Journal of Medical Economics最新文献

{"title":"Economic impact of in-office pediatric tympanostomy tube placement using a tympanostomy tube delivery device in a commercially- and Medicaid-insured US population.","authors":"Meghan Moynihan, Ryan Ross, Joseph Delgado, Mark Gerber, Gene Liu","doi":"10.1080/13696998.2025.2514925","DOIUrl":"10.1080/13696998.2025.2514925","url":null,"abstract":"<p><strong>Aims: </strong>Pediatric tympanostomy tube placement (TTP) is performed primarily in operating room (OR) settings under general anesthesia. Recent studies have demonstrated TTP completed in the otolaryngologist's office with a topical anesthetic and delivery device to be safe and effective. This study estimated the financial impact to commercial and Medicaid health plans by shifting pediatric TTP from the OR to an office setting.</p><p><strong>Materials and methods: </strong>This budget impact model estimated the financial impact of using the Hummingbirdⁱ Tympanostomy Tube System (HTTS) for in-office pediatric tympanostomy tube placement by shifting 25% of utilization away from the OR - hospital outpatient department (HOPD) or ambulatory surgery center (ASC) - in a commercially- and Medicaid-insured population.</p><p><strong>Results: </strong>The annual TTP estimate in a one-million-member health plan was 2,872 for a commercial plan and 4,534 for a Medicaid plan. Cost inputs for HOPD and ASC procedures were $5,743 and $3,609 (Commercial) and $2,199 and $1,663 (Medicaid), respectively. Cost inputs for in-office procedures utilizing HTTS were $2,000 (Commercial) and $1,600 (Medicaid). All surgical cost estimates assumed a bilateral (two ear) procedure. A 25% shift led to cost savings of $1,991,790 (14.5%) for commercial plans and $365,562 (4.2%) for Medicaid plans; translating into $0.17 per member per month (PMPM) and $58 per treated member per month (PTMPM) savings in a commercial plan and $0.03 PMPM and $7 PTMPM in Medicaid. Per patient cost of TTP decreased by $3,743/65.2% (HOPD) or $1,609/44.6% (ASC) for Commercial and $519/24.5% (HOPD) and $63/3.8% (ASC) for Medicaid.</p><p><strong>Limitations: </strong>Costs in HOPD and ASC setting were based on averages in the MarketScan administrative claims databases which are limited by contributor, data coding, and entry errors.</p><p><strong>Conclusions: </strong>In-office TTP utilizing HTTS substantially reduces cost for U.S. commercial and Medicaid health insurers, offering a significant opportunity to deliver value-based care.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"899-909"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144234314","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}

{"title":"Use of external data to inform overall survival extrapolation in NICE technology appraisals for oncology drugs.","authors":"Audrey Petitjean, Huiyu Shang, Ash Bullement, Nicholas Latimer","doi":"10.1080/13696998.2025.2506968","DOIUrl":"10.1080/13696998.2025.2506968","url":null,"abstract":"<p><strong>Aim: </strong>To assess use of external evidence for overall survival (OS) estimation in oncology single-technology appraisals (STAs) by the National Institute for Health and Care Excellence (NICE).</p><p><strong>Methods: </strong>STAs for oncology drugs appraised by NICE between January 2021 and March 2023 were identified. For each eligible STA, OS extrapolation methods used, the rationale for using external data, the source and type of data, and information on acceptance by the evidence review group (ERG) and the appraisal committee were extracted.</p><p><strong>Results: </strong>Initially, 215 STAs were identified, of which 82 were eligible for the study. Of these, 32 STAs used external data for OS extrapolation, including trial data (44%), real-world data (47%), clinical opinion (25%), meta-analysis (1%) and previous STA (1%). External data were used more frequently in state-transition models for post-event transitions and cure assumptions, and in partitioned-survival models to replace pivotal trial OS, inform long-term survival estimates or to estimate OS based on surrogacy analysis. Sensitivity analyses on use of external data was explored in 16 (50%) of the STAs. The committee accepted use of external data in half of the analysed STAs, acknowledging uncertainty in OS extrapolation.