What is off-label medication use?

Before a medication reaches the U.S. market, it must obtain approval from the FDA. 

The approval demonstrates that the medication has received rigorous testing for effectiveness and safety in a specific population and for one or more uses. FDA approval means the benefits of using the medication for that use outweigh any potential risks. 

What does it mean to use a medication off label?

Unapproved or off-label use is when a healthcare professional prescribes medication for conditions or symptoms other than the ones the FDA originally approved it for.

Examples of off-label drug uses may involve:

• Dosage: prescribing a higher or lower dosage or changing how often you take the drug
• Health condition: using the medication for a health condition that is not included in the drug label or box
• Administration form: administering the medication in a different way, like taking a powder instead of a pill
• Population: recommending the drug to someone who’s not in the FDA-approved population, like gender, age, or body mass index (BMI)

Is off-label use safe?

The Agency for Healthcare Research and Quality estimates that in the United States, 1 in 5 prescriptions is for an off-label use. 

Off-label uses may not have undergone the same clinical scrutiny, or for the same length, as FDA-approved uses. 

Still, many off-label uses are supported by robust clinical evidence and have become widely accepted within the medical community. A 2021 review explains that often, off-label uses are a starting point for future research and sometimes progress into approved uses. 

If a healthcare professional recommends that you use a medication for a non-FDA-approved use, you can discuss the risks and benefits with them.  

How is off-label use determined?

Doctors often base the decision to prescribe a medication off-label on emerging scientific research, clinical experience, and patient needs. The following are some reasons why a healthcare professional may recommend off-label use: 

• There are no approved medications for your health condition.
• You already tried the FDA-approved options, and they didn’t work.
• You experience significant side effects from the approved alternatives.
• You have no access to the approved medications.
• The approved options interact with other medications you take.

Common off-label uses for medications

Some medications that are commonly prescribed off-label include:

• Gabapentin (Neurontin): In addition to its FDA-approved uses for epilepsy, neuropathic pain, and restless leg syndrome, gabapentin has a wealth of off-label uses. These include treating bipolar disorder, postmenopausal hot flashes, anxiety, irritable bowel syndrome (IBS), alcohol withdrawal, and itching. 
• Amitriptyline: While approved for major depressive disorder (MDD) in adults, amitriptyline is also prescribed off-label for conditions like post-traumatic stress disorder (PTSD), insomnia, chronic pain, IBS, and headaches.
• Metformin (Riomet): Metformin is FDA-approved to treat type 2 diabetes. It also has off-label uses, like treating polycystic ovary syndrome (PCOS), managing weight gain associated with antipsychotics, and supporting cancer treatment. 
• Quetiapine (Seroquel): Approved uses include treating MDD, schizophrenia, and bipolar disorder, while off-label uses include treating anxiety disorder, insomnia, and PTSD.
• Mirtazapine (Remeron): This drug is FDA-approved to treat depression and has a large variety of off-label uses, such as managing insomnia, anxiety, PTSD, migraine, and methamphetamine use disorder.
• Botox: Botox is FDA-approved for various uses, like reducing the appearance of wrinkles and treating overactive bladder and chronic migraine. Off-label uses may include supporting wound healing and rosacea treatment. 
• Fluoxetine (Prozac): This drug is approved to treat MDD, obsessive-compulsive disorder (OCD), and bulimia. Off-label uses of fluoxetine include treating PTSD, social anxiety, and borderline personality disorder.
• Semaglutide (Ozempic): Ozempic is FDA-approved for blood sugar management in people with diabetes, but some healthcare professionals prescribe it off-label for weight loss. For reference, the alternative semaglutide medication, Wegovy, is FDA-approved for weight loss, though there have been shortages of this and other semaglutide products in recent years.
• Colchicine (Colcrys): This drug is approved for the management of gout attacks and the autoinflammatory condition familial Mediterranean fever. It also has many off-label uses, for example, treating inflammation of the sac around the heart, liver disease, autoimmune blistering rash, and thickening of the lung tissues. 

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Summary

Doctors typically prescribe medications off-label when a drug has shown effectiveness and overall safety in clinical settings or research. It is a common practice among healthcare professionals. Off-label prescriptions may help treat health conditions where there are no suitable FDA-approved options.  

Off-label uses may not always undergo the same level of clinical testing as FDA-approved uses for a medication. While off-label uses can be safe, this is not always the case. That’s why it’s important to discuss any off-label uses with a healthcare professional and to follow their directions.