a. What is a rare disease?

The Orphan Drug Act defines a rare disease as a disease or condition that affects less than 200,000 people in the United States.

b. What is the Orphan Drug Act?

The Orphan Drug Act is a law passed by Congress in 1983 that incentivizes the development of drugs to treat rare diseases. Companies and other drug developers can request orphan drug designation and FDA will grant such designation if the drug meets specific criteria. Orphan designation qualifies sponsors for various incentives, including:

• Tax credits for qualified clinical (in humans) testing
• Waiver of the Prescription Drug User Fee (currently at almost $3 million for a new drug)
• Potential 7 years of market exclusivity after approval

In addition, the Orphan Drug Act established the Orphan Product Grants Program to provide funding for developing products for rare diseases or conditions.

c. What is an orphan drug?

An orphan drug is a drug for a rare disease or condition. Some rare disease treatments have been “orphaned” or discontinued because there was not enough financial incentive to continue development or production. The Orphan Drug Act incentivizes drug development for rare diseases.