Value in Health最新文献_第10页

Guiding Principles for Evaluating Vaccines in Joint Health Technology Assessment in the European Union: Preparing for the European Union’s Regulation on Health Technology Assessment for Vaccines 欧盟联合卫生技术评估中的疫苗评估指导原则》：为欧盟疫苗 HTA 法规做准备。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.011

Objectives

The appraisal of vaccines in the European Union (EU) currently involves many different decision-making bodies and processes. The objective of this study was to help inform the development of standardized methodology and vaccine-specific processes for use in the EU Regulation on health technology assessment (HTA).

Methods

Literature reviews and expert consultation were conducted to identify current practices and gaps related to vaccine appraisals and to develop guiding principles for the joint clinical assessment of vaccines.

Results

We found that significant variation exists across the EU member states in the decision-making processes when clinically evaluating vaccines. Three guiding principles consisting of 13 recommendations were developed to help inform the development of decision-making frameworks for the joint clinical assessment of vaccines in the EU: (1) support the creation of appropriate terminology and measurements for clinical appraisals of vaccines; (2) develop inclusive, timely, and transparent vaccine appraisal processes to support stronger evidence generation for vaccine decision making and appraisal; and (3) improve the collection and interoperability of real-world data, including robust surveillance, to foster evidence generation and support the standardization of vaccine clinical appraisals.

Conclusions

Given the significance of vaccines for public health, there is an urgency to develop standardized and vaccine-specific methodologies and processes for use in the EU joint HTA framework. The proposed guiding principles could support the effective implementation of the EU Regulation on HTA for vaccines and have the potential to ensure consistent, transparent, and timely access to new vaccines in the EU.

目标：欧盟（EU）目前对疫苗的评估涉及许多不同的决策机构和流程。本研究的目的是为欧盟卫生技术评估 (HTA) 法规中使用的标准化方法和疫苗特定流程的开发提供信息：方法：进行文献综述和专家咨询，以确定与疫苗评估相关的现行做法和差距，并制定疫苗联合临床评估的指导原则：结果：我们发现欧盟各成员国在疫苗临床评估的决策过程中存在很大差异。为帮助欧盟制定疫苗联合临床评估的决策框架，我们制定了由 13 项建议组成的三项指导原则：（1）支持为疫苗临床评估创建适当的术语和衡量标准；（2）制定包容、及时和透明的疫苗评估流程，为疫苗决策和评估提供更有力的证据支持；以及（3）改善真实世界数据的收集和互操作性，包括强有力的监测，以促进证据的生成并支持疫苗临床评估的标准化：鉴于疫苗对公共卫生的重要性，迫切需要制定标准化的疫苗专用方法和流程，供欧盟联合 HTA 框架使用。建议的指导原则可支持欧盟《疫苗 HTA 法规》的有效实施，并有可能确保欧盟对新疫苗的一致、透明和及时使用。

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引用次数: 0

Methods for Measuring and Valuing Informal Care: A Systematic Review and Meta-Analysis in Stroke. 衡量和评估非正式护理的方法：中风的系统回顾和荟萃分析。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.007

Neha Das, Phuong Nguyen, Thi Quynh Anh Ho, Peter Lee, Suzanne Robinson, Lan Gao

Objectives: To accurately capture informal care in healthcare evaluations, rigorous approaches are required to measure and value this important care component. In this systematic review and meta-analysis, we intended to summarize the current methods of measuring and valuing informal care costs in healthcare evaluations (full and partial healthcare evaluations, including cost of illness and cost analysis) in stroke.

Methods: A systematic search was conducted in MEDLINE, Embase, EconLit, and CINAHL. We used EndNote 20, Research Screener, and Covidence platforms for screening and data extraction. A meta-analysis was performed on informal care hours, and a subgroup meta-analysis was conducted based on stroke severity.

Results: A total of 31 articles were included in the qualitative synthesis. There was variation among the studies in the informal care measurement and valuation approaches. The meta-analysis of studies where data on informal care hours were available showed an estimate of informal care hours of 25.76 per week (95% CI 13.36-38.16) with a high heterogeneity (I² = 99.97%). The overall risk of bias in the studies was assessed as low.

Conclusions: Standardizing the measurement and valuation of informal care costs is essential for improving the consistency and comparability of economic evaluations. Pilot studies that incorporate standardized informal care cost valuation methods can help identify any practical challenges and capture the impact of informal care more accurately.

