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.

ASSESS-DHT will increase the adoption of trustworthy and effective Digital Health Technologies (DHT) across Europe, enabling a more coherent digital single market, for health systems and patients to access DHT from all over Europe and giving industry a European market. We will develop a new assessment framework, beyond the existing models, capable of uniform HTA adoption across Europe, addressing new assessment challenges in Digital Therapeutics, AI and telehealth. Our consortium comprises 4 HTA bodies, the European Observatory on Health Systems and Policies, 5 academic and not for profit organisations with expertise in EU Regulation, DHT development and assessment methodologies, data protection, data quality, interoperability and cybersecurity. Patients and health professionals are included through the International Diabetes Federation and European Society of Cardiology. We include the DHT industry through companies with products for us to assess through a formal methodology mimicking actual HTA assessments – 3 as partners, some through an open call and industry-wide consultation via DIGITAL EUROPE. We will engage healthcare payers and ministries through our Advisory Board. We will co-create our overarching assessment framework with specialised pathways for different categories of DHT specified through a novel evidence-based typology. We will cater for phased adoption (going beyond fast track models like DiGA), complex life-cycles, iteratively developed AI, consolidated in a comprehensive HTA assessment manual plus specific guides in topics like cybersecurity. We will develop a sustainable repository containing the ASSESS-DHT framework, pathways and unambiguous criteria, a semantically searchable evidence library, checklists and tools to support companies with evidence generation, evidence of health system value from DHT, and online communities of practice.

HTx will create a framework for next generation Health Technology Assessment (HTA) that supports patient-centred, societally oriented, and real-time decision-making for integrated healthcare throughout Europe. HTx will focus on therapeutic areas with high unmet need for which HTA information has to be provided on complex and personalised combinations of health technologies. Based on a select number of relevant case studies, HTx will enhance methods for integrating evidence from RCTs and real-world data (RWD). HTx will also augment statistical and econometric methods for generating robust estimates of effectiveness and cost-effectiveness in order to support relevant HTA decision-making for these complex and personalised combinations of health technologies. HTx will also contribute to improving methods to support personalised treatment advice fitted for sharing with patients and their physicians. This includes the development of statistical and econometric approaches and artificial intelligence/machine learning methods for forecasting treatment effects in specific groups of patients. Simultaneously, in close collaboration with the European Network for HTA (EUnetHTA), HTx will improve synergies between regulatory agencies, HTA bodies and clinical guideline developers. This will include the translation of HTx methods into already existing European guidelines, most prominently those developed by EUnetHTA. HTx will also support initial efforts to discuss reimbursement and funding models that facilitate controlled access to and the pricing of these complex health technologies. Finally, we will evaluate the transferability of HTx results into all EU Member Countries especially in Central and Eastern European (CEE) Countries and promote the dissemination of HTx results to the different European stakeholders with a special focus on the patient community.

IMPACT HTA proposes new and improved methods, tools and guidance for decision-makers across ten research areas in the context of HTA and health system performance measurement, which, overall, contribute to the understanding of costs and health outcomes variations within and across countries and to costs and health outcomes data integration from different sources. IMPACT HTA researches factors on the supply and demand side of health systems that have major effects on the costs and outcomes of health-related interventions. It includes methods for more robust measures of quality of life, patient preferences, patient-reported outcomes, better incorporation of stakeholder views into value assessments, developing methods for cost comparability leading to better generalisability of economic evaluations as well as measuring broader economic and societal impacts and incorporating these in economic evaluations and improvements in performance at health system and provider level. The perspectives of different stakeholder groups in the health system and the broader economy are taken into account, including patients, HTA agencies, the macro-economy, hospitals and hospital networks. These perspectives are obtained through participation in research and preference elicitation (patients, hospitals, hospital networks), or derived explicitly through participation in this consortium (HTA agencies). With respect to data integration, IMPACT HTA develops new methods to integrate data on direct and indirect costs of illness and on health outcomes from various sources, such as randomised controlled trials, observational studies and registries. New methods proposed make use of the strengths of real-world data and address their limitations. The consortium calibre in terms of research and research planning track record, continuous interaction with decision-makers and the patient community will ensure project objectives are realistic and achievable within the project’s duration.