What are FDA-approved 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 is off-label medication use?
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
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.1,2
How does a doctor decide to use a medication off-label?
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
Is it safe to use a medication off-label?
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
Examples of off-label medication use for mental health conditions:
- Amitriptyline: insomnia, posttraumatic stress disorder (PTSD)
- Clonidine: smoking cessation, excessive saliva caused by clozapine
- Gabapentin: alcohol dependence, social anxiety
- Prazosin: posttraumatic stress disorder (PTSD) nightmares
- Topiramate: alcohol dependence, weight gain caused by antipsychotic medication, binge eating disorder, bulimia
- Trazodone: insomnia
- 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.
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.