目标：为了在医疗保健评估中准确捕捉非正式护理，需要采用严格的方法来衡量和估价这一重要的护理组成部分。在本系统综述和荟萃分析中，我们旨在总结目前在中风的医疗评估（全面和部分医疗评估，包括疾病成本和成本分析）中衡量和估价非正式护理成本的方法：在 Medline、Embase、EconLIT 和 CINAHL 中进行了系统检索。我们使用 EndNote 20、Research Screener 和 Covidence 平台进行筛选和数据提取。对非正式护理时间进行了荟萃分析，并根据中风严重程度进行了分组荟萃分析：定性综合共纳入 31 篇文章。各研究在非正式照护的测量和评估方法上存在差异。对有非正规照护时数数据的研究进行的荟萃分析表明，每周非正规照护时数估计为 25.76 小时（95% 置信区间 [CI]：13.36-38.16）：13.36-38.16），异质性较高（I2=99.97%）。研究的总体偏倚风险被评估为低：要提高经济评估的一致性和可比性，必须对非正规护理成本的测量和估值进行标准化。采用标准化非正规护理成本估价方法的试点研究有助于确定任何实际挑战，并更准确地捕捉非正规护理的影响。

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引用次数: 0

Sales Revenues for New Therapeutic Agents Approved by the United States Food and Drug Administration From 1995 to 2014 1995 年至 2014 年美国食品药品管理局批准的新治疗药物的销售收入：一项回顾性研究。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.015

Objectives

This study aimed to analyze worldwide sales of new therapeutic agents and to estimate the time it takes for product sales to exceed industry-wide average drug development costs.

Methods

Data obtained from company reports were analyzed to track worldwide sales of new medicines approved by the US Food and Drug Administration from 1995 to 2014. All sales figures were reported in 2019 US dollars. Kaplan-Meier curves were used to evaluate the time it took for discounted product sales to exceed the average costs associated with developing 1 new drug (accounting for the costs of failed trials), using published estimates of these costs.

Results

Based on data for 361 of 558 new therapeutic agents approved over the study period (median follow-up 13.2 years), mean sales revenue per product was $15.2 billion through the end of 2019; the median was $6.7 billion. These products jointly generated global sales of $5.5 trillion since approval. Revenues were highly skewed, with the 25 best selling products (7%, 25 of 361) accounting for 38% of this amount ($2.1 trillion of $5.5 trillion). Approximately 47% of products had discounted sales that exceeded the estimated industry-wide average costs of development within 5 years of approval, and 75% within 10 years. After attributing potential production, marketing, and other costs, these numbers dropped to 21% of products within 5 years of approval, and 46% within 10 years.

Conclusions

Sales of new medicines approved from 1995 to 2014 were highly skewed, but many products had net discounted sales that exceeded the industry-wide average costs of development within 10 years of approval. An understanding of how sales revenues accrue in the years after initial approval, alongside data on business costs, can inform discussions about how to incentivize private investment in innovation while ensuring affordable prices for patients and the healthcare system.

目的分析全球新治疗药物的销售情况，并估算产品销售额超过整个行业平均药物开发成本所需的时间：分析从公司报告中获取的数据，追踪 1995 年至 2014 年美国食品药品管理局批准的新药在全球的销售情况。所有销售额均以 2019 美元为单位。采用 Kaplan-Meier 曲线，利用已公布的成本估算值，评估折扣后的产品销售额超过开发一种新药的平均成本（考虑试验失败的成本）所需的时间：根据研究期间（中位数跟踪期：13.2 年）批准的 558 种新治疗药物中 361 种药物的数据，截至 2019 年底，每种产品的平均销售收入为 152 亿美元；中位数为 67 亿美元。自批准以来，这些产品共创造了 5.5 万亿美元的全球销售额。收入高度倾斜，25 种最畅销产品（7%，25/361 种）的收入占这一总额的 38%（2.1 万亿美元/5.5 万亿美元）。约 47% 的产品在获得批准后 5 年内的折扣销售额超过了整个行业的估计平均开发成本，75% 的产品在 10 年内的折扣销售额超过了整个行业的估计平均开发成本。在归因于潜在的生产、营销和其他成本后，这些数字在批准后 5 年内下降到 21%，在 10 年内下降到 46%：结论：1995 年至 2014 年期间获批的新药销售额偏差很大，但大多数产品在获批后 10 年内的净折扣销售额超过了整个行业的平均开发成本。了解销售收入在首次批准后的几年内是如何累积的，同时了解商业成本的数据，可以为讨论如何激励私人投资创新，同时确保患者和医疗系统能够负担得起价格提供参考。

