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Comment on: “Comparison of Caregiver and General Population Preferences for Dependency‑Related Health States” 评论:“护理人员和一般人群对依赖相关健康状态的偏好比较”。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-05-16 DOI: 10.1007/s40258-025-00973-w
Sadia Farhana
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引用次数: 0
Authors’ Reply to Sadia Farhana’s Comment on: “Comparison of Caregiver and General Population Preferences for Dependency-Related Health States” 作者对Sadia Farhana评论的回复:“护理人员和一般人群对依赖相关健康状态的偏好的比较”。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-05-16 DOI: 10.1007/s40258-025-00974-9
Eva Rodríguez-Míguez, Antonio Sampayo
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引用次数: 0
The Cost Effectiveness of Elective Surgical Procedures with Longer NHS Waiting Lists: A Targeted Review 选择性外科手术的成本效益与较长的NHS等待名单:有针对性的审查。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-05-09 DOI: 10.1007/s40258-025-00975-8
Laura A. Trigg, Caroline Farmer, Madhusubramanian Muthukumar, Edward C. F. Wilson, Alan Lovell, Dawn Lee

Objectives

Our aim was to review the evidence for the cost effectiveness of elective surgeries with long waiting lists within the NHS in England. This is to inform understanding of national spending priorities in the context of significant demand for elective surgeries and to inform the debate on appropriate cost-effectiveness thresholds across healthcare decision making.

Methods

We conducted a targeted literature review to identify published cost-effectiveness analyses for nine elective procedures with long waiting lists in the NHS, selected based on previous reviews. These were percutaneous coronary intervention (PCI), coronary artery bypass graft surgery (CABG), hysterectomy, cholecystectomy, knee replacement, groin hernia repair, hip replacement, prostatectomy, and cataract surgery. We made comparisons adjusted for currency and price year (2024).

Results

We identified 21 evaluations; in these, the cost effectiveness of surgeries was compared with no surgery (n = 9), medical management (n = 5), and between early and delayed surgery (n = 10). The evaluations reported that almost all procedures would be considered cost effective yielding incremental cost-effectiveness ratios (ICERs) below £20,000 per quality-adjusted life-year gained. Cholecystectomy, prostatectomy, hip and knee replacement surgeries were associated with ICERs of between £5,000 and £10,000.

Conclusions

These findings offer insights for policymakers on optimising finite healthcare resources, particularly post-COVID-19, with surgical waiting lists a priority for the NHS. Prioritising these elective procedures is likely to be a highly cost-effective use of NHS resources. Allocation of investment to areas that are more cost effective than others is likely to increase the efficiency of the NHS, resulting in a net health gain compared with the reimbursement of less cost-effective interventions.

目的:我们的目的是回顾证据的成本效益选择性手术与长等待名单在英国国民健康保险制度。这是为了了解在选择性手术需求巨大的背景下,国家支出的优先事项,并为医疗保健决策中适当的成本效益阈值的辩论提供信息。方法:我们进行了一项有针对性的文献综述,以确定已发表的成本效益分析,其中9项选择性手术在NHS中有很长的等待名单,选择基于先前的综述。包括经皮冠状动脉介入治疗(PCI)、冠状动脉旁路移植术(CABG)、子宫切除术、胆囊切除术、膝关节置换术、腹股沟疝修补术、髋关节置换术、前列腺切除术和白内障手术。我们进行了汇率和价格调整后的比较(2024年)。结果:我们确定了21个评价;在这些研究中,比较了手术与不手术(n = 9)、医疗管理(n = 5)以及早期和延迟手术(n = 10)的成本效益。评估报告称,几乎所有程序都被认为具有成本效益,每个质量调整生命年的增量成本效益比(ICERs)低于20,000英镑。胆囊切除术、前列腺切除术、髋关节和膝关节置换手术与ICERs相关的费用在5000英镑到1万英镑之间。结论:这些发现为政策制定者提供了优化有限医疗资源的见解,特别是在covid -19之后,手术等待名单是NHS的优先事项。优先考虑这些选择性程序可能是NHS资源的高成本效益使用。将投资分配给比其他领域更具成本效益的领域,可能会提高国民保健制度的效率,与报销成本效益较低的干预措施相比,产生净健康收益。
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引用次数: 0
Antimicrobial Resistance (AMR) Development Map: A Conceptual Map and a Tool to Support Economic Evaluation of AMR Interventions 抗菌素耐药性(AMR)发展地图:支持AMR干预措施经济评估的概念地图和工具。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-05-09 DOI: 10.1007/s40258-025-00969-6
Kristina Aluzaite, Marta O. Soares, Catherine Hewitt, William Hope, Julie Robotham, Beth Woods

