Supporting the implementation of the Cancer Mission · 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

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,…

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…