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Correction: Systematic Literature Review of Access Pathways to Drugs for Patients with Rare Diseases 更正:罕见病患者药物获取途径的系统文献综述。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-03-21 DOI: 10.1007/s40258-025-00959-8
Constanza Vargas, Richard De Abreu Lourenco, Manuel Espinoza, Stephen Goodall
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引用次数: 0
A Scoping Review Mapping Economic Evaluations of Midwifery Service Provision and the Midwifery Workforce 助产服务提供和助产人员经济评估的范围综述。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-03-19 DOI: 10.1007/s40258-025-00962-z
Boe Calvert, Caroline S. E. Homer, Sarah Bar-Zeev, Alicia Ferguson, Vanessa Scarf

Background

Midwives are essential in achieving universal health coverage targets and the health targets of the Sustainable Development Goals, yet a significant global shortfall exists in the midwifery workforce. Economic evaluations of midwifery are scarce but can assist in supporting evidence-informed decision-making for sustainable and equitable health care for women and girls.

Objectives

This review aimed to systematically identify, map and report on available literature regarding economic evaluations conducted on midwifery service provision and the midwifery workforce in all settings.

Methods

A scoping review was conducted following the Joanna Briggs Institute methodology. A comprehensive search strategy was developed and run in six health databases. Peer-reviewed studies and unpublished research theses conducting economic evaluations on midwifery service provision or midwifery workforce strategies were included. Sources were limited to English-language literature published in the past 20 years. Identified sources were screened and reviewed, and data from included sources were extracted, reviewed, mapped and synthesised to report findings. Quality appraisal was conducted on all included sources using the Joanna Briggs Institute Critical Appraisal Checklist for Economic Evaluations.

Results

A total of 32 studies were included in the review. Most were from high-income countries (26/32), and very few were from low- and middle-income countries (6/32). The quality of included studies varied greatly. Under half of the studies conducted full economic evaluations (15/32), and the remainder were partial economic evaluations (17/32). Most studies evaluated midwifery service provision (29/32) through either midwife-led models of care (15/29) or by place of birth (13/29), mostly for low-risk women (23/29) from the perspective of healthcare funders. Evaluation of midwifery education programs was less common, and these were all conducted in low- and middle-income countries (3/32). Most studies concluded that midwifery service provision was cost-saving, cost-effective or cost-beneficial.

Conclusions

Our review identified a significant gap in economic evaluation of midwifery from low- and middle-income countries. However, there is ongoing need for robust, quality economic evaluations on midwifery service provision and workforce strategies in all global regions. Such studies would further support health policymakers and governments to make evidence-informed decisions to address midwifery workforce shortages and provision of evidence-based and respectful care that meets the healthcare needs of women and girls.

背景:助产士对于实现全民健康覆盖具体目标和可持续发展目标的健康具体目标至关重要,但全球助产士队伍存在严重短缺。助产的经济评价很少,但可以帮助支持循证决策,为妇女和女童提供可持续和公平的保健服务。目的:本综述旨在系统地识别、绘制和报告有关在所有环境中对助产服务提供和助产人员进行经济评估的现有文献。方法:根据乔安娜布里格斯研究所的方法进行范围审查。制定了一项综合搜索战略,并在六个卫生数据库中运行。包括对助产服务提供或助产劳动力战略进行经济评估的同行评议研究和未发表的研究论文。资料来源仅限于过去20年出版的英语文学。筛选和审查已确定的来源,并从纳入的来源提取、审查、绘制和综合数据,以报告发现。使用乔安娜布里格斯研究所经济评估关键评估清单对所有纳入的资源进行质量评估。结果:本综述共纳入32项研究。大多数来自高收入国家(26/32),很少来自低收入和中等收入国家(6/32)。纳入研究的质量差异很大。不到一半的研究进行了全面的经济评价(15/32),其余的是部分经济评价(17/32)。大多数研究通过助产士主导的护理模式(15/29)或按出生地(13/29)评估助产服务的提供(29/32),主要是从保健资助者的角度评估低风险妇女(23/29)。对助产教育项目的评估不太常见,这些都是在低收入和中等收入国家进行的(3/32)。大多数研究的结论是,提供助产服务是节省成本、具有成本效益或具有成本效益的。结论:我们的综述发现了低收入和中等收入国家在助产经济评估方面的显著差距。然而,目前仍需要对全球所有区域的助产服务提供和劳动力战略进行强有力、高质量的经济评估。这些研究将进一步支持卫生政策制定者和政府作出循证决策,解决助产人员短缺问题,并提供循证和尊重的护理,满足妇女和女童的保健需求。
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引用次数: 0
Beyond the Diagnosis: Valuing Genome-Wide Sequencing for Rare Disease Diagnosis Using Contingent Valuation 诊断之外:利用偶然价值评估全基因组测序对罕见疾病诊断的价值。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2025-03-14 DOI: 10.1007/s40258-025-00948-x
Michael Abbott, Mandy Ryan, Rodolfo Hernández, Sebastian Heidenreich, Zosia Miedzybrodzka