Introduction

Antimicrobial resistance (AMR) is a complex, inter-sectoral and international problem. Economic evaluation (EE) methods offer systematic, evidence-driven approaches to inform policy decisions about which AMR interventions to fund. EE of AMR interventions is complicated owing to diffuse effects, complex mechanics of the problem and high levels of uncertainty. Current AMR EE literature restricts the analytical scope, potentially resulting in omissions of effects that may limit the utility of EE to inform policy decisions. We aimed to systemise the key evolutionary and ecological processes of AMR to elucidate the paths through which AMR interventions impact population health and healthcare costs to support EE design and to support decision makers in understanding the limitations of EE evidence for decision-making.

Methods

A conceptual map and a corresponding tool were developed on the basis of a literature review in consultation with experts across the relevant disciplines of molecular biology, infectious disease modelling, health economics and ecology.

Results

The AMR development map: (1) distils the key AMR processes and process drivers behind AMR development and maps the available types of AMR interventions to AMR process drivers; (2) proposes a way to conceptualise the spatial scope of analysis through considering the connectivity of the wider ecosystem and (3) outlines the key dimensions that AMR burden and intervention effects could be measured across. An AMR development map tool was developed to support conceptual modelling, with the focus on the choice of scope in the EE of AMR interventions, and an illustrative case study was provided.

Discussion

This work summarises the key underlying biological principles of AMR development to provide mechanistical grounding for considering the scope of effects of AMR interventions and the appropriate system of analysis to support conceptual modelling in EE of AMR interventions. In addition, this map can facilitate the identification of effects that cannot be considered or quantified, thus enabling transparency about these omissions within decision-making.

抗菌素耐药性(AMR)是一个复杂的跨部门和国际问题。经济评价(EE)方法提供了系统的、证据驱动的方法,为政策决策提供信息,以确定应该资助哪些抗菌素耐药性干预措施。抗菌素耐药性干预措施由于影响分散、问题机制复杂和高度不确定性而变得复杂。当前的AMR EE文献限制了分析范围,潜在地导致忽略了可能限制EE为政策决策提供信息的效用。我们旨在系统化AMR的关键进化和生态过程,阐明AMR干预措施影响人口健康和医疗保健成本的途径,以支持EE设计,并支持决策者理解EE证据对决策的局限性。方法:在文献综述的基础上,与分子生物学、传染病建模、卫生经济学和生态学等相关学科的专家协商,制定了概念图和相应的工具。结果:抗菌素耐药性发展图谱:(1)提炼出抗菌素耐药性发展背后的关键过程和过程驱动因素,并将抗菌素耐药性干预措施的可用类型映射为抗菌素耐药性过程驱动因素;(2)通过考虑更广泛生态系统的连通性,提出了一种概念化分析空间范围的方法;(3)概述了可以测量抗菌素耐药性负担和干预效果的关键维度。开发了一种抗菌素耐药性发展地图工具,以支持概念建模,重点关注抗菌素耐药性干预措施EE范围的选择,并提供了一个说明性案例研究。讨论:本工作总结了抗菌素耐药性发展的关键潜在生物学原理,为考虑抗菌素耐药性干预措施的影响范围和适当的分析系统提供了机制基础,以支持抗菌素耐药性干预措施的概念建模。此外,该地图有助于确定无法考虑或量化的影响,从而使决策过程中对这些遗漏具有透明度。
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引用次数: 0
Aggregate Distributional Cost-Effectiveness Analysis of Biologics for the Treatment of Ankylosing Spondylitis in Chile 智利治疗强直性脊柱炎生物制剂的总体分布成本-效果分析。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-05-06 DOI: 10.1007/s40258-025-00972-x
Magdalena Walbaum, Nicolas Jana-Valencia

Background and Objective

Ankylosing spondylitis is a complex rheumatic disease, characterised by chronic and progressive inflammation of the spine, causing an important health and economic burden for the person with the condition. Evidence shows the unequal impact of the disease in different groups of people, with a higher burden for lower socioeconomic groups. The objective of this study is to evaluate the impact of the use of biologics for the treatment of ankylosing spondylitis on health inequities in Chile.

