What is off-Label Drug Use?

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  • Written By: Mary McMahon
  • Edited By: Bronwyn Harris
  • Last Modified Date: 28 November 2019
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When a doctor prescribes a drug for a condition which it has not been approved for, this is known as off-label drug use. Around the world, off-label use is an accepted and common practice, especially in certain medical disciplines. At times, off-label use represents an innovative and fresh approach to a condition, while in other cases it reflects the standard of care and many years of customary use. In most cases, off-label prescribing is perfectly legal, although it may not always be safe for the patient.

Before a drug is released, it goes through a lengthy testing and approval process. The drug is usually targeted at a specific condition, and the testing is designed to ensure that the drug is effective against the condition. During the approval period, the best doses and method of delivery are also determined. At the end of the testing period, the regulatory agency such as the United States Food and Drug Administration (FDA) will either approve or deny the drug application.


Once approved, the pharmaceutical company can market the drug for its approved use, which is also sometimes called an “indication.” For example, a drug company may develop a drug which is meant to target depression. Advertising for the drug can only indicate that it is effective for depression. However, doctors may discover that the drug is also effective for treating pain, in which case they may prescribe it off-label to patients who could benefit from the type of pain relief the drug offers.

Off-label drug use is especially common in pediatrics, since many drugs are tested only on adults, and not on children. Treatment of cancer also involves a great deal of off-label use, since most cancer drugs are formulated for a specific type and stage of cancer, but they may be more widely applicable. Veterinarians also engage in off-label drug use, especially when they are dealing with exotic species.

Certain drugs such as opiates are heavily regulated, and doctors may not prescribe them for off-label use. The majority of drugs, however, are perfectly legal to prescribe off-label, because regulatory agencies do not generally tell doctors how, precisely, to practice medicine, although authorities may step in in obvious cases of malpractice. Medical science advances at such a rapid rate that it can be difficult for regulatory agencies to keep up with innovations in pharmaceuticals, and many drug companies would rather not go through the lengthy testing and approval process for every potential use of a drug. As a result, as many as 60% of prescriptions may be off-label.

If a doctor prescribes an off-label drug for you, do not be afraid to ask questions about it. You should feel comfortable asking about how effective the off-label use will be, and if any studies have been published about the use of the drug in an off-label context. Be sure to ask about benefits and risks of the drug, and ask about side effects and potential drug interactions. If you are concerned about paying for the drug, you may want to find out if your insurance company covers off-label drug use or not, as some companies will not cover off-label prescriptions.


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Post 3

@Sierra02 – Physicians have been prescribing Neurontin off label for treating bi-polar disorder, drug and alcohol withdrawal, attention deficit disorder, and numerous pain disorders.

The drug was originally tested and approved by the FDA for the safe treatment of epilepsy. It was only allowed after all other treatments rendered useless.

Post 2

@Bfree – I agree with you some doctors have no choice but to use off label drugs. My doctor has prescribed medicine for me this way and they’ve always done the trick.

I’ve been taking Neurontin for the past 2 years for an extreme social anxiety disorder. This may be a wonder drug as I’ve seen it prescribed for so many different uses. I know it helped reduce my anxiety attacks because I get a little crazy if I miss just one dose and everyone around me knows it.

Post 1

I don’t doubt that there’s such a high percentage of off label medications being prescribed. Physicians and pharmacist’s who are basically out in the field in the medical industry, are the ones who have one on one contact with patients. They know which approved drugs are working and which ones are not. It’s all those approved but not working drugs that leave our physicians no choice but to offer an off label alternative.

I understand that it’s the FDA’s responsibility to ensure the public that our medications are safe for their prescribed purposes. But it seems to me, and this is only my opinion, that something needs to be done with the high cost and lengthy testing process from the FDA. The drug manufacturers have to pay a high price for approval on each drug and for every indication they want to have it approved for.

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