</p><p><strong>Limitations: </strong>The analysis was limited to the STAs published between 2021 and 2023 and publicly available materials on the NICE website.</p><p><strong>Conclusion: </strong>This study provides an overview of external data used to estimate OS in oncology STAs conducted by NICE in recent years. External data, including trial data, real-world data and clinical opinions, were incorporated into recent oncology STAs at various modelling stages. ERGs and appraisal committees were generally accepting of the use of external data. However, it is crucial to conduct a sensitivity analysis and provide a justification for the methods and data source selection.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"803-813"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144101977","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}

{"title":"Cost-effectiveness of enzalutamide with androgen-deprivation therapy (ADT) versus ADT alone for the treatment of high-risk biochemically recurrent non-metastatic castration-sensitive prostate cancer in Canada.","authors":"Armen Aprikian, Fred Saad, Ewa Wywial, Thomas McLean, Karissa Johnston, Yuxin Li, Andrew Chilelli","doi":"10.1080/13696998.2025.2503660","DOIUrl":"10.1080/13696998.2025.2503660","url":null,"abstract":"<p><strong>Aim: </strong>The EMBARK (NCT02319837) trial demonstrated that enzalutamide with androgen-deprivation therapy (ADT; enzalutamide combination) or without ADT (enzalutamide monotherapy) significantly improved metastasis-free survival compared with ADT alone in high-risk biochemically recurrent (BCR) non-metastatic castration-sensitive prostate cancer (nmCSPC; also known as non-metastatic hormone-sensitive prostate cancer [nmHSPC]). No new safety signals were observed during the trial. The findings of EMBARK led to Health Canada's approval of enzalutamide for this patient population. The aim of this analysis was to assess the cost-effectiveness of enzalutamide combination versus ADT alone in patients with high-risk BCR nmCSPC from the Canadian payer's perspective.</p><p><strong>Material and methods: </strong>A semi-Markov model was created to represent the treatment and disease progression of patients with high-risk BCR nmCSPC over a 30-year horizon. Costs and outcomes were discounted at 1.5% annually. Treatment effects for high-risk BCR nmCSPC were informed by data from the EMBARK trial. Life-years (LYs), quality-adjusted life-years (QALYs), lifetime costs and incremental cost-effectiveness ratio (ICER) were estimated for enzalutamide combination and ADT alone. A one-way sensitivity analysis (OWSA) and scenario analyses were conducted to assess the robustness of results.</p><p><strong>Results: </strong>In the base-case deterministic analysis over the modeled time horizon, enzalutamide combination accumulated 11.85 LYs and 8.96 QALYs versus 8.75 LYs and 6.24 QALYs for ADT alone. The total cost for enzalutamide combination was Canadian dollars (CAD) 166,199, compared with CAD 95,146 for ADT alone. When combining cost and clinical outcome data, enzalutamide combination was associated with an ICER of CAD 26,129 per QALY gained. The OWSA and scenario analysis results were consistent with the base-case results.</p><p><strong>Conclusion: </strong>The study findings suggest that enzalutamide combination was associated with an ICER of CAD 26,129 per QALY gained, which is within the standard Canadian thresholds for willingness-to-pay (i.e. <CAD 50,000 per QALY gained), making enzalutamide combination a preferred treatment option for patients with high-risk BCR nmCSPC in Canada.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"766-777"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110959","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}

