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Population normative data for OxCAP-MH capability scores. OxCAP-MH 能力评分的人群常模数据。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 Epub Date: 2024-05-24 DOI: 10.1007/s10198-024-01696-w
Péter György Balázs, Agata Łaszewska, Judit Simon, Valentin Brodszky

Aim: The study aims to establish the first set of normative data for OxCAP-MH capability instrument and to examine its association with sociodemographic and anxiety/depression severity variables.

Methods: A large-sample cross-sectional online survey was conducted among the Hungarian adult general population in 2021. OxCAP-MH standardized mean scores were compared across age, sex, education level, residence, employment, and marital status. Linear regression analysis was employed to determine the impact of sociodemographic and anxiety/depression severity on the OxCAP-MH score.

Results: In total, N = 2000 individuals completed the survey. The sample mean age was 47.1, with female majority (53.4%). Most respondents had completed primary education (51%), were active on labour market (52.4%), lived in larger cities (70.0%), and were married/in relationship (61.1%). Nearly half of the participants reported experiencing depression (48.5%), anxiety (44.3%), and 38.6% reported having both. The mean OxCAP-MH score for the total sample was 67.2 (SD = 14.4), the highest in the non-depressed (74.4) and non-anxious (73.6) subgroups, the lowest among those with extremely severe depression (45.0) and severe anxiety (47.7). Regression results indicated that older individuals (by β = 0.1), males (β = 2.3), those with secondary or higher education (β = 2.7 and 4.5) and students (β = 6.8) had significantly (p<0.01) higher mental capabilities. Respondents with mild, moderate, severe, or extremely severe depression (β =  -6.6, -9.6, -13.8, -18.3) and those with mild, moderate, or severe anxiety (β =  -4.1, -7.7, -10.3) had lower capability scores.

Conclusion: The OxCAP-MH instrument effectively differentiated capabilities across sociodemographic groups and highlighting the impact of depression and anxiety severity on general population's mental capability.

目的:本研究旨在为 OxCAP-MH 能力工具建立第一套标准数据,并研究其与社会人口学和焦虑/抑郁严重程度变量之间的关联:方法: 2021 年对匈牙利成年普通人群进行了一次大样本横截面在线调查。比较了不同年龄、性别、教育程度、居住地、就业和婚姻状况的 OxCAP-MH 标准化平均得分。采用线性回归分析确定社会人口统计学和焦虑/抑郁严重程度对 OxCAP-MH 分数的影响:共有 N = 2000 人完成了调查。样本平均年龄为 47.1 岁,女性占多数(53.4%)。大多数受访者完成了初等教育(51%),活跃于劳动力市场(52.4%),居住在大城市(70.0%),已婚/恋爱中(61.1%)。近一半的受访者表示患有抑郁症(48.5%)和焦虑症(44.3%),38.6%的受访者表示同时患有抑郁症和焦虑症。总样本的 OxCAP-MH 平均得分为 67.2(标准差 = 14.4),非抑郁(74.4)和非焦虑(73.6)亚组的得分最高,极度严重抑郁(45.0)和严重焦虑(47.7)亚组的得分最低。回归结果表明,年龄较大者(β = 0.1)、男性(β = 2.3)、受过中等或高等教育者(β = 2.7 和 4.5)和学生(β = 6.8)的焦虑程度明显(p 结论):OxCAP-MH工具有效区分了不同社会人口群体的能力,并突出了抑郁和焦虑严重程度对普通人群心理能力的影响。
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引用次数: 0
Setting incentives right with long-term risk adjustment. 通过长期风险调整制定正确的激励措施。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 Epub Date: 2024-12-20 DOI: 10.1007/s10198-024-01751-6
Simon Reif, Sabrina Schubert, Achim Wambach
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引用次数: 0
Workload and short sickness absences in a cohort of Finnish hospital employees. 芬兰医院员工群体的工作量与短期病假。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 Epub Date: 2024-05-23 DOI: 10.1007/s10198-024-01698-8
Tero Kuusi, Martti Kulvik, Mikko Härmä, Annina Ropponen

This article used register data on day-to-day working hours of hospital employees combined with patient characteristics at work unit (wards) level to measure workload and its implications for short, self-certified sickness absences. We measured statistically the average nursing treatment burden of different patient mixes in hospital wards, and then analyzed the overall workload (intensity) of working days by comparing it to the actual available nursing workforce. We found that a significant part of the workload variation occurred between working days, and it was related to unexpected changes in the number of employees. In atypical situations a long work shift was associated with caring for patients with fewer resources. The high workload of a day increased the risk of short sickness absences along the following 3-week period. The results show that managing short-term workload variability should be a key aim from the perspective of well-being at work, and that combining different data sources can provide novel, important insights to the measurement of workload.

