Value in Health最新文献_第3页

Informing the United States Medicare Drug Price Negotiation for Apixaban and Rivaroxaban: Methodological Considerations for Value Assessments Many Years After Launch 为阿哌沙班和利伐沙班的美国医疗保险药品价格谈判提供信息：上市多年后价值评估的方法考虑。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.011

Marina Richardson PhD, MSc , Abigail C. Wright PhD, MSc , Jeffrey A. Tice MD , David M. Rind MD, MSc , Matt Seidner BS , Sarah Emond MPP , Steven D. Pearson MD, MSc

Objectives

To demonstrate how health technology assessment methods can be used to support Medicare’s price negotiations for apixaban and rivaroxaban.

Methods

Following the statutory outline of evidence that will be considered by Medicare, we conducted a systematic literature review, network meta-analyses, and decision analyses to evaluate the health outcomes and costs associated with apixaban and rivaroxaban compared with warfarin and dabigatran for patients with nonvalvular atrial fibrillation. Our methods inform discussions about the therapeutic impact of apixaban and rivaroxaban and suggest price premiums above their therapeutic alternatives over a range of cost-effectiveness thresholds.

Results

Network meta-analyses found apixaban resulted in a lower risk of major bleeding compared with warfarin and dabigatran and a lower risk of stroke/systemic embolism compared with warfarin but not compared with dabigatran. Rivaroxaban resulted in a lower risk of stroke/systemic embolism versus warfarin but not dabigatran, and there was no difference in major bleeding. Decision-analytic modeling of apixaban suggested annual price premiums up to $4350 above the price of warfarin and up to $530 above the price for dabigatran at cost-effectiveness thresholds up to $200 000 per equal value of life-years gained. Analyses of rivaroxaban showed an annual price premium of up to $3920 above warfarin and no premium above that paid for dabigatran.

Conclusions

Although health technology assessment is typically performed near the time of regulatory approval, with modifications, we produced comparative clinical and relative cost-effectiveness findings to help guide negotiations on a “fair” price for drugs on the market for over a decade.

目标：展示如何利用卫生技术评估方法支持医疗保险对阿哌沙班和利伐沙班的价格谈判：展示如何利用卫生技术评估 (HTA) 方法支持医疗保险对阿哌沙班和利伐沙班的价格谈判：根据医疗保险将考虑的法定证据大纲，我们进行了系统性文献综述、网络荟萃分析 (NMA) 和决策分析，以评估阿哌沙班和利伐沙班与华法林和达比加群相比，对非瓣膜性心房颤动患者的健康结果和相关成本。我们的方法为有关阿哌沙班和利伐沙班治疗效果的讨论提供了信息，并提出了在一系列成本效益阈值下高于其治疗替代品的价格溢价：NMA发现，与华法林和达比加群相比，阿哌沙班导致大出血的风险较低，与华法林相比，阿哌沙班导致中风/系统性栓塞的风险较低，但与达比加群相比，阿哌沙班导致中风/系统性栓塞的风险较低。与华法林相比，利伐沙班导致中风/全身性栓塞的风险较低，但与达比加群相比较，利伐沙班导致中风/全身性栓塞的风险较低，但与达比加群相比较，利伐沙班导致大出血的风险没有差异。阿哌沙班的决策分析模型表明，在成本效益阈值为每等值生命年收益 20 万美元时，阿哌沙班的年价格比华法林高出 4350 美元，比达比加群高出 530 美元。对利伐沙班的分析表明，每年的价格比华法林高出 3,920 美元，而没有比达比加群高：尽管 HTA 通常是在监管部门批准时进行，但经过修改后，我们得出了临床和相对成本效益的比较结果，有助于指导市场上已上市十多年的药物的 "公平 "价格谈判。

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引用次数: 0

Assessing the Health and Welfare Benefits of Interventions Using the Wider Societal Impacts Framework 利用更广泛的社会影响框架评估干预措施的健康和福利效益。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.014

Shainur Premji PhD , Susan Griffin PhD

Objectives

Health technology assessment bodies advocate capturing the value of interventions in terms of their benefits to health and broader welfare. The wider societal impacts (WSI) framework considers how changes in health alter a person’s net contribution to society—that is, what they produce minus what they consume. In this research, we review this framework and explore the scope to differentiate WSI by equity-relevant sociodemographic characteristics.

