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Genomic Testing in Australia: A Budget Impact Analysis Using Diffusion Modeling From a Healthcare System Perspective 基因组测试在澳大利亚:从医疗保健系统的角度使用扩散模型的预算影响分析。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-10-15 DOI: 10.1016/j.jval.2025.09.3067
Dylan A. Mordaunt MB, ChB, MPH , Zornitza Stark DM , Adam G. Elshaug PhD , Chris Schilling PhD

Objectives

Genomic testing can shorten the diagnostic odyssey for people with rare diseases, yet clinical uptake has lagged funding policy in Australia. Therefore, we evaluated the 10-year budget impact of alternative implementation strategies for publicly funded genomic testing using national claims data and diffusion modeling.

Methods

Monthly Medicare Benefits Schedule claims (1993–2025) were analyzed for chromosomal microarray analysis (CMA), Fragile X (FMR1) testing, and genomic tests across 7 rare-disease groups (syndromic and non-syndromic intellectual disability, neuromuscular, inherited cardiac, renal ciliopathies/tubulopathies, congenital hearing loss, mitochondrial). Logistic, Gompertz, and Bass diffusion functions and Seasonal Autoregressive Integrated Moving-Average models were fitted to uptake and used to forecast 2025 to 2034 volumes. Scenarios included status quo, broadened second-line eligibility, and first-line exome sequencing/genome sequencing (ES/GS) replacing CMA/FMR1 (60:40 ES:GS). Costs used were the 1 July 2024 Medicare Benefits Schedule fees; the results are in Australian (AUD) dollars.

Results

Observed genomic testing volumes were below diffusion-implied trajectories. The 10-year cumulative spending was as follows: status quo AUD 1.1 million; broadened second-line AUD 7.5 million (incremental +6.4 million vs status quo); and first-line ES/GS AUD 6.2 million (incremental +5.1 million). In 2028, it was status quo AUD 0.23 million, second-line AUD 1.21 million, and first-line AUD 0.97 million. ES/GS achieved lower cumulative spend than the broadened second-line despite higher per-test fees, reflecting substitution from CMA/FMR1 and efficient diagnostic pathways.

Conclusions

Indication-by-indication funding has yielded slower-than-expected uptake and likely under-budgeting. A first-line genomic testing pathway, aligned with CMA criteria, could better match clinical need while constraining spend versus expanding second-line eligibility. Harmonized eligibility and streamlined implementation would improve access and planning.
背景:基因组检测可以缩短罕见病患者的诊断过程,但临床应用滞后于澳大利亚的资助政策。我们使用国家索赔数据和扩散模型评估了公共资助基因组检测的替代实施策略的10年预算影响。方法:对每月医疗保险福利计划(MBS)索赔(1993-2025)进行染色体微阵列分析(CMA)、脆性X染色体(FMR1)检测和基因组检测,涵盖7个罕见疾病组(综合征和非综合征性智力残疾、神经肌肉、遗传性心脏、肾纤毛病/小管病、先天性听力损失、线粒体)。Logistic、Gompertz和Bass扩散函数和SARIMA拟合了吸收量,并用于预测2025-2034年的产量。场景包括:现状;扩大二线资格;一线ES/GS取代CMA/FMR1 (60:40 ES:GS)。成本使用2024年7月1日MBS时间表费用;结果以澳元表示。结果:观察到的基因组检测量低于扩散隐含轨迹。十年累计支出为:现状110万澳元;扩大二线750万澳元(比现状增加640万澳元);一线ES/GS 620万澳元(增量+ 510万)。2028年,现状AUD 0.23m,二线AUD 121 m,一线AUD 0.97m。尽管每次检测费用较高,但ES/GS的累积支出低于扩大二线,这反映了CMA/FMR1的替代和有效的诊断途径。结论:各适应症的资金投入慢于预期,可能预算不足。与CMA标准一致的一线基因组检测途径可以更好地满足临床需求,同时限制支出,而不是扩大二线资格。统一的资格和精简的执行将改善获取和规划。
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引用次数: 0
Comparative Responsiveness of Preference-Based Health-Related Quality of Life, Social Care, and Well-Being Measures in the Context of Multiple Sclerosis 在多发性硬化症的背景下,基于偏好的健康相关生活质量、社会关怀和福利措施的比较响应性
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-10-14 DOI: 10.1016/j.jval.2025.09.3063
Elizabeth Goodwin PhD , Amy Heather MSc , Nia Morrish MSc , Jenny Freeman PhD , Kate Boddy MSc , Sarah Thomas PhD , Jeremy Chataway PhD , Rod Middleton PhD , Annie Hawton PhD

Objectives

To provide evidence on the responsiveness of social care and well-being preference-based measures (PBMs) compared with health-related quality of life PBMs in the context of multiple sclerosis (MS).

Methods

The ICEpop CAPability measure for Adults (ICECAP-A) and Adult Social Care Outcomes Toolkit (ASCOT) were completed online in September 2019, March 2020, September 2020, via the UK MS Register. Responses were linked to EQ-5D-3L and MS Impact Scale-8 Dimensions (MSIS-8D) values, and to MS Walking Scale-12, Hospital Anxiety and Depression Scale (HADS), and Fatigue Severity Scale scores. Responsiveness was assessed in relation to minimal important differences on MS Walking Scale-12, Hospital Anxiety and Depression Scale, and Fatigue Severity Scale between time points, using mean change scores, t tests, standardized effect sizes, standardized response means, and multivariable regression analyses.