本文利用医院员工日常工作时间的登记数据,结合工作单位(病房)层面的患者特征,来衡量工作量及其对短期自证病假的影响。我们对医院病房中不同病人组合的平均护理治疗负担进行了统计测量,然后通过与实际可用的护理劳动力进行比较,分析了工作日的总体工作量(强度)。我们发现,很大一部分工作量变化发生在工作日之间,这与员工人数的意外变化有关。在非典型情况下,长时间轮班与用较少的资源护理病人有关。在接下来的 3 周内,一天的高工作量会增加短期病假的风险。研究结果表明,从工作幸福感的角度来看,管理短期工作量的可变性应该是一个关键目标,而结合不同的数据来源可以为工作量的测量提供新颖、重要的见解。
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引用次数: 0
A budget impact analysis of a digital monitoring solution in patients treated with oral anticancer agents: a medico-economic analysis of the randomized phase 3 CAPRI trial. 口服抗癌药患者数字监测解决方案的预算影响分析:CAPRI 随机三期试验的医学经济分析。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 Epub Date: 2024-06-28 DOI: 10.1007/s10198-024-01702-1
Etienne Minvielle, Henri Leleu, Marc Masseti, Arlindo Ferreira, Gérard de Pouvourville, Mario Di Palma, Florian Scotté

Background/objectives: Remote patient monitoring (RPM) has demonstrated numerous benefits in cancer care, including improved quality of life, overall survival, and reduced medical resource use. This study presents a budget impact analysis of a nurse navigator-led RPM program, based on the CAPRI trial, from the perspective of the French national health insurance (NHI). The study aimed to assess the impact of the program on medical resource utilization and costs.

Methods: Medical resource utilization data were collected from both medico-administrative sources and patient-reported questionnaires. Costs were calculated by applying unit costs to resource utilization and estimating the average monthly cost per patient. Sensitivity analyses were conducted to explore different perspectives and varying resource consumption.

Results: The analysis included 559 cancer patients participating in the CAPRI program. From the NHI perspective, the program resulted in average savings of €377 per patient over the 4.58-month follow-up period, mainly due to reduced hospitalizations. The all-payers perspective yielded even greater savings of €504 per patient. Sensitivity analyses supported the robustness of the findings.

Conclusion: The budget impact analysis demonstrated that the CAPRI RPM program was associated with cost savings from the perspective of the NHI. The program's positive impact on reducing hospitalizations outweighed the additional costs associated with remote monitoring. These findings highlight the potential economic benefits of implementing RPM programs in cancer care. Further research is warranted to assess the long-term cost-effectiveness and scalability of such programs in the real-world settings.

背景/目的:远程患者监护(RPM)已在癌症护理中显示出诸多益处,包括改善生活质量、提高总生存率和减少医疗资源的使用。本研究以 CAPRI 试验为基础,从法国国家医疗保险(NHI)的角度出发,对一项由护士导航员主导的 RPM 计划进行了预算影响分析。研究旨在评估该计划对医疗资源利用率和成本的影响:方法:医疗资源利用率数据由医疗行政部门和患者报告问卷收集而来。成本的计算方法是将单位成本应用于资源利用率,并估算出每位患者每月的平均成本。进行了敏感性分析,以探讨不同的观点和不同的资源消耗:分析包括 559 名参加 CAPRI 计划的癌症患者。从国家医疗保险的角度来看,该计划在 4.58 个月的跟踪期内为每位患者平均节省了 377 欧元,这主要归功于住院次数的减少。从所有纳税人的角度来看,每位患者节省的费用甚至更高,达到 504 欧元。敏感性分析证实了研究结果的可靠性:预算影响分析表明,从国家医疗保险的角度来看,CAPRI RPM 计划与成本节约有关。该计划对减少住院人数的积极影响超过了与远程监控相关的额外成本。这些发现凸显了在癌症治疗中实施 RPM 计划的潜在经济效益。我们有必要开展进一步的研究,以评估此类计划在实际环境中的长期成本效益和可扩展性。
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引用次数: 0
The hidden cost of sophistication: economic complexity and obesity. 复杂性的隐性成本:经济复杂性与肥胖症。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 Epub Date: 2024-06-11 DOI: 10.1007/s10198-024-01699-7
Ronald Djeunankan, Sosson Tadadjeu, Henri Njangang, Ummad Mazhar