Methods

This research updates previous calculations using publicly available data from population-based surveys in the United Kingdom. We then estimate for 199 chronic conditions: (1) WSI for the average person with the condition and (2) gain in WSI for an improvement of 0.1 in health-related quality of life score.

Results

The nature and availability of information varied across population-based surveys and precluded analyses to examine WSI by population subgroup. Our updated estimates mirrored earlier findings that consideration of the broader societal impacts of health would reprioritize interventions compared with assessment on health alone. For example, for the same improvement in health, a woman experiencing diseases of the circulatory system has the highest potential gain in WSI (£354/month) whereas a man experiencing HIV has the lowest potential gain (£233/month).

Conclusions

The WSI framework provides a simple, indirect method to inform resource allocation decisions. Understanding the equity implications of this approach was hindered by differences in the information collected across population-based surveys. Findings demonstrate the potential reprioritization that may occur if the broader welfare benefits of health interventions were used to inform coverage decisions.

目标：卫生技术评估机构主张根据干预措施对健康和更广泛福利的益处来衡量其价值。更广泛的社会影响（WSI）框架考虑的是健康的变化如何改变一个人对社会的净贡献--即他们的生产量减去他们的消费量。在本研究中，我们回顾了这一框架，并探讨了根据与公平相关的社会人口特征来区分 WSI 的范围：本研究利用英国人口调查的公开数据更新了之前的计算结果。然后，我们对 199 种慢性疾病进行了估算：(i) 普通患者的 WSI；(ii) 健康相关生活质量（HRQoL）每提高 0.1 分所获得的 WSI：不同人群调查的信息性质和可用性各不相同，因此无法按人群分组对 WSI 进行分析。我们的最新估计结果与之前的研究结果一致，即与仅评估健康状况相比，考虑健康对社会的广泛影响将重新确定干预措施的优先次序。例如，在健康状况得到相同改善的情况下，患有循环系统疾病的女性的 WSI 潜在收益最高（354 英镑/月），而患有艾滋病的男性的潜在收益最低（233 英镑/月）：WSI 框架为资源分配决策提供了一种简单、间接的方法。由于基于人口的调查所收集的信息存在差异，因此无法了解这种方法对公平的影响。研究结果表明，如果利用健康干预措施的更广泛福利效益来为覆盖决策提供信息，就有可能重新确定优先次序。

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引用次数: 0

Choice Consistency in Discrete Choice Experiments: Does Numeracy Skill Matter? 离散选择实验中的选择一致性：计算能力重要吗？(VIH-2023-0494.R2）。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.001

Mesfin G. Genie PhD , Nabin Poudel PhD , Francesco Paolucci PhD , Surachat Ngorsuraches PhD

Objectives

This study investigated the relationship between numeracy skills (NS) and choice consistency in discrete choice experiments (DCEs).

Methods

A DCE was conducted to explore patients’ preferences for kidney transplantation in Italy. Patients completed the DCE and answered 3-item numeracy questions. A heteroskedastic multinomial logit model was used to investigate the effect of numeracy on choice consistency.

Results

Higher NS were associated with greater choice consistency, increasing the scale to 1.63 (P < .001), 1.39 (P < .001), and 1.18 (P < .001) for patients answering 3 of 3, 2 of 3, and 1 of 3 questions correctly, respectively, compared with those with no correct answers. This corresponded to 63%, 39%, and 18% more consistent choices, respectively. Accounting for choice consistency resulted in varying willingness-to-wait (WTW) estimates for kidney transplant attributes. Patients with the lowest numeracy (0/3) were willing to wait approximately 42 months [95% CI: 29.37, 54.68] for standard infectious risk, compared with 33 months [95% CI: 28.48, 38.09] for 1 of 3, 28 months [95% CI: 25.13, 30.32] for 2 of 3, and 24 months [95% CI: 20.51, 27.25] for 3 of 3 correct answers. However, WTW differences for an additional year of graft survival and neoplastic risk were not statistically significant across numeracy levels. Supplementary analyses of 2 additional DCEs on COVID-19 vaccinations and rheumatoid arthritis, conducted online, supported these findings: higher NS were associated with more consistent choices across different disease contexts and survey formats.