Background and Objective

The utility of genome-wide sequencing is often quantified in terms of its diagnostic yield. Although obtaining a diagnosis is a fundamental aspect of value, service users also value broader clinical, informational, process and psychological factors in the provision of genomic testing. This study aims to value genome-wide sequencing from the user perspective in Scotland.

Methods

A survey was developed and administered to 1014 patients and families with experience of genome-wide sequencing to diagnose a rare condition in Scotland. Participants’ willingness to pay for genomic testing was elicited using a contingent valuation payment card. The survey included two genomic-related patient-reported outcome measures: (i) the Personal Utility Scale (PrU) to generate scores for the personal utility of genome-wide sequencing; and (ii) a subscale of the Feelings About Genomic Testing Results (FACTOR) questionnaire to measure negative psychological outcomes. Data were also collected on participants’ prior experiences of genomic testing services. A double-hurdle regression model investigated the predictors of patients' willingness to pay for genomic testing.

Results

Of the 1014 invitations sent, 171 contingent valuation questionnaires were returned. Diagnosed participants reported higher personal utility on PrU than undiagnosed participants. However, both groups reported similar negative psychological outcomes on FACTOR. Diagnosed participants were willing to pay £2043 for genome-wide sequencing, compared with £835 for undiagnosed participants. Diagnostic status, waiting time for results and FACTOR scores (negative psychological outcomes) influenced users’ valuations of genome-wide sequencing.

Conclusions

Obtaining a diagnosis is a fundamental component of utility in the provision of genomic testing. However, there is still value to those who do not receive a diagnosis. These results have implications for service delivery, such as providing targeted pre-test and post-test genetic counselling, and investing in efficient genome sequencing pipelines to reduce waiting times. Valuing the user experience of genomic testing aligns with patient-centred approaches to the provision of healthcare.

背景和目的:全基因组测序的效用通常是根据其诊断率来量化的。虽然获得诊断是价值的一个基本方面,但服务用户也重视提供基因组检测的更广泛的临床、信息、过程和心理因素。本研究旨在从苏格兰用户的角度来评估全基因组测序。方法:一项调查被开发和管理的1014名患者和家庭与全基因组测序的经验,以诊断罕见的条件在苏格兰。参与者支付基因组测试的意愿是通过一张条件评估支付卡来激发的。该调查包括两项与基因组相关的患者报告的结果测量:(i)个人效用量表(PrU),用于为全基因组测序的个人效用生成分数;以及(ii)关于基因组测试结果的感受(FACTOR)问卷的子量表,以测量负面心理结果。还收集了参与者先前的基因组检测服务经验的数据。一个双栏回归模型调查了患者愿意为基因组检测付费的预测因素。结果:在发出的1014份邀请中,回收了171份条件评估问卷。确诊的参与者在PrU上的个人效用高于未确诊的参与者。然而,两组在FACTOR上报告的负面心理结果相似。确诊的参与者愿意为全基因组测序支付2043英镑,而未确诊的参与者愿意支付835英镑。诊断状态、结果等待时间和因子得分(负面心理结果)影响用户对全基因组测序的评价。结论:获得诊断是提供基因组检测的基本组成部分。然而,对于那些没有得到诊断的人来说,这仍然是有价值的。这些结果对提供服务具有影响,例如提供有针对性的检测前和检测后遗传咨询,以及投资于有效的基因组测序管道以减少等待时间。重视基因组测试的用户体验与提供医疗保健的以患者为中心的方法是一致的。
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引用次数: 0
A Head-On Comparison of EQ-VT- and Crosswalk-Based EQ-5D-5L Value Sets EQ-VT和基于人行横道的EQ-5D-5L值集的正面比较
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-03-11 DOI: 10.1007/s40258-025-00954-z
Henry Bailey, Bram Roudijk

Background

No systematic country-level comparison has been undertaken between crosswalk- and EQ-VT-derived EQ-5D-5L value sets. Crosswalk values can differ from EQ-VT-based EQ-5D-5L value sets owing to valuation protocols, changes in societal preferences over time, and a change in the label of the highest level on mobility in moving from EQ-5D-3L to EQ-5D-5L. This study aimed to compare the five-level (5L) crosswalk and EQ-VT value sets to explore differences between them at the country level.

