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Exome Sequencing in the Diagnostic Pathway for Suspected Rare Genetic Diseases: Does the Order of Testing Affect its Cost-Effectiveness? 疑似罕见遗传病诊断途径中的外显子组测序:检测顺序会影响成本效益吗?
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-30 DOI: 10.1007/s40258-024-00936-7
Koen Degeling, Toni Tagimacruz, Karen V MacDonald, Trevor A Seeger, Katharine Fooks, Viji Venkataramanan, Kym M Boycott, Francois P Bernier, Roberto Mendoza-Londono, Taila Hartley, Robin Z Hayeems, Deborah A Marshall

Background: Patients with suspected rare diseases often experience lengthy and uncertain diagnostic pathways. This study aimed to estimate the cost-effectiveness of exome sequencing (ES) in different positions in the diagnostic pathway for patients suspected of having a rare genetic disease.

Methods: Data collected retrospectively from 305 patients suspected of having a rare genetic disease (RGD), who received clinical-grade ES and participated in the Canadian multicentre Care4Rare-SOLVE study, informed a discrete event simulation of the diagnostic pathway. We distinguished between tests that can lead to the diagnosis of a specific RGD ('indicator tests') and more routine non-RGD diagnostic tests ('non-indicator tests'). Five strategies were considered: no-ES, and ES as 1st, 2nd, 3rd, or 4th test (Tier 1, Tier 2, Tier 3, and Tier 4, respectively), where ES was the final test in the diagnostic pathway if included. Outcomes included the diagnostic yield, time-to-diagnosis, time on the diagnostic pathway, and test costs for each strategy. The cost-effectiveness analysis from a Canadian healthcare system perspective was conducted with diagnostic yield as the primary outcome of interest. Probabilistic analyses and expert-defined scenario analyses quantified uncertainty.

Results: Implementing ES increases the diagnostic yield by 16 percentage points from 20% with no-ES to 36%. Exome sequencing, as the first test (Tier 1), resulted in the shortest time to a diagnosis and the lowest testing cost. Mean testing costs per patient were CAD4347 (95% CI 3925, 4788) for no-ES, CAD2458 (95% CI 2406, 2512) for Tier 1, CAD3851 (95% CI 3684, 4021) for Tier 2, CAD5246 (95% CI 4956, 5551) for Tier 3 and CAD6422 (95% CI 5954, 6909) for Tier 4, with Tier 1 having the highest diagnostic yield at the lowest cost. The scenario analyses yielded results consistent with those of the base case.

Conclusions: Implementing ES to diagnose patients suspected of having a RGD can result in a higher diagnostic yield. Although a limitation of our study was that the yield for the non-ES indicator tests was estimated using expert opinion due to a lack of available data, the results underscore the value of ES as a first-line diagnostic test, offering reduced time to diagnosis and lower overall testing costs.

