ideasIdea
A fast route to the matched drug when it is licensed for another cancer
Sometimes a progression biopsy shows exactly which drug would help, but it is licensed for another cancer and cannot be obtained. A standing pathway would fix that.
Mechanism-matched treatment recommendations frequently fail at the access step. A national pathway modelled on the Dutch DRUP and UK-style access schemes would combine a molecular tumour board decision, company-supplied drug, mandatory outcome registration and payer participation, so that every off-label use generates evidence. This converts scattered compassionate use into a structured cohort study.
Hypothesis
A standing mechanism-matched access pathway raises the proportion of actionable resistance findings that result in treatment from a small minority to over half, and generates interpretable efficacy data per mechanism-drug pair.
Rationale
DRUP-type studies have shown that structured off-label access with outcome collection is feasible and produces usable evidence for rare biomarker-drug combinations.
What would test it
Launch a pathway in one country covering ten drugs for three years; measure treatment rate for actionable findings, response rates by cohort, and time from report to first dose.
Maturity
speculative
Who has to act
regulator
Cost to try
Medium ($1M to $50M)
Years to first evidence
4
Bottlenecks it attacks
- Acquired resistance to every therapy · Nearly every targeted therapy stops working within months to a few years as the tumour adapts.
- 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.
- Regulatory divergence between regions · Regulatory divergence means a drug approved in one country can take years to reach another, or never arrive.