The next time your doctor writes you a prescription, consider this: The medication may not be approved for your specific condition or age group.
But you probably shouldn't call the medical board. The practice, called "off-label" prescribing, is entirely legal and very common. More than one in five outpatient prescriptions written in the U.S. are for off-label therapies.
Medicine is rigorously tested for safety and effectiveness before becoming available to the consumer. In the U.S., the FDA makes sure this happens. Once on the market, the FDA, along with the makers of the drug, continue to monitor the medicine for any unforeseen problems. Should an issue develop, or the safety of a medication come into question, a recall may be initiated.
"Off-label" means the medication is being used in a manner not specified in the FDA's approved packaging label, or insert. Every prescription drug marketed in the U.S. carries an individual, FDA-approved label. This label is a written report that provides detailed instructions regarding the approved uses and doses, which are based on the results of clinical studies that the drug maker submitted to the FDA.
“Many people may be surprised to know that the FDA regulates drug approval, not drug prescribing, and ... doctors are free to prescribe a drug for any [reason they think is medically appropriate],” says G. Caleb Alexander, MD, MS, a medical ethics advocate and assistant professor of medicine at the University of Chicago Medical Center. "Off-label use is so common, that virtually every drug is used off-label in some circumstances."
Unaware of Off-Label Usage
Despite the prominence of off-label drug use, experts say few patients are aware that they are receiving a drug off-label. And doctors are not required to tell a patient that a drug is being used off-label.
Atlanta suburbanite Michelle Murphy was stunned to learn nadolol, the beta-blocker medicine she had been taking every day for several years to prevent migraines, was not actually approved for that use.
"It’s almost like we are test subjects, being reassured that everything will be fine because it worked to help people in studies that were taking it, but not exactly for what we are taking it for," Murphy says.
Benefits of Off-Label Drug Use
Off-label prescribing isn't necessarily bad. It can be beneficial, especially when patients have exhausted all other approved options, as may be the case with rare diseases or cancer.
According to the American Cancer Society, cancer treatment often involves using certain chemotherapy drugs off-label, because a chemotherapy drug approved for one type of cancer may actually target many different types of tumors. Off-label use of a drug or combination of drugs often represents the standard of care.