FDA Awards 21 Grants for Trials on Rare Diseases

The FDA awarded clinical trial grants, totaling more than $23 million over four years, for research on 21 rare diseases, including amyotrophic lateral sclerosis, brain tumors, cystic fibrosis and drug-resistant HIV.

Over 40 percent of the awards fund studies in pediatric patients, with two focused on transplantation and related indications. One project is a medical device trial developing a fully implantable neuroprosthesis in individuals with spinal cord injuries, aiming to help patients use their hands more effectively. About one-quarter of the funded studies focus on different adult and childhood cancers.

The FDA awarded the funds through its Orphan Products Clinical Trials Grants Program. The grants are intended for clinical studies that could contribute to agency approval. In 2015, five funded studies supported product approvals, including one treatment for neuroblastoma.