The PharmaLedger project will create a blockchain-based framework for the efficient digitization of the healthcare industry. The goal of the project is to provide a widely trusted platform that will support the design and adoption of blockchain-enabled healthcare solutions while accelerating delivery of innovation that will benefit the entire ecosystem, from manufacturers to patients. PharmaLedger will serve as a single source of truth for the healthcare ecosystem and will be designed for efficient decentralized governance, wide adoption by the stakeholders of the ecosystem, compliance with extant and emerging standards and regulation, and end-to-end connectivity and interoperability. Sustainability of the platform will be ensured by leveraging existing, successful blockchain technologies; open source reference implementation; and a fully documented, actionable methodology for evolutionary digitization of the healthcare industry. The project will address the key challenges of the healthcare ecosystem through prioritized delivery of applications and validation of business use cases, including but not be limited to end-to-end product tracking for combating counterfeit medicines and medical supplies; supply chain integrity; efficiency of recruitment and submission in clinical trials; and machine-learning health data marketplaces. The platform will support integrated use of medical devices across the use cases. The PharmaLedger project brings together 28 partners from 10 EU Member States, including 11 large pharmaceutical companies; highly innovative technology SMEs specializing in blockchain development, security, privacy and business intelligence; universities and research institutes specializing in pharmacoeconomic analysis, research of patient requirements, big data analytics and electronic health records; leading clinical trials companies; supply chain partners; patient representatives; and leading healthcare service providers.

Progress in the life sciences and related technologies offer great potential for therapeutic benefits to patients in need. However, major adaptations to current paradigms of bringing medicines to patients are required in order to realize that potential and to address important challenges in the healthcare ecosystem. Against this background, several initiatives are exploring new pathways to market, collectively referred to as Medicines Adaptive Pathways to Patients (MAPPs). The ADAPT-SMART consortium is aligning a limited number of major stakeholders eager to progress towards MAPPs implementation. It will act as a neutral collaborative platform that will engage industry, SMEs, regulators, Health Technology Assessment bodies (HTAs), payers, governments, clinicians and patients. The ADAPT-SMART consortium will contribute to align understanding of the impact of MAPPs, to share learnings between all stakeholders, and to allow the field to actively work towards MAPPs implementation. The impact of the ADAPT-SMART CSA will be a result of the delivery of • actionable advice/recommendations to IMI on how to best leverage results from past/current projects; • concrete proposals for future (IMI) projects; • actionable advice/recommendations and information to other actors in the healthcare environment; • synthesis of learnings from pilot projects and case studies with relevance to MAPPs; • communication of CSA outcomes by way of publications and conference presentations. This CSA will increase the probability of successful implementation of MAPPs and accelerate access to crucial therapies, thus improving the position of both the patients in need of novel treatments and the research-based pharmaceutical industry.

Europe is generating huge amounts of patient-level information contained in Electronic Health Record (EHR) systems and other types of health databases. These include structured data in the form of diagnoses, medications, laboratory test results, etc., and unstructured data in clinical narratives. The Electronic Health Data in a European Network (EHDEN) Consortium leverages these vast volumes of data to improve future clinical practice and individual patient outcomes by increasing our understanding of disease and treatment pathways. EHDEN will galvanize transparent and reproducible analytics that will generate valid real-world evidence to improve patient care, and enable medical outcomes-based research at an unprecedented scale. The EHDEN Consortium provides the infrastructure and eco-system supporting disease-specific projects in the IMI Big Data for Better Outcomes (BD4BO) programme. The core of EHDEN is the use of a common data model (OMOP-CDM), standardised outcome assessment (ICHOM), and transparent open-source analytics (OHDSI). The objective of the EHDEN consortium is to provide all the necessary services that enable a distributed European data network to perform fast, scalable and highly reproducible research, while respecting privacy regulations, local data provenance and governance. This will include services and tools to perform data standardization, analytical pipelines, tools to share study results, and tools for stakeholder engagement and training. The EHDEN Consortium combines active participation of stakeholder representatives with proven experience in: a) integrating different data types, methods and technologies to utilize diverse clinical datasets; b) platform development to make methods and datasets Findable, Accessible, Interoperable and Reusable (FAIR); and c) engaging a wide variety of stakeholders, including health technology assessment agencies, regulators and patients.

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).

Scientific excellence, and acceptance by health authorities of results derived from research on personalised medicine require standards ensuring validity and reproducibility. The objective of PERMIT is to establish, with all relevant stakeholders and invited experts, recommendations ensuring the robustness of personalised medicine trials, which also requires validation of the stratification methods. In a series of workshops participants, partners and selected experts will address the various aspects of methodology, design, data management, analysis and interpretation in personalised medicine research programmes, with the objective to reach consensus and publish recommendations. A mapping of the literature will inventory methodological practice in the various steps of personalised medicine programmes (WP2) and identify needs in terms of standardised methodology. WP3 will address the design of the stratification and validation cohorts, including the issue of the statistical power and the quality of data. WP4 will focus on the use of the stratification algorithms and the robustness the stratification methods. WP5 will address the translational step needed to select treatments to be tested in each of the clusters. WP6 will be dedicated to randomized clinical trials testing treatments in each cluster (including umbrella / basket designs), or comparing the personalised vs. non-personalised approach. Recommendations and publications in scientific journals, a final meeting and webinars will foster adoption and implementation (WP7). The PERMIT consortium is composed of participants: pan-European research infrastructures (ECRIN, EATRIS, ELIXIR-LU/UNILU), funders (DLR), HTAs (KCE, ISCIII), patients (EPF), regulatory (ISS), data protection (TMF) and scientific experts, whereas partners represent stakeholders interested in the quality of evidence generated by personalised medicine research (industry, medicine agencies, ethics committees, funders, journal editors, HTAs, BBMRI).