Some residents of Massachusetts may be among the 25 to 30 million Americans who have a rare disease. In the U.S., any disease that affects less than 200,000 people is considered "rare." There are about 6,000 known rare diseases in the world, and the diagnosis and treatment of these conditions can be difficult.
In order to find effective treatments for rare diseases, new drugs are constantly being developed. Orphan drugs, which are medicines that are not yet being commercially developed but are in testing, are often the best hope for treating rare diseases. Since the Orphan Drug Act was passed in the U. S. in 1983, the FDA has approved more than 500 drugs that were classified as orphan drugs.