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Off-label drugs and medical devices: Get the facts

Drugs and medical devices are sometimes used off-label. See what it means when you're prescribed a drug or medical device for off-label use.

By Mayo Clinic staff

You've heard the term "off-label" used to describe the drug you've been prescribed. Or maybe you had a stent inserted after your heart attack and now are reading that some stents are used off-label.

You might be wondering what off-label means, and whether it's safe to use a drug or medical device described as off-label. Off-label use of drugs or medical devices isn't unusual. By some estimates, more than one out of every five drugs prescribed in the United States is for off-label use.

Don't be alarmed. It's usually safe and effective to use drugs or medical devices off-label. However, it pays to get informed about just what the drug or device your doctor prescribed is supposed to do.

What does off-label mean?

There are two basic ways a drug or medical device can be used:

  • Approved use. When a drug or medical device is used to treat a specific condition or disease described in its label — and prescribed in a way that follows those guidelines — its use is said to be an "approved" use, or on-label use. The Food and Drug Administration is responsible for drug approvals in the United States.
  • Off-label. When a drug or medical device is used to treat a disease or condition not listed on its label, or used in such a way that's not outlined in the label, it's said to be used off-label. This off-label use is also sometimes referred to as extra-label use, nonapproved use or unapproved use.

How the drug approval process works

When discussing off-label use, it's helpful to first know how a drug or medical device gets approved in the United States. Before a drug is marketed, it goes through three stages of research known as clinical trials. These trials are designed to prove whether a drug treats a specific condition effectively, does what it's supposed to do and is safe when used as directed.

A federal agency called the Food and Drug Administration (FDA) is responsible for evaluating new drugs. The FDA decides whether a drug is safe and effective for treating a specific condition. The drug or device manufacturer, along with the FDA, comes up with the label (or in the case of a device, a device label).

The label doesn't refer to the actual material on the drug package. Instead, it refers to information about the drug that is used as a guide to doctors and other health professionals. Information on the drug label includes:

  • Mechanism of action. This refers to how the medication works in the body.
  • Uses. Also called indications, this refers to what the drug has been approved to treat. This also includes the group of people, such as adults, children, men or women, who can use the medication.
  • Dosage. How much of a drug to give and how often.
  • Adverse effects. Also called side effects, this refers to what symptoms may occur from taking the medication.

Label information is usually lengthy. You'll receive a condensed version — often in the form of a patient information sheet when you get your prescription. Over-the-counter medications typically contain labeling information on the package itself.

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Oct. 1, 2007

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