Methods

We conducted an aggregate distributional cost-effectiveness analysis. Data on health outcomes and costs were derived from a cost-effectiveness model of secukinumab, etanercept, certolizumab pegol, infliximab, adalimumab and golimumab versus treatment as usual for the treatment of ankylosing spondylitis from the Chilean healthcare system perspective. Health gains and health opportunity costs were distributed across socioeconomic subgroups. Health and equity impacts, measured using the Atkinson index, were assessed on an equity-efficiency impact plane.

Results

All treatments had a positive impact on equity relative to treatment as usual. At an opportunity cost threshold of 1 Gross Domestic Product per capita/quality-adjusted life-year, secukinumab improved societal welfare irrespective of the Atkinson index value. When varying thresholds (2 and 3 Gross Domestic Product), all assessed technologies contributed to an increase in societal welfare, regardless of the Atkinson index.

Conclusions

Biologic treatment for ankylosing spondylitis, such as secukinumab, may reduce health inequity in the Chilean population. An aggregate distributional cost-effectiveness analysis framework is feasible to implement alongside a cost-effectiveness analysis in the context of the Chilean healthcare system to provide additional information of equity impacts for health technology assessment recommendations and policy making.

背景和目的:强直性脊柱炎是一种复杂的风湿性疾病,以脊柱慢性进行性炎症为特征,对患者造成重要的健康和经济负担。有证据表明,该疾病对不同人群的影响不平等,社会经济地位较低的群体负担更重。本研究的目的是评估使用生物制剂治疗强直性脊柱炎对智利卫生不公平现象的影响。方法:我们进行了总体分配成本-效果分析。健康结果和成本数据来自智利医疗保健系统角度的成本-效果模型,该模型将secukinumab、依那西普、certolizumab pegol、英夫利昔单抗、阿达木单抗和戈利木单抗与常规治疗相比用于治疗强直性脊柱炎。健康收益和健康机会成本分布在社会经济亚群体中。使用阿特金森指数衡量健康和公平影响,在公平-效率影响层面进行评估。结果:与常规治疗相比,所有治疗均对公平有积极影响。在1人均国内生产总值/质量调整生命年的机会成本门槛下,无论阿特金森指数值如何,secukinumab都能改善社会福利。当不同的阈值(国内生产总值2和3)时,所有被评估的技术都对社会福利的增加做出了贡献,而不考虑阿特金森指数。结论:强直性脊柱炎的生物治疗,如secukinumab,可能会减少智利人群的健康不平等。在智利卫生保健系统的背景下,与成本效益分析一起实施总分配成本效益分析框架是可行的,可以为卫生技术评估建议和政策制定提供额外的公平影响信息。
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引用次数: 0
Cost-Effectiveness Analysis of Evolocumab or Inclisiran in Combination with Statins Versus Statin Monotherapy Among Patients with ASCVD in China 中国ASCVD患者Evolocumab或Inclisiran联合他汀类药物与他汀类药物单药的成本-效果分析
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-05-05 DOI: 10.1007/s40258-025-00971-y
Pengpeng Wang, Le Liang, Yamei Li
<div><h3>Background and objective</h3><p>Several innovative proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors have been approved for combination lipid-lowering therapy in China. Compared with their high initial launch prices, some PCSK9 inhibitors have been subjected to the National Drug Price Negotiation (NDPN) policy with substantial price reductions, such as a 78.14% reduction for evolocumab (EVO). Others, such as inclisiran (INC), have not been included in this policy and maintain high prices. This study aimed to: (1) assess the cost-effectiveness of representative PCSK9 inhibitors (EVO and INC) in combination with statins versus statin monotherapy for Chinese patients with atherosclerotic cardiovascular disease (ASCVD), and (2) evaluate the influence of the NDPN policy on cost-effectiveness.