Obesity has become a global health crisis, affecting people of all ages, regions, and socio-economic backgrounds. While individual behaviour and genetic factors contribute to obesity, the role of economic complexity in the evolution of obesity rates has not yet been empirically studied. Using a large panel of 110 countries over the period 1976-2015, this article estimates the linear and non-linear links between obesity and economic complexity. According to baseline results, an improvement in economic complexity will lead to an increase in obesity up to a certain threshold. Beyond this turning point, any further increase in economic complexity will significantly contribute to obesity reduction. The issue of simultaneity is tackled using the two-stage instrumental variable method. Our findings support the Obesity Kuznets Curve (OKC) pattern, which suggests that economic progress and obesity have an inverted U-shaped relationship. Our results suggest that greater embeddedness of knowledge in the products produced and exported by a country increases the likelihood of obesity in society, at least up to a threshold. From these results, some important policy implications are discussed.

肥胖症已成为全球健康危机,影响着不同年龄、地区和社会经济背景的人群。虽然个人行为和遗传因素会导致肥胖,但经济复杂性在肥胖率演变中的作用尚未得到实证研究。本文利用 1976-2015 年间 110 个国家的大型面板数据,估算了肥胖与经济复杂性之间的线性和非线性联系。根据基线结果,经济复杂度的提高会导致肥胖症的增加,直至达到某个临界点。超过这一转折点后,经济复杂性的进一步提高将大大有助于减少肥胖。利用两阶段工具变量法解决了同时性问题。我们的研究结果支持肥胖库兹涅茨曲线(OKC)模式,即经济进步与肥胖之间存在倒 U 型关系。我们的结果表明,一个国家生产和出口的产品中知识的嵌入程度越高,社会中肥胖的可能性就越大,至少会达到一个临界点。根据这些结果,我们讨论了一些重要的政策影响。
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引用次数: 0
Incentivizing adherence to pre-exposure prophylaxis for HIV prevention: a randomized pilot trial among male sex workers in Mexico. 激励坚持暴露前预防措施以预防艾滋病毒:在墨西哥男性性工作者中开展的随机试点试验。
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-03-01 Epub Date: 2024-07-13 DOI: 10.1007/s10198-024-01705-y
Omar Galárraga, Marta Wilson-Barthes, Carlos Chivardi, Nathalie Gras-Allain, Fernando Alarid-Escudero, Monica Gandhi, Kenneth H Mayer, Don Operario

Low adherence to preventative medications against life-long health conditions is a major contributor to global morbidity and mortality. We implemented a pilot randomized controlled trial in Mexico to measure the extent to which conditional economic incentives help male sex workers increase their adherence to pre-exposure prophylaxis (PrEP) for HIV prevention. We followed n = 110 male sex workers over 6 months. At each quarterly visit (at months 0, 3, and 6), all workers received a $10 transport reimbursement, a free 3-month PrEP supply, and completed socio-behavioral surveys. The primary outcome was an objective biomarker of medication adherence based on tenofovir (TFV) drug concentration levels in hair collected at each visit. Individuals randomized to the intervention received incentives based on a grading system as a function of PrEP adherence: those with high (> 0.043 ng/mg TFV concentration), medium (0.011 to 0.042 ng/mg), or low (< 0.011 ng/mg) adherence received $20, $10, or $0, respectively. Six-month pooled effects of incentives on PrEP adherence were analyzed using population-averaged gamma generalized estimating equation models. We estimated heterogeneous treatment effects by sex worker characteristics. The incentive intervention led to a 28.7% increase in hair antiretroviral concentration levels over 6 months consistent with increased PrEP adherence (p = 0.05). The effect of incentives on PrEP adherence was greater for male sex workers who were street-based (vs. internet) workers (p < 0.10). These pilot findings suggest that modest conditional economic incentives could be effective, at scale, for improving PrEP adherence among male sex workers, and should be tested in larger implementation trials. ClinicalTrials.gov Identifier: NCT03674983.