背景:疑似罕见病的患者往往经历漫长而不确定的诊断过程。本研究旨在评估外显子组测序(ES)在疑似患有罕见遗传病的患者诊断途径中不同位置的成本效益。方法:回顾性收集305名疑似患有罕见遗传病(RGD)的患者的数据,这些患者接受了临床级ES,并参加了加拿大多中心Care4Rare-SOLVE研究,提供了诊断途径的离散事件模拟。我们区分了可导致诊断特定RGD的测试(“指标测试”)和更常规的非RGD诊断测试(“非指标测试”)。考虑了五种策略:无ES, ES作为第一,第二,第三或第四测试(分别为1级,2级,3级和4级),如果包括ES,则是诊断途径中的最终测试。结果包括诊断率、诊断时间、诊断途径时间和每种策略的检测成本。从加拿大医疗保健系统的角度进行成本效益分析,诊断率作为主要结果感兴趣。概率分析和专家定义的情景分析量化了不确定性。结果:实施ES将诊断率从无ES的20%提高到36%,提高了16个百分点。外显子组测序作为第一种检测方法(一级),诊断时间最短,检测成本最低。每位患者的平均检测成本为no-ES的CAD4347 (95% CI 3925, 4788), 1级的CAD2458 (95% CI 2406, 2512), 2级的CAD3851 (95% CI 3684, 4021), 3级的CAD5246 (95% CI 4956, 5551)和4级的CAD6422 (95% CI 5954, 6909),其中1级以最低的成本具有最高的诊断率。情景分析产生的结果与基本情况一致。结论:采用ES诊断疑似RGD的患者可获得更高的诊断率。虽然我们研究的一个局限性是,由于缺乏可用数据,非ES指标测试的产量是使用专家意见来估计的,但结果强调了ES作为一线诊断测试的价值,缩短了诊断时间,降低了总体测试成本。
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引用次数: 0
Cost of Carbon in the Total Cost of a Healthcare Procedure: Example of Micro-Costing Study in a French Setting 医疗程序总成本中的碳成本:法国微观成本研究实例。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-30 DOI: 10.1007/s40258-024-00933-w
Paul-Simon Pugliesi, Hervé Frick, Stéphanie Guillot, Karine Ferrare, Catherine Renzullo, Alexandre Benoist, Serge Ribes, Guillaume Beltramo, Thomas Maldiney, Romain Ter Schiphorst, Caroline Abdul Malak, Adrien Bevand, Laurie Marrauld, Catherine Lejeune

Background

Economic evaluation aims to compare the costs and results of health strategies to inform public decision making. Although sometimes suggested, until now no national evaluation agency has recommended formally incorporating the cost of greenhouse gas (GHG) emissions generated by health interventions into the estimation of healthcare costs.

Objective

The objective of this study was to test and discuss the feasibility of estimating and including the contribution of GHG emissions cost to the total cost of a surgical intervention, with the example of robot-assisted total knee arthroplasty (RTA), using a micro-costing approach.

Methods

The study was conducted in June 2022 at the William Morey Hospital (France). Data regarding all of the resources (labor, medical equipment, consumables), as well as energy consumption, staff commuting and waste treatment were collected and valued from the hospital point of view. Greenhouse gas emissions were valued using a cost-effectiveness approach. Several sensitivity analyses were performed.

Results

The mean cost per patient of an RTA was estimated to be €4755.65, of which €152.64 (3.21 %) would be attributable to GHG emissions. The contribution of GHG emissions in the overall cost of a health intervention was highly dependent on the convention used for the price of carbon.

Conclusion

Despite persistent theoretical and practical challenges, adding the estimation of GHG emission costs in the economic evaluation of health interventions may provide institutional decision makers with information that allows them to allocate the public healthcare resources more efficiently.

背景:经济评价的目的是比较卫生战略的成本和结果,为公共决策提供信息。虽然有时会提出建议,但迄今为止没有任何国家评估机构正式建议将卫生干预措施产生的温室气体排放成本纳入卫生保健费用的估计。目的:本研究的目的是以机器人辅助全膝关节置换术(RTA)为例,采用微观成本计算方法,测试和讨论估算和包括温室气体排放成本对手术干预总成本贡献的可行性。方法:研究于2022年6月在法国William Morey医院进行。收集了有关所有资源(劳动力、医疗设备、消耗品)以及能源消耗、工作人员通勤和废物处理的数据,并从医院的角度对其进行了评估。使用成本效益方法评估温室气体排放。进行了一些敏感性分析。结果:RTA每位患者的平均费用估计为4755.65欧元,其中152.64欧元(3.21%)将归因于温室气体排放。温室气体排放对卫生干预总成本的贡献在很大程度上取决于用于碳定价的公约。结论:尽管存在理论和实践上的挑战,但在卫生干预措施的经济评估中加入温室气体排放成本的估计可能为机构决策者提供信息,使他们能够更有效地分配公共卫生资源。
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引用次数: 0
The Gift of Time, How Do I Want to Spend It? Exploring Preferences for Time Allocation Among Women with and without a Breast Cancer Diagnosis 时间的礼物,我想如何使用它?探讨有和没有乳腺癌诊断的妇女的时间分配偏好。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-28 DOI: 10.1007/s40258-024-00934-9
Ni Gao, Mandy Ryan, Suzanne Robinson, Richard Norman

Background

Women’s preferences for time allocation reveal how they would like to prioritise market work, family life, and other competing activities. Whilst preferences may not always directly translate to behaviour, they are an important determinant of intention to act.

