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Rare cancers together make up a fifth of all cancers, but no single one supports its own trial, so most patients get off-label therapy with no data capture. One permanent national umbrella trial, with molecular screening for all comers and arms opened by mechanism, would give every rare cancer 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.
Shares Rare cancers are not so rare: the rare cancer burden in Europe, Basket, umbrella, and platform trials, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
Shares Rare cancers are not so rare: the rare cancer burden in Europe, ClinicalTrials.gov, Basket, umbrella, and platform trials, Rare and paediatric cancers without markets.
Shares Basket, umbrella, and platform trials, Trials enrol too few, too slowly, Rare and paediatric cancers without markets, Neuroendocrine tumours.
Shares AI trial matching & clinical decision support, Basket, umbrella, and platform trials, Trial modernisation roadmap: the randomised trial → platforms and adaptive designs → decentralised, pragmatic and always-on, Trials enrol too few, too slowly.
Shares Basket, umbrella, and platform trials, Trial modernisation roadmap: the randomised trial → platforms and adaptive designs → decentralised, pragmatic and always-on, Trials enrol too few, too slowly, Trial design, endpoints and cost.
Shares AI trial matching & clinical decision support, Basket, umbrella, and platform trials, Trials enrol too few, too slowly, Rare and paediatric cancers without markets.
Shares Rare cancers are not so rare: the rare cancer burden in Europe, Basket, umbrella, and platform trials, Rare and paediatric cancers without markets, Trial design, endpoints and cost.
Shares Basket, umbrella, and platform trials, Trial modernisation roadmap: the randomised trial → platforms and adaptive designs → decentralised, pragmatic and always-on, Cancer Research UK, Trial design, endpoints and cost.