Conclusions

The findings suggested that combining patients with varying NS could bias WTW estimates, highlighting the need to consider numeracy in DCE data analysis and interpretation.

研究目的本研究调查了离散选择实验（DCE）中计算能力与选择一致性之间的关系：在意大利进行了一项离散选择实验，以探讨患者对肾移植的偏好。患者在完成离散选择实验的同时回答了三个算术问题。采用异方差多项式Logit（HMNL）模型研究计算能力对选择一致性的影响：结果：计算能力越高，选择一致性越大，量表增加到 1.63（p结论：研究结果表明，将不同计算能力的患者组合在一起，可以提高选择一致性：研究结果表明，将具有不同计算能力的患者合并在一起可能会使WTW估计值出现偏差，这突出说明了在DCE数据分析和解释中考虑计算能力的必要性。

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引用次数: 0

Measuring Healthcare Experiences Among People With Intellectual Disability: A Rapid Evidence Synthesis of Tools and Methods 衡量智障人士的医疗保健经历：工具和方法的快速证据综述。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.05.018

Objectives

Patient-reported experience measures (PREMs) collect essential data for service and system-wide quality improvement and performance monitoring toward value-based care. However, the experiences of people with intellectual disability, who have high healthcare utilization couple with poorer outcomes, are often omitted from system-wide PREMs and service-wide PREMs data. The use of PREMs instruments for data collection among people with intellectual disability has not been explored. This review aimed to identify and synthesize measurement tools and approaches that have been used to gather patient-reported experience data from people with intellectual disability.

Methods

Rapid evidence assessment was used, in which comprehensive search strategies were applied to electronic databases and gray literature. Narrative synthesis was used with the included articles to address the review aim.

Results

A total of 48 documents were included; 26 peer-reviewed journal articles and 22 articles from gray literature. Patient-reported experiences have been gathered from people with intellectual disabilities in relation to specific services or encounters, predominantly using qualitative methods. To date, there is an absence of targeted service- or system-wide surveys. Existing clinic- and condition-specific instruments provide insight for broader application.

Conclusions

Patient experience assessment among people with intellectual disability requires consideration of (1) how individuals are identified and approached, (2) the content, design and structure of measurement instruments, and (3) the process by which data are collected, and (4) how it may be applied to create change. Despite the collection of patient experience data from people with intellectual disability, there is little research available about how this information is later used to support health service improvement. Applying PREMs for quality improvement is critical to realize the improvements to healthcare provision required for people with intellectual disability toward equitable care quality.

目标：患者报告体验测量（PREMs）为服务和整个系统的质量改进和绩效监控收集重要数据，以实现以价值为基础的医疗保健。然而，全系统范围的 PREMs 和全服务范围的 PREMs 数据往往忽略了智障人士的体验，而智障人士的医疗保健使用率较高，治疗效果较差。在智障人士中使用 PREMs 工具收集数据的问题尚未得到探讨。本综述旨在确定和综合用于收集智障人士患者报告体验数据的测量工具和方法：方法：采用快速证据评估（REA），对电子数据库和灰色文献采用综合搜索策略。对纳入的文章进行叙述性综合，以实现综述目的：结果：共纳入 48 篇文献，其中 26 篇为同行评审期刊论文，22 篇为灰色文献。主要采用定性方法从智障人士那里收集了与特定服务或遭遇有关的患者报告经验。迄今为止，还没有针对性的服务或全系统调查。现有的针对特定诊所和病症的工具可为更广泛的应用提供启示：智障人士的患者体验评估需要考虑 a) 如何识别和接触患者；b) 测量工具的内容、设计和结构；c) 数据收集过程；d) 如何应用这些数据来创造改变。尽管从智障人士那里收集了患者体验数据，但关于这些信息日后如何用于支持医疗服务改进的研究却很少。将 PREMs 应用于质量改进，对于实现改善智障人士医疗保健服务、提高公平护理质量至关重要。