Objective

We present the first study to apply a discrete choice experiment (DCE) to investigate time allocation preferences among women diagnosed with breast cancer and women without a cancer diagnosis.

Methods

Time attributes were paid work, household work, caregiving, passive leisure and physical leisure. An income attribute was included to estimate the monetary value of time. The study took place in the UK and the DCE was completed by 191 women diagnosed with breast cancer and 347 women without a cancer diagnosis. Responses were analysed using a mixed logit model.

Results

Women diagnosed with breast cancer have stronger positive preferences for daily activities compared to women without a cancer diagnosis. They require less compensation (not significant) for an additional hour of paid work (£5.58), household work (£7.92), and caregiving (£8.53). They are willing to pay more for an additional hour of passive leisure (£1.70, not significant) and physical leisure (£13.66, significant).

Conclusion

The heterogeneous preferences for time allocation among women have policy implications and are significant for welfare analysis.

背景:女性对时间分配的偏好揭示了她们如何优先考虑市场工作、家庭生活和其他竞争性活动。虽然偏好可能并不总是直接转化为行为,但它们是行动意图的重要决定因素。目的:我们提出了第一个应用离散选择实验(DCE)来调查诊断为乳腺癌的妇女和没有癌症诊断的妇女的时间分配偏好的研究。方法:时间属性为有偿工作、家务劳动、照料、被动休闲和体力休闲。收入属性是用来估计时间的货币价值的。这项研究在英国进行,191名确诊为乳腺癌的女性和347名未确诊为癌症的女性完成了DCE。使用混合logit模型分析响应。结果:与未患乳腺癌的女性相比,被诊断为乳腺癌的女性对日常活动有更强的积极偏好。他们对额外的有偿工作(5.58英镑)、家务(7.92英镑)和护理(8.53英镑)要求的报酬较少(不显著)。他们愿意为额外一小时的被动休闲(1.70英镑,不显著)和体力休闲(13.66英镑,显著)支付更多费用。结论:女性对时间分配的异质性偏好具有政策意义,对福利分析具有重要意义。
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引用次数: 0
Systematic Literature Review of Access Pathways to Drugs for Patients with Rare Diseases 罕见病患者药物获取途径的系统文献综述。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-28 DOI: 10.1007/s40258-024-00939-4
Constanza Vargas, Richard De Abreu Lourenco, Manuel Espinoza, Stephen Goodall

Objective

This article reviews the assessment pathways that have been implemented worldwide to facilitate access to drugs for patients with rare diseases.

Methods

The Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) guidelines were used to conduct a systematic literature review. The Ovid (Embase/MEDLINE), Cochrane, Web of Science, Econlit, National Institute of Health Research, Centre for Reviews and Dissemination, and International Network of Agencies for Health Technology Assessment databases were searched. Two independent reviewers screened all titles and abstracts; one reviewer did the full-text review and data extraction. Data were extracted on study general characteristics, general aspects of rare diseases, source of funding, allocation of public resources (e.g., use of health technology assessment), and pricing strategies. Assessment pathways were classified as: (1) separate processes; (2) exception to standard process; (3) standard process with no change; and (4) alternative process. Each assessment pathway was characterized based on its unique characteristics specific to rare diseases focusing on whether they targeted specific aspects of the process, utilized particular methodologies during the evaluation of the evidence, or considered specific attributes in the recommendation.

