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Cost of Carbon in the Total Cost of Healthcare Procedures: A Methodological Challenge 医疗程序总成本中的碳成本:方法论挑战。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-06-11 DOI: 10.1007/s40258-024-00890-4
Paul-Simon Pugliesi, Laurie Marrauld, Catherine Lejeune
{"title":"Cost of Carbon in the Total Cost of Healthcare Procedures: A Methodological Challenge","authors":"Paul-Simon Pugliesi,&nbsp;Laurie Marrauld,&nbsp;Catherine Lejeune","doi":"10.1007/s40258-024-00890-4","DOIUrl":"10.1007/s40258-024-00890-4","url":null,"abstract":"<div><p>Economic evaluations aim to compare the costs and the results of health strategies to guide the public decision-making process. Cost estimation is, thus, a cornerstone of this approach. At present, few national evaluation agencies recommend incorporating the cost of greenhouse gas (GHG) emissions from healthcare actions into the calculation of healthcare costs. Our main goal is to describe and discuss the methodology for integrating the cost of GHG emissions into the field of applied economic evaluations. To estimate this cost, three steps are required: (1) identifying and quantifying the physical flows linked to the production and management of the outputs of healthcare interventions, (2) estimating the quantity of GHG that can be attributed to each physical flow, and (3) valuing these GHG emissions in monetary terms. Integrating the cost of GHG emissions into the calculation of the costs of healthcare interventions is both useful and relevant from a perspective of collective intergenerational well-being. This approach has been made possible thanks to the existence of accounting and monetary valuation methods for emissions. Agencies specialized in health economic evaluations could take up this issue to resolve ongoing questions, thus providing researchers with a methodological framework and public decision-makers with some key insights.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 5","pages":"599 - 607"},"PeriodicalIF":3.1,"publicationDate":"2024-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141305240","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
Employing Real-World Evidence for the Economic Evaluation of Non-Vitamin K Antagonist Oral Anticoagulants in Patients with Atrial Fibrillation in Thailand 采用真实世界的证据对泰国心房颤动患者使用非维生素 K 拮抗剂口服抗凝药进行经济评估。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-06-10 DOI: 10.1007/s40258-024-00891-3
Rungroj Krittayaphong, Unchalee Permsuwan
{"title":"Employing Real-World Evidence for the Economic Evaluation of Non-Vitamin K Antagonist Oral Anticoagulants in Patients with Atrial Fibrillation in Thailand","authors":"Rungroj Krittayaphong,&nbsp;Unchalee Permsuwan","doi":"10.1007/s40258-024-00891-3","DOIUrl":"10.1007/s40258-024-00891-3","url":null,"abstract":"<div><h3>Background</h3><p>This study aimed to assess the cost-effectiveness of non-vitamin K antagonist oral anticoagulants (NOACs) in comparison with warfarin using data from real practice based on the perspective of the health care system in Thailand.</p><h3>Methods</h3><p>A four-state Markov model encompassing well-controlled atrial fibrillation (AF), stroke and systemic embolism, major bleeding and death was utilised to forecast clinical and economic outcomes. Transitional probabilities, direct medical costs and utilities were derived from the real-world data of the ‘COOL-AF Thailand’ registry, Thailand’s largest nationwide registry spanning 27 hospitals. The cohort comprised AF patients. The primary outcomes assessed were total costs, life years, quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio. All costs and outcomes were subject to an annual discount rate of 3.0%. A spectrum of sensitivity analyses was conducted.</p><h3>Results</h3><p>The mean age of the cohort was 68.8 ± 10.7 years. The NOACs group incurred a marginally lower total lifetime cost than the warfarin group (247,857 Thai baht [THB] vs 253,654 THB or 7137 USD vs 7304 USD) and experienced gains of 0.045 life years and 0.043 QALYs over the warfarin group. Given the lower cost and higher benefits associated with NOACs, this implies that NOAC treatment is a dominant strategy compared to warfarin for AF patients. At a ceiling ratio of 160,000 THB (4607 USD) per QALY, NOACs presented a 61.2% probability of being cost effective.</p><h3>Conclusions</h3><p>Non-vitamin K antagonist oral anticoagulants represent a cost-saving alternative to warfarin in the real clinical practice. However, with a probability of being cost effective below 65%, it suggests some parameter uncertainty regarding their overall cost effectiveness compared to warfarin.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 5","pages":"725 - 734"},"PeriodicalIF":3.1,"publicationDate":"2024-06-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141299884","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
