Off-label use

Regulation of therapeutic goods in the United States
Prescription drugs Over-the-counter drugs
Law Federal Food, Drug, and Cosmetic Act CDAPCA Controlled Substances Act Prescription Drug Marketing Act Hatch-Waxman Act exemptions Marihuana Tax Act of 1937
Government agencies Department of Health and Human Services Food and Drug Administration Department of Justice DEA
Process Drug discovery Drug design Drug development New Drug Application Investigational New Drug Clinical trial phases Randomized controlled trial Pharmacovigilance Abbreviated New Drug Application Fast track approval Off-label use
International coordination ICH Uppsala Monitoring Centre WHO CIOMS Single Convention on Narcotic Drugs
Non-governmental organizations National Academy of Medicine RADAR NORML
v t e

Off-label use is the use of pharmaceutical drugs for an unapproved indication or in an unapproved age group, dosage, or route of administration.^[1] Both prescription drugs and over-the-counter drugs (OTCs) can be used in off-label ways, although most studies of off-label use focus on prescription drugs.

Off-label use is very common and generally legal unless it violates ethical guidelines or safety regulations. The ability to prescribe drugs for uses beyond the officially approved indications is commonly used to good effect by healthcare providers. For example, methotrexate is commonly used off-label because its immunomodulatory effects relieve various disorders.^[2] However, off-label use can entail health risks and differences in legal liability. Marketing of pharmaceuticals for off-label use is usually prohibited.