Orphan drug

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Orphan drug is a designation by the United States FDA for a drug that is used to treat, prevent, or diagnose an orphan disease.

Orphan disease

An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States.

Importance

Orphan diseases are often serious or life threatening.

US law

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.

Partial list of orphan drugs


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