Value in Health最新文献_第3页

Prescription Medication Use and Expenditure for Atrial Fibrillation in the United States 美国房颤的处方药使用和支出。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.11.012

Harshith Thyagaturu MD , Karthik Seetharam MD , Nicholas Roma MD , Neel Patel MD , Jordan Lacoste PharmD , Vikram Padala BS , Karthik Gonuguntla MD , Muhammad Bilal Munir MD , Sudarshan Balla MD

Objectives

To study the national trends of anticoagulants, antiarrhythmic drugs (AADs), and expenditures in the civilian noninstitutionalized atrial fibrillation (AF) population.

Methods

The Medical Expenditure Panel Survey was queried from January 2016 to December 2021 to identify adults (age ≥18 years) with a diagnosis of AF utilizing the International Classification of Diseases, Tenth Revision, Clinical Modification code I48. Prevalence of anticoagulants (AAD) and its expenditure and AF expenditure across clinical settings in the United States were estimated. The predictors of anticoagulant use were identified utilizing multivariate logistic regression analysis.

Results

A total of 17.3 million AF adults were identified, of which 46.5% were female, 89.6% were White, and ∼70% were middle/high income with prevalent comorbidities of hypertension (75.3%) and coronary heart disease (30%). The mean CHA₂DS₂ VASc score was 3.2, and 40% had a score of ≥4. In the United States, an average of $26 103 (2021 inflation adjusted) was spent per year per adult with AF for health-related expenditures. The prevalence of direct oral anticoagulants (DOACs) and class I AAD use has increased; in contrast, vitamin K antagonists use has declined. DOAC-related per person annual expenses increased from $849 in 2016 to $1929 in 2021. In those with a CHA₂DS₂ VASc score of ≥2, female sex and the presence of coronary heart disease were associated with a lower likelihood of anticoagulant use.

Conclusions

AF is a costly condition in which prescription medication use, such as DOACs and class III AADs, are significant contributors.

目的：了解全国非住院房颤人群抗凝剂、抗心律失常药物（AAD）及支出趋势。方法：对2016年1月至2021年12月的医疗支出小组调查（MEPS）进行查询，使用ICD-10-CM代码“I48”识别诊断为房颤的成年人（年龄≥18岁）。估计了美国临床环境中抗凝血剂、AAD的患病率及其支出和AF支出。使用多变量logistic回归分析确定抗凝剂使用的预测因素。结果：共鉴定出1730万名房颤成年人，其中46.5%为女性，89.6%为白人，约70%为中等/高收入人群，普遍存在高血压（75.3%）和冠心病（30%）合并症。平均CHA2DS2 VASc评分为3.2分，40%评分≥4分。在美国，每位患有房颤的成年人每年平均花费26103美元（经2021年通货膨胀调整后）用于健康相关支出。直接口服抗凝剂（DOAC）和I类AAD使用的流行率有所增加；相反，维生素K拮抗剂（VKA）的使用有所下降。doac相关的人均年支出从2016年的849美元增加到2021年的1929美元。在CHA2DS2 VASc评分≥2的患者中，女性和存在冠心病与使用抗凝血剂的可能性较低相关。结论：房颤是一种昂贵的疾病，处方药的使用，如DOACs和III类aad，是重要的贡献者。

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引用次数: 0

Value Framework Based on Multiple-Criteria Decision Analysis for Assessment of New Health Technologies Under Universal Healthcare Coverage System in Taiwan 基于多准则决策分析的台湾全民健保新技术评估价值架构。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.11.009

Thi Thuy Dung Nguyen MS , Yu-Hsuan Lee MPH , Yu-Jr Lin MS , Shu-Chen Chang PhD , Fei-Yuan Hsiao PhD , Chee-Jen Chang PhD , Huang-Tz Ou PhD

Objectives

Given the lack of a value framework for assessing health technologies in Asian settings, a value framework incorporating multiple-criteria decision analysis for new drugs under universal healthcare coverage in Taiwan was established.

Methods

The development process included (1) the adoption of 5 value domains (ie, Overall clinical benefit, Disease burden, Alignment with patient concerns, Economic value, and Feasibility of adoption into the health system) and 26 corresponding indicators, derived from the literature and expert discussions; (2) the creation of separate weighting schemes for 3 drug types—new oncology, new orphan, and other new drugs—based on inputs from multiple stakeholders (n = 86) using various weighting methods; and (3) the application of the value framework to cases of new oncology drugs.

Results

Overall clinical benefit had the highest preference weight, irrespective of drug type, (ie, mean values [95% CIs] for new oncology, new orphan, and other new drugs: 32.5 [30.4–34.6], 30.6 [28.1–33.1], and 30.6 [28.7–32.6], respectively), weighting method, and stakeholder type. The 5 domain-derived weights (from the point allocation method) were comparable to the 26 indicator-derived weights (from the direct rating method), suggesting that the value framework with a short-form (domain-derived) weighting scheme is sufficient to support decision making under time and resource constraints.

