Molecular profiling and access to treatment for rare cancers in Europe

Arcagen is a clinical study proposing a comprehensive analysis of molecular and clinical characteristics of rare adult solid cancers (11 domains: sarcomas, gynecology, uro-genital, GI, NET, head and neck, thoracic, skin and eyes, CNS and CUP) across Europe, utilizing the EORTC-SPECTA platform with the EURACAN ERN. This study enrolled 1235 advanced stage patients. 893 were evaluable for molecular profiling toward a potential access to personalized therapy.In the 11 different domains, 69.4% (620/893) of patients had actionable mutations, among whom 93 (15% of 620 patients with currently actionable alterations) received targeted- or immune (IO)-therapies. The median overall survival of the patient population without treatment adaptation, with switch to targeted therapy, or immunotherapy was 1.1, 3.3 years, and not reached, respectively.Across Europe, high prevalence and diversity of molecular alterations for rare solid tumors were observed as well as significant disparities regarding access to personalized medicine approaches. Molecular characterization of rare cancer types can be challenging, and this can limit application of personalized therapy. Here, the authors compile a large clinical cohort of rare adult solid cancers to find 69% harbour actionable mutations.