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引用次数: 0

Does Willingness to Pay Differ for Mental and Physical Health? 心理健康和身体健康的支付意愿是否不同？

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.009

Objectives

The urban environment can have a significant impact on mental and physical health. Health impact appraisal of new developments should address these issues. However, transferable economic valuation evidence for urban planners in the United Kingdom is thin, especially around mental health, making it harder to estimate the cost-efficiency of public health interventions to address these conditions. A further complication is that mental health may be perceived differently from physical health. This study examines willingness to pay (WTP) to avoid depression and lower back pain.

Methods

WTP estimates were obtained by applying contingent valuation tasks in an online survey with a representative sample in the United Kingdom (N = 1553). Interval regression models were used to estimate the effects of disease severity, payment frequency, and respondent characteristics on WTP.

Results

Respondents’ WTP to avoid both conditions was relatively high (around 5%-6% of stated income to return to current health state). Depression was rated as being twice as burdensome on quality of life than pain, and bids to avoid depression were 20% to 30% more than pain. Analysis of motivation responses suggests mental health treatment is perceived as less easy to access and less effective than the equivalent for pain, and respondents expect a larger burden on their family and relationships as they try to manage their condition themselves.

Conclusions

Results suggest that depression bids may be affected by uncertainty around access to effective treatment in the healthcare system. This has implications for how mental illness may be prioritized in resource allocation toward public health interventions.

目标：城市环境会对身心健康产生重大影响。新开发项目的健康影响评估应解决这些问题。然而，在英国，城市规划者可转移的经济评估证据很少，尤其是在心理健康方面，因此很难估算针对这些问题的公共卫生干预措施的成本效益。另一个复杂因素是，人们对心理健康的认识可能不同于身体健康。本研究探讨了避免抑郁症和腰背痛的支付意愿（WTP）：方法：通过对英国具有代表性的样本（n=1553）进行在线调查，采用或然估价任务获得 WTP 估计值。采用区间回归模型估算疾病严重程度、支付频率和受访者特征对WTP的影响：结果：受访者避免两种疾病的 WTP 相对较高（约为恢复当前健康状况所需的 5-6% 的收入）。抑郁症对生活质量造成的负担是疼痛的两倍，避免抑郁症的出价比避免疼痛的出价高 20-30%。对动机反应的分析表明，心理健康治疗被认为不如疼痛治疗容易获得，也不如疼痛治疗有效，而且受访者在试图自己控制病情的过程中，预计会给家庭和人际关系带来更大的负担：结果表明，抑郁症的治疗可能会受到医疗保健系统中有效治疗的不确定性的影响。这对如何在公共卫生干预的资源分配中优先考虑精神疾病产生了影响。

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引用次数: 0

Traditional Methods Hold Their Ground Against Machine Learning in Predicting Potentially Inappropriate Medication Use in Older Adults 在预测老年人可能的用药不当方面，传统方法与机器学习相比仍有优势。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.005

Objectives

Machine learning methods have gained much attention in health sciences for predicting various health outcomes but are scarcely used in pharmacoepidemiology. The ability to identify predictors of suboptimal medication use is essential for conducting interventions aimed at improving medication outcomes. It remains uncertain whether machine learning methods could enhance the identification of potentially inappropriate medication use among older adults compared with traditional methods. This study aimed to (1) to compare the performances of machine learning models in predicting use of potentially inappropriate medications and (2) to quantify and compare the relative importance of predictors in a population of community-dwelling older adults (>65 years) in the province of Québec, Canada.

Methods

We used the Québec Integrated Chronic Disease Surveillance System and selected a cohort of 1 105 295 older adults of whom 533 719 were potentially inappropriate medication users. Potentially inappropriate medications were defined according to the Beers list. We compared performances between 5 popular machine learning models (gradient boosting machines, logistic regression, naive Bayes, neural networks, and random forests) based on receiver operating characteristic curves and other performance criteria, using a set of sociodemographic and medical predictors.

Results

No model clearly outperformed the others. All models except neural networks were in agreement regarding the top predictors (sex and anxiety-depressive disorders and schizophrenia) and the bottom predictors (rurality and social and material deprivation indices).

