On signing the Orphan Drug Act into law on January 4, 1983, President Ronald Reagan lamented, “I only wish with the stroke of this pen I could also decree that the pain and heartache of people who suffer from these diseases would cease.” More than a quarter-century later, the prospect of Washington relieving that pain and heartache any time soon remains elusive. The orphan drug law served its initial purpose of facilitating R&D, and ultimately treatments, focused on rare diseases. However, as drug developers and patient advocates correctly note, much more remains to be done. Some 25 million Americans have one of the more than 7,000 diseases given orphan status. Only 2,400 drugs have orphan designations, though, and 367 have been approved, Timothy R. Coté, M.D., who until recently headed FDA’s Office of Orphan Products Development, told GEN.
