首页 > 最新文献

European Journal of Health Economics最新文献

英文 中文
Cost-effectiveness of two online interventions supporting self-care for eczema for parents/carers and young people. 支持家长/护理人员和青少年自我护理湿疹的两种在线干预措施的成本效益。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2024-01-09 DOI: 10.1007/s10198-023-01649-9
Tracey H Sach, Mary Onoja, Holly Clarke, Miriam Santer, Ingrid Muller, Taeko Becque, Beth Stuart, Julie Hooper, Mary Steele, Sylvia Wilczynska, Matthew J Ridd, Amanda Roberts, Amina Ahmed, Lucy Yardley, Paul Little, Kate Greenwell, Katy Sivyer, Jacqui Nuttall, Gareth Griffiths, Sandra Lawton, Sinéad M Langan, Laura Howells, Paul Leighton, Hywel C Williams, Kim S Thomas

Objective: To estimate the cost-effectiveness of online behavioral interventions (EczemaCareOnline.org.uk) designed to support eczema self-care management for parents/carers and young people from an NHS perspective.

Methods: Two within-trial economic evaluations, using regression-based approaches, adjusting for baseline and pre-specified confounder variables, were undertaken alongside two independent, pragmatic, parallel group, unmasked randomized controlled trials, recruiting through primary care. Trial 1 recruited 340 parents/carers of children aged 0-12 years and Trial 2 337 young people aged 13-25 years with eczema scored ≥ 5 on Patient-Oriented Eczema Measure (POEM). Participants were randomized (1:1) to online intervention plus usual care or usual care alone. Resource use, collected via medical notes review, was valued using published unit costs in UK £Sterling 2021. Quality-of-life was elicited using proxy CHU-9D in Trial 1 and self-report EQ-5D-5L in Trial 2.

Results: The intervention was dominant (cost saving and more effective) with a high probability of cost-effectiveness (> 68%) in most analyses. The exception was the complete case cost-utility analysis for Trial 1 (omitting participants with children aged < 2), with adjusted incremental cost savings of -£34.15 (95% CI  - 104.54 to 36.24) and incremental QALYs of - 0.003 (95% CI  - 0.021 to 0.015) producing an incremental cost per QALY of £12,466. In the secondary combined (Trials 1 and 2) cost-effectiveness analysis, the adjusted incremental cost was -£20.35 (95% CI  - 55.41 to 14.70) with incremental success (≥ 2-point change on POEM) of 10.3% (95% CI 2.3-18.1%).

Conclusion: The free at point of use online eczema self-management intervention was low cost to run and cost-effective.

Trial registration: This trial was registered prospectively with the ISRCTN registry (ISRCTN79282252). URL www.EczemaCareOnline.org.uk .

目的:从英国国家医疗服务体系(NHS)的角度评估在线行为干预(EczemaCareOnline.org.uk)的成本效益:从英国国家医疗服务体系(NHS)的角度评估旨在支持父母/照护者和青少年湿疹自我护理管理的在线行为干预(EczemaCareOnline.org.uk)的成本效益:在进行两项独立、务实、平行组、无掩蔽随机对照试验的同时,还进行了两项试验内经济评估,采用基于回归的方法,对基线和预先指定的混杂变量进行调整,并通过初级保健进行招募。试验1招募了340名0-12岁儿童的家长/监护人,试验2招募了337名13-25岁的年轻人,这些年轻人的湿疹在 "以患者为导向的湿疹测量"(POEM)中得分≥5分。参与者被随机分配(1:1)接受在线干预加常规护理或仅接受常规护理。通过病历审查收集的资源使用情况采用已公布的单位成本进行估价,单位为 2021 英镑。在试验 1 中,使用代用 CHU-9D 对生活质量进行评估;在试验 2 中,使用自我报告 EQ-5D-5L 对生活质量进行评估:在大多数分析中,干预占主导地位(节约成本且更有效),成本效益概率高(> 68%)。但试验 1 的全病例成本效用分析是个例外(省略了有年幼子女的参与者):免费在线湿疹自我管理干预的运行成本低且具有成本效益:该试验已在ISRCTN登记处进行了前瞻性登记(ISRCTN79282252)。网址:www.EczemaCareOnline.org.uk 。
{"title":"Cost-effectiveness of two online interventions supporting self-care for eczema for parents/carers and young people.","authors":"Tracey H Sach, Mary Onoja, Holly Clarke, Miriam Santer, Ingrid Muller, Taeko Becque, Beth Stuart, Julie Hooper, Mary Steele, Sylvia Wilczynska, Matthew J Ridd, Amanda Roberts, Amina Ahmed, Lucy Yardley, Paul Little, Kate Greenwell, Katy Sivyer, Jacqui Nuttall, Gareth Griffiths, Sandra Lawton, Sinéad M Langan, Laura Howells, Paul Leighton, Hywel C Williams, Kim S Thomas","doi":"10.1007/s10198-023-01649-9","DOIUrl":"10.1007/s10198-023-01649-9","url":null,"abstract":"<p><strong>Objective: </strong>To estimate the cost-effectiveness of online behavioral interventions (EczemaCareOnline.org.uk) designed to support eczema self-care management for parents/carers and young people from an NHS perspective.</p><p><strong>Methods: </strong>Two within-trial economic evaluations, using regression-based approaches, adjusting for baseline and pre-specified confounder variables, were undertaken alongside two independent, pragmatic, parallel group, unmasked randomized controlled trials, recruiting through primary care. Trial 1 recruited 340 parents/carers of children aged 0-12 years and Trial 2 337 young people aged 13-25 years with eczema scored ≥ 5 on Patient-Oriented Eczema Measure (POEM). Participants were randomized (1:1) to online intervention plus usual care or usual care alone. Resource use, collected via medical notes review, was valued using published unit costs in UK £Sterling 2021. Quality-of-life was elicited using proxy CHU-9D in Trial 1 and self-report EQ-5D-5L in Trial 2.</p><p><strong>Results: </strong>The intervention was dominant (cost saving and more effective) with a high probability of cost-effectiveness (> 68%) in most analyses. The exception was the complete case cost-utility analysis for Trial 1 (omitting participants with children aged < 2), with adjusted incremental cost savings of -£34.15 (95% CI  - 104.54 to 36.24) and incremental QALYs of - 0.003 (95% CI  - 0.021 to 0.015) producing an incremental cost per QALY of £12,466. In the secondary combined (Trials 1 and 2) cost-effectiveness analysis, the adjusted incremental cost was -£20.35 (95% CI  - 55.41 to 14.70) with incremental success (≥ 2-point change on POEM) of 10.3% (95% CI 2.3-18.1%).</p><p><strong>Conclusion: </strong>The free at point of use online eczema self-management intervention was low cost to run and cost-effective.</p><p><strong>Trial registration: </strong>This trial was registered prospectively with the ISRCTN registry (ISRCTN79282252). URL www.EczemaCareOnline.org.uk .</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11377600/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139405177","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Exploring non-iterative time trade-off methods for valuation of EQ-5D-5L health states. 探索用于评估 EQ-5D-5L 健康状况的非迭代时间权衡方法。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2023-12-17 DOI: 10.1007/s10198-023-01647-x
Zhihao Yang, Kim Rand, Elly Stolk, Jan Busschbach, Nan Luo