{"title":"Comparison of enteral nutritional therapy to induce and maintain remission in paediatric patients with mild-to-moderate Crohn's disease: a cost-effectiveness analysis in the UK.","authors":"Minal Patel, Rita Shergill-Bonner, Preeti Sharma, Maria Elisa Capobianco, Carolyn Steeds","doi":"10.1080/13696998.2025.2539644","DOIUrl":"10.1080/13696998.2025.2539644","url":null,"abstract":"<p><strong>Objectives: </strong>This study assessed the cost-effectiveness (CE) of a specialised formula versus standard oral nutritional supplements (ONS) for the dietary management of paediatric mild-to-moderate Crohn's disease (CD), from the UK National Health Service (NHS) perspective.</p><p><strong>Methods: </strong>We developed a Markov model with five health states (remission, mild-to-moderate, moderate-to-severe, surgery and death), using 8-week cycles and a one-year horizon. A systematic literature review identified treatment effectiveness (clinical remission rates) and health state utilities (Child Health Utility-9 Dimension). Costs were sourced from the British National Formulary and National Tariff Payment System (2024 prices) and included costs of intervention and healthcare resources such as primary and outpatient care, diagnostic tests, accident and emergency care and hospitalisations. Clinical experts validated the model structure and inputs. Deterministic and probabilistic sensitivity analyses (DSA and PSA) assessed the robustness of findings.</p><p><strong>Results: </strong>The base case results demonstrated that specialised formula saves £591 per patient annually while maintaining similar quality-adjusted life-years (QALYs) compared to standard ONS. Additionally, in a cohort of 1,000 patients, 552 would achieve remission with the specialised formula compared to 496 with standard ONS. DSA identified the 8-week intervention costs and utility scores as key drivers of CE. PSA indicated that at a willingness-to-pay threshold of £20,000 per QALY, specialised formula has a 78% likelihood of being CE compared with standard ONS.</p><p><strong>Limitations: </strong>The main limitation is the lack of published clinical trials comparing specialised formula with standard ONS, along with the overall scarcity of published data on standard ONS. Meta-analysis would be beneficial to validate and strengthen the comparative data; however, due to the limitations of the published data, meta-analysis is not possible at present.</p><p><strong>Conclusions: </strong>Specialised formula is a less costly and more effective treatment compared to standard ONS in mild-to-moderate paediatric CD patients over one year.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1214-1225"},"PeriodicalIF":3.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144742208","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}

{"title":"The impact of <i>KRAS</i> mutational status on patient-reported outcomes in advanced non-small-cell lung cancer: a cross sectional study in France and Germany.","authors":"Christos Chouaid, Andromachi Giannopoulou, Alexandra Starry, Björn Stollenwerk, Farastuk Bozorgmehr","doi":"10.1080/13696998.2024.2437324","DOIUrl":"10.1080/13696998.2024.2437324","url":null,"abstract":"<p><strong>Objectives: </strong>Health-related quality of life (HRQoL) studies in patients with advanced non-small-cell lung cancer (NSCLC) according to <i>KRAS</i> mutational status are limited. This study aimed to report real-world evidence on HRQoL outcomes based on <i>KRAS</i> mutational status in patients with advanced NSCLC tumors receiving second-line or later (2L+) treatment in France and Germany.</p><p><strong>Methods: </strong>In this real-world, non-interventional, cross-sectional, multicenter, patient-reported outcome (PRO) study conducted in France (15 contributing sites) and Germany (8 contributing sites), physicians enrolled adult patients with locally advanced and unresectable or metastatic NSCLC with known <i>KRAS</i> mutation status (<i>KRAS</i> G12C, <i>KRAS</i> non-G12C, or <i>KRAS</i> wildtype [WT]), who received a 2L + treatment. Study outcomes included sociodemographic characteristics; HRQoL evaluations based on EORTC Global Health Status QoL scores (QLQ-C30) and EQ-5D-5L scores. Data were analyzed descriptively.