Results

Data from 1742 people with MS were available for analysis. When using standardized values, MSIS-8D showed the greatest responsiveness and EQ-5D-3L the least. In contrast, when absolute utility values were used, EQ-5D-3L performed similarly to MSIS-8D and better than ICECAP-A and ASCOT. Standardized regression analyses indicated the MSIS-8Ds to be the most responsive, followed by the ASCOT, ICECAP-A, and EQ-5D-3L.

Conclusions

The ICECAP-A, ASCOT, and MSIS-8D were more responsive than the EQ-5D-3L in the context of MS when compared using standardized scores. The increased responsiveness of EQ-5D-3L when absolute values were used seems an artefact of the wide-ranging scale of this measure. This illustrates how the maximum potential range of values for a given PBM tariff could influence whether an intervention is found to be cost-effective.
目的:在多发性硬化症(MS)的背景下,与健康相关的生活质量PBMs相比,提供社会关怀和基于健康偏好的措施(PBMs)的响应性的证据。方法:通过英国MS Register于2019年9月、2020年3月和2020年9月在线完成成人icpop能力量表(ICECAP-A)和成人社会护理结果工具包(ASCOT)。反应与EQ-5D-3L和MS影响量表-8维度(MSIS-8D)值、MS步行量表-12 (msw -12)、医院焦虑和抑郁量表(HADS)和疲劳严重程度量表(FSS)得分相关联。使用平均变化评分、t检验、标准化效应量、标准化反应均值和多变量回归分析,评估与时间点之间MSWS-12、HADS和FSS的最小重要差异相关的响应性。结果:来自1742名多发性硬化症患者的数据可供分析。当使用标准值时,MSIS-8D表现出最大的响应性,EQ-5D-3L表现出最小的响应性。相比之下,当使用绝对效用值时,EQ-5D-3L的表现与MSIS-8D相似,优于ICECAP-A和ASCOT。标准化回归分析表明,msis - 8d反应最积极,其次是ASCOT、ICECAP-A和EQ-5D-3L。结论:在使用标准化评分进行比较时,ICECAP-A、ASCOT和MSIS-8D比EQ-5D-3L对MS的反应更敏感。当使用绝对值时,EQ-5D-3L的响应性增加似乎是该测量范围广泛的人工产物。这说明了给定PBM关税的最大潜在值范围如何影响干预措施是否具有成本效益。
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引用次数: 0
Evaluating Long-Term Health Disparity Impacts of Clinical Algorithms Using a Patient-Level Simulation Framework 使用患者级模拟框架评估临床算法的长期健康差异影响。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-10-15 DOI: 10.1016/j.jval.2025.09.3066
Sara Khor PhD , Anirban Basu PhD , Veena Shankaran MD , Kyueun Lee PhD , Eric C. Haupt ScM , Erin E. Hahn PhD , Josh J. Carlson PhD , Aasthaa Bansal PhD

Objectives

This study applies a simulation framework to evaluate the long-term effects of omitting race from a colon cancer decision algorithm for adjuvant chemotherapy, assessing impacts on health outcomes, costs, and disparities while accounting for measurement errors across racial groups.

Methods

We developed a patient-level state-transition model using electronic health records from a large Southern California health system to project outcomes for 4839 adults with stage II and III colon cancer after surgery. We compared 30-year quality-adjusted life-years (QALYs), healthcare costs, and QALY distribution among racial groups under 3 chemotherapy treatment scenarios: (1) current practice, (2) treatment guided by an algorithm that includes race, and (3) the same algorithm with race omitted. An additional health state addressed racial bias in cancer recurrence ascertainment, and probabilistic sensitivity analysis (PSA) assessed uncertainty.

Results

The clinical algorithm, compared with current practice, could improve average health by 0.048 QALYs and reduce racial health disparity by 0.20 QALYs at an incremental cost of $3221, with the disparity gap decreasing in 96% of PSA iterations. Omitting race showed minimal effects on overall health or costs but resulted in 13% fewer Black patients receiving treatment, decreasing their QALYs by 0.07 and widening the disparity gap by 0.13 QALY. Health disparity increased in 94% of PSA iterations.

Conclusions

A cancer decision algorithm can improve population health and reduce health disparities, but omitting race may harm disadvantaged groups and limit reductions in disparities. Patient-level simulations can be routinely used to evaluate the potential health disparity impacts of algorithms before implementation.
目的:本研究应用模拟框架来评估从辅助化疗的结肠癌决策算法中忽略种族的长期影响,评估对健康结果、成本和差异的影响,同时考虑种族群体之间的测量误差。方法:我们开发了一个患者层面的状态转换模型,使用来自南加州大型卫生系统的电子健康记录来预测4,839名II期和III期结肠癌成人手术后的结果。我们比较了三种化疗方案下不同种族群体的30年质量调整生命年(QALYs)、医疗费用和QALY分布:1)当前实践,2)由包含种族的算法指导的治疗,以及3)相同的算法忽略种族。另一项健康状况研究解决了癌症复发确定中的种族偏见,概率敏感性分析(PSA)评估了不确定性。结果:与目前的实践相比,临床算法可以提高平均健康水平0.048个QALYs,减少种族健康差距0.20个QALYs,增量成本为3,221美元,差异差距在96%的PSA迭代中减少。忽略种族对整体健康或成本的影响很小,但导致接受治疗的黑人患者减少了13%,使他们的质量aly降低了0.07,并使差距扩大了0.13。在94%的PSA迭代中,健康差距增加了。结论:癌症决策算法可以改善人群健康,缩小健康差距,但忽略种族可能会损害弱势群体,限制差距的缩小。患者水平的模拟可常规用于评估算法实施前的潜在健康差异影响。
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引用次数: 0
Psychometric Performance of Skin, Self-Confidence, and Social Health-Related EQ-5D-5L Bolt-Ons in Patients With Atopic Dermatitis and Psoriasis in Germany 德国特应性皮炎和牛皮癣患者皮肤、自信和社会健康相关EQ-5D-5L的心理测量表现
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-10-31 DOI: 10.1016/j.jval.2025.10.008
Ines Buchholz PhD , Carsten Spitzer MD , Alexander Thiem MD , Laura Lübke MSc , Clara Wülfing MSc , Steffen Emmert MD , Fanni Rencz DSc

