Off-label use
Clinical reviewer: Jacob Moore, MD · Last checked September 24, 2026
What does off-label mean?
Off-label use is prescribing an FDA-approved drug in a way its label does not cover: for a different disease, in a different way, or at a different dose than approved. FDA has not determined that the drug is safe and effective for that unapproved use. A healthcare provider may still judge it medically appropriate for a patient.
What counts as off-label use?
The National Cancer Institute defines off-label use as "the legal use of a prescription drug to treat a disease or condition for which the drug has not been approved by the U.S. Food and Drug Administration" [1]. FDA describes it more broadly: once a drug is approved, a provider may prescribe it for an unapproved use if they judge it medically appropriate, covering a different disease or condition, a different way of giving the drug, or a different dose than the one approved [2].
Why might a drug be used off-label?
FDA names two reasons: there might not be an approved drug for a patient's disease or condition, or the patient may have tried all approved treatments without seeing a benefit [2]. The same FDA page explains what "safe" means in an approval: "does not mean that the drug has no side effects. Instead, it means the FDA has determined the benefits of using the drug for a particular use outweigh the potential risks" [2]. That determination doesn't extend to unapproved uses. Ozempic and Mounjaro, for instance, have indications that all involve adults (and, for Mounjaro, some children) with type 2 diabetes [3][4].
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This entry is for education and is not medical advice. Talk with a licensed clinician about your own situation before starting, stopping, or changing any treatment.
