The U.S. 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.
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.
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 October 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:
Prospective Natural History Studies
- 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.
Retrospective Natural History Studies or Survey Studies
- 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.
Further information is available at the FDA Orphan Products Natural History Grants Program website.