Off-label Drug Use: Is It Safe?

When the doctor prescribes a medication, most Americans assume the medication 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

Getting Approval

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.

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 use some medications 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 eventually lead to formal research.

Off-label use of drugs is usually done when the accepted drug is not effective and nothing else is available. This is also true for treating different types of patients or using different dosing and duration of use.

For example, in pediatrics the most common reason for off-label use occurs when an effective drug approved for children does not exist and the patient needs treatment. A lot of pediatric drugs were initially used in this way. You also have to bear in mind that most of the time, as noted in the example above, the other drug is tried only after and nothing else is available.

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. Both physicians and patients may incorrectly attribute benefits to a drug simply out of enthusiasm. The net effect is that the patient may be placed at risk for side effects without real chance for benefit. This kind of information gets identified in randomized trials. Randomized trials are controlled studies that can distinguish between improvement from the medication or from expectation and the power of suggestion (placebo effect).

Benefits Versus Risks

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 potential harms. This is because the drugs are being used for longer time frames than used in the original clinical studies. This can sometimes result in unintended side effects coming to light. For example, some antipsychotic drugs were being used to treat dementia symptoms in the elderly. Today this is not the case. Labels now carry strict warnings to avoid their use for this purpose because of serious health complications and death.

What Is a Consumer to Do?

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

• 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. —Consumers should ask the pharmacist to review the package insert with them before using the prescription.
• Do some research. —Use reputable sources to find information about your medication. 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 they think you need this particular drug and if there is a similar drug that has been approved that you can take.

Revision Information

• Reviewer: Michael Woods, MD, FAAP
• Review Date: 03/2017
• Update Date: 03/23/2015