Objectives

The European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) has shown potential limitations in capturing key aspects of health-related quality of life in dermatological diseases. This study investigated the psychometric properties of the EQ-5D-5L and 5 bolt-ons—skin irritation (itching, IT), self-confidence (SE), social relationships (SR), social participation (SP), and feeling connected (FC)—in 2 prevalent dermatological conditions.

Methods

A cross-sectional online survey was conducted with n = 180 atopic dermatitis (mean age: 37 years) and n = 285 psoriasis patients (46 years) in Germany. Outcome measures included the EQ-5D-5L, 5 bolt-ons, and Dermatology Life Quality Index, Skin Shame Scale, Patient-Oriented Eczema Measure, and Psoriasis Signs and Symptoms Diary. Analyses comprised ceiling, construct and known-groups validity, and EQ VAS explanatory power.

Results

Adding IT led to the largest reduction in ceiling of the EQ-5D-5L (from 15.5% to 5.6% in atopic dermatitis, and 18.7% to 10.7% in psoriasis), followed by SE (atopic dermatitis) and FC (psoriasis). Overall, 72% of atopic dermatitis and 54% of psoriasis patients reporting no pain/discomfort reported problems with IT. IT correlated strongly with conceptually overlapping items of the Dermatology Life Quality Index, Patient-Oriented Eczema Measure, and Psoriasis Signs and Symptoms Diary. Adding IT improved known-groups validity and explanatory power, whereas additional bolt-ons offered limited additional impact. No meaningful differences emerged between SR and SP bolt-ons.

Conclusions

In this patient sample from Germany, IT performed best, with minimal added value from other bolt-ons. IT is a promising candidate bolt-on for recommendation in chronic inflammatory skin diseases. Future research may explore the usefulness of other potentially relevant bolt-ons (eg, sleep) in these patient populations.
目的:EQ-5D-5L在捕捉皮肤病患者与健康相关的生活质量的关键方面显示出潜在的局限性。本研究考察了EQ-5D-5L和皮肤刺激(瘙痒、IT)、自信(SE)、社会关系(SR)、社会参与(SP)和联系感(FC)在两种常见皮肤病中的心理测量特性。方法:对德国180例特应性皮炎患者(平均年龄37岁)和285例银屑病患者(46岁)进行横断面在线调查。结果测量包括EQ-5D-5L、5项附加指标、皮肤病生活质量指数(DLQI)、皮肤羞耻感量表、患者导向湿疹测量(POEM)、牛皮癣体征和症状日记(PSSD)。分析包括上限、结构效度和已知组效度,以及EQ VAS解释力。结果:加入IT后EQ-5D-5L的上限降低幅度最大(特应性皮炎从15.5%降至5.6%,牛皮癣从18.7%降至10.7%),其次是SE(特应性皮炎)和FC(牛皮癣)。总体而言,72%的特应性皮炎患者和54%的牛皮癣患者报告没有疼痛/不适,报告了IT问题。IT与DLQI、POEM和PSSD的概念重叠项有很强的相关性。添加IT提高了已知组的有效性和解释力,而附加的附加影响有限。SR和SP螺栓连接之间没有显著差异。结论:在这个来自德国的患者样本中,IT表现最好,其他螺栓的附加价值最小。它是一种很有前途的候选补强剂,推荐用于慢性炎症性皮肤病。未来的研究可能会探索在这些患者群体中其他潜在相关的补充(如睡眠)的有用性。
{"title":"Psychometric Performance of Skin, Self-Confidence, and Social Health-Related EQ-5D-5L Bolt-Ons in Patients With Atopic Dermatitis and Psoriasis in Germany","authors":"Ines Buchholz PhD ,&nbsp;Carsten Spitzer MD ,&nbsp;Alexander Thiem MD ,&nbsp;Laura Lübke MSc ,&nbsp;Clara Wülfing MSc ,&nbsp;Steffen Emmert MD ,&nbsp;Fanni Rencz DSc","doi":"10.1016/j.jval.2025.10.008","DOIUrl":"10.1016/j.jval.2025.10.008","url":null,"abstract":"<div><h3>Objectives</h3><div>The European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) has shown potential limitations in capturing key aspects of health-related quality of life in dermatological diseases. This study investigated the psychometric properties of the EQ-5D-5L and 5 bolt-ons—skin irritation (itching, IT), self-confidence (SE), social relationships (SR), social participation (SP), and feeling connected (FC)—in 2 prevalent dermatological conditions.</div></div><div><h3>Methods</h3><div>A cross-sectional online survey was conducted with <em>n</em> = 180 atopic dermatitis (mean age: 37 years) and <em>n</em> = 285 psoriasis patients (46 years) in Germany. Outcome measures included the EQ-5D-5L, 5 bolt-ons, and Dermatology Life Quality Index, Skin Shame Scale, Patient-Oriented Eczema Measure, and Psoriasis Signs and Symptoms Diary. Analyses comprised ceiling, construct and known-groups validity, and EQ VAS explanatory power.</div></div><div><h3>Results</h3><div>Adding IT led to the largest reduction in ceiling of the EQ-5D-5L (from 15.5% to 5.6% in atopic dermatitis, and 18.7% to 10.7% in psoriasis), followed by SE (atopic dermatitis) and FC (psoriasis). Overall, 72% of atopic dermatitis and 54% of psoriasis patients reporting no pain/discomfort reported problems with IT. IT correlated strongly with conceptually overlapping items of the Dermatology Life Quality Index, Patient-Oriented Eczema Measure, and Psoriasis Signs and Symptoms Diary. Adding IT improved known-groups validity and explanatory power, whereas additional bolt-ons offered limited additional impact. No meaningful differences emerged between SR and SP bolt-ons.</div></div><div><h3>Conclusions</h3><div>In this patient sample from Germany, IT performed best, with minimal added value from other bolt-ons. IT is a promising candidate bolt-on for recommendation in chronic inflammatory skin diseases. Future research may explore the usefulness of other potentially relevant bolt-ons (eg, sleep) in these patient populations.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 3","pages":"Pages 429-440"},"PeriodicalIF":6.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145432189","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Empirical Evidence and Conceptual Framework to Address the Indian Health Service Underfunding Challenge 解决印度卫生服务资金不足挑战的经验证据和概念框架。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-10-30 DOI: 10.1016/j.jval.2025.10.003
Junying Zhao PhD, PhD , Rashmi Jaggad MPH , James R. Kennedye MD , Pallab Ghosh PhD , Kylie Stewart MHA , Janis E. Campbell PhD

