When the doctor pulls out the prescription pad, most Americans assume the medicine has been approved by the United States Food and Drug Administration (FDA) for this particular condition. However, that may not be the case. The doctor may be prescribing it for what is called "off-label use."

Prescribing Off-label

Off-label drug use can mean prescribing a drug:

• For a condition for which it has not received FDA approval
• For a different population group, such as children instead of adults
• At a different dose or duration than was determined by the FDA to have a favorable risk-benefit ratio

"Consumers should be told when they are receiving a drug for off-label use and make an informed decision if they want to accept the risk," says pharmacist Larry Sasich.

Getting Approval    TOP

When seeking approval for a new drug, manufacturers submit results from a series of clinical studies, indicating a specific use for the drug in a specific group of people (such as women or people with diabetes). The agency only approves a drug to treat conditions specified in the research.

Although the FDA reviews the scientific data very carefully, some side effects will not show up until very large numbers of patients start taking the medicine. This is far more people than could ever be studied prior to approval.

After a drug has been on the market for awhile and new uses become identified, the drug company can conduct scientific research to confirm the safety and effectiveness of the new use and apply for a supplemental approval.

Often long before this happens, however, doctors begin to experiment with the use of medicines for other purposes that seem logical, often based on analogies to similar drugs. For example, if one antidepressant is approved as a treatment for obsessive-compulsive disorder, doctors may try other antidepressants for the same purposes, even if they have not been approved for that use. This type of informal use may lead to formal research.

This is an accepted practice. However, it has some significant problems. One of the most significant is that doctors may incorrectly come to believe that a drug is effective for a given condition. Only double-blind, placebo-controlled studies can distinguish between expectation and the power of suggestion, and specific benefits. Both physicians and patients may incorrectly attribute benefits to a drug simply out of enthusiasm. The net effect is that the patient is placed at risk for side effects without real chance for benefit.

Benefits Versus Risks    TOP

Sometimes off-label use is the best possible treatment for a patient. For instance, a chemotherapy drug approved to fight one type of cancer may be given to someone with a different cancer. In this life-threatening situation, waiting for the additional research is not in the patient's best interest, and the potential benefits outweigh the risks.

While off-label prescribing is generally considered safe when done by competent doctors, it has also contributed to some well-publicized harms. For example, fenfluramine and dexfenfluramine, two popular and effective anti-obesity drugs, were withdrawn from the market after reports suggested that the drugs could lead to heart valve disease. While off-label prescribing was not the only issue, this example illustrates how the FDA approval process does not guarantee protection from serious adverse events.

What Is a Consumer to Do?    TOP

Here are some tips for the next time your doctor prescribes a medicine:

Ask about risks and benefits. —Ask your doctor if the drug has been approved for your condition and about the potential benefits and risks involved.
Talk to the pharmacist. —Sasich recommends that consumers ask the pharmacist to review the package insert with them before filling the prescription.
Do some research. —Use reputable sources to find information about your medicine. Check for three things: whether your condition is an approved use, what the proper dose is, and what the proper duration of use is. You should also check if there are any known interactions with drugs you regularly take.
Inquire about other options. —If you are not comfortable with a drug that has not been approved by the FDA for your condition, tell your doctor. Ask why she thinks you need this particular drug and if there is a similar drug that has been approved that you can take.