对终身健康状况的预防性药物依从性低是导致全球发病率和死亡率的主要原因。我们在墨西哥开展了一项试点随机对照试验,以衡量有条件的经济激励措施能在多大程度上帮助男性性工作者提高对暴露前预防药物(PrEP)的依从性,从而预防艾滋病。我们对 n = 110 名男性性工作者进行了为期 6 个月的跟踪调查。在每个季度的访问中(第 0、3 和 6 个月),所有性工作者都会收到 10 美元的交通费报销、3 个月的 PrEP 免费供应,并完成社会行为调查。主要结果是根据每次就诊时收集的头发中替诺福韦(TFV)药物浓度水平作为坚持用药的客观生物标志物。根据 PrEP 依从性的分级系统,随机接受干预的人将获得奖励:TFV 浓度高(> 0.043 ng/mg)、中(0.011 至 0.042 ng/mg)或低(0.011 至 0.042 ng/mg)的人将获得奖励。
{"title":"Incentivizing adherence to pre-exposure prophylaxis for HIV prevention: a randomized pilot trial among male sex workers in Mexico.","authors":"Omar Galárraga, Marta Wilson-Barthes, Carlos Chivardi, Nathalie Gras-Allain, Fernando Alarid-Escudero, Monica Gandhi, Kenneth H Mayer, Don Operario","doi":"10.1007/s10198-024-01705-y","DOIUrl":"10.1007/s10198-024-01705-y","url":null,"abstract":"<p><p>Low adherence to preventative medications against life-long health conditions is a major contributor to global morbidity and mortality. We implemented a pilot randomized controlled trial in Mexico to measure the extent to which conditional economic incentives help male sex workers increase their adherence to pre-exposure prophylaxis (PrEP) for HIV prevention. We followed n = 110 male sex workers over 6 months. At each quarterly visit (at months 0, 3, and 6), all workers received a $10 transport reimbursement, a free 3-month PrEP supply, and completed socio-behavioral surveys. The primary outcome was an objective biomarker of medication adherence based on tenofovir (TFV) drug concentration levels in hair collected at each visit. Individuals randomized to the intervention received incentives based on a grading system as a function of PrEP adherence: those with high (> 0.043 ng/mg TFV concentration), medium (0.011 to 0.042 ng/mg), or low (< 0.011 ng/mg) adherence received $20, $10, or $0, respectively. Six-month pooled effects of incentives on PrEP adherence were analyzed using population-averaged gamma generalized estimating equation models. We estimated heterogeneous treatment effects by sex worker characteristics. The incentive intervention led to a 28.7% increase in hair antiretroviral concentration levels over 6 months consistent with increased PrEP adherence (p = 0.05). The effect of incentives on PrEP adherence was greater for male sex workers who were street-based (vs. internet) workers (p < 0.10). These pilot findings suggest that modest conditional economic incentives could be effective, at scale, for improving PrEP adherence among male sex workers, and should be tested in larger implementation trials. ClinicalTrials.gov Identifier: NCT03674983.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":"299-311"},"PeriodicalIF":3.1,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11725604/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141604495","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 health and long-term care costs in the last year of life in The Netherlands.
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-02-26 DOI: 10.1007/s10198-025-01763-w
Christel E van Dijk, Tristan Langereis, Jan-Willem H Dik, Trynke Hoekstra, Bernard van den Berg

Knowing the determinants of rising health and long-term care costs is crucial to support cost containment policies and to predict future expenditures. According to the "red herring" debate, not ageing per se, but proximity to death is the most important determinant of future expenditures. This study aims to update and expand the existing Dutch literature after two major reforms in health and long-term care. Insurance claims data from 2018-2019 of 13,738,193 insured individuals were included. Using negative binomial regression analyses, the association between deceased individuals and survivors on total health and long-term care costs was investigated, as well as per health care sector. Costs rose sharply in the two months prior to death. Regression models showed an association with total health and long-term care costs of 10.8 for deceased individuals compared with survivors (crude model) and 3.3 (adjusted model). Especially including age and chronic diseases decreased the association. The largest differences in costs between deceased individuals and survivors in the adjusted model were found for geriatric rehabilitation care and primary care stays (16.7), home nursing (10,6), and long-term care (9.3). Not just the costs of deceased individuals are important for health care costs, but also age, as measured by being in the highest age category, and chronic diseases. The costs of deceased individuals were heterogeneous across health care sectors.