Introduction: The composite time trade-off (cTTO) method is used as the primary method for valuing EQ-5D-5L health states, but it requires intensive interviewer training and stringent quality control, which increases the burden of conducting cTTO studies. In this study, two non-iterative variants of the TTO method, non-stopping TTO (nTTO) and open-ended TTO (oTTO), were tested head-to-head with the cTTO method aiming to reduce the administration burden.

Methods: 31 EQ-5D-5L health states from an orthogonal array was selected and valued by a general public sample in China. Data were collected by 7 interviewers with all interviewers performed an equal number of interviews using all three TTO methods. We compared the value distribution, logical consistency, administration burden, and modeling performance of these three TTO methods.

Results: In total, 422 participants participated in the valuation interviews, with 139 using the nTTO method, 140 using the oTTO method, and 143 using the cTTO method. Both oTTO and nTTO methods saved around 10 min for conducting an interview. The mean values of three methods were similar with each method showed different characteristics in their value distributions. cTTO outperformed the other two methods in terms of modeling performance.

Discussion: Both non-iterative TTO methods showed potential for valuing EQ-5D health states, although their data distributions and modeling performance were inferior to the cTTO method. The results of this study showed the potential of these two alternative non-iterative TTO methods.

简介复合时间权衡法(cTTO)被用作评估 EQ-5D-5L 健康状况的主要方法,但它需要对访谈者进行强化培训和严格的质量控制,这增加了进行 cTTO 研究的负担。在本研究中,两种非迭代 TTO 方法的变体,即非停止 TTO(nTTO)和开放式 TTO(oTTO),与 cTTO 方法进行了正面交锋测试,旨在减少管理负担。数据由 7 名访问员收集,所有访问员使用三种 TTO 方法进行了相同次数的访问。我们比较了这三种 TTO 方法的价值分布、逻辑一致性、管理负担和建模性能:共有 422 名参与者参加了估值访谈,其中 139 人使用了 nTTO 方法,140 人使用了 oTTO 方法,143 人使用了 cTTO 方法。oTTO 和 nTTO 方法都节省了约 10 分钟的访谈时间。cTTO 在建模性能方面优于其他两种方法:讨论:两种非迭代 TTO 方法都显示出对 EQ-5D 健康状况进行估值的潜力,尽管它们的数据分布和建模性能都不如 cTTO 方法。本研究结果显示了这两种非迭代 TTO 方法的潜力。
{"title":"Exploring non-iterative time trade-off methods for valuation of EQ-5D-5L health states.","authors":"Zhihao Yang, Kim Rand, Elly Stolk, Jan Busschbach, Nan Luo","doi":"10.1007/s10198-023-01647-x","DOIUrl":"10.1007/s10198-023-01647-x","url":null,"abstract":"<p><strong>Introduction: </strong>The composite time trade-off (cTTO) method is used as the primary method for valuing EQ-5D-5L health states, but it requires intensive interviewer training and stringent quality control, which increases the burden of conducting cTTO studies. In this study, two non-iterative variants of the TTO method, non-stopping TTO (nTTO) and open-ended TTO (oTTO), were tested head-to-head with the cTTO method aiming to reduce the administration burden.</p><p><strong>Methods: </strong>31 EQ-5D-5L health states from an orthogonal array was selected and valued by a general public sample in China. Data were collected by 7 interviewers with all interviewers performed an equal number of interviews using all three TTO methods. We compared the value distribution, logical consistency, administration burden, and modeling performance of these three TTO methods.</p><p><strong>Results: </strong>In total, 422 participants participated in the valuation interviews, with 139 using the nTTO method, 140 using the oTTO method, and 143 using the cTTO method. Both oTTO and nTTO methods saved around 10 min for conducting an interview. The mean values of three methods were similar with each method showed different characteristics in their value distributions. cTTO outperformed the other two methods in terms of modeling performance.</p><p><strong>Discussion: </strong>Both non-iterative TTO methods showed potential for valuing EQ-5D health states, although their data distributions and modeling performance were inferior to the cTTO method. The results of this study showed the potential of these two alternative non-iterative TTO methods.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138810354","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Lifecycle model-based evaluation of infant 4CMenB vaccination in the UK. 基于生命周期模型的英国婴儿 4CMenB 疫苗接种评估。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2024-01-05 DOI: 10.1007/s10198-023-01654-y
J P Sevilla, Daniel Tortorice, David Kantor, John Regan, Kinga H Meszaros, Ekkehard C Beck, Najida Begum, David E Bloom

Objectives: Invasive meningococcal disease, an uncommon but severe disease, imposes catastrophic health and economic burdens. Cost-utility analysis (CUA) assumes separability in lifetime health and economic variables and cannot capture the full value of preventing such burdens. We overcome these limitations with a retrospective societal perspective cost-benefit analysis (CBA) of meningococcal serogroup B vaccination (4CMenB) of one infant cohort in the United Kingdom using a health-augmented lifecycle model (HALM) incorporating health's interactions with consumption, earnings, non-market time and financial risk.

Methods: We used a static Markov model of vaccination's health impact and an HALM to estimate the private willingness to pay (PWTP) for the intrinsic and instrumental value of health under perfect capital markets, financial risk protection in the absence of insurance against permanent disability, parental spillovers, and acute phase disability. We estimated social WTP (SWTP) incorporating social severity preferences. We estimated rates of return that inform health payer reimbursement decisions, finance ministry budgeting decisions, and legislature taxation decisions. An expert Advisory Board investigated the validity of applying the HALM to infant 4CMenB.

Results: The PWTP for a 2 + 1 vaccination schedule is £395, comprising £166 of disability insurance value, £79 of positive parental spillover value, £28 in the value of averting acute phase disability, and £122 in residual intrinsic and instrumental value of health. SWTP is £969.