Objectives

The Indian Health Service (IHS) provides services to American Indian and Alaska Native individuals. However, IHS faces a longstanding underfunding problem. This article examines the extent, reasons, and alternative solutions for the IHS underfunding problem.

Methods

We used the most recent 15-year quantitative and qualitative data from a fiscal administration perspective and established a conceptual framework from an economic perspective to better understand the nature of financing IHS. We applied the framework, especially efficiency and equity criteria, to examine the justifiability of proposed benchmarks for IHS budgeting and compare proposed solutions.

Results

First, we identified budget planning and approving steps where significant IHS budget cuts occurred and why. Second, the United States has a federal Indian trust responsibility for American Indian and Alaska Native healthcare and thus mandatory spending in principle. However, in practice, it has been a discretionary spending decision for the United States to maximize social welfare subject to limited fiscal resources. Third, between the 2 economic criteria for resource allocation, vertical equity, instead of allocative efficiency, justifies more IHS appropriations. Fourth, entitlement mandatory spending on IHS was proposed by legislators during 2001 to 2004 but failed to receive Congressional votes. Nonentitlement mandatory spending and sequestration exemption were proposed by the President but were unapproved for fiscal year 2024.

Conclusions

To our knowledge, this article is the first to evaluate discrepancies in IHS budget proposals, before and during enactment, to elucidate the stages at which underfunding may occur and the implications for future research and policy making by the IHS, Tribal Nations, and US Congress.
目标:印第安人健康服务(IHS)为美洲印第安人和阿拉斯加原住民(AIAN)个人提供服务。然而,IHS长期面临资金不足的问题。本文探讨了IHS资金不足问题的程度、原因和替代解决方案。方法从财政管理的角度,利用最近15年的定量和定性数据,从经济学的角度建立了一个概念框架,以更好地理解IHS融资的本质。我们应用该框架,特别是效率和公平标准,来检查IHS预算的拟议基准的合理性,并比较拟议的解决方案。结果:首先,我们确定了预算计划和批准步骤,其中发生了重大的IHS预算削减,以及原因。其次,美国对印度的医疗保健负有联邦印度信托责任,因此原则上是强制性支出。然而,在实践中,在有限的财政资源条件下,实现社会福利最大化一直是美国的自由支配性支出决策。第三,在资源配置的两个经济标准之间,纵向公平而不是配置效率证明了更多的IHS拨款是合理的。第四,2001年至2004年期间,立法者曾提议在IHS上强制支出福利,但未能获得国会投票。非福利性强制性支出和自动封存豁免是由总统提出的,但在2024财年未获批准。结论:本文首次评估了IHS预算提案在立法前和立法期间的差异,以阐明可能出现资金不足的阶段,以及对IHS、部落国家和美国国会未来研究和政策制定的影响。
{"title":"Empirical Evidence and Conceptual Framework to Address the Indian Health Service Underfunding Challenge","authors":"Junying Zhao PhD, PhD ,&nbsp;Rashmi Jaggad MPH ,&nbsp;James R. Kennedye MD ,&nbsp;Pallab Ghosh PhD ,&nbsp;Kylie Stewart MHA ,&nbsp;Janis E. Campbell PhD","doi":"10.1016/j.jval.2025.10.003","DOIUrl":"10.1016/j.jval.2025.10.003","url":null,"abstract":"<div><h3>Objectives</h3><div>The Indian Health Service (IHS) provides services to American Indian and Alaska Native individuals. However, IHS faces a longstanding underfunding problem. This article examines the extent, reasons, and alternative solutions for the IHS underfunding problem.</div></div><div><h3>Methods</h3><div>We used the most recent 15-year quantitative and qualitative data from a fiscal administration perspective and established a conceptual framework from an economic perspective to better understand the nature of financing IHS. We applied the framework, especially efficiency and equity criteria, to examine the justifiability of proposed benchmarks for IHS budgeting and compare proposed solutions.</div></div><div><h3>Results</h3><div>First, we identified budget planning and approving steps where significant IHS budget cuts occurred and why. Second, the United States has a federal Indian trust responsibility for American Indian and Alaska Native healthcare and thus mandatory spending in principle. However, in practice, it has been a discretionary spending decision for the United States to maximize social welfare subject to limited fiscal resources. Third, between the 2 economic criteria for resource allocation, vertical equity, instead of allocative efficiency, justifies more IHS appropriations. Fourth, entitlement mandatory spending on IHS was proposed by legislators during 2001 to 2004 but failed to receive Congressional votes. Nonentitlement mandatory spending and sequestration exemption were proposed by the President but were unapproved for fiscal year 2024.</div></div><div><h3>Conclusions</h3><div>To our knowledge, this article is the first to evaluate discrepancies in IHS budget proposals, before and during enactment, to elucidate the stages at which underfunding may occur and the implications for future research and policy making by the IHS, Tribal Nations, and US Congress.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 3","pages":"Pages 403-412"},"PeriodicalIF":6.0,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145422774","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Priority for Self or Others? Incorporating Equity Considerations in a Preference-Based Health Value Assessment 优先考虑自己还是别人?在基于偏好的健康价值评估中纳入公平考虑。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-11-03 DOI: 10.1016/j.jval.2025.10.011
Mesfin G. Genie PhD , Surachat Ngorsuraches PhD