{"title":"The health and long-term care costs in the last year of life in The Netherlands.","authors":"Christel E van Dijk, Tristan Langereis, Jan-Willem H Dik, Trynke Hoekstra, Bernard van den Berg","doi":"10.1007/s10198-025-01763-w","DOIUrl":"https://doi.org/10.1007/s10198-025-01763-w","url":null,"abstract":"<p><p>Knowing the determinants of rising health and long-term care costs is crucial to support cost containment policies and to predict future expenditures. According to the \"red herring\" debate, not ageing per se, but proximity to death is the most important determinant of future expenditures. This study aims to update and expand the existing Dutch literature after two major reforms in health and long-term care. Insurance claims data from 2018-2019 of 13,738,193 insured individuals were included. Using negative binomial regression analyses, the association between deceased individuals and survivors on total health and long-term care costs was investigated, as well as per health care sector. Costs rose sharply in the two months prior to death. Regression models showed an association with total health and long-term care costs of 10.8 for deceased individuals compared with survivors (crude model) and 3.3 (adjusted model). Especially including age and chronic diseases decreased the association. The largest differences in costs between deceased individuals and survivors in the adjusted model were found for geriatric rehabilitation care and primary care stays (16.7), home nursing (10,6), and long-term care (9.3). Not just the costs of deceased individuals are important for health care costs, but also age, as measured by being in the highest age category, and chronic diseases. The costs of deceased individuals were heterogeneous across health care sectors.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143505826","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
Price transparency in the Dutch market-based health care system: did price dispersion for similar hospital services reduce over time?
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-02-22 DOI: 10.1007/s10198-025-01759-6
Frédérique Franken, Rudy Douven, Stéphanie van der Geest, Marco Varkevisser

In market-based health care systems, insurers negotiate prices of hospital care products with providers. While few countries disclose these negotiated prices, in 2016, the Dutch government required the disclosure of insurer-provider negotiated prices for hospital products up to €885 - the maximum deductible in the Netherlands - to enhance price transparency. This aimed to increase price awareness among and price transparency for consumers, insurers, and providers, fostering price competition. We study if price dispersion for relatively homogeneous hospital care products decreased post-publication, resulting in price convergence. We used negotiated price data from three major Dutch health insurers on over 200 hospital products. Using descriptive statistics and linear regression, with the coefficient of variation (a measure of dispersion) regressed on the year, we examined the development of price dispersion and the occurrence of price convergence. Price dispersion for the studied sample of hospital products decreased by an average of 29% between 2016 and 2022. This decrease was not accompanied by a price level increase that was larger than expected based on general inflation. Regression analysis showed a significant negative association between year and the coefficient of variation, indicating price convergence. These findings support our hypothesis that price dispersion decreased after mandatory price disclosure. The government mandate potentially increased awareness of largely unexplainable price differences for products priced below €885, encouraging insurers and providers to reduce these through the negotiation process. The observed price convergence likely benefits patients, as it results in less random out-of-pocket payments across providers for the same hospitals products.

{"title":"Price transparency in the Dutch market-based health care system: did price dispersion for similar hospital services reduce over time?","authors":"Frédérique Franken, Rudy Douven, Stéphanie van der Geest, Marco Varkevisser","doi":"10.1007/s10198-025-01759-6","DOIUrl":"https://doi.org/10.1007/s10198-025-01759-6","url":null,"abstract":"<p><p>In market-based health care systems, insurers negotiate prices of hospital care products with providers. While few countries disclose these negotiated prices, in 2016, the Dutch government required the disclosure of insurer-provider negotiated prices for hospital products up to €885 - the maximum deductible in the Netherlands - to enhance price transparency. This aimed to increase price awareness among and price transparency for consumers, insurers, and providers, fostering price competition. We study if price dispersion for relatively homogeneous hospital care products decreased post-publication, resulting in price convergence. We used negotiated price data from three major Dutch health insurers on over 200 hospital products. Using descriptive statistics and linear regression, with the coefficient of variation (a measure of dispersion) regressed on the year, we examined the development of price dispersion and the occurrence of price convergence. Price dispersion for the studied sample of hospital products decreased by an average of 29% between 2016 and 2022. This decrease was not accompanied by a price level increase that was larger than expected based on general inflation. Regression analysis showed a significant negative association between year and the coefficient of variation, indicating price convergence. These findings support our hypothesis that price dispersion decreased after mandatory price disclosure. The government mandate potentially increased awareness of largely unexplainable price differences for products priced below €885, encouraging insurers and providers to reduce these through the negotiation process. The observed price convergence likely benefits patients, as it results in less random out-of-pocket payments across providers for the same hospitals products.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-02-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143477245","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
Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands.
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-02-21 DOI: 10.1007/s10198-024-01754-3
Simon van der Schans, Rimma Velikanova, Diana Weidlich, Ruth Howells, Anish Patel, Matthias Bischof, Maarten J Postma, Cornelis Boersma

