The U.S. Food and Drug Administration (FDA) recently awarded six research grants for natural history studies in rare diseases.
One of the grants was awarded to Alicia Gerke, assistant professor of pulmonary, critical care, and occupational medicine at the University of Iowa. Gerke will receive approximately $300,000 over two years to conduct a retrospective study in sarcoidosis.
The aim of this study is to establish a large, wide-ranging, retrospective group of sarcoidosis patients.
The natural history of a disease is defined as the course it takes from onset through presymptomatic and clinical stages to a final outcome. Gerke’s study will focus on the natural history of disease development in sarcoidosis patients. The goal is to find important predictive markers for treatment responsiveness and improve treatment protocols.
For the first time, the FDA has awarded four grants totaling $6.3 million to be used over the next five years for natural history studies in rare diseases. The funds are distributed through its Orphan Products Grants Program.
In addition to Gerke, three other proposals received approximately $2 million each over five years:
- David Lynch, from the Children’s Hospital of Philadelphia, to study Friedreich’s ataxia;
- Adi Cohen, from the Columbia University Medical Center, to study pregnancy and lactation-associated osteoporosis;
- Kenneth Ataga, at the University of North Carolina at Chapel Hill, to study sickle cell anemia.
Two additional grants primarily being funded by the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) include:
- Wen-Hann Tan, from the Children’s Hospital Corporation, to study Angelman syndrome;
- Nicholas Johnson, from the University of Utah, to study myotonic dystrophy type 1.
NCATS is funding these grants through its Therapeutics for Rare and Neglected Diseases (TRND) program.
“We’re grateful to NCATS for its support of this important program, which enabled us to extend our support to two additional studies,” Scott Gottlieb, FDA commissioner, said in a press release.
“We believe these important studies will provide key information about how these rare diseases develop and progress and can ultimately help in the development of models of disease behavior that can make the development process more efficient,” Gottlieb added.
Congress assigned funding to the FDA for the study of rare diseases, which have a prevalence of less than 200,000 people in the United States, as defined by the Orphan Drug Act. There are 7,000 known rare diseases that together affect approximately 30 million Americans.
All 80 grant applications received by the FDA were reviewed and evaluated for scientific and technical merit by rare disease and natural history experts from academia, patient groups, the NIH, and the FDA.
According to the FDA, the six winning applications address unmet needs in a broad spectrum of diseases.
“One of the challenges we encounter developing therapies for rare diseases is the lack of natural history data to guide the design of successful clinical trials,” said Nora Yang, PhD, director of portfolio management and strategic operations in NCATS’ Division of Pre-Clinical Innovation.
“We are pleased that we can collaborate with the FDA to fund two natural history studies this year that will help NCATS develop novel treatments for patients who suffer from these devastating diseases,” Yang added.
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