# A structured registry for every off-label cancer drug use

Source: https://onco.cc/ideas/idea-data-off-label-outcomes-registry/  
OnCo record `idea-data-off-label-outcomes-registry` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- 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.
- Proposed 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

## Sources

- DRUP trial (archived copy): https://web.archive.org/web/20140517065619/http://drup.nl/
- ASCO TAPUR: https://www.tapur.org/

## Connected records

- fronts: [AI & Computation](https://onco.cc/fronts/ai-computation/)
- institutions: [American Society of Clinical Oncology (ASCO)](https://onco.cc/institutions/asco/), [Netherlands Cancer Institute (NKI-AvL)](https://onco.cc/institutions/nki/)
- terms: [Real-world evidence](https://onco.cc/terms/real-world-evidence/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/)
- bottlenecks: [No incentive to repurpose cheap drugs](https://onco.cc/bottlenecks/b-generic-repurposing/), [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Weak real-world evidence and registries](https://onco.cc/bottlenecks/b-real-world-evidence/)

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