Conclusions: HALM-based CBA provides an empirically richer, more utility-theoretically grounded approach to vaccine evaluation than CUA, demonstrating good value for money for legislatures (based on private values) and for all decision-makers (based on social values).

目标:侵袭性脑膜炎球菌病是一种不常见但严重的疾病,会带来灾难性的健康和经济负担。成本效用分析(CUA)假定终生健康和经济变量是可分离的,因此无法体现预防此类负担的全部价值。我们采用健康增强生命周期模型(HALM),结合健康与消费、收入、非市场时间和金融风险的相互作用,对英国一个婴儿队列的脑膜炎球菌血清 B 群疫苗接种(4CMenB)进行了回顾性社会视角成本效益分析(CBA),从而克服了这些局限性:我们使用疫苗接种对健康影响的静态马尔可夫模型和 HALM 来估算在完善的资本市场、没有永久性残疾保险的金融风险保护、父母溢出效应和急性期残疾的情况下,私人对健康内在价值和工具价值的支付意愿(PWTP)。我们估算了包含社会严重性偏好的社会 WTP(SWTP)。我们估算的回报率可为医疗支付方的报销决策、财政部的预算决策和立法机构的税收决策提供参考。一个专家咨询委员会调查了将 HALM 应用于婴儿 4CMenB 的有效性:结果:2 + 1 疫苗接种计划的公共收益预测值为 395 英镑,其中包括 166 英镑的残疾保险价值、79 英镑的父母正溢出价值、28 英镑的避免急性期残疾价值以及 122 英镑的剩余健康内在价值和工具价值。SWTP 为 969.英镑:与 CUA 相比,基于 HALM 的成本效益分析为疫苗评估提供了一种经验更丰富、效用理论基础更扎实的方法,为立法机构(基于私人价值)和所有决策者(基于社会价值)展示了良好的性价比。
{"title":"Lifecycle model-based evaluation of infant 4CMenB vaccination in the UK.","authors":"J P Sevilla, Daniel Tortorice, David Kantor, John Regan, Kinga H Meszaros, Ekkehard C Beck, Najida Begum, David E Bloom","doi":"10.1007/s10198-023-01654-y","DOIUrl":"10.1007/s10198-023-01654-y","url":null,"abstract":"<p><strong>Objectives: </strong>Invasive meningococcal disease, an uncommon but severe disease, imposes catastrophic health and economic burdens. Cost-utility analysis (CUA) assumes separability in lifetime health and economic variables and cannot capture the full value of preventing such burdens. We overcome these limitations with a retrospective societal perspective cost-benefit analysis (CBA) of meningococcal serogroup B vaccination (4CMenB) of one infant cohort in the United Kingdom using a health-augmented lifecycle model (HALM) incorporating health's interactions with consumption, earnings, non-market time and financial risk.</p><p><strong>Methods: </strong>We used a static Markov model of vaccination's health impact and an HALM to estimate the private willingness to pay (PWTP) for the intrinsic and instrumental value of health under perfect capital markets, financial risk protection in the absence of insurance against permanent disability, parental spillovers, and acute phase disability. We estimated social WTP (SWTP) incorporating social severity preferences. We estimated rates of return that inform health payer reimbursement decisions, finance ministry budgeting decisions, and legislature taxation decisions. An expert Advisory Board investigated the validity of applying the HALM to infant 4CMenB.</p><p><strong>Results: </strong>The PWTP for a 2 + 1 vaccination schedule is £395, comprising £166 of disability insurance value, £79 of positive parental spillover value, £28 in the value of averting acute phase disability, and £122 in residual intrinsic and instrumental value of health. SWTP is £969.</p><p><strong>Conclusions: </strong>HALM-based CBA provides an empirically richer, more utility-theoretically grounded approach to vaccine evaluation than CUA, demonstrating good value for money for legislatures (based on private values) and for all decision-makers (based on social values).</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11377635/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139106810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Screening strategy to advance HCV elimination in Italy: a cost-consequence analysis. 意大利消除丙型肝炎病毒的筛查策略:成本-后果分析。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2024-01-27 DOI: 10.1007/s10198-023-01652-0
Andrea Marcellusi, Francesco Saverio Mennini, Massimo Andreoni, Loreta A Kondili

Background and aims: Italy has the greatest burden of hepatitis C virus (HCV) infection in Western Europe. The screening strategy represents a crucial prevention tool to achieve HCV elimination in Italy. We evaluated the cost-consequences of different screening strategies for the diagnosis of HCV active infection in the birth cohort 1948-1968 to achieve the HCV elimination goal.

Methods: We designed a probabilistic model to estimate the clinical, and economic outcomes of different screening coverage uptakes, considering the direct costs of HCV management according to each liver fibrosis stage, in the Italian context. A decision probabilistic tree simulates 4 years of HCV testing of the 1948-1968 general population birth cohort, (15,485,565 individuals to be tested) considering different coverage rates. A No-screening scenario was compared with two alternative screening scenarios that represented different coverage rates each year: (1) Incremental approach (coverage rates equal to 5%, 10%, 30%, and 50% at years 1, 2, 3, and 4, respectively) and (2) Fast approach (50% coverage rate at years 1, 2, 3 and 4). Overall 106,200 cases were previously estimated to have an HCV active infection. A liver disease progression Markov model was considered for an additional 6 years (horizon-time 10 years).

Results: The highest increased number of deaths and clinical events are reported for the No-screening scenario (21,719 cumulative deaths at the end of ten years; 10,148 cases with HCC and/or 7618 cases with Decompensated Cirrhosis). Following the Fast-screening scenario, the reductions in clinical outcomes and deaths were higher compared with No-screening and Incremental-screening. At ten years time horizon, less than 5696 liver deaths (PSA CI95%: - 3873 to 7519), 3,549 HCC (PSA CI95%: - 2413 to 4684) and less than 3005 liver decompensations (PSA CI 95%: - 2104 to 3907) were estimated compared with the Incremental-scenario. The overall costs of the Fast-screening, including the costs of the DAA and liver disease management of the infected patients for 10 years, are estimated to be € 43,107,543 more than no-investment in screening and € 62,289,549 less compared with the overall costs estimated by the Incremental-scenario.

Conclusion: It is necessary to guarantee dedicated funds and efficiency of the system for the cost-efficacious screening of the 1948-1968 birth cohort in Italy. A delay in HCV diagnosis and treatment in the general population, yet not addressed for the HCV free-of-charge screening, will have important clinical and economic consequences in Italy.

