Rare Disease Research Project Hopes to Help Product Development

The FDA is offering $2 million in new grants for natural history studies of rare disease.

Their goal is to collect data on how specific rare diseases progress in individuals over time so that the findings can help support product development and approval.

The results can help identify subpopulations, and develop and/or validate clinical outcome measures, biomarkers and companion diagnostics, according to the agency.

“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.”

Natural history is the course a disease takes in affected individuals from 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.

Grant applications will be due Oct. 14. The anticipated start date of funding is March 2017.