Alzheimer’s disease (AD) and Parkinson’s disease (PD) are common neurodegenerative conditions, posing a major societal burden. There is a lack of treatments to slow disease progression, and therapeutic development has been impeded by a lack of biomarkers that can detect individuals early in the disease, measure treatment effects, and stratify patients. European cohorts recruited for research on aging and neurodegeneration provide a huge potential for biomarker discovery and validation by providing bio-samples along with deep clinical and imaging phenotypes. However, these cohorts are difficult to access. An overview of the availability of data and samples is lacking, and protocols and regulations for data and sample collection, storage, and sharing vary. The European Platform for Neurodegenerative Diseases (EPND) will tackle the above issues by developing a self-sustaining European-based platform to facilitate discovery and access of relevant bio-samples and data. EPND will be built on an existing informatics infrastructure, the AD Workbench, which EPND will adapt to support resource- and participant-level discovery, data harmonisation, central and federated data and sample storage, and data analysis. The sample and data discovery tools will be connected to a network of over 60 cohorts on AD, PD, and related disorders. Together, these cohorts will facilitate access to data and samples of over 120,000 research participants including CSF (n=30,000), plasma (n=120,000), stools (n=6,000), urine (n=27,000), saliva (n=17,000) and digital biomarkers (n=2,000). Prospective data collection will also occur during the project. This approach provides the community with a new and powerful environment for collaborative cross study analysis of harmonised biomarkers, datasets and samples. EPND will provide visibility into the quality and standardization of the data and samples available in the platform from the cohorts available and will also provide protocols for ongoing data and sample collection. This will guarantee quality of samples available, an important factor for validation and regulatory approval for biomarkers. EPND will be guided by ethical, legal and regulatory experts, patients, and other stakeholders to ensure responsible practices and processes underpin all discovery, sharing and access of data and samples, whilst simultaneously ensuring the platform is self-sustainable by the end of the project. Thereby, EPND will provide the community with a long-term, powerful environment to aid biomarker research for neurodegenerative disorders, enabling critical advances in the development of treatments for AD and PD.

Chronic Kidney Disease (CKD) is one of the major causes of death in high income countries and its prevalence is growing rapidly. New therapies for the treatment of CKD have shown clear efficacy at a population level. Yet, individual patients respond differently with many patients showing suboptimal efficacy. This indicates that a ‘one size fits all’ approach is no longer sustainable. Currently there are no validated pharmacodynamic biomarkers in CKD that aid in guiding optimal therapy for individual patients. Furthermore, there remains a large gap between biomarker research and actual implementation of biomarker based care in daily clinical practice. Thus, there is an urgent need to validate and translate biomarkers as tools for daily clinical practice. PRIME-CKD has a unique advantage to address this challenge as it builds on recently discovered pharmacodynamic biomarkers for CKD treatment, and has access to a vast amount of clinical data, bio-samples. PRIME CKD will: 1) Provide a breakthrough in the pharmacotherapy of CKD with the validation and implementation of pharmacodynamic biomarkers which will advance personalized medicine. 2) Scientifically validate innovative clinical trial elements, pipelines and qualification procedures that can be used by researchers, diagnostics industries and regulatory authorities. 3) Establish a Stakeholder Network to truly engage patients, clinicians and other stakeholders in implementation of personalized medicines 4) Develop a roadmap for biomarker use in daily clinical practice for the optimal treatment of CKD. These results will enable clinicians to match patients with CKD with the most effective available drug within the shortest possible timeframe leading to less disease burden and progression of disease, enhanced quality of life and increased cost-efficiency. PRIME-CKD results will thus unlock the potential of biomarkers in healthcare and close the gap between biomarker research and clinical use in daily practice.

