{"entity":{"id":"idea-data-off-label-outcomes-registry","kind":"idea","name":"A structured registry for every off-label cancer drug use","aka":[],"tldr":"Doctors often use cancer drugs outside their approved use based on a hunch or a small study. Record what happens every time so the hunches become evidence.","summary":"Off-label use is common in oncology, especially for rare tumours and molecularly matched therapy, and the outcomes are almost never captured. DRUP (Netherlands) and TAPUR (ASCO) showed that a pragmatic protocol can turn off-label use into evidence and even into reimbursement (DRUP's personalised reimbursement model). The proposal extends this to all off-label anticancer use: a light-touch registration with structured indication, biomarker and outcome capture as a condition of reimbursement.","asOf":"2026-09-08","links":[{"label":"DRUP trial (archived copy)","url":"https://web.archive.org/web/20140517065619/http://drup.nl/"},{"label":"ASCO TAPUR","url":"https://www.tapur.org/"}],"tags":[],"related":[],"cancers":[],"sections":["ai-computation"],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":["asco","nki"],"pathways":[],"terms":["real-world-evidence","tumour-agnostic"],"trials":[],"people":[],"bottlenecks":["b-real-world-evidence","b-generic-repurposing","b-rare-cancers"],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"An off-label registry will generate evidence supporting reimbursement or label expansion for at least five drug-indication pairs within four years, and will identify at least as many with no benefit that should stop being used.","rationale":"DRUP identified active cohorts (for example, nivolumab in dMMR tumours) and led to conditional reimbursement; the model has worked but only for molecularly selected patients in a few countries.","test":"Implement registration-as-condition-of-reimbursement for off-label targeted and immune therapies in one national payer; report cohorts reaching the pre-specified efficacy threshold at 24 months.","maturity":"early-clinical","actor":"payer","cost":"medium","horizonYears":3},"route":"/ideas/idea-data-off-label-outcomes-registry/","neighbours":{"section":[{"id":"ai-computation","kind":"section","name":"AI & Computation","route":"/fronts/ai-computation/"}],"institution":[{"id":"asco","kind":"institution","name":"American Society of Clinical Oncology (ASCO)","route":"/institutions/asco/"},{"id":"nki","kind":"institution","name":"Netherlands Cancer Institute (NKI-AvL)","route":"/institutions/nki/"}],"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-generic-repurposing","kind":"bottleneck","name":"No incentive to repurpose cheap drugs","route":"/bottlenecks/b-generic-repurposing/"},{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-real-world-evidence","kind":"bottleneck","name":"Weak real-world evidence and registries","route":"/bottlenecks/b-real-world-evidence/"}]}}