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Real world Outcomes across the AD spectrum for better care: Multimodal data Access Platform - Sofia ref.: 116020

Periodic Reporting for period 2 - ROADMAP (Real world Outcomes across the AD spectrum for better care: Multimodal data Access Platform - Sofia ref.: 116020)

Reporting period: 2017-11-01 to 2018-10-31

In the context of demographic change, Europe is facing an acute healthcare challenge. In no area is this more apparent than in Alzheimer’s disease (AD) and dementia. AD and related dementias affect nearly 50 million individuals worldwide with prevalence projected to double over the next twenty years. So far there is no cure for AD or a therapy to slow the progression of the disease and challenges in the evaluation of early disease interventions within the current assessment systems have not changed. Consequently, the precision medicine approach to health funding requires new models that encompass all the available evidence for the identification of best suited treatments for different groups within the AD continuum. ROADMAP Phase I aimed to create the conditions for open collaboration between stakeholders (regulatory/HTA bodies, payers, patient organisations, pharmaceutical companies, academia), allowing consensual, efficient use of available real-world evidence (RWE) to assess the value of new AD treatments being evaluated within the ethical, legal, and social structures.

Specifically, the ROADMAP has worked toward these objectives:

- To define and reach consent on AD priority outcomes and classify their relevance within the stages of the disease.

- To identify potential data sources across Europe and developed technical solutions to facilitate the data harmonisation and integration, allowing the combination of Randomized Controlled Trials, AD-cohort and Electronic Health Records -based data for real-world evidence studies, assessing pharmacoeconomic evaluation.

- To establish a landscape of AD-outcomes across Europe and their relevance in each stage of the disease, by developing the Data Cube. To perform external validation of selected core disease progression models, combining diverse datasets, facilitating the analysis of disease trajectories and effect of interventions on disease trajectories.

- To elucidate the specifications for an economic model to evaluate the C-E of disease-modifying and other pharmacological interventions to treat patients with AD predementia or dementia.

- To develop guiding principles and recommendations from HTA/payers/regulators for the development and incorporation of RWE into clinical and market access development plans for AD.

- To design and implement a communication strategy focusing on the needs of patients and professionals, by coordinating external communication and dissemination activities. Facilitated liaison with patients and dementia community, as well as other relevant stakeholders.

- Identifying ethical, legal, and social issues arising from the combination of datasets from multiple countries for disease, health economic, and treatment modeling for AD, including requirements set out in national ethical and legal frameworks governing local ethics approval.

- To assess a full plan for ROADMAP phase 2 that addresses identified gaps and pitfalls, and exploits promising solutions to their full potential for development of a European RWE platform in AD.
ROADMAP has been highly active in establishing the framework for its activities. Some of the key results include:

- Defining a minimum set of measurable real-world AD outcomes, by means of pragmatic literature reviews, systemic literature reviews, group consultation, surveys and individual interviews with patients, supporters and healthcare professionals
- Development of a matrix of prioritised outcomes according to stakeholder group and mapping them against the data available from DPUK cohorts and other relevant European datasets.
- Developing recommendations on RWE-appropriate, AD-related cognitive, functional, and behavioural outcomes.
- Assessment of cohorts and other data sources to identify intermediate markers that may be transferable to clinical practice.
- Identifying data sources and outlining a data strategy for RWE outcomes.
- Providing an overview of data sources available throughout Europe, the tools available for identifying and combining these data, and opportunities and strategies for pooling data.
- Developing new methods for collecting RWE to improve how new treatments can be valued in AD.
- Engagement with stakeholders. The use of smart devices for self-report, social media, and direct objective assessment applications was of particular interest.
- Providing recommendations for disease progression modelling.
- Evaluation of both hypothesis-driven and machine-learning approaches. Both were informed by a review of current models of AD pathology, and by using available datasets for validation of selected models.
- Developing the concept of an integrated core health economic model of healthcare use/costs and health outcomes in AD.
- Assessment of quality of life, resource utilisation, and costs associated with AD, and previous AD economic modelling studies, and used systematic reviews to improve the evidence base of long-term AD cost-effectiveness modeling.
- Identifying guiding principles on the use of RWE in AD in the regulatory/HTA context.
- Collection and collation of regulatory and HTA-related data to identify possible recommendations for the development and incorporation of RWE into clinical and market-access development plans for AD.
- Exploring patients' attitudes towards combination of data sets via a focus group interview and several literature review.

All of these high-impact results have been shared and disseminated by means of more than 150 dissemination activities, including scientific contributions (articles in journals, chapters in books, oral and poster presentations at scientific meetings, publications in conference proceeding…) as well as nonscientific and non-peer reviewed publications, the organisation of conferences/workshops, external newsletters, press releases, flyers/brochures, media (TV, radio, press, multimedia), video/film and participation in activities organised jointly with other projects.
By creating a framework for research and dialogue building data integration tools tailored to European health systems and by developing RWE data standards for AD, ROADMAP has the potential to establish Europe and European scientists as world leading. The output of this collaboration will also provide pragmatic recommendations to guide industry to invest in capturing outcome measures that have the potential to better prepare innovative AD therapies, reducing attrition and allowing for improved practice towards approval and launch.

ROADMAP project pursued producing more transparent, open and widely accepted outcomes which will immediately yield substantial benefit for all stakeholders involved in AD research and healthcare by making the pathway to patient benefit. The immediate impacts from this project are largely instrumental with patient health and wealth benefits observed indirectly through quicker and more streamlined decision making to foster patient access to new therapies. However, through the integration of use cases across work packages, there will also be improved understanding of disease progression and treatment benefits.