Journal of Medical Economics最新文献

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Addressing the challenges of health economic modelling in the context of suboptimal evidence base - case study based on a comparison between photodynamic diagnosis and narrow band imaging in non-muscle invasive bladder cancer. 在非肌肉浸润性膀胱癌的光动力学诊断和窄带成像比较的基础上,解决健康经济模型在次优证据基础背景下的挑战。
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-04-25 DOI: 10.1080/13696998.2026.2661553
Jonathan Belsey, Wojciech Chrosny, Jane Lapon
{"title":"Addressing the challenges of health economic modelling in the context of suboptimal evidence base - case study based on a comparison between photodynamic diagnosis and narrow band imaging in non-muscle invasive bladder cancer.","authors":"Jonathan Belsey, Wojciech Chrosny, Jane Lapon","doi":"10.1080/13696998.2026.2661553","DOIUrl":"https://doi.org/10.1080/13696998.2026.2661553","url":null,"abstract":"<p><strong>Introduction: </strong>Standard economic models, which have been developed to reflect typical data availability for pharmaceutical products, often struggle with non-pharmaceutical interventions like diagnostics and medical devices, where clinical trial data is more likely to be short-term or non-comparative. This paper explores methodological challenges in evaluating Photodynamic Diagnosis (PDD) <i>via</i> blue light cystoscopy (BLC) versus Narrow Band Imaging (NBI) for non-muscle invasive bladder cancer (NMIBC).</p><p><strong>Methods: </strong>A semi-Markov state-transition cost-utility model was developed for a health technology appraisal (HTA) by the Danish Treatment Council. The basis of the model was a differential effect of the two technologies on the risk of early local cancer recurrence. Due to a lack of direct comparative trials, an indirect hazard-function-based approach was used. Using TreeAge Pro software, baseline hazard curves were derived from ten-year survival data for conventional white light cystoscopy. Hazard ratios (HRs) from meta-analyses were then applied to these curves to derive simulated time-to-event curves for BLC and NBI.</p><p><strong>Results: </strong>The base-case analysis yielded an incremental cost-effectiveness ratio (ICER) of DKK 70,707/QALY for BLC-TURBT versus NBI-TURBT. Sensitivity analyses confirmed results remained robust and well below the DKK 500,000/QALY willingness-to-pay threshold. The model was most sensitive to HRs for time to first recurrence. Scenario analyses, including traditional parametric extrapolation, yielded consistent ICERs between DKK 36,775 and DKK 215,965/QALY.</p><p><strong>Discussion: </strong>The hazard-based workflow effectively integrated survival data from disparate sources, using a software-based method was quicker, simpler and more intuitive to use than conventional statistical methods. The approach used is equally applicable to a partitioned survival structure. The alignment between hazard-based and traditional parametric methods suggests this is a valid, efficient alternative for developing models in the face of evidence gaps.</p><p><strong>Conclusions: </strong>A hazard-function approach provides a transparent, practical solution for building robust economic models when clinical data is limited or incompatible with standard approaches.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1341-1354"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147773854","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 system dynamics modelling protocol to evaluate the impact of a health financing mechanism for breast cancer pharmacotherapies in Malaysia. 评估马来西亚乳腺癌药物治疗卫生筹资机制影响的系统动力学建模方案。
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-04-21 DOI: 10.1080/13696998.2026.2660601
Jing Sheng Lim, Weng Hong Fun, Kenneth Kwing Chin Lee, Renukha Sellappans
{"title":"A system dynamics modelling protocol to evaluate the impact of a health financing mechanism for breast cancer pharmacotherapies in Malaysia.","authors":"Jing Sheng Lim, Weng Hong Fun, Kenneth Kwing Chin Lee, Renukha Sellappans","doi":"10.1080/13696998.2026.2660601","DOIUrl":"https://doi.org/10.1080/13696998.2026.2660601","url":null,"abstract":"<p><strong>Introduction: </strong>The rising cost of targeted breast cancer therapies challenges financial sustainability and equitable access in dual-tier health systems. In Malaysia, public cancer care is highly subsidized but budget constrained, shifting patients toward private services that are typically financed through out-of-pocket payments, private health insurance or employer-sponsored insurance. Rakan KKM (MOH's Friend) is a fee-for service initiative in selected public hospitals designed to provide lower-cost private care, with revenues reinvested into the public system. However, its financial implications for breast cancer pharmacotherapy remain uncertain. This manuscript presents a methodological protocol for a system dynamics (SD) model developed to evaluate the financial implications of Rakan KKM for breast cancer pharmacotherapy in Malaysia.