Orphanage at the FDA

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In the late 1970s and early 1980s, social and political forces gave birth to a new movement to serve the needs of people with rare diseases, a movement that has only accelerated with the penetrance of internet connectivity. The FDA's Office of Orphan Products Development (OOPD) was established in 1982 to promote the development of products that demonstrate promise for the diagnosis, prevention, and/or treatment of rare diseases or conditions. Additional governmental responses included passage of the 1983 Orphan Drug Act (ODA) as well as subsequent legislation for medical devices. This article describes how OOPD assists in the development of orphan products (drugs, biologics, medical devices, and medical foods) for patients with rare diseases. The 40 clinicians, pharmacists, scientists, engineers, and others who perform OOPD's mission activities are broadly committed to providing useful new therapies to people with rare diseases. However, OOPD does not actually decide what products (drugs, biologics devices, and medical foods) are licensed for marketing and which applications are denied. Rather, OOPD has three key functions: certifying that indeed a product has bona fide promise for use in a rare disease, granting funds for the development of such products and advocating with stakeholders for the creation of new therapies in rare disease therapies.