Results

A total of 5604 unique citations were screened and 158 were included for data extraction. Sixty-one assessment pathways were identified in 43 countries, categorized as separate processes (37%), exceptions to standard processes (32%), standard processes with no changes (26%), and alternative processes (5%). Some countries (10/43; 23%) have more than one assessment pathway available. Assessment pathways varied in their inclusion of a health technology assessment, source of funding, consideration of uncertainty, and pricing strategies.

Conclusions

The diversity of assessment pathways reflects the complexity of addressing access to treatments for rare diseases. Furthermore, most assessment pathways are from high-income countries; therefore, there is less clarity on what is happening in low- and middle-income countries.

目的:本文综述了世界范围内为促进罕见病患者获得药物而实施的评估途径。方法:采用系统评价和荟萃分析首选报告项目(PRISMA)指南进行系统文献综述。检索了Ovid (Embase/MEDLINE)、Cochrane、Web of Science、Econlit、National Institute of Health Research、Centre for Reviews and Dissemination和国际Network of Agencies for Health Technology Assessment数据库。两位独立审稿人对所有标题和摘要进行了筛选;一位审稿人进行全文审查和数据提取。提取了关于研究的一般特征、罕见病的一般方面、资金来源、公共资源的分配(例如卫生技术评估的使用)和定价策略的数据。评估途径分为:(1)独立过程;(二)标准程序的例外;(3)无变化的标准工艺;(4)替代工艺。每一种评估途径都是根据其特有的罕见疾病特征来确定其特征的,重点是它们是否针对该过程的特定方面,在评估证据时是否使用了特定方法,或在建议中是否考虑了特定属性。结果:共筛选到5604条独特引文,其中158条纳入数据提取。在43个国家确定了61种评估途径,分为单独的过程(37%)、标准过程的例外(32%)、没有变化的标准过程(26%)和替代过程(5%)。一些国家(10/43;23%)有一个以上的评估途径。评估途径在包括卫生技术评估、资金来源、考虑不确定性和定价策略方面各不相同。结论:评估途径的多样性反映了解决罕见病治疗可及性问题的复杂性。此外,大多数评估途径来自高收入国家;因此,低收入和中等收入国家的情况不太清楚。
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引用次数: 0
Cost-Effective and Sustainable Drug Use in Hospitals: A Systematic and Practice-Based Approach 医院中具有成本效益和可持续的药物使用:系统和基于实践的方法。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-19 DOI: 10.1007/s40258-024-00937-6
Michiel Zietse, Shannon L. van der Zeeuw, Anne-Sophie Klein Gebbink, Annemarie C. de Vries, Marie-Rose B. S. Crombag, Roelof W. F. van Leeuwen, Maaike J. Hoedemakers

Background and Objective

Rising healthcare costs challenge the financial sustainability of healthcare systems. Interventional pharmacoeconomics has emerged as a vital discipline to improve the cost-effective and sustainable use of drugs in clinical practice. However, current efforts are often fragmented, highlighting the need for an integrated hospital-wide approach. This study aimed to develop a scalable framework to systematically identify and implement cost-effective and sustainable drug use practices in hospitals.

Methods

This study was conducted at the Erasmus University Medical Centre in Rotterdam between December 2022 and July 2023. A novel ‘8-Step Efficiency Model’ was designed to systematically identify and evaluate strategies for cost-effective and sustainable drug use. The process involved identifying high-expenditure drugs, systematically assessing these drugs using the Efficiency Model, and conducting a multi-disciplinary evaluation of the proposed cost-effectiveness strategies.

Results

The study assessed 39 high-cost drugs, representing 57% of the Dutch national expensive drug expenditure in 2021. Initiatives for enhancing cost-effectiveness and sustainability were identified or developed for 27 out of the 39 assessed drugs (51% of the national drug expenditure in 2021). Case examples of infliximab (e.g., wastage prevention) and intravenous immunoglobulins (e.g., lean body weight dosing) illustrate practical applications of the framework, resulting in substantial cost savings and improved sustainability.