</p><p><strong>Methodology: </strong>A system dynamics model has been developed to model breast cancer disease progression across stages and patient movement between public, private and Rakan KKM care settings, integrating associated healthcare expenditures and revenue flows from the perspective of the Ministry of Health. An influence diagram was constructed through stakeholder engagement to identify key feedback mechanisms influencing access, affordability and system sustainability. Model parameterisation is complete, using national epidemiological data, published registries data, national drug acquisition cost estimates and expert elicitation from oncology clinicians and pharmacists. This protocol details the simulation framework where a status quo scenario is compared against intervention scenarios over a 10-year horizon. One-way sensitivity analysis and monte-carlo simulations address parameter and clinical uncertainties, while scenario analyses guided by the Diffusion of Innovation framework examine alternative uptake rates and capacity constraints.</p><p><strong>Conclusion: </strong>This protocol describes a transparent and adaptable SD modelling approach to assess the fiscal sustainability of financing high-cost breast cancer therapies in mixed public-private systems by projecting how patient switching influences pharmacotherapy expenditure and the net resources potentially available to support public service investment through Rakan KKM.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1279-1291"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147773868","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
Annual vaccination with BNT162b2 in Germany can avoid substantial clinical and economic burden of COVID-19 disease. 在德国,每年接种BNT162b2疫苗可以避免COVID-19疾病带来的重大临床和经济负担。
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2025-12-27 DOI: 10.1080/13696998.2025.2604970
Amy Barber, Julie Roiz, Sam Greenall, Edward Church, Andrea Schmetz, Stefan Riebel, Jingyan Yang, Oliver Witzke, Afschin Gandjour
{"title":"Annual vaccination with BNT162b2 in Germany can avoid substantial clinical and economic burden of COVID-19 disease.","authors":"Amy Barber, Julie Roiz, Sam Greenall, Edward Church, Andrea Schmetz, Stefan Riebel, Jingyan Yang, Oliver Witzke, Afschin Gandjour","doi":"10.1080/13696998.2025.2604970","DOIUrl":"https://doi.org/10.1080/13696998.2025.2604970","url":null,"abstract":"<p><strong>Aims: </strong>To quantify the clinical and economic burden of Coronavirus disease 19 (COVID-19), and burden potentially avoided with annual vaccination, in German adults in an endemic setting.</p><p><strong>Materials and methods: </strong>A decision tree model was constructed to estimate the clinical and economic impact of COVID-19 and projected burden avoided with BNT162b2, a seasonally adapted mRNA vaccine against severe COVID-19 disease. The majority of cost inputs and clinical event probabilities were informed by German real-world evidence from seasons 2022/23 and 2023/24. Vaccine efficacy was derived from US and German studies. Long/Post COVID impact was assessed in scenario analysis.</p><p><strong>Results: </strong>The model estimated up to 20.8 million symptomatic infections per year in adults, including 7.5 million in people aged 60+, and 6.0 million in comorbid adults. This translates to an estimated 198,598 hospitalizations and 24,626 COVID-attributable deaths, with 93.6% of deaths occurring in people aged 60+. The total economic burden including productivity loss was estimated at €1.4 billion in people aged 60+, €2.0 billion in comorbid adults, and €3.9 billion in all working adults. Long/Post COVID increased the economic burden by 1.7 times.</p><p><p>Assuming vaccination of 100% of recommended groups, 11.0 million symptomatic infections could be prevented, with the greatest impact in people aged 60+ (estimated 110,362 hospitalizations and 13,685 deaths avoided). For people aged 60+, comorbid adults, and all working adults, the number needed to vaccinate to prevent one symptomatic infection was 7, 4, and 4 people; to prevent one hospitalization, 231, 2,363, and 4,549; and to avoid one death, 1,862, 19,055, and 36,683, respectively.