Pub Date : 2024-12-30DOI: 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。
{"title":"Exome Sequencing in the Diagnostic Pathway for Suspected Rare Genetic Diseases: Does the Order of Testing Affect its Cost-Effectiveness?","authors":"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","doi":"10.1007/s40258-024-00936-7","DOIUrl":"https://doi.org/10.1007/s40258-024-00936-7","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909111","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}
Pub Date : 2024-12-30DOI: 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.
{"title":"Cost of Carbon in the Total Cost of a Healthcare Procedure: Example of Micro-Costing Study in a French Setting","authors":"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","doi":"10.1007/s40258-024-00933-w","DOIUrl":"10.1007/s40258-024-00933-w","url":null,"abstract":"<div><h3>Background</h3><p>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.</p><h3>Objective</h3><p>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.</p><h3>Methods</h3><p>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.</p><h3>Results</h3><p>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.</p><h3>Conclusion</h3><p>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.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"265 - 275"},"PeriodicalIF":3.1,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909106","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}
Pub Date : 2024-12-28DOI: 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.
{"title":"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","authors":"Ni Gao, Mandy Ryan, Suzanne Robinson, Richard Norman","doi":"10.1007/s40258-024-00934-9","DOIUrl":"10.1007/s40258-024-00934-9","url":null,"abstract":"<div><h3>Background</h3><p>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.</p><h3>Objective</h3><p>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.</p><h3>Methods</h3><p>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.</p><h3>Results</h3><p>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).</p><h3>Conclusion</h3><p>The heterogeneous preferences for time allocation among women have policy implications and are significant for welfare analysis.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"253 - 264"},"PeriodicalIF":3.1,"publicationDate":"2024-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142891468","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}
Pub Date : 2024-12-28DOI: 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。
{"title":"Systematic Literature Review of Access Pathways to Drugs for Patients with Rare Diseases","authors":"Constanza Vargas, Richard De Abreu Lourenco, Manuel Espinoza, Stephen Goodall","doi":"10.1007/s40258-024-00939-4","DOIUrl":"10.1007/s40258-024-00939-4","url":null,"abstract":"<div><h3>Objective</h3><p>This article reviews the assessment pathways that have been implemented worldwide to facilitate access to drugs for patients with rare diseases.</p><h3>Methods</h3><p>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.</p><h3>Results</h3><p>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.</p><h3>Conclusions</h3><p>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.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"209 - 229"},"PeriodicalIF":3.1,"publicationDate":"2024-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142891466","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}
Pub Date : 2024-12-19DOI: 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.
{"title":"Cost-Effective and Sustainable Drug Use in Hospitals: A Systematic and Practice-Based Approach","authors":"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","doi":"10.1007/s40258-024-00937-6","DOIUrl":"10.1007/s40258-024-00937-6","url":null,"abstract":"<div><h3>Background and Objective</h3><p>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.</p><h3>Methods</h3><p>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.</p><h3>Results</h3><p>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.</p><h3>Conclusions</h3><p>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.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"183 - 195"},"PeriodicalIF":3.1,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-024-00937-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142862748","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-13DOI: 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.
{"title":"Quality-Adjusted Life Expectancy Norms Based on the EQ-5D-5L and SF-6Dv2 for China","authors":"Shitong Xie, Xiaoning He, Weihua Guo, Jing Wu","doi":"10.1007/s40258-024-00925-w","DOIUrl":"10.1007/s40258-024-00925-w","url":null,"abstract":"<div><h3>Objectives</h3><p>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.</p><h3>Methods</h3><p>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.</p><h3>Results</h3><p>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.</p><h3>Conclusion</h3><p>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.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"291 - 310"},"PeriodicalIF":3.1,"publicationDate":"2024-12-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142821844","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}
Pub Date : 2024-12-12DOI: 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.
