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Valuing Alzheimer's disease drugs: a health technology assessment perspective on outcomes

Published online by Cambridge University Press:  27 August 2020

Annette Bauer*
Affiliation:
Care Policy and Evaluation Centre, London School of Economics and Political Science, Houghton Street, LondonWC2A 2AE, UK
Raphael Wittenberg
Affiliation:
Care Policy and Evaluation Centre, London School of Economics and Political Science, Houghton Street, LondonWC2A 2AE, UK
Amanda Ly
Affiliation:
Centre for Medical Informatics, Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Nine Edinburgh BioQuarter, 9 Little France Road, EdinburghEH16 4UX, UK
Anders Gustavsson
Affiliation:
Quantify Research, Hantverkargatan 8, 112 21Stockholm, Sweden Department of Neurobiology, Care Sciences and Society, Karolinska Institute, Alfred Nobels allé 23, 141 83Stockholm, Sweden
Christin Bexelius
Affiliation:
ROCHE. F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, 4070Basel, Switzerland
Claire Tochel
Affiliation:
Centre for Medical Informatics, Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Nine Edinburgh BioQuarter, 9 Little France Road, EdinburghEH16 4UX, UK
Martin Knapp
Affiliation:
Care Policy and Evaluation Centre, London School of Economics and Political Science, Houghton Street, LondonWC2A 2AE, UK
Mia Nelson
Affiliation:
Centre for Medical Informatics, Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Nine Edinburgh BioQuarter, 9 Little France Road, EdinburghEH16 4UX, UK
Catherine Sudlow
Affiliation:
Centre for Medical Informatics, Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Nine Edinburgh BioQuarter, 9 Little France Road, EdinburghEH16 4UX, UK
*
Author for correspondence: Annette Bauer, E-mail: a.bauer@lse.ac.uk

Abstract

Objectives

Due to the nature of Alzheimer's disease (AD), health technology assessment (HTA) agencies might face considerable challenges in choosing appropriate outcomes and outcome measures for drugs that treat the condition. This study sought to understand which outcomes informed previous HTAs, to explore possible reasons for prioritizations, and derive potential implications for future assessments of AD drugs.

Method

We conducted a literature review of studies that analyzed decisions made in HTAs (across disease areas) in three European countries: England, Germany, and The Netherlands. We then conducted case studies of technology assessments conducted for AD drugs in these countries.

Results

Overall, outcomes measured using clinical scales dominated decisions or recommendations about whether to fund AD drugs, or price negotiations. HTA processes did not always allow the inclusion of outcomes relevant to people with AD, their carers, and families. Processes did not include early discussion and agreement on what would constitute appropriate outcome measures and cut-off points for effects.

Conclusions

We conclude that in order to ensure that future AD drugs are valued appropriately and timely, early agreement with various stakeholders about outcomes, outcome measures, and cut-offs is important.

Type
Article Commentary
Copyright
Copyright © The Author(s), 2020. Published by Cambridge University Press

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