</p><h3>Methods</h3><p>A Markov model was developed from the Chinese healthcare system perspective. Atorvastatin monotherapy was used as the standard of care (SOC), and the combinations of EVO or INC with SOC were employed as the intervention regimens. The transition probabilities and treatment effects were based on the Asian subgroup of the FOURIER trial and pooled analysis of the ORION-8, -9, and -10 trials, or calculated with results from a meta-analysis. Costs and utilities were derived from published literature or calculated with methods from literature and assumptions. Sensitivity and scenario analyses were conducted to evaluate the robustness of the model and the influence of the NDPN policy. Finally, the cost threshold analyses were conducted to estimate the annual costs required for EVO and INC to achieve a 75% probability of cost-effectiveness.</p><h3>Results</h3><p>The base-case incremental cost-effectiveness ratios (ICERs) of EVO + SOC and INC + SOC were 168,066 Chinese yuan (CNY) [23,652 US dollars (USD)] and 586,119 CNY (82,485 USD) per quality-adjusted life year (QALY), respectively. At WTP thresholds of three and one times GDP per capita (268,200 CNY [37,744 USD] and 89,400 CNY [12,581 USD]) per QALY gained, the probabilities that the intervention regimens were cost-effective would be 89.86% and 0.30% for EVO + SOC and 0.54% and 0% for INC + SOC, respectively. To achieve a 75% probability of cost-effectiveness, the required annual costs would be 9851 CNY (1386 USD) and 3434 CNY (483 USD) for EVO and 6554 CNY (922 USD) and 2096 CNY (295 USD) for INC, respectively. When the price reduction caused by the NDPN policy was removed from EVO and added to INC (assuming that the price of INC also reduces by 78.14%), the ICERs were 782,954 CNY (110,185 USD) and 130,877 CNY (18,418 USD) per QALY, respectively.</p><h3>Conclusions</h3><p>With the intervention of the NDPN policy, EVO + SOC has been a cost-effective option for Chinese patients with ASCVD at the WTP threshold of three times GDP per capita. However, INC + SOC is not a cost-effective regimen at the current price, and the NDPN policy may be an appropriate intervention measure.<
背景与目的:几种新型蛋白转化酶subtilisin/kexin type 9 (PCSK9)抑制剂已在中国被批准用于联合降脂治疗。与最初的高价格相比,一些PCSK9抑制剂已经受到国家药品价格谈判(NDPN)政策的大幅降价,例如evolocumab (EVO)的降价幅度为78.14%。其他的,如inclisiran (INC),没有包括在这一政策中,并保持高价。本研究旨在:(1)评估具有代表性的PCSK9抑制剂(EVO和INC)联合他汀类药物与他汀类药物单药治疗中国动脉粥样硬化性心血管疾病(ASCVD)患者的成本-效果;(2)评估NDPN政策对成本-效果的影响。方法:从中国医疗卫生系统角度建立马尔可夫模型。采用阿托伐他汀单药治疗作为标准护理方案(SOC), EVO或INC联合SOC作为干预方案。转移概率和治疗效果基于FOURIER试验的亚洲亚组和ORION-8、-9和-10试验的汇总分析,或根据荟萃分析的结果计算。成本和效用是从已发表的文献中得出的,或者用文献和假设中的方法计算出来的。通过敏感性和情景分析来评估模型的稳健性和NDPN政策的影响。最后,进行成本阈值分析,以估计EVO和INC达到75%成本效益概率所需的年成本。结果:EVO + SOC和INC + SOC的基本情况增量成本-效果比(ICERs)分别为每质量调整生命年(QALY) 168,066元(23,652美元)和586,119元(82,485美元)。在WTP阈值为人均GDP的3倍和1倍时(每个QALY分别为268,200元人民币(37,744美元)和89,400元人民币(12,581美元)),EVO + SOC的干预方案具有成本效益的概率分别为89.86%和0.30%,INC + SOC的干预方案为0.54%和0%。为了达到75%的成本效益,EVO的年成本分别为9851元人民币(1386美元)和3434元人民币(483美元),INC的年成本分别为6554元人民币(922美元)和2096元人民币(295美元)。当从EVO中去除NDPN政策导致的价格下降并加入INC(假设INC的价格也下降了78.14%)时,ICERs分别为782,954 CNY (110,185 USD)和130,877 CNY (18,418 USD) / QALY。结论:在NDPN政策的干预下,EVO + SOC已成为中国ASCVD患者在人均GDP三倍的WTP阈值下的成本效益选择。然而,在目前的价格下,INC + SOC并不是一个具有成本效益的方案,NDPN政策可能是一个合适的干预措施。
{"title":"Cost-Effectiveness Analysis of Evolocumab or Inclisiran in Combination with Statins Versus Statin Monotherapy Among Patients with ASCVD in China","authors":"Pengpeng Wang,&nbsp;Le Liang,&nbsp;Yamei Li","doi":"10.1007/s40258-025-00971-y","DOIUrl":"10.1007/s40258-025-00971-y","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background and objective&lt;/h3&gt;&lt;p&gt;Several innovative proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors have been approved for combination lipid-lowering therapy in