Is it legal for a doctor to prescribe an "off-label" medication?

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Q: My father saw a doctor about frequent pain, and was prescribed an "off-label" prescription. The doctor said the drug hasn't actually been approved to treat this problem but was okay. Is this proper? Legal?

A: "Off-label" prescriptions are certainly legal. Once the Food And Drug Administration (FDA) has approved a medication for one purpose, doctors may then prescribe it for other purposes. Whether or not an off-label use of a medication is appropriate depends on a number of factors which your father's physician has likely considered.

By permitting off-label prescriptions, the FDA enables medications to be used without subjecting each new use of a product to the lengthy and rigorous test procedures mandated in the agency's initial approval process. But research does not end with the approval of a medication. Interested parties may subsequently fund further studies to assess whether the newly approved drug might have benefits other than those for which it was approved. These other benefits are referred to as "new uses."

Until roughly two years ago, it was illegal for a pharmaceutical company to advertise or promote off-label uses of their products. If a doctor inquired about off-label prescribing, the company could answer--but could be fined heavily if it offered such information on its own.

Since passage of the Food and Drug Administration Modernization Act of 1997, however, manufacturers of drugs, biologics (products derived from living sources, such as vaccines) and FDA-regulated devices could disseminate certain information regarding off-label use of their products so long as they satisfied a number of criteria, including the following:

  • The information cannot be false or misleading
  • Information is comprised of unabridged reprints or copies of articles (referencing scientifically conducted studies) published in professional publications such as medical journals
  • The FDA is given 60 days prior to the information's dissemination to review it, requiring the manufacturer to include additional objective information along with whatever is disseminated
  • The product's manufacturer has both applied for formal FDA approval of the off-label usage, and has completed whatever studies might be needed to support the application.

The recent fen-phen tragedy is one example of an off-label use gone awry. This case involved doctors' off-label prescribing of the diet fenfluramine and phentermine in combination that may have resulted in heart-valve damage for some patients As a result of this unfortunate situation, many critics renewed their longtime objections to all off-label prescribing. However, off-label prescribing such as the common use of amitriptyline for chronic pain, although initially approved for treatment of depression, demonstrates the benefit such use can provide. Off-label prescribing may even save lives by, for a period of time, bypassing the lengthy and complex FDA approval process.

How can you protect your father and yourself? Ask your doctor, whenever a new drug is prescribed, whether the drug has been approved for that purpose. If not, do not hesitate to question him about how safe and effective the particular off-label use has been, and how long the drug has been prescribed off-label for this purpose. After all, it may take years of use before problems are recognized.

If you need another opinion, consult a trusted pharmacist or another physician. Keep in mind the seriousness with which your father's doctor considers prescribing off-label. This is because the physician, depending on what research exists to support the off-label use, may be legally going out on a limb when he prescribes off-label.

Further background about a medication and its uses is attainable through your own research, but above all remember: if it's not an emergency situation, anytime you are uncomfortable with a drug, whether or not it is being prescribed off-label, learn about all other alternatives before accepting the prescription.


John Connolly is President and CEO of Castle Connolly Medical Ltd., the trusted source for finding top doctors. Read his full biography

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