</p><p><strong>Results: </strong>Of 156 enrolled patients, data from 149 patients were included in the final analysis (France, <i>n</i> = 103; Germany, <i>n</i> = 46). Median (quartile [Q]1, Q3) age was 67.0 (61.0, 71.0) years; 56.4% of patients were male. In total, 38.9% (<i>n</i> = 58), 26.2% (<i>n</i> = 39), and 34.9% (<i>n</i> = 52) of patients had tumors with <i>KRAS</i> G12C mutation, <i>KRAS</i> non-G12C mutation and WT <i>KRAS</i>, respectively. Mean (±SD) QLQ-C30 Global Health Status QoL scores were 56.99 (20.30) for the overall population, and 56.03 (22.55), 58.97 (18.67) and 56.57 (19.05) for <i>KRAS</i> G12C, non-G12C, and WT subpopulations. In the overall population, moderate-to-extreme problems were reported in all EQ-5D-5L dimensions (range: overall population, 15.5%-39.6%; <i>KRAS</i> G12C, 15.6%-46.6%; non-G12C, 7.8%-23.1%; WT, 21.1%-44.2%).</p><p><strong>Conclusion: </strong>HRQoL was broadly similar across <i>KRAS</i> G12C, non-G12C, and WT subpopulations.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"13-24"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769607","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}

{"title":"Healthcare resource utilization and costs among treatment-naïve people with HIV in state Medicaids: analysis of multi-tablet vs. single-tablet antiretroviral regimen initiators with and without concurrent mental health disorders.","authors":"M Chen, M J Christoph, S Park, F Turkistani, A R Weinberg, C Trom, W Zachry, J Gruber, U Mordi, K Rajagopalan","doi":"10.1080/13696998.2024.2436288","DOIUrl":"10.1080/13696998.2024.2436288","url":null,"abstract":"<p><strong>Background: </strong>Research is needed to understand the impact of mental health disorders (MHD) on healthcare resource utilization (HCRU) and costs among people with human immunodeficiency virus (PWH).</p><p><strong>Objectives: </strong>Examine the HCRU and cost burden among treatment-naïve PWH with and without MHD initiating single tablet antiretroviral regimens (STRs) and multi-tablet regimens (MTRs).</p><p><strong>Methods: </strong>A retrospective database analysis of the US Medicaid population from Anlitiks' All Payor Claims database between 1 January 2016 and 30 June 2023 was conducted. Treatment-naïve MTR-initiators vs STR-initiators (the index was the first prescription fill claim date) with ≥ 12-months pre- and post-index continuous enrollment and no pre-index HIV-2 diagnosis among PWH/MHD and PWH/no-MHD during 1 January 2017-30 June 2022 were selected. Demographics, clinical characteristics, HCRU and costs between MTR-initiators vs STR-initiators among PWH/MHD and PWH/no-MHD were described using Chi-square tests and Wilcoxon rank-sum or t-tests for categorical and continuous variables, as appropriate. HCRU and costs were examined using multivariable logistic and gamma-log link regression models, controlling for potential confounders.</p><p><strong>Results: </strong>MTR-initiators (PWH/MHD: <i>n</i> = 7,874, PWH/no-MHD: <i>n</i> = 3,612) vs. STR-initiators (PWH/MHD: 46,024, PWH/no-MHD: 23,452) were significantly younger (PWH/MHD: 43.6 vs. 47.2 years; PWH/no-MHD: 39.2 vs. 43.3 years) and more likely to be female (PWH/MHD: 46.4% vs. 35.7%; PWH/no-MHD: 42.3% vs 29.7%) in both groups (all <i>p</i>-values < 0.05). MTR-initiators vs. STR-initiators had significantly higher rates of inpatient (IP) hospitalizations (PWH/MHD: 28.9% vs. 27.1%; PWH/no-MHD:13.9% vs. 11.9%) and emergency department (ED) visits (PWH/MHD: 53.3% vs. 49.2%; PWH/no-MHD: 35.2% vs. 31.8%) among both those with and without MHD (all <i>p</i>-values < 0.05). MTR-initiators vs. STR-initiators also had significantly higher adjusted all-cause medical costs (PWH/MHD: $60,228 vs $40,634; PWH/no-MHD: $33,623 vs. $17,996) (all <i>p</i>-values < 0.05).</p><p><strong>Conclusions: </strong>Among PWH/MHD and PWH/no-MHD, MTR-initiators experienced significantly higher HCRU, and 1.5 times greater costs compared to STR-initiators. In both MTR and STR-initiator groups, the PWH/MHD cohort consistently demonstrated a greater HCRU and cost burden than the PWH/no-MHD.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"25-35"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142769664","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}