China. Compared with their high initial launch prices, some PCSK9 inhibitors have been subjected to the National Drug Price Negotiation (NDPN) policy with substantial price reductions, such as a 78.14% reduction for evolocumab (EVO). Others, such as inclisiran (INC), have not been included in this policy and maintain high prices. This study aimed to: (1) assess the cost-effectiveness of representative PCSK9 inhibitors (EVO and INC) in combination with statins versus statin monotherapy for Chinese patients with atherosclerotic cardiovascular disease (ASCVD), and (2) evaluate the influence of the NDPN policy on cost-effectiveness.&lt;/p&gt;&lt;h3&gt;Methods&lt;/h3&gt;&lt;p&gt;A Markov model was developed from the Chinese healthcare system perspective. Atorvastatin monotherapy was used as the standard of care (SOC), and the combinations of EVO or INC with SOC were employed as the intervention regimens. The transition probabilities and treatment effects were based on the Asian subgroup of the FOURIER trial and pooled analysis of the ORION-8, -9, and -10 trials, or calculated with results from a meta-analysis. Costs and utilities were derived from published literature or calculated with methods from literature and assumptions. Sensitivity and scenario analyses were conducted to evaluate the robustness of the model and the influence of the NDPN policy. Finally, the cost threshold analyses were conducted to estimate the annual costs required for EVO and INC to achieve a 75% probability of cost-effectiveness.&lt;/p&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;p&gt;The base-case incremental cost-effectiveness ratios (ICERs) of EVO + SOC and INC + SOC were 168,066 Chinese yuan (CNY) [23,652 US dollars (USD)] and 586,119 CNY (82,485 USD) per quality-adjusted life year (QALY), respectively. At WTP thresholds of three and one times GDP per capita (268,200 CNY [37,744 USD] and 89,400 CNY [12,581 USD]) per QALY gained, the probabilities that the intervention regimens were cost-effective would be 89.86% and 0.30% for EVO + SOC and 0.54% and 0% for INC + SOC, respectively. To achieve a 75% probability of cost-effectiveness, the required annual costs would be 9851 CNY (1386 USD) and 3434 CNY (483 USD) for EVO and 6554 CNY (922 USD) and 2096 CNY (295 USD) for INC, respectively. When the price reduction caused by the NDPN policy was removed from EVO and added to INC (assuming that the price of INC also reduces by 78.14%), the ICERs were 782,954 CNY (110,185 USD) and 130,877 CNY (18,418 USD) per QALY, respectively.&lt;/p&gt;&lt;h3&gt;Conclusions&lt;/h3&gt;&lt;p&gt;With the intervention of the NDPN policy, EVO + SOC has been a cost-effective option for Chinese patients with ASCVD at the WTP threshold of three times GDP per capita. However, INC + SOC is not a cost-effective regimen at the current price, and the NDPN policy may be an appropriate intervention measure.&lt;","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 5","pages":"855 - 867"},"PeriodicalIF":3.3,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143968647","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Evaluating Access Improving Interventions: An Economic Evaluation of Surgical Task-Shifting for C-Sections in Sierra Leone 评估获取改善干预措施:塞拉利昂剖腹产手术任务转移的经济评估。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-04-30 DOI: 10.1007/s40258-025-00965-w
Bryony Dawkins, Bethany Shinkins, Tim Ensor, David Jayne, Thomas Ashley, Alex J. van Duinen, Håkon A. Bolkan, David Meads

