Off-Label Drug Use: What You Need to Know

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.

"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.   

Beta-blockers are another example of beneficial off-label prescribing. Such medications are FDA-approved for the treatment of high blood pressure, but are widely recognized by cardiologists as a standard of care for patients with heart failure. And in fact, some beta blockers are now formally approved to treat heart failure. It's not uncommon for off-label uses to eventually get approved by the FDA.

Other drugs commonly prescribed off-label include tricyclic antidepressants for chronic pain, and antipsychotics for attention deficit hyperactivity disorder (ADHD) "Name the drug, and one can come up with off-label uses," Alexander says.