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.
What are off-label medications?
This means it 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 an antidepressant medication 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 different dosage form than approved. For example, a medication that is approved for use with an oral tablet but prescribed with an oral solution.
Why are medications used off-label?
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.
How does a doctor decide to use a medication off-label?
Once a medicine is approved for one use, doctors can decide whether it is right for other uses. Several factors are considered when a physician decides to prescribe a medication. This includes deciding whether the medication is safe and effective for a specific patient.
Is it safe to take off-label medications?
In most cases, taking a medication off-label 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. And sometimes a medication could help with symptoms of conditions that it is not approved for. There are many off-label uses that most doctors agree are safe and effective.
Off-label use means that an approved drug is used in a way that is not approved by the FDA.
This may include:
- use of a medication for an unapproved indication or disease
- use of a medication at an unapproved dose
- use of a medication in an unapproved dosage form
Consider asking your doctor about off-label use of medications that you are prescribed.
©2017 The College of Psychiatric and Neurologic Pharmacists (CPNP). Aimee Patterson, PharmD, January 2018
This information is being provided as a community outreach effort of the College of Psychiatric and Neurologic 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 College of Psychiatric and Neurologic Pharmacists disclaims any and all liability alleged as a result of the information provided herein.
To view the references for this resource, please visit cpnp.org/366992.