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将偏好纳入基因组医学健康经济模型的方法:批判性解释性综合与概念框架

Approaches to Incorporation of Preferences into Health Economic Models of Genomic Medicine: A Critical Interpretive Synthesis and Conceptual Framework.

作者信息

Smith Hadley Stevens, Regier Dean A, Goranitis Ilias, Bourke Mackenzie, IJzerman Maarten J, Degeling Koen, Montgomery Taylor, Phillips Kathryn A, Wordsworth Sarah, Buchanan James, Marshall Deborah A

机构信息

Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, 401 Park Drive Suite 401, Boston, MA, USA, 02215.

School of Population and Public Health, University of British Columbia, Vancouver, BC, Canada.

出版信息

Appl Health Econ Health Policy. 2025 May;23(3):337-358. doi: 10.1007/s40258-025-00945-0. Epub 2025 Jan 20.

Abstract

INTRODUCTION

Genomic medicine has features that make it preference sensitive and amenable to model-based health economic evaluation. Preferences of patients, caregivers, and clinicians related to the uptake and delivery of genomic medicine technologies and services that are not captured in health state utility weights can affect the intervention's cost-effectiveness and budget impact. However, there is currently no established or agreed-on approach for integrating preference information into economic evaluations. The objective of this study was to explore approaches for incorporating preferences into model-based economic evaluations of genomic medicine and to develop a conceptual framework to consider preferences in health economic models.

METHODS

We conducted a critical interpretive synthesis of published literature guided by the following question: how have preferences been incorporated into model-based economic evaluations of genomic medicine interventions? We integrated findings from the literature and expert opinion to develop a conceptual framework of ways in which preferences influence economic value in the context of genomic medicine.

RESULTS

Our synthesis included 14 articles. Revealed and stated preference data were used to estimate choice probabilities and to value outcomes. Our conceptual framework situates preference data in the context of health system, patient, clinician, and family characteristics. Preference data were sourced from clinicians, patients and families impacted by a condition or intervention, and the general public. Evaluations employed various types of models, including discrete event simulation, microsimulation, Markov, and decision tree models.

CONCLUSION

When evaluating the broad benefits and costs of implementing new interventions, sufficiently accounting for preferences in the form of model inputs and valuation of outcomes in economic evaluations is important to avoid biased implementation decisions. Incorporation of preference data may improve alignment between predicted and real-world uptake and more accurately estimate welfare impacts, and this study provides critical insights to support researchers who seek to incorporate preference information into model-based health economic evaluations.

摘要

引言

基因组医学具有一些特性,使其具有偏好敏感性,适合基于模型的卫生经济评估。患者、护理人员和临床医生对基因组医学技术与服务的采用和提供的偏好,在健康状态效用权重中未得到体现,却会影响干预措施的成本效益和预算影响。然而,目前尚无既定的或得到认可的方法将偏好信息纳入经济评估。本研究的目的是探索将偏好纳入基于模型的基因组医学经济评估的方法,并开发一个概念框架,以在卫生经济模型中考虑偏好。

方法

我们以以下问题为导向,对已发表的文献进行了批判性解释性综合分析:偏好是如何纳入基于模型的基因组医学干预经济评估的?我们整合了文献研究结果和专家意见,以构建一个概念框架,阐述在基因组医学背景下偏好影响经济价值的方式。

结果

我们的综合分析纳入了14篇文章。显示性偏好数据和陈述性偏好数据被用于估计选择概率和评估结果价值。我们的概念框架将偏好数据置于卫生系统、患者、临床医生和家庭特征的背景下。偏好数据来源于临床医生、受某种疾病或干预影响的患者及其家庭,以及普通公众。评估采用了各种类型的模型,包括离散事件模拟、微观模拟、马尔可夫模型和决策树模型。

结论

在评估实施新干预措施的广泛效益和成本时,在经济评估中以模型输入和结果估值的形式充分考虑偏好,对于避免有偏差的实施决策很重要。纳入偏好数据可能会改善预测的与实际的采用情况之间的一致性,并更准确地估计福利影响,本研究为寻求将偏好信息纳入基于模型的卫生经济评估的研究人员提供了重要见解。

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