Off-Label Drug Use: What Does It Mean?

Posted on: January 27th, 2016 by Risa Blair, M.S. Ed., MHC 1 Comment
What Is Off-Label Drug Use

Off-label drug use refers to the practice of prescribing medication for purposes, populations, or in doses not approved by the FDA. Although the FDA must clear all new medications in order for them to be marketed, once a drug becomes available it can be used for non-approved reasons, as long as a medical professional determines it to be safe. Drugs may be prescribed for non-approved uses in desperate circumstances and may offer hope where other drugs have failed, or a particular drug may not have been tested in a specific population. Despite the dangerous side effects that may result from off-label drug use, prescribing medications for non-approved reasons is a fairly common practice. According to a 2006 study, 21% of common prescriptions were being taken for reasons not approved by the FDA.

A similar 2007 study reported that nearly 80% of children that were discharged from hospitals were taking at least one medication for an off-label purpose. Off-label use is so common, that doctors often prescribe medications for unapproved purposes without even knowing it.

Is it Legal?

Thanks to the Food, Drug, and Cosmetic Act of 1938, the FDA is the regulating authority over promotional materials for medications. In most cases, marketing and pharmaceutical companies are not permitted to promote their drugs. As previously mentioned, the FDA must approve all medication labels before the products become commercially available, which include safe use and scary side effects. Brochures, television, radio, and print advertisements, and other educational materials are all considered to contain labeling information. Including misleading information, intentionally omitting data, and promoting medications for off-label uses are considered “misbranding”, and are not allowed. Drug manufacturers may respond, however, to unsolicited inquiries from medical professionals regarding non-approved uses, as well as provide informational literature that discusses off-label uses (source).

Sometimes pharmaceutical companies intentionally conceal valuable information so that they may continue to expand their market and profit even more. In 2012, GlaxoSmithKline paid a $3 billion fine in a settlement that resulted from, among other shady things, their marketing of Wellbutrin and Paxil for off-label purposes (including to treat depression in children).

Why is it Important for a Drug to be Approved by the FDA?

Once a pharmaceutical company submits proof to the FDA that their drug has been tested and is effective and safe, the FDA’s Center for Drug Evaluation and Research experts review this information and determine if it can then become available to doctors and consumers. If the FDA didn’t oversee large drug companies, there would be less quality control and a greater chance of corruption being overlooked. Without sufficient proof that a medication can be successful, it is difficult to determine if the benefits will outweigh the risks, what those risks are, and what warning signs to look out for.

How do I Know if I’m Taking a Medication for a Non-approved Use?

You can always ask your doctor if the medication that he or she has prescribed you has been approved for that purpose. If they don’t know, ask your pharmacist. If you are to take it for an off-label use, ask if there have been well-designed trials that prove the treatment is effective for individuals with your diagnosis. You should also ask your doctor if there are any medications available that have already been approved for your condition.

You may also visit and search for your prescription. Once you’ve reached the medication page, check out the “Indications & Usage” section to see if the drug has been approved to treat your medical condition or search Recall Guide for this and additional information.

Additional Resources

About Author

Risa Blair, M.S. Ed. has written for various professional websites including While attending the Mental Health Counseling Graduate Program at The College of Saint Rose in Albany, N.Y., she worked as a research assistant at the Institute for Community Research and Training. Additionally, Risa has also worked as a research assistant for the Psychology Department at the University of Albany, where she studied the effects of drugs, addiction, social policy, and their influences on behavior and health. Risa also enjoys oil painting, cooking, and reading.


  1. DanielShaw1

    Thoughtful, thorough and well researched. Also fully up to date, highly relevant and well written. Unique style; scholarly yet highly readable. Ms. Blair, I waited several days to visit this site. I knew you had it in you to write like this. But now that I have an account, I’ll consider this my first “Go To” site for this kind of information. Great work!


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