{"title":"The burden of venous thromboembolism in ten countries: a cost-of-illness Markov model on surgical and ICU patients.","authors":"Kim Seemann, Ubong Silas, Antonia Bosworth Smith, Tobias Münch, Sita J Saunders, Alex Veloz, Rhodri Saunders","doi":"10.1080/13696998.2024.2436797","DOIUrl":"10.1080/13696998.2024.2436797","url":null,"abstract":"<p><strong>Aim: </strong>The objective of this study was to assess the burden of hospital-acquired venous thromboembolism (VTE) on healthcare systems and patients across ten countries.</p><p><strong>Methods: </strong>A multi-methodological approach was taken to estimate the burden of hospital-acquired VTE across five key clinical specialties and ten countries (Australia, Brazil, China, France, Mexico, South Korea, Spain, Taiwan, Thailand, and the United Kingdom). Surveys with healthcare professionals (surgeons, hematologists, and hospital management) were conducted to identify clinical specialties of interest. A systematic literature review and interviews were conducted to identify data for incidences and costs. A health-economic model was developed, using a decision tree and Markov model to estimate 1-year costs. Costs are presented in 2022 USD.</p><p><strong>Results: </strong>Orthopedics, oncology, long-term ICU, cardiology, and obstetrics and gynecology were identified as the clinical specialties of interest. The total cost burden of hospital-acquired VTE was estimated to be $41,280 million, which equals $503 per patient at risk. Expressed as a share of 2022 GDP, an average spending per country of 0.05% to 0.18% was observed. The VTE-associated mortality was substantial, accounting for 150,081 deaths in a 74.2 million population, translating into an average mortality rate of 2.02 (0.64-3.05) per 1,000 patients at risk.</p><p><strong>Limitations: </strong>There were limited data available concerning VTE incidences in some countries and clinical specialties. Where data were available, there was heterogeneity of incidence definitions across the identified studies. Generalizations, imputations, and the country-agnostic structure of the model might have contributed to biases.</p><p><strong>Conclusions: </strong>The burden of hospital-acquired VTE is substantial both from an economic and from a patient perspective in all countries evaluated.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1-12"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750933","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}

{"title":"The role of sodium zirconium cyclosilicate drug utilization in managing hyperkalemia: impact on healthcare resource utilization and on maintenance of renin-angiotensin-aldosterone system inhibitor therapy in Italian clinical practice.","authors":"Marco Gnesi, Francesca Daniel, Valentina Mongelli, Andrea Merlo, Nicola Cosentino, Anna Rita Maurizi, Marta Nugnes, Melania Leogrande, Luca Degli Esposti","doi":"10.1080/13696998.2025.2487357","DOIUrl":"https://doi.org/10.1080/13696998.2025.2487357","url":null,"abstract":"<p><strong>Aims: </strong>Hyperkalemia (HK) is prevalent in patients with chronic kidney disease (CKD) and heart failure, particularly those on renin-angiotensin-aldosterone system inhibitors (RAASi). However, HK treatment often necessitates RAASi discontinuation. Sodium zirconium cyclosilicate (SZC), reimbursed in Italy since 2021, offers a new treatment option for HK. This study aimed to assess real-world SZC use and resulting economic rebounds in Italy.</p><p><strong>Methods: </strong>Using administrative databases of healthcare entities covering about 6 million residents, patients with at least one prescription of SZC from Jan-2022 to Jun-2023 were identified. Patients receiving other potassium binders after SZC initiation were excluded. A logistic regression model estimated odds ratios (OR) with 95% confidence interval (95%CI) for predictors of long-term SZC use (>90 days), including age, sex, CKD status, and comorbidities. Univariate regression identified the potential association between each individual predictor and the likelihood of long-term treatment, followed by multivariate analysis adjusted for confounders. A backward stepwise logistic regression method retained only significant predictors, enhancing model accuracy.