Conclusions

This study presents a systematic scalable model for enhancing the cost-effectiveness of high-expenditure drugs in hospital settings. This approach not only addresses financial sustainability but also promotes the quality of patient care and sustainable drug use. This model could serve as a generic blueprint for other institutions to identify and implement cost-effective and sustainable drug use strategies.

背景与目的:不断上升的医疗成本对医疗系统的财务可持续性提出了挑战。介入药物经济学已成为提高临床实践中药物成本效益和可持续使用的重要学科。然而,目前的努力往往是分散的,这突出表明需要采取全医院的综合办法。本研究旨在开发一个可扩展的框架,以系统地确定和实施具有成本效益和可持续的医院用药做法。方法:本研究于2022年12月至2023年7月在鹿特丹伊拉斯谟大学医学中心进行。设计了一个新的“8步效率模型”,以系统地确定和评估具有成本效益和可持续的药物使用战略。该过程包括确定高支出药物,使用效率模型系统地评估这些药物,并对拟议的成本效益战略进行多学科评估。结果:该研究评估了39种高成本药物,占2021年荷兰国家昂贵药物支出的57%。为39种评估药物中的27种(占2021年国家药物支出的51%)确定或制定了提高成本效益和可持续性的举措。英夫利昔单抗(例如,预防浪费)和静脉注射免疫球蛋白(例如,瘦体重剂量)的案例说明了该框架的实际应用,从而节省了大量成本并提高了可持续性。结论:本研究提出了一个系统的可扩展模型,以提高医院环境中高费用药物的成本效益。这种方法不仅解决了财务可持续性问题,而且还促进了患者护理质量和可持续用药。这一模式可作为其他机构确定和执行具有成本效益和可持续的药物使用战略的通用蓝图。
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引用次数: 0
Quality-Adjusted Life Expectancy Norms Based on the EQ-5D-5L and SF-6Dv2 for China 基于 EQ-5D-5L 和 SF-6Dv2 的中国预期寿命质量调整规范。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-13 DOI: 10.1007/s40258-024-00925-w
Shitong Xie, Xiaoning He, Weihua Guo, Jing Wu

Objectives

Quality-adjusted life expectancy (QALE) norms reflect the normative profiles or reference data of QALE of the general population and provide a meaningful anchor for comparison to inform healthcare decision-making. This study aimed to develop the QALE norms for the Chinese population by using a representative dataset of health utility values collected using the EQ-5D-5L and short-form 6-dimension version 2 (SF-6Dv2) instruments.

Methods

Age-specific population norms of health utility values calculated using the EQ-5D-5L and SF-6Dv2 were used. Both utility norms were combined with the latest version of the National Life Tables of China published in 2021 to calculate QALE estimates on the basis of age, sex, and urban/rural residence area. QALE estimates were further discounted using 1.5%, 3.5%, 5.0%, and 8.0% discount rates.

Results

When using the health utility values evaluated by the SF-6Dv2, the QALE at age 0 years was 66.34 years at the discount rate of 0% and 16.65 years at the discount rate of 5%. For the EQ-5D-5L, the QALE at age 0 years was 76.50 years at the discount rate of 0% and 19.45 years at the discount rate of 5%. At birth, females exhibited a higher QALE, while the difference between females and males initially increased before subsequently declining overtime, ultimately resulting in females having a lower QALE. Rural population had a monotonically lower QALE than urban population.

Conclusion

This study constructed age-stratified QALE norms for the Chinese population categorized by sex and residence area using mortality data alongside corresponding health utility values derived from the EQ-5D-5L and SF-6Dv2.