背景和目的:意大利是西欧丙型肝炎病毒(HCV)感染负担最重的国家。筛查策略是意大利消除丙型肝炎病毒的重要预防工具。为实现消除丙型肝炎病毒(HCV)的目标,我们对 1948-1968 年出生队列中诊断丙型肝炎病毒(HCV)活动性感染的不同筛查策略的成本后果进行了评估:我们设计了一个概率模型来估算不同筛查覆盖率的临床和经济结果,同时考虑到在意大利每个肝纤维化阶段的 HCV 管理的直接成本。决策概率树模拟了对 1948-1968 年出生的普通人群(15,485,565 人接受检测)进行为期 4 年的 HCV 检测,并考虑了不同的覆盖率。将无筛查方案与代表每年不同覆盖率的两种备选筛查方案进行了比较:(1) 递增法(第 1、2、3 和 4 年的覆盖率分别为 5%、10%、30% 和 50%);(2) 快速法(第 1、2、3 和 4 年的覆盖率为 50%)。据此估算,共有 106,200 例患者感染了丙型肝炎病毒。肝病进展马尔可夫模型考虑了额外 6 年(水平时间 10 年):结果:在无筛查方案中,死亡人数和临床事件增加最多(10 年后累计死亡 21719 例;10148 例 HCC 和/或 7618 例失代偿期肝硬化)。与不筛查和增量筛查相比,快速筛查方案降低的临床结果和死亡人数更高。与增量筛查方案相比,在十年的时间跨度内,估计肝脏死亡病例少于 5696 例(PSA CI95%:- 3873 至 7519),HCC 病例少于 3549 例(PSA CI95%:- 2413 至 4684),肝脏失代偿病例少于 3005 例(PSA CI 95%:- 2104 至 3907)。与不投资筛查相比,快速筛查的总成本(包括 DAA 和受感染患者 10 年肝病管理的成本)估计增加 43 107 543 欧元,与增量方案估计的总成本相比,减少 62 289 549 欧元:结论:有必要保证专项资金和系统效率,以便在意大利对 1948-1968 年出生的人群进行具有成本效益的筛查。普通人群中的 HCV 诊断和治疗延迟问题尚未在 HCV 免费筛查中得到解决,这将对意大利的临床和经济产生重要影响。
{"title":"Screening strategy to advance HCV elimination in Italy: a cost-consequence analysis.","authors":"Andrea Marcellusi, Francesco Saverio Mennini, Massimo Andreoni, Loreta A Kondili","doi":"10.1007/s10198-023-01652-0","DOIUrl":"10.1007/s10198-023-01652-0","url":null,"abstract":"<p><strong>Background and aims: </strong>Italy has the greatest burden of hepatitis C virus (HCV) infection in Western Europe. The screening strategy represents a crucial prevention tool to achieve HCV elimination in Italy. We evaluated the cost-consequences of different screening strategies for the diagnosis of HCV active infection in the birth cohort 1948-1968 to achieve the HCV elimination goal.</p><p><strong>Methods: </strong>We designed a probabilistic model to estimate the clinical, and economic outcomes of different screening coverage uptakes, considering the direct costs of HCV management according to each liver fibrosis stage, in the Italian context. A decision probabilistic tree simulates 4 years of HCV testing of the 1948-1968 general population birth cohort, (15,485,565 individuals to be tested) considering different coverage rates. A No-screening scenario was compared with two alternative screening scenarios that represented different coverage rates each year: (1) Incremental approach (coverage rates equal to 5%, 10%, 30%, and 50% at years 1, 2, 3, and 4, respectively) and (2) Fast approach (50% coverage rate at years 1, 2, 3 and 4). Overall 106,200 cases were previously estimated to have an HCV active infection. A liver disease progression Markov model was considered for an additional 6 years (horizon-time 10 years).</p><p><strong>Results: </strong>The highest increased number of deaths and clinical events are reported for the No-screening scenario (21,719 cumulative deaths at the end of ten years; 10,148 cases with HCC and/or 7618 cases with Decompensated Cirrhosis). Following the Fast-screening scenario, the reductions in clinical outcomes and deaths were higher compared with No-screening and Incremental-screening. At ten years time horizon, less than 5696 liver deaths (PSA CI95%: - 3873 to 7519), 3,549 HCC (PSA CI95%: - 2413 to 4684) and less than 3005 liver decompensations (PSA CI 95%: - 2104 to 3907) were estimated compared with the Incremental-scenario. The overall costs of the Fast-screening, including the costs of the DAA and liver disease management of the infected patients for 10 years, are estimated to be € 43,107,543 more than no-investment in screening and € 62,289,549 less compared with the overall costs estimated by the Incremental-scenario.</p><p><strong>Conclusion: </strong>It is necessary to guarantee dedicated funds and efficiency of the system for the cost-efficacious screening of the 1948-1968 birth cohort in Italy. A delay in HCV diagnosis and treatment in the general population, yet not addressed for the HCV free-of-charge screening, will have important clinical and economic consequences in Italy.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139572023","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
The impact of an 'evergreening' strategy nearing patent expiration on the uptake of biosimilars and public healthcare costs: a case study on the introduction of a second administration form of trastuzumab in The Netherlands. 临近专利到期的 "常青化 "战略对生物仿制药吸收和公共医疗成本的影响:荷兰引入曲妥珠单抗第二给药形式的案例研究。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2024-01-08 DOI: 10.1007/s10198-023-01648-w
Ghyli Kirshner, Peter Makai, Chiara Brouns, Lonneke Timmers, Ron Kemp

In this paper, we explore dynamic market share and public healthcare costs of trastuzumab's evergreening (subcutaneous) variant during introduction of trastuzumab's competitive biosimilar variants in the Netherlands. We used a time series design to assess dynamic market share of trastuzumab's evergreening variant after introducing trastuzumab's biosimilar variants, focusing on the number of treatments and patients. The public healthcare costs of this evergreening strategy were estimated using administrative claims data. Our results show that the original trastuzumab was completely replaced by the subcutaneous and biosimilar variants. The uptake of the subcutaneous form peaked at 50% market share but after the introduction of biosimilars progressively reduced to a market share of 20%, resulting in a more competitive market structure. The public healthcare costs for trastuzumab significantly decreased after the introduction of the biosimilars. After the introduction of the biosimilars, a substantial price drop is visible, with the subcutaneous version, still under patent, also falling sharply in price but less strongly than the iv/biosimilar version. As the costs are publicly funded, we recommend a more explicit societal debate to consider if the potential benefits of subcutaneous Herceptin® (and other similar medicines) are worth the additional costs, and at which price it should be reimbursed as the part of the benefit package.