Pricing, Procurement and Reimbursement Policies for Incentivizing Market Entry of Novel Antibiotics and Diagnostics: Learnings from 10 Countries Globally 激励新型抗生素和诊断方法进入市场的定价、采购和报销政策:全球 10 个国家的经验教训。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-06-05 DOI: 10.1007/s40258-024-00888-y
Sabine Vogler, Katharina Habimana, Manuel Alexander Haasis, Stefan Fischer
{"title":"Pricing, Procurement and Reimbursement Policies for Incentivizing Market Entry of Novel Antibiotics and Diagnostics: Learnings from 10 Countries Globally","authors":"Sabine Vogler,&nbsp;Katharina Habimana,&nbsp;Manuel Alexander Haasis,&nbsp;Stefan Fischer","doi":"10.1007/s40258-024-00888-y","DOIUrl":"10.1007/s40258-024-00888-y","url":null,"abstract":"<div><h3>Background</h3><p>Fostering market entry of novel antibiotics and enhanced use of diagnostics to improve the quality of antibiotic prescribing are avenues to tackle antimicrobial resistance (AMR), which is a major public health threat. Pricing, procurement and reimbursement policies may work as AMR ‘pull incentives’ to support these objectives. This paper studies pull incentives in pricing, procurement and reimbursement policies (e.g., additions to, modifications of, and exemptions from standard policies) for novel antibiotics, diagnostics and health products with a similar profile in 10 study countries. It also explores whether incentives for non-AMR health products could be transferred to AMR health products.</p><h3>Methods</h3><p>This research included a review of policies in 10 G20 countries based on literature and unpublished documents, and the production of country fact sheets that were validated by country experts. Initial research was conducted in 2020 and updated in 2023.</p><h3>Results</h3><p>Identified pull incentives in pricing policies include free pricing, higher prices at launch and price increases over time, managed-entry agreements, and waiving or reducing mandatory discounts. Incentives in procurement comprise value-based procurement, pooled procurement and models that delink prices from volumes (subscription-based schemes), whereas incentives in reimbursement include lower evidence requirements for inclusion in the reimbursement scheme, accelerated reimbursement processes, separate budgets that offer add-on funding, and adapted prescribing conditions.</p><h3>Conclusions</h3><p>While a few pull incentives have been piloted or implemented for antibiotics in recent years, these mechanisms have been mainly used to incentivize launch of certain non-AMR health products, such as orphan medicines. Given similarities in their product characteristics, transferability of some of these pull incentives appears to be possible; however, it would be essential to conduct impact assessments of these incentives. Trade-offs between incentives to foster market entry and thus potentially improve access and the financial sustainability for payers need to be addressed.</p><h3>Graphical Abstract</h3><div><figure><div><div><picture><source><img></source></picture></div></div></figure></div></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 5","pages":"629 - 652"},"PeriodicalIF":3.1,"publicationDate":"2024-06-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141247569","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
Cost-Effectiveness of Test-and-Treat Strategies to Reduce the Antibiotic Prescription Rate for Acute Febrile Illness in Primary Healthcare Clinics in Africa 降低非洲初级保健诊所急性发热病抗生素处方率的试验和治疗策略的成本效益
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-05-25 DOI: 10.1007/s40258-024-00889-x
Pim W. M. van Dorst, Simon van der Pol, Piero Olliaro, Sabine Dittrich, Juvenal Nkeramahame, Maarten J. Postma, Cornelis Boersma, Antoinette D. I. van Asselt