</p><p><strong>Conclusion: </strong>COVID-19 imposes a substantial clinical and economic burden on the German population, which could be mitigated with an expanded COVID-19 vaccination program. Further research into Long/Post COVID is needed. Our study presents considerations highlighting the value of broad vaccination especially for working adults.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"16-28"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846571","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 pasireotide long-acting release in acromegaly: a systematic literature review and methodology assessment. 肢端肥大症pasireotide长效释放的成本-效果:系统的文献回顾和方法学评估。
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-01-10 DOI: 10.1080/13696998.2025.2609506
Aleksandra Gilis-Januszewska, Małgorzata Bronikowska, Grzegorz Binowski, Michał Jachimowicz, Beatrice Gueron, Fabian Schmidt, Sika Dédé Kossi
{"title":"Cost-effectiveness of pasireotide long-acting release in acromegaly: a systematic literature review and methodology assessment.","authors":"Aleksandra Gilis-Januszewska, Małgorzata Bronikowska, Grzegorz Binowski, Michał Jachimowicz, Beatrice Gueron, Fabian Schmidt, Sika Dédé Kossi","doi":"10.1080/13696998.2025.2609506","DOIUrl":"10.1080/13696998.2025.2609506","url":null,"abstract":"<p><strong>Background: </strong>Acromegaly is a rare and progressive condition caused by excessive secretion of growth hormone and insulin-like growth factor type 1. Pasireotide long-acting release (LAR) is indicated as a second-line therapy for adults with acromegaly who are unsuitable for or unresponsive to surgery and inadequately controlled with first-generation somatostatin receptor ligands (FGSRLs). Although its efficacy and safety have already been established, its cost-effectiveness remains unclear. The primary objective of this study was to systematically assess the cost-effectiveness of pasireotide LAR compared to other second-line medical options, particularly pegvisomant.</p><p><strong>Methods: </strong>A systematic literature review was conducted in May 2024 in Medline, Medline In Process, Web of Science, and the Centre for Reviews and Dissemination (CRD), York. Studies were eligible if they were full or partial economic analyses of pasireotide LAR as a second-line pharmacological treatment for adult patients with acromegaly. Studies not available in English and publications prior to 2009 were excluded. Included articles were assessed for transparency using the appropriate checklists. Data extraction focused on costs, incremental cost-effectiveness ratios and quality-adjusted life years. Formal data synthesis of outcomes was not undertaken due to the small number of studies and notable heterogeneity between them. Methodological assessment involving the evaluation of model inputs, data sources, and their alignment with the evidence on the course of disease and clinical practice was performed to examine the main factors contributing to discrepancies in cost-effectiveness outcomes and assess the credibility of the results.</p><p><strong>Results: </strong>Of 160 records identified, six publications met the inclusion criteria. Their findings demonstrated variability. The critical assessment highlighted considerable variability in methodological rigor among the included studies.</p><p><strong>Conclusions: </strong>Evidence on pasireotide LAR's cost-effectiveness versus pegvisomant regimens remains inconclusive. Although two studies indicated that pegvisomant is more cost-effective, the study with the highest methodological credibility found that pasireotide LAR is a cost-effective alternative.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"135-154"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145949003","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
Budget impact model of nadofaragene firadenovec for the treatment of high-risk non-muscle-invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin immunotherapy. nadofaragene firadenovec治疗对卡介苗-谷氨酰胺免疫疗法无反应的高危非肌浸润性膀胱癌的预算影响模型
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-03-04 DOI: 10.1080/13696998.2026.2630599
Min Yang, Xinglei Chai, Dongni Ye, Angela Zhao, Ashton Moradi, Yair Lotan