Methods

From the countries with both time trade-off (TTO)- or discrete choice experiment (DCE) + TTO-based EQ-5D-3L value sets and EQ-VT-based EQ-5D-5L value sets, 19 pairs of EQ-5D-3L/EQ-5D-5L sets were found. For each of these EQ-5D-3L value sets, 5L crosswalk sets were developed and compared with the corresponding national EQ-5D-5L valuation set using correlation analysis, ranges, values of specific states, Bland–Altman plots, and scatter plots. Three of the countries have EQ-5D-3L and EQ-5D-5L valuation data for the same set of respondents. These three cases were analyzed separately, as they provide a “true” test of the differences between the two value sets.

Results

Spearman correlation between the crosswalk and valuation sets ranged from 0.831 to 0.989, being below 0.9 in 11 pairs of value sets. The difference in the percentage of negative values ranged from +22.5 to −18.8%, and the difference in the ranges within each pair of value sets ranged from +42.7 to −18.4%. The average mean absolute difference of values (crosswalk versus EQ-VT) was 0.149. This was below 0.1 in only 5 of the 19 EQ-VT/crosswalk set pairs. For the states comprising one level 5 and four level 1s, no country preserved its ranking of importance of the five dimensions in moving from crosswalk to EQ-VT values. Most of the Bland–Altman plots and scatterplots revealed a pattern that placed states with the highest level on mobility as a separate band from other states.

Discussion

All of the criteria showed poor agreement between the crosswalk- and EQ-VT-based value sets. The differences in labels for the most extreme response option for the mobility dimension leads to substantial differences in values between these value sets.

Conclusions

Crosswalk and EQ-VT value sets should not be used interchangeably, except under circumstances where it is not possible or feasible to conduct a direct EQ-5D-5L valuation study.

背景:没有对人行横道和eq - vt衍生的EQ-5D-5L值集进行系统的国家级比较。人行横道的价值可能不同于基于eq - v的EQ-5D-5L价值集,这是由于评估协议、随着时间的推移社会偏好的变化,以及从EQ-5D-3L到EQ-5D-5L的最高流动性水平标签的变化。本研究旨在比较五层(5L)人行横道和EQ-VT值集,以探讨它们在国家层面上的差异。方法:从同时存在时间权衡(TTO)或离散选择实验(DCE) +基于时间权衡(TTO)的EQ-5D-3L值集和基于eq - v的EQ-5D-5L值集的国家中,发现了19对EQ-5D-3L/EQ-5D-5L值集。对于每一个EQ-5D-3L价值集,我们开发了5L人行横道价值集,并使用相关分析、范围、特定状态值、Bland-Altman图和散点图将其与相应的国家EQ-5D-5L价值集进行比较。其中三个国家对同一组受访者拥有EQ-5D-3L和EQ-5D-5L估值数据。这三种情况分别进行了分析,因为它们提供了对两个值集之间差异的“真实”测试。结果:人行横道与评价集的Spearman相关系数在0.831 ~ 0.989之间,有11对评价集的Spearman相关系数小于0.9。负值百分比的差异从+ 22.5%到-18.8%不等,每对值集范围的差异从+42.7到-18.4%不等。平均绝对差值(人行横道与EQ-VT)为0.149。在19对EQ-VT/人行横道组中,只有5对低于0.1。对于包含一个5级和四个15级的州,没有一个国家保留了从人行横道到EQ-VT值的五个维度的重要性排名。大多数Bland-Altman图和散点图揭示了一种模式,即将流动性水平最高的州与其他州分开作为一个单独的带。讨论:所有的标准都显示基于人行横道和eq - vt的值集之间的一致性很差。流动性维度的最极端响应选项的标签差异导致这些值集之间的值存在实质性差异。结论:人行横道和EQ-VT值不应该互换使用,除非在不可能或不可行进行直接EQ-5D-5L评估研究的情况下。
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引用次数: 0
GaitSmart Rehabilitation Exercise Programme for Gait and Mobility Issues: A NICE Medical Technologies Guidance 步态和行动问题的GaitSmart康复运动计划:NICE医疗技术指南。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-03-10 DOI: 10.1007/s40258-025-00955-y
Huey Yi Chong, Michal Pruski, Megan Dale, Rhys Morris

