Off-Label Usage of Medications

The United States Food & Drug Administration (FDA) is responsible for regulating both prescription and non-prescription medications. One of their roles in regulation is to ensure that medications are safe and effective. This is done through the FDA approval process.

This means the medication is used in a way that is not stated in the FDA labeling. Medications may be used off-label for several reasons such as:

• For a disease or condition that it is not approved to treat. For example, using some antidepressant medications to treat nerve pain.
• At a different dose than approved. For example, a lower or higher dose of the medication may be used for some patients.
• In a different dosage form than approved. For example, a medication that is approved for use with an oral tablet but prescribed with an oral solution.¹

Off-label use of medications is common. Although common, many patients may not know that a medication is prescribed off-label. So why are medications used off-label?

One reason may be that the FDA approval process is expensive and time-consuming. If the company would like to add an indication to a medication, an additional application is required. For this reason, a medication may still be helpful for off-label use even if it is not approved by the FDA. Off-label use of medications can be particularly useful for patients who have tried all other medications for a disease. Another reason a medication may be prescribed off-label is because there might not be an approved drug to treat a disease. One example is cancer medication. Often a cancer medication may be approved to treat one type of cancer, but is used off-label to treat other types of cancers. Although a medication is not approved for a condition, it may have been studied for its benefits in that condition.^1,2

Once a medication is approved for one use, doctors can decide whether it is right for other uses. Several factors are considered when a health care provider decides to prescribe a medication. This includes deciding whether the medication is safe and effective for a specific patient.^2,3

In most cases, taking a medication for off-label use is safe. Drug companies are required to prove that a medication is safe for people to use. They just don’t need to prove the drug works for treating an off-label condition. Sometimes a medication could help with symptoms of conditions that it was not approved for. There are many off-label uses that most doctors agree are safe and effective.⁴

Provided by

(January 2023)

Aimee Patterson, PharmD, March 2019

To view the references for this resource, please visit https://aapp.org/resource/patients/offlabel.

©2022 The American Association of Psychiatric Pharmacists (AAPP). AAPP makes this document available under the Creative Commons Attribution-No Derivatives 4.0 International License. Last Updated: January 2016.​

This information is being provided as a community outreach effort of the American Association of Psychiatric Pharmacists. This information is for educational and informational purposes only and is not medical advice. This information contains a summary of important points and is not an exhaustive review of information about the topic. Always seek the advice of a physician or other qualified medical professional with any questions you may have regarding medications or medical conditions. Never delay seeking professional medical advice or disregard medical professional advice as a result of any information provided herein. The American Association of Psychiatric Pharmacists disclaims any and all liability alleged as a result of the information provided herein.