Periodic Reporting for period 2 - EPND (European platform for neurodegenerative disorders)
Berichtszeitraum: 2022-11-01 bis 2023-10-31
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 ~70 cohorts on AD, PD, and related disorders.
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 ~70 cohorts on AD, PD, and related disorders.
Objective 1. Create a set of agreed ethical, legal, regulatory, business and sustainability principles to establish governance and operational processes to enable sharing and access to data and samples
On the ethical and legal side of this objective, WP2 has continued to inform the project with the development of the detailed exploration of the ecosystem that EPND operates in. In developing this first version of the output (D2.2) entitled “The Current State of Biomedical Data and Sample Sharing for Research Purposes in Europe: Perspectives from the European Neurodegenerative Disease Community” the team has developed insights and recommendations that will inform the implementation of necessary governance processes and is helping steer the project.
The contribution of the WP2 team also continues with support for assessing compliance needs for the technical hub and the further contractual developments that are facilitating sample and data exchange for the case studies.
Additionally, documented in M11 “White Paper v1.1”, governance aspects that will be important to the ability of EPND to facilitate data/sample sharing such as the model of management of a data and sample access committee are being refined to ensure operational viability.
WP7 has engaged in extensive research during year 2 to understand the roles and operating models of other platforms in the data and sample sharing ecosystem and have developed a benchmarking report. We expect this in partnership with work championed by the project leadership team to help us refine the operating models, and consequential business operations, which will ultimately lead to EPNDs sustainability.
In year 2, the PMT co-leads convened a cross-WP taskforce to identify the issues that need to be resolved for broader case study data permissioning. The outcome of the taskforce deliberations led to a set of recommendations by the PMT, which will be carried forward and implemented in partnership with WP2, WP4 and WP5. These recommendations will also guide case study 5 and 6 selection criteria.
Objective 2. Establish a network that provides central and federated access to high quality samples and data from ~70 cohorts by combining existing data discovery and sharing initiatives made interoperable as part of the AD Workbench.
The initial cohort network established at the Bronze level has been augmented by establishing an automatic 'syndication' feed of cohort metadata from the EMIF project. And with a Silver level catalogue now built and populated this provides a solid basis that we can further enhanced in scale and utility during the remaining years of the project, and beyond. Technical work has focused on practical requirements like the Data Access Request (DAR) process and connectivity, with some efforts now extending into biobanks (NCER-PD, IBBL, EPAD, and partner cohort biobanks) to formulate a similar design specification for the Sample Access Request (SAR) process. These initiatives pave the way for EPND to leverage our network and provide access to samples and data.
Objective 3. Provide visibility into the quality and standardization of the data and samples available in the platform from existing cohorts.
As planned, Year 2 has had a focus on dissemination of the SOPs developed with some extension of the best practice guidelines and the development of input on digital biomarkers, a successful workshop was performed and received very positive feedback. The development of the Silver and Gold platform categories will enable the integration of these qualitative assessments to enhance visibility.
Objective 4. Perform a robust evaluation of the developing EPND platform through the execution of case studies on the Amyloid-Tau-Neurodegeneration (ATN) staging.
During the second year, much more progress has been made in evaluation of the evolving platform despite continues delays due to contractual challenges. We have been able to explore the contractual needs of a range of partners, which will be an important component of the future platform. We were able to test our logistical capabilities, through shipping samples from a range of cohorts to a central partner and then out to partners to perform assays. Data standardisation and harmonisation ahead of ultimate integration as well as exploring permissioning and access management, has been the final step in evaluating the needs and demands on a functional platform.
Objective 5. Embrace sustainability from the start of the project by designing and piloting exploitation strategies such that the platform is self-sustainable by the end of the project
EPND’s value proposition and initial set of offerings (to help inform sustainability discussions) were developed by the PMT and refined based on feedback from the Management Board and relevant work packages. WP1 helped shape the offerings with respect to the technical hub and provide temporality to them based on the technical hub roadmap and timelines.
Year two for the sustainability efforts has focused on several key areas.
· Lead by the leadership team the development of a clearer scope and a more focused understanding of the ‘customers’ of EPND and how their use cases might be addressed by an ultimate sustainable delivery of the project.
· This has synergised with an extensive benchmarking exercise to explore exploitation strategies of similar platforms and projects.
