What is an orphan drug?

• A. Drugs used to treat a rare disease that would otherwise be overlooked by commercial manufacturers
• B. Drugs that are not approved by the FDA
• C. Drugs with no therapeutic effect
• D. Drugs that treat common diseases with existing therapies

Answer: (A)

An orphan drug is a medication developed to treat a rare disease or condition. Because the patient population is small, drug sponsors may not see enough potential return on investment to pursue development through normal market channels. Policies like the Orphan Drug Act provide incentives—such as market exclusivity, tax credits for research, grants, and FDA support—to encourage development of therapies for these overlooked diseases. This explains why such drugs exist and are prioritized despite limited patient numbers. It isn’t about drugs that haven’t been approved by the FDA, or drugs with no therapeutic effect, or drugs that treat common diseases with established therapies.