Background: Spinal muscular atrophy (SMA) is a rare genetic disease resulting in loss of motor function and, in severe cases (e.g., SMA type 1), infantile death. While treatments like nusinersen and onasemnogene abeparvovec improve prognosis for patients with SMA, costs for these medications can contribute to economic burden.

Objective: Direct costs were compared for onasemnogene abeparvovec, a one-time gene replacement therapy, versus nusinersen, a lifelong therapy, for patients with SMA type 1 and/or three or more survival motor neuron 2 (SMN2) gene copies in the Netherlands.

Methods: A cost comparison analysis model of 1-year incident patient population from the Netherlands was used to compare costs of onasemnogene abeparvovec versus nusinersen for patients eligible for onasemnogene abeparvovec immediately after diagnosis. Multiple analyses were conducted for economic outcomes (e.g., base-case, break-even, deterministic sensitivity, probabilistic sensitivity, scenario analyses).

Results: Cost differences of -€2.9 million (undiscounted) and -€1.5 million (discounted) per patient with SMA type 1 treated with onasemnogene abeparvovec versus nusinersen over a 20-year time horizon were identified (base-case). Reduced costs with onasemnogene abeparvovec versus nusinersen were evident after 8.25 years.

Conclusion: Onasemnogene abeparvovec was less costly than nusinersen after 8.25 years of treatment of patients with SMA type 1 in the Netherlands.

背景:脊髓性肌萎缩症(SMA)是一种罕见的遗传疾病,会导致患者丧失运动功能,严重者(如 SMA 1 型)会导致婴儿死亡。尽管纽西奈森和奥那西喹阿替巴韦克等治疗方法可改善 SMA 患者的预后,但这些药物的费用也会加重患者的经济负担:目的:比较了荷兰SMA 1型和/或三个或更多存活运动神经元2(SMN2)基因拷贝患者的一次性基因替代疗法onasemnogene abeparvovec和终身疗法nusinersen的直接成本:方法:采用荷兰1年发病患者人群的成本比较分析模型,比较符合条件的患者在确诊后立即接受onasemnogene abeparvovec与nusinersen治疗的成本。对经济结果进行了多重分析(如基本情况分析、盈亏平衡分析、确定性敏感性分析、概率敏感性分析、情景分析):结果:在 20 年的时间跨度内,每名接受 onasemnogene abeparvovec 治疗的 1 型 SMA 患者与接受 nusinersen 治疗的患者的成本差异分别为-290 万欧元(未贴现)和-150 万欧元(贴现)(基础案例)。8.25 年后,onasemnogene abeparvovec 与 nusinersen 相比明显降低了成本:结论:在荷兰,onasemnogene abeparvovec 治疗 1 型 SMA 患者 8.25 年后的费用低于 nusinersen。
{"title":"Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands.","authors":"Simon van der Schans, Rimma Velikanova, Diana Weidlich, Ruth Howells, Anish Patel, Matthias Bischof, Maarten J Postma, Cornelis Boersma","doi":"10.1007/s10198-024-01754-3","DOIUrl":"https://doi.org/10.1007/s10198-024-01754-3","url":null,"abstract":"<p><strong>Background: </strong>Spinal muscular atrophy (SMA) is a rare genetic disease resulting in loss of motor function and, in severe cases (e.g., SMA type 1), infantile death. While treatments like nusinersen and onasemnogene abeparvovec improve prognosis for patients with SMA, costs for these medications can contribute to economic burden.</p><p><strong>Objective: </strong>Direct costs were compared for onasemnogene abeparvovec, a one-time gene replacement therapy, versus nusinersen, a lifelong therapy, for patients with SMA type 1 and/or three or more survival motor neuron 2 (SMN2) gene copies in the Netherlands.</p><p><strong>Methods: </strong>A cost comparison analysis model of 1-year incident patient population from the Netherlands was used to compare costs of onasemnogene abeparvovec versus nusinersen for patients eligible for onasemnogene abeparvovec immediately after diagnosis. Multiple analyses were conducted for economic outcomes (e.g., base-case, break-even, deterministic sensitivity, probabilistic sensitivity, scenario analyses).</p><p><strong>Results: </strong>Cost differences of -€2.9 million (undiscounted) and -€1.5 million (discounted) per patient with SMA type 1 treated with onasemnogene abeparvovec versus nusinersen over a 20-year time horizon were identified (base-case). Reduced costs with onasemnogene abeparvovec versus nusinersen were evident after 8.25 years.</p><p><strong>Conclusion: </strong>Onasemnogene abeparvovec was less costly than nusinersen after 8.25 years of treatment of patients with SMA type 1 in the Netherlands.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143469923","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
Routine measurement in low back pain; towards a pragmatic patient-reported productivity cost outcome measurement using the institute for medical technology assessment productivity cost questionnaire.
IF 3.1 3区 医学 Q1 ECONOMICS Pub Date : 2025-02-21 DOI: 10.1007/s10198-025-01756-9
Adekunle Z Ademiluyi, Antoinette D I van Asselt, Michiel F Reneman