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引用次数: 0

How Many Monte Carlo Samples Are Needed for Probabilistic Cost-Effectiveness Analyses? 概率成本效益分析需要多少蒙特卡罗样本？

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.06.016

Objectives

Probabilistic sensitivity analysis (PSA) is conducted to account for the uncertainty in cost and effect of decision options under consideration. PSA involves obtaining a large sample of input parameter values (

N

) to estimate the expected cost and effect of each alternative in the presence of parameter uncertainty. When the analysis involves using stochastic models (eg, individual-level models), the model is further replicated

P

times for each sampled parameter set. We study how

N

and

P

should be determined.

Methods

We show that PSA could be structured such that

P

can be an arbitrary number (say,

P = 1

). To determine

N

, we derive a formula based on Chebyshev’s inequality such that the error in estimating the incremental cost-effectiveness ratio (ICER) of alternatives (or equivalently, the willingness-to-pay value at which the optimal decision option changes) is within a desired level of accuracy. We described 2 methods to confirm, visually and quantitatively, that the

N

informed by this method results in ICER estimates within the specified level of accuracy.

Results

When

N

is arbitrarily selected, the estimated ICERs could be substantially different from the true ICER (even as

P

increases), which could lead to misleading conclusions. Using a simple resource allocation model, we demonstrate that the proposed approach can minimize the potential for this error.

Conclusions

The number of parameter samples in probabilistic cost-effectiveness analyses should not be arbitrarily selected. We describe 3 methods to ensure that enough parameter samples are used in probabilistic cost-effectiveness analyses.

目的：进行概率敏感性分析（PSA）是为了考虑所考虑的决策方案在成本和效果方面的不确定性。概率敏感性分析包括获取大量输入参数值样本（N），以估算在参数不确定的情况下每个备选方案的预期成本和效果。如果分析涉及使用随机模型（如个人层面的模型），则要对每个抽样参数集进行 P 次模型复制。我们将研究如何确定 N 和 P：我们表明，PSA 的结构可以使 P 成为一个任意数（例如，P=1）。为了确定 N，我们根据切比雪夫不等式推导出一个公式，使替代方案的增量成本效益比（ICER）（或等同于最优决策方案发生变化时的支付意愿值）的估算误差在所需的准确度范围内。我们介绍了两种方法，以直观和定量的方式确认这种方法所告知的 N 会导致 ICER 估计值在指定的准确度范围内：当 N 被任意选择时，估算的 ICER 可能与真实的 ICER 相差甚远（甚至随着 P 的增加），这可能导致误导性结论。通过使用一个简单的资源分配模型，我们证明了所提出的方法可以最大限度地减少这种误差的可能性：概率 CEA 中的参数样本数量不应随意选择。我们介绍了三种确保在概率 CEA 中使用足够参数样本的方法。

