Background
What challenge is ROADMAP facing?
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 and challenges in the evaluation of early disease interventions within the current assessment systems pertain. At the same time the relevance of a precision medicine approach to health funding grows, giving rise for the need of new models that encompass all the available evidence for the identification of best suited treatments for different groups.  

Read more about the state of precision medicine in AD
What is the EXAG?
The ROADMAP Regulatory‐HTA‐Payer Expert Advisory Group (EXAG) has the following aims for the duration of the project:

Objectives

  • Engage actively in discussions, provide feedback and input into topics brought forth by the Work Packages, including on research activities and outputs;
  • Ensure that the ROADMAP outputs are not only of high scientific quality, but will also have meaningful applicability in the regulatory, HTA, and payer contexts.
What is Alzheimer's disease (AD)?
AD (the most common cause of dementia) is a progressive disease characterised by loss of function and death of nerve cells in several areas of the brain. The neurodegeneration leads to loss of cognitive functions such as memory and learning. The new Alzheimer’s Diagnostic Guidelines refer to the spectrum of AD in three stages; (1) preclinical AD, (2) mild cognitive impairment (MCI) due to AD, and (3) dementia due to AD.

Read more about the changing definition of AD
What is Dementia?
“Dementia” is a syndrome (i.e. a pattern of symptoms) and typically involves loss of memory, mood changes and problems with thinking, orientation, comprehension, calculation, learning capacity, language and judgement. It is the umbrella term used to describe the symptoms that occur when the brain is damaged as a result of one or more diseases or conditions, resulting in memory and intellectual impairments which are sufficiently severe as to interfere with daily life.

Read more about the prevalence of dementia in Europe
What is Real-World Evidence (RWE)?
Real-World Evidence (RWE) is derived out of Real-World Data (RWD) by the analysation of this kind of data. RWD is a broad concept, which depends on differing conceptualisations across its research paradigm. In the realm of ROADMAP, we have a definition of data which are coming from structured data sources, and are organised in a pyramid of evidence. These include large broad population-based databases, for example from Nordic countries, the Netherlands and UK. These are national registries and health care registries, together with available electronic medical records, data from general practitioners (GP’s) covering what is documented during daily care of patients when they are visiting the GP’s. We also have dedicated cohort data which are rich in disease-specific information, these are coming from research on patients that have dementia or AD. Last but not least, there are also data from clinical trials which one might not see as RWE, but are complementing as evidence.

Read more:

What Is Real-World Data? A Review of Definitions Based on Literature and Stakeholder Interviews

Real-World Evidence — What Is It and What Can It Tell Us?