{"title":"Cost-Effectiveness of Test-and-Treat Strategies to Reduce the Antibiotic Prescription Rate for Acute Febrile Illness in Primary Healthcare Clinics in Africa","authors":"Pim W. M. van Dorst,&nbsp;Simon van der Pol,&nbsp;Piero Olliaro,&nbsp;Sabine Dittrich,&nbsp;Juvenal Nkeramahame,&nbsp;Maarten J. Postma,&nbsp;Cornelis Boersma,&nbsp;Antoinette D. I. van Asselt","doi":"10.1007/s40258-024-00889-x","DOIUrl":"10.1007/s40258-024-00889-x","url":null,"abstract":"<div><h3>Background</h3><p>Inappropriate antibiotic use increases selective pressure, contributing to antimicrobial resistance. Point-of-care rapid diagnostic tests (RDTs) would be instrumental to better target antibiotic prescriptions, but widespread implementation of diagnostics for improved management of febrile illnesses is limited.</p><h3>Objective</h3><p>Our study aims to contribute to evidence-based guidance to inform policymakers on investment decisions regarding interventions that foster more appropriate antibiotic prescriptions, as well as to address the evidence gap on the potential clinical and economic impact of RDTs on antibiotic prescription.</p><h3>Methods</h3><p>A country-based cost-effectiveness model was developed for Burkina Faso, Ghana and Uganda. The decision tree model simulated seven test strategies for patients with febrile illness to assess the effect of different RDT combinations on antibiotic prescription rate (APR), costs and clinical outcomes. The incremental cost-effectiveness ratio (ICER) was expressed as the incremental cost per percentage point (ppt) reduction in APR.</p><h3>Results</h3><p>For Burkina Faso and Uganda, testing all patients with a malaria RDT was dominant compared to standard-of-care (SoC) (which included malaria testing). Expanding the test panel with a C-reactive protein (CRP) test resulted in an ICER of $ 0.03 and $ 0.08 per ppt reduction in APR for Burkina Faso and Uganda, respectively. For Ghana, the pairwise comparison with SoC—including malaria and complete blood count testing—indicates that both testing with malaria RDT only and malaria RDT + CRP are dominant.</p><h3>Conclusion</h3><p>The use of RDTs for patients with febrile illness could effectively reduce APR at minimal additional costs, provided diagnostic algorithms are adhered to. Complementing SoC with CRP testing may increase clinicians’ confidence in prescribing decisions and is a favourable strategy.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 5","pages":"701 - 715"},"PeriodicalIF":3.1,"publicationDate":"2024-05-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-024-00889-x.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141149592","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}
引用次数: 0
Informing Structural Assumptions for Three State Oncology Cost-Effectiveness Models through Model Efficiency and Fit 通过模型效率和拟合度为三个州肿瘤成本效益模型的结构假设提供依据。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-05-21 DOI: 10.1007/s40258-024-00884-2
Dominic Muston
{"title":"Informing Structural Assumptions for Three State Oncology Cost-Effectiveness Models through Model Efficiency and Fit","authors":"Dominic Muston","doi":"10.1007/s40258-024-00884-2","DOIUrl":"10.1007/s40258-024-00884-2","url":null,"abstract":"<div><p>The characteristics and relative strengths and weaknesses of partitioned survival models (PSMs) and state transition models (STMs) for three state oncology cost-effectiveness models have previously been studied. Despite clear and longstanding economic modeling guidelines, more than one structure is rarely presented, and the choice of structure appears correlated more with audience or precedent than disease, decision problem, or available data. One reason may be a lack of guidance and tools available to readily compare measures of internal validity such as the model fit and efficiency of different structures, or sensitivity of results to those choices. To address this gap, methods are presented to evaluate the fit and efficiency of three structures, with an accompanying R software package, <i>psm3mkv</i>. The methods are illustrated by analyzing interim and final analysis datasets of the KEYNOTE-826 randomized controlled trial. At both interim and final analyses, the STM Clock Reset structure provided the best and most efficient fit. Structural uncertainties had been reduced from interim to final analysis. Beyond measures of internal validity, guidelines highlight the importance of reflecting all available data, avoiding model selection purely on the basis of goodness of fit and strongly considering external validity. The method and software allow modelers to more easily evaluate and report model fit and efficiency, examine implicit assumptions, and reveal sensitivities to structural choices.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 5","pages":"619 - 628"},"PeriodicalIF":3.1,"publicationDate":"2024-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070248","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
