Scientific Publications by FDA Staff

Expert Opin Orphan Drugs 2014 Aug;2(8):807-15

Fueling the development of products for rare diseases: the impact of the FDA's orphan products grants program.

Imoisili MA, Mueller C, Needleman KIM

Introduction: The US Congress enacted the Orphan Drug Act in 1983 to provide incentives to accelerate the development of products for the treatment, prevention or diagnosis of rare diseases and conditions. The support of clinical research continues to be an important incentive to assist in finding treatments for rare diseases. Our aim was to provide a descriptive analysis of the US FDA's Orphan Products Grants Program and its impact on bringing products for rare diseases to market. Areas covered: Data from the Program was analyzed from 1983 to the end of 2013. The main outcome measures included the yearly dollar amount invested in the Program in actual dollars and adjusted for inflation; the number of grant applications submitted and awards made overall and by year; the product types investigated and their therapeutic class; and grants awarded which have led to marketing approvals of products, by therapeutic class and indication. Expert opinion: Over $320 million have been awarded to fund clinical studies through the Program. The amount of funding appropriated was $500,000 in 1983 and has remained constant at $14 - $15 million since 2005; $6 - $8 million when adjusted for inflation. Funding received from the Program has served an unmet medical need and has contributed to approximately 10% of orphan product approvals in the US. This and many other programs are important to assist in finding treatments for rare diseases.