GaitSmart (Dynamic Metrics Ltd) is a class I CE-marked sensor-based digital technology designed to measure lower limb movement via sensors placed on the body. GaitSmart aims to identify any problems with gait, and provide a personalised rehabilitation programme via the integrated vGym app. The company identified people referred for knee or hip replacement and people at risk of falling as potentially being able to benefit from GaitSmart. The clinical evidence identified was limited in its applicability to the scope of the assessment, and there was large variability in the comparators used in these studies and in the current care pathways, especially for people at risk of falls. There is some limited evidence that patients like the report generated by the technology and found it helpful in understanding their condition. Clinical experts thought GaitSmart had the potential to improve outcomes for patients and could be used in the community setting by a range of healthcare professionals. Economic modelling over a 1-year time horizon from a UK National Health Service (NHS) perspective for the falls model and a 17-week horizon for the rehabilitation model indicate that GaitSmart is cost saving; however, this is dependent on the model of standard care. Where GaitSmart is delivered by trained healthcare assistants, it would lead to a reduction in staff time costs compared to most alternative standard care options. Uncertainty is greater in the falls model given the high variability in standard care. In April 2024, the National Institute for Health and Care Excellence recommended that GaitSmart can be used to treat gait and mobility issues in adults at risk of falls, while more evidence is gathered. For adults having hip or knee replacements, access to GaitSmart should be via the company, research or non-core NHS funding, with appropriate management of any risks.

GaitSmart (Dynamic Metrics Ltd)是一种I类ce认证的基于传感器的数字技术,旨在通过放置在身体上的传感器来测量下肢运动。GaitSmart旨在识别任何步态问题,并通过集成的vGym应用程序提供个性化的康复计划。该公司确定了需要膝关节或髋关节置换术的人,以及有跌倒风险的人,这些人可能会从GaitSmart中受益。所确定的临床证据在评估范围的适用性方面是有限的,并且在这些研究中使用的比较物和当前的护理途径中存在很大的可变性,特别是对于有跌倒风险的人。有一些有限的证据表明,患者喜欢这项技术生成的报告,并发现它有助于了解他们的病情。临床专家认为,GaitSmart有可能改善患者的预后,可以在社区环境中被一系列医疗保健专业人员使用。从英国国民健康服务(NHS)的角度来看,跌倒模型的1年时间跨度和康复模型的17周时间跨度的经济模型表明,GaitSmart节省了成本;然而,这取决于标准护理的模式。如果GaitSmart由训练有素的医疗保健助理提供,与大多数替代标准护理方案相比,它将减少员工的时间成本。考虑到标准护理的高度可变性,falls模型的不确定性更大。2024年4月,美国国家健康与护理卓越研究所(National Institute for Health and Care Excellence)建议,GaitSmart可用于治疗有跌倒风险的成年人的步态和行动能力问题,同时还收集了更多证据。对于接受髋关节或膝关节置换术的成年人,应该通过公司、研究机构或非核心NHS基金获得GaitSmart,并对任何风险进行适当的管理。
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引用次数: 0
New Horizons? Assessing General Public Preferences for a Wellbeing Economy in the Post-COVID-19 World 新视野号吗?评估公众对后covid -19世界福祉经济的普遍偏好。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 DOI: 10.1007/s40258-025-00951-2
Rachel Milte, Matthew Crocker, Gang Chen, Gordon Duff, Julie Ratcliffe

Objective

As societies emerge from the COVID-19 pandemic, governments are increasingly moving away from a focus on economic growth at any cost towards the principles of a wellbeing economy, focused on achieving a more equitable distribution of wealth and wellbeing. This study aimed to assess the relative importance to the Australian general public of the key principles of a wellbeing economy and to investigate heterogeneity in preferences between demographic subgroups.