Cost Effectiveness of Adding Fenfluramine to Standard of Care for Patients with Dravet Syndrome in Sweden 瑞典在标准治疗基础上增加芬氟拉明治疗垂视综合征患者的成本效益。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-05-17 DOI: 10.1007/s40258-024-00886-0
Chiara Malmberg, Magnus Värendh, Patric Berling, Mata Charokopou, Erik Eklund
{"title":"Cost Effectiveness of Adding Fenfluramine to Standard of Care for Patients with Dravet Syndrome in Sweden","authors":"Chiara Malmberg,&nbsp;Magnus Värendh,&nbsp;Patric Berling,&nbsp;Mata Charokopou,&nbsp;Erik Eklund","doi":"10.1007/s40258-024-00886-0","DOIUrl":"10.1007/s40258-024-00886-0","url":null,"abstract":"<div><h3>Objective</h3><p>This study evaluated, in a Swedish setting, the cost effectiveness of fenfluramine (FFA) as an add-on to standard of care (SoC) for reducing seizure frequency in Dravet syndrome, a severe developmental epileptic encephalopathy.</p><h3>Methods</h3><p>Cost effectiveness of FFA+SoC compared with SoC only was evaluated using a patient-level simulation model with a lifetime horizon. Patient characteristics and treatment effects, including convulsive seizures, seizure-free days and mortality, were derived from FFA clinical trials. Resource use and costs included cost of drug acquisition, routine care and monitoring, as well as ongoing and emergency resources. Quality of life (QoL) estimates for patients and their caregivers were derived from clinical trial data. Robustness was evaluated by one-way sensitivity analysis, probabilistic sensitivity analysis and scenario analyses.</p><h3>Results</h3><p>Lifetime cost of FFA+SoC was ~3 million SEK per patient compared with ~1.5 million SEK for SoC only. FFA+SoC generated 15% more QALYs than SoC only (21.2 vs 18.5 over a lifetime), resulting in an incremental cost-effectiveness ratio (ICER) of ~540,000 SEK. Moreover, FFA+SoC had a higher probability of being cost effective than SoC only from a willingness-to-pay threshold of 710,000 SEK. Results remained generally consistent across scenario analyses, with only few exceptions (exclusions of carer utility or FFA effect on sudden unexpected death in epilepsy).</p><h3>Conclusion</h3><p>Due to better seizure control, FFA is a clinically meaningful add-on therapy and was estimated to be a cost-effective addition to current SoC for patients with this rare disease in Sweden at a willingness-to-pay threshold of 1,000,000 SEK.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 4","pages":"543 - 554"},"PeriodicalIF":3.1,"publicationDate":"2024-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140954995","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
A structured process for the validation of a decision-analytic model: application to a cost-effectiveness model for risk-stratified national breast screening 验证决策分析模型的结构化流程:应用于风险分层国家乳腺筛查的成本效益模型。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-05-16 DOI: 10.1007/s40258-024-00887-z
Stuart J. Wright, Ewan Gray, Gabriel Rogers, Anna Donten, Katherine Payne
{"title":"A structured process for the validation of a decision-analytic model: application to a cost-effectiveness model for risk-stratified national breast screening","authors":"Stuart J. Wright,&nbsp;Ewan Gray,&nbsp;Gabriel Rogers,&nbsp;Anna Donten,&nbsp;Katherine Payne","doi":"10.1007/s40258-024-00887-z","DOIUrl":"10.1007/s40258-024-00887-z","url":null,"abstract":"<div><h3>Background</h3><p>Decision-makers require knowledge of the strengths and weaknesses of decision-analytic models used to evaluate healthcare interventions to be able to confidently use the results of such models to inform policy. A number of aspects of model validity have previously been described, but no systematic approach to assessing the validity of a model has been proposed. This study aimed to consolidate the different aspects of model validity into a step-by-step approach to assessing the strengths and weaknesses of a decision-analytic model.