What is being done to develop treatments for rare diseases?
Researchers have made considerable progress in recent years in figuring out ways to diagnose, treat and even prevent a variety of rare diseases. Still, much more remains to be done because there are no treatments for the vast majority of rare diseases. The Orphan Drug Act of 1983 provides incentives for drug companies to develop treatments for rare diseases. In the 25 years since the Act was signed into federal law, the U.S. Food and Drug Administration (FDA) has approved more than 340 treatments for rare diseases. Recently, the National Institutes of Health (NIH) launched a new effort, called the Therapeutics for Rare and Neglected Diseases (TRND) program, to create an integrated research pipeline to jump start the development of new treatments for rare and neglected disorders. The NIH Office of Rare Diseases Research (ORDR) handles oversight and governance of TRND. The laboratory work for TRND will be performed in a facility administered by the intramural program of the National Huma