{"title":"How Many Monte Carlo Samples Are Needed for Probabilistic Cost-Effectiveness Analyses?","authors":"","doi":"10.1016/j.jval.2024.06.016","DOIUrl":"10.1016/j.jval.2024.06.016","url":null,"abstract":"<div><h3>Objectives</h3><div>Probabilistic sensitivity analysis (PSA) is conducted to account for the uncertainty in cost and effect of decision options under consideration. PSA involves obtaining a large sample of input parameter values (<span><math><mrow><mi>N</mi></mrow></math></span>) to estimate the expected cost and effect of each alternative in the presence of parameter uncertainty. When the analysis involves using stochastic models (eg, individual-level models), the model is further replicated <span><math><mrow><mi>P</mi></mrow></math></span> times for each sampled parameter set. We study how <span><math><mrow><mi>N</mi></mrow></math></span> and <span><math><mrow><mi>P</mi></mrow></math></span> should be determined.</div></div><div><h3>Methods</h3><div>We show that PSA could be structured such that <span><math><mrow><mi>P</mi></mrow></math></span> can be an arbitrary number (say, <span><math><mrow><mi>P</mi><mo>=</mo><mn>1</mn></mrow></math></span>). To determine <span><math><mrow><mi>N</mi></mrow></math></span>, we derive a formula based on Chebyshev’s inequality such that the error in estimating the incremental cost-effectiveness ratio (ICER) of alternatives (or equivalently, the willingness-to-pay value at which the optimal decision option changes) is within a desired level of accuracy. We described 2 methods to confirm, visually and quantitatively, that the <span><math><mrow><mi>N</mi></mrow></math></span> informed by this method results in ICER estimates within the specified level of accuracy.</div></div><div><h3>Results</h3><div>When <span><math><mrow><mi>N</mi></mrow></math></span> is arbitrarily selected, the estimated ICERs could be substantially different from the true ICER (even as <span><math><mrow><mi>P</mi></mrow></math></span> increases), which could lead to misleading conclusions. Using a simple resource allocation model, we demonstrate that the proposed approach can minimize the potential for this error.</div></div><div><h3>Conclusions</h3><div>The number of parameter samples in probabilistic cost-effectiveness analyses should not be arbitrarily selected. We describe 3 methods to ensure that enough parameter samples are used in probabilistic cost-effectiveness analyses.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"27 11","pages":"Pages 1553-1563"},"PeriodicalIF":4.9,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559836","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

Linking Reimbursement to Patient Benefits for Advanced Therapy Medicinal Products and Other High-Cost Innovations: Policy Recommendations for Outcomes-Based Agreements in Europe 将先进治疗药物产品和其他高成本创新产品的报销与患者利益挂钩：欧洲基于结果的协议的政策建议。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-11-01 DOI: 10.1016/j.jval.2024.07.007

Tuba Saygın Avşar PhD , Jamie Elvidge MSc , Claire Hawksworth MSc , Juliet Kenny MSc , Bertalan Németh PhD , Marcelien Callenbach MSc , Johanna Ringkvist MSc , Dalia Dawoud PhD

Objectives

Health technology assessment (HTA) of advanced therapy medicinal products (ATMPs), such as high-cost and one-time cell and gene therapies, is particularly challenging. Outcomes-based agreements (OBAs) are a potential solution to mitigate the risks while providing access to patients but are not widely used across Europe. This study aimed to develop policy recommendations to support the acceptability and implementation of OBAs in Europe.

Methods

A policy sandbox approach was used to engage with stakeholders and explore how HTA organizations can support reimbursement decisions regarding OBAs for ATMPs. A panel of 38 experts from across the European region was convened in 2 workshops, representing payers, HTA organizations, patients, registries, and an industry trade body.

Results

Policy recommendations were developed to support the appropriate consideration of OBAs for reimbursing highly uncertain technologies, such as ATMPs. If a positive HTA recommendation cannot be made at the proposed price, then a simple price discount reflecting the uncertainty is preferred over complex solutions such as OBAs. If an OBA is pursued, it should be designed collaboratively with all stakeholders to understand data collection feasibility and minimize burden to patients and providers. Payers are encouraged to approach OBAs as a tool for informed decision making, including a readiness to make negative reimbursement decisions based on unfavorable evidence.

Conclusions

The study presents a policy framework for using OBAs in reimbursement decisions. OBAs must be carefully designed, focusing on appropriateness and the burden of implementation. The relevant authorities should be committed to making decisions in light of the resulting evidence.