Conclusions

Including other types of predictors (eg, unstructured data) may be more useful for increasing performance in prediction of potentially inappropriate medication use.

目的：在健康科学领域，机器学习方法在预测各种健康结果方面备受关注，但在药物流行病学领域却鲜有应用。识别次优用药预测因素的能力对于开展旨在改善用药效果的干预措施至关重要。与传统方法相比，机器学习方法能否提高识别老年人潜在用药不当的能力仍不确定。本研究的目的是：1）比较机器学习模型在预测潜在用药不当方面的性能；2）量化和比较加拿大魁北克省社区老年人（65 岁以上）群体中预测因素的相对重要性：我们使用了魁北克慢性病综合监测系统，选择了 1,105,295 名老年人作为研究对象，其中 533,719 人可能使用了不适当的药物。潜在的不恰当用药是根据 Beers 清单定义的。我们根据 ROC 曲线和其他性能标准，使用一组社会人口学和医学预测因子，比较了五种流行的机器学习模型（梯度提升机、逻辑回归、天真贝叶斯、神经网络和随机森林）的性能：结果：没有一个模型明显优于其他模型。除神经网络外，所有模型在首要预测因子（性别、焦虑抑郁障碍和精神分裂症）和次要预测因子（农村、社会和物质匮乏指数）方面的表现一致：结论：纳入其他类型的预测因子（如非结构化数据）可能更有助于提高潜在不当用药的预测效果。

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引用次数: 0

A Tutorial on Value-Based Adaptive Designs: Could a Value-Based Sequential 2-Arm Design Have Created More Health Economic Value for the Big CACTUS Trial? 基于价值的适应性设计教程：基于价值的连续双臂设计能否为大型 CACTUS 试验创造更多的健康经济价值？

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.004

Objectives

Value-based trials aim to maximize the expected net benefit by balancing technology adoption decisions and clinical trial costs. Adaptive trials offer additional efficiency. This article provides guidance on determining whether a value-based sequential design is the best option for an adaptive 2-arm trial, illustrated through a case study.

Methods

We outlined 4 steps for the value-based sequential approach. The case study re-evaluates the Big CACTUS trial design using pilot trial data and a model-based health economic analysis. Expected net benefit is computed for (1) original fixed design, (2) value-based design with fixed sample size, and (3) optimal value-based sequential design with adaptive stopping. We compare pretrial modeling with the actual Big CACTUS trial results.

Results

Over 10 years, the adoption decision would affect approximately 215 378 patients. Pretrial modeling shows that the expected net benefit minus costs are (1) £102 million for the original fixed design, (2) £107 million (+5.3% higher) for the value-based design with optimal fixed sample size, and (3) £109 million (+6.7% higher) for the optimal value-based sequential design with maximum sample size of 435 per arm. Post hoc analysis using actual Big CACTUS trial data indicates that the value-adaptive trial with a maximum sample size of 95 participant pairs would not have stopped early. Bootstrap simulations reveal a 9.76% probability of early completion with n = 95 pairs compared with 31.50% with n = 435 pairs.

Conclusions

The 4-step approach to value-based sequential 2-arm design with adaptive stopping was successfully implemented. Further application of value-based adaptive approaches could be useful to assess the efficiency of alternative study designs.

目标：基于价值的试验旨在通过平衡技术采用决策和临床试验成本，最大限度地提高预期净效益。适应性试验可提高效率。本文通过一个案例研究，为确定基于价值的序列设计是否是适应性双臂试验的最佳选择提供指导：方法：我们概述了基于价值的序列方法的四个步骤。案例研究利用试点试验数据和基于模型的卫生经济分析重新评估了 Big CACTUS 试验设计。计算了(a)原始固定设计、(b)基于价值的固定样本量设计和(c)基于价值的最优顺序设计以及自适应停止的预期净效益。我们将试验前的模型与 Big CACTUS 的实际试验结果进行了比较：结果：十年间，采用该疗法的决定将影响约 215,378 名患者。试验前建模显示：(a) 原始固定设计的预期净收益减去成本为 1.02 亿英镑；(b) 最佳固定样本量的基于价值的设计为 1.07 亿英镑（高出 5.3%）；(c) 每臂最大样本量为 435 个的最佳基于价值的序列设计为 1.09 亿英镑（高出 6.7%）。使用 Big CACTUS 实际试验数据进行的事后分析表明，最大样本量为 95 对参与者的价值自适应试验不会提前停止。Bootstrap 模拟显示，n=95 时提前结束试验的概率为 9.76%，而 n=435 时为 31.50%：基于价值的顺序双臂设计的四步骤自适应停止方法已成功实施。进一步应用基于价值的自适应方法可能有助于评估替代研究设计的效率。

