Off-label use
Also called Off-label prescribing, unapproved use of an approved drug
Off-label use is the prescribing of an FDA-approved drug in a way its approved labeling does not cover: for a different condition, at a different dose, in a different form, or for a group of patients it was not approved for. The FDA's position is that, once a drug is approved, a licensed clinician generally may prescribe it for an unapproved use when they judge it medically appropriate for the patient.
Off-label use is legal and common, but it means something specific. The FDA has not determined that the drug is safe and effective for that use, so the evidence has to come from elsewhere, from trials, guidelines or clinical experience, and its strength varies widely. It is a separate question from compounding: an off-label drug is an approved product used outside its label, while a compounded medication was never approved.
Why it matters after 45
Several treatments discussed in midlife are off-label: testosterone for women, where no FDA-approved product exists in the United States, clomiphene and enclomiphene in men, and drugs such as rapamycin or methylene blue discussed for aging. Each is a reasonable thing to ask about, and each deserves the question of what evidence supports that particular use.
Where it comes up
Related terms
References
- U.S. Food and Drug Administration. Understanding Unapproved Use of Approved Drugs "Off Label". https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/understanding-unapproved-use-approved-drugs-label
- U.S. Food and Drug Administration. Is It Really "FDA Approved"? https://www.fda.gov/consumers/consumer-updates/it-really-fda-approved
Definitions here are educational and are not medical advice. They explain what a term means; the pages linked above cover what it means for you, and a clinician reviews your own results. Editorial & medical review policy · All glossary terms