The Gravitate-Health mission is to equip and empower citizens with digital information tools that make them confident, active, and responsive in their patient journey, specifically encouraging safe use of medicines for better health outcomes and quality of life. It is our vision that engagement of citizens in their own health can only be achieved with access to actionable, understandable, relevant, reliable and evidence-based information that meets their specific needs, health context, and literacy level. This project's ambition is to provide a key piece to advance this vision: the Gravitate Lens (G-lens), which focuses (but does not conceal or filter) approved electronic product information (ePI) content, and offers a route for patients to access trustworthy, up-to-date information that better meet their individual needs. Gravitate-Health is an integrated digital health information project. The principle objective is to demonstrate how use of an integrated, digital, user-centric health information solution with two-way communication could enable tangible improvements in availability and understanding of health information from a set of trusted sources, starting with regulator-approved medicinal product information (e.g. package leaflet content) and EHR-IPS (International Patient Summary). The secondary objectives are to demonstrate that the improved availability and understanding of health information from trusted sources translate to higher levels of adherence to treatment, safer use of medication (Pharmacovigilance), better health outcomes and quality of life, and to develop new and deeper insights into how use of available health information can be optimized to act as effective risk minimization measures. The project allows for efficient and timely development of the G-lens, provides testing grounds for new services and an evaluation framework to test the efficiency, efficacy and safety of Gravitate-Health services. Our main outputs will be an open source digital platform supporting G-lens functionally, demonstrated in a number of testing scenarios, and a White Paper with recommendations on realistic strategies to strengthen access, understanding and future use of digital services like ePIs as a tool for Risk Minimization. The Gravitate-Health is a public – private partnership with 45 members from Europe and the US, co-led by University of Oslo (coordinator) and Pfizer (industry lead), funded by the Innovative Medicines Initiative (IMI) – a joint undertaking of the European Commission, the European Federation of Pharmaceutical Industries and Associations (EFPIA), IMI Associated Partners.

Lack of specific relevant know-how in regulatory science delays the development of new treatment strategies or limits the chances that promising innovations will reach patients. STARS aims to improve the direct regulatory impact of results obtained in medical research. Seventeen European countries are represented in the consortium through their national competent authorities, alongside academic and industry representatives, and associations with relevant experience. The work plan includes the development of a Comprehensive Inventory of existing support activities based on a detailed analysis of the currently established programmes. This analysis is also the basis for development of a Common Strategy to strengthen regulatory sciences and two curricula, the Core Curriculum specifying essential knowledge for the professional training of clinical scientists and the Comprehensive Curriculum defining relevant knowledge for specific post-graduate programmes. Three pilot projects aim (i) to transfer an identified best practice example for training programmes to other EEA countries, (ii) to implement a new support activity addressing a gap in regulatory knowledge of significant relevance and (iii) to implement the Comprehensive Curriculum. STARS will deliver consensual recommendations ensuring sustainable support of academic research and will propose additional support mechanisms based on a comprehensive analysis of needs. STARS has the objective and the potential to complement, coordinate and harmonise regulatory efforts among Member States and at European level to support academic health research for the benefit of patients. The aim is to reach academic researchers very early in the planning of relevant grant applications. A further aim is to strengthen regulatory knowledge in general by reaching clinical scientists during professional training and qualification.

This innovation action will give a powerful impulse to implementation of ISO IDMP (ID of Medicinal Products) standards in EU Member States drug databases, supporting safe cross-border ePrescription/eDispensation and effective pharmacovigilance. Once EU-interoperable data on medicines taken by patients become available, further benefits will accrue through better health data for improved clinical decision support, patient empowerment, public health and clinical research. New opportunities will arise for pharma industry, software developers, SMEs providing smart apps and others, thereby fostering their innovation capacity and competitiveness. The many challenges still to be faced on this road will be tackled by a powerful consortium assembling all relevant actors, with critical mass for impact throughout the EU. After 10 years of development, the IDMP suite of standards is ready for implementation. Though some isolated implementation work has started, the time is now ripe for a more concerted effort towards large-scale implementation, contributing to this global interoperability endeavour and delivering benefits to EU citizens. Project ambition centres on conversion of key regulatory and clinical processes to use IDMP. These information value chains must be converted over their full length from data input to data repositories to data usage. Project work spans all three areas, focussing on the most challenging, the implementation of EU and national SPOR (substances, products, organisations, referentials) data bases, including establishing an EU Substance Reference System (EU-SRS). Such information is fundamental to cross-border ePrescription where safe dispensation may require reliable identification of substances in available products. 19 countries are represented, including 26 national Drug and eHealth Agencies. Stakeholders are involved through their associations. Duration is 4 years, budget € 21 m, with requested funding € 19 m.