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引用次数: 0

A New and Improved Experimental Design for the Discrete Choice Experiment Module of the EuroQol Valuation Technology Protocol 对 EQ-VT 估值协议的离散选择实验模块进行新的改进实验设计。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.05.024

Objectives

The EuroQol Valuation Technology (EQ-VT) protocol, which is used to value the EQ-5D-5L instrument, comprises a composite time trade-off and a discrete choice experiment (DCE) module. Despite significant limitations, the DCE module has not been updated since its inception in 2012. This study aimed to update the EQ-VT DCE design using state-of-the-art methods.

Methods

DCE data from 19 EQ-5D-5L valuation studies were summarized using a Bayesian hierarchical meta-analysis model, which created the priors for our Bayesian efficient DCE design. This design comprised 20 subdesigns, each with 12 choice tasks, and included 2 levels that overlapped to reduce the complexity of the choice tasks. The relative efficiency and robustness of the new design were established by comparing the D-errors and minimal sample size requirements for the 19 within-sample and 7 out-of-sample countries with the previous DCE design.

Results

The updated DCE design shows large reductions in the D-error: by 20% and 22% for the 19 within-sample and 7 out-of-sample countries, respectively. Sample size requirements were also reduced, resulting in an average reduction of 45% for both the within and out-of-sample countries.

Conclusions

The updated DCE design outperforms the current EQ-VT design. Given its enhanced performance and reduced complexity, it is set to replace the existing DCE design in future EQ-5D-5L valuation studies using the EQ-VT protocol.

目的：用于评估 EQ-5D-5L 仪器的 EuroQol 估值技术（EQ-VT）协议由综合时间权衡（cTTO）和离散选择实验（DCE）模块组成。尽管离散选择实验模块有很大的局限性，但自 2012 年推出以来一直没有更新。本研究旨在采用最先进的方法更新 EQ-VT DCE 设计：方法：使用贝叶斯分层荟萃分析模型总结了来自 19 项 EQ-5D-5L 估值研究的 DCE 数据，为我们的贝叶斯高效 DCE 设计创建了先验。该设计包括 20 个子设计，每个子设计有 12 个选择任务，其中有两个层次重叠，以降低选择任务的复杂性。通过比较 19 个样本内国家和 7 个样本外国家的 D 错误和最小样本量要求与之前的 DCE 设计，确定了新设计的相对效率和稳健性：更新后的 DCE 设计显示 D 误差大幅降低：19 个样本内国家和 7 个样本外国家的 D 误差分别降低了 20% 和 22%。对样本量的要求也有所降低，因此样本内国家和样本外国家的样本量平均降低了 45%：更新后的 DCE 设计优于当前的 EQ-VT 设计。结论：更新后的 DCE 设计优于当前的 EQ-VT 设计，鉴于其性能的提高和复杂性的降低，在未来使用 EQ-VT 协议进行的 EQ-5D-5L 估值研究中，它将取代现有的 DCE 设计。

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引用次数: 0

Three Pillars of Support for Orphan Drug Programs: Individual Valuations, Societal Valuations, and Anonymous Altruism. 支持孤儿药计划的三大支柱：个人价值、社会价值和匿名利他主义。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.014

Darius N Lakdawalla, Charles E Phelps, Hong-Hanh Nguyen

Objectives: To identify and describe potential societal and individual sources of support for orphan drug programs.

Methods: The Generalized Risk-Adjusted Cost-Effectiveness method shows that acute illness and disability severity increase individuals' willingness to pay for health gains. We develop a social welfare function (SWF) that incorporates individuals' own values, combined with politically or ethically determined weights. We introduce the concept of horizontal equity-that individuals in similar situations should be treated similarly-into the SWF. Finally, we introduce anonymous altruism into individuals' utility functions-the desire to help others, without knowing their identity.