Conclusions

A country-specific value framework incorporating multiple-criteria decision analysis for new drugs was developed in an Asian setting under universal healthcare coverage. It allows multiple stakeholders to systematically appraise all drug value attributes and provides a structured process for adapting and refining value assessments.

目的：考虑到亚洲地区缺乏评估医疗技术的价值框架，本文建立了台湾全民医疗覆盖下新药多标准决策分析（MCDA）的价值框架。方法：制定过程包括：1)采用5个价值域（即“总体临床效益”、“疾病负担”、“与患者关注的一致性”、“经济价值”和“纳入卫生系统的可行性”）和26个相应指标，这些指标来源于文献和专家讨论；2)基于多个利益相关者（n=86）使用各种加权方法的输入，为三种药物类型（新肿瘤药物、新孤儿药和其他新药）创建单独的加权方案；3)价值框架在肿瘤新药案例中的应用。结果：无论药物类型（即新肿瘤药、新孤儿药和其他新药的平均值[95%置信区间]分别为32.5[30.4-34.6]、30.6[28.1-33.1]和30.6[28.7-32.6]）、加权方法和利益相关者类型，“总体临床获益”的偏好权重最高。5个领域衍生权重（来自点分配法）与26个指标衍生权重（来自直接评级法）具有可比性，表明具有简短形式（领域衍生）权重方案的价值框架足以支持时间和资源约束下的决策。结论：在全民医疗覆盖的亚洲环境中，开发了一个包含新药MCDA的国家特定价值框架。它允许多个利益相关者系统地评估所有药物价值属性，并提供一个结构化的过程来调整和改进价值评估。

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引用次数: 0

Financial Impacts of Paying for Gene Therapy for Sickle Cell Disease Under Alternative Pricing and Financing Mechanisms 在替代价格和融资机制下支付镰状细胞病基因治疗费用的财务影响。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.10.3848

Anirban Basu PhD

Objectives

This study aims to understand the role of alternative pricing and financing mechanisms on the budget impact for payers and the risks and returns of manufacturers for gene therapies.

Methods

This article uses fundamental economic principles to interpret the implications of alternative pricing mechanisms in terms of the share of value appropriated by the manufacturer and how alternative financing mechanisms alter it. It demonstrates these concepts by studying the financial impacts for a payer and the manufacturer across alternative pricing and financing mechanisms that could be used by the US Centers for Medicare and Medicaid Services to pay for gene therapy for sickle cell disease.

Results

Unlike value-based and manufacturer-set monopoly prices, an effective monopoly price can be derived to guarantee monopoly profits for manufacturers during their exclusivity period, thereby providing a high appropriation share and substantially lowering price and budget impact for a payer. For sickle cell disease gene therapy, the 10-year budget impact for the US Centers for Medicare and Medicaid Services would range from US dollar $8.6 billion to $12.8 billion under a value-based price, to $10.2 billion to $15.2 billion under a monopoly price, but reduce to $7.7 billion under an effective monopoly price. The latter price would still fetch over 50% of the total surplus to the manufacturer while mitigating their risk of sales volume.

Conclusions

Significant budget impacts for funding gene therapy are not mitigated across alternative financing mechanisms at any given price. The price determines most of the budget impact. The option of a patent buyout may help negotiate down prices to effective monopoly prices.

目的了解替代价格和融资机制对支付方预算影响的作用，以及基因疗法制造商的风险和收益：本文利用基本经济学原理，从生产商的价值分配份额以及替代融资机制如何改变价值分配份额的角度来解释替代定价机制的影响。本文通过研究美国医疗保险与医疗补助服务中心（CMS）在支付镰状细胞病（SCD）基因治疗费用时可能采用的替代定价和融资机制对支付方和制造商的财务影响来证明这些概念：与基于价值的垄断价格和制造商设定的垄断价格不同，有效的垄断价格可以保证制造商在独占期的垄断利润，从而提供高额的拨款份额，并大大降低对支付方的价格和预算影响。就 SCD 基因疗法而言，在基于价值的价格下，对 CMS 的 10 年预算影响将从 86 亿美元到 128 亿美元不等，在垄断价格下将从 102 亿美元到 152 亿美元不等，但在有效垄断价格下将降至 77 亿美元。后一种价格仍可为制造商带来超过 50%的总盈余，同时降低其销售量风险：我的研究表明，在任何给定的价格下，其他融资机制都无法减轻基因治疗对预算的重大影响。价格决定了大部分预算影响。选择专利买断可能有助于通过谈判将价格降至有效垄断价格。

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引用次数: 0

Healthcare Costs in the United States by Demographic Characteristics and Comorbidity Status 按人口特征和合并症状况分列的美国医疗费用。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.10.3847

Naomi N. Adjei MD, MPH , Allen Haas MS , Charlotte C. Sun DrPH, MPH , Hui Zhao PhD , Paul G. Yeh MD, DrPH , Sharon H. Giordano MD, MPH , Iakovos Toumazis PhD , Larissa A. Meyer MD, MPH

Objectives

Current, real-world healthcare cost information is needed to project future expenditures and inform policy. We estimated the healthcare costs for adults in 2019 in the United States by age, sex, race/ethnicity, geographic region, and comorbidity.