Objectives

To investigate patient preferences for health insurance plans covering newer type 2 diabetes treatments when equity is considered.

Methods

A discrete choice experiment with 701 patients with type 2 diabetes tested 2 experimental conditions: (1) personal health outcomes only (experiment 1) and (2) equal health outcomes for self and other patients with poorer health (experiment 2). Preferences were estimated using latent-class logit models in willingness-to-pay (WTP) space.

Results

Two distinct classes emerged under each experimental condition. In experiment 1, a “Risk-Averse” class (68.6%) displayed high WTP for a 1-percentage-point improvement in efficacy ($26.06, P < .01) and required large compensation for a 1-percentage-point increase in risk of side effects (−$28.39, P < .01), with low price sensitivity and a low likelihood of opting out. In contrast, a “Cost-Sensitive” class (31.4%) valued efficacy at $6.89 (P < .01), showed weaker risk aversion (−$5.15, P < .01), greater price sensitivity (−0.39, P < .01), and a higher tendency to opt-out. In experiment 2, an “Equity-Concerned” class (71.6%) showed a high WTP for efficacy ($20.84, P < .01), lower risk aversion (−$14.33, P < .01), and relatively lower price sensitivity (−0.10, P < .01). The “Cost-Sensitive” class (28.4%) valued efficacy at $7.28 (P < .01) and was insensitive to risk ($0.03, P = .98). Older respondents tended to align with cost-sensitive classes, whereas those with childcare responsibilities were more likely to belong to risk averse/equity-concerned classes.

Conclusions

Patients exhibited strong preferences for treatment efficacy, risk aversion, and varying levels of cost sensitivity. However, risk tolerance increased when equity was considered.
目的:探讨在考虑公平性的情况下,患者对新发2型糖尿病(T2D)治疗的健康保险计划的偏好。方法:采用离散选择实验(DCE)对701例T2D患者进行两种实验条件:(1)只有个人健康结果(实验1)和(2)自我健康结果与其他较差患者健康结果相同(实验2)。在支付意愿(WTP)空间中使用潜在类logit模型估计偏好。结果:在不同的实验条件下,出现了两个不同的类别。在实验1中,“风险厌恶”类患者(68.6%)对1个百分点(1-pp)的疗效改善表现出较高的WTP(26.06美元)。结论:患者对治疗效果、风险厌恶和不同程度的成本敏感性表现出强烈的偏好。然而,当考虑到股权时,风险承受能力增加了。
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引用次数: 0
Health Utility Decrements Associated With Social Isolation and Loneliness: Evidence From Australian Longitudinal Data 与社会隔离和孤独相关的健康效用下降:来自澳大利亚纵向数据的证据。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-03-01 Epub Date: 2025-10-30 DOI: 10.1016/j.jval.2025.10.006
Muhammad Fikru Rizal PhD , Cathy Mihalopoulous PhD , Sharon Clifford MPH , Arul Earnest PhD , Matthew P. Hamilton MSc , Long K.D. Le PhD , Michelle H. Lim PhD , Lidia Engel PhD , Connecting for Better Health (C4BH) Working Group

Objectives

This study estimates health utility decrements associated with social isolation and loneliness (SIL) using nationally representative longitudinal data from Australia.

Methods

Four waves of the Household, Income and Labour Dynamics in Australia survey were analyzed. Health utility was measured using the Short Form 6-Dimension with the Australian weighting algorithm. Social isolation was defined as living alone with infrequent social contact, and loneliness as a score ≥5 on a 1 to 7 scale. Respondents were classified as neither, socially isolated only, lonely only, or both. Individual fixed-effects panel regressions were applied to estimate within-person associations, with adjustment for time-varying health and sociodemographic factors.

