The goal of FDA's Office of Orphan Products Development (OOPD) Orphan Products Clinical Trials Grants Program (formerly known as Orphan Products Grants) is to support the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products. Applicants must include in the application's Background and Significance section documentation to support the estimated prevalence of the orphan disease or condition (or in the case of a vaccine or diagnostic, information to support the estimates of how many people will be administered the diagnostic or vaccine annually) and an explanation of how the proposed study will either help gain product approval or provide essential data needed for product development.
Orphan products clinical trials grants are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases/conditions. Since the program’s inception in 1983, OOPD has received over 2500 applications (generally, about 100 applications/year), reviewed over 2200, and funded over 590 studies. The Orphan Products Clinical Trials Grants Program has been used to bring more than 55 products to marketing approval.
Grants ensure that product development occurs in a timely manner with a very modest investment. In general, OOPD grant funding lasts for three-four years. At any one time, there are typically 60 to 85 ongoing grant-funded projects. A major portion of the appropriated funds (typically approximately $15 million) for a given fiscal year go towards continued funding of prior approved grants. The rapid increase in the cost of clinical trials in recent years has precluded an increase in the number of new OOPD grants. OOPD usually funds between 12-18 new grants per fiscal year. OOPD conducts site visits to grantees to ensure extramural funded studies, which involve human subjects, are consistent with grant agreement terms and minimize FDA’s exposure to risk of violations in human subjects’ protection requirements.
Please use our website which provides helpful information on our program and useful tips when applying for a grant. Our grant application process is similar to NIH, however, there are many differences as discussed on these web pages.