Are You Taking an Off-Label Medication?

Off label drug use is way too common

/ Author:  / Reviewed by: Robert Carlson, M.D

Off-label drug use happens when a drug is used for a condition for which it does not have approval from the Food and Drug Administration (FDA). Doctors may give an off-label prescription for many reasons.

A recent report by Mayo Clinic doctors explains that off-label use is common and patients may not know they are taking a drug off-label. Doctors and patients can work together to be informed about all the risks and benefits of any medication.

What is off-label use and when does it happen?

Medications must be approved by the FDA for each illness or condition. Clinical trials provide guidance to the FDA so they can assess the drug for safety and effectiveness in specific conditions.

Not only is approval needed for each condition, but a drug's safety and effectiveness must be assessed for different demographic groups. For example, a drug must be tested in adults, children and pregnant women separately in order to be approved for use in those groups.

Any time a doctor prescribes a medication for an illness, condition or person that does not have FDA approval, it is an off-label use. 

Off-label use is common. A study in 2006 looked at commonly used medications and found that about one in five were prescribed for off-label uses.

Patients may be using a drug off-label without even knowing it. Current laws do not require that a doctor tell the patient that the use is off-label. 

Why and how do drugs get prescribed off-label?

There can be many reasons a doctor may choose to prescribe a drug off-label.  Research on off-label uses for drugs is often published in scientific journals. 

Doctors can learn about other uses for a drug through these reports. They can take what is known about the drug and apply it to other conditions or groups of people.

For example, most medications are not tested in children, even though children may suffer from the illnesses they are designed to treat.

Morphine is a classic example. It is approved for use in adults but is also commonly used in children to treat pain. In this case, off-label use gives doctors a valuable tool to relieve pain in children.

Why aren’t drugs approved for all their uses?

Getting FDA approval can be costly. Money often limits who seeks FDA approval for new conditions and groups of people.

Generic drug manufacturers may not have the money to seek FDA approval. For rare conditions even the companies that have the money are unlikely to spend it seeking approval from the FDA.

This is why some off-label uses never gain FDA approval.

What are the benefits of off-label use?

Off-label use of medications allows doctors to treat a patient’s individual needs. Doctors can take an array of symptoms and conditions and decide which medication will be most helpful for a person.

If drugs could only be used for approved uses, people might have trouble getting treatments they need.

Also, off-label uses for drugs can lead to discoveries about new uses for a drug.  

What are the risks of off-label use?

Off-label uses may have not been tested in large groups of people. 

Less is known about the safety and effectiveness of the drug for an off-label condition.

Sometimes studies about off-label uses of a drug are funded by the companies that make the drug.  In some cases, these studies are small and may be very favorable for the new use of the drug.

What should you be doing?

As a patient, the most important thing to do is talk to your doctor and pharmacist about any drugs you are taking.

Your doctor can tell you about the approved uses of a drug and explain why she is prescribing it to you. Your pharmacist can also give good information about the drug and its possible side effects.

The FDA website ( also has valuable consumer information to help you stay informed.

In a recent press release, Christopher Wittich, MD, at the Mayo Clinic said, “Healthcare providers and patients should educate themselves about off-label drugs to weigh the risks and benefits before a physician prescribes one or a patient takes one.” 

Review Date: 
August 6, 2012