{"title":"Budget impact model of nadofaragene firadenovec for the treatment of high-risk non-muscle-invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin immunotherapy.","authors":"Min Yang, Xinglei Chai, Dongni Ye, Angela Zhao, Ashton Moradi, Yair Lotan","doi":"10.1080/13696998.2026.2630599","DOIUrl":"10.1080/13696998.2026.2630599","url":null,"abstract":"<p><strong>Aims: </strong>To estimate the budget impact of adopting nadofaragene firadenovec for the treatment of adults with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma <i>in situ</i> with or without papillary tumors (CIS ± Ta/T1) from a US third-party payer's perspective.</p><p><strong>Methods: </strong>A budget impact model was developed to compare total healthcare costs under two scenarios: (1) with nadofaragene firadenovec included in the treatment mix, and (2) without it. The model simulated a hypothetical US health plan covering one million members over a 3-year time horizon. Costs assessed included drug acquisition and administration, surveillance and disease management, cystectomy, and treatment-related adverse events (AEs). Base-case inputs were informed by trial data, published literature, and real-world evidence. Scenario analyses explored alternative population definitions, epidemiology and clinical inputs; one-way sensitivity analyses tested parameter uncertainty.</p><p><strong>Results: </strong>In the base-case, the estimated per-member-per-month (PMPM) budget impact of adopting nadofaragene firadenovec ranged from $0.0010 in Year 1 to $0.0084 in Year 3. Increased drug and administration costs were partially offset by cost savings from reduced disease progression, fewer cystectomies, and lower AE rates. Scenario analyses demonstrated that even examining a broader population including papillary disease-only patients, the budget impact remained modest ($0.0064-$0.0548 PMPM from Years 1-3). Sensitivity analyses confirmed that results were most influenced by uptake rate, drug cost, and target population size.</p><p><strong>Limitations: </strong>Key limitations include reliance on projected market shares for nadofaragene firadenovec, omission of recently approved treatments (nogapendekin alfa inbakicept-pmln and TAR-200) due to the lack of publicly available market share data, and assumptions to derive clinical outcomes for certain treatments due to lack of reported data.</p><p><strong>Conclusions: </strong>Despite its higher acquisition cost, the inclusion of nadofaragene firadenovec resulted in a minimal budget impact as bladder-sparing option for patients with BCG-unresponsive NMIBC with CIS ± Ta/T1.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"672-687"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147355392","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
The indirect economic burden caused by premature mortality from lung cancer in Asia-Pacific countries; years of life lost and productivity costs. 亚太国家肺癌过早死亡造成的间接经济负担多年的生命损失和生产力成本。
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-03-06 DOI: 10.1080/13696998.2026.2635297
Manoj Gambhir, Vishnu Subash, Deepak Alexander, Robert Hughes, Aimee Fox, Sameer Gokhale
{"title":"The indirect economic burden caused by premature mortality from lung cancer in Asia-Pacific countries; years of life lost and productivity costs.","authors":"Manoj Gambhir, Vishnu Subash, Deepak Alexander, Robert Hughes, Aimee Fox, Sameer Gokhale","doi":"10.1080/13696998.2026.2635297","DOIUrl":"10.1080/13696998.2026.2635297","url":null,"abstract":"<p><strong>Background: </strong>Lung cancer caused 2.2 million new cases and 1.8 million deaths globally in 2020, accounting for 11.4% of global cancer. In the Asia-Pacific (AP) region, lung cancer is the leading cause of cancer mortality, contributing to 60% of global lung cancer deaths. This study assesses the economic impact of premature lung cancer deaths in 12 AP countries with different economic profiles by estimating productivity losses.</p><p><strong>Methods: </strong>The human capital approach was used to estimate productivity losses from premature lung cancer deaths (ICD-10 C33-34) across Singapore, Australia, Hong Kong, New Zealand, South Korea, Taiwan, Malaysia, Thailand, Indonesia, Philippines, Vietnam, and India. Years of productive life lost (YPLL) and the present value of future lost productivity (PVFLP) were calculated based on age-specific mortality, wage, and employment data. Results were analyzed by grouping countries into high- and middle-income categories.</p><p><strong>Findings: </strong>In 2019, 221,293 lung cancer deaths led to 617,574 YPLL and over $2.3 billion in productivity losses across the 12 AP countries. High-income countries experienced greater losses ($1.5 billion, $34,359 per death) compared to middle-income countries ($816 million, $4,660 per death).