Methods

We aggregated and summarized the healthcare costs in 2021 US dollars using claims data derived from Optum’s deidentified Clinformatics® Data Mart Database, which includes inpatient, outpatient, and prescription claims for commercial and Medicare Advantage beneficiaries nationwide.

Results

A total of 9 227 901 adults were included in the analysis. The largest group represented was 71 to 75 years old (13%), female (53%), White (68%), received care in the South (41%), and had commercial health insurance (56%). There was a positive relationship between healthcare cost and age. Females had a 1.3-fold multiplicative increase in costs than males (95% CI 1.33–1.34). There were 92.5% of individuals who had health claims in the Northeast, 89.6% in the Midwest, 88.9% in the South, 77.1% in the West, and 12.7% with unknown geographic region. Patients with severe renal failure, heart failure, or metastatic cancer incurred the highest mean yearly costs ($139 844, $113 031, and $85 299, respectively). Metastatic cancer and severe renal failure were associated with a 5.3-fold multiplicative increase in costs than not having these conditions, after adjusting for potential confounders (95% CI 5.26–5.41 and 4.98–5.16, respectively).

Conclusions

We identified patient characteristics and medical conditions that are associated with high healthcare cost burden and could benefit from tailored interventions. We provided detailed cost estimates to aid healthcare modeling, cost projection, and cost-minimizing interventions.

目的：需要当前真实世界的医疗成本信息来预测未来支出并为政策提供信息。我们按年龄、性别、种族/人种、地理区域和合并症估算了美国成年人 2019 年的医疗成本：我们使用 Optum 的去标识 Clinformatics® Data Mart 数据库中的理赔数据，以 2021 年美元为单位对医疗费用进行了汇总：共有 9,227,901 名成年人参与了分析。其中最大的群体为 71-75 岁（13%）、女性（53%）、白人（68%）、在南方接受医疗服务（41%）、拥有商业医疗保险（56%）。医疗费用与年龄呈正相关。与男性相比，女性的费用增加了 1.3 倍（95% CI 1.33-1.34）。92.5% 的人在东北部、89.6% 的人在中西部、88.9% 的人在南部、77.1% 的人在西部，还有 12.7% 的人地域不详。严重肾功能衰竭、心力衰竭或转移性癌症患者的年平均费用最高（分别为 139844 美元、113031 美元和 85299 美元）。在对潜在的混杂因素进行调整后，转移性癌症和严重肾功能衰竭与没有这些病症的患者相比，费用增加了 5.3 倍（95% CI 分别为 5.26-5.41 和 4.98-5.16）：我们发现了与高医疗成本负担相关的患者特征和医疗条件，这些特征和条件可从有针对性的干预措施中获益。我们提供了详细的成本估算，以帮助进行医疗建模、成本预测和成本最小化干预。

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引用次数: 0

Measurement Equivalence of Standard and Zoom-Enabled Electronic Clinical Outcome Assessments: Implications for Patient Accessibility in Clinical Trials 标准电子临床结果评估与缩放电子临床结果评估的测量等效性：对临床试验中患者可及性的影响》。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.10.3801

Bryan McDowell MBA , Kelly M. Dumais PhD , Sarah T. Gary PhD , Helen A. Doll PhD , Gauri Nagrani , Tomás Ward PhD , Willie Muehlhausen DVM

Objectives

Diversity and inclusion in clinical trials remains an important topic, particularly for participants with disabilities such as vision impairment. With advances in smartphone and tablet technologies and their increasing use in clinical trials, accessibility features, such as pinch to zoom, are now at our fingertips. However, implementing such accessibility features when collecting electronic clinical outcomes assessments (eCOA) does not come without risks and must be designed with careful consideration and scientifically tested to ensure no impact to data integrity. Therefore, the objectives of this study were to determine the measurement equivalence of an eCOA questionnaire with and without a zoom accessibility feature and test its usability.

Methods

An eCOA app with a zoom accessibility feature was designed following industry standards for eCOA best design. Participants (n = 53) with chronic or recent pain completed a questionnaire with standard response scales (verbal rating scale, numerical rating scale, and visual analog scale), with and without the zoom accessibility feature enabled, in a randomized crossover design. Intraclass correlation coefficients were determined. A subset of participants (n = 10) with vision impairment participated in a usability testing interview.

