Workshop to focus on FDA process that grants orphan drug designation

The U.S. Food and Drug Administration has scheduled the third in a series of workshops on orphan drug designation for academics, biotechnology companies, and those unfamiliar with the process for Nov. 4-5, 2010, in Lansdowne, Va.

 The workshop, in collaboration with the Drug Information Association, will focus on the process used by the FDA to grant a special status, known as orphan designation, for drug products intended to treat rare diseases. Similar workshops were held earlier this year in Claremont, Calif., and Minneapolis.

Orphan drugs are either drug or biologic products used to treat diseases or conditions affecting fewer than 200,000 people in the United States. Orphan drug designation is available for drugs already approved for another disease or condition or for experimental drugs that have not been previously approved.

During the workshop, 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. FDA staff will provide one-on-one guidance to help participants develop strong applications.

 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 scientific 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 orphan drug may then be submitted for review through the FDA marketing approval process, which evaluates the drug for safety and efficacy.

 Since 1983, when the Orphan Drug Act was passed, the FDA has received 3,212 applications for orphan drug designation and has granted orphan drug designation to about 2,250 drugs, of which 361 have become approved products.