</p><p><strong>Limitations: </strong>The analysis excluded direct healthcare costs and productivity losses from morbidity and caregiver burden. Assumptions such as uniform labor force participation and mortality distribution may limit precision.</p><p><strong>Interpretation: </strong>Lung cancer imposes a significant burden across the AP region, with economic disparities between high- and middle-income countries. Findings highlight the need for continued investment in prevention, early detection, and equitable access to treatment, especially in middle-income nations.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"726-739"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147365549","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 treatment and care of patients with gastrointestinal inflammatory diseases: a systematic review. 胃肠道炎症性疾病患者治疗和护理的成本效益:系统综述
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-03-20 DOI: 10.1080/13696998.2026.2637360
Kristina Randlová, Petra Marešová, Lukáš Režný, Jan Hruška, Sonja Srdanović, Guðmundur H Guðmundsson, Egill Másson, Kamil Kuča
{"title":"Cost-effectiveness of treatment and care of patients with gastrointestinal inflammatory diseases: a systematic review.","authors":"Kristina Randlová, Petra Marešová, Lukáš Režný, Jan Hruška, Sonja Srdanović, Guðmundur H Guðmundsson, Egill Másson, Kamil Kuča","doi":"10.1080/13696998.2026.2637360","DOIUrl":"https://doi.org/10.1080/13696998.2026.2637360","url":null,"abstract":"<p><strong>Aims: </strong>The aim of this study was to systematically review and descriptively synthesize published cost-effectiveness evidence for biologic versus non-biologic treatments as well as comparisons among different biologic therapies and treatment sequencing strategies, in inflammatory bowel disease, highlighting patterns and variability across disease types, treatment strategies, and study designs.</p><p><strong>Materials and methods: </strong>We conducted a systematic review following the PRISMA guidelines, searching the Web of Science and PubMed databases for studies published between 2013 and 2024. Studies comparing the cost-effectiveness of treatments for inflammatory bowel disease were included.</p><p><strong>Results: </strong>Eighteen studies met the inclusion criteria, covering Crohn's disease and/or ulcerative colitis. Biologic therapies were generally associated with superior health outcomes compared with conventional treatments. Reported quality-adjusted life years (QALYs) ranged from 2.23 to 18.12 for biologic therapies and from 1.69 to 17.99 for non-biologic treatments. Although biologic therapies have higher costs, they are generally considered cost-effective. For Crohn's disease, infliximab was reported as a cost-effective biologic option, while findings for ulcerative colitis varied. Surgical intervention (colectomy) was identified as a cost-effective in selected clinical scenarios.</p><p><strong>Conclusion: </strong>Biologic therapies for inflammatory bowel disease are cost-effective, providing significant health benefits that offset higher costs. Substantial methodological heterogeneity and reliance on model-based economic evaluations limit direct comparability across studies. Future economic evaluations should focus on methodological consistency and transparency in assumptions to strengthen the interpretability of cost-effectiveness evidence.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"919-939"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147491152","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 exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United States. 在美国,夸张基因自体细胞基因编辑治疗镰状细胞病复发性血管闭塞危像患者的成本效益
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-02-23 DOI: 10.1080/13696998.2026.2624971
Andrea Lopez, Michael Gargano, Hongbo Yang, Foluso Joy Ogunsile, Nanxin Li, Yanwen Xie, Sushanth Jeyakumar, Chuka Udeze
{"title":"Cost-effectiveness of exagamglogene autotemcel gene-edited therapy in patients with sickle cell disease with recurrent vaso-occlusive crises in the United States.","authors":"Andrea Lopez, Michael Gargano, Hongbo Yang, Foluso Joy Ogunsile, Nanxin Li, Yanwen Xie, Sushanth Jeyakumar, Chuka Udeze","doi":"10.1080/13696998.2026.2624971","DOIUrl":"https://doi.org/10.1080/13696998.2026.2624971","url":null,"abstract":"<p><strong>Objective: </strong>Exagamglogene autotemcel (exa-cel) is a one-time nonviral gene-edited therapy approved in the United States (US) for treatment of patients aged ≥12 years with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). Standard of care (SOC) for SCD includes symptomatic care, hydroxyurea and/or red blood cell transfusions. This study estimated the long-term clinical outcomes and cost-effectiveness of exa-cel relative to SOC among patients with SCD with recurrent VOCs.