{"entity":{"id":"idea-tr2-off-label-outcome-registry","kind":"idea","name":"Record what happens when doctors use cancer drugs off-label","aka":[],"tldr":"Cancer drugs are often prescribed outside their approved use based on hope or small studies. Capturing outcomes of these uses would reveal which ones fail so they can be stopped.","summary":"Off-label use is common in oncology, particularly for targeted agents matched to molecular alterations in other cancers. Structured outcome capture at the point of off-label prescribing (indication, rationale, response, duration, toxicity) through a simple registry, as the Dutch DRUP trial and the US TAPUR study do within trial frameworks, but extended to routine practice, would document failures that are otherwise invisible.","asOf":"2026-09-08","links":[{"label":"Bottleneck evidence (Failures are hidden): Anderson et al., Compliance with results reporting at ClinicalTrials.gov (NEJM 2015)","url":"https://doi.org/10.1056/NEJMsa1409364"}],"tags":[],"related":["idea-tr2-payer-combo-cwe"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["tumour-agnostic","real-world-evidence"],"trials":[],"people":[],"bottlenecks":["b-negative-results","b-real-world-evidence"],"keyPapers":["paper-anderson-n-engl-j-med"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"A national off-label registry will identify at least five drug-alteration-cancer combinations with response rates below 10% in over 50 patients within three years, leading to guideline statements against their use.","rationale":"DRUP and TAPUR have both closed cohorts for futility, generating negative evidence that changed practice. Most off-label use happens outside such studies and is never counted.","test":"Mandate registry entry as a condition of off-label reimbursement in one health system; report cohort response rates annually.","maturity":"early-clinical","actor":"clinic","cost":"medium","horizonYears":3},"route":"/ideas/idea-tr2-off-label-outcome-registry/","neighbours":{"idea":[{"id":"idea-tr2-payer-combo-cwe","kind":"idea","name":"Payers cover off-label combinations only inside registry-randomised trials","route":"/ideas/idea-tr2-payer-combo-cwe/"}],"term":[{"id":"real-world-evidence","kind":"term","name":"Real-world evidence","route":"/terms/real-world-evidence/"},{"id":"tumour-agnostic","kind":"term","name":"Tumour-agnostic (tissue-agnostic) approval","route":"/terms/tumour-agnostic/"}],"bottleneck":[{"id":"b-negative-results","kind":"bottleneck","name":"Failures are hidden","route":"/bottlenecks/b-negative-results/"},{"id":"b-real-world-evidence","kind":"bottleneck","name":"Weak real-world evidence and registries","route":"/bottlenecks/b-real-world-evidence/"}],"paper":[{"id":"paper-anderson-n-engl-j-med","kind":"paper","name":"Compliance with results reporting at ClinicalTrials.gov","route":"/key-papers/paper-anderson-n-engl-j-med/"}]}}