# One standing umbrella trial for all rare cancers in a country

Source: https://onco.cc/ideas/idea-bio2-rare-cancer-umbrella-platform/  
OnCo record `idea-bio2-rare-cancer-umbrella-platform` (Idea). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Summary

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.

## Fields

- Kind: Idea
- Last checked: 2026-09-08
- Hypothesis: A permanent rare cancer umbrella platform enrols over 2,000 patients per year in a mid-sized country, delivers analysable results for at least ten mechanism-defined arms in five years, and reduces the cost per evaluable patient by half.
- Rationale: Shared control arms, shared screening and shared infrastructure are the only ways to make small populations statistically and economically tractable, as demonstrated by platform trials in genomic screening programmes and in paediatric oncology consortia.
- Proposed test: Fund a three-year platform pilot with pre-specified success metrics on enrolment, arms completed and cost per evaluable patient, compared with the preceding period of standalone rare cancer trials.
- Maturity: being-tested-at-scale
- Actor: policy

## Sources

- Bottleneck evidence (Rare and paediatric cancers without markets): Gatta et al., Rare cancers are not so rare: the rare cancer burden in Europe (EJC 2011): https://doi.org/10.1016/j.ejca.2011.08.008

## Connected records

- collections: [ClinicalTrials.gov](https://onco.cc/collections/clinicaltrials-gov/)
- cancers: [Biliary tract cancer (cholangiocarcinoma)](https://onco.cc/cancers/cholangiocarcinoma/), [Neuroendocrine tumours](https://onco.cc/cancers/neuroendocrine/), [Sarcomas (soft tissue, bone, GIST)](https://onco.cc/cancers/sarcoma/)
- technologies: [AI trial matching & clinical decision support](https://onco.cc/technologies/ai-trial-matching/), [Comprehensive genomic profiling](https://onco.cc/technologies/cgp/)
- companies: [UNICANCER](https://onco.cc/companies/unicancer/)
- institutions: [Cancer Research UK](https://onco.cc/institutions/cruk/), [National Cancer Institute (NIH)](https://onco.cc/institutions/nci/)
- terms: [Basket, umbrella, and platform trials](https://onco.cc/terms/basket-umbrella-platform/), [Tumour-agnostic (tissue-agnostic) approval](https://onco.cc/terms/tumour-agnostic/)
- bottlenecks: [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Trial design, endpoints and cost](https://onco.cc/bottlenecks/b-trial-design/), [Trials enrol too few, too slowly](https://onco.cc/bottlenecks/b-trial-enrolment/)
- key papers: [Rare cancers are not so rare: the rare cancer burden in Europe](https://onco.cc/key-papers/paper-gatta-eur-j-cancer/)
- roadmaps: [Trial modernisation roadmap: the randomised trial → platforms and adaptive designs → decentralised, pragmatic and always-on](https://onco.cc/roadmaps/trial-modernisation-roadmap/)

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