Results

The intraclass correlation coefficients analysis showed high agreement (0.894-0.982) between zoomed and non-zoomed completions of the verbal rating scale, numerical rating scale, and visual analog scale. Participant usability testing showed good ease of use, ability to read the screen, and usefulness of the zoom feature, especially when not wearing corrective measures for vision impairment.

Conclusions

These findings support the use of a specially designed eCOA zoom accessibility feature for use in clinical trials.

目的：临床试验中的多样性和包容性仍然是一个重要课题，尤其是对于视力障碍等残疾参与者而言。随着智能手机和平板电脑技术的进步，以及它们在临床试验中越来越多的应用，"捏合缩放 "等无障碍功能现在已变得触手可及。然而，在收集电子临床结果评估（eCOA）时使用此类无障碍功能并非没有风险，必须经过仔细考虑和科学测试，以确保不会影响数据的完整性。因此，本研究的目的是确定带有和不带有缩放无障碍功能的电子临床结果评估问卷的测量等效性，并测试其可用性：方法：按照电子COA最佳设计的行业标准，设计了一款具有缩放无障碍功能的电子COA应用程序。在随机交叉设计中，患有慢性疼痛或近期疼痛的参与者（n=53）在启用和未启用缩放无障碍功能的情况下完成了一份带有标准反应量表（口头评分量表 [VRS]、数字评分量表 [NRS]、视觉模拟量表 [VAS]）的问卷调查。测定了类内相关系数（ICC）。一部分有视力障碍的参与者（10 人）参加了可用性测试访谈：ICC分析表明，在完成VRS、NRS和VAS时，缩放与非缩放之间的一致性很高（0.894-0.982）。参与者的可用性测试表明，缩放功能具有良好的易用性、读屏能力和实用性，尤其是在未佩戴视力矫正器的情况下：这些研究结果支持在临床试验中使用专门设计的 eCOA 缩放辅助功能。

{"title":"Measurement Equivalence of Standard and Zoom-Enabled Electronic Clinical Outcome Assessments: Implications for Patient Accessibility in Clinical Trials","authors":"Bryan McDowell MBA , Kelly M. Dumais PhD , Sarah T. Gary PhD , Helen A. Doll PhD , Gauri Nagrani , Tomás Ward PhD , Willie Muehlhausen DVM","doi":"10.1016/j.jval.2024.10.3801","DOIUrl":"10.1016/j.jval.2024.10.3801","url":null,"abstract":"<div><h3>Objectives</h3><div>Diversity and inclusion in clinical trials remains an important topic, particularly for participants with disabilities such as vision impairment. With advances in smartphone and tablet technologies and their increasing use in clinical trials, accessibility features, such as pinch to zoom, are now at our fingertips. However, implementing such accessibility features when collecting electronic clinical outcomes assessments (eCOA) does not come without risks and must be designed with careful consideration and scientifically tested to ensure no impact to data integrity. Therefore, the objectives of this study were to determine the measurement equivalence of an eCOA questionnaire with and without a zoom accessibility feature and test its usability.</div></div><div><h3>Methods</h3><div>An eCOA app with a zoom accessibility feature was designed following industry standards for eCOA best design. Participants (<em>n</em> = 53) with chronic or recent pain completed a questionnaire with standard response scales (verbal rating scale, numerical rating scale, and visual analog scale), with and without the zoom accessibility feature enabled, in a randomized crossover design. Intraclass correlation coefficients were determined. A subset of participants (<em>n</em> = 10) with vision impairment participated in a usability testing interview.</div></div><div><h3>Results</h3><div>The intraclass correlation coefficients analysis showed high agreement (0.894-0.982) between zoomed and non-zoomed completions of the verbal rating scale, numerical rating scale, and visual analog scale. Participant usability testing showed good ease of use, ability to read the screen, and usefulness of the zoom feature, especially when not wearing corrective measures for vision impairment.</div></div><div><h3>Conclusions</h3><div>These findings support the use of a specially designed eCOA zoom accessibility feature for use in clinical trials.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 269-274"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142547773","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Modeling the Potential Health, Health Economic, and Health Inequality Impact of a Large-Scale Rollout of the Drink Less App in England 模拟在英国大规模推出“少喝酒”应用程序对健康、健康经济和健康不平等的潜在影响。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-02-01 DOI: 10.1016/j.jval.2024.11.007

Colin Angus MSc , Melissa Oldham PhD , Robyn Burton PhD , Larisa-Maria Dina PhD , Matt Field PhD , Mattew Hickman PhD , Eileen Kaner PhD , Gemma Loebenberg MSc , Marcus Munafò PhD , Elena Pizzo PhD , Jamie Brown PhD , Claire Garnett PhD

Objectives

Alcohol places a significant burden on the National Health Service (NHS); yet, uptake of cost-effective approaches remains low. Digital interventions may overcome some barriers to delivery. The Drink Less app has evidence of being effective at supporting heavier drinkers to reduce their alcohol intake. In this study, we estimate the longer-term health impacts, cost-effectiveness, and health inequality impact of a large-scale rollout of the Drink Less app.

