On 16 November, the members of the ROADMAP initiative -Real world Outcomes across the Alzheimer’s Disease (AD) – announced the start of a new collaborative pan-European research effort. Its aim is deliver a series of scalable, transferable data integration methods and tools for patient outcomes, developed and tested through pilot projects, which will provide the foundation for a Europe-wide RWE platform on AD. In parallel, ROADMAP will develop tools for stakeholder engagement and for better understanding of the ELSI (ethical, legal and social implications) context and health economics impact of a RWE approach in AD.
Successfully delivering therapies to people with AD will also depend on building a sustainable global payer strategy to address challenges around drug access to markets. This will be dependent on the integration of RWE in health care systems to support approval as well as HTA/payer evidence. The development of a sustainable global payers strategy is one of ROADMAP’s objectives.
As the result of an ageing population, rising costs and more specialised treatments, Europe is now facing an acute healthcare challenge. In no area is this more apparent than in AD and dementia. The European Union (EU), with the world’s most diverse and sophisticated health care systems, is uniquely placed to develop and exploit technologies to support RWE; addressing operational issues of data access, integration and security, alongside hypothesis testing, ELSI, health economic (HE) and budget impact, at a level of detail and complexity not possible elsewhere.
ROADMAP, led by the University of Oxford and Novartis (on behalf of EFPIA), has been designed to combine active participation of stakeholder representatives with exemplar pilot exercises in different settings that allow the identification of the opportunities and threats for widespread adoption of RWE and to enhance clinical and health policy decision-making in AD.
It also addresses the challenge of how to best inform clinical and health policy decisions by conceptualising the entire population as the decision-making laboratory and building a population-based integrated data environment which is optimised for precision and personalised medicine. By developing tools enabling this laboratory to conduct hypothesis specific in-silico experiments, ROADMAP provides a context for identifying and addressing the many unknown challenges of AD. In this process, the interests of healthcare providers, industry, regulators, payers, patients and governments can be addressed transparently, leading to shared best practice, greater efficiency and responsiveness to increasingly fine-grained healthcare questions.
In order to achieve its objectives, ROADMAP has been designed as a highly pragmatic initiative. It combines active stakeholder participation (with special emphasis on patients, HTA, regulators and payers) with the application of established technologies across diverse data types and across healthcare systems, to optimise the use of RWE in AD.
ROADMAP aims to provide the foundation for a much-needed integrated data environment and framework for RWE in AD including: 1) The development of consensual key outcome measures across stakeholder groups; 2) Enabling data integration tools for dataset characterisation and outcome classification, data standards, data sourcing, software application repository; 3) Guidelines on the handling and interpretation of RWE data.
In addition, the following priorities have been identified: Outcome measurement; Identifying the best natural history model; Identifying the best pharmacoeconomic modelling strategy; Using short-term data to model long-term effects; Using short-term data to model long-term economic impact; Association between cognition, QoL (quality of life) and function over the AD spectrum; Identifying combination markers for diagnosis in asymptomatic patient population; Identifying combination markers for prognosis; and consolidating the regulatory/HTA/payer evidence requirements.
ROADMAP is mainly sponsored by the European Union’s Horizon 2020 research and innovation programme and the European pharmaceutical industry (via EFPIA) under the auspices of the Innovative Medicines Initiative (IMI) 2 Joint Undertaking.