Value in Health最新文献_第5页

Predicting Healthcare Utilization Outcomes With Artificial Intelligence: A Large Scoping Review 用人工智能预测医疗保健利用结果：大范围回顾。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.007

Carlos Gallego-Moll MSc , Lucía A. Carrasco-Ribelles PhD , Marc Casajuana MSc , Laia Maynou PhD , Pablo Arocena PhD , Concepción Violán MD, PhD , Edurne Zabaleta-del-Olmo PhD

Objectives

To broadly map the research landscape to identify trends, gaps, and opportunities in data sets, methodologies, outcomes, and reporting standards for artificial intelligence (AI)-based healthcare utilization prediction.

Methods

We conducted a scoping review following the Joanna Briggs Institute methodology. We searched 3 major international databases (from inception to January 2025) for studies applying AI in predictive healthcare utilization. Extracted data were categorized into data sets characteristics, AI methods and performance metrics, predicted outcomes, and adherence to the Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis (TRIPOD) + AI reporting guidelines.

Results

Among 1116 records, 121 met inclusion criteria. Most were conducted in the United States (62%). No study incorporated all 6 relevant variable groups: demographic, socioeconomic, health status, perceived need, provider characteristics, and prior utilization. Only 7 studies included 5 of these groups. The main data sources were electronic health records (60%) and claims (28%). Ensemble models were the most frequently used (66.9%), whereas deep learning models were less common (16.5%). AI methods were primarily used to predict future events (90.1%), with hospitalizations (57.9%) and visits (33.1%) being the most predicted outcomes. Adherence to general reporting standards was moderate; however, compliance with AI-specific TRIPOD + AI items was limited.

Conclusions

Future research should broaden predicted outcomes to include process- and logistics-oriented events, extend applications beyond prediction—such as cohort selection and matching—and explore underused AI methods, including distance-based algorithms and deep neural networks. Strengthening adherence to TRIPOD-AI reporting guidelines is also essential to enhance the reliability and impact of AI in healthcare planning and economic evaluation.

目的：广泛地描绘研究前景，以确定基于人工智能的医疗保健利用预测的数据集、方法、结果和报告标准方面的趋势、差距和机会。方法：我们按照乔安娜布里格斯研究所的方法进行了范围审查。我们检索了三个主要的国际数据库（从成立到2025年1月），寻找将人工智能应用于预测性医疗保健利用的研究。提取的数据分为数据集特征、人工智能方法和性能指标、预测结果以及对TRIPOD+AI报告指南的遵守情况。结果：1116例病例中，符合纳入标准的121例。大多数是在美国进行的（62%）。没有一项研究纳入了所有六个相关变量组：人口统计学、社会经济、健康状况、感知需求、提供者特征和既往使用情况。只有7项研究包括了其中的5个群体。主要数据来源是电子健康记录（60%）和索赔（28%）。集成模型是最常用的（66.9%），而深度学习模型则不太常用（16.5%）。人工智能方法主要用于预测未来事件（90.1%），其中住院（57.9%）和就诊（33.1%）是预测最多的结果。对一般报告标准的遵守程度中等，但对AI特定TRIPOD+AI项目的遵守程度有限。结论：未来的研究应扩大预测结果，包括以过程和物流为导向的事件，将应用扩展到预测之外，如队列选择和匹配，并探索未充分利用的人工智能方法，包括基于距离的算法和深度神经网络。加强对TRIPOD-AI报告准则的遵守对于提高AI在医疗保健规划和经济评估中的可靠性和影响也至关重要。

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引用次数: 0

Adult Social Care Outcomes Toolkit and ICEpop Capability Measure in Decision Making: A Review of NICE Social Care and Public Health Guidelines ASCOT和ICECAP在决策中的作用：NICE社会关怀和公共卫生指南综述

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.021

Zhixin Zhang MSc , Tuba Saygın Avşar PhD , Sophie Cooper BSc , Jeremy Dietz MSc

Objectives

When the National Institute for Health and Care Excellence (NICE) assesses whether interventions in health and social care offer value for money, where possible, it considers health effects expressed in quality-adjusted life-years. NICE’s preferred measure of health-related quality of life is EQ-5D. For nonhealth effects, NICE cites Adult Social Care Outcomes Toolkit (ASCOT) and ICEpop Capability Measure (ICECAP) as possible outcomes. To date, to our knowledge, their use in NICE guidelines has not been reviewed. The objectives of this study were to (1) review how ASCOT and ICECAP have been used in NICE social care and public health guideliens and (2) contextualize the review via expert interviews.