目的：对高成本、一次性细胞和基因疗法等高级治疗药物产品（ATMP）进行卫生技术评估（HTA）尤其具有挑战性。基于结果的协议 (OBA) 是一种潜在的解决方案，既能降低风险，又能为患者提供治疗机会，但在欧洲尚未得到广泛应用。本研究旨在制定政策建议，以支持欧洲接受和实施基于结果的协议：方法：采用政策沙盒的方法与利益相关者接触，探讨 HTA 组织如何支持有关 ATMP 的 OBA 的报销决策。在两次研讨会上，来自欧洲地区的 38 位专家组成了一个专家小组，他们分别代表支付方、HTA 组织、患者、注册机构和一个行业贸易机构：制定了政策建议，以支持在对 ATMP 等高度不确定的技术进行报销时适当考虑 OBA。如果无法按照建议的价格提出积极的 HTA 建议，那么反映不确定性的简单价格折扣比 OBA 等复杂的解决方案更可取。如果要采用 OBA，则应与所有利益相关者合作设计，以了解数据收集的可行性，并尽量减轻患者和医疗服务提供者的负担。鼓励支付方将 OBAs 作为知情决策的工具，包括随时准备根据不利证据做出否定的报销决定：本研究提出了在报销决策中使用 OBA 的政策框架。必须精心设计 OBA，重点关注适宜性和执行负担。相关部门应致力于根据所获得的证据做出决策。

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引用次数: 0

An attribution of value framework for combination treatments. 综合疗法的价值归属框架。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-10-29 DOI: 10.1016/j.jval.2024.08.012

Andrew H Briggs, Alexis Doyle-Connolly, John Schneider, Tanja Podkonjak, Helen Taylor, Emma Roffe, Eric Low, Sarah Davis, Martin Kaiser, Anthony J Hatswell, Neil Rabin

Objective: The use of cost-effectiveness methods to support policy decisions has become well established but difficulties can arise when evaluating a new treatment which is indicated to be used in combination with an established "backbone treatment." If the latter has been priced close to the decision maker's willingness to pay threshold, this may mean that there is no headroom for the new treatment to demonstrate value, at any price, even if the combination is clinically effective. Without a mechanism for attributing value to component treatments within a combination therapy, the health system risks generating negative funding decisions for combinations of proven clinical benefit to patients. The aim of this work was to define a value attribution methodology which could be used to allocate value between the components of any combination treatment.

Methods: The framework is grounded in the standard decision rules of cost-effectiveness analysis and provides solutions according to key features of the problem: perfect/imperfect information about component treatment monotherapy effects and balanced/unbalanced market power between their manufacturers.

Results: The share of incremental value varies depending on whether there is perfect/imperfect information and balance/imbalance of market power, with some scenarios requiring the manufacturers to negotiate a share of the incremental value within a range defined by the framework.

Conclusions: It is possible to define a framework that is independent of price and focuses on benefits expressed as Quality-Adjusted Life-Year (QALY) gains (and/or QALY equivalents for cost-savings), a standard metric used by many HTA agencies to evaluate novel treatments.

目的：使用成本效益方法来支持政策决策的做法已经非常成熟，但在评估一种新疗法时可能会遇到困难，因为这种新疗法需要与已确立的 "骨干疗法 "联合使用。如果 "骨干疗法 "的定价已接近决策者的支付意愿阈值，这可能意味着新疗法在任何价格下都没有展示价值的余地，即使这种组合在临床上是有效的。如果没有一种机制来为联合疗法中的成分治疗赋予价值，医疗系统就有可能对已证明对患者有临床益处的联合疗法做出负面的资助决定。这项工作的目的是确定一种价值归属方法，用于在任何联合疗法的各组成部分之间分配价值：方法：该框架以成本效益分析的标准决策规则为基础，并根据问题的关键特征提供解决方案：关于组合疗法单一疗法效果的完美/不完美信息，以及其制造商之间平衡/不平衡的市场力量：增量价值的份额因信息是否完全/不完全和市场力量是否平衡/不平衡而不同，有些方案要求制造商在框架定义的范围内协商增量价值的份额：可以定义一个独立于价格的框架，该框架侧重于以质量调整生命年（QALY）收益（和/或成本节约的 QALY 当量）表示的效益，这是许多 HTA 机构用于评估新型疗法的标准指标。

