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
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.
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,"