Methods

NICE social care and public health guidelines published before August 26, 2025 were reviewed, and information on the use of ASCOT and ICECAP was extracted. Five experts were interviewed to contextualize the review findings.

Results

Of the eligible guidelines, ASCOT appeared as an outcome in 4% and ICECAP in 1%. Neither measure significantly affected committee’s decision making. Interview findings were grouped into 2 themes: (1) reasons behind the limited use of these measures (with 3 subthemes: conceptual, system-wide issues, and implementation challenges) and (2) ongoing developments and future opportunities.

Conclusions

ASCOT and ICECAP appeared infrequently in the NICE guidelines reviewed, and when used, their impact on committee decision making was limited—either because of trial-specific limitations or reliance on other forms of evidence. Experts suggested several barriers to the use of these measures, and although these barriers are not insurmountable, it is unclear whether such measures may appear more in future NICE social care and public health guidelines.

背景：当国家健康和护理卓越研究所（NICE）评估健康和社会护理干预措施是否物有所值时，在可能的情况下，它会考虑以质量调整生命年（QALYs）表示的健康影响。NICE对健康相关生活质量的首选测量是EQ-5D。对于非健康影响，NICE将ASCOT和ICECAP列为可能的结果。迄今为止，它们在NICE指南中的使用尚未被审查。目的：本研究的目的是1)回顾ASCOT和ICECAP在NICE社会护理和公共卫生指南中的应用情况，2)通过专家访谈将回顾置于背景中。方法：回顾2025年8月26日之前发布的NICE社会护理和公共卫生指南，提取有关ASCOT和ICECAP使用的信息。五位专家接受了采访，以了解审查结果的背景。结果：在符合条件的指南中，ASCOT作为结局出现的比例为4%，ICECAP为1%。这两项措施都没有显著影响委员会的决策。访谈结果分为两个主题：(1)这些措施有限使用背后的原因（有3个分主题：概念、全系统问题和实施挑战）；(2)持续的发展和未来的机会。结论：ASCOT和ICECAP在NICE审查的指南中很少出现，当使用时，它们对委员会决策的影响是有限的，要么是由于试验特定的限制，要么是依赖于其他形式的证据。专家们提出了使用这些措施的几个障碍，尽管这些障碍并非不可克服，但尚不清楚这些措施是否会更多地出现在未来的NICE社会护理和公共卫生指南中。

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引用次数: 0

Methodological Insights From Health Valuation Studies in Low- and Middle-Income Countries: A Scoping Review 中低收入国家健康评估研究的方法学见解：范围审查。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.014

Thomas Gadsden PhD , Janine Verstraete PhD , Audrey Moyo MPhil , Stanley Carries PhD , Nokwanda Sithole BA , Eugene Lee Davids PhD , Donela Besada PhD , Blake Angell PhD , Stephen Jan PhD , Darshini Govindasamy PhD

Objectives

Valuation studies generate utility values for health states using stated preference methods. These studies are complex and resource-intensive, posing implementation challenges in low- and middle-income countries (LMICs). This review aimed to examine how valuation studies in LMICs have navigated these challenges.

Methods

A scoping review was conducted, with database (MEDLINE, EMBASE, and CINAHL) and gray literature searches performed between April and June 2024. Inclusion was limited to valuation studies in LMICs using generic or disease-specific instruments and stated preference techniques with adult respondents and published in English. Results were narratively synthesized.

