One standing umbrella trial for all rare cancers in a country
Rare cancers together are a fifth of all cancers, but each is too small for its own trial. One permanent trial with many arms would give all of them a route.
Rare cancer patients face a structural problem: no single disease supports a trial, so most receive off-label therapy with no data capture. A standing national umbrella — one master protocol, molecular screening for all comers, arms opened by mechanism, shared infrastructure and Bayesian analysis — has precedent in genomically driven platforms and in national molecular screening programmes, but is rarely made permanent or made the default route.
- 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.
- Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.
- Trials enrol too few, too slowly · Fewer than one in ten adults with cancer joins a trial. Trials close for lack of patients, not lack of ideas.
Pages like this
not linked directly; found by shared links- IdeaTrial-in-a-box: a preconfigured protocol kit any hospital can open for a rare cancer
Shares Basket, umbrella, and platform trials, Trials enrol too few, too slowly, Biliary tract cancer (cholangiocarcinoma), Neuroendocrine tumours.
- IdeaOne global rare cancer network with n-of-1 and Bayesian trial frameworks
Shares Basket, umbrella, and platform trials, Biliary tract cancer (cholangiocarcinoma), Neuroendocrine tumours, Rare and paediatric cancers without markets.
- IdeaRandomise inside the registry that already follows every patient
Shares ClinicalTrials.gov, Basket, umbrella, and platform trials, Neuroendocrine tumours, Rare and paediatric cancers without markets.
- IdeaJust-in-time site activation: open a site in two weeks when a patient appears
Shares AI trial matching & clinical decision support, Basket, umbrella, and platform trials, Trials enrol too few, too slowly, Rare and paediatric cancers without markets.
- IdeaA national platform trial that every ctDNA-positive patient can join
Shares AI trial matching & clinical decision support, Basket, umbrella, and platform trials, Trials enrol too few, too slowly, Cancer Research UK.
- IdeaA standing platform trial that assigns treatment by how the tumour escaped
Shares Basket, umbrella, and platform trials, Trials enrol too few, too slowly, Comprehensive genomic profiling, Trial design, endpoints and cost.
- IdeaOne national master contract and budget template for all cancer trials
Shares ClinicalTrials.gov, Trials enrol too few, too slowly, Cancer Research UK, National Cancer Institute (NIH).
- InstitutionInstitut National du Cancer
Shares UNICANCER, Tumour-agnostic (tissue-agnostic) approval, Basket, umbrella, and platform trials, Trials enrol too few, too slowly.