Skip to main content
An official website of the United States government
Español

orphan drug

(OR-fun …)
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.
Search NCI's Dictionary of Cancer Terms