Enhanced International Research on Very Rare Cancers ofPaediatric & Adolescent and Young Adult Age: TheCollaborative Observational Study for Pancreatoblastomaand Adrenocortical Carcinoma

01/01/2026 to 31/12/2027

Representatives from the EU Beating Cancer Plan and the US NCI Cancer Moonshot established a technical working group aiming at EU-US
synergistic collaborative efforts to develop joint definitions of rare paediatric and AYA cancers and to identify common data elements for
observational rare tumour studies. Pancreatoblastoma (PBL) and Adrenocortical Carcinoma (ACC) are both aggressive rare malignancies with dismal
outcome particularly in advanced and metastatic stages. Early diagnosis, surgical management and ideally identification of new active therapies based
on molecular signals and the understanding of mode of actions to address the high unmet medical needs are critical to improve patients’ prognosis.
A pilot collaborative observational study will be established as proof of concept to accelerate progress by joining forces to increase accrual of
sufficient numbers of patients for meaningful analyses. The target cohorts will be analysed by age, sex race, ethnicity, and clinical presentation.
Genetic, clinical, and treatment-specific characteristics will be collected to enable the identification of potential biomarkers for possible early
detection, diagnosis, prognosis and risk classification, therapeutic response, and drug development. Patient-centric research will be fostered by
involving advocacy groups and patients in designing ethical data-sharing protocols as well as patient-reported outcomes (PROs).
This smaller-scale project will address practical and legal challenges related to data collection, regulatory alignment, and integration of complex data
types such as molecular data, imaging, and PROs. The goal is to establish a framework for future expansion to additional rare paediatric tumours
allowing international collaborations. The direct grant will involve the European EXPERT Group for Very Rare Tumours and include relevant
stakeholders (Legal and IT experts, CCI-E, SIOPE, EUROCAN) and will use the “Partner” Registry maintained by ERNPaedCan.