The University Hospital Vall d'Hebron is highly specialised in rare diseases at the regional, national, and international level, with its incorporation as a full member of 20 of the 24 European Reference Networks (ERNs). The scientific coordination of the ERN-EuroBloodNet, www.eurobloodnet.eu , the ERN dedicated to Rare Haematological Diseases (RHD), is led by Dr Mañú Pereira, head of the Rare Anaemia Disorders research laboratory within the "CANCER Y ENFERMEDADES HEMATOLOGICAS INFANTILES" group at Vall d'Hebron Research Institute (VHIR). The research strategy of our research laboratory is focused on the development and validation of innovative methodological approaches for better characterisation of patients affected by rare anemia disorders according to their individual features to allow personal risk profiles and personalised medicine. Within the ERN-EuroBloodNet and in line with the strategy at European level, the team is leading several projects on European patients' registries for rare haematological diseases, namely,



the European Patients Registries on Rare Haematological Diseases, ENROL , and on Rare Anaemia Disorders, RADeep , aiming at avoiding fragmentation of data by promoting the standards for patient registries' interoperability. Building on this expertise, the group is now launching RADiANT, a new research initiative that extends our data infrastructure beyond patient registries into clinical trial-grade research. RADiANT encompasses clinical outcomes research, including the definition of endpoints and study populations and the use of patient-reported outcome measures (PROMs); the design of external control arms; and Post-Authorisation Efficacy & Safety Studies (PAES & PASS). It also supports the re-use and integration of clinical data with -omics and functional analysis, the development of prognosis models combining advanced statistical and AI-based methods, federated approaches combining algorithms and platform infrastructure, and the extraction of

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