Results

A total of 36 studies from 22 LMICs were included. Studies were conducted across low (n = 2), lower-middle (n = 11), and upper middle-income countries (n = 9). Half were published since 2020. There were 33 studies that developed nationally representative value sets, 2 of which were based on patient preferences. Two pilot studies and 1 cancer-specific value set were also included. The EQ-5D-5L was used most (n = 16), followed by the EQ-5D-3L (n = 14), Short Form-6 Dimension (n = 4), Chinese medicine Quality of life-11 Dimensions (n = 1), and European Organization for Research and Treatment of Cancer-8 dimension (n = 1). Methodological adaptations included “lite” protocols, portable tools, and crosswalk methodology. Comprehension aids were reported in 11 studies; 5 included illiterate participants, and 7 were conducted in multiple languages.

Conclusions

Valuation studies are increasing rapidly in LMICs, and there is growing experimentation to reduce resource demands and enhance inclusivity. Although this is promising, the resource demands of valuation studies still limit their implementation in low-income settings. Therefore, these countries may still find it more cost-efficient to adapt value sets from neighboring countries rather than develop their own.

目的：评估研究使用既定偏好方法生成健康状态的效用值。这些研究复杂且资源密集，对低收入和中等收入国家的实施构成挑战。本综述旨在探讨中低收入国家的估值研究如何应对这些挑战。方法：采用数据库（MEDLINE、EMBASE和CINAHL）和灰色文献检索（2024年4 - 6月）进行范围综述。纳入的研究仅限于在低收入和中等收入国家进行的评估研究，这些研究使用通用或特定疾病的工具和成人受访者的陈述偏好技术，并以英语发表。对结果进行叙述性综合。结果：纳入了来自22个低收入国家的36项研究。研究在低收入国家（n=2）、中低收入国家（n=11）和中高收入国家（n=9）进行。其中一半是在2020年以后出版的。33项研究开发了具有全国代表性的价值集，其中两项基于患者的偏好。还包括两项试点研究和一项癌症特异性值集。使用最多的是EQ-5D-5L (n=16)，其次是EQ-5D-3L （n=14）、SF-6D （n=4）、CQ-11D （n=1）和EORTC-8D （n=1）。方法上的调整包括“精简”协议、便携式工具和人行横道方法。11项研究报告了理解辅助工具；其中5个是文盲，7个是用多种语言进行的。结论：估值研究在中低收入国家迅速增加，减少资源需求和增强包容性的实验也越来越多。虽然这是有希望的，但估价研究的资源需求仍然限制了它们在低收入环境中的执行。因此，各国可能仍会发现，采用邻国的价值体系比开发自己的价值体系更具成本效益。

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引用次数: 0

Integrating Price Benchmarks and Comparative Clinical Effectiveness to Predict Initial Price Offers for Medicare Drug Price Negotiation (Initial Price Applicability Year 2027) 整合价格基准和比较临床有效性预测医疗保险药品价格谈判的初始报价（IPAY 2027）。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.09.3054

Kevin H. Li PharmD, MSc , Emma M. Cousin PharmD , Nico Gabriel MA , Sean D. Sullivan BScPharm, PhD

Objectives

This study estimated initial price offers for the 15 drugs selected for the Medicare Drug Price Negotiation Program in the Initial Price Applicability Year 2027.

Methods

We applied the Centers for Medicare and Medicaid Services guidance to construct a list of therapeutic alternatives for each drug. Price benchmarks included the statutory discount, Big 4/Federal Supply Schedule prices, estimated Medicare Part D net prices, and wholesale acquisition cost. Comparative effectiveness evidence was extracted from peer-reviewed network meta-analyses, clinical guidelines, and Institute for Clinical and Economic Review assessments. Drugs were rated on a 4-tier scale (A-D) based on comparative net health benefit. Initial offers were then estimated by applying market-based discounts depending on the availability and type (branded vs generic) of therapeutic alternatives.

Results

For 6 drugs, statutory or Big 4/Federal Supply Schedule prices anchored the estimated initial offers. Four drugs were informed by clinically comparable branded alternatives, leading to approximately 20% reductions from net price. Two drugs with primarily generic alternatives received larger discounts of approximately 30%. Three drugs with therapeutic alternatives previously negotiated in Initial Price Applicability Year 2026 were assigned either the established maximum fair price or a market-based premium (semaglutide). Across all 15 drugs, estimated discounts ranged from 32% to 78% off list price and 16% to 56% off net price.

