Sometimes a medication will be approved for use by the Food and Drug Administration (FDA) for a particular condition or patient group, but will also show success in treating another condition or group. Until the medication is approved by the FDA for treatment of this additional condition or group, this new use is called an “off-label use.” An off-label use is any use of the medication that is not specifically mentioned in the FDA approval labeling (including use for a different condition, different dose, or in a different population than the one that has been approved).
For example, the medication Seroquel (quetiapine) has two FDA-approved uses: to treat schizophrenia and bipolar I disorder. However, Seroquel is also frequently prescribed to people with borderline personality disorder (BPD) in order to reduce symptoms such as paranoid thinking, anger/hostility, and impulsivity. The use of Seroquel to treat these symptoms of BPD is an off-label use.
Once the FDA has approved a medication for use in any condition, a physician can legally prescribe that medication for off-label use. It is the physician’s responsibility to be well-informed about medication, to only prescribe a medication for an off-label use if there is a good scientific or medical reason for doing so, and to track the patient’s response to the medication.
Nightingale SL. Off-label use of prescription drugs. American Family Physician. 68:425-7, 2003.
U.S. Food and Drug Administration, Center for Drug Evaluation and Research, http://www.fda.gov/cder/present/diamontreal/regappr/sld001.htm, retrieved October 22, 2008.