本文探讨了在荷兰引入曲妥珠单抗竞争性生物类似药变体期间,曲妥珠单抗常青(皮下注射)变体的动态市场份额和公共医疗成本。在引入曲妥珠单抗的生物类似药变体后,我们采用时间序列设计来评估曲妥珠单抗常青变体的动态市场份额,重点关注治疗次数和患者人数。我们利用行政报销数据估算了这一常青战略的公共医疗成本。我们的结果表明,原来的曲妥珠单抗完全被皮下注射和生物类似物变体所取代。皮下注射剂的市场占有率最高时达到 50%,但在引入生物仿制药后,其市场占有率逐渐降至 20%,从而形成了一个更具竞争性的市场结构。引入生物仿制药后,曲妥珠单抗的公共医疗成本大幅下降。引入生物仿制药后,价格大幅下降,仍在专利期内的皮下注射版价格也大幅下降,但降幅小于静脉注射/生物仿制药。由于费用由政府资助,我们建议开展更明确的社会辩论,以考虑皮下注射 Herceptin®(及其他类似药物)的潜在益处是否值得支付额外费用,以及作为一揽子福利的一部分应按何种价格报销。
{"title":"The impact of an 'evergreening' strategy nearing patent expiration on the uptake of biosimilars and public healthcare costs: a case study on the introduction of a second administration form of trastuzumab in The Netherlands.","authors":"Ghyli Kirshner, Peter Makai, Chiara Brouns, Lonneke Timmers, Ron Kemp","doi":"10.1007/s10198-023-01648-w","DOIUrl":"10.1007/s10198-023-01648-w","url":null,"abstract":"<p><p>In this paper, we explore dynamic market share and public healthcare costs of trastuzumab's evergreening (subcutaneous) variant during introduction of trastuzumab's competitive biosimilar variants in the Netherlands. We used a time series design to assess dynamic market share of trastuzumab's evergreening variant after introducing trastuzumab's biosimilar variants, focusing on the number of treatments and patients. The public healthcare costs of this evergreening strategy were estimated using administrative claims data. Our results show that the original trastuzumab was completely replaced by the subcutaneous and biosimilar variants. The uptake of the subcutaneous form peaked at 50% market share but after the introduction of biosimilars progressively reduced to a market share of 20%, resulting in a more competitive market structure. The public healthcare costs for trastuzumab significantly decreased after the introduction of the biosimilars. After the introduction of the biosimilars, a substantial price drop is visible, with the subcutaneous version, still under patent, also falling sharply in price but less strongly than the iv/biosimilar version. As the costs are publicly funded, we recommend a more explicit societal debate to consider if the potential benefits of subcutaneous Herceptin<sup>®</sup> (and other similar medicines) are worth the additional costs, and at which price it should be reimbursed as the part of the benefit package.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11377641/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139378796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Looking inside the lab: a systematic literature review of economic experiments in health service provision. 观察实验室内部:关于医疗服务经济实验的系统文献综述。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2024-01-11 DOI: 10.1007/s10198-023-01662-y
Massimo Finocchiaro Castro, Calogero Guccio, Domenica Romeo

Experimental economics is, nowadays, a well-established approach to investigate agents' behavior under economic incentives. In the last decade, a fast-growing number of studies have focused on the application of experimental methodology to health policy issues. The results of that stream of literature have been intriguing and strongly policy oriented. However, those findings are scattered between different health-related topics, making it difficult to grasp the overall state-of-the-art. Hence, to make the main contributions understandable at a glance, we conduct a systematic literature review of laboratory experiments on the supply of health services. Of the 1248 articles retrieved from 2011, 56 articles published in peer-review journals have met our inclusion criteria. Thus, we have described the experimental designs of each of the selected papers and we have classified them according to their main area of interest.

如今,实验经济学已成为研究经济激励下行为主体行为的一种行之有效的方法。在过去十年中,越来越多的研究集中于将实验方法应用于卫生政策问题。这些研究成果引人入胜,并具有强烈的政策导向性。然而,这些研究成果分散在不同的健康相关主题中,因此很难把握整体的最新进展。因此,为了使主要贡献一目了然,我们对有关医疗服务供应的实验室实验进行了系统的文献综述。在 2011 年检索到的 1248 篇文章中,有 56 篇发表在同行评审期刊上的文章符合我们的纳入标准。因此,我们对每篇入选论文的实验设计进行了描述,并根据其主要关注领域进行了分类。
{"title":"Looking inside the lab: a systematic literature review of economic experiments in health service provision.","authors":"Massimo Finocchiaro Castro, Calogero Guccio, Domenica Romeo","doi":"10.1007/s10198-023-01662-y","DOIUrl":"10.1007/s10198-023-01662-y","url":null,"abstract":"<p><p>Experimental economics is, nowadays, a well-established approach to investigate agents' behavior under economic incentives. In the last decade, a fast-growing number of studies have focused on the application of experimental methodology to health policy issues. The results of that stream of literature have been intriguing and strongly policy oriented. However, those findings are scattered between different health-related topics, making it difficult to grasp the overall state-of-the-art. Hence, to make the main contributions understandable at a glance, we conduct a systematic literature review of laboratory experiments on the supply of health services. Of the 1248 articles retrieved from 2011, 56 articles published in peer-review journals have met our inclusion criteria. Thus, we have described the experimental designs of each of the selected papers and we have classified them according to their main area of interest.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11377526/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139425979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
On spillovers in economic evaluations: definition, mapping review and research agenda. 经济评价中的溢出效应:定义、绘图审查和研究议程。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2024-01-23 DOI: 10.1007/s10198-023-01658-8
María J Mendoza-Jiménez, Job van Exel, Werner Brouwer

An important issue in economic evaluations is determining whether all relevant impacts are considered, given the perspective chosen for the analysis. Acknowledging that patients are not isolated individuals has important implications in this context. Increasingly, the term "spillovers" is used to label consequences of health interventions on others. However, a clear definition of spillovers is lacking, and as a result, the scope of the concept remains unclear. In this study, we aim to clarify the concept of spillovers by proposing a definition applicable in health economic evaluations. To illustrate the implications of this definition, we highlight the diversity of potential spillovers through an expanded impact inventory and conduct a mapping review that outlines the evidence base for the different types of spillovers. In the context of economic evaluations of health interventions, we define spillovers as all impacts from an intervention on all parties or entities other than the users of the intervention under evaluation. This definition encompasses a broader range of potential costs and effects, beyond informal caregivers and family members. The expanded impact inventory enables a systematic approach to identifying broader impacts of health interventions. The mapping review shows that the relevance of different types of spillovers is context-specific. Some spillovers are regularly included in economic evaluations, although not always recognised as such, while others are not. A consistent use of the term "spillovers", improved measurement of these costs and effects, and increased transparency in reporting them are still necessary. To that end, we propose a research agenda.