</p><p><strong>Methods: </strong>A Markov model was used to compare the expected lifetime costs and clinical outcomes of patients with SCD with recurrent VOCs treated with exa-cel versus SOC from the US payer and societal perspectives. The model structure is based on disease severity, characterized by VOC frequency, which impacts the risk of developing SCD-related complications and mortality. The model incorporated data from the phase 3 pivotal CLIMB SCD-121 trial alongside published literature. Model outcomes included number of VOCs and other acute complications, proportion of patients developing chronic complications, life years (LYs), quality-adjusted LYs (QALYs), costs, and incremental cost-effectiveness ratios (ICERs).</p><p><strong>Results: </strong>Over a lifetime horizon, exa-cel was projected to improve survival by 30.8 years (mean age of death, exa-cel: 74.5 vs. SOC: 43.6), reduce the number of VOC events by 77 (7 vs. 84), and reduce undiscounted disease-related costs by $3.34 M ($0.55 M vs. $3.89 M) compared to treatment with SOC. Patients treated with exa-cel also were less likely to experience acute complications or develop chronic complications compared to SOC. The ICER per discounted QALY for exa-cel versus SOC was $16,800 from the payer perspective; exa-cel was dominant (less costly, more effective than SOC) from the societal perspective.</p><p><strong>Conclusions: </strong>Compared to SOC, exa-cel was projected to considerably reduce the number of VOCs, improve survival, and reduce disease-related costs in patients with SCD. Exa-cel was projected to be a cost-effective treatment option.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"547-562"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147276172","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
Breaking barriers in women's pelvic health: claims-based economic analysis and healthcare utilization of an AI care program compared to usual care. 打破女性盆腔健康的障碍:与常规护理相比,基于索赔的经济分析和人工智能护理计划的医疗保健利用
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-02-24 DOI: 10.1080/13696998.2026.2627835
Ana P Pereira, Andrea M Seet, Beatriz Domingues, Dora Janela, Akshat Pradhan, Anabela C Areias, Xin Tong, Virgílio Bento, Vijay Yanamadala, Steven P Cohen, Jennesa Atherton, Fernando Dias Correia, Luke Belz, Fabíola Costa
{"title":"Breaking barriers in women's pelvic health: claims-based economic analysis and healthcare utilization of an AI care program compared to usual care.","authors":"Ana P Pereira, Andrea M Seet, Beatriz Domingues, Dora Janela, Akshat Pradhan, Anabela C Areias, Xin Tong, Virgílio Bento, Vijay Yanamadala, Steven P Cohen, Jennesa Atherton, Fernando Dias Correia, Luke Belz, Fabíola Costa","doi":"10.1080/13696998.2026.2627835","DOIUrl":"https://doi.org/10.1080/13696998.2026.2627835","url":null,"abstract":"<p><strong>Aim: </strong>To compare healthcare utilization and spending among women enrolled in an employer-sponsored, artificial intelligence (AI) structured pelvic care program with those receiving usual in-person care for pelvic floor dysfunction (PFD) in routine clinical settings.</p><p><strong>Methods: </strong>This retrospective payor-perspective economic evaluation used exact and propensity score-matched cohorts derived from a third-party U.S. nationwide claims database from July 2022 to May 2025. Eligible participants were adult females with a pelvic-related condition, at least 24 months of continuous health-insurance coverage, and a minimum of one pelvic claim in the prior year. Intervention group (IG) comprised women who participated in the AI pelvic care program (consisting of biofeedback-mediated pelvic floor muscle training asynchronously monitored by a physical therapist specialized in pelvic health). Comparator group (CG) included women who sought a medical or physical therapy evaluation visit for PFD. Self-reported clinical outcomes available for the IG were assessed using latent-basis growth analysis.</p><p><strong>Results: </strong>The matched cohort included 602 women (301 per group). Relative to CG, IG patients had substantially lower healthcare spending over 12 months, with mean gross per-person pelvic-related savings of $3,082.4 (95% CI $1,270.2 to $4,894.7, <i>p</i><.001). Savings were primarily associated with fewer surgical procedures (per-person difference of $2,534.2; 95% CI $831.2 to $4,237.2, <i>p</i>=.004), with differences also noted in medical office visits and imaging utilization. IG participants demonstrated significant improvements in pelvic floor symptom burden, work productivity, and mental health.