Methods

We used the Sheffield Alcohol Policy Model to estimate changes in alcohol consumption, hospital admissions, mortality, and NHS costs of 2 rollout scenarios over a 20-year time horizon: (1) a mass media awareness campaign and (2) a targeted drive to embed referral to Drink Less within primary care. We modeled the cost-effectiveness and inequality impact of each approach in a distributional cost-effectiveness analysis.

Results

A mass media campaign is estimated to reduce per capita alcohol consumption by 0.07 units/week and avert 108 556 hospital admissions and 2606 deaths over 20 years, gaining 24 787 quality-adjusted life-years at a net saving to the NHS of £417 million. Embedding in primary care is estimated to reduce consumption by 0.13 units/week, saving 188 452 admissions and 4599 deaths and gaining 38 897 quality-adjusted life-years at a net saving of £590 million. Both scenarios are estimated to reduce health inequalities, with a larger reduction for the primary care approach.

Conclusions

A large-scale rollout of the Drink Less app is estimated to be health improving, cost saving, and reducing health inequalities. Embedding the use of Drink Less within primary care is likely to be the more effective approach.

目标：酒精对国民保健服务造成了重大负担，但采用具有成本效益的方法仍然很低。数字干预措施可能会克服一些交付障碍。有证据表明，“少喝酒”应用程序可以有效地帮助重度饮酒者减少酒精摄入量。在本研究中，我们估计了大规模推出“少喝酒”应用程序的长期健康影响、成本效益和健康不平等影响。方法：我们使用谢菲尔德酒精政策模型来估计20年时间内两种推出方案的酒精消费、住院率、死亡率和NHS成本的变化：i)大众媒体宣传运动和ii)有针对性地推动将转介到“少喝酒”纳入初级保健。我们在分配成本效益分析中模拟了每种方法的成本效益和不平等影响。结果：据估计，一项大众媒体运动可以使人均酒精消费量减少0.07单位/周，并在20年内避免108,556例住院病例和2,606例死亡，获得24,787个QALYs，为NHS净节省4.17亿英镑。据估计，纳入初级保健每周可减少0.13个单位的消耗，节省188,452人次入院和4,599人死亡，获得38,897个质量年，净节省5.9亿英镑。据估计，这两种情况都能减少卫生不平等现象，初级保健方法的减少幅度更大。结论：估计大规模推出“少喝”应用程序可以改善健康，节省成本，并减少健康不平等。将减少饮酒纳入初级保健可能是更有效的方法。

{"title":"Modeling the Potential Health, Health Economic, and Health Inequality Impact of a Large-Scale Rollout of the Drink Less App in England","authors":"Colin Angus MSc , Melissa Oldham PhD , Robyn Burton PhD , Larisa-Maria Dina PhD , Matt Field PhD , Mattew Hickman PhD , Eileen Kaner PhD , Gemma Loebenberg MSc , Marcus Munafò PhD , Elena Pizzo PhD , Jamie Brown PhD , Claire Garnett PhD","doi":"10.1016/j.jval.2024.11.007","DOIUrl":"10.1016/j.jval.2024.11.007","url":null,"abstract":"<div><h3>Objectives</h3><div>Alcohol places a significant burden on the National Health Service (NHS); yet, uptake of cost-effective approaches remains low. Digital interventions may overcome some barriers to delivery. The Drink Less app has evidence of being effective at supporting heavier drinkers to reduce their alcohol intake. In this study, we estimate the longer-term health impacts, cost-effectiveness, and health inequality impact of a large-scale rollout of the Drink Less app.</div></div><div><h3>Methods</h3><div>We used the Sheffield Alcohol Policy Model to estimate changes in alcohol consumption, hospital admissions, mortality, and NHS costs of 2 rollout scenarios over a 20-year time horizon: (1) a mass media awareness campaign and (2) a targeted drive to embed referral to Drink Less within primary care. We modeled the cost-effectiveness and inequality impact of each approach in a distributional cost-effectiveness analysis.</div></div><div><h3>Results</h3><div>A mass media campaign is estimated to reduce per capita alcohol consumption by 0.07 units/week and avert 108 556 hospital admissions and 2606 deaths over 20 years, gaining 24 787 quality-adjusted life-years at a net saving to the NHS of £417 million. Embedding in primary care is estimated to reduce consumption by 0.13 units/week, saving 188 452 admissions and 4599 deaths and gaining 38 897 quality-adjusted life-years at a net saving of £590 million. Both scenarios are estimated to reduce health inequalities, with a larger reduction for the primary care approach.</div></div><div><h3>Conclusions</h3><div>A large-scale rollout of the Drink Less app is estimated to be health improving, cost saving, and reducing health inequalities. Embedding the use of Drink Less within primary care is likely to be the more effective approach.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 215-223"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Using outcome information during consultation yields better shared decision-making, better patient experiences, and more positive expectations: a comparative effectiveness study.

