A structured registry for every off-label cancer drug use
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.
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.
- Weak real-world evidence and registries · We do not reliably know what happens to patients after approval, so we cannot tell which drugs deliver in practice.
- No incentive to repurpose cheap drugs · Old, cheap drugs with anti-cancer signals never get the trials they need because no one profits from the result.
- Rare and paediatric cancers without markets · Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.
Pages like this
not linked directly; found by shared links- IdeaA fast route to the matched drug when it is licensed for another cancer
Shares Tumour-agnostic (tissue-agnostic) approval, Netherlands Cancer Institute (NKI-AvL), No incentive to repurpose cheap drugs, Real-world evidence.
- IdeaRequire genetic and target-trial evidence before funding any repurposing phase 3
Shares No incentive to repurpose cheap drugs, Weak real-world evidence and registries, Real-world evidence.
- IdeaAn automated pipeline emulating trials of every common drug against every cancer
Shares No incentive to repurpose cheap drugs, Weak real-world evidence and registries, Real-world evidence.
- IdeaStandards for external control arms built from federated real-world data
Shares Weak real-world evidence and registries, Real-world evidence, Rare and paediatric cancers without markets.
- IdeaRecord what happens when doctors use cancer drugs off-label
Shares Tumour-agnostic (tissue-agnostic) approval, Weak real-world evidence and registries, Real-world evidence.
- IdeaA DRUP-style protocol for off-label generic targeted drugs in rare tumours
Shares Netherlands Cancer Institute (NKI-AvL), No incentive to repurpose cheap drugs, Rare and paediatric cancers without markets.
- IdeaA public rulebook for when an external or synthetic control arm is acceptable
Shares Weak real-world evidence and registries, Real-world evidence, Rare and paediatric cancers without markets.
- IdeaLinked prescribing and outcome data to find drug interactions with cancer therapy
Shares Weak real-world evidence and registries, Real-world evidence.