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.

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.