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 FDA is in a unique position to help those who suffer from rare diseases by offering several important incentives to promote the development of products for rare diseases, one of which is this grants program,” said Gayatri R. Rao, M.D., director of the FDA’s Office of Orphan Product Development. “The grants awarded this year support much-needed research in difficult-to-treat diseases that have little, or no, available treatment options.”

The program is administered through the FDA’s Orphan Products Grants Program, 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.

The 2014 grant recipients are:

• Denise Adams, of Cincinnati Children's Hospital Medical Center. The phase II study of vincristine versus sirolimus for the treatment of high risk kaposiform hemangioendothelioma will be funded with $1.6 million over four years.
• Mitesh Borad, Mayo Clinic Arizona, receives approximately $600,000 over three years for a phase I study of VSV-hIFN-B for the treatment of hepatocellular carcinoma.
• Andrew Brenner, University of Texas Health Center San Antonio, will run a phase II study of TH-302 for the treatment of glioblastoma to be funded by approximately $1.6 million over four years.
• Kelly Dooley, Johns Hopkins University, Baltimore, Md., receives $1.6 million over four years for a phase II study of PA-824 for the treatment of pulmonary tuberculosis.
• Donald Durden, University of California San Diego, receives $1.6 million over four years for a phase II study of Poly-ICLC for the treatment of pediatric low grade gliomas.
• Alfred Lane, Stanford University, California, will run a phase II study of sildenafil for the treatment of lymphatic malformations with approximately $1.6 million over four years.
• Dung Le, of Johns Hopkins University, receives $1.6 million over four years for a phase II study of folfirinox followed by ipilimumab/GVAX for the treatment of pancreatic cancer.
• Phillip Low, Mayo Clinic Rochester, Minnesota, will run a phase I study of intrathecal autologous mesenchymal stem cell therapy for the treatment of multiple system atrophy with $600,000 over three years.
• Guido Magni, River Vision Development, New York, N.Y., receives $1.2 million over three years for a phase II study of RV001 for the treatment of thyroid eye disease.
• Michael Portman, Seattle Children's Hospital, will run a phase III study of triiodothyronine supplementation for the treatment of young infants after cardiopulmonary bypass will receive approximately $1.6 million over four years.
• Jana Portnow, City of Hope Beckman Research Institute, Duarte, Calif., receives $600,000 over three years for a phase I study of neural stem cells & 5-FC/leucovorin for the treatment of recurrent high grade gliomas.
• Scott Rollins, Selexys Pharmaceuticals, Oklahoma City, Okla., will run a phase II study of SelG1 for the treatment of sickle cell disease with $1.6 million over four years.
• Beena Sood, Wayne State University, Detroit, Mich., will run a phase I/II study of aerosolized survanta for the treatment of neonatal respiratory distress syndrome with approximately $1.4 million over four years.
• Warren Stern, Aesrx, ofNewton, Mass., receives approximately $1.6 million over four years for a phase II study of Aes103 (5-HMF) for the treatment of stable sickle cell disease.
• Pamela Zeitlin, Johns Hopkins University, will run a phase II study of digitoxin for the treatment of cystic fibrosis with approximately $290,000 for one year.

For the grants program therapies, a disease or condition is considered rare if it affects less than 200,000 persons in the U.S. There are about 7,000 rare diseases and conditions, according to the NIH. In total, nearly 30 million Americans suffer from at least one rare disease.