Conclusions

Our analysis highlights how Centers for Medicare and Medicaid Services may incorporate statutory discounts, prior maximum fair prices, and comparative effectiveness evidence into initial price offers, although uncertainty remains. An explicit health technology assessment framework could strengthen future negotiation cycles unless international price referencing policy intercedes.

目的：本研究估计了在初始价格适用年（IPAY） 2027年为医疗保险药品价格谈判项目选择的15种药物的初始价格。方法：我们应用美国医疗保险和医疗补助服务中心（CMS）的指导，构建每种药物的治疗方案清单。价格基准包括法定折扣、四大/联邦供应计划（FSS）价格、估计的医疗保险D部分净价格和批发采购成本。比较有效性的证据是从同行评议的网络荟萃分析、临床指南和临床与经济评论研究所评估中提取的。根据相对净健康效益，将药物分为四级（a - d）。然后根据治疗替代方案的可用性和类型（品牌vs.仿制药）应用基于市场的折扣来估计初始报价。结果：对于六种药物，法定价格或四大/FSS价格锚定了估计的初始报价。有四种药物被告知具有临床可比性的品牌替代品，导致净价降低约20%。两种主要具有非专利替代品的药物获得了约30%的较大折扣。先前在IPAY 2026中协商的具有治疗替代方案的三种药物被指定为既定的最高公平价格（MFP）或基于市场的溢价（semaglutide）。在所有15种药物中，估计折扣幅度从目录价格的32-78%到净价的16-56%不等。结论：我们的分析强调了CMS如何将法定折扣、先前的mfp和比较有效性证据纳入初始报价，尽管仍存在不确定性。除非国际价格参考政策介入，明确的卫生技术评估框架可加强未来的谈判周期。

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引用次数: 0

Operationalizing Distributional Cost-Effectiveness in Genomic Medicine: A United States–Focused Comment 在基因组医学中实施分配成本效益：对Smith等人的美国关注评论（2025）。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.09.3068

Quan Zhang PhD

引用次数: 0

Incorporating Informal Caregiving Time Into Cost-Effectiveness Analysis for Adults Aged 50 and Older: Estimating From Health-Related Quality of Life and Functional Status 将非正式护理时间纳入50岁及以上成年人的成本-效果分析：从健康相关生活质量和功能状态估计

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.07.026

Boshen Jiao PhD, MPH , Xinran Lu MS , Darius N. Lakdawalla PhD , William V. Padula PhD , Fangli Geng PhD, MS

Objectives

Informal caregiving, often provided by family and friends, is essential for supporting individuals with functional impairments. However, these caregiving costs are frequently excluded from cost-effectiveness analyses (CEAs), potentially biasing the evaluation of medical interventions dependent on informal caregiving. Although guidelines recommend including these costs in CEA from a societal perspective, implementation has been inconsistent because of limited data. To address this gap, we developed US-based models estimating informal caregiving time by linking it to health-related quality of life (HrQoL) of care recipients.

Methods

Using data from the 2010 to 2018 waves of the Health and Retirement Study, a nationally representative survey of Americans aged 50+, we focused on individuals with limitations in activities of daily living (ADLs) or instrumental ADLs (IADLs). We performed regression analyses to estimate the association between informal caregiving time and HrQoL, developing separate models with and without ADL/IADL predictors. Additionally, we created models with both preference-based health utility scores from the Health Utilities Index Mark 3 and visual analog scale scores as the HrQoL measures.

Results

The models demonstrated strong predictive accuracy based on cross-validation. Findings from the models using HrQoL as the primary predictor indicate that lower Health Utilities Index or visual analog scale scores are associated with increased caregiving time. The models incorporating the number of ADL and IADL limitations as additional predictors further highlight the growing demand for informal caregiving as functional impairments worsen.

