SILVER SPRING, Md., Oct. 13 /PRNewswire-USNewswire/ — 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