经济评估中的一个重要问题是,根据分析所选的角度,确定是否考虑了所有相关影响。承认患者不是孤立的个体在这方面具有重要意义。人们越来越多地使用 "溢出效应 "一词来描述医疗干预措施对他人造成的影响。然而,目前还缺乏对外溢效应的明确定义,因此,这一概念的范围仍不明确。在本研究中,我们提出了一个适用于卫生经济评估的定义,旨在澄清外溢效应的概念。为了说明该定义的含义,我们通过扩大影响清单来强调潜在溢出效应的多样性,并进行了一次绘图审查,概述了不同类型溢出效应的证据基础。在对健康干预措施进行经济评价时,我们将外溢效应定义为干预措施对被评价干预措施使用者以外的所有各方或实体产生的所有影响。这一定义涵盖了非正式护理人员和家庭成员之外的更广泛的潜在成本和影响。扩大的影响清单能够以系统的方法确定卫生干预措施的更广泛影响。图谱审查表明,不同类型外溢效应的相关性因具体情况而异。有些外溢效应经常被纳入经济评价,尽管并不总是得到认可,而有些则没有。仍有必要统一使用 "外溢效应 "一词,改进对这些成本和效应的衡量,并提高报告的透明度。为此,我们提出了一项研究议程。
{"title":"On spillovers in economic evaluations: definition, mapping review and research agenda.","authors":"María J Mendoza-Jiménez, Job van Exel, Werner Brouwer","doi":"10.1007/s10198-023-01658-8","DOIUrl":"10.1007/s10198-023-01658-8","url":null,"abstract":"<p><p>An important issue in economic evaluations is determining whether all relevant impacts are considered, given the perspective chosen for the analysis. Acknowledging that patients are not isolated individuals has important implications in this context. Increasingly, the term \"spillovers\" is used to label consequences of health interventions on others. However, a clear definition of spillovers is lacking, and as a result, the scope of the concept remains unclear. In this study, we aim to clarify the concept of spillovers by proposing a definition applicable in health economic evaluations. To illustrate the implications of this definition, we highlight the diversity of potential spillovers through an expanded impact inventory and conduct a mapping review that outlines the evidence base for the different types of spillovers. In the context of economic evaluations of health interventions, we define spillovers as all impacts from an intervention on all parties or entities other than the users of the intervention under evaluation. This definition encompasses a broader range of potential costs and effects, beyond informal caregivers and family members. The expanded impact inventory enables a systematic approach to identifying broader impacts of health interventions. The mapping review shows that the relevance of different types of spillovers is context-specific. Some spillovers are regularly included in economic evaluations, although not always recognised as such, while others are not. A consistent use of the term \"spillovers\", improved measurement of these costs and effects, and increased transparency in reporting them are still necessary. To that end, we propose a research agenda.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11377364/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139520634","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Impact of Long COVID on productivity and informal caregiving. 长 COVID 对生产力和非正式护理的影响。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-09-01 Epub Date: 2023-12-26 DOI: 10.1007/s10198-023-01653-z
Joseph Kwon, Ruairidh Milne, Clare Rayner, Román Rocha Lawrence, Jordan Mullard, Ghazala Mir, Brendan Delaney, Manoj Sivan, Stavros Petrou

Background: Around 2 million people in the UK suffer from Long COVID (LC). Of concern is the disease impact on productivity and informal care burden. This study aimed to quantify and value productivity losses and informal care receipt in a sample of LC patients in the UK.

Methods: The target population comprised LC patients referred to LC specialist clinics. The questionnaires included a health economics questionnaire (HEQ) measuring productivity impacts, informal care receipt and service utilisation, EQ-5D-5L, C19-YRS LC condition-specific measure, and sociodemographic and COVID-19 history variables. Outcomes were changes from the incident infection resulting in LC to the month preceding the survey in paid work status/h, work income, work performance and informal care receipt. The human capital approach valued productivity losses; the proxy goods method valued caregiving hours. The values were extrapolated nationally using published prevalence data. Multilevel regressions, nested by region, estimated associations between the outcomes and patient characteristics.

Results: 366 patients responded to HEQ (mean LC duration 449.9 days). 51.7% reduced paid work hours relative to the pre-infection period. Mean monthly work income declined by 24.5%. The average aggregate value of productivity loss since incident infection was £10,929 (95% bootstrap confidence interval £8,844-£13,014) and £5.7 billion (£3.8-£7.6 billion) extrapolated nationally. The corresponding values for informal caregiving were £8,726 (£6,247-£11,204) and £4.8 billion (£2.6-£7.0 billion). Multivariate analyses found significant associations between each outcome and health utility and C19-YRS subscale scores.

Conclusion: LC significantly impacts productivity losses and provision of informal care, exacerbated by high national prevalence of LC.

