Require genetic and target-trial evidence before funding any repurposing phase 3
The big metformin cancer trial failed after years and millions, despite strong observational hints. Cheaper checks on causality should be passed before funding the next one.
MA.32 (metformin in breast cancer) was negative despite extensive observational support that was later attributed to immortal-time and confounding biases. Mendelian randomisation using drug-target genetic proxies, target trial emulation with active-comparator new-user designs in large health records, and pre-registered replication across independent databases can test causal plausibility for a fraction of a trial's cost. The proposal is a funding rule: repurposing phase 3 trials receive public funding only after a standardised evidence gate (genetic support where a proxy exists, at least two concordant target trial emulations, and a plausible dose-response) has been passed and published.
- 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.
- 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.
Pages like this
not linked directly; found by shared links- 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.
- IdeaLinked prescribing and outcome data to find drug interactions with cancer therapy
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaWearable activity data as a validated real-world endpoint
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaNo mCODE, no payment: tie oncology reimbursement to a minimal structured record
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaGood practice standards and inspection for real-world data sources
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaOne international registry, not one per country, for conditional approvals
Shares Weak real-world evidence and registries, Real-world evidence.
- IdeaA structured registry for every off-label cancer drug use
Shares No incentive to repurpose cheap drugs, Weak real-world evidence and registries, Real-world evidence.
- IdeaReal-world dose intensity and toxicity monitoring to revise labelled doses
Shares Weak real-world evidence and registries, Real-world evidence.