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-31 DOI: 10.1016/j.jval.2025.01.009

Nina L Loos, Ruud W Selles, Marloes Hp Ter Stege, Grada R Danée Arends, Lisa Hoogendam, Yara E van Kooij, Joris Veltkamp, Robbert M Wouters

Objectives: Value-based healthcare has gained recognition. Part of this framework is using outcome information in daily care. This study evaluated the effects of the patients' perceived use of outcome information on shared decision-making, patient experiences with healthcare, treatment credibility, and outcome expectations.

Methods: Data collection occurred at 25 clinics for hand surgery and therapy. We created two groups based on whether patients indicated that outcome information was used (Outcome Information group) or was not used (control group) during the clinician consultation. The patients' experience with healthcare was assessed after the first consultation with digitally distributed patient-reported experience measure (PREM) and a questionnaire to measure treatment credibility and expectations. We controlled for confounders using propensity score matching on a 3:1 basis. We calculated Cliff's delta as an effect size measure (0.11-0.27 small, 0.28-0.42 medium, >0.43 large).

Results: After propensity score matching, we included 636 patients in the Outcome Information group and 212 in the control group. The Outcome Information group experienced more shared decision-making (Cliff's delta 0.33 [0.24-0.40], p<0.001) and scored better on all PREM items. Patients in the Outcome Information group had more positive expectations of the treatment outcome (Cliff's delta: 0.21 [0.12-0.29], p<0.001) and found their treatment more credible (Cliff's delta: 0.26 [0.18-0.34], p<0.001) compared to patients in the control group.

Conclusions: The perceived use of outcome information by patients leads to more shared decision-making, better experiences with healthcare, and more positive outcome expectations and treatment credibility. We therefore recommend using outcome information in daily care to fulfill the promise of value-based healthcare.

{"title":"Using outcome information during consultation yields better shared decision-making, better patient experiences, and more positive expectations: a comparative effectiveness study.","authors":"Nina L Loos, Ruud W Selles, Marloes Hp Ter Stege, Grada R Danée Arends, Lisa Hoogendam, Yara E van Kooij, Joris Veltkamp, Robbert M Wouters","doi":"10.1016/j.jval.2025.01.009","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.009","url":null,"abstract":"<p><strong>Objectives: </strong>Value-based healthcare has gained recognition. Part of this framework is using outcome information in daily care. This study evaluated the effects of the patients' perceived use of outcome information on shared decision-making, patient experiences with healthcare, treatment credibility, and outcome expectations.</p><p><strong>Methods: </strong>Data collection occurred at 25 clinics for hand surgery and therapy. We created two groups based on whether patients indicated that outcome information was used (Outcome Information group) or was not used (control group) during the clinician consultation. The patients' experience with healthcare was assessed after the first consultation with digitally distributed patient-reported experience measure (PREM) and a questionnaire to measure treatment credibility and expectations. We controlled for confounders using propensity score matching on a 3:1 basis. We calculated Cliff's delta as an effect size measure (0.11-0.27 small, 0.28-0.42 medium, >0.43 large).</p><p><strong>Results: </strong>After propensity score matching, we included 636 patients in the Outcome Information group and 212 in the control group. The Outcome Information group experienced more shared decision-making (Cliff's delta 0.33 [0.24-0.40], p<0.001) and scored better on all PREM items. Patients in the Outcome Information group had more positive expectations of the treatment outcome (Cliff's delta: 0.21 [0.12-0.29], p<0.001) and found their treatment more credible (Cliff's delta: 0.26 [0.18-0.34], p<0.001) compared to patients in the control group.</p><p><strong>Conclusions: </strong>The perceived use of outcome information by patients leads to more shared decision-making, better experiences with healthcare, and more positive outcome expectations and treatment credibility. We therefore recommend using outcome information in daily care to fulfill the promise of value-based healthcare.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143081184","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

Development and testing the psychometric properties of 20 bolt-on items for the EQ-5D-5L across 31 rare diseases.

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.006

Richard Huan Xu, Fanni Rencz, Ruiqi Sun, Dong Dong, Shuyang Zhang

Objective: Our objective was to develop and assess the psychometric properties of relevant bolt-on items for the EQ-5D-5L in patients with rare diseases (RDs).

Methods: Nineteen new EQ-5D-5L bolt-ons were developed based on literature review, expert input and qualitative interviews and focus groups with patients, caregivers and representatives of patient associations. A nationwide, cross-sectional, web-based survey in China included patients or caregivers of patients with 31 RDs in China (n=9,190). In each RDs, participants completed the EQ-5D-5L and three out of 20 [one existing and 19 newly-developed] bolt-ons. Ceiling, explanatory power, convergent, divergent and known-group validity were examined.