</p><h3>Methods</h3><p>A pre-defined set of steps were used to conduct the validation process of an exemplar early decision-analytic-model-based cost-effectiveness analysis of a risk-stratified national breast cancer screening programme [UK healthcare perspective; lifetime horizon; costs (£; 2021)]. Internal validation was assessed in terms of descriptive validity, technical validity and face validity. External validation was assessed in terms of operational validation, convergent validity (or corroboration) and predictive validity.</p><h3>Results</h3><p>The results outline the findings of each step of internal and external validation of the early decision-analytic-model and present the validated model (called ‘MANC-RISK-SCREEN’). The positive aspects in terms of meeting internal validation requirements are shown together with the remaining limitations of MANC-RISK-SCREEN.</p><h3>Conclusion</h3><p>Following a transparent and structured validation process, MANC-RISK-SCREEN has been shown to have satisfactory internal and external validity for use in informing resource allocation decision-making. We suggest that MANC-RISK-SCREEN can be used to assess the cost-effectiveness of exemplars of risk-stratified national breast cancer screening programmes (NBSP) from the UK perspective.</p><h3>Implications</h3><p>A step-by-step process for conducting the validation of a decision-analytic model was developed for future use by health economists. Using this approach may help researchers to fully demonstrate the strengths and limitations of their model to decision-makers.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 4","pages":"527 - 542"},"PeriodicalIF":3.1,"publicationDate":"2024-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11178649/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140954809","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}
引用次数: 0
Measurement of Catastrophic Health Expenditure in India: A Systematic Review and Meta-Analysis 印度灾难性医疗支出的衡量:系统回顾与元分析》。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-05-10 DOI: 10.1007/s40258-024-00885-1
Umenthala Srikanth Reddy
{"title":"Measurement of Catastrophic Health Expenditure in India: A Systematic Review and Meta-Analysis","authors":"Umenthala Srikanth Reddy","doi":"10.1007/s40258-024-00885-1","DOIUrl":"10.1007/s40258-024-00885-1","url":null,"abstract":"<div><h3>Introduction</h3><p>The escalating burden of catastrophic health expenditure (CHE) poses a significant threat to individuals and households in India, where out-of-pocket expenditure (OOP) constitutes a substantial portion of healthcare financing. With rising OOP in India, a proper measurement to track and monitor CHE due to health expenditure is of utmost important. This study focuses on synthesizing findings, understanding measurement variations, and estimating the pooled incidence of CHE by health services, reported diseases, and survey types.</p><h3>Method</h3><p>Following the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines, a thorough search strategy was employed across multiple databases, between 2010 and 2023. Inclusion criteria encompassed observational or interventional studies reporting CHE incidence, while exclusion criteria screened out studies with unclear definitions, pharmacy revenue-based spending, or non-representative health facility surveys. A meta-analysis, utilizing a random-effects model, assessed the pooled CHE incidence. Sensitivity analysis and subgroup analyses were conducted to explore heterogeneity.</p><h3>Results</h3><p>Out of 501 initially relevant articles, 36 studies met inclusion criteria. The review identified significant variations in CHE measurements, with incidence ranging from 5.1% to 69.9%. Meta-analysis indicated the estimated incidence of CHE at a 10% threshold is 0.30 [0.25–0.35], indicating a significant prevalence of financial hardship due to health expenses. The pooled incidence is estimated by considering different sub-groups. No statistical differences were found between inpatient and outpatient CHE. However, disease-specific estimates were significantly higher (52%) compared to combined diseases (21%). Notably, surveys focusing on health reported higher CHE (33%) than consumption surveys (14%).</p><h3>Discussion</h3><p>The study highlights the intricate challenges in measuring CHE, emphasizing variations in recall periods, components considered in out-of-pocket expenditure, and diverse methods for defining capacity to pay. Notably, the findings underscore the need for standardized definitions and measurements across studies. The lack of uniformity in reporting exacerbates the challenge of comparing and comprehensively understanding the financial burden on households.</p></div>","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 4","pages":"471 - 483"},"PeriodicalIF":3.1,"publicationDate":"2024-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140897006","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