背景:英国约有 200 万人患有长期慢性阻塞性肺病 (LC)。令人担忧的是疾病对生产力和非正式护理负担的影响。本研究旨在对英国 LC 患者的生产力损失和接受非正规护理的情况进行量化和估价:目标人群包括转诊至 LC 专科诊所的 LC 患者。调查问卷包括一份健康经济学调查问卷(HEQ),用于测量生产力影响、接受非正规护理和服务利用率、EQ-5D-5L、C19-YRS LC 条件特异性测量以及社会人口学和 COVID-19 病史变量。结果是指从导致 LC 的感染事件到调查前一个月期间,有偿工作状况/h、工作收入、工作表现和接受非正规护理情况的变化。人力资本法估算的是生产力损失;替代品法估算的是护理时间。这些数值是利用已公布的流行率数据推断出的全国性数值。按地区嵌套的多层次回归估算了结果与患者特征之间的关联:366名患者对HEQ做出了回应(平均LC持续时间为449.9天)。与感染前相比,51.7%的患者减少了带薪工作时间。平均每月工作收入减少了 24.5%。自感染事件发生以来,生产力损失的平均总值为 10,929 英镑(95% 引导置信区间为 8,844 - 13,014 英镑),推断全国范围内的生产力损失为 57 亿英镑(38 - 76 亿英镑)。非正式护理的相应数值为 8726 英镑(6247-11204 英镑)和 48 亿英镑(26-70 亿英镑)。多变量分析发现,每种结果与健康效用和 C19-YRS 分量表评分之间都存在显著关联:LC 严重影响生产力损失和非正规护理的提供,而全国 LC 的高患病率又加剧了这一影响。
{"title":"Impact of Long COVID on productivity and informal caregiving.","authors":"Joseph Kwon, Ruairidh Milne, Clare Rayner, Román Rocha Lawrence, Jordan Mullard, Ghazala Mir, Brendan Delaney, Manoj Sivan, Stavros Petrou","doi":"10.1007/s10198-023-01653-z","DOIUrl":"10.1007/s10198-023-01653-z","url":null,"abstract":"<p><strong>Background: </strong>Around 2 million people in the UK suffer from Long COVID (LC). Of concern is the disease impact on productivity and informal care burden. This study aimed to quantify and value productivity losses and informal care receipt in a sample of LC patients in the UK.</p><p><strong>Methods: </strong>The target population comprised LC patients referred to LC specialist clinics. The questionnaires included a health economics questionnaire (HEQ) measuring productivity impacts, informal care receipt and service utilisation, EQ-5D-5L, C19-YRS LC condition-specific measure, and sociodemographic and COVID-19 history variables. Outcomes were changes from the incident infection resulting in LC to the month preceding the survey in paid work status/h, work income, work performance and informal care receipt. The human capital approach valued productivity losses; the proxy goods method valued caregiving hours. The values were extrapolated nationally using published prevalence data. Multilevel regressions, nested by region, estimated associations between the outcomes and patient characteristics.</p><p><strong>Results: </strong>366 patients responded to HEQ (mean LC duration 449.9 days). 51.7% reduced paid work hours relative to the pre-infection period. Mean monthly work income declined by 24.5%. The average aggregate value of productivity loss since incident infection was £10,929 (95% bootstrap confidence interval £8,844-£13,014) and £5.7 billion (£3.8-£7.6 billion) extrapolated nationally. The corresponding values for informal caregiving were £8,726 (£6,247-£11,204) and £4.8 billion (£2.6-£7.0 billion). Multivariate analyses found significant associations between each outcome and health utility and C19-YRS subscale scores.</p><p><strong>Conclusion: </strong>LC significantly impacts productivity losses and provision of informal care, exacerbated by high national prevalence of LC.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11377524/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139038200","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
The added value of the cognition, dining, gastrointestinal problems, sleep and tiredness bolt-on dimensions to the EQ-5D-5L in patients with coeliac disease. 认知、就餐、胃肠道问题、睡眠和疲劳等方面对 EQ-5D-5L 的附加价值。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-08-30 DOI: 10.1007/s10198-024-01719-6
M Mercédesz Angyal, Mathieu F Janssen, Péter L Lakatos, Valentin Brodszky, Fanni Rencz

Objectives: Multiple studies suggest that the EQ-5D may overestimate health-related quality of life (HRQoL) in patients with coeliac disease (CD). We aimed to develop and psychometrically test potentially relevant bolt-on dimensions to improve the measurement performance of the EQ-5D-5L in CD patients.

Methods: The development and selection of bolt-ons were informed by a literature review on HRQoL in CD, expert and patient input. A cross-sectional online survey was conducted amongst 312 adult CD patients. Respondents completed the EQ-5D-5L, two condition-specific bolt-ons newly-developed for the present study [dining (DI) and gastrointestinal problems (GI)] and three existing bolt-ons [cognition (CO), sleep (SL) and tiredness (TI)]. The following psychometric properties were tested: ceiling, informativity, convergent and known-group validity, and dimensionality (confirmatory factor analysis).

Results: Adding the TI, SL, GI, DI and CO individual bolt-ons reduced the ceiling of the EQ-5D-5L (39%) to 17%, 23%, 24%, 26% and 37%, respectively. GI excelled with strong convergent validity with the Gastrointestinal Symptom Rating Scale total score (rs=0.71) and improved the discriminatory power for all known-groups. GI was the only bolt-on loading on a different factor from the five core dimensions, whereas the other four bolt-ons loaded onto the same 'psychosocial health' factor as the EQ-5D-5L anxiety/depression dimension.

Conclusion: The DI, GI, SL and TI bolt-ons, especially the GI, enhance the validity of EQ-5D-5L in patients with CD, suggesting their value in capturing important HRQoL aspects potentially missed by the five core dimensions. These bolt-ons can be used in sensitivity analyses supporting health technology assessments and subsequent resource allocation decisions.