Results: Among the bolt-ons, itching had the lowest ceiling (6.5%), while social relationships had the highest (42.2%). The absolute reduction in the ceiling of the EQ-5D-5L with the addition of any bolt-ons was limited, ranging from 0 (respiratory problems) to 8.3%-points (isolation). Dignity and vitality resulted in the largest increase in explained variance in EQ VAS. The isolation, fertility and visual acuity bolt-ons showed good divergent validity from the EQ-5D-5L items. There was strong convergent validity between SF-12 and conceptually-related bolt-ons (e.g., physical health composite and muscle problems bolt-on). Various bolt-ons improved the known-groups validity in specific patient groups, e.g., Huntington's disease (oral expressions), scleroderma (dexterity), myasthenia gravis (muscle problems), neuromyelitis optica and multiple sclerosis (fatigue), Marfan syndrome (self-image) and Pompe disease (safety).

Conclusion: The EQ-5D-5L shows sufficient validity in most RDs, but incorporating relevant specific bolt-ons could enhance its ability to more comprehensively assess health-related quality of life in these patients.

{"title":"Development and testing the psychometric properties of 20 bolt-on items for the EQ-5D-5L across 31 rare diseases.","authors":"Richard Huan Xu, Fanni Rencz, Ruiqi Sun, Dong Dong, Shuyang Zhang","doi":"10.1016/j.jval.2025.01.006","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.006","url":null,"abstract":"<p><strong>Objective: </strong>Our objective was to develop and assess the psychometric properties of relevant bolt-on items for the EQ-5D-5L in patients with rare diseases (RDs).</p><p><strong>Methods: </strong>Nineteen new EQ-5D-5L bolt-ons were developed based on literature review, expert input and qualitative interviews and focus groups with patients, caregivers and representatives of patient associations. A nationwide, cross-sectional, web-based survey in China included patients or caregivers of patients with 31 RDs in China (n=9,190). In each RDs, participants completed the EQ-5D-5L and three out of 20 [one existing and 19 newly-developed] bolt-ons. Ceiling, explanatory power, convergent, divergent and known-group validity were examined.</p><p><strong>Results: </strong>Among the bolt-ons, itching had the lowest ceiling (6.5%), while social relationships had the highest (42.2%). The absolute reduction in the ceiling of the EQ-5D-5L with the addition of any bolt-ons was limited, ranging from 0 (respiratory problems) to 8.3%-points (isolation). Dignity and vitality resulted in the largest increase in explained variance in EQ VAS. The isolation, fertility and visual acuity bolt-ons showed good divergent validity from the EQ-5D-5L items. There was strong convergent validity between SF-12 and conceptually-related bolt-ons (e.g., physical health composite and muscle problems bolt-on). Various bolt-ons improved the known-groups validity in specific patient groups, e.g., Huntington's disease (oral expressions), scleroderma (dexterity), myasthenia gravis (muscle problems), neuromyelitis optica and multiple sclerosis (fatigue), Marfan syndrome (self-image) and Pompe disease (safety).</p><p><strong>Conclusion: </strong>The EQ-5D-5L shows sufficient validity in most RDs, but incorporating relevant specific bolt-ons could enhance its ability to more comprehensively assess health-related quality of life in these patients.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143067692","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

A Comparison of the Performance of Six Surrogacy Models, Including Weighted Linear Regression, Meta-regression, and Bivariate Meta-Analysis.

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.005

Adrian D Vickers

Objectives: Several trial-level surrogate methods have been proposed in the literature. However, often only one method is presented in practice. By plotting trial-level associations between surrogate and final outcomes with prediction intervals and by presenting results from cross-validation procedures, this research demonstrates the value of comparing a range of model predictions.

Methods: Two oncology data sets were used as examples. One contained 34 trials and had an overall moderate surrogate association; the other contained 14 trials and had an overall strong association. The models fitted included weighted linear regression, meta-regression, and Bayesian bivariate random-effects meta-analysis (BRMA).

Results: Predictions from the models showed a high degree of variation when there was a moderate association (surrogate threshold effect [STE] of 0.413-0.906) and less variation when there was a strong association (STE of 0.696-0.887). For both data sets, BRMA provided the most robust results, although informative priors for the heterogeneity distribution were needed for the smaller data set. Weighted linear regression models provided reasonable predictions in cases of moderate association. However, in the case of strong association, Bayesian BRMA demonstrated greater uncertainty in predictions.

Conclusion: Weighted linear regression provides a useful reference because prediction intervals represent 95% of variance in the data. However, the weights used in such a model must include information on follow-up time. In cases with small data sets, as well as in cases where there appeared to be a strong association, Bayesian BRMA provided predictions that were more robust than those provided by weighted linear regression.