Methods

An online survey was developed and delivered to a general public sample of 2042 Australian adults (aged 18 years and above). Respondents were invited to rank the key principles of a wellbeing economy (dignity, nature and climate, social connection, fairness and participation) plus two additional traditional economic indicators of societal success (‘economic growth’ and ‘economic prosperity’) in order of their relative importance for informing future policy directions. Data analysis was conducted using simple summative scoring, which involved the use of a point system allocated to rankings as a dependent variable. In addition, a rank-ordered logit model was used to explore preferences for the entire sample and subgroups defined by key socio-demographic characteristics.

Results

‘Dignity’ (people have enough to live in comfort, safety and happiness) and ‘fairness’ (equal opportunity for all Australians and the gap between richest and poorest greatly reduced) were ranked as the most important priorities for the total sample and for key socio-demographic subgroups differentiated by age, level of education and level of socio-economic advantage. Traditional economic indicators of societal success including ‘economic prosperity’ and ‘economic growth’ were considered important but generally ranked below the principles of ‘dignity’ and ‘fairness’.

Conclusions

The findings indicate that government movements away from traditional economic indicators and towards new broader wellbeing economy measures of societal success are likely to be supported by the general public.

目标:随着社会从COVID-19大流行中复苏,各国政府正越来越多地从不惜一切代价关注经济增长转向福祉经济原则,重点是实现更公平的财富和福祉分配。本研究旨在评估福利经济的关键原则对澳大利亚公众的相对重要性,并调查人口亚组之间偏好的异质性。方法:开发了一项在线调查,并向2042名澳大利亚成年人(18岁及以上)提供了一般公众样本。受访者被邀请对福利经济的关键原则(尊严、自然和气候、社会联系、公平和参与)以及另外两个社会成功的传统经济指标(“经济增长”和“经济繁荣”)进行排名,以确定它们对未来政策方向的相对重要性。数据分析使用简单的总结性评分进行,其中涉及使用分配给排名的积分系统作为因变量。此外,使用秩序logit模型来探索由关键社会人口特征定义的整个样本和子群体的偏好。结果:“尊严”(人们有足够的舒适、安全和幸福的生活)和“公平”(所有澳大利亚人机会平等,贫富差距大大缩小)被列为总样本和按年龄、教育水平和社会经济优势水平区分的关键社会人口亚群体的最重要优先事项。“经济繁荣”和“经济增长”等衡量社会成功的传统经济指标被认为很重要,但通常排在“尊严”和“公平”原则之后。结论:研究结果表明,政府从传统的经济指标转向新的更广泛的社会成功福利经济指标的行动可能会得到公众的支持。
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引用次数: 0
Is the Use of Unanchored Matching-Adjusted Indirect Comparison Always Superior to Naïve Indirect Comparison on Survival Outcomes? A Simulation Study 非锚定匹配调整间接比较在生存结果上总是优于Naïve间接比较吗?模拟研究。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-02-23 DOI: 10.1007/s40258-025-00952-1
Ying Liu, Xiaoning He, Jia Liu, Jing Wu

Objective

To compare the performance of matching-adjusted indirect comparison (MAIC) and naïve indirect comparison (NIC) under a wide range of data scenarios on survival outcome.

Methods

A simulation study included 729 (36) single-arm trial data scenarios, which were created by performing a three-level full factorial arrangement of six situational variables, including individual patient data (IPD) sample size, aggregate data (AgD) sample size, covariate strength, covariate correlation, covariate overlap, and relative treatment effect. In each scenario, 1000 repetitions of simulated datasets were generated using the Monte Carlo approach. MAIC and NIC methods were used to estimate the relative treatment effect of each simulated dataset. The performance was evaluated in terms of bias, empirical standard error (ESE), mean squared error (MSE), and confidence interval coverage, respectively.

Results

MAIC yielded relatively unbiased estimates of relative treatment effect compared with NIC in most scenarios, with better coverage and MSE but higher ESE. None of the situational variables had a significant impact on the bias and coverage of MAIC. However, increasing IPD sample size and covariate overlap significantly reduced the ESE and MSE of MAIC. In scenarios with low covariate overlap and high covariate strength, the bias of MAIC was larger and even greater than that of NIC.

