ideasIdea
Record what happens when doctors use cancer drugs off-label
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.
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.
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.
What would test it
Mandate registry entry as a condition of off-label reimbursement in one health system; report cohort response rates annually.
Maturity
early clinical
Who has to act
clinic
Cost to try
Medium ($1M to $50M)
Years to first evidence
3
Bottlenecks it attacks
- Failures are hidden · Negative trials, failed drugs and abandoned programmes are rarely published, so the same mistakes are repeated.
- 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.