Background

Access to safe, timely and affordable surgical care is lacking globally. Less than 6% of all surgical operations are carried out in low- and middle-income countries, where over a third of the world’s population lives. CapaCare, an NGO operating in Sierra Leone, have developed a surgical training programme (STP) for Associate Clinicians based on principles of task-shifting to improve access. Interventions to increase healthcare access have the same value evidence requirements as new technologies but their evaluation presents methodological challenges as access is not routinely incorporated explicitly in economic evaluations.

Objective

To evaluate the cost-effectiveness of surgical task-shifting in Sierra Leone, implemented through the CapaCare STP, to increase provision of caesarean section (C-section).

Methods

We evaluated the impact of the STP on the provision of C-section and subsequent maternal and child outcomes, measured in disability-adjusted life-years (DALYs), relative to the costs using a healthcare system perspective and decision-tree model parameterised using data from surgical logbooks, national data, and the literature.

Results

Results indicate that the surgical task-shifting programme in Sierra Leone would be considered cost-effective in increasing provision for C-section. It is cost saving (USD − 16.77) and results in 2.14 DALYs averted, per women with an indication for C-section, due to avoidance of maternal and child deaths as well as reduced complications.

Conclusion

Investment in surgical task-shifting initiatives should be considered by policymakers as a potentially cost-effective way to increase access to quality surgical services. Future evaluations of access-increasing interventions should seek to capture the distributional impact of this strategy and system benefits.

背景:全球缺乏安全、及时和负担得起的外科护理。不到6%的外科手术是在占世界人口三分之一以上的低收入和中等收入国家进行的。在塞拉利昂运作的非政府组织CapaCare根据任务转移原则为助理临床医生制定了一项外科培训计划(STP),以改善获取机会。增加医疗保健可及性的干预措施与新技术具有相同的价值证据要求,但其评估存在方法上的挑战,因为可及性通常没有明确纳入经济评估。目的:评估塞拉利昂通过CapaCare STP实施的手术任务转移的成本效益,以增加剖腹产(C-section)的提供。方法:我们评估STP对提供剖腹产和随后的孕产妇和儿童结局的影响,以残疾调整生命年(DALYs)衡量,相对于成本,使用医疗保健系统视角和决策树模型参数化,使用手术日志、国家数据和文献数据。结果:结果表明,在塞拉利昂的手术任务转移方案将被认为是具有成本效益的增加提供剖腹产。由于避免了孕产妇和儿童死亡以及减少了并发症,因此每名有剖腹产指征的妇女节省了费用(- 16.77美元),避免了2.14个伤残调整生命年。结论:决策者应考虑对手术任务转移计划的投资,将其作为增加获得高质量手术服务的潜在成本效益途径。今后对增加获取的干预措施的评价应设法抓住这一战略和系统效益的分配影响。
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引用次数: 0
The Health Economics of Genomic Technologies: A Growing Evidence Base on Value 基因组技术的健康经济学:基于价值的越来越多的证据
IF 3.1 4区 医学 Q1 ECONOMICS Pub Date : 2025-04-28 DOI: 10.1007/s40258-025-00970-z
James Buchanan, Ilias Goranitis, Deirdre Weymann
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引用次数: 0
Cost-Effectiveness and Value of Information Analyses of Caplacizumab for the Treatment of Thrombotic Thrombocytopenic Purpura in Brazil 巴西卡普拉珠单抗治疗血栓性血小板减少性紫癜的成本-效果和信息价值分析
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-04-24 DOI: 10.1007/s40258-025-00968-7
Julia Simões Correa Galendi, Hannes Rasch, Carlos Antonio Caramori, Rafael Dezen Gaiolla, Dirk Müller, Vania Santos Nunes Nogueira

Objectives

The objective was to evaluate the cost effectiveness of caplacizumab for the treatment of patients with acquired immune thrombocytopenic thrombotic purpura (iTTP) compared to standard of care from the perspective of the Brazilian Unified Health System (SUS).

Methods

A decision tree followed by a Markov model with a lifetime horizon was developed. Patients entered the model with an acute iTTP event. All patients were assumed to be admitted to hospital where they either respond to treatment or die. The model offered three health states: remission, relapse or death. Input data were obtained from literature searches with the data on efficacy of caplacizumab based on the HERCULES trial. The incremental cost-effectiveness ratio (ICER) was compared to the willingness-to-pay threshold for rare diseases of Brazilian reais (R$)120,000/quality-adjusted life years (QALYs). In addition to various sensitivity analyses, a value of information (VOI) analysis was conducted.

Results

In the base case, caplacizumab resulted in 0.70 QALYs gained, and cost R$1,333,601 more, with an ICER of R$1,901,729/QALY. The cost of the caplacizumab vial was the most influential parameter. Probabilistic analysis showed that caplacizumab was not cost effective in any iterations for the threshold of the rare disease. The expected value of perfect information per year is R$0.

