UMBRELLA is a holistic approach to progress, reshape, and benchmark the overall stroke care pathway and set new and improved standards of care in terms of primary and secondary prevention, rapid access to treatments, early accurate diagnosis, stratification, management and real-time monitoring, therapeutic targets identification, and rehabilitation, recurrent stroke and related cardiovascular events. This innovative approach will transform healthcare systems by improving and harmonizing professionals' workflows in a more patient-centred, digitalized, and communicative manner. UMBRELLA aims to revolutionize stroke management by implementing a comprehensive approach that addresses gaps along the whole continuum of the stroke care pathway. The key paradigm in the project is the multicentric, synergistic "umbrella" strategy for local data collection, harmonization, and standardization along the entire pre-, in-, and post-hospitalization pathway. By establishing specific common data models (CMDs) implemented in each of the 7 top-tier European clinical centres, UMBRELLA will create a federated data platform (U-platform) where Real World Data (RWD)-based AI algorithms can be locally created and validated, to advance personalised diagnosis, risk prediction, and treatment decisions in the acute and post-acute phases of stroke. The algorithms will be then trained in a decentralized manner through a federated learning infrastructure (FL-platform), which preserve data security and privacy, avoiding data centralization or exchange across centres but fostering collective AI-models training. On the other hand, standardized stroke management protocols and procedures will be created and implemented across the participating centres, including the validated usage of advanced digital technologies as solutions to facilitate data collection, visualization, patient engagement, monitoring, outcomes integration, and decision-making across the whole stroke pathway.

EU-PEARL has the ambition of transforming the current approach of conducting single-compound clinical trials into the use of cross-company Integrated Research Platforms (IRPs), taking into consideration both patients’ interests and the opportunities from novel molecules for addressing medical needs. Patient-centric data and knowledge sharing have the potential to accelerate the development of new treatments and reduce the operational costs of clinical trials. EU-PEARL will improve clinical effectiveness, patients’ satisfaction and societal access to timely and affordable medicines and it will shape the clinical trials of the future. This will change the industry paradigm from competition to cooperation in four disease areas and provide the framework for designing IRPs in other disease areas. The main objectives of EU-PEARL are: (1) To create a reusable, accessible and sustainable modular IRP for the design and execution of patient-centric, cross- company IRP in any disease area with unmet needs; (2) To set up the open, dynamic, patient inclusive IRP governance structure that will manage the appropriate regulatory, ethical, legal, statistical and data utilisation requirements of the IRP; (3) To disseminate and exploit the EU-PEARL paradigm through the provision of the necessary common tools, procedures, expertise and operational skills working to the highest scientific, regulatory and ethical standards and best practices, developed jointly by public and industry partners in a consensus-based approach; and (4) To create trial-ready IRP networks in the four disease areas of Major Depressive Disorder (MDD), Tuberculosis (TB), Non-Alcoholic Steatohepatitis (NASH) and Neurofibromatosis (NF).

The European Rare Diseases Research Alliance (ERDERA) aims to improve the health and well-being of the 30 million people living with a rare disease in Europe, by making Europe a world leader in Rare Disease (RD) research and innovation, to support concrete health benefits to rare disease patients, through better prevention, diagnosis and treatment. This Partnership will deliver a RD ecosystem that builds on the successes of previous programmes by supporting robust patient need-led research, developing new diagnostic methods and pathways, spearheading the digital transformational change connecting the dots between care, patient data and research, while ensuring strong alignment of strategies in RD research across countries and regions. Structuring goal-oriented public-private collaborations targeted at interventions all along the R&D value chain will ensure that the journey from knowledge to patient impact is expedited, thereby optimising EU innovation potential in RD. To support its ambition and missions ERDERA has been designed as a comprehensive and integrated ecosystem of which structure can be compared to an institute encompassing three main parts: (i) funding, (ii) internal (in house) Clinical Research Network that implements research activities targeting clinical trial readiness of RDs and accelerating diagnosis and translation of research discovery into improved patient care, and (iii) related supporting services (Data, Expertise, Education and Training) as well as an acceleration hub that serve external and internal RD community, all supported by all-embracing coordination and strategy and foundational (inter)national alignment.

Patients' outcomes and experience of health care can be improved through the systematic capture and use of information from their perspective. We are currently not using all the information we could gain from Patients' outcomes and experience of health care can be improved through the systematic capture and use of information from their perspective. We are currently not using all the information we could gain from Patient-Reported Outcomes (PROs) to accurately measure value from the patient perspective. This is due to the lack of standardisation, interoperability and implementation of PRO measurement schemes. We urgently need a European scale network of outcomes data collection, analysis and evidence sharing to inform clinical practice and healthcare decisions. To tackle these issues, this public-private consortium brings together scientists, clinicians and professionals to design and set up independent, patient-centred, Health Outcomes Observatories (H2Os). With input from patients, providers and health care decision makers (health ministries, health insurers, regional health authorities, public health agencies, medicines regulators, health technology assessment agencies), these Observatories will collect data and provide information not only for individual clinical care, but also for evaluation of new technologies and for healthcare decision making. We will establish ethically and legally sound national, or regional, H2Os and run these initially in four countries for three diseases. Based on a hybrid model of federated and centralised data collection, management and analysis, these Observatories will operate under a governance model that will guarantee that data are protected under jurisdictional data protection law. H2Os will be connected to a pan-European umbrella H2O to facilitate interoperability, guide reproducibility in other countries, and promote the benefit of measuring and using patient-centred outcomes at regional, national, European and global levels.

PARADIGM will provide a unique framework that enables structured, effective, meaningful, ethical, innovative, and sustainable patient engagement (PE) and demonstrates the ‘return on the engagement’ for all players. Much needed processes and tools for three key decision-making points (research priority setting, design of clinical trials and early dialogue) will be developed by consensus. Building on advances at international level, PARADIGM will integrate the needs, perspectives and expectations of all actors (including vulnerable populations) involved and will also produce a stakeholder-tailored set of metrics to measure the impact of PE. PARADIGM will deliver to the community, recommendations and tools to support the implementation of PE, leveraging pre-existing resources. These will address capacity and capability questions, managing potential conflict of interest, the compensation of individuals and organisations, and the ethics of engagement. A targeted communication, dissemination and engagement stra