Conclusions

These models provide practical tools to facilitate the inclusion of informal caregiving costs in US-based CEAs.

目的：通常由家人和朋友提供的非正式照料对于支持有功能障碍的个人至关重要。然而，这些护理费用经常被排除在成本效益分析（cea）之外，这可能会对依赖于非正式护理的医疗干预措施的评估产生偏见。尽管指南建议从社会角度将这些成本包括在CEA中，但由于数据有限，实施一直不一致。为了解决这一差距，我们开发了基于美国的模型，通过将非正式护理时间与护理对象的健康相关生活质量（HrQoL）联系起来，来估计非正式护理时间。方法：使用2010-2018年健康与退休研究（HRS）的数据，这是一项针对50岁以上美国人的全国代表性调查，我们重点关注日常生活活动（ADLs）或工具性ADLs （IADLs）受限的个体。我们进行了回归分析，以估计非正式护理时间与HrQoL之间的关系，并开发了单独的模型，包括ADL/IADL预测因子和不包括ADL/IADL预测因子。此外，我们创建了基于健康效用指数（HUI）标记3的基于偏好的健康效用评分和视觉模拟量表（VAS）评分作为HrQoL测量的模型。结果：基于交叉验证的模型显示出较强的预测准确性。使用HrQoL作为主要预测因子的模型结果表明，较低的HUI或VAS评分与护理时间的增加有关。将ADL和IADL限制数量作为额外预测因素的模型进一步强调，随着功能障碍的恶化，对非正式护理的需求日益增长。结论：这些模型提供了实用的工具，有助于在美国的cea中纳入非正式护理成本。

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引用次数: 0

Toward a Common Ground for Defining Digital Health Interventions, Mapping Digital Health Frameworks to PICOTS-ComTeC: An ISPOR Special Interest Group Report 迈向定义数字健康干预措施的共同基础，将数字健康框架映射到picot - comtec: ISPOR特别兴趣小组报告。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.07.022

Annette Champion MBA , Anita Burrell MA , Anke-Peggy Holtorf PhD , Rossella Di Bidino PhD , Jagadeswara Rao Earla PhD , Artem T. Boltyenkov PhD , Masami Tabata-Kelly MA , Carl Asche PhD , Brian Seal PhD , Zsombor Zrubka PhD

Objectives

Digital health interventions (DHIs) should be defined in a comparable, structured manner to facilitate research informing clinical and financial decisions. The Population, Intervention, Comparator, Outcomes, Timing, Setting, Communication, Technology, Context (PICOTS-ComTeC) framework was developed to define patient-facing DHIs for health economics and outcomes research. Our objective was to compare PICOTS-ComTeC with established DHI frameworks and guidelines to determine the degree of overlap, additional value of PICOTS-ComTeC, and how the frameworks might be used together.

Methods

An expert group selected comparator frameworks. Reviewer pairs extracted information and mapped DHI definitions to 9 domains and 32 subcategories of PICOTS-ComTeC. A third reviewer checked for consistency across frameworks and missing data.

Results

A diverse group of 16 frameworks (9 international, regarding DHI classification, quality, labeling, and reporting; 6 national DHI health technology assessment and payer; 1 international health economic reporting) were compared with PICOTS-ComTeC. Across all frameworks, 81% (116/144) of PICOTS-ComTeC domains matched (range 44%-100%). The mean number of domain matches for a framework was 7.3. Comparator frameworks matched 48% (247/512) of PICOTS-ComTeC subcategories (range 16%-81%).

Conclusions

The degree to which PICOTS-ComTeC is congruous with items in diverse DHI frameworks suggests that PICOTS-ComTeC represents a common ground for defining patient-facing DHIs for research, reporting, and assessment purposes, thereby improving patient care by accelerating adoption of effective DHIs. PICOTS-ComTeC contains items not uniformly present in comparator frameworks. PICOTS-ComTeC can be used to define patient-facing DHIs by adding missing PICOTS-ComTeC items to comparator frameworks or using information from comparator frameworks to describe PICOTS-ComTeC items.