Conclusions

The performance of MAIC consistently demonstrates advantage over NIC across various scenarios. MAIC often provides more unbiased estimates and achieves confidence interval coverage close to nominal values compared with NIC. While MAIC may exhibit higher ESE in specific scenarios, this additional uncertainty can offer a more accurate reflection of variability, enhancing the robustness of the results. Researchers should thoroughly comprehend the influencing factors and interactions affecting the performance of these methods and judiciously apply research findings.

目的比较匹配调整间接比较法(MAIC)和天真间接比较法(NIC)在多种数据情景下对生存结果的影响:模拟研究包括 729 个(36 个)单臂试验数据情景,这些情景是通过对六个情景变量(包括单个患者数据(IPD)样本量、总体数据(AgD)样本量、协变量强度、协变量相关性、协变量重叠和相对治疗效果)进行三级全因子排列而创建的。在每种情况下,使用蒙特卡罗方法生成 1000 次重复的模拟数据集。使用 MAIC 和 NIC 方法估算每个模拟数据集的相对治疗效果。分别从偏差、经验标准误差(ESE)、均方误差(MSE)和置信区间覆盖率等方面对其性能进行了评估:在大多数情况下,与 NIC 相比,MAIC 对相对治疗效果的估计相对无偏,覆盖率和 MSE 更高,但 ESE 更高。没有一个情景变量对 MAIC 的偏差和覆盖率有显著影响。然而,增加 IPD 样本规模和共变量重叠度会显著降低 MAIC 的 ESE 和 MSE。在协变量重叠度低、协变量强度高的情况下,MAIC 的偏差更大,甚至超过了 NIC:结论:在各种情况下,MAIC 的性能始终优于 NIC。与 NIC 相比,MAIC 通常能提供更无偏的估计值,并实现接近名义值的置信区间覆盖率。虽然 MAIC 在特定情况下可能表现出更高的 ESE,但这种额外的不确定性可以更准确地反映变异性,从而增强结果的稳健性。研究人员应全面了解影响这些方法性能的影响因素和相互作用,并明智地应用研究成果。
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引用次数: 0
Cost-Utility Analysis of Genomic Profiling in Directing Targeted Therapy in Advanced NSCLC in Thailand 基因组谱在泰国指导晚期NSCLC靶向治疗中的成本-效用分析。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2025-02-07 DOI: 10.1007/s40258-025-00950-3
Saowalak Turongkaravee, Surakit Nathisuwan, Thanyanan Baisamut, Jennis Meanwatthana

Background

Sequential next-generation sequencing (NGS) testing has demonstrated cost-effectiveness in guiding targeted therapy with tyrosine kinase inhibitors (TKIs) for advanced non-small cell lung cancer (aNSCLC) in developed countries. However, its cost-effectiveness in developing countries remains uncertain.

Objective

The aim was to conduct a cost-utility analysis comparing sequential NGS testing with the current approach of single epidermal growth factor receptor (EGFR) testing combined with first-line targeted therapy, as implemented under Thailand's Universal Health Coverage scheme for aNSCLC.

Method

Hybrid decision tree and Markov models were developed to estimate the lifetime costs and quality-adjusted life years (QALYs) associated with each strategy. The models simulate cohorts of aNSCLC patients who receive platinum-based chemotherapy or TKIs based on identified gene alterations. Patients enter the model at 60 years of age. The incremental cost-effectiveness ratio (ICER) was computed from a societal perspective. The analysis employed a lifetime horizon and discounted costs and outcomes at a rate of 3%. Furthermore, uncertainty and scenario analyses were conducted.

Findings

A sequential NGS testing strategy could identify an additional 19% of patients with biomarker-positive findings who subsequently received biomarker-driven targeted therapy compared to a single EGFR testing strategy. The number needed to screen to identify a single gene mutation and administer first-line TKI was six for the sequential NGS testing strategy. Compared to the single EGFR testing, the ICER of the sequential NGS testing strategy was 1,851,150 THB/QALY (US$51,335). At a willingness-to-pay threshold of 160,000 THB/QALY (US$4437), the single EGFR testing strategy demonstrated 100% cost-effectiveness. In contrast, the sequential NGS testing was not deemed cost-effective. The sensitivity of the ICER was influenced by the overall survival rates associated with anaplastic lymphoma kinase (ALK) inhibitors and platinum-based chemotherapy.

Interpretation

Sequential NGS testing identified a greater number of patients with aNSCLC eligible for targeted therapies, resulting in improved survival rates and enhanced QALYs compared to single EGFR testing. However, in the context of Thailand, sequential NGS testing was not cost-effective. The single EGFR testing strategy emerged as the most cost-effective option for guiding first-line targeted therapy.

