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Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives

Objectives: Inclusion of patient preference (PP) data in decision making has been largely discussed in recent years. Healthcare decision makers—regulatory and health technology assessment (HTA)—are more and more conscious of the need for a patient-centered approach to decide on optimal allocation of...

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Autores principales: Chachoua, Lylia, Dabbous, Monique, François, Clément, Dussart, Claude, Aballéa, Samuel, Toumi, Mondher
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7649266/
https://www.ncbi.nlm.nih.gov/pubmed/33195294
http://dx.doi.org/10.3389/fmed.2020.543046
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author Chachoua, Lylia
Dabbous, Monique
François, Clément
Dussart, Claude
Aballéa, Samuel
Toumi, Mondher
author_facet Chachoua, Lylia
Dabbous, Monique
François, Clément
Dussart, Claude
Aballéa, Samuel
Toumi, Mondher
author_sort Chachoua, Lylia
collection PubMed
description Objectives: Inclusion of patient preference (PP) data in decision making has been largely discussed in recent years. Healthcare decision makers—regulatory and health technology assessment (HTA)—are more and more conscious of the need for a patient-centered approach to decide on optimal allocation of scarce money, time, and technological resources. This literature review aims to examine the use of and recommendations for the integration of PP in decision making. Methods: A literature search was conducted through PubMed/Medline in May 2019 to identify publications on PP studies used to inform benefit–risk assessments (BRAs) and HTAs and patient-centered projects and guidelines related to the inclusion of PPs in health policy decision making. After title and abstract screening and full-text review, selected publications were analyzed to retrieve data related to the collection, use, and/or submission of PPs informing BRA or HTA as well as attempts and initiatives in recommendations for PPs integration in decision-making processes. Results: Forty-nine articles were included: 24 attempts and pilot project discussions and 25 PP elicitation studies. Quantitative approaches, particularly discrete choice experiments, were the most used (24 quantitative elicitation studies and 1 qualitative study). The objective of assessing PPs was to prioritize outcome-specific information, to value important treatment characteristics, to provide patient-focused benefit–risk trade-offs, and to appraise the patients' willingness to pay for new technologies. Moreover, attempts and pilot projects to integrate PPs in BRAs and HTAs were identified at the European level and across countries, but no clear recommendations have been issued yet. No less than seven public and/or private initiatives have been undertaken by governmental agencies and independent organizations to set guidance targeting improvement of patients' involvement in decision making. Conclusion: Despite the initiatives undertaken, the pace of progress remains slow. The use of PPs remains poorly implemented, and evidence of proper use of these data in decision making is lacking. Guidelines and recommendations formalizing the purpose of collecting PPs, what methodology should be adopted and how, and who should be responsible for generating these data throughout the decision-making processes are needed to improve and empower integration of PPs in BRA and HTA.
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spelling pubmed-76492662020-11-13 Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives Chachoua, Lylia Dabbous, Monique François, Clément Dussart, Claude Aballéa, Samuel Toumi, Mondher Front Med (Lausanne) Medicine Objectives: Inclusion of patient preference (PP) data in decision making has been largely discussed in recent years. Healthcare decision makers—regulatory and health technology assessment (HTA)—are more and more conscious of the need for a patient-centered approach to decide on optimal allocation of scarce money, time, and technological resources. This literature review aims to examine the use of and recommendations for the integration of PP in decision making. Methods: A literature search was conducted through PubMed/Medline in May 2019 to identify publications on PP studies used to inform benefit–risk assessments (BRAs) and HTAs and patient-centered projects and guidelines related to the inclusion of PPs in health policy decision making. After title and abstract screening and full-text review, selected publications were analyzed to retrieve data related to the collection, use, and/or submission of PPs informing BRA or HTA as well as attempts and initiatives in recommendations for PPs integration in decision-making processes. Results: Forty-nine articles were included: 24 attempts and pilot project discussions and 25 PP elicitation studies. Quantitative approaches, particularly discrete choice experiments, were the most used (24 quantitative elicitation studies and 1 qualitative study). The objective of assessing PPs was to prioritize outcome-specific information, to value important treatment characteristics, to provide patient-focused benefit–risk trade-offs, and to appraise the patients' willingness to pay for new technologies. Moreover, attempts and pilot projects to integrate PPs in BRAs and HTAs were identified at the European level and across countries, but no clear recommendations have been issued yet. No less than seven public and/or private initiatives have been undertaken by governmental agencies and independent organizations to set guidance targeting improvement of patients' involvement in decision making. Conclusion: Despite the initiatives undertaken, the pace of progress remains slow. The use of PPs remains poorly implemented, and evidence of proper use of these data in decision making is lacking. Guidelines and recommendations formalizing the purpose of collecting PPs, what methodology should be adopted and how, and who should be responsible for generating these data throughout the decision-making processes are needed to improve and empower integration of PPs in BRA and HTA. Frontiers Media S.A. 2020-10-26 /pmc/articles/PMC7649266/ /pubmed/33195294 http://dx.doi.org/10.3389/fmed.2020.543046 Text en Copyright © 2020 Chachoua, Dabbous, François, Dussart, Aballéa and Toumi. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Medicine
Chachoua, Lylia
Dabbous, Monique
François, Clément
Dussart, Claude
Aballéa, Samuel
Toumi, Mondher
Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives
title Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives
title_full Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives
title_fullStr Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives
title_full_unstemmed Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives
title_short Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives
title_sort use of patient preference information in benefit–risk assessment, health technology assessment, and pricing and reimbursement decisions: a systematic literature review of attempts and initiatives
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7649266/
https://www.ncbi.nlm.nih.gov/pubmed/33195294
http://dx.doi.org/10.3389/fmed.2020.543046
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