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引用次数: 0

Estimating the Economic Impacts for Caregivers of Young People With Mental Health Problems in a Brazilian Cohort. 估算巴西队列中精神健康问题青少年照顾者的经济影响。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-10-28 DOI: 10.1016/j.jval.2024.10.3802

Carolina Ziebold, David McDaid, Derek King, Renee Romeo, Wagner S Ribeiro, Pedro M Pan, Euripedes C Miguel, Rodrigo A Bressan, Luis A Rohde, Giovanni A Salum, Sara Evans-Lacko

Objectives: To assess the indirect economic impacts on caregivers resulting from mental health problems in their children and to explore the association with characteristics of the young people and their caregivers.

Methods: Data from 1158 caregivers of young people aged 14 to 23 years with mental health problems in a Brazilian cohort were analyzed. We assessed productivity losses, additional household tasks, out-of-pocket expenses, and own healthcare utilization because of the young person's mental health problems over the past 6 months. The costs of productivity losses and household tasks were estimated in terms of caregivers' earnings. Logistic regression models identified factors associated with reported impacts. Generalized linear models evaluated clinical and caregiver characteristics associated with the economic impact on caregivers.

Results: Nearly 40% of caregivers (n = 458) experienced economic impacts because of mental health issues in their children over the previous 6 months. The total economic impact among these 458 caregivers who reported incurring costs amounted to half of their earnings, and this was consistent across socioeconomic groups. Factors associated with reporting impacts differed from those affecting their costs. Externalizing and comorbid diagnoses, service use, higher impairment, and female caregivers increased the likelihood of impacts, whereas the greatest economic impacts were associated with internalizing conditions and service use.

Conclusions: Though these findings need to be interpreted with caution because of inherent limitations, they underscore the substantial economic impacts borne by caregivers of young people with mental health problems, suggesting the need for targeted policy interventions to promote equitable caregiving and provide more comprehensive childcare support.

目的评估子女的精神健康问题对照顾者造成的间接经济影响，并探讨与青少年及其照顾者的特征之间的关联：方法：我们分析了巴西队列中 1,158 名 14-23 岁有精神健康问题青少年的照顾者的数据。我们对过去 6 个月中因青少年精神健康问题而导致的生产力损失、额外家务劳动、自付费用和自身医疗保健使用情况进行了评估。生产力损失和家务劳动的成本按照顾者的收入估算。逻辑回归模型确定了与所报告的影响相关的因素。广义线性模型研究了与对照顾者的经济影响相关的临床和照顾者特征：近 40% 的照顾者（n=458）在过去 6 个月中因子女的心理健康问题而受到经济影响。在这 458 名报告产生费用的照顾者中，总经济影响相当于其收入的一半，而且在不同的社会经济群体中都是如此。与报告影响相关的因素与影响费用的因素有所不同。外显性诊断和合并诊断、服务使用、较高的损伤程度以及女性照顾者增加了产生影响的可能性，而最大的经济影响则与内显性疾病和服务使用有关：虽然由于固有的局限性，这些研究结果需要谨慎解读，但它们强调了有精神健康问题的年轻人的照顾者所承担的巨大经济影响，表明有必要采取有针对性的政策干预措施，以促进公平的照顾和提供更全面的儿童保育支持。

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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 : 2024-10-28 DOI: 10.1016/j.jval.2024.10.3801

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

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 [VRS], numerical rating scale [NRS], visual analog scale [VAS]), with and without the zoom accessibility feature enabled, in a randomized crossover design. Intraclass Correlation Coefficients (ICCs) were determined. A subset of participants (n=10) with vision impairment participated in a usability testing interview.