目的:质量调整预期寿命(QALE)规范反映了一般人群QALE的规范性概况或参考数据,并为比较提供了有意义的锚点,以告知医疗保健决策。本研究旨在通过使用EQ-5D-5L和SF-6Dv2短格式6维仪器收集的具有代表性的健康效用值数据集,制定中国人群的QALE规范。方法:采用EQ-5D-5L和SF-6Dv2计算的年龄人群健康效用值规范。这两个效用标准与2021年发布的最新版《中国国家生命表》相结合,以年龄、性别和城乡居住区域为基础计算QALE估值。QALE估计进一步使用1.5%、3.5%、5.0%和8.0%的贴现率进行贴现。结果:使用SF-6Dv2评估的健康效用值时,0岁时的QALE在贴现率为0%时为66.34岁,贴现率为5%时为16.65岁。EQ-5D-5L在0岁时的QALE在贴现率为0%时为76.50岁,在贴现率为5%时为19.45岁。在出生时,雌性表现出更高的QALE,而雌性和雄性之间的差异最初增加,随后随着时间的推移而下降,最终导致雌性的QALE降低。农村人口的QALE单调低于城市人口。结论:本研究利用死亡率数据以及EQ-5D-5L和SF-6Dv2得出的相应健康效用值,构建了按性别和居住区域分类的中国人口年龄分层的QALE规范。
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引用次数: 0
Value is Gendered: The Need for Sex and Gender Considerations in Health Economic Evaluations 价值是性别化的:在卫生经济评估中需要考虑性别和性别因素。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-12 DOI: 10.1007/s40258-024-00930-z
Martina Mchenga, Lavanya Vijayasingham, Rajalakshmi RamPrakash, Michelle Remme

Economic evaluations play a crucial role in health resource allocation by assessing the costs and effects of various interventions. However, existing methodologies often overlook significant differences related to sex and gender, leading to a ‘blind spot’ in understanding patient heterogeneity. This paper highlights how biological and social factors influence costs and health outcomes differently for women, emphasising the need for a more explicit consideration of these differences in economic evaluations to ensure efficient and equitable resource allocation. The paper is structured to first outline how sex and gender factors impact costs and outcomes. It then identifies biases in current economic evaluation methods and practices, using real-world examples to illustrate the implications of these biases on policymaking and health equity. Notably, we argue that neglecting gender considerations can lead to inefficiencies and inequities in healthcare resource distribution. Key areas of gender bias include the estimation of productivity losses, quality of life variations and the secondary household effects of interventions. The analysis reveals that women often face higher healthcare costs and experience different health outcomes due to systemic biases in treatment and care. The paper concludes with practical recommendations for analysts, decision makers and research funders, advocating for the integration of sex and gender-responsive methodologies in health economic evaluations. Ultimately, this work calls for a paradigm shift in health economics to better reflect the complexities of sex and gender and improve health outcomes for all.

经济评价通过评估各种干预措施的成本和效果,在卫生资源分配中起着至关重要的作用。然而,现有的方法往往忽略了与性别和性别相关的显著差异,导致在理解患者异质性方面存在“盲点”。本文强调了生物和社会因素对妇女的成本和健康结果的不同影响,强调需要在经济评估中更明确地考虑到这些差异,以确保有效和公平的资源分配。本文的结构首先概述了性别和性别因素如何影响成本和结果。然后,它确定了当前经济评估方法和实践中的偏见,并使用现实世界的例子来说明这些偏见对决策和卫生公平的影响。值得注意的是,我们认为忽视性别因素可能导致医疗资源分配效率低下和不公平。性别偏见的主要领域包括估计生产力损失、生活质量变化和干预措施对家庭的次要影响。分析显示,由于治疗和护理方面的系统性偏见,妇女往往面临更高的医疗保健费用,并经历不同的健康结果。该文件最后为分析人员、决策者和研究资助者提出了实际建议,倡导将性别和促进性别平等的方法纳入卫生经济评价。最终,这项工作呼吁卫生经济学的范式转变,以更好地反映性别和社会性别的复杂性,并改善所有人的健康结果。
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引用次数: 0
Assessing the Direct Impact of Death on Discrete Choice Experiment Utilities 评估死亡对离散选择实验效用的直接影响。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-06 DOI: 10.1007/s40258-024-00929-6
Hossein Ameri, Thomas G. Poder

Background

The dead state can affect the value sets derived from discrete choice experiments (DCEs). Our aim was to empirically assess the direct impact of the immediate death state on health utilities using discrete choice experiment with time (DCETTO).

