Understanding Off-Label Drugs
The FDA approves medications to treat specific diseases. But when a drug is used to treat a condition it's not approved for, it is called "off-...

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Picture of woman looking at label Understanding Off-Label Drugs

Beth suffers from migraines. Her doctor gave her a prescription that he said would help her condition. Beth carefully read the pamphlet that came in the pill box. The insert said the drug is used to treat high blood pressure. But she doesn't have high blood pressure. Why did her doctor give her this drug?


Off-label prescribing
Beth's doctor is using the drug "off-label." This means that the drug has been approved by the Food and Drug Administration (FDA) for a certain disease, but it was prescribed for a different, unapproved reason. Beth's medication is approved to treat high blood pressure, but is used off-label to treat migraines.

Doctors prescribe off-label drugs often. In fact, it's estimated that one out of every five prescriptions written today is for an off-label drug.

The approval process
Before a drug can be sold, it has to be deemed safe and effective by the FDA for treating a specific disease. After clinical trials and a long testing process, the FDA approves the drug. The FDA then works with the drug manufacturer to create a "drug label." This is not the same drug label that's attached to the pill bottle or box. This drug label is used by doctors and it gives detailed information about the medication. It includes:

  • Medical condition(s) it's approved to treat.
  • Approved dosages.
  • How it should be taken. For example, by mouth (orally) or applied to your skin (topically).
  • Approved groups. Many drugs are not safe for children or pregnant women.

When a medicine is prescribed for a different reason, dose, route or group from what is stated on the drug label, it is being used off-label. Prescribing drugs off-label is legal, but is not regulated by the FDA.

Why off-label?
Experts are constantly finding new uses for existing drugs based on medical studies.

Off-label drugs can be life-saving. For example, some chemotherapy and cancer drugs work well on many types of tumors. Most doctors only choose an off-label medicine when they are confident it is safe and will work for your condition. Doctors may also prescribe off-label medicines for rare illnesses, when there is no drug approved to treat the condition.

Often the prescribed drug is an older, generic medication whose off-label use is backed by medical evidence (but not always.) Your doctor bases this treatment decision on a network of knowledge:

  • His or her own experiences with the medication
  • What colleagues have reported
  • Studies in medical journals and presentations at professional meetings

Safety concerns
Some experts say off-label drug use is controversial. Since drugs are not studied to treat other conditions, there may be more possible risks when they are taken off-label.

If your doctor gives you a prescription, ask if it's for an off-label drug. If it is, follow up with these questions:

  • Is there medical evidence that supports the use of this drug for my illness?
  • Is this medicine more effective than the drug approved to treat my condition?
  • What are the risks and benefits for taking this medicine?
  • Does my health insurance cover it? Some insurance companies will not pay for a drug if it is used for a different reason than what is stated on the drug label.

Medication safety still stands when taking off-label medicines:

  • Only take an off-label drug when it's prescribed by your doctor.
  • Always take all medicine as prescribed.
  • Tell your doctor all prescriptions, over-the-counter drugs, vitamins and supplements you take.
By Jenilee Matz, MPH, Staff Writer
Created on 02/17/2005
Updated on 02/03/2010
Sources:
  • Agency for Healthcare Research and Quality. Off-label drugs: what you need to know.
  • Poole S, Dooley M. Prescribing. In: Walsh D, Caraceni AT, Fainsinger R, Foley K, Glare P, Goh C, et al., eds. Palliative Medicine, 1st ed. Philadelphia, PA: Saunders Elsevier; 2009.
  • American Cancer Society. Off-label drug use.
Copyright © OptumHealth.
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