Results: Combined with the empirical link between disease severity and rarity, the Generalized Risk-Adjusted Cost-Effectiveness method demonstrates heightened willingness to pay for health gains for people with rare diseases, leading rational individuals to support orphan drug programs, our first pillar of support. Adding horizontal equity to the SWF further increases societal support for orphan drug programs. Anonymous altruism, focusing most strongly on those in the most-dire circumstances, leads to altruistic support for those with severe disorders. Because innovators' economic incentives lead them to focus on larger markets, anonymous altruistic individuals will increasingly prefer public investments into rare diseases over time, as private markets systematically produce gains for common diseases.

Conclusions: We identified 3 supporting pillars for orphan drug programs: (1) individuals' propensity to prefer treatments for severe diseases; (2) the preference for horizontal equity in our social welfare; (3) anonymous altruism, the desire to help strangers, coupled with market incentives that underserve strangers with rare diseases.

目的确定并描述支持孤儿药项目的潜在社会和个人来源：广义风险调整成本效益法（GRACE）显示，急性病和残疾的严重程度会增加个人对健康收益的支付意愿（WTP）。我们开发了一种社会福利函数（SWF），将个人自身的价值与政治或道德决定的权重相结合。我们在 SWF 中引入了横向公平的概念，即处于类似情况的个人应得到类似的待遇。最后，我们在个人效用函数中引入了匿名利他主义--在不知道他人身份的情况下帮助他人的愿望：结果：结合疾病严重性与稀有性之间的经验联系，GRACE 显示了对健康收益的更高 WTP，从而引导理性个体支持孤儿药项目，即我们的第一支持支柱。将横向公平添加到 SWF 中会进一步增加社会对孤儿药项目的支持。匿名利他主义最强烈地关注那些处于最恶劣环境中的人，从而导致对严重失调患者的利他主义支持。由于创新者的经济动机促使他们关注更大的市场，因此匿名利他主义者会特别支持孤儿药项目。搭便车问题的存在将其转化为对公共项目的支持：我们确定了支持孤儿药项目的三大支柱：（1）个人对治疗罕见疾病的渴望，这些疾病通常病情严重、危及生命；（2）我们的社会福利基金中的横向公平概念；（3）匿名利他主义，即对处于困境中的人的渴望，即使他们并不为人所知。

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引用次数: 0

Mapping the Lequesne Functional Index Into the EQ-5D-5L Utility Index in Patients With Knee Osteoarthritis 将勒克斯功能指数映射到膝关节骨关节炎患者的 EQ-5D-5L 实用性指数中。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-06 DOI: 10.1016/j.jval.2024.06.017

Objective

This study aimed to map the Lequesne index onto the EuroQol 5 Dimension (EQ-5D-5L) utility index for patients with knee osteoarthritis.

Methods

Baseline data from a previous randomized controlled trial were used; 461 patients were involved in the mapping development, and 230 in the validation phase. Various modeling techniques, including generalized linear models, tobit, and beta regression, were used. Factors such as age, sex, and body mass index were considered as covariates. Model selection was based on criteria such as Akaike and Bayesian information criteria, adjusted R², mean absolute error, and root mean squared error. Validation involved assessing the preselected models using mean absolute error, root mean squared error, and intraclass correlation coefficient. This study follows the Mapping Onto Preference-Based Measures Reporting Standards statement.

Results

Five models were developed, with 2 incorporating age, sex, with or without body mass index along with the Lequesne index showing the best fit across regressions. Validation results were similar for the 3 regressions, with beta regression models exhibiting wider ranges closer to the validation data set. Intraclass correlation coefficient values were better for beta regression models. Both models tended to overpredict for lower EQ-5D-5L values and underpredict for better health status.

Conclusion

These mapping functions, the first of their kind, effectively translate the Lequesne index to EQ-5D-5L values in patients with knee osteoarthritis. They demonstrate satisfactory fit and precision, providing valuable tools for clinicians and researchers, particularly in situations where generic preference-based health-related quality of life instruments are inaccessible for utility derivation in cost-effectiveness studies.