</p><p><strong>Limitations: </strong>Claims-based analyses cannot exclude unmeasured confounding, misclassification, or selection bias. The one-year follow-up limits assessment of long-term economic impact.</p><p><strong>Conclusions: </strong>Participation in this AI pelvic care program was associated with markedly lower healthcare utilization and spending compared with usual care, largely linked to fewer surgical interventions. These findings highlight the potential of accessible, guideline-concordant AI pelvic care to lessen healthcare spending associated with PFD and inform payor-oriented care delivery models.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"516-531"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147283933","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
Comparing deep learning and classical regression approaches for predicting healthcare expenditure and spending: a systematic review. 比较深度学习和经典回归方法预测医疗保健支出和支出:系统回顾。
IF 3 4区 医学
Journal of Medical Economics Pub Date : 2026-12-01 Epub Date: 2026-03-04 DOI: 10.1080/13696998.2026.2630598
John Tayu Lee, Melody Hsiao-San Yeh, Vincent Cheng-Sheng Li, Hsiao-Hui Chen, Yi-Hsuan Liu, Yu-Chun Chen, David Bin-Chia Wu
{"title":"Comparing deep learning and classical regression approaches for predicting healthcare expenditure and spending: a systematic review.","authors":"John Tayu Lee, Melody Hsiao-San Yeh, Vincent Cheng-Sheng Li, Hsiao-Hui Chen, Yi-Hsuan Liu, Yu-Chun Chen, David Bin-Chia Wu","doi":"10.1080/13696998.2026.2630598","DOIUrl":"10.1080/13696998.2026.2630598","url":null,"abstract":"<p><strong>Aims: </strong>This study compares deep learning architectures with traditional regression and tree-based models for individual-level healthcare cost prediction, with particular attention to performance differences across data contexts.</p><p><strong>Methods: </strong>We conducted a preregistered systematic review (PROSPERO CRD420251129440). Web of Science, PubMed, Embase, and Scopus were searched through August 2025. Eligible studies used real-world individual-level data (claims, electronic health records, or registries) to predict cost-related outcomes with at least one deep learning architecture and one classical regression comparator, and reported quantitative performance. Data were extracted on population, predictors, outcome horizon, model type, validation strategy, performance metrics, calibration, and interpretability.</p><p><strong>Results: </strong>Eight studies met inclusion criteria, spanning the United States, Europe, and Asia. In longitudinal designs-such as multi-year claims prediction and medication or hospitalization time-series forecasting-sequential deep learning models, particularly LSTM and CNN-LSTM hybrids, consistently outperformed regression and tree-based algorithms. Reported gains included approximately 10-20% reductions in RMSE/MAE, R<sup>2</sup> improvements of 0.01-0.15, and AUROC values up to 0.78 for high-risk classification. Across studies, prior costs and utilization were consistently the strongest predictors, while social determinants and free-text features were rarely incorporated. In contrast, for low-dimensional, structured, cross-sectional medical data, generalized linear models and tree-based approaches remain robust baseline models due to their interpretability and calibration stability.</p><p><strong>Limitations: </strong>Evidence is based on a small and heterogeneous set of eight studies, with limited external or temporal validation, short prediction horizons, and sparse assessment of calibration, economic interpretability, and fairness, warranting cautious interpretation.</p><p><strong>Conclusions: </strong>Deep learning offers clear gains for longitudinal, sequence-rich cost forecasting, whereas tree-based methods remain highly competitive for cross-sectional tabular prediction. Overall, these findings are consistent with the proposed Complexity-Performance Hypothesis, which posits that the predictive advantages of deep learning emerge primarily when model capacity is well matched to data complexity.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"654-671"},"PeriodicalIF":3.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147355320","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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