Results

A total of 53 108 observations from 21 965 individuals (mean age 44.9; 53% male) were included, with 20% experiencing SIL (3% socially isolated only, 15% lonely only, 2% both). Compared with no SIL, adjusted models showed utility decrements of 0.020 for social isolation, 0.061 for loneliness, and 0.102 for both (all P < .001). The combined decrements significantly exceeded the sum of individual estimates (P < .05), suggesting interaction effects. Loneliness had the greatest impact on young adults (15-24 years), whereas social isolation affected middle-aged females (25-44 years) the most. Results were robust across sensitivity analyses, except that applying the UK weighting algorithm yielded smaller estimates and no significant interaction effects.

Conclusions

SIL is independently associated with reduced health utility, with some variation by age and sex. Potential interaction effects of SIL on health utility should be considered in economic evaluation, while recognizing their sensitivity to the choice of weighting algorithm.
目的:本研究使用来自澳大利亚的具有全国代表性的纵向数据来估计与社会隔离和孤独(SIL)相关的健康效用下降。方法:对澳大利亚家庭、收入和劳动力动态(HILDA)调查的四次浪潮进行分析。使用SF-6D和澳大利亚加权算法测量健康效用。社会隔离被定义为独居,缺乏频繁的社会接触,孤独感在1-7的量表中得分≥5。受访者被归类为两者都不是,只是社会孤立,只是孤独,或两者兼而有之。应用个体固定效应面板回归来估计人内关联,并对随时间变化的健康和社会人口因素进行调整。结果:来自21,965个人(平均年龄44.9岁,53%为男性)的53,108次观察被纳入,其中20%经历SIL(3%仅社会孤立,15%仅孤独,2%两者都有)。与没有SIL相比,调整后的模型显示,社会隔离的效用减少0.020,孤独感的效用减少0.061,两者的效用减少0.102(均p < 0.001)。综合降幅显著超过个体估计值之和(p < 0.05),提示交互作用。孤独对年轻人(15-24岁)的影响最大,而社会孤立对中年女性(25-44岁)的影响最大。敏感性分析的结果是稳健的,除了应用UK加权算法产生较小的估计值和没有显著的相互作用效应。结论:SIL与健康效用降低独立相关,随年龄和性别而有所差异。在经济评价中应考虑SIL对卫生效用的潜在交互效应,同时认识到它们对权重算法选择的敏感性。
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引用次数: 0
Evaluating the validity of the EQ-HWB-9 in a large UK general population sample. 评估EQ-HWB-9在英国大型普通人群样本中的有效性。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-02-28 DOI: 10.1016/j.jval.2026.02.006
Emily McDool, Clara Mukuria, Nyantara Wickramasekera, Aki Tsuchiya

Objectives: The EQ Health and Wellbeing (EQ-HWB-9) is a preference-weighted instrument with an interim value set for assessing health and wellbeing in patients, social care users and carers. This study evaluated the experimental (2022) version's psychometric properties and compared it with established measures in a UK general population sample.

Methods: Data were drawn from a large cross-sectional survey of the UK general population (n = 11,383). Ceiling/floor effects were assessed at item (>50% extreme responses) and instrument (>15% at min/max score) levels. Convergent validity was assessed using Spearman correlations between EQ-HWB-9 items and conceptually similar items from the Short Warwick-Edinburgh Mental Wellbeing Scale (SWEMWBS), ICEpop CAPability measure for Adults (ICECAP-A), Health Utilities Index Mark 3 (HUI3), Office for National Statistics Four Personal Wellbeing Questions (ONS-4) and SIPHER-7. Pearson correlations were used for utility scores. Known-group validity was assessed using effect sizes to examine differences by mental wellbeing, disability, life satisfaction, caregiving status, self-reported health, and age. Agreement with comparator instruments was assessed using Bland-Altman plots and Lin's concordance correlation coefficient (CCC).

Results: Potential ceiling effects were noted for EQ-HWB-9 mobility and activity items, but not at the instrument level. Strong correlations (rs≥0.5, p<.001) with measures hypothesised a priori to assess related constructs (SWEMWBS, ICECAP-A, HUI3, ONS-4 and SIPHER-7) supported convergent validity. The EQ-HWB-9 effectively distinguished between relevant population subgroups (effect sizes≥0.8). Agreement with HUI3 utilities was higher (CCC >0.75) than other comparators.

Conclusions: The EQ-HWB-9 shows strong psychometric performance and is supported for use in UK health and wellbeing assessments.

