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Orphan drug designation
Pronunciation
A status given to certain drugs called orphan drugs, which show promise in the treatment, prevention, or diagnosis of orphan diseases. 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. This law is meant to help bring more drugs to patients with rare diseases. (NCI Dictionary)
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