Methods

A sample of the general population in Quebec, Canada, completed two approaches: DCETTO followed by a best-worst scaling with time (BWSTTO) (hereafter referred to as DCEBWS), versus DCETTO followed by the dominated option and the immediate death state (hereafter referred to as DCEDOD), both designed with the SF-6Dv2. In DCEBWS, all participants first completed 10 DCETTO choices (i.e., option A vs B), followed by 3 BWSTTO. In DCEDOD, the same participants first completed the same 10 DCETTO choices, followed by a repeated choice between the dominated option (i.e., A or B) and the immediate death state. A conditional logit model was used to estimate value sets. The performance of models was assessed using goodness of fit using Bayesian information criterion, parameters’ logical consistency, and levels’ significance. The direct impact of the death state on DCE latent utilities was evaluated by examining the magnitude of coefficients, assessing the agreement among the value sets estimated by DCETTO with DCEBWS and with DCEDOD using Bland-Altman plots, the proportion of worst-than-dead (WTD) health states, and analyzing the range of estimated values.

Results

From 398 participants, a total of 348 participants were included for final analysis. The number of parameters with illogical consistency and non-significant coefficients was lower in DCEBWS. The observed consistency in the relative importance of dimensions across all approaches suggests a stable and reliable ranking. The utility range for DCEDOD (− 0.921 to 1) was narrower than for DCETTO (− 1.578 to 1) and DCEBWS (− 1.150 to 1). The DCEDOD estimated a lower percentage of WTD health states (20.01 %) compared to DCETTO (47.19 %) and DCEBWS (33.73 %). The agreement between DCETTO and DCEBWS was slightly stronger than between DCETTO and DCEDOD, and the mean utility values were higher in DCEDOD than in DCEBWS.

Conclusions

The inclusion of the immediate death state directly within DCE increased utility values. This increase was higher when the immediate death was included in a sequence within a DCETTO (i.e., DCEDOD) than when it was included in a continuum of DCETTO (i.e., DCEBWS). The use of DCEDOD was potentially better suited to incorporate the dead state into a DCE.

背景:死态会影响离散选择实验(dce)的值集。我们的目的是利用时间离散选择实验(DCETTO)实证评估即时死亡状态对医疗效用的直接影响。方法:在加拿大魁北克省的一个普通人群样本中,完成了两种方法:采用SF-6Dv2设计的DCETTO和最佳最差随时间尺度(BWSTTO)(以下简称DCEBWS),以及采用优势选项和立即死亡状态(以下简称DCEDOD)的DCETTO。在DCEBWS中,所有参与者首先完成10个dceto选项(即选项A与选项B),然后完成3个BWSTTO。在DCEDOD中,相同的参与者首先完成相同的10个dceto选项,然后在主导选项(即a或B)和立即死亡状态之间重复选择。使用条件logit模型估计值集。使用贝叶斯信息准则、参数逻辑一致性和水平显著性来评估模型的性能。死亡状态对DCE潜在效用的直接影响是通过检查系数的大小、使用Bland-Altman图评估DCETTO与DCEBWS和DCEDOD估计的值集之间的一致性、比死亡严重的健康状态(WTD)的比例以及分析估计值的范围来评估的。结果:从398名参与者中,共有348名参与者被纳入最终分析。DCEBWS中具有非逻辑一致性和非显著性系数的参数数量较少。在所有方法中观察到的维度相对重要性的一致性表明了一个稳定可靠的排名。DCEDOD的效用范围(- 0.921比1)比DCETTO(- 1.578比1)和DCEBWS(- 1.150比1)窄。与DCETTO(47.19%)和DCEBWS(33.73%)相比,DCEDOD估计的WTD健康状态百分比(20.01%)较低。DCETTO和DCEBWS之间的一致性略强于DCETTO和DCEDOD之间,并且DCEDOD的平均效用值高于DCEBWS。结论:直接将立即死亡状态纳入DCE增加了实用价值。当立即死亡被包括在DCETTO(即DCEDOD)序列中时,这种增加高于被包括在DCETTO连续序列(即DCEBWS)中。使用DCEDOD可能更适合将死状态合并到DCE中。
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引用次数: 0
Acknowledgement to Referees 给推荐人的确认函。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-05 DOI: 10.1007/s40258-024-00931-y
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引用次数: 0
Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA. 在美国评估Etranacogene Dezaparvovec基因治疗血友病B的成本-效果。
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2024-12-02 DOI: 10.1007/s40258-024-00932-x
Jyotirmoy Sarker, Jeffrey A Tice, David M Rind, Surrey M Walton