Results: The ICC analysis showed high agreement (0.894-0.982) between zoomed and non-zoomed completions of the VRS, NRS and VAS. 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, Kelly M Dumais, Sarah T Gary, Helen A Doll, Gauri Nagrani, Tomás Ward, Willie Muehlhausen","doi":"10.1016/j.jval.2024.10.3801","DOIUrl":"https://doi.org/10.1016/j.jval.2024.10.3801","url":null,"abstract":"<p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>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 [VRS], numerical rating scale [NRS], visual analog scale [VAS]), with and without the zoom accessibility feature enabled, in a randomized crossover design. Intraclass Correlation Coefficients (ICCs) were determined. A subset of participants (n=10) with vision impairment participated in a usability testing interview.</p><p><strong>Results: </strong>The ICC analysis showed high agreement (0.894-0.982) between zoomed and non-zoomed completions of the VRS, NRS and VAS. 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.</p><p><strong>Conclusions: </strong>These findings support the use of a specially designed eCOA zoom accessibility feature for use in clinical trials.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2024-10-28","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Deriving a Preference-Weighted Measure for People with Hypoglycaemia from the Hypo-RESOLVE-QoL. 从 Hypo-RESOLVE-QoL 中得出低血糖患者的偏好加权测量值。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-10-28 DOI: 10.1016/j.jval.2024.10.3800

Donna Rowen, Emily McDool, Jill Carlton, Philip Powell, Richard Norman

Objective: Hypoglycaemia impacts the health-related quality of life (HRQoL) of people living with diabetes (PwD), and existing preference-weighted measures do not capture all important aspects. The study aimed to generate a preference-weighted measure capturing the HRQoL impact of hypoglycaemia in PwD.

Methods: Items for the health state classification system were selected from the hypoglycaemia-specific Hypo-RESOLVE QoL measure using: relevance in cognitive interviews, translatability, suitability for valuation, endorsement by patient advisors and experts, and psychometric performance in a large survey of PwD. Second, an online valuation survey using discrete choice experiment (DCE) with survival attribute was conducted with members of the UK public. DCE data was modelled using conditional logit analysis, and results scaled to produce preference weights for the classification system on a scale where 1 is equivalent to full health, 0 is equivalent to dead, and below zero is worse than dead.

Results: The health state classification system consists of eight items reflecting the factors of the Hypo-RESOLVE QoL (psychological, social and physical aspects). The valuation survey was completed by 1000 members of the UK public, representative for age and sex. Good understanding of DCE tasks was demonstrated. The item "do what I want to do in my life" had the largest preference weight, and "find it hard to stop thinking about my glucose levels" had the smallest.

Conclusions: This study generated Hypo-RESOLVE QoL-8D, a preference-weighted measure capturing the HRQoL impact of hypoglycaemia in PwD, with UK general public preference-weights. The measure can be generated from Hypo-RESOLVE QoL data.

目的：低血糖会影响糖尿病患者（PwD）的健康相关生活质量（HRQoL），而现有的偏好加权测量方法并不能捕捉到所有重要方面。本研究旨在制定一种偏好加权测量方法，以捕捉低血糖对糖尿病患者 HRQoL 的影响：健康状态分类系统的项目是从低血糖症专用的 Hypo-RESOLVE QoL 测量中筛选出来的，筛选时考虑了以下因素：认知访谈中的相关性、可翻译性、评估的适用性、患者顾问和专家的认可度，以及大型残疾人调查中的心理测量表现。其次，对英国公众进行了一项在线估价调查，采用的是带有生存属性的离散选择实验（DCE）。利用条件对数分析法对离散选择实验数据进行建模，并对结果进行缩放，以产生分类系统的偏好权重，其中 1 表示完全健康，0 表示死亡，低于 0 表示比死亡更糟：健康状况分类系统由 8 个项目组成，反映了 Hypo-RESOLVE QoL 的各因素（心理、社会和生理方面）。估值调查由 1000 名英国公众完成，他们的年龄和性别均具有代表性。他们对 DCE 任务有很好的理解。其中，"做我一生中想做的事 "的偏好权重最大，而 "发现很难停止思考我的血糖水平 "的偏好权重最小：本研究利用英国普通公众的偏好权重，生成了低血糖影响残疾人 HRQoL 的偏好权重测量指标 Hypo-RESOLVE QoL-8D。该指标可从 Hypo-RESOLVE QoL 数据中生成。

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引用次数: 0