What does the Orphan Drug Act do?

Congress passed the Orphan Drug Act of 1983 to stimulate the development of drugs for rare diseases. A rare disease is defined as a disease that affects fewer than 200,000 people in the United States.

How long does orphan drug designation last?

The purpose of the designation was to create financial incentives for companies to develop new drugs and biologics for rare diseases. These incentives include a partial tax credit for clinical trial expenditures, waived user fees, and eligibility for 7 years of marketing exclusivity [1].

How do I find out my orphan drug status?

Requests for an orphan drug designation can be submitted through the FDA Form 4035. The FDA will complete a review of the orphan drug designation request within 90 days of its receipt.

Who is responsible for Orphan Drug Act development?

The Orphan Drug Act and the Development of Products for Rare Diseases The Orphan Drug Act and the Development of Products for Rare Diseases Mathew T. Thomas, MD Office of Orphan Products Development Food and Drug Administration Telephone: 301-827-3666 Email: [email protected] The Office of Orphan Drug Development

Is there such a thing as an orphan drug?

Orphan Drugs. For rural referral centers, sole community hospitals, critical access hospitals, and free-standing cancer hospitals participating in the 340B Program, the term “covered outpatient drug” does not include a drug designated by the Secretary under section 526 of the Federal Food, Drug, and Cosmetic Act for a rare disease or condition.

How are clinical testing expenses determined under the Orphan Drug Act?

CLINICAL TESTING EXPENSES FOR CERTAIN DRUGS FOR RARE DISEASES OR CONDITIONS For purposes of section 38, the credit determined under this section for the taxable year is an amount equal to 50 percent of the qualified clinical testing expenses for the taxable year.

Is the 340B program required to provide orphan drugs?

Therefore, manufacturers are not required to provide these covered entities orphan drugs under the 340B Program. A manufacturer may, at its sole discretion, offer discounts on orphan drugs to these hospitals.