Background: Hemophilia B, a severe genetic disorder, involves substantial treatment costs and frequent interventions. Etranacogene dezaparvovec (EDZ) is a recently approved gene therapy for hemophilia B.

Objective: This study evaluates the cost-effectiveness of EDZ compared with conventional factor IX (FIX) prophylaxis.

Methods: A semi-Markov model simulated a cohort of adult males with severe hemophilia B to assess the economic impact of EDZ versus FIX prophylaxis over a lifetime horizon from a health system perspective in the USA. Inputs derived from clinical trials included therapy durability and transition probabilities based on Pettersson Scores. Scenario analyses incorporated frameworks suggested by the Institute for Clinical and Economic Review for single or short-term transformative therapies.

Results: Base-case analysis showed that at a cost of US$3.5 million, EDZ led to lifetime cost savings of US$11 million and an additional 0.64 quality-adjusted life years (QALYs) compared with FIX. However, FIX has extremely high annual costs. When annual cost offsets attributed to EDZ were capped at US$150,000, EDZ was found to have a threshold price of US$3.1 million at a willingness-to-pay of US$150,000 per QALY.

Conclusion: EDZ proved to be a dominant strategy over FIX prophylaxis in the base-case scenario, providing large cost savings and slightly better outcomes. The substantial costs associated with FIX are a primary driver behind these results. The introduction of cost-offset caps significantly affects the value-based price of EDZ. Using caps on cost offsets in considering price can help to balance affordability and value in the health system.

背景:血友病B是一种严重的遗传性疾病,需要大量的治疗费用和频繁的干预。dezaparvovec (Etranacogene dezaparvovec, EDZ)是最近批准的一种治疗b型血友病的基因疗法。目的:本研究比较EDZ与传统因子IX (FIX)预防的成本-效果。方法:半马尔可夫模型模拟了一组患有严重B型血友病的成年男性,从美国卫生系统的角度评估EDZ与FIX预防在一生中的经济影响。来自临床试验的输入包括基于Pettersson评分的治疗持久性和转移概率。情景分析纳入了临床和经济评论研究所建议的单一或短期变革性治疗框架。结果:基本案例分析表明,与FIX相比,EDZ以350万美元的成本节省了1100万美元的生命周期成本,并增加了0.64个质量调整生命年(QALYs)。然而,FIX的年成本极高。当归因于EDZ的年度成本抵消上限为15万美元时,EDZ的门槛价格为310万美元,每个QALY的支付意愿为15万美元。结论:在基本病例情况下,EDZ被证明是一种优于FIX预防的主要策略,可以节省大量成本,并且效果略好。与FIX相关的大量成本是这些结果背后的主要驱动因素。成本抵消上限的引入显著影响了经济开发区基于价值的价格。在考虑价格时使用成本抵消上限有助于平衡卫生系统的可负担性和价值。
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引用次数: 0
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Applied Health Economics and Health Policy
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