FDA provides guidance on applying for orphan drug designation

As part of its continuing effort to make the agency more transparent and accessible, the FDA has scheduled a series of workshops about orphan drug designation for academics, biotechnology companies, and those unfamiliar with the process. The FDA can grant a special status, known as orphan designation, for drug products intended to treat rare diseases.

Orphan drugs are either drug or biologic products used to treat conditions affecting fewer than 200,000 people in the United States. Orphan drugs may be already-approved or experimental drugs.

 The workshops will be held Feb. 25-26, 2010, in Claremont, Calif., and Aug. 3-4, 2010, in Minneapolis. At the workshops, participants will propose a specific drug for a specific rare disease and work on an orphan designation application to submit to the FDA at the conclusion of the workshop. To help participants develop strong applications, FDA staff will provide one-on-one regulatory help.

To obtain orphan drug designation, drugs must be for the treatment, prevention or diagnosis of a rare disease or condition. Designation also requires there be a medical rationale for expecting the proposed drug to be effective in the treatment, prevention, or diagnosis of that disease or condition.

Orphan designation qualifies the applicant to receive certain benefits, such as tax credits and marketing incentives, from the federal government in exchange for developing the drug. After designation, the drug must then be submitted for review through the FDA marketing approval process, which evaluates the drug for safety and efficacy.

http://www.fda.gov