On the ethical and legal side of this objective, WP2 has continued to inform the project with the development of the detailed exploration of the ecosystem that EPND operates in. In developing this first version of the output (D2.2) entitled “The Current State of Biomedical Data and Sample Sharing for Research Purposes in Europe: Perspectives from the European Neurodegenerative Disease Community” the team has developed insights and recommendations that will inform the implementation of necessary governance processes and is helping steer the project.
The contribution of the WP2 team also continues with support for assessing compliance needs for the technical hub and the further contractual developments that are facilitating sample and data exchange for the case studies.
Additionally, documented in M11 “White Paper v1.1”, governance aspects that will be important to the ability of EPND to facilitate data/sample sharing such as the model of management of a data and sample access committee are being refined to ensure operational viability.
WP7 has engaged in extensive research during year 2 to understand the roles and operating models of other platforms in the data and sample sharing ecosystem and have developed a benchmarking report. We expect this in partnership with work championed by the project leadership team to help us refine the operating models, and consequential business operations, which will ultimately lead to EPNDs sustainability.
In year 2, the PMT co-leads convened a cross-WP taskforce to identify the issues that need to be resolved for broader case study data permissioning. The outcome of the taskforce deliberations led to a set of recommendations by the PMT, which will be carried forward and implemented in partnership with WP2, WP4 and WP5. These recommendations will also guide case study 5 and 6 selection criteria.
Objective 2. Establish a network that provides central and federated access to high quality samples and data from ~70 cohorts by combining existing data discovery and sharing initiatives made interoperable as part of the AD Workbench.
The initial cohort network established at the Bronze level has been augmented by establishing an automatic 'syndication' feed of cohort metadata from the EMIF project. And with a Silver level catalogue now built and populated this provides a solid basis that we can further enhanced in scale and utility during the remaining years of the project, and beyond. Technical work has focused on practical requirements like the Data Access Request (DAR) process and connectivity, with some efforts now extending into biobanks (NCER-PD, IBBL, EPAD, and partner cohort biobanks) to formulate a similar design specification for the Sample Access Request (SAR) process. These initiatives pave the way for EPND to leverage our network and provide access to samples and data.
Objective 3. Provide visibility into the quality and standardization of the data and samples available in the platform from existing cohorts.
As planned, Year 2 has had a focus on dissemination of the SOPs developed with some extension of the best practice guidelines and the development of input on digital biomarkers, a successful workshop was performed and received very positive feedback. The development of the Silver and Gold platform categories will enable the integration of these qualitative assessments to enhance visibility.
Objective 4. Perform a robust evaluation of the developing EPND platform through the execution of case studies on the Amyloid-Tau-Neurodegeneration (ATN) staging.
During the second year, much more progress has been made in evaluation of the evolving platform despite continues delays due to contractual challenges. We have been able to explore the contractual needs of a range of partners, which will be an important component of the future platform. We were able to test our logistical capabilities, through shipping samples from a range of cohorts to a central partner and then out to partners to perform assays. Data standardisation and harmonisation ahead of ultimate integration as well as exploring permissioning and access management, has been the final step in evaluating the needs and demands on a functional platform.
Objective 5. Embrace sustainability from the start of the project by designing and piloting exploitation strategies such that the platform is self-sustainable by the end of the project
EPND’s value proposition and initial set of offerings (to help inform sustainability discussions) were developed by the PMT and refined based on feedback from the Management Board and relevant work packages. WP1 helped shape the offerings with respect to the technical hub and provide temporality to them based on the technical hub roadmap and timelines.
Year two for the sustainability efforts has focused on several key areas.
· Lead by the leadership team the development of a clearer scope and a more focused understanding of the ‘customers’ of EPND and how their use cases might be addressed by an ultimate sustainable delivery of the project.
· This has synergised with an extensive benchmarking exercise to explore exploitation strategies of similar platforms and projects.
EPND’s year 2 has built on the running start enabled by the existing cohort relationships as well as the in-kind data platform contribution (AD WorkBench). The launch of the enhanced cohort catalogue in November/December 2023 has positioned the project to be able to enable data and sample discovery well before the end of year three. The case studies 5 and 6 (as part of WP5) will serve as proof of concepts for the platform’s functionality, which will be put to test in year three. Industry/external funding for case study 5 will be explored, and if successfully implemented, we will have a clear example of one sustainable offering from EPND in year three.