{"entity":{"id":"off-label","kind":"term","name":"Off-label","aka":["off label","off-label use","off-label prescribing","on-label","labelled indication","labeled indication","approved indication","approved indications","drug label","prescribing information"],"tldr":"Prescribing an approved drug for a use not listed on its official label, for example a different cancer or an earlier stage. Legal and common in oncology, but insurers may refuse to pay and the evidence is often thinner.","summary":"A drug's label (its licence) specifies the diseases, stages, biomarkers and prior treatments for which regulators have judged the evidence sufficient; anything else is off-label. In oncology perhaps a third of prescribing is off-label, driven by tumour-agnostic biology (a KRAS G12C inhibitor approved in lung cancer used in colorectal cancer), by rare cancers where trials are infeasible, and by guideline recommendations that run ahead of regulators. Compendia such as NCCN largely determine which off-label uses insurers will reimburse in the United States, and tumour-agnostic approvals and basket trials are ways of bringing rational off-label use onto the label.","asOf":"2026-09-09","wikipedia":"https://en.wikipedia.org/wiki/Off-label_use","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Off-label_use"}],"tags":[],"related":["standard-of-care","tumour-agnostic","basket-umbrella-platform","accelerated-approval","orphan-drug","rare-cancers","companion-diagnostic-term"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"category":"Clinic basics"},"route":"/terms/off-label/","neighbours":{"term":[{"id":"accelerated-approval","kind":"term","name":"Accelerated approval","route":"/terms/accelerated-approval/"},{"id":"basket-umbrella-platform","kind":"term","name":"Basket, umbrella, and platform trials","route":"/terms/basket-umbrella-platform/"},{"id":"companion-diagnostic-term","kind":"term","name":"Companion diagnostic","route":"/terms/companion-diagnostic-term/"},{"id":"expanded-access","kind":"term","name":"Expanded access (compassionate use)","route":"/terms/expanded-access/"},{"id":"medicare-ced","kind":"term","name":"Medicare coverage with evidence development","route":"/terms/medicare-ced/"},{"id":"orphan-drug","kind":"term","name":"Orphan drug","route":"/terms/orphan-drug/"},{"id":"rare-cancers","kind":"term","name":"Rare cancers","route":"/terms/rare-cancers/"},{"id":"standard-of-care","kind":"term","name":"Standard of care","route":"/terms/standard-of-care/"},{"id":"tumour-agnostic","kind":"term","name":"Tumour-agnostic (tissue-agnostic) approval","route":"/terms/tumour-agnostic/"}],"drug":[{"id":"fenbendazole","kind":"drug","name":"Fenbendazole (veterinary anthelmintic)","route":"/drugs/fenbendazole/"},{"id":"ivermectin","kind":"drug","name":"Ivermectin","route":"/drugs/ivermectin/"},{"id":"mebendazole","kind":"drug","name":"Mebendazole","route":"/drugs/mebendazole/"}],"trial":[{"id":"nct07487805","kind":"trial","name":"ICONIC: ivermectin combined with immune checkpoint inhibition in cancer (University of Florida phase 2)","route":"/trials/nct07487805/"}],"paper":[{"id":"paper-powderly-fenbendazole-ivermectin-liver-injury-2026","kind":"paper","name":"Drug-induced liver injury following co-ingestion of veterinary fenbendazole and ivermectin for prostate cancer: a case report","route":"/key-papers/paper-powderly-fenbendazole-ivermectin-liver-injury-2026/"},{"id":"paper-patel-ivermectin-cancer-curr-oncol-rep-2025","kind":"paper","name":"Ivermectin in cancer treatment: should healthcare providers caution or explore its therapeutic potential?","route":"/key-papers/paper-patel-ivermectin-cancer-curr-oncol-rep-2025/"},{"id":"paper-saperstein-ivermectin-neurotoxicity-breast-cancer-2026","kind":"paper","name":"Life-threatening neurotoxicity following off-label ivermectin use in metastatic breast cancer: a case report","route":"/key-papers/paper-saperstein-ivermectin-neurotoxicity-breast-cancer-2026/"},{"id":"paper-hulscher-ivermectin-mebendazole-cohort-anticancer-res-2026","kind":"paper","name":"Real-world clinical outcomes of ivermectin and mebendazole in cancer patients: results from a prospective observational cohort (with 2026 expression of concern)","route":"/key-papers/paper-hulscher-ivermectin-mebendazole-cohort-anticancer-res-2026/"}]}}