A drug used to treat, prevent, or diagnose an orphan disease. An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan diseases are often serious or life threatening. In 1983, the U.S. government passed a law, called the Orphan Drug Act, to give drug companies certain financial benefits for developing orphan drugs that are safe and effective. In the European Union (EU), a disease is defined as rare if it affects fewer than 5 in 10,000 people across the EU.
