphan Products Grants Programme for clinical trials, which has provided more than $350 million to fund more than 570 new clinical studies and has supported the marketing approval of more than 55 products since its creation in 1983.

Grant applications will be due 14 October 2016. All responsive applications will be reviewed and evaluated for scientific and technical merit by a panel of rare disease and natural history experts.