目标：应以可比较的、结构化的方式定义数字卫生干预措施（DHIs），以促进为临床和财务决策提供信息的研究。PICOTS-ComTeC（人口、干预、比较、结果、时间、设置、沟通、技术、环境）框架被开发用于定义面向患者的DHIs，用于卫生经济学和结果研究。我们的目标是比较PICOTS-ComTeC与已建立的DHI框架和指南，以确定重叠的程度，PICOTS-ComTeC的附加价值，以及如何将这些框架一起使用。方法：专家组选择比较框架。审稿人对提取信息并将DHI定义映射到PICOTS-ComTeC的9个域和32个子类别。第三个审稿人检查了框架之间的一致性和丢失的数据。结果：16个框架(9个国际框架，关于DHI分类、质量、标签和报告；6 .国家卫生技术评估与支付人；一份国际卫生经济报告)与PICOTS-ComTeC进行比较。在所有框架中，81%（116/144）的PICOTS-ComTeC域匹配（范围为44%-100%）。一个框架的平均域匹配数为7.3。比较框架匹配48%（247/512）的PICOTS-ComTeC子类别（范围16%-81%）。结论：PICOTS-ComTeC与不同DHI框架项目的一致程度表明，PICOTS-ComTeC代表了为研究、报告和评估目的定义面向患者的DHIs的共同基础，从而通过加速采用有效的DHIs来改善患者护理。PICOTS-ComTeC包含的项目在比较器框架中并不统一。PICOTS-ComTeC可用于定义面向患者的DHIs，方法是将缺少的PICOTS-ComTeC项目添加到比较器框架中，或者使用比较器框架中的信息来描述PICOTS-ComTeC项目。

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引用次数: 0

Implications of Value Set Choice on EQ-5D-Y-3L Child and Proxy Health-Related Quality of Life Ratings: What to Do When a Country-Specific “Y” Value Set Is Unavailable? 值集选择对EQ-5D-Y-3L儿童和代理HRQoL评分的影响：当国家特定的“Y”值集不可用时该怎么办？

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.004

Diana Khanna PhD , Jyoti Khadka PhD , Christine Mpundu-Kaambwa PhD , Rachel Milte PhD , Julie Ratcliffe PhD , Quality of Life in Kids: Key evidence to strengthen decisions in Australia (QUOKKA) project team

Objectives

There is limited guidance on whether to apply an available EQ-5D-Y-3L “Y” value set from another country or use a country-specific EQ-5D-3L “adult” value set when a country-specific “Y” value set is unavailable. This study aims to examine how the choice of value set (ie, “Y” or “adult”) influences the interrater gap between child-self and proxy-reported health-related quality of life (HRQoL).

Methods

An online sample of 845 dyads (children aged 6-10 years and parents) independently completed the self and proxy versions of the EQ-5D-Y-3L. Corresponding HRQoL values were derived using the “Y” and the “adult” value sets for 5 countries: Germany, Hungary, Japan, The Netherlands, and Spain. Analyses were stratified by age (6-7 vs 8-10-year-olds), gender (boys vs girls), and health condition (no vs yes). Group differences were identified using paired t tests. The percentage of directional consistency in child-proxy discrepancies across value sets was also examined as a secondary analysis.

Results

Proxies significantly overestimated HRQoL values across most “Y” value sets (Hungary, Japan, and Spain). Significant discrepancies using the corresponding “adult” value sets were observed only for Germany. Additionally, significant interrater differences were observed for children without health conditions across all value sets. Proportional agreement in direction was marginally higher when using “Y” value sets, except for Germany.

Conclusions

The choice of value set influences child-proxy HRQoL assessments. In the absence of a country-specific “Y” value set, using an alternative “Y” value set is preferable to relying solely on a country-specific “adult” value set.

