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Rare cancers are those with fewer than about 6 new cases per 100,000 people per year. Individually rare, together they are a quarter of all cancers and have worse survival because of late diagnosis, few trials and scattered expertise.
RARECARE (EU) defines rare cancers as incidence <6/100,000/year; there are ~200 such entities making up ~24% of European cancers, with 5-year survival ~48% versus ~63% for common cancers. Causes of the gap: diagnostic delay and misdiagnosis, absence of standard treatments, difficulty running trials (basket/umbrella designs, registries, real-world evidence help), and dispersed care. Responses: European Reference Networks (EURACAN, EuroBloodNet, PaedCan), centralisation (e.g. sarcoma centres), the NCI Rare Tumor Patient Engagement Network, Rare Cancers Europe, orphan-drug incentives and tumour-agnostic approvals (NTRK, MSI-H, RET, BRAF) that serve rare histologies. Paediatric cancers are all rare cancers.
Shares ComboMATCH (EAY191), DETERMINE arm 04: trastuzumab with pertuzumab in HER2-amplified or mutated rare cancers, I-PREDICT, DART (SWOG S1609): nivolumab plus ipilimumab in rare tumours.
Shares Orphan drug, Regulation (EC) No 1901/2006: EU paediatric medicines, Orphan Drug Act 1983, Regulation (EC) No 141/2000: EU orphan medicines.
Shares Regulation (EC) No 1901/2006: EU paediatric medicines, Parathyroid carcinoma, Orphan Drug Act 1983, Regulation (EC) No 141/2000: EU orphan medicines.
Shares Regulation (EC) No 1901/2006: EU paediatric medicines, Orphan drug designation, Orphan Drug Act 1983.
Shares I-PREDICT, DART (SWOG S1609): nivolumab plus ipilimumab in rare tumours, WINTHER.
Shares I-PREDICT, WINTHER, NCI-MATCH (EAY131), TAPUR (Targeted Agent and Profiling Utilization Registry).
Shares Regulation (EC) No 1901/2006: EU paediatric medicines, Orphan drug designation, Regulation (EC) No 141/2000: EU orphan medicines.
Shares Dermatofibrosarcoma protuberans, Chordoma, Inflammatory myofibroblastic tumour (IMT), Gastrointestinal stromal tumour (GIST).