{"entity":{"id":"b-rare-cancers","kind":"bottleneck","name":"Rare and paediatric cancers without markets","aka":[],"tldr":"Taken together rare cancers are a fifth of all cancers, but each one alone is too small for a company to invest in.","summary":"Rare cancers (incidence below 6 per 100,000 per year in the European definition) together make up about a fifth of all cancer diagnoses and have worse survival than common cancers, yet each individual entity is too small to support a conventional development programme. Sarcomas, paediatric solid tumours, rare haematological malignancies and rare molecular subtypes of common cancers depend on academic cooperative groups, repurposing of adult drugs, and basket trials. Children have historically received new agents a decade after adults; the RACE for Children Act (2020) now obliges sponsors to test molecularly relevant drugs in paediatric populations, and tumour-agnostic approvals (NTRK, RET, BRAF, dMMR) create a route for rare subtypes. Diagnosis is also a bottleneck: pathology expertise for rare entities is concentrated in a few reference centres.","asOf":"2026-09-08","links":[{"label":"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"},{"label":"FDA, Pediatric Oncology and the RACE for Children Act","url":"https://www.fda.gov/about-fda/oncology-center-excellence/pediatric-oncology"},{"label":"WHO Global Initiative for Childhood Cancer","url":"https://www.who.int/initiatives/the-global-initiative-for-childhood-cancer"}],"tags":[],"related":["idea-menin-infant-all","idea-gd2-car-t-frontline-consolidation","idea-tcr-t-beyond-hla-a2","idea-bio2-rare-cancer-umbrella-platform","idea-bio2-n-of-1-framework","idea-bio2-paediatric-first-development","idea-bio2-rare-cancer-telepathology-network","idea-bio2-functional-precision-rare","idea-bio2-rare-cancer-prize-fund","idea-bio2-registry-embedded-randomisation","idea-bio2-patient-partnered-rare-commons","idea-bio2-mechanism-defined-baskets","idea-bio2-shared-compound-access-pool","idea-bio2-bayesian-borrowing-acceptance","idea-bio2-rare-tumour-model-bank","idea-moon-rare-cancer-global-network","idea-moon-open-source-oncology-drug-discovery"],"cancers":["sarcoma","neuroblastoma","mesothelioma","cholangiocarcinoma","neuroendocrine","all-leukemia","thyroid"],"sections":[],"technologies":["mibg-theranostics","limb-salvage-surgery","tandem-transplant"],"targets":["ntrk","gd2","ret","kmt2a"],"drugs":["tovorafenib","dinutuximab","naxitamab","nirogacestat","eflornithine","selpercatinib","revumenib"],"companies":["y-mabs","day-one-biopharmaceuticals","springworks","us-worldmeds","childrens-oncology-group","curie-nki-eortc"],"institutions":["nci","cruk"],"pathways":[],"terms":["tumour-agnostic","basket-umbrella-platform","sarcoma-histotype-tailoring","inrg-staging","mycn-amplification","fnclcc-grade"],"trials":["defi","gd2-cart01","euro-ewing-2012","anbl0032","hr-nbl1","interfant-06","naxitamab-201","nmtrc003","aall1731"],"people":[],"bottlenecks":[],"keyPapers":["paper-gatta-eur-j-cancer"],"journals":[],"dependsOn":[],"notes":[],"stage":"biology","severity":"major","metrics":[{"label":"Share of all cancer diagnoses in Europe that are rare cancers (RARECARE)","value":"22%","source":"Gatta et al., European Journal of Cancer 2011","url":"https://doi.org/10.1016/j.ejca.2011.08.008"},{"label":"Five-year relative survival for rare vs common cancers in Europe (RARECARE)","value":"47% vs 65%","source":"Gatta et al., European Journal of Cancer 2011","url":"https://doi.org/10.1016/j.ejca.2011.08.008"},{"label":"Children (0-14) diagnosed with cancer worldwide each year","value":"~400,000","source":"WHO fact sheet, Cancer in children","url":"https://www.who.int/news-room/fact-sheets/detail/cancer-in-children"}],"causes":["Small patient numbers make randomised trials slow or impossible and the commercial return too small to justify development.","Paediatric cancers have distinct biology (fusion-driven, low mutational burden) so adult drugs often do not apply.","Reference pathology and molecular diagnosis for rare entities is concentrated in a few centres, delaying correct diagnosis.","Regulatory precedent requires disease-specific evidence, which rare cancers cannot easily generate.","Preclinical models for many rare and paediatric tumours do not exist."],"currentEfforts":["The RACE for Children Act (FDARA 2017, in force 2020) requires paediatric investigation plans for adult oncology drugs directed at relevant molecular targets.","Tumour-agnostic approvals (larotrectinib and entrectinib for NTRK fusions, selpercatinib for RET, dabrafenib-trametinib for BRAF V600E) and basket trials such as NCI-MATCH and Pediatric MATCH reach rare subtypes.","The Children's Oncology Group, SIOPEN, Euro Ewing and ITCC run international trials that make paediatric randomisation possible.","The International Rare Cancers Initiative (Cancer Research UK, NCI, EORTC) designs trials for individual rare cancers.","Y-mAbs (naxitamab), Day One (tovorafenib), SpringWorks (nirogacestat) and US WorldMeds (eflornithine) show that small companies can bring rare-cancer drugs to approval.","The EU Orphan Regulation and US Orphan Drug Act provide exclusivity, fee waivers and tax credits."],"successLooksLike":"Every rare cancer entity has a reference diagnostic pathway and at least one open trial, children receive new molecularly matched drugs within two years of adult approval, and survival for rare cancers converges with common cancers."},"route":"/bottlenecks/b-rare-cancers/","neighbours":{"idea":[{"id":"idea-gbc-burden-to-funding-audit-and-dedicated-call","kind":"idea","name":"A burden-to-funding audit for gallbladder cancer and a dedicated research call","route":"/ideas/idea-gbc-burden-to-funding-audit-and-dedicated-call/"},{"id":"idea-data-72-hour-second-opinion-network","kind":"idea","name":"A digital second-opinion network answering community oncologists within 72 hours","route":"/ideas/idea-data-72-hour-second-opinion-network/"},{"id":"idea-reg-generic-targeted-therapy-drup","kind":"idea","name":"A DRUP-style protocol for off-label generic targeted 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