目的：对于是否应用来自其他国家的可用EQ-5D-Y-3L“Y”值集，或者在无法获得特定国家的“Y”值集时使用特定国家的EQ-5D-3L“成人”值集，目前的指导有限。本研究旨在检验值集（即“Y”或“成人”）的选择如何影响儿童自我和代理报告的健康相关生活质量（HRQoL）之间的评分差距。方法：在线抽样845对（6-10岁儿童和家长）独立完成EQ-5D-Y-3L的自我版和代理版。相应的HRQoL值是使用“Y”和“成人”值集得出的，这五个国家是：德国、匈牙利、日本、荷兰和西班牙。分析按年龄（6-7岁vs 8-10岁）、性别和健康状况分层。使用配对t检验确定组间差异。跨值集的儿童代理差异的方向一致性百分比也作为次要分析进行了检查。结果：代理在大多数“Y”值集（匈牙利、日本、西班牙）中显著高估了HRQoL值。使用相应的“成人”值集，仅在德国观察到显著差异。此外，在所有值集中，对没有健康状况的儿童观察到显著的评分间差异。当使用“Y”值集时，除德国外，方向上的比例一致性略高。结论：值集的选择影响儿童代理HRQoL的评价。在没有特定国家的“Y”值集的情况下，使用替代的“Y”值集比完全依赖特定国家的“成人”值集更可取。

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引用次数: 0

A Generalized Risk-Adjusted Cost-Effectiveness Economic Model for Measuring the Value of Interventions That Delay Mobility Impairment Across Neurological Conditions 一个广义风险调整成本效益（GRACE）经济模型，用于测量延迟神经系统疾病的行动障碍干预的价值。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.08.006

Jason Shafrin PhD , Jaehong Kim PhD , Jacob Fajnor BA , Kyi-Sin Than MPH , Elizabeth S. Mearns PharmD , Stacey L. Kowal MSc , Thomas Majda PharmD , Jakub P. Hlávka PhD

Objectives

To quantify how incorporating patient risk preferences and severity adjustments affect the value of a hypothetical treatment for mobility impairments caused by neurological conditions.

Methods

A 5-state Markov model was developed to measure the health economic value of a hypothetical treatment delaying the progression of mobility impairments by 30.7% versus standard of care for patients who were 45-year-old, minimally impaired, and had received a diagnosis of a neurological condition. A generalized and risk-adjusted cost-effectiveness (GRACE) model was implemented using relative risk aversion estimates from a US general population survey. Treatment value was measured as risk-aversion and severity-adjusted net monetary benefit (NMB), defined as (1) risk-adjusted health gains (generalized risk-adjusted quality-adjusted life-years [GRA-QALYs]) monetized by (2) risk-aversion and severity-adjusted willingness to pay less (3) incremental costs. Risk-neutral results (traditional cost-effectiveness analysis [TCEA]) were compared.

Results

Incorporating risk preferences and disease severity increased the value of health benefits. Incremental health gains from using the hypothetical treatment (vs standard of care) were valued more when accounting for risk preferences with GRACE (1.358 GRA-QALYs vs 1.199 QALY). Willingness to pay for these health gains was higher when computed under GRACE compared with TCEA ($109 656 per GRA-QALY vs $100 000 per QALY). Overall, NMB increased by 11.6% (risk-aversion and severity-adjusted NMB = $278 324 vs TCEA NMB = $249 311) using GRACE versus TCEA. Results were sensitive to risk-aversion estimates and the functional form of patient utility.

Conclusions

In the first application of GRACE within neurology, GRACE increased the health economic value of a hypothetical neurology treatment, suggesting that TCEA may undervalue treatments for mobility-related neurological impairments.

