August 13, 2014
With support from the FDA’s Office of Orphan Products Development, the authors of an article published in Expert Opinion on Orphan Drugs studied the impact of the FDA’s Orphan Products Grants Program on the development of medicines and products for rare diseases. Since the Orphan Drug Act launch in 1983, US$320 million have been awarded to clinical studies on rare disease products. From an initial US$500,000 in 1983, funding has continued to rise, reaching US$12 million in 2013, a slight decrease from the US$14 to US$15 million allocated annually from 2005 to 2012.
The grants program receives on average 90 to 100 applications a year. Though Imosili et al. note that applicants are US-based essentially, foreign public and private entities are also eligible to apply under an active Investigational New Drug Application (IND) or Investigational Device Exemptions (IDE). In fact, the program funds a number of grants concerning multiple international sites.
Based on scientific and technical criteria, applications are reviewed and evaluated by an independent panel of experts. This independent review process avoids funding projects that are not regulatory sound or likely to meet market approval. Out of 567 awarded grants, the program’s funds have contributed to the authorization of 34 drugs, 9 biologics and 8 devices, representing around 10% of orphan products. Imosili et al. highlight that incentives such as the FDA’s grants program are effective to foster research on rare diseases. Grants provide a significant source of funding for investigators with limited resources. While FDA’s Orphan Products Grants Program is one of the main programs to fund rare disease clinical trials, others such as NIH’s Therapeutics for Rare and Neglected Diseases (TRND) program and IRDiRC aim to accelerate the development of drugs and diagnostic tools for rare diseases.