Engaging with patient organisations within IMI consortia to inform quality, relevance and value in Alzheimer’s research – insights from MOPEAD, EPAD and ROADMAP.

ROADMAP partners Alzheimer Europe and Lilly joined forces recently at the AE annual conference in Berlin on 2^nd – 4^th October, at a symposium bringing together three IMI projects: EPAD, MOPEAD, and ROADMAP. ROADMAP representative, Catherine Reed gave a talk entitled ‘ROADMAP: best use of Real World Evidence to address specific healthcare challenges’ focusing on the importance of the real-world evidence (RWE) within AD that characterises the project and the role of patient representatives to achieve project objectives. Here we report briefly on this presentation.

Firstly, Catherine emphasised the characteristic distinguishing ROADMAP from the other IMI projects that were part of the symposium, stressing ROADMAP’s emphasis on the use of incorporating RWE into the evidence base for healthcare decision -making. This included an explanation of what RWE is, what it can be used for, what the challenges are in using RWE within AD and how ROADMAP has been designed to meet these challenges.

In relation to challenges in particular, Catherine went into greater detail, explaining that while RWE comes from numerous data sources (including patient registries, insurance data, clinical trials, economic studies, and so on), there is no consensus regarding what information should be collected or on which should be considered of greatest significance for different stakeholders. In particular, this challenge is compounded by the diagnostic and prognostic significance of different kinds of information throughout the progressive stages of AD, as different indicators and measures are used throughout the course of the disease. For example, at a pre-symptomatic stage it is AD biomarkers (e.g. brain imaging) is necessary to identify the presence of AD, whereas later on in the disease, tracking cognitive, functional, and behavioural changes become important, and consideration of long-term care aspects are more relevant to for those most severely affected. Stakeholders (e.g. clinicians, regulators, payers, researchers, patient and caregivers) will have all place different value on the type of information gathered at different stages of disease.

The talk then moved on to present ROADMAP’s data pyramid which visually represents the hierarchy of RWE data sources, from health registries (most representative of the general population) to clinical trials (highly selected samples), and posed the question of how the potential benefit of these diverse data sets can be leveraged. Catherine went on to present ROADMAP’s proposed answer to this question, by explaining the ‘data cube’ a visual tool which allows the identification of key AD outcomes across the disease stages within available data sources.

After giving this overview of ROADMAP’s goals using RWE, Catherine provided some insight into the structure of the eight Work Packages that will deliver the multiple objectives of the project before making some concluding remarks emphasising ROADMAP’s importance with healthcare decision-making process. In particular, emphasis was given to the need for initiatives such as ROADMAP to have a strong communications strategy that can reach beyond the clinical and academic communities to grow public understanding of this important work. It was also emphasised that ROADMAP will only be successful if there is widespread acceptance of its findings and recommendations across all key stakeholders – not just academia and industry, but also regulators, payers, patients and their carers –  and if the project’s output are useable by decision-makers in understanding AD and the impact of new treatments.

The session concluded with a question and answer panel discussion across EPAD, MOPEAD, and ROADMAP, in which Catherine took part. In the questions directed to her about ROADMAP, Catherine made it clear that a key aim of the project is for it to have a clear voice from patient organisations and their members, as by listening carefully we can be sure of identifying the outcomes of the most value for them, at the right times, as they experience the course of AD.

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