{"title":"Value is Gendered: The Need for Sex and Gender Considerations in Health Economic Evaluations","authors":"Martina Mchenga, Lavanya Vijayasingham, Rajalakshmi RamPrakash, Michelle Remme","doi":"10.1007/s40258-024-00930-z","DOIUrl":"10.1007/s40258-024-00930-z","url":null,"abstract":"<div><p>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.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"171 - 181"},"PeriodicalIF":3.1,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-024-00930-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142811932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-06DOI: 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.
{"title":"Assessing the Direct Impact of Death on Discrete Choice Experiment Utilities","authors":"Hossein Ameri, Thomas G. Poder","doi":"10.1007/s40258-024-00929-6","DOIUrl":"10.1007/s40258-024-00929-6","url":null,"abstract":"<div><h3>Background</h3><p>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 (DCE<sub>TTO</sub>).</p><h3>Methods</h3><p>A sample of the general population in Quebec, Canada, completed two approaches: DCE<sub>TTO</sub> followed by a best-worst scaling with time (BWS<sub>TTO</sub>) (hereafter referred to as DCE<sub>BWS</sub>), versus DCE<sub>TTO</sub> followed by the dominated option and the immediate death state (hereafter referred to as DCE<sub>DOD</sub>), both designed with the SF-6Dv2. In DCE<sub>BWS</sub>, all participants first completed 10 DCE<sub>TTO</sub> choices (i.e., option A vs B), followed by 3 BWS<sub>TTO</sub>. In DCE<sub>DOD</sub>, the same participants first completed the same 10 DCE<sub>TTO</sub> 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 DCE<sub>TTO</sub> with DCE<sub>BWS</sub> and with DCE<sub>DOD</sub> using Bland-Altman plots, the proportion of worst-than-dead (WTD) health states, and analyzing the range of estimated values.</p><h3>Results</h3><p>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 DCE<sub>BWS</sub>. The observed consistency in the relative importance of dimensions across all approaches suggests a stable and reliable ranking. The utility range for DCE<sub>DOD</sub> (− 0.921 to 1) was narrower than for DCE<sub>TTO</sub> (− 1.578 to 1) and DCE<sub>BWS</sub> (− 1.150 to 1). The DCE<sub>DOD</sub> estimated a lower percentage of WTD health states (20.01 %) compared to DCE<sub>TTO</sub> (47.19 %) and DCE<sub>BWS</sub> (33.73 %). The agreement between DCE<sub>TTO</sub> and DCE<sub>BWS</sub> was slightly stronger than between DCE<sub>TTO</sub> and DCE<sub>DOD</sub>, and the mean utility values were higher in DCE<sub>DOD</sub> than in DCE<sub>BWS</sub>.</p><h3>Conclusions</h3><p>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 DCE<sub>TTO</sub> (i.e., DCE<sub>DOD</sub>) than when it was included in a continuum of DCE<sub>TTO</sub> (i.e., DCE<sub>BWS</sub>). The use of DCE<sub>DOD</sub> was potentially better suited to incorporate the dead state into a DCE.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 2","pages":"319 - 327"},"PeriodicalIF":3.1,"publicationDate":"2024-12-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142791077","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}
Pub Date : 2024-12-05DOI: 10.1007/s40258-024-00931-y
{"title":"Acknowledgement to Referees","authors":"","doi":"10.1007/s40258-024-00931-y","DOIUrl":"10.1007/s40258-024-00931-y","url":null,"abstract":"","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"23 1","pages":"1 - 3"},"PeriodicalIF":3.1,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142783619","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}
Pub Date : 2024-12-02DOI: 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.
{"title":"Evaluating the Cost-Effectiveness of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B Treatment in the USA.","authors":"Jyotirmoy Sarker, Jeffrey A Tice, David M Rind, Surrey M Walton","doi":"10.1007/s40258-024-00932-x","DOIUrl":"https://doi.org/10.1007/s40258-024-00932-x","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>This study evaluates the cost-effectiveness of EDZ compared with conventional factor IX (FIX) prophylaxis.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142765795","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}