A DRUP-style protocol for off-label generic targeted drugs in rare tumours
Cheap generic versions of targeted cancer drugs like imatinib exist, but patients with rare tumours carrying the matching mutation often cannot get them. A structured programme would treat them and collect the evidence.
The Netherlands' Drug Rediscovery Protocol (DRUP) gives patients with actionable mutations access to approved targeted drugs outside their label, with cohorts expanding on response and payers covering successful cohorts; it has been replicated in several countries. Now that imatinib, erlotinib, gefitinib, sunitinib, abiraterone and others are generic, an analogous protocol for generic targeted drugs would be extremely cheap. The proposal is a DRUP-generic arm run across countries including middle-income ones, with genomic profiling, cohort-based Simon two-stage designs, and pre-agreed payer coverage for cohorts meeting response thresholds.
- 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 structured registry for every off-label cancer drug use
Shares Netherlands Cancer Institute (NKI-AvL), No incentive to repurpose cheap drugs, Rare and paediatric cancers without markets.
- IdeaA RECOVERY-style permanent platform trial of cheap drugs added to cancer care
Shares Basket, umbrella, and platform trials, No incentive to repurpose cheap drugs.
- InstitutionKWF Dutch Cancer Society
Shares Netherlands Cancer Institute (NKI-AvL), No incentive to repurpose cheap drugs.
- IdeaStandards for external control arms built from federated real-world data
Shares Basket, umbrella, and platform trials, Rare and paediatric cancers without markets.
- IdeaA fast route to the matched drug when it is licensed for another cancer
Shares Netherlands Cancer Institute (NKI-AvL), No incentive to repurpose cheap drugs.
- IdeaJust-in-time site activation: open a site in two weeks when a patient appears
Shares Basket, umbrella, and platform trials, Rare and paediatric cancers without markets.
- CollectionPancreatic Cancer Action Network (PanCAN)
Shares Basket, umbrella, and platform trials, Comprehensive genomic profiling.
- IdeaModel-informed individual dosing with drug-level monitoring for every oral cancer drug
Shares Imatinib, Small-molecule kinase inhibitors.