目的：量化纳入患者风险偏好和严重程度调整如何影响神经系统疾病引起的活动障碍的假设治疗价值。方法：建立了一个五状态马尔可夫模型来衡量一种假设的治疗方法的健康经济价值，这种治疗方法比标准护理（SoC）延迟30.7%的运动障碍进展，诊断为神经系统疾病的45岁轻度损伤患者。使用来自美国普通人群调查的相对风险厌恶（RRA）估计，实现了广义和风险调整成本效益（GRACE）模型。治疗价值以风险厌恶和严重程度调整后的净货币效益（RASA-NMB）来衡量，定义为：(i)风险调整后的健康收益（GRA-QALYs）货币化（ii）风险厌恶和严重程度调整后的支付意愿（RASA-WTP）减去（iii）增量成本。风险中性结果（传统成本效益分析（TCEA））进行比较。结果：纳入风险偏好和疾病严重程度增加了健康益处的价值。当考虑GRACE的风险偏好时，使用假设治疗（vs. SoC）的增量健康收益更有价值（1.358 gra -QALY vs. 1.199 QALY）。与TCEA相比，GRACE下计算这些健康收益的支付意愿更高（每个GRA-QALY为109,656美元，每个QALY为100,000美元）。总体而言，使用GRACE与TCEA相比，净货币效益增加了11.6% （RASA-NMB = 278,324美元，而传统CEA NMB = 249,311美元）。结果对风险规避估计和患者效用的功能形式敏感。结论：在GRACE在神经病学中的首次应用中，GRACE增加了一种假设的神经病学治疗的健康经济价值，这表明TCEA可能低估了运动相关神经损伤的治疗价值。

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引用次数: 0

Combining Simulation Model-Based Outcomes With County-Level Data for Geographic Health Equity Impact Evaluations of New Interventions 将基于模拟模型的结果与县级数据相结合，用于新干预措施的地理卫生公平影响评估。

IF 6 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2026-01-01 DOI: 10.1016/j.jval.2025.07.025

Jeroen P. Jansen PhD , Michael P. Douglas MS , Kathryn A. Phillips PhD

Objectives

Geographic health disparities persist across the United States, with substantial variations in health outcomes between regions. Evaluating how emerging health technologies might affect these disparities is crucial for developing equitable health policies. This article introduces an approach for geographic health equity impact evaluation by combining predicted outcomes by equity-relevant subgroup from a simulation model with US county-level data on subgroup proportions.

Methods

The approach involves the following steps: (1) create a data set with county-level information on equity-relevant factors and lifetime risk of the target indication; (2) estimate quality-adjusted life-years (QALYs) and costs with and without the intervention for different combinations of equity-relevant factors with the simulation model; (3) calculate expected and incremental QALYs in target population, incremental net health benefits per 100 000 general population, and quality-adjusted life expectancy at birth without and with liquid biopsy for each county based on its distribution of equity-relevant factors and step 2 estimates; and (4) quantify inequality in QALYs and quality-adjusted life expectancies between counties with and without the technology and the corresponding health equity impact.

Results

We illustrate the approach using liquid biopsy for first-line treatment in non-small cell lung cancer. Future applications should incorporate more detailed information on the equity-relevant groups by county.

Conclusions

Combining simulation model outcomes with county-level data on equity-relevant subgroups provides a novel approach for health equity impact evaluations of new interventions. It facilitates examining how introducing a new health technology can affect geographic disparities in health and can help identify areas that may benefit most from a new intervention.

目的：美国各地的地理健康差异持续存在，各地区之间的健康结果存在很大差异。评估新兴卫生技术可能如何影响这些差异，对于制定公平的卫生政策至关重要。本文介绍了一种地理健康公平影响评估方法，该方法将模拟模型中与公平相关的子组预测结果与美国县级子组比例数据相结合。方法：该方法包括以下步骤：1)创建县级股票相关因素和目标适应症终身风险信息数据集；2)利用仿真模型估计有和没有干预的不同股权相关因素组合的质量年和成本；3)根据公平相关因素的分布和第2步估计，计算每个县目标人口的预期和增量质量预期寿命、每10万普通人口的增量净健康福利，以及没有和有LB的质量调整出生预期寿命（QALEs）；4)量化有技术县与无技术县在质量质量年和质量质量方面的不平等以及相应的卫生公平影响。结果：我们阐述了液体活检在非小细胞肺癌的一线治疗中的方法。未来的申请应按县纳入更多有关股权相关群体的详细信息。结论：将模拟模型结果与县级公平相关亚组数据相结合，为新干预措施的卫生公平影响评估提供了一种新方法。它有助于审查引进一项新的卫生技术如何影响卫生领域的地域差异，并有助于确定可能从一项新的干预措施中获益最多的地区。

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引用次数: 0