The U.S. FDA’s Office of Special Medical Programs/Office of Orphan Products Development (OSMP/OOPD) has launched a new grants program that will provide $2 million in funding to research the natural history of rare diseases. The natural history of the disease includes the time immediately prior to its inception, through a pre-symptomatic phase and different clinical stages, to a final outcome in the absence of treatment. The goals of the research program are to characterize the natural history of rare diseases; identify sub-populations; and develop clinical outcome measures, biomarkers, and companion diagnostics.
This is the first time the U.S. FDA has provided funding for this type of study.
“Not understanding how a rare disease progresses is often a major obstacle in the development of life-saving medical products,” said Gayatri Rao, MD, JD, director of the OOPD, in a press release from the agency. “Information about a disease’s natural history can aid in clinical trial design, identify study endpoints, and lead to faster, better trials – hopefully leading to new and effective diagnostics and treatments.”
Grant applications are due by October 14, 2016, with an anticipated starting fund date of March 2017.
Source: U.S. FDA press release, February 29, 2016.