目的：文献中提出了几种试验水平代用方法。然而，在实践中往往只提出一种方法。本研究通过绘制代用结果与最终结果之间的试验级关联预测区间图，并展示交叉验证程序的结果，证明了比较一系列模型预测的价值：方法：以两个肿瘤数据集为例。方法：以两个肿瘤数据集为例，其中一个包含 34 项试验，总体上具有中等程度的代用关联性；另一个包含 14 项试验，总体上具有较强的关联性。拟合的模型包括加权线性回归、元回归和贝叶斯双变量随机效应元分析（BRMA）：结果：当存在中度关联时，模型的预测结果显示差异较大（代阈效应 [STE] 为 0.413-0.906），而当存在强关联时，预测结果的差异较小（STE 为 0.696-0.887）。对于这两个数据集，BRMA 提供了最稳健的结果，尽管对于较小的数据集来说，异质性分布需要有信息丰富的先验。加权线性回归模型为中度关联提供了合理的预测。然而，在强关联的情况下，贝叶斯 BRMA 预测的不确定性更大：加权线性回归提供了有用的参考，因为预测区间代表了数据中 95% 的方差。然而，这种模型中使用的权重必须包括随访时间的信息。与加权线性回归相比，贝叶斯 BRMA 在数据集较小的情况下，以及在似乎存在较强关联的情况下，所提供的预测结果更为稳健。

{"title":"A Comparison of the Performance of Six Surrogacy Models, Including Weighted Linear Regression, Meta-regression, and Bivariate Meta-Analysis.","authors":"Adrian D Vickers","doi":"10.1016/j.jval.2025.01.005","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.005","url":null,"abstract":"<p><strong>Objectives: </strong>Several trial-level surrogate methods have been proposed in the literature. However, often only one method is presented in practice. By plotting trial-level associations between surrogate and final outcomes with prediction intervals and by presenting results from cross-validation procedures, this research demonstrates the value of comparing a range of model predictions.</p><p><strong>Methods: </strong>Two oncology data sets were used as examples. One contained 34 trials and had an overall moderate surrogate association; the other contained 14 trials and had an overall strong association. The models fitted included weighted linear regression, meta-regression, and Bayesian bivariate random-effects meta-analysis (BRMA).</p><p><strong>Results: </strong>Predictions from the models showed a high degree of variation when there was a moderate association (surrogate threshold effect [STE] of 0.413-0.906) and less variation when there was a strong association (STE of 0.696-0.887). For both data sets, BRMA provided the most robust results, although informative priors for the heterogeneity distribution were needed for the smaller data set. Weighted linear regression models provided reasonable predictions in cases of moderate association. However, in the case of strong association, Bayesian BRMA demonstrated greater uncertainty in predictions.</p><p><strong>Conclusion: </strong>Weighted linear regression provides a useful reference because prediction intervals represent 95% of variance in the data. However, the weights used in such a model must include information on follow-up time. In cases with small data sets, as well as in cases where there appeared to be a strong association, Bayesian BRMA provided predictions that were more robust than those provided by weighted linear regression.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068294","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

Screening for Type 2 Diabetes Mellitus: A Systematic Review of Recent Economic Evaluations.

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2025-01-27 DOI: 10.1016/j.jval.2025.01.001

Zixuan Jin, Joshua Rothwell, Ka Keat Lim

Objectives: To examine recent economic evaluations, whether any type 2 diabetes mellitus (T2DM) screening designs may represent better value for money, and to rate their methodological qualities.

Methods: We systematically searched three concepts (economic evaluations (EEs), T2DM, screening) in three databases (Medline, Embase, and EconLit) for EEs published between 2010 and 2023. Two independent reviewers screened for and rated their methodological quality (using CHEC-Extended).

Results: Of 32 EEs, a majority were from high-income countries (69%). Most used single biomarkers (54%) to screen adults ≥30-<60 years old (60%) but did not report locations (69%), treatments for those diagnosed (66%), diagnostic methods (57%) and screening intervals (54%). Compared to no screening, T2DM screening using single biomarkers was found not cost-effective (23/54 comparisons), inconclusive (16/54), dominant (11/54), or cost-effective (4/54). Compared to no screening, screening with a risk score and single biomarkers was found cost-effective (21/40) or dominant (19/40). Risk score alone was mostly dominant (6/10). Compared to universal screening, targeted screening among obese, overweight, or older people may be cost-effective or dominant. Compared to fasting plasma glucose (FPG) or fasting capillary glucose, screening using risk scores was found mostly dominant or cost-effective. Expanding screening location or lowering HbA1c or FPG thresholds was found dominant or cost-effective. Each EE had 4-17 items (median 14/20) on CHEC-Extended rated "Yes/Rather Yes".

Conclusion: EE findings varied based on screening tools, intervals, locations, minimum screening age, diagnostic methods and treatment. Future EEs should more comprehensively report screening designs and evaluate T2DM screening in low-income countries.

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