背景:在发达国家,连续下一代测序(NGS)测试已经证明了在指导使用酪氨酸激酶抑制剂(TKIs)治疗晚期非小细胞肺癌(aNSCLC)的靶向治疗方面的成本效益。然而,其在发展中国家的成本效益仍然不确定。目的:目的是进行成本-效用分析,比较序贯NGS检测与目前的单表皮生长因子受体(EGFR)检测结合一线靶向治疗的方法,后者是在泰国的aNSCLC全民健康覆盖计划下实施的。方法:采用混合决策树和马尔可夫模型来估计与每种策略相关的生命周期成本和质量调整寿命年(QALYs)。该模型模拟了基于已鉴定基因改变接受铂类化疗或TKIs的aNSCLC患者队列。患者在60岁时进入模型。增量成本效益比(ICER)是从社会角度计算的。该分析采用了生命周期,并以3%的比率贴现成本和结果。此外,还进行了不确定性和情景分析。研究结果:与单一EGFR检测策略相比,序列NGS检测策略可以额外识别出19%的生物标志物阳性患者,这些患者随后接受了生物标志物驱动的靶向治疗。序列NGS检测策略需要筛选确定单个基因突变并给予一线TKI的数量为6。与单一EGFR检测相比,序列NGS检测策略的ICER为1,851,150 THB/QALY(51,335美元)。在16万THB/QALY(4437美元)的支付意愿阈值下,单一EGFR检测策略显示出100%的成本效益。相比之下,连续的NGS测试被认为不具有成本效益。ICER的敏感性受间变性淋巴瘤激酶(ALK)抑制剂和铂基化疗相关的总生存率的影响。解释:与单一EGFR检测相比,序贯NGS检测确定了更多适合靶向治疗的aNSCLC患者,从而提高了生存率和QALYs。然而,在泰国的情况下,连续的NGS测试并不具有成本效益。单一EGFR检测策略成为指导一线靶向治疗的最具成本效益的选择。
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引用次数: 0
Predicting Health Utilities Using Health Administrative Data: Leveraging Survey-linked Health Administrative Data from Ontario, Canada 使用健康管理数据预测健康效用:利用来自加拿大安大略省的与调查相关的健康管理数据。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-02-06 DOI: 10.1007/s40258-025-00947-y
Yue Niu, Nazire Begen, Guangyong Zou, Sisira Sarma

Background

The quality-adjusted life year (QALY) is widely used to measure health outcome that combines the length of life and health-related quality of life (HRQoL). To be a reliable QALY measure, HRQoL measurements with a preference-based scoring algorithm need to be converted into health utilities on a scale from zero (dead) to one (perfect health). However, preference-based health utility data are often not available. We address this gap by developing a predictive model for health utilities.

Objectives

To develop a predictive model for health utilities using available demographic and morbidity variables in a health administrative dataset for non-institutionalised populations in Ontario, Canada.

Methods

The data were obtained from the 2009 to 2010 Canadian Community Health Survey containing Health Utilities Index Mark3 (HUI3), a generic multi-attribute preference-based health utility instrument linked with Ontario health administrative (OHA) data that were collected for administrative or billing purposes for patient encounters with the health care system. We employed four regression models (linear, Tobit, single-part beta mixture, and two-part beta mixture) and a calibration technique to identify the best-fit regression model.

Results

Our findings indicate that the two-part beta mixture model is the best-fit for predicting health utilities in the OHA data. The proposed predictive model reflects the original distribution of HUI3 in the population.

Conclusion

Our proposed predictive model generates reasonably accurate health utility predictions from OHA data. Our model-based prediction approach is a useful strategy for real-world applications, particularly when preference-based utility data are unavailable.