目的:情商健康与福祉(EQ- hwb -9)是一种偏好加权工具,具有中间值集,用于评估患者,社会护理使用者和护理人员的健康和福祉。这项研究评估了实验版本(2022)的心理测量特性,并将其与英国普通人群样本的既定测量结果进行了比较。方法:数据来自英国普通人群的大型横断面调查(n = 11,383)。天花板/地板效应在项目(>50%极端反应)和工具(>15%最低/最高评分)水平上进行评估。采用EQ-HWB-9项目与来自沃里克-爱丁堡心理健康量表(SWEMWBS)、成人ICEpop能力测量(ICECAP-A)、健康效用指数标记3 (HUI3)、国家统计局四个个人健康问题(ONS-4)和SIPHER-7的概念相似项目之间的Spearman相关性来评估收敛效度。效用评分采用Pearson相关性。已知组效度采用效应量来评估心理健康、残疾、生活满意度、照顾状况、自我报告的健康状况和年龄的差异。使用Bland-Altman图和Lin’s一致性相关系数(CCC)评估与比较仪器的一致性。结果:EQ-HWB-9的移动性和活动项目存在潜在的上限效应,但在工具水平上没有。强相关性(rs≥0.5,p0.75)。结论:EQ-HWB-9显示出强大的心理测量性能,支持在英国健康和福祉评估中使用。
{"title":"Evaluating the validity of the EQ-HWB-9 in a large UK general population sample.","authors":"Emily McDool, Clara Mukuria, Nyantara Wickramasekera, Aki Tsuchiya","doi":"10.1016/j.jval.2026.02.006","DOIUrl":"https://doi.org/10.1016/j.jval.2026.02.006","url":null,"abstract":"<p><strong>Objectives: </strong>The EQ Health and Wellbeing (EQ-HWB-9) is a preference-weighted instrument with an interim value set for assessing health and wellbeing in patients, social care users and carers. This study evaluated the experimental (2022) version's psychometric properties and compared it with established measures in a UK general population sample.</p><p><strong>Methods: </strong>Data were drawn from a large cross-sectional survey of the UK general population (n = 11,383). Ceiling/floor effects were assessed at item (>50% extreme responses) and instrument (>15% at min/max score) levels. Convergent validity was assessed using Spearman correlations between EQ-HWB-9 items and conceptually similar items from the Short Warwick-Edinburgh Mental Wellbeing Scale (SWEMWBS), ICEpop CAPability measure for Adults (ICECAP-A), Health Utilities Index Mark 3 (HUI3), Office for National Statistics Four Personal Wellbeing Questions (ONS-4) and SIPHER-7. Pearson correlations were used for utility scores. Known-group validity was assessed using effect sizes to examine differences by mental wellbeing, disability, life satisfaction, caregiving status, self-reported health, and age. Agreement with comparator instruments was assessed using Bland-Altman plots and Lin's concordance correlation coefficient (CCC).</p><p><strong>Results: </strong>Potential ceiling effects were noted for EQ-HWB-9 mobility and activity items, but not at the instrument level. Strong correlations (rs≥0.5, p<.001) with measures hypothesised a priori to assess related constructs (SWEMWBS, ICECAP-A, HUI3, ONS-4 and SIPHER-7) supported convergent validity. The EQ-HWB-9 effectively distinguished between relevant population subgroups (effect sizes≥0.8). Agreement with HUI3 utilities was higher (CCC >0.75) than other comparators.</p><p><strong>Conclusions: </strong>The EQ-HWB-9 shows strong psychometric performance and is supported for use in UK health and wellbeing assessments.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147345265","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Early TAVR cost-effectiveness compared to clinical surveillance in patients with asymptomatic severe aortic stenosis in the US. 美国无症状严重主动脉瓣狭窄患者早期TAVR成本-效果与临床监测的比较
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-02-25 DOI: 10.1016/j.jval.2026.02.005
Phillippe Généreux, Matthew R Reynolds, Marcella A Kelley, Christin Thompson, Suzanne J Baron

Objectives: The EARLY TAVR trial demonstrated improved clinical outcomes for patients with asymptomatic severe aortic stenosis (aSAS) treated with transcatheter aortic valve replacement (eTAVR) compared with clinical surveillance (CS). The cost effectiveness of an eTAVR strategy for patients with aSAS in the United States (US) is unknown.

Methods: A Markov model with 30-day cycles was developed from the US healthcare payor perspective to estimate the cost-effectiveness of eTAVR vs. CS over a lifetime horizon. Inputs for population characteristics and health outcomes were derived from the EARLY TAVR trial. Costs were derived from US Medicare reimbursement rates. Probabilistic and deterministic sensitivity analyses were performed to evaluate the effect of parameter uncertainty on model output.

Results: When compared to CS, eTAVR was associated with 0.21 additional life years (LY) and 0.24 additional quality-adjusted life years (QALYs) over a lifetime due to more time spent in the alive and well health state with eTAVR. Lifetime costs were estimated to be $8,812 lower, due primarily to reductions in costs associated with the AVR procedure, stroke, and heart failure hospitalizations. Accordingly, eTAVR was projected to be economically dominant over CS. In probabilistic sensitivity analysis, a large majority of iterations (95.9%) produced cost-effective results ($100,000 threshold) for eTAVR versus CS, with most simulations (90.3%) showing dominance, confirming the robustness of the base case results. These findings were consistent over a variety of scenario analyses.

Conclusions: An eTAVR strategy for the treatment of aSAS may be a cost-saving approach for US healthcare payors, when compared to CS.

