What are the incentives for orphan drug development?

The law provides three incentives: (1) 7-year market exclusivity to sponsors2 of approved orphan products, (2) a tax credit of 50 percent of the cost of conducting human clinical trials, and (3) Federal research grants for clinical testing of new therapies to treat and/or diagnose rare diseases.

What are the benefits of orphan drug status?

Incentives of Orphan Drug Designation

• 7-year marketing exclusivity to sponsors of approved orphan products.
• 25% federal tax credit for expenses incurred in conducting clinical research within the United States.
• Waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs.

What is the FDA fast track for drug approval?

Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.

How do you qualify for orphan drug designation?

Criteria for Orphan Drug Designation

1. The product must be intended for use in a rare disease or condition.
2. Adequate documentation or prevalence data must demonstrate that the intended condition is rare.

What does it mean when a drug is designated as a Fast Track drug?

Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.

What is orphan status of a drug?

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.

What are the tax incentives for orphan drugs?

Tax incentives: The Orphan Drug Tax Credit (ODTC): The sponsors, after obtaining orphan drug designation, can collect tax credits for expenses incurred while conducting a clinical trial. User fee: Orphan drug products are exempt from the usual new drug application or user fees charged by the FDA.

How does the Orphan Drug Designation program work?

The Orphan Drug Designation Program provides for research grants, tax credits for clinical research, and protocol assistance for the development of drugs for rare diseases and disorders. The program also provides marketing exclusivity for approved orphan drug products.

How are economic incentives used in drug development?

Economic incentives are granted post-approval to reward innovation in drug development. FDA’s Office of Orphan Products Development (OOPD) Grants Program supports the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy.

Can a generic drug be approved during an orphan market?

During the orphan market exclusivity period, the FDA cannot approve a NDA (new drug application) or a generic drug application for the same product and for the same rare disease indication. The FDA could approve a second application for the same drug for a different disease indication.