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 bes

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.

The aim of ROADMAP is to provide the foundation for a Europe-wide real world evidence (RWE) platform in AD by piloting multi-modal data integration tools and engaging with all key stakeholder groups for consensual definition of patient outcomes, tools and methods that are actionable and relevant. ROADMAP will leverage best practice and exploit synergies with other projects and initiatives at the national and European levels in pursue of scalable and transferable solutions for dataset characterisation, outcome classification, data standards, data sourcing, software application and guidelines on the handling and interpretation of RWE data. In parallel, the project will deepen understanding of the ethical, legal and social implications (ELSI) and health economics (HE) impact of a RWE approach for a meaningful contribution to the Big Data for Better Outcomes programme in IMI2. The ROADMAP programme will consist of 8 integrated work packages by which the Consortium will work to (1) identify relevant AD outcomes and progression markers; (2) identify and pool AD-related RWE across data sources; (3) develop and validate disease progression models combining diverse datasets and strategies; (4) develop risk/value-based costing and health economics models for HTA/regulators, service providers, industry and carers; (5) establish stakeholder-based guidelines for RWE; (6) develop a communication strategy focussing on the needs of patients and professionals; (7) develop an ELSI framework for the development and application of RWE in AD. The ROADMAP consortium brings together outstanding expertise, experience and traction in the field, including many of Europe’s top institutions, authorities, companies, experts in AD, biomedical informatics and analysis of epidemiologic and routinely collected health data sources, providing direct connections to key initiatives such as DPUK, EMIF, EPAD and others.

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.

There is an urgent need for novel approaches assessing functional decline in early AD. The main goal of the RADAR-AD project is to develop a digital platform to detect subtle functional deficits in early Alzheimer’s disease (AD) individuals by integrating a meaningful combination of smartphone, wearable and/or home sensor based parameters. The system developed will be suitable for future longitudinal studies, including trials. The objectives are to 1) Identify the most relevant functional domains and the most promising remote measurement tools (RMTs) for these domains based on reviewing the literature and piloting of RMTs in small studies; 2) Optimise the RADAR-CNS platform for use in AD studies; 3) Test the platform and selected RMTs in a real world environment clinical study with 240 participants across the AD spectrum ranging from the preclinical AD to the dementia stage; 4) Perform statistical modelling to estimate longitudinal predictions; 5) Discuss results with regulatory agencies in order to obtain guidance about how to develop a path for formal qualification as outcome measurements in future therapeutic interventions. Considering the limited budget and project duration, we will rely upon already available technology platforms and on available longitudinal datasets where possible. The consortium includes experts in clinical dementia studies, computer science, bioinformatics, regulatory policies, ethics, and patient and public involvement (PPI). Additional strengths of the consortium are the deep and broad interface with RADAR-CNS and related IMI projects, and the access to large amount of patient-level data from key European cohort studies for modelling purposes.