Evolving Evidence-Based Value Assessment of One-Time Therapies: Tisagenlecleucel as a Case Study 基于证据的一次性疗法价值评估的演变:作为案例研究的 Tisagenlecleucel
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-04-29 DOI: 10.1007/s40258-024-00882-4
Theodore Laetsch, Jie Zhang, Hongbo Yang, Yanwen Xie, Dudan Zhang, Louis Garrison
{"title":"Evolving Evidence-Based Value Assessment of One-Time Therapies: Tisagenlecleucel as a Case Study","authors":"Theodore Laetsch,&nbsp;Jie Zhang,&nbsp;Hongbo Yang,&nbsp;Yanwen Xie,&nbsp;Dudan Zhang,&nbsp;Louis Garrison","doi":"10.1007/s40258-024-00882-4","DOIUrl":"10.1007/s40258-024-00882-4","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;p&gt;Economic evaluation of one-time therapies during reimbursement decision-making is challenging due to uncertain long-term outcomes. The availability of 5-year outcome data from the ELIANA trial and real-world evidence of tisagenlecleucel, the first chimeric antigen receptor T-cell (CAR-T) therapy, presents an opportunity to re-evaluate the predictions of prior cost-effectiveness analyses (CEAs).&lt;/p&gt;&lt;h3&gt;Objective&lt;/h3&gt;&lt;p&gt;To conduct a systematic literature review (SLR) of prior CEAs of tisagenlecleucel for pediatric/young adult relapsed or refractory acute lymphoblastic leukemia (r/r ALL) and evaluate the impact of recently available 5-year efficacy data from ELIANA and advances in CAR-T manufacturing in an updated CEA model.&lt;/p&gt;&lt;h3&gt;Methods&lt;/h3&gt;&lt;p&gt;OVID MEDLINE/Embase and health technology assessment (HTA) databases were searched for full-text economic evaluations in English reporting cost-effectiveness results for tisagenlecleucel for r/r ALL. Evaluations with publicly reported incremental cost-effectiveness ratios (ICERs) were included in the SLR. Study screening and data abstraction were conducted following PRISMA guidelines&lt;b&gt;.&lt;/b&gt; Data extracted included the country/currency, perspective, clinical trial evidence, model structures, long-term efficacy extrapolation approaches (i.e., overall survival [OS]), time horizon, discount rates, and outcomes (i.e., life years [LY], quality-adjusted LY [QALY], and ICERs). The CEA model reported in Wakase et al. was updated using 5-year OS data from ELIANA and the CAR-T infusion rate informed by real-world practice.&lt;/p&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;p&gt;Sixteen records corresponding to 15 unique studies were included in the SLR (11 publications and 5 HTA reports); all were conducted from the health care system perspective of the respective countries. Most studies found tisagenlecleucel to be cost effective, but all studies’ projected 3- and 5-year OS rates for tisagenlecleucel were lower than the observed 3- and 5-year rates, respectively, derived from 5-year ELIANA data. When applying updated OS projections from the most recent ELIANA data cut and higher infusion rates of 92.5% (per the real-world infusion rate)—96.0% (per the manufacturer success rate) to the CEA of Wakase et al., the associated QALYs for tisagenlecleucel increased from 11.6 to 14.6–15.0, and LYs increased from 13.3 to 17.0–17.5. Accordingly, the ICERs for tisagenlecleucel decreased from ¥2,035,071 to ¥1,787,988–¥1,789,048 versus blinatumomab and from ¥2,644,702 to ¥2,257,837–¥2,275,181 versus clofarabine combination therapy in the updated CEA model.&lt;/p&gt;&lt;h3&gt;Conclusions and Relevance&lt;/h3&gt;&lt;p&gt;Projections at launch of the likely cost effectiveness of tisagenlecleucel appear to have underestimated its ultimate economic value given more recent trial and real-world data. To balance uncertainty in initial valuation with the need to provide access to novel oncology therapies, payers can consider flexible reimbursement policies alongside on","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 5","pages":"749 - 765"},"PeriodicalIF":3.1,"publicationDate":"2024-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s40258-024-00882-4.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140811782","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}
