Neurodegenerative diseases are one of the most important contributors to morbidity and mortality in the elderly. In Europe, over 14 million people are currently living with dementia, at a cost of over 400 billion EUR annually. Comorbidities with these conditions are frequent and a major obstacle to optimal diagnosis and management. Recent advances in diagnostic technologies and the advent of disease-modifying therapies (DMT) for Alzheimer’s disease (AD), the most common aetiology of dementia, heralds the beginning of precision medicine in this disease area. PROMINENT will develop a digital platform for precision medicine that will remove barriers that currently exists for leveraging these technological advancements in the routine care of patients with neurodegenerative disorders and co-morbidities. The platform gives clinicians access to prediction models leveraging multimodal diagnostic data automatically derived from multiple sources (imaging repositories, medical records, mobile devices), helping them choose optimal care pathways and improving diagnostic precision. It will provide personalized, relevant and meaningful information on diagnosis and prognosis in a format understandable by patients and care partners. Further, it will support the introduction of new health technologies such as DMT for AD, by ensuring adherence to appropriate use guidelines and facilitating the prospective collection of data on real-world usage, safety and effectiveness. The expected impact of the project is to increase diagnostic accuracy and optimized use of existing and new treatment options. It will empower patients and caregivers by engaging them in more person-centric health care decisions, leading to improved adherence and patient experience. Ultimately this is expected to lead to cost-effective care, improved health outcomes and quality of life.

Immunotherapy (IO) is the new standard of care for many patients with advanced Non-Small Cell Lung Cancer (aNSCLC), yet only around 30-50% of treated patients benefit from IO in the long term. Programmed Death-Ligand 1 (PD-L1) remains the only biomarker used to predict patient outcome to IO, though its efficacy is limited. Other potential biomarkers have been identified, yet not validated in prospective randomized clinical trials, providing only partial evidence. Due to the dynamic complexity of the immune system-tumour microenvironment, its interaction with the host and patient behaviour, it?s unlikely for a single biomarker to accurately predict patient outcome. Artificial Intelligence (AI) and machine learning (ML) frameworks, that synthetize and correlate information from multiple sources, are essential to develop powerful decision-making tools able to deal with this highly complex context and provide individualized predictions to improve patient outcomes reducing the economic burden of health care systems in NSCLC. The aim of the I3LUNG project is to develop such AI-based tools to assist in improving survival and quality of life, preventing undue toxicity, and reducing treatment costs. I3LUNG adopts a two-pronged approach: setting up a transnational platform of available data from 2000 patients in order to validate the AI models, and generating a multi-omics prospective data collection in 200 NSCLC patients integrating diverse -omic information then validate its usefulness in leading IO therapeutic decisions. A psychological study will help in defining the impact of AI-guided decisions on patients, eliciting their preference, and physicians comparing AI with Human Intuition. The final goal is the construction of a novel integrated AI-assisted Data Storage and Elaboration Platform backed up by Trustworthy Explainable AI methodology, ensuring its accessibility and ease of use by healthcare providers and patients alike.

Prostate Cancer (PCa) is the second leading cause of cancer, among men in Europe. There are currently major unmet needs in this field, such as insufficient knowledge on risk factors that contribute to PCa and on patient characteristics (including genetic profiles) that could facilitate patient stratification. Finally, there is lack of meaningful engagement of all key stakeholders, while the knowledge currently gained from clinical practice and real life data is not being fed back into PCa patients’ care pathways. There is thus a need for better definition of PCa across all stages, improved patient’s stratification at diagnosis, and standardisation of PCa-related outcomes based on real life data. PIONEER’s unique dual approach is to first identify critical evidence gaps in PCa by respected Key Opinion Leaders, and then embark on a research priority setting exercise that reflects the needs of all key stakeholders in PCa management. To achieve this, PIONEER has brought together comprehensive datasets that consists of the most relevant prostate clinical trials and registries, large epidemiological cohorts, electronic heath records, and real-life data from different European (and non-European) patient populations. These unique data sets will be integrated, standardised, harmonised and analysed using approaches that are built on our experience of similar previous IMI projects i.e EMIF, and eTRIKS, and analysed using a unique set of methodologies and advanced analytics methods (OMOP, eHS). PIONEER has already performed a first PCa research priority setting survey, where major stakeholders were asked to identify the current unmet needs in PCa. The five most important open questions will be used as pilot studies to verify PIONEER’s research framework. As such, PIONEER’s deliverables will be outcome-driven, value-based and patient-centric, and relevant to all key stakeholders, as they would have been meaningfully involved from the inception of the project.

The overall goal of the Big Data for Better Outcomes (BD4BO) programme is to facilitate the use of ‘big data’ to promote the development of value-based, outcomes-focused healthcare systems in Europe. To fully exploit the transformative potential of big data, consideration will need to be taken of the use of detailed personal and biological information across the spectrum of care delivery, starting from the development of innovative medicines and treatments, to market access and adoption, diffusion, and use in healthcare systems by providers and patients. This paradigm shift requires shared understanding and standards among healthcare stakeholders including patients, providers, payers, regulators, policy makers, pharmaceutical industry, and academia. OBJECTIVES The proposed Coordination and Support Action (CSA) will establish an enabling platform that brings together these stakeholder groups across the BD4BO programme to ensure quality and consistency of individual projects in line with the overarching programme objective. Our consortium therefore aims to promote the use of big Data for better Outcomes, policy Innovation and healthcare system Transformation (DO->IT). Accordingly, we will: • Define a programme strategy that ensures quality, consistency and sustainability of health outcomes related activities across individual BD4BO projects. • Integrate, synthesise, and manage knowledge from all BD4BO projects, making it easily accessible via a single knowledge exchange platform. • Act as pivotal point of collaboration, stakeholder engagement and communication for all BD4BO projects. • Provide transparency and enable the use of patient health data and human biological samples for research purposes by developing minimum data privacy standards for Informed Consent Forms (ICFs) and supporting materials for use by individual BD4BO disease-specific projects and more widely in the Research and Development (R&D) sector.

Millions of people in the EU are negatively affected by cancer, and cancer is one of the leading causes of death and morbidity in Europe (ecis.jrc.europa.eu). The PRIME-ROSE consortium will contribute to the Mission Cancer goal to optimise treatment and support quality of life of more than 3 million people by 2030. The PRIME-ROSE consortium envisions to support Mission Cancer through the following objectives: 1) increase access to optimised and affordable treatment interventions for cancer patients that prolong life and increase quality of life, 2) generate clinical evidence and evaluate effectiveness in Precision Cancer Medicine (PCM)-relevant and synthetically randomised, controlled academic investigator-initiated pragmatic clinical trials, and 3) work together across Europe with relevant experts to implement evidence-based PCM in routine practice and address inequalities in access. This consortium builds on the truly bottom-up, clinician-initiated family of DRUP-like innovative trials that provide broad access to precision medicine for cancer patients. This action is part of the Cancer Mission cluster of projects on ‘Diagnosis and treatment’.