Regulatory and Health Technology Assessment Considerations for Disease-Modifying Drugs in Alzheimer’s Disease

28 Nov 2018

Although there are a growing number of well-reported, late-stage clinical trial failures in Alzheimer’s disease, the introduction of a disease-modifying therapy within the next 5 years may be anticipated. These treatments are likely to target Alzheimer’s disease in the earlier disease stages, unlike drugs that are currently available that treat symptoms of moderate-to-severe dementia. Therefore, there is a need to establish a consensus on regulatory and health technology assessment requirements for Alzheimer’s disease, as a new drug will need to undergo regulatory and health technology assessments before it becomes available to patients. This article reports the discussions and activities of the regulatory and health technology assessment expert advisory group of the 2-year ROADMAP (real-world outcomes across the Alzheimer’s disease spectrum: a multimodal data access platform) project. The expert advisory group discussions identified a lack of consensus on validated outcomes in the earliest Alzheimer’s disease stages, the need for filling gaps between outcomes used across clinical trials and real-world settings, and the role that real-world evidence might have in characterising the impact of a possible disease-modifying therapy on caregivers, resource use and long-term outcomes. This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License ( Bouvy JC, Jonsson P, O’Rourke D, Chadha AS, Hedberg N, Makady A, et al. Regulatory and Health Technology Assessment Considerations for Disease-Modifying Drugs in Alzheimer’s Disease. CNS Drugs [Internet]. 2018 Nov 22 [cited 2018 Nov 28]; Available from:


Systematic literature review of methodologies and data sources of existing economic models across the full spectrum of Alzheimer’s disease and dementia from apparently healthy through disease progression to end of life care: a systematic review protocol

08 Jun 2018

Dementia is one of the greatest health challenges the world will face in the coming decades, as it is one of the principal causes of disability and dependency among older people. Economic modelling is used widely across many health conditions to inform decisions on health and social care policy and practice. The aim of this literature review is to systematically identify, review and critically evaluate existing health economics models in dementia. We included the full spectrum of dementia, including Alzheimer’s disease (AD), from preclinical stages through to severe dementia and end of life. This review forms part of the Real world Outcomes across the Alzheimer’s Disease spectrum for better care: multimodal data Access Platform (ROADMAP) project.


Measuring quality of life of people with predementia and dementia and their caregivers: a systematic review protocol

30 Mar 2018

Dementia is the fastest growing major cause of disability globally and may have a profound impact on the health-related quality of life (HRQoL) of both the patient with dementia and those who care for them. This review aims to systematically identify and synthesise the measurements of HRQoL for people with, and their caregivers across the full spectrum of, dementia from its preceding stage of predementia to end of life.


Resource utilisation and costs in predementia and dementia: a systematic review protocol

23 Jan 2018

Dementia is the fastest growing major cause of disability globally with a mounting social and financial impact for patients and their families but also to health and social care systems. This review aims to systematically synthesise evidence on the utilisation of resources and costs incurred by patients and their caregivers and by health and social care services across the full spectrum of dementia, from its preceding preclinical stage to end of life. The main drivers of resources used and costs will also be identified.



Big Data For Better Outcomes

12 Mar 2017

Large amounts of data from multiple sources have led to the opportunity of deriving health benefits through using sophisticated technologies. Regardless of the frequently cited revolution of data-driven health care, promises remain to be fulfilled. The IMI2 BD4BO programme recognises this in representative disease areas, providing a framework to guide research and invite stakeholders to discuss the future of health systems shaped by big data. The projects will impact he research environment through shared definitions and methods to avoid duplication of work, while transforming health care systems in terms of clinical operations, research and development, evidence-based personalised medicine and public health.