Conclusion

Although caplacizumab results in incremental QALYs, based on the proposed cost, caplacizumab is not cost effective from the SUS perspective, and VOI results indicate that further research would not be worthwhile.

目的:目的是从巴西统一卫生系统(SUS)的角度评估卡普拉珠单抗治疗获得性免疫性血小板减减性血栓性紫癜(iTTP)患者的成本效益。方法:建立具有生命周期视界的决策树和马尔可夫模型。患者进入模型时伴有急性iTTP事件。所有的病人都被假定住进医院,在那里他们要么对治疗有反应,要么死亡。该模型提供了三种健康状态:缓解、复发或死亡。输入数据来自文献检索,其中包括基于HERCULES试验的caplacizumab疗效数据。将增量成本-效果比(ICER)与巴西雷亚尔(R$)120,000/质量调整生命年(QALYs)的罕见病支付意愿阈值进行比较。除了各种敏感性分析外,还进行了信息值(VOI)分析。结果:在基本病例中,卡普拉珠单抗获得0.70个QALY,成本增加1,333,601雷亚尔,ICER为1,901,729雷亚尔/QALY。卡普拉珠单抗药瓶的成本是影响最大的参数。概率分析显示,对于罕见病的阈值,卡普拉珠单抗在任何迭代中都没有成本效益。每年完全信息的期望值为0雷亚尔。结论:虽然卡placizumab的QALYs增加,但基于拟议的成本,从SUS的角度来看,卡placizumab不具有成本效益,VOI结果表明不值得进一步研究。
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引用次数: 0
Compulsory Licensing as an Instrument to Tackle High Medicine Prices: A Realist Review of Industrial and Health Consequences 强制许可作为解决高药价的工具:对工业和健康后果的现实主义审查。
IF 3.3 4区 医学 Q1 ECONOMICS Pub Date : 2025-04-19 DOI: 10.1007/s40258-025-00956-x
Steven Simoens, Walter Van Dyck, Rosanne Janssens, Liese Barbier, Jeroen Luyten

Although compulsory licensing of medicines is traditionally discussed in the context of low- and middle-income countries tackling high prices, it has recently sparked debate in several high-income countries. This study aims to examine the industrial and health consequences of compulsory licensing when applied by a high-income country. Our literature review found that the impact of compulsory licensing is challenging to predict as it can have multiple (opposing) consequences in terms of economic activity, patient outcomes and public health. Compulsory licensing can, under particular circumstances, serve as a lever of industrial policy in a country that wishes to develop its domestic generic pharmaceutical industry. However, originator pharmaceutical companies and other industries may reduce investment, which can be negative for countries with a high presence of innovators, adversely impacting long-term economic activity. Compulsory licensing may also induce state retaliation against the license-issuing country. From a health policy perspective, compulsory licensing likely increases patient access to expensive medicines and frees up resources that can be invested in other (health) programs. However, pharmaceutical companies may delay medicine launches or cancel clinical trials in the license-issuing country. Although there are benefits resulting from a credible threat to use compulsory licensing, the overall desirability of actually using it depends on the specific context and needs to be assessed on a case-by-case basis.

虽然药品强制许可传统上是在低收入和中等收入国家应对高价格的背景下讨论的,但最近在几个高收入国家引发了辩论。本研究旨在考察高收入国家实施强制许可的工业和健康后果。我们的文献综述发现,强制许可的影响很难预测,因为它可能在经济活动、患者预后和公共卫生方面产生多重(相反的)后果。在特殊情况下,强制许可可以作为希望发展其国内非专利制药工业的国家的工业政策杠杆。然而,创始者制药公司和其他行业可能会减少投资,这对拥有大量创新者的国家可能是不利的,对长期经济活动产生不利影响。强制许可也可能引起国家对发证国的报复。从卫生政策的角度来看,强制许可可能会增加患者获得昂贵药物的机会,并释放可投资于其他(卫生)计划的资源。然而,制药公司可能会推迟药物上市或取消在发证国的临床试验。虽然使用强制许可的可信威胁会带来好处,但实际使用强制许可的总体可取性取决于具体情况,需要逐个评估。
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Applied Health Economics and Health Policy
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