| gen. |
rare diseases (A rare disease presents a unique problem in treatment because the number of patients with the disease is so small (fewer than 200,000 in the United States) that it is not worthwhile for companies to go through the lengthy and expensive process required for approval and marketing. Drugs produced for such cases are made available under the Orphan Drug Act of 1983, which was intended to stimulate the development of drugs for rare diseases. More than 400 orphan drugs have been designated, but there are about 5,000 rare diseases that remain without treatment. Britannica Alexander Demidov) |
