A Mayo Clinic analysis revealed that even though numerous people have heard of off-label drug use, i.e. a physician prescribing medications prior to their approval by the Food and Drug Administration, they might not be aware that it applies to prescriptions they currently take.

Researchers have now asked and answered 10 questions about off-label drug use in an article featured in Mayo Clinic Proceedings.

Leading author Christopher Wittich, M.D., an internal medicine physician at the Mayo Clinic, explains:

“Since the Food and Drug Administration does not regulate the practice of medicine, off-label drug use has become very common. Health care providers and patients should educate themselves about off-label drugs to weigh the risks and benefits before a physician prescribes one or a patient takes one.”

Key points of the article include:

  • Off-label drug use is common practice. According to a 2006 report, around 1 in 5 prescriptions within a group of commonly used medications were for off-label use. Another study reports that 79% of children discharged from pediatric hospitals were taking at least one off-label medication.
  • Patients may be unaware that the drugs they have been prescribed are being used off-label. So far, there is no court decision to ensure that physicians must disclose using off-label drugs through informed consent. The FDA points out that it does not regulate the practice of medicine and that the federal Food, Drug, and Cosmetic Act of 1938 is not responsible to make physicians liable for off-label drug use.
  • Off-label drug use can become the predominant treatment for a condition. For instance, some none-FDA approved antidepressants are used to treat neuropathic pain, whilst some drugs in this class are considered a first-line treatment option.
  • Morphine, which is extensively used to treat pain in hospitalized pediatric patients is another example of widely practiced off-label drug use, whilst many inhaled bronchodilators, antimicrobials, anticonvulsants, and proton pump inhibitors also are used in children without formal FDA approval. Seeking FDA approval for a new medication can be costly and time-consuming, whilst adding additional indications for an already approved medication require another drug application, which, if approval is granted, may not be cost-effective, since the drug’s revenue may not offset the expense and effort for obtaining approval.
  • Generic medications may not have the necessary funding to conduct FDA-approval studies, which means that drug manufacturers may never seek FDA approval for a new drug indication.

Whilst it is not permitted for pharmaceutical manufacturers to promote off-label uses of medications, they are allowed to respond to unsolicited questions from health care providers and distribute peer-reviewed publications about off-label use. For instance, this year, GlaxoSmithKline agreed to pay a record $3 billion to settle a Justice Department case involving alleged off-label drug use marketing, and Merck Sharp & Dohme was fined $322 million over its alleged promotion of the painkiller Vioxx for being used off-label.

Written by Petra Rattue