The U.S. Food and Drug Administration (FDA) recently stated that there is now a total of $2 million in research grants available to finance natural history studies of rare diseases.
The goal is to gather data about how certain rare disease progress in people over time. This is the best way that scientists can collect knowledge that will inform as well as support their researcher, development and approval of new products.
The funds are part of the Orphan Products Grants, and it is the first time the FDA has taken from this fund to finance different kinds of studies about rare diseases.
“Rare diseases are often poorly understood,” Gayatri Rao, director of the FDA’s Office of Orphan Products Development, said. “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 is to develop the identity of subpopulations, the natural history of diseases, companion diagnostics, biomarkers and create or confirm clinical outcome measures.
“Despite their importance, it is very difficult for groups, such as patient advocacy organizations, to find funding to conduct natural history studies,” Rao said. “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.”