Off-label drugs and medical devices: Get the facts

(MayoClinic.com) 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.

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.

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.

Drugs are usually prescribed off-label when doctors discover new uses for a drug. There's nothing inherently wrong with this. Doctors may discover that a device or drug can be beneficial in more instances than just those the FDA evaluated when it was first developed.

Once a drug is approved in the United States (and in many other countries), it can legally be used by doctors to treat any condition or disease a doctor determines is medically appropriate. Some common off-label drug uses include:

• Treating a disease other than the drug is approved for
• Giving a drug at a different dose or frequency than it's been approved for
• Using a drug to treat children when its approval is limited to adults

Off-label prescribing is typically done for drugs that are older and have been on the market awhile; generic drugs are commonly used off-label. That's because after a drug has been used awhile, new uses for the drug may have been discovered.

Cancer drugs are frequently used off-label. A cancer drug is usually not approved to treat just cancer overall. Instead, a cancer drug is usually approved to treat a specific type of cancer, often at a specific stage of the cancer. However, doctors may try a drug on another type, or stage, of cancer and discover it works well. It can then become the "standard of care."

Even though new uses have been found for the drugs, drug makers seldom choose to put the already approved drug through another lengthy approval process. A drug maker can't advertise that their drug is approved to treat an off-label use unless the drug maker submits new information to the FDA for review. If information is submitted and approved, the FDA will add this new information to the drug label and it is no longer considered to be off-label. The FDA encourages drug makers to add new information like this, but doesn't require it.

While off-label use of drugs or medical devices is relatively common, some experts have raised concerns. These include:

• Lack of evidence. While there is reliable information that some drugs are effective when used off-label, for others there is little to no evidence of effectiveness. As a result, the FDA and others are concerned that some off-label use might be ineffective or even dangerous. While the FDA doesn't prevent off-label use, it doesn't promote off-label use, either.
• Reimbursement. Some insurance companies won't cover the cost of a drug — most often if it's a costly drug — if it's prescribed for off-label use. Depending on your state's laws, insurers could be required to cover care that is "medically appropriate." If your doctor can prove — through the use of peer-reviewed medical studies or other reliable information — that off-label is appropriate, that should help bolster your case in getting reimbursement.

It's helpful to have a conversation with your doctor about off-label use and its appropriateness. Some questions you'll want to ask include:

• Is there any evidence the drug you're suggesting be used off-label is effective? What is that evidence, and how reliable is it? Ask for references or information supporting the off-label use.
• Is the off-label use of this drug likely to work better than the standard or approved treatment?
• What are the potential side effects and other risks associated with this drug if I use it off-label?

Off-label use of drugs or medical devices is relatively common and can provide you with helpful options in treating your condition or disease. However, it pays to get informed about what your treatment choices are and discuss off-label treatment with your doctor.

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