目的:早期TAVR试验表明,与临床监测(CS)相比,经导管主动脉瓣置换术(eTAVR)治疗无症状严重主动脉瓣狭窄(aSAS)患者的临床结果有所改善。在美国,对asa患者使用eTAVR策略的成本效益尚不清楚。方法:从美国医疗保健支付者的角度开发了一个30天周期的马尔可夫模型,以估计eTAVR与CS在整个生命周期内的成本效益。人群特征和健康结果的输入来自EARLY TAVR试验。费用来源于美国医疗保险报销率。采用概率和确定性敏感性分析来评估参数不确定性对模型输出的影响。结果:与CS相比,eTAVR与一生中0.21额外的生命年(LY)和0.24额外的质量调整生命年(QALYs)相关,因为使用eTAVR在存活和健康状态下度过的时间更长。终生成本估计降低了8,812美元,主要是由于AVR手术、中风和心力衰竭住院治疗相关成本的降低。因此,预计eTAVR在经济上优于CS。在概率敏感性分析中,绝大多数迭代(95.9%)对eTAVR和CS产生了具有成本效益的结果(100,000美元阈值),大多数模拟(90.3%)显示出优势,证实了基本情况结果的鲁棒性。这些发现在各种情景分析中是一致的。结论:与CS相比,eTAVR治疗asa的策略可能是美国医疗保健支付者节省成本的方法。
{"title":"Early TAVR cost-effectiveness compared to clinical surveillance in patients with asymptomatic severe aortic stenosis in the US.","authors":"Phillippe Généreux, Matthew R Reynolds, Marcella A Kelley, Christin Thompson, Suzanne J Baron","doi":"10.1016/j.jval.2026.02.005","DOIUrl":"https://doi.org/10.1016/j.jval.2026.02.005","url":null,"abstract":"<p><strong>Objectives: </strong>The EARLY TAVR trial demonstrated improved clinical outcomes for patients with asymptomatic severe aortic stenosis (aSAS) treated with transcatheter aortic valve replacement (eTAVR) compared with clinical surveillance (CS). The cost effectiveness of an eTAVR strategy for patients with aSAS in the United States (US) is unknown.</p><p><strong>Methods: </strong>A Markov model with 30-day cycles was developed from the US healthcare payor perspective to estimate the cost-effectiveness of eTAVR vs. CS over a lifetime horizon. Inputs for population characteristics and health outcomes were derived from the EARLY TAVR trial. Costs were derived from US Medicare reimbursement rates. Probabilistic and deterministic sensitivity analyses were performed to evaluate the effect of parameter uncertainty on model output.</p><p><strong>Results: </strong>When compared to CS, eTAVR was associated with 0.21 additional life years (LY) and 0.24 additional quality-adjusted life years (QALYs) over a lifetime due to more time spent in the alive and well health state with eTAVR. Lifetime costs were estimated to be $8,812 lower, due primarily to reductions in costs associated with the AVR procedure, stroke, and heart failure hospitalizations. Accordingly, eTAVR was projected to be economically dominant over CS. In probabilistic sensitivity analysis, a large majority of iterations (95.9%) produced cost-effective results ($100,000 threshold) for eTAVR versus CS, with most simulations (90.3%) showing dominance, confirming the robustness of the base case results. These findings were consistent over a variety of scenario analyses.</p><p><strong>Conclusions: </strong>An eTAVR strategy for the treatment of aSAS may be a cost-saving approach for US healthcare payors, when compared to CS.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147318131","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Why Object to Inequalities in Health and Well-Being? A Mixed-Methods Exploration of Inequality Aversion With Members of the General Public. 为什么反对健康和福祉方面的不平等?一个混合方法的探索不平等厌恶与普通公众的成员。
IF 6 2区 医学 Q1 ECONOMICS Pub Date : 2026-02-25 DOI: 10.1016/j.jval.2026.01.029
Becky Field, Katherine E Smith, Nyantara Wickramasekera, Aki Tsuchiya

Objectives: To examine whether: (1) reasoning for distributional preferences depends on the domain of inequality; (2) reasoning for distributional preferences is affected by cause of inequality; (3) participants provide and explain responses that violate "monotonicity" (the welfare economics principle that, other things being equal, social welfare improves when at least one person is better-off); and (4) the above vary across the digital divide.

Methods: We used mixed-methods to collect qualitative and quantitative data, via online discussion groups with a survey (11 groups, n = 53) and telephone interviews (n = 15) with digital minority individuals. Participants considered scenarios comparing equal and unequal health and wellbeing outcomes for an imaginary island. Well-being was framed as "equivalent income" (described to participants as household spending money, with other life aspects being good).

Results: Distributional preferences varied by domain and cause of inequality but not digital status. Health inequality caused by financial inequality was widely unaccepted. Some preferred equal distributions, even when violating "monotonicity," citing fairness and social cohesion.

Conclusions: Recruiting across the digital divide and using mixed-methods enriches inequality aversion research, enhancing the inclusivity and legitimacy of DCEA.

分配成本效益分析(dcea)有助于决策者在医疗资源分配中纳入公平考虑。公众对卫生不平等的反感有据可查,但很少探讨其根本原因。现有的研究往往会引起社会经济群体对不平等的厌恶,可能会将对健康不平等的厌恶与家庭财务或福祉的不平等混为一谈。此外,在线调查系统地排除了不使用互联网的人,这是一个弱势群体。目的:检查是否:i)分配偏好的推理取决于不平等的领域;Ii)分配偏好的推理受到不平等原因的影响;Iii)参与者提供并解释违反“单调性”的回答(福利经济学原则,在其他条件相同的情况下,当至少有一个人变得更富裕时,社会福利就会改善);iv)上述内容因数字鸿沟而异。方法:我们采用混合方法收集定性和定量数据,通过在线讨论小组调查(11组,n=53)和电话访谈(n=15)与数字少数民族个人。参与者考虑了在一个虚构的岛屿上比较平等和不平等的健康和福祉结果的情景。幸福感被定义为“同等收入”(向参与者描述为家庭支出,生活的其他方面都很好)。结果:分配偏好随领域和不平等的原因而变化,而不是数字状态。由财政不平等造成的健康不平等普遍不被接受。有些人更喜欢平均分配,即使违反了“单调性”,理由是公平和社会凝聚力。结论:跨数字鸿沟招聘和使用混合方法丰富了不平等厌恶研究,增强了DCEA的包容性和合法性。
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引用次数: 0
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Value in Health
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