FDA announces availability of $2 million for rare disease research

The U.S. Food and Drug Administration today 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 individualsfrom the time immediately prior to its inception, progressing through a presymptomatic 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, within the Office of Special Medical Programs. "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."

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

"Despite their importance, it is very difficult for groups, such as patient advocacy organizations, to find funding to conduct natural history studies," said Dr. Rao. "As we observe Rare Disease Day today, we are excited to announce a program that will bridge some of this funding gap and complement existing efforts to help bring new and important therapies to rare disease patients."

Congress appropriates funding for the study of rare diseases. FDA is using the funds for this new Orphan Products Natural History Grants Program as well as the existing Orphan Products Grants Program 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 Oct. 14, 2016. All responsive applications will be reviewed and evaluated for scientific and technical merit by a panel of rare disease and natural history experts. The anticipated start date of funding for grantees is March 2017.

The funding levels and durations of these grants will include:

• A maximum of $400,000 in total costs per year for up to five years for prospective (looking forward) natural history studies involving clinical examination of affected individuals.
• A maximum of $150,000 in total costs per year for up to two years for retrospective (looking back) natural history studies (i.e., chart review) or survey studies (i.e., questionnaire).

Rare diseases, as defined in the Orphan Drug Act, are diseases or conditions with a prevalence of less than 200,000 persons in the United States. Though the diseases are individually rare, together, the 7,000 known rare diseases affect approximately 30 million Americans.