{"entity":{"id":"idea-moon-rare-cancer-global-network","kind":"idea","name":"One global rare cancer network with n-of-1 and Bayesian trial frameworks","aka":[],"tldr":"Rare cancers are collectively common but each is too rare for normal trials. Link every rare cancer patient worldwide into one network with registries and trial designs built for small numbers.","summary":"Rare cancers make up roughly a quarter of cancer diagnoses and have worse survival; each is too infrequent for conventional trials and expertise is scattered. Networks such as EURACAN and the International Rare Cancers Initiative show partial solutions. The proposal is a global federated rare cancer network: a single patient-facing registration and referral pathway, standardised molecular workup, a shared registry with outcomes, and pre-agreed trial frameworks (Bayesian borrowing across related histologies, n-of-1 crossover designs, external control arms from the registry) accepted by regulators for approval decisions.","asOf":"2026-09-08","links":[{"label":"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)","url":"https://doi.org/10.1016/j.ejca.2011.08.008"}],"tags":[],"related":[],"cancers":["sarcoma","cholangiocarcinoma","mesothelioma","neuroendocrine"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["curie-nki-eortc"],"institutions":[],"pathways":[],"terms":["basket-umbrella-platform"],"trials":[],"people":[],"bottlenecks":["b-rare-cancers","b-trial-design","b-regulatory-fragmentation"],"keyPapers":["paper-gatta-eur-j-cancer"],"journals":[],"dependsOn":[],"notes":[],"hypothesis":"The network doubles the number of rare cancer patients in trials and achieves at least five regulatory approvals in rare histologies within a decade using its trial frameworks.","rationale":"Aggregating small populations across borders is the only route to adequate sample sizes, and regulators have signalled openness to novel designs when data infrastructure is credible.","test":"Launch in sarcoma subtypes and rare gastrointestinal cancers; measure registration coverage, time to trial availability and approvals using network-generated evidence.","maturity":"early-clinical","actor":"research","cost":"medium","horizonYears":5},"route":"/ideas/idea-moon-rare-cancer-global-network/","neighbours":{"cancer":[{"id":"cholangiocarcinoma","kind":"cancer","name":"Biliary tract cancer (cholangiocarcinoma)","route":"/cancers/cholangiocarcinoma/"},{"id":"mesothelioma","kind":"cancer","name":"Mesothelioma","route":"/cancers/mesothelioma/"},{"id":"neuroendocrine","kind":"cancer","name":"Neuroendocrine tumours","route":"/cancers/neuroendocrine/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"}],"company":[{"id":"curie-nki-eortc","kind":"company","name":"EORTC","route":"/companies/curie-nki-eortc/"}],"term":[{"id":"basket-umbrella-platform","kind":"term","name":"Basket, umbrella, and platform trials","route":"/terms/basket-umbrella-platform/"}],"bottleneck":[{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","route":"/bottlenecks/b-rare-cancers/"},{"id":"b-regulatory-fragmentation","kind":"bottleneck","name":"Regulatory divergence between regions","route":"/bottlenecks/b-regulatory-fragmentation/"},{"id":"b-trial-design","kind":"bottleneck","name":"Trial design, endpoints and cost","route":"/bottlenecks/b-trial-design/"}],"paper":[{"id":"paper-gatta-eur-j-cancer","kind":"paper","name":"Rare cancers are not so rare: the rare cancer burden in Europe","route":"/key-papers/paper-gatta-eur-j-cancer/"}]}}