背景:质量调整生命年(QALY)被广泛用于衡量生命长度和健康相关生活质量(HRQoL)相结合的健康结局。要成为可靠的QALY度量,使用基于偏好的评分算法的HRQoL度量需要转换为从0(死亡)到1(完美健康)的健康效用。然而,基于偏好的健康效用数据往往无法获得。我们通过开发卫生公用事业预测模型来解决这一差距。目的:利用加拿大安大略省非机构人口的卫生管理数据集中现有的人口统计学和发病率变量,为卫生事业开发一个预测模型。方法:数据来自2009年至2010年加拿大社区卫生调查,该调查包含健康效用指数Mark3 (HUI3),这是一种基于多属性偏好的通用健康效用工具,与安大略省卫生管理(OHA)数据相关联,这些数据是为管理或计费目的收集的,用于患者与卫生保健系统的接触。我们采用了四种回归模型(线性、Tobit、单部分β混合和两部分β混合)和校准技术来确定最适合的回归模型。结果:我们的研究结果表明,两部分贝塔混合模型最适合预测OHA数据中的健康效用。所提出的预测模型反映了HUI3在种群中的原始分布。结论:我们提出的预测模型根据OHA数据产生了相当准确的健康效用预测。我们基于模型的预测方法对于实际应用程序是一种有用的策略,特别是当基于偏好的效用数据不可用时。
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引用次数: 0
Health Economic Evaluations of Obesity Interventions: Expert Views on How We Can Identify, Interpret, Analyse and Translate Effects 肥胖干预措施的健康经济评估:如何识别、解释、分析和转化效果的专家观点。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2025-02-01 DOI: 10.1007/s40258-025-00946-z
Samira B. Jabakhanji, Gintare Valentelyte, Fabian Manke-Reimers, Vidar Halsteinli, Rønnaug Ødegård, Adam Martin, Grace O’Malley, Jan Sorensen, Emilia Hagman

Economic evaluations of obesity interventions are critical to informing policymakers and clinical practitioners about best-value prevention and treatment interventions. However, existing studies often fail to measure appropriate outcomes over sufficient time periods and to adequately address the complexity of data, environments and outcomes. An international, multidisciplinary workshop in Ireland (May 2023) addressed these issues through scientific presentations on obesity modelling, group discussions and interactive small-group exercises. Nineteen presenters and participants co-created a list of research needs, priorities and strategies for the long-term study of obesity and its complications. To support availability of relevant outcome and cost data for health economic analyses, participants highlighted a need to define standards for data collection, data sharing, modelling, and integrating a systems perspective. For example, regarding data collection, careful consideration must be given to selecting valid and relevant health-related outcomes for determining future health risk. Although these issues have been previously highlighted, they remain critical barriers to comprehensive economic obesity studies. To identify best-value obesity interventions, researchers should prioritise strategies to overcome these barriers. This includes early engagement with multidisciplinary stakeholders to integrate diverse perspectives. Developing infrastructure to support international collaborations between researchers, policymakers and patient representatives was also recommended. Additionally, establishing best-practice guidelines could help researchers navigate the complexities of obesity data, environments and outcomes, particularly in data-scarce research environments. The creation of a core outcomes set for obesity would standardise measures for economic evaluations, thereby facilitating more robust cross-country comparisons of intervention effects and improving the evidence base and overall quality of future obesity research.

肥胖干预措施的经济评估对于告知决策者和临床从业人员关于最有价值的预防和治疗干预措施至关重要。然而,现有的研究往往未能在足够的时间内衡量适当的结果,也未能充分处理数据、环境和结果的复杂性。爱尔兰(2023年5月)的一个国际多学科研讨会通过肥胖模型、小组讨论和互动小组练习的科学介绍解决了这些问题。19位演讲者和参与者共同制定了一份关于肥胖及其并发症长期研究的研究需求、优先事项和策略的清单。为了支持获得卫生经济分析所需的相关结果和成本数据,与会者强调需要从系统的角度确定数据收集、数据共享、建模和整合的标准。例如,在数据收集方面,必须仔细考虑选择有效和相关的健康相关结果,以确定未来的健康风险。尽管这些问题之前已经被强调过,但它们仍然是全面经济肥胖研究的关键障碍。为了确定最有价值的肥胖干预措施,研究人员应该优先考虑克服这些障碍的策略。这包括尽早与多学科利益相关者接触,以整合不同的观点。还建议发展基础设施以支持研究人员、决策者和患者代表之间的国际合作。此外,建立最佳实践指南可以帮助研究人员驾驭肥胖数据、环境和结果的复杂性,特别是在数据稀缺的研究环境中。建立肥胖的核心结果集将使经济评估的衡量标准标准化,从而促进对干预效果进行更有力的跨国比较,并改善证据基础和未来肥胖研究的总体质量。
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引用次数: 0
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Applied Health Economics and Health Policy
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