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Alzheimers Dement (N Y). 2018 Dec 12;4:694-702. doi: 10.1016/j.trci.2018.10.013. eCollection 2018.

Participant outcomes and preferences in Alzheimer's disease clinical trials: The electronic Person-Specific Outcome Measure (ePSOM) development program.

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Centre for Clinical Brain Sciences, University of Edinburgh, Edinburgh, UK.
Edinburgh Centre for Research on the Experience of Dementia (ECRED), School of Health in Social Science, University of Edinburgh, Edinburgh, UK.
Usher Institute of Population Health Sciences and Informatics, Molecular, Genetic and Population Health Sciences, University of Edinburgh, Edinburgh, UK.
Alzheimer's Research UK, Cambridge, UK.



Current pharmacological interventions for Alzheimer's dementia delay symptom progression for about a year. Although the outcomes in earlier disease states may include changes in biomarkers, the clinical effectiveness of any intervention can ultimately only be assessed by a patient's self-reported well-being. A better understanding of earlier manifestations of Alzheimer's disease and the drive for relevant outcome measures, allied to technological advances in artificial intelligence, have mediated the electronic Person-Specific Outcome Measure (ePSOM) development program.


There are 4 sequential stages in the ePSOM development program-(1) literature review, (2) focus group study, (3) national survey, and (4) development of an app for capturing person-specific outcomes. Here, we report the overall approach to the program incorporating our literature review on patient-reported outcome measures and patient preferences in the Alzheimer's disease population.


Alzheimer's disease trials do not use any patient-reported outcome measures. Quality of life measures are often used as proxies for this, but they do not capture individual needs. Therefore, trials currently fail to reflect the participant's aspirations for effect but rather default to clinicostatistical measure of cognition and function. There is no implementation of patient preferences despite evidence that understanding preferences may influence adherence to treatment.


It is important to consider preferences for an intervention and use PROMs for the measure of effectiveness given that both risk and benefit are judged by the recipient of the treatment. The ePSOM development program will deliver the methodology for incorporating meaningful outcomes in clinical trials to expand upon current biological and clinical measurements of effectiveness.


Alzheimer's disease clinical trials; Clinical effectiveness; Dementia; Patient preferences; Patient-reported outcome measures

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