An orphan drug is a pharmaceutical agent that is developed specifically to treat a rare disease or medical condition. There are many key factors, as well as, FDA rules and regulations to be aware of when developing an orphan drug.
1) Summary of the Orphan Drug Act
The Orphan Drug Act of 1982 was designed to promote the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. The Orphan Drug Act defines a disease or condition as “rare” if it affects fewer than 200,000 people in the US, or if the cost of developing a drug and making it available in the United States for such diseases or conditions will exceed any profits from its sale in the United States. The resulting Orphan Drug Regulations (21 CFR Part 316) outline the procedures and requirements for submitting requests for designation of a drug for a rare disease or condition.
2) Incentives for Developing Orphan Products
- 7-year marketing exclusivity to sponsors of approved orphan products, with an additional 6 months for a pediatric indication.
- 50% federal tax credit for expenses incurred in conducting clinical research within the US (tax credits may be applied to prior year or applied over as many as 20 years to future taxes).
- Waiver of PDUFA (Prescription Drug User Fee Act) fees for orphan drugs.
- Qualify to compete for research grants from the OOPD (Office of Orphan Products Development) to support clinical studies for developing orphan drugs.
- Eligible to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan.
3) Eligibility of a product for orphan drug designation
Product must have a sponsor (ie, company, individual, institution, government agency). A sponsor may request orphan drug designation of a previously unapproved drug, or of a new orphan indication for an already marketed drug.
A sponsor of a drug that is otherwise the same drug as an already approved orphan drug may seek and obtain orphan drug designation for the subsequent drug for the same rare disease or condition if it can present a plausible hypothesis that its drug may be clinically superior to the first drug.
>More than one sponsor may receive orphan drug designation of the same drug for the same rare disease or condition, but each sponsor seeking orphan drug designation must file a complete request for designation.Sponsor’s product cannot have approval under a New Drug application (NDA), Biologic License Application (BLA) or Premarket Approval (PMA) application for the disease or condition for which orphan status is requested. A marketing application for the product has not been filed prior to filing the orphan designation application.
4) Content of a Request for Orphan-Drug Designation
The FDA issued 13 proposed amendments to the Orphan Drug Regulations on October 19, 2011, intended to clarify some of the regulatory provisions and address issues that have arisen since implementation. Public comments about the proposed amendments were collected through January 17, 2012, and 12 comments were provided by pharmaceutical companies, trade associations, attorneys, health care organizations, and individuals. These amendments and the public comments are currently under review by the FDA.