Barcelona, 16 November 2016 – The members of the ROADMAP initiative today announced the start of a new collaborative pan-European research effort to 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 Alzheimer’s disease (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.

About Alzheimer’s disease and dementia

Alzheimer’s disease is a progressive degenerative disease which causes loss of neurons in the brain. The symptoms may eventually manifest as Alzheimer’s dementia which impacts cognition, function and behaviour, becomes progressively worse over time and cannot be reversed. There are 7.7 million new cases of dementia globally each year, suggesting one new case every four seconds. There are an estimated 46.8 million people worldwide living with dementia in 2015. This number will almost double every 20 years, reaching 74.7 million in 2030 and 131.5 million in 2050.[1] Currently approved treatments may temporarily stabilise or slow the worsening of symptoms, but do not alter the course of the disease. Attempts to bring new drugs to market for the treatment and prevention of Alzheimer’s dementia have been disappointing despite massive commercial, public and academic investment of time and resources.

About ROADMAP

ROADMAP aims to provide the foundation for a much-needed integrated data environment and framework for RWE in AD including:

• The development of consensual key outcome measures across stakeholder groups;
• Enabling data integration tools for dataset characterisation and outcome classification, data standards, data sourcing, software application repository;
• 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;
• 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 2 Joint Undertaking.

The ROADMAP programme has budget of €7.77M distributed across a total of 22 partners from the private and academic sectors:

For more information, check out www.roadmap-alzheimer.org

About the Innovative Medicines Initiative

The Innovative Medicines Initiative (IMI) is Europe's largest public-private initiative aiming to speed up the development of better and safer medicines for patients. IMI supports collaborative research projects and builds networks of industrial and academic experts in order to boost pharmaceutical innovation in Europe. IMI is a joint undertaking between the European Union and the European Federation of Pharmaceutical Industries and Associations, EFPIA.

More information can be found at www.imi.europa.eu

Acknowledgement

The project leading to this application has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 116020. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA

Disclaimer

This communication reflects the views of the ROADMAP Consortium and neither IMInor the European Unionand EFPIAare liablefor any use that may be made of the information contained herein.

CONTACTS:

Prof. John Gallacher                                                       Frederic de Reydet de Vulpillieres

John.gallacher@psych.ox.ac.uk                                 Frederic.de_reydet_de_vulpillieres@novartis.com