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

From the Commission's own budget table for this call. Confirm against the topic page before you plan around it: these move when a work programme is amended.

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 repurposing should be considered [6] . Data should be disaggregated by tumour biology, sex, gender, age and other relevant variables, such as by measures of socio-economic status or ethnicity; Validate early safety and efficacy of innovative medicines or as part of multi-modal treatment interventions in phase 1, including in first-in-human, multi-centre clinical trials, considering new trial designs [7] , for and with patients with rare or very rare cancers and their caregivers/families; The primary and secondary endpoints of the clinical trial(s) should support safety, efficacy…

3 projects have been funded under this line (HORIZON-MISS-CANCER-03) in Horizon Europe and H2020. This is what they were made of — not a recommendation, just what the Commission signed.

The mix of organisations

Who coordinated them

Coordinating an EU project is a demanding, specific job. These organisations have done it on this topic line before.