研究目的本研究旨在将膝关节骨性关节炎（OA）患者的勒克恩指数映射到欧洲质量标准 5 维（EQ-5D-5L）效用指数上：方法：利用之前一项随机对照试验的基线数据。461名患者参与了绘图开发，230名患者参与了验证阶段。采用了多种建模技术，包括一般线性模型、Tobit 和贝塔回归。年龄、性别和体重指数等因素被视为协变量。模型的选择基于 Akaike 和 Bayesian 信息标准、调整后 R2、平均绝对误差 (MAE) 和均方根误差 (RMSE) 等标准。验证包括使用 MAE、RMSE 和类内相关系数 (ICC) 评估预选模型。这项研究遵循了基于偏好的测量报告标准（MAPS）声明：共建立了五个模型，其中两个模型结合了年龄、性别和/或体重指数以及勒克森指数，显示出最佳的回归拟合效果。三种回归的验证结果相似，Beta 回归模型的范围更接近验证数据集。Beta 回归模型的 ICC 值更高。两种模型都倾向于对较低的 EQ-5D-5L 值预测过高，而对较好的健康状况预测过低：这些映射函数是同类模型中的首个，可有效地将膝关节 OA 患者的勒克斯恩指数转换为 EQ-5D-5L 值。它们显示出令人满意的拟合度和精确度，为临床医生和研究人员提供了有价值的工具，尤其是在成本效益研究中无法使用基于一般偏好的健康相关生活质量工具进行效用推导的情况下。

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引用次数: 0

Uncertainty in Long-Term Relative Effectiveness of Medicines in Health Technology Assessment 健康技术评估中药物长期相对有效性的不确定性：比较脊髓肌肉萎缩症、囊性纤维化和高胆固醇血症药物的评估。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health

Pub Date : 2024-07-04 DOI: 10.1016/j.jval.2024.05.023

Objectives

Uncertainty regarding the long-term relative effectiveness is an important factor in health technology assessment (HTA) of medicines. This study investigated how different HTA bodies address this uncertainty in their assessments.

Methods

A total of 49 HTA reports from 6 national HTA bodies, assessing 9 medicines for spinal muscular atrophy, cystic fibrosis, and hypercholesterolemia, were included. In these reports, 81 relative effectiveness assessments and 45 cost-effectiveness assessments were performed on an indication level. We collected information on included trials, assessment outcomes, uncertainty regarding the long-term effectiveness, proposed managed entry agreements, and reassessments.

Results

Uncertainty regarding the long-term effectiveness was an important consideration in almost all cost-effectiveness assessments (91%) and three-quarters of relative effectiveness assessments (74%), despite differences in methodologies among HTA bodies. There were considerable differences in the amount and type of long-term effectiveness data included by HTA bodies due to timing and inclusion criteria. In total 23 managed entry agreements were proposed of which 14 were linked to uncertainty regarding the long-term effectiveness. In addition, 13 reassessments were performed of which 4 led to an increase in patient access because of more available long-term effectiveness data.

Conclusions

Uncertainty regarding the long-term effectiveness is an important challenge for HTA bodies. There are large differences in the acceptance of evidence among HTA bodies, which leads to heterogeneity in the inclusion of available long-term effectiveness data for decision making. In cases with large uncertainty regarding the long-term effectiveness, outcome-based agreements and reassessments are used by HTA bodies, but differently between HTA bodies and indications.

目的：长期相对有效性的不确定性是药品卫生技术评估（HTA）中的一个重要因素。本研究调查了不同的 HTA 机构在评估中是如何处理这种不确定性的。方法：研究纳入了 6 个国家 HTA 机构的 49 份 HTA 报告，评估了脊髓性肌萎缩症、囊性纤维化和高胆固醇血症等 9 种药物。在这些报告中，按适应症级别进行了 81 项相对有效性评估 (REA) 和 45 项成本效益评估 (CEA)。我们收集了有关纳入试验、评估结果、长期有效性的不确定性、拟议的管理下进入协议（MEA）和重新评估的信息：结果：尽管各 HTA 机构采用的方法不同，但长期有效性的不确定性几乎是所有 CEA（91%）和四分之三 REA（74%）的重要考虑因素。由于时间和纳入标准的不同，各 HTA 机构所纳入的长期有效性数据的数量和类型也存在很大差异。总共提出了 23 项多边环境评估，其中 14 项与长期有效性的不确定性有关。此外，还进行了 13 次重新评估，其中 4 次由于获得了更多的长期有效性数据而增加了患者的可及性：结论：长期有效性的不确定性是 HTA 机构面临的一个重要挑战。结论：长期有效性的不确定性是 HTA 机构面临的重要挑战。各 HTA 机构在接受证据方面存在巨大差异，这导致了在将可用的长期有效性数据纳入决策方面的不一致性。在长期有效性存在较大不确定性的情况下，HTA 机构会采用基于结果的协议和重新评估，但不同的 HTA 机构和适应症会有不同的做法。

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引用次数: 0