Phase 1 including first-in-human clinical trials to test biomarker-guided medicines or multi-modal treatment interventions for patients with rare or very rare cancers or cancer subtypes

Expected Outcome:Proposals under this topic should aim to deliver results that are directed and tailored towards, and contribute to all of the following expected outcomes:Patients with rare or very rare cancers or cancer subtypes have access to tailored, promising biomarker-guided medicines or multi-modal treatment interventions via participation in subsequent clinical trials or national or regional compassionate use programmes by health authorities;Researchers, physicians, innovators[1], startup, spin-off and spin-out companies, SMEs, charities or foundations and other professionals from different disciplines and sectors have access to innovative technology, medical devices or promising biomarker-guided medicines for further validation or commercialisation;National healthcare providers, policymakers and authorities in European regions, EU Member States and Associated Countries have early safety and efficacy evidence to support further testing of affordable biomarker-guided medicines or multi-modal treatment interventions that benefit patients with rare or very rare cancers in their healthcare systems; Scope:Patients with rare and very rare[2] cancers across EU Member States and Associated Countries often present with advanced disease due to late diagnosis and have access to few treatment options. Hence, these patients typically have a lower 5-year overall survival than those with more common cancers and face challenges with timely access to a small number of appropriate phase 1 clinical trials, despite good disease control rates[3], to validate biomarker-guided medicines or multi-modal treatment interventions targeting their disease and adapted to an increasingly precision oncology healthcare landscape.Proposals should address all the following:When still relevant and required, finalise the preclinical validation of promising[4] biomarker-guided drugs[5], for rare or very rare cancer indications through in vivo and/or ex vivo and/or in silico research models. Drug