Ultimately, EHDEN is striving to ensure 21st century research can be conducted with 21st century tools, engaging with the wider community in terms of performing observational research, supporting R&D, trial optimisation and a new clinical trial paradigm.
One would imagine that real-world data (RWD), data that is captured predominately in the clinical setting and environment of a patient, versus a clinical trial, and the real-world evidence (RWE) derived from RWD via analytics, is new. Indeed, we have been using observational data since the ancient Egyptians, Greeks and Romans, whereas the ‘modern’ clinical trial has been refined since 1946.
Right now, we are facing unprecedented expectations of our healthcare systems and technologies to reduce increasing morbidity and to avoid mortality. And this within an international environment of socio-political/economic unrest, and burgeoning environmental collapse. Furthermore, developers and manufacturers of pharmaceuticals, medical devices and new health technologies are being asked to substantiate value and to link outcomes to reimbursement requiring more and more evidence generation.
Moreover, data generation, either within a clinical environment, for instance within electronic health records, claims databases, regional databases, registries or cohorts, is being complemented by patient-generated data, whether human (e.g. via an app) or machine-generated (e.g. a wearable or sensor). Greater, and deeper molecular, genotypic knowledge, complementary to phenotypic knowledge is now accelerating after the initial stutters following the Human Genome Project. The human genome has also been complemented by multiple systemic biological frameworks, such as the connectome, microbiome, proteome, etc., further enhancing our understanding of ourselves. All of this explosion in data is a challenge, but also an opportunity to create ever higher resolution phenomic profiling of patients for clinical care decision making, and for research.
Despite this, and with such rapidly accelerating data growth there are challenges around data parochialism, or ‘ownership’, privacy and security challenges, further complicated by factors such as the interpretation of guidance such as the EU’s General Data Protection Regulation, and overall in protecting the sanctity of citizens data, whilst deriving knowledge from it. Unfortunately, our resolution of a digital avatar is the merest shadow today due to the difficulty of interoperability, governance and transparency in intended use.
Very topical is the ascent of machine learning, or ‘Artificial Intelligence’, ascending the hype cycle with the promise of algorithms augmenting clinical intelligence, providing clinical decision making or indeed replacing clinicians altogether. It remains to be seen as to what extent in the near future algorithms will impact on healthcare, but increasing regulatory scrutiny will focus on their efficacy and in particular the quality of data input driving the training and validation sets. Certainly, the volume of labelled data is insufficient in diversity and variety today.
Against this backdrop, we, the research and clinical community need solutions that can deliver on expectations for the 21st century to support evidence generation and clinical decision-making, as well as research and development.
The European Health Data & Evidence Network (EHDEN) project, funded via the Innovative Medicines Initiative (IMI), is the largest of its kind in Europe working in the domain of RWD/RWE. It is a public-private partnership consortium of 22 partners, from 2018 to 2024, led by Erasmus Medical Centre (EMC) and Janssen, working to create an open science community symbiotic with the Observational Health Data Science and Informatics (OHDSI) global framework to facilitate observational/RWD-based research at scale and acceleration, without impinging on quality.
At its core is the standardisation of RWD via the use of the Observational Medical Outcomes Partnership (OMOP) common data model (CDM), standardised analytics and a sustainable research community for the coming decades. EHDEN can be characterised within three pillars:
(1) Infrastructure, focusing on the architectural development of a platform to support FAIR RWD, security and privacy, and a federated network with locally OMOP-mapped data from diverse Data Partners (e.g. secondary/tertiary care, primary care, claims, registry/cohorts, et al) utilisation of OHDSI-standardised research tools. Open calls to invite Data Partners to join the network and be mapped, with financial support from a Harmonisation Fund, and for small/medium enterprises (SMEs) to be trained and certified in a consistent approach to ETL/mapping to the CDM, thus creating a new technical marketplace
(2) Research, EHDEN will run multiple use cases of convergent interest in the project, such as drug utilisation, drug safety, or health technology assessment (HTA) with Data Partners, and with, e.g., the European Medicines Agency, to validate and verify the mapping and the architecture of the federated network, as well as developing methodologies further. EHDEN will also work on incorporating the ICHOM health standards into the OMOP CDM to speed up adoption and utilisation within the network of relevant outcome standards
(3) Education and community-building, the project is launching the EHDEN Academy as a free educational resource to support the project, our partners and the European RWD community, as well as developing value propositions, sustainability plans and facilitation of an open science community longer term.
Critical to the output of EHDEN is evidence generation, in terms of using research-grade RWD to impact on clinical practice now, not in years. Its success will be measured by the ability of the project, and its sustainable legacy to do this in Europe, linked within the OHDSI global framework to other global regions in parallel development.
Interoperability and standardisation are technical challenges, but EHDEN is working within a socio-technical construct which also requires the trust of all involved, especially Data Partners and Researchers, whilst ensuring compliance with GDPR, local and regional governance and approval processes.
Ultimately, EHDEN is striving to ensure 21st century research can be conducted with 21st century tools, engaging with the wider community in terms of performing observational research, supporting R&D, trial optimisation and a new clinical trial paradigm. As Europe prioritises digital healthcare over the term of the new Parliament, we hope EHDEN will become a centre point for a strategic development of a federated network and open science community supporting research, clinical care, evidence generation and better outcomes for its citizens.