Tim Coté on Orphan Drugs and the Fight Against Rare Diseases

Dr. Tim Coté is director of the FDA's Office of Orphan Products Development. Coté's office is responsible for implementing the 1983 Orphan Drug Act, originally the brainchild of a Connecticut woman whose children suffered from a rare disease. In the years since that Act was passed, says Coté, 357 new therapies have gone to full marketing approval and another 2,100 compounds that are "promising" have been labeled as orphan drugs. The Office of Orphan Product Development also serves as an ombudsman for everyone from patients groups to industry, expediting the development of drugs to fight approximately 7,000 rare diseases, including Gaucher's, Pompe's, Muscular Dystrophy, and Cystic Fibrosis -- diseases that affect roughly 30 million Americans. But while Coté sees progress in the battle against rare diseases, he adds:"Things can't expand fast enough for the people who really need [orphan drugs].... The single most important thing is that we have to hurry because people have children who need therapies right now."