The FDA Office of Orphan Products Development (OOPD) was created to identify and promote the development of orphan products. Orphan products are drugs, biologics, medical devices, and medical foods that are indicated for a rare disease or condition. Rare diseases, as generally defined in the US Orphan Drug Act (ODA), are diseases or conditions with a prevalence of fewer than 200,000 persons in the US. Approximately 30 million Americans are affected by 7,000 known rare diseases but only a few hundred of these rare diseases have approved treatments. OOPD provides incentives for sponsors to develop products for rare diseases.
OOPD Clinical Trials Grants:
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. The Orphan Products Grants Program has been supporting clinical trial research since 1983 and has facilitated the marketing approval of more than 60 products. OOPD funded grants ensure that product development occurs in a timely manner with a very modest investment. At any one time, there are typically 60 to 85 ongoing grant-funded projects. A major portion of the appropriated funds (typically approximately $15.5 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 conducts ongoing grant evaluations to ensure extramural funded studies, which involve human subjects, maintain grant agreement terms and minimize FDA’s exposure to risk of violations in human subjects’ protection requirements.
To address the remaining unmet need and the lack of treatments for the majority of rare diseases, FDA is focusing their efforts with a new funding opportunity to facilitate and move new therapies along in drug development in safe yet efficient means by encouraging innovative clinical trial methods such as adaptive and seamless trial designs, modeling and simulations, and basket and umbrella trials. These methods are vital to efficient trials and data evaluation which can expedite drug development.