目的:多项研究表明,EQ-5D 可能会高估乳糜泻(CD)患者的健康相关生活质量(HRQoL)。我们旨在开发潜在的相关附加维度并对其进行心理测试,以提高 EQ-5D-5L 在 CD 患者中的测量性能:方法:根据有关 CD 患者 HRQoL 的文献综述、专家和患者的意见,开发并选择了附加维度。对 312 名成年 CD 患者进行了横断面在线调查。受访者填写了 EQ-5D-5L、两个为本研究新开发的疾病特异性指标(就餐(DI)和胃肠道问题(GI))和三个现有指标(认知(CO)、睡眠(SL)和疲劳(TI))。对以下心理测量特性进行了测试:上限、信息性、收敛性和已知组有效性以及维度(确证因子分析):结果:添加 TI、SL、GI、DI 和 CO 单项后,EQ-5D-5L 的上限(39%)分别降至 17%、23%、24%、26% 和 37%。胃肠道症状量表与胃肠道症状量表总分(rs=0.71)具有很强的收敛有效性,并提高了所有已知组别的判别能力。胃肠道症状是唯一一个与五个核心维度不同的因子,而其他四个因子与 EQ-5D-5L 焦虑/抑郁维度一样,都是 "社会心理健康 "因子:结论:DI、GI、SL 和 TI 附加因子(尤其是 GI)提高了 EQ-5D-5L 在 CD 患者中的有效性,表明它们在捕捉五个核心维度可能遗漏的重要 HRQoL 方面具有价值。这些附加项目可用于支持健康技术评估和后续资源分配决策的敏感性分析。
{"title":"The added value of the cognition, dining, gastrointestinal problems, sleep and tiredness bolt-on dimensions to the EQ-5D-5L in patients with coeliac disease.","authors":"M Mercédesz Angyal, Mathieu F Janssen, Péter L Lakatos, Valentin Brodszky, Fanni Rencz","doi":"10.1007/s10198-024-01719-6","DOIUrl":"https://doi.org/10.1007/s10198-024-01719-6","url":null,"abstract":"<p><strong>Objectives: </strong>Multiple studies suggest that the EQ-5D may overestimate health-related quality of life (HRQoL) in patients with coeliac disease (CD). We aimed to develop and psychometrically test potentially relevant bolt-on dimensions to improve the measurement performance of the EQ-5D-5L in CD patients.</p><p><strong>Methods: </strong>The development and selection of bolt-ons were informed by a literature review on HRQoL in CD, expert and patient input. A cross-sectional online survey was conducted amongst 312 adult CD patients. Respondents completed the EQ-5D-5L, two condition-specific bolt-ons newly-developed for the present study [dining (DI) and gastrointestinal problems (GI)] and three existing bolt-ons [cognition (CO), sleep (SL) and tiredness (TI)]. The following psychometric properties were tested: ceiling, informativity, convergent and known-group validity, and dimensionality (confirmatory factor analysis).</p><p><strong>Results: </strong>Adding the TI, SL, GI, DI and CO individual bolt-ons reduced the ceiling of the EQ-5D-5L (39%) to 17%, 23%, 24%, 26% and 37%, respectively. GI excelled with strong convergent validity with the Gastrointestinal Symptom Rating Scale total score (r<sub>s</sub>=0.71) and improved the discriminatory power for all known-groups. GI was the only bolt-on loading on a different factor from the five core dimensions, whereas the other four bolt-ons loaded onto the same 'psychosocial health' factor as the EQ-5D-5L anxiety/depression dimension.</p><p><strong>Conclusion: </strong>The DI, GI, SL and TI bolt-ons, especially the GI, enhance the validity of EQ-5D-5L in patients with CD, suggesting their value in capturing important HRQoL aspects potentially missed by the five core dimensions. These bolt-ons can be used in sensitivity analyses supporting health technology assessments and subsequent resource allocation decisions.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142114596","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
Trends in NICE technology appraisals of non-small cell lung cancer drugs over the last decade. 过去十年 NICE 对非小细胞肺癌药物的技术评估趋势。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2024-08-30 DOI: 10.1007/s10198-024-01711-0
Lotte Westerink, Sharon Wolters, Guiling Zhou, Arjan Postma, Cornelis Boersma, Job Frank Martien van Boven, Maarten Jacobus Postma

Objectives: The aim of this study is to analyse the trends in technology appraisals for non-small cell lung cancer (NSCLC) treatments performed by the National Institute for Health and Care Excellence (NICE) over the last ten years.

Methods: A systematic search was conducted for single technology appraisals of NSCLC drugs in the online NICE database from 2012 to 2022. Search terms used were 'non small cell lung cancer', and 'NSCLC'. Appraisals that were under development or terminated as well as multiple technology appraisals were considered out of scope.

Results: In the 30 included appraisals for targeted therapies and immunotherapies within NSCLC, a total of 53 different comparators were included by NICE for 41 assorted indications or subgroups. Partitioned survival models were most frequently used, often including three health states and time horizons of up to 30 years. Throughout the decade the use of indirect comparisons was high and became more established and complex over time. Of all appraisals, 90% positively recommended the treatment for use in the UK.

Conclusion: Technology appraisals became more complex over time due to the emergence of targeted therapies and immunotherapies, leading to multiple different indications, subpopulations and comparators that needed to be included in appraisals. Partitioned Survival Analysis (PartSA) models became the cornerstone within NSCLC, with time horizons up to 30 years and over time methods for indirect treatment comparisons became more established. The majority of the appraisals resulted in a positive recommendation for reimbursement.

研究目的本研究旨在分析美国国家健康与护理卓越研究所(NICE)在过去十年间对非小细胞肺癌(NSCLC)治疗进行技术鉴定的趋势:在NICE在线数据库中对2012年至2022年NSCLC药物的单项技术评估进行了系统检索。搜索关键词为 "非小细胞肺癌 "和 "NSCLC"。正在开发或已终止的评估以及多项技术评估均被视为超出范围:在纳入的 30 项 NSCLC 靶向疗法和免疫疗法评估中,NICE 针对 41 种不同的适应症或亚组共纳入了 53 种不同的参照物。最常使用的是分区生存模型,通常包括三种健康状态和长达 30 年的时间跨度。在这十年中,间接比较法的使用率很高,而且随着时间的推移变得越来越成熟和复杂。在所有评估中,90%的评估积极推荐在英国使用该疗法:结论:由于靶向疗法和免疫疗法的出现,技术评估随着时间的推移变得更加复杂,导致评估中需要纳入多种不同的适应症、亚人群和比较对象。分区生存分析(PartSA)模型成为 NSCLC 的基石,时间跨度长达 30 年,随着时间的推移,间接治疗比较的方法也越来越成熟。大多数评估都提出了积极的报销建议。
{"title":"Trends in NICE technology appraisals of non-small cell lung cancer drugs over the last decade.","authors":"Lotte Westerink, Sharon Wolters, Guiling Zhou, Arjan Postma, Cornelis Boersma, Job Frank Martien van Boven, Maarten Jacobus Postma","doi":"10.1007/s10198-024-01711-0","DOIUrl":"https://doi.org/10.1007/s10198-024-01711-0","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of this study is to analyse the trends in technology appraisals for non-small cell lung cancer (NSCLC) treatments performed by the National Institute for Health and Care Excellence (NICE) over the last ten years.</p><p><strong>Methods: </strong>A systematic search was conducted for single technology appraisals of NSCLC drugs in the online NICE database from 2012 to 2022. Search terms used were 'non small cell lung cancer', and 'NSCLC'. Appraisals that were under development or terminated as well as multiple technology appraisals were considered out of scope.</p><p><strong>Results: </strong>In the 30 included appraisals for targeted therapies and immunotherapies within NSCLC, a total of 53 different comparators were included by NICE for 41 assorted indications or subgroups. Partitioned survival models were most frequently used, often including three health states and time horizons of up to 30 years. Throughout the decade the use of indirect comparisons was high and became more established and complex over time. Of all appraisals, 90% positively recommended the treatment for use in the UK.</p><p><strong>Conclusion: </strong>Technology appraisals became more complex over time due to the emergence of targeted therapies and immunotherapies, leading to multiple different indications, subpopulations and comparators that needed to be included in appraisals. Partitioned Survival Analysis (PartSA) models became the cornerstone within NSCLC, with time horizons up to 30 years and over time methods for indirect treatment comparisons became more established. The majority of the appraisals resulted in a positive recommendation for reimbursement.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":null,"pages":null},"PeriodicalIF":3.1,"publicationDate":"2024-08-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142114597","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"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
期刊
European Journal of Health Economics
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1