Purpose: The iMTA productivity cost questionnaire (iPCQ) has been recommended as a measurement tool for productivity cost, however, its use in routine care is hindered by the length of this questionnaire (18 questions). This study developed and tested a short-form (SF-) iPCQ.

Method: A secondary analysis of the Groningen Spine Cohort's baseline data from patients with low back pain referred for tertiary care was performed. Six SFs were evaluated against the comprehensive iPCQ. Spearman correlation (r), intraclass correlation coefficient (ICC, agreement), standard error of measurement (SEM), and Bland-Altman's plot tested the congruence of the SFs with the comprehensive iPCQ.

Results: The sample consisted of 1220 patients with low back pain. The SF version with the highest correlation (SF-3; 7 items) with the comprehensive iPCQ had r = 0.99, ICC = 0.99, SEM = 295, while the SF with the least number of items (SF-6; 5 items) had r = 0.84, ICC = 0.91, SEM = 2063. The mean productivity cost estimates of SF-3 and SF-6 were €3414 (95% CI: 3036-3791) and €3333 (95% CI: 2970-3696) respectively while that for the comprehensive iPCQ amounted to €3456 (95% CI: 3189-3720).

Conclusion: A SF with seven questions was developed with a high agreement with the comprehensive iPCQ. Initial clinimetric testing was satisfactory. Further assessment is recommended.

{"title":"Routine measurement in low back pain; towards a pragmatic patient-reported productivity cost outcome measurement using the institute for medical technology assessment productivity cost questionnaire.","authors":"Adekunle Z Ademiluyi, Antoinette D I van Asselt, Michiel F Reneman","doi":"10.1007/s10198-025-01756-9","DOIUrl":"https://doi.org/10.1007/s10198-025-01756-9","url":null,"abstract":"<p><strong>Purpose: </strong>The iMTA productivity cost questionnaire (iPCQ) has been recommended as a measurement tool for productivity cost, however, its use in routine care is hindered by the length of this questionnaire (18 questions). This study developed and tested a short-form (SF-) iPCQ.</p><p><strong>Method: </strong>A secondary analysis of the Groningen Spine Cohort's baseline data from patients with low back pain referred for tertiary care was performed. Six SFs were evaluated against the comprehensive iPCQ. Spearman correlation (r), intraclass correlation coefficient (ICC, agreement), standard error of measurement (SEM), and Bland-Altman's plot tested the congruence of the SFs with the comprehensive iPCQ.</p><p><strong>Results: </strong>The sample consisted of 1220 patients with low back pain. The SF version with the highest correlation (SF-3; 7 items) with the comprehensive iPCQ had r = 0.99, ICC = 0.99, SEM = 295, while the SF with the least number of items (SF-6; 5 items) had r = 0.84, ICC = 0.91, SEM = 2063. The mean productivity cost estimates of SF-3 and SF-6 were €3414 (95% CI: 3036-3791) and €3333 (95% CI: 2970-3696) respectively while that for the comprehensive iPCQ amounted to €3456 (95% CI: 3189-3720).</p><p><strong>Conclusion: </strong>A SF with seven questions was developed with a high agreement with the comprehensive iPCQ. Initial clinimetric testing was satisfactory. Further assessment is recommended.</p>","PeriodicalId":51416,"journal":{"name":"European Journal of Health Economics","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143469940","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
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