引用次数: 0
Comparison of the Measurement Properties of EQ-5D-5L and SF-6Dv2 in COVID-19 Patients in China 中国 COVID-19 患者的 EQ-5D-5L 和 SF-6Dv2 测量特性比较。
IF 3.1 4区 医学
Applied Health Economics and Health Policy Pub Date : 2024-04-19 DOI: 10.1007/s40258-024-00881-5
Ningxin Ding, Huixuan Zhou, Chen Chen, Hui Chen, Yunfeng Shi
{"title":"Comparison of the Measurement Properties of EQ-5D-5L and SF-6Dv2 in COVID-19 Patients in China","authors":"Ningxin Ding,&nbsp;Huixuan Zhou,&nbsp;Chen Chen,&nbsp;Hui Chen,&nbsp;Yunfeng Shi","doi":"10.1007/s40258-024-00881-5","DOIUrl":"10.1007/s40258-024-00881-5","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background and Objectives&lt;/h3&gt;&lt;p&gt;There are limited studies comparing the health utility values of EQ-5D-5L and SF-6Dv2 within the same patient cohorts. The widespread transmission and recurring infections associated with Omicron variants amid the COVID-19 pandemic have resulted in substantial health detriments and increased utilisation of health care resources. This highlights the crucial need to assess the loss in quality-adjusted life years (QALYs). Therefore, this study aims to compare the ceiling and floor effects, agreement, correlation and responsiveness between EQ-5D-5L and SF-6Dv2 based on COVID-19 patients during the Omicron outbreak in China.&lt;/p&gt;&lt;h3&gt;Methods&lt;/h3&gt;&lt;p&gt;We recruited 694 COVID-19 patients across mainland China to participant in an online questionnaire survey from January to February 2023. The questionnaire encompassed queries concerning the sociodemographic and health details of the participants, who were requested to recollect their health status during and after experiencing COVID-19 using the EQ-5D-5L and SF-6Dv2 questionnaires. Epanechnikov kernel density plots were used to visualise the ceiling and floor effects for both instruments. Agreement was assessed by Bland–Altman graph and intraclass correlation coefficient (ICC). Correlation was evaluated using linear regression, Pearson’s correlation and Spearman’s correlation. The standardised response mean (SRM) and relative efficiency (RE) were used to examine the responsiveness of EQ-5D-5L and SF-6Dv2 at detecting the health improvement after COVID-19 infection and the difference in dichotomous health indicators.&lt;/p&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;p&gt;In total, 648 valid responses from patients aged 35.6 ± 15.0 years were involved in analysis. The EQ-5D-5L utility indices were 0.58 ± 0.33 and 0.92 ± 0.14 during and after COVID-19 infection, respectively, which were significantly higher than indices of the SF-6Dv2 utility (0.43 ± 0.31 and 0.81 ± 0.19, &lt;i&gt;p&lt;/i&gt; &lt; 0.001). A ceiling effect of EQ-5D-5L larger than that of SF-6Dv2 was observed during COVID-19 infection (49.5% vs 21.6%). Intraclass correlation coefficients between EQ-5D-5L and SF-6Dv2 during and after COVID-19 infection were 0.69 and 0.55, respectively. The utility indices of EQ-5D-5L and SF-6Dv2 were highly correlated, with Pearson’s correlation coefficients of 0.76 and 0.70 (&lt;i&gt;p&lt;/i&gt; &lt; 0.001) during and after COVID-19 infection, respectively. The spearman’s correlations were moderate to high between dimensions of EQ-5D-5L and SF-6Dv2 (&lt;i&gt;p&lt;/i&gt; &lt; 0.01). Both EQ-5D-5L and SF-6Dv2 were responsive to detect health improvement after COVID-19 and the differences in dichotomous health indicators.&lt;/p&gt;&lt;h3&gt;Conclusions&lt;/h3&gt;&lt;p&gt;The utility indices generated by EQ-5D-5L and SF-6Dv2 in COVID-19 patients demonstrate strong correlation and responsiveness. However, the agreement between these two instruments does not reach a satisfactory level. Consequently, these two measures cannot be used interchangeably. In situations where ","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":"22 4","pages":"555 - 568"},"PeriodicalIF":3.1,"publicationDate":"2024-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140683328","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
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