The FDA has awarded 15 grants totaling more than $19 million to boost the development of medical device, drug, and biological products for patients with rare diseases, with at least a quarter of the funding going to studies focused solely on pediatrics.
The FDA awards grants for clinical studies on safety and/or effectiveness of products that could either result in, or substantially contribute to, approval of the products.
The program is administered through the FDA’s Orphan Products Grants Program. This program was created by the Orphan Drug Act, passed in 1983, to promote the development of products for rare diseases. Since its inception, the program has given more than $330 million to fund more than 530 new clinical studies on developing treatments for rare diseases and has been used to bring more than 50 products to marketing approval.
For the grants program therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the United States. There are about 7,000 rare diseases and conditions, according to the National Institutes of Health. In total, nearly 30 million Americans suffer from at least one rare disease.
The 2014 grant recipients included:
- Kelly Dooley, The Johns Hopkins University (Baltimore, Md), Phase 2 Study of PA-824 for the Treatment of Pulmonary Tuberculosis— $1.6 million over four years
- Michael Portman, Seattle Children’s Hospital (Seattle, Wash.), Phase 3 Study of Triiodothyronine Supplementation for the Treatment of Young Infants After Cardiopulmonary Bypass— approximately $1.6 million over four years
- Scott Rollins, Selexys Pharmaceuticals Corporation (Oklahoma City, Okla.), Phase 2 Study of SelG1 for the Treatment of Sickle Cell Disease— $1.6 million over four years
- Beena Sood, Wayne State University (Detroit, Mich.), Phase 1/2 Study of Aerosolized Survanta for the Treatment of Neonatal Respiratory Distress Syndrome— approximately $1.4 million over four years
- Warren Stern, Aesrx, LLC (Newton, Mass.), Phase 2 Study of Aes103 (5-HMF) for the Treatment of Stable Sickle Cell Disease—approximately $1.6 million over four years
- Pamela Zeitlin, The Johns Hopkins University (Baltimore, Md.), Phase 2 Study of Digitoxin for the Treatment of Cystic Fibrosis— approximately $290,000 for one year