</p><p><strong>Results: </strong>The study identified 355 SZC-treated patients (mean age 70.4 years, 64.2% male). CKD was found in 69.3% (47.6% on dialysis), with common comorbidities including hypertension (57.5%), diabetes (43.4%), and heart failure (23.4%). RAASi use was observed in 68.7% before SZC initiation, and RAASi discontinuation was lower in long-term SZC users compared to short-term SZC users (41.2 vs. 56.6%, <i>p</i> = 0.048). Short-term SZC treatment (≤90 days) was more frequent (83.1%) and predicted by dialysis (OR = 0.22). Healthcare costs over 6 months averaged €7,943 for short-term users (dialysis: €3,452) and €6,647 for long-term users (dialysis: €1,130).</p><p><strong>Conclusions: </strong>This real-world study showed that nearly 17% of patients continued SZC therapy for ≥90 days. Long-term therapy was associated with lower RAASi discontinuation and reduced healthcare costs due to hospitalizations and outpatient specialist services, suggesting that SZC can potentially provide clinical and economic benefits for HK management.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"28 1","pages":"576-585"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144022468","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}

{"title":"Cost-effectiveness analysis of regorafenib dose optimization for refractory metastatic colorectal cancer.","authors":"Tanios Bekaii-Saab, Sang Kyu Cho, Brian Hocum, Jamie Grossman, Sreevalsa Appukkuttan, Svetlana Babajanyan, Marisca Marian, Woojung Lee, Afsaneh Barzi, Min Yang","doi":"10.1080/13696998.2025.2496068","DOIUrl":"https://doi.org/10.1080/13696998.2025.2496068","url":null,"abstract":"<p><strong>Background: </strong>New regimens have emerged as third-line or later therapies for metastatic colorectal cancer (mCRC), including regorafenib dose optimization (ReDO), trifluridine/tipiracil and bevacizumab (TAS-BEV) combination therapy, and fruquintinib. We evaluated relative cost-effectiveness of these therapies in patients with mCRC from a US payer's perspective.</p><p><strong>Materials and methods: </strong>A partitioned survival model (PSM) was constructed to estimate total costs and quality-adjusted life years (QALYs). Clinical parameters were obtained from pivotal trials of the respective therapies and incremental cost-effectiveness ratios (ICERs) were estimated to assess relative cost-effectiveness of these treatments. Model robustness was assessed using deterministic (DSA) and probabilistic sensitivity analysis (PSA). Three scenario analyses were conducted: (1) assuming equal efficacy across treatments, (2) with prior exposure to anti-vascular endothelial growth factor (VEGF) therapy, and (3) alternative clinical inputs for fruquintinib from a different clinical trial.</p><p><strong>Results: </strong>Under the conventional willingness-to-pay (WTP) threshold in US ($150,000 per QALY gained), ReDO was cost-effective when compared with TAS-BEV and was dominant over fruquintinib. TAS-BEV was associated with an incremental QALY of 0.197 over ReDO, resulting in an ICER at $554,567 per QALY gained. The base case results were robust in DSA and PSA. Most influential parameters were treatment cost and effectiveness. In patients with prior anti-VEGF therapy, ReDO remained cost-effective compared to TAS-BEV and fruquintinib under the conventional WTP threshold.</p><p><strong>Limitation: </strong>Differences in trial populations may affect the comparability of the outcomes. Sensitivity and scenario analyses were conducted to address these limitations.</p><p><strong>Conclusion: </strong>ReDO was cost-effective compared with TAS-BEV from the US payer's perspective despite a higher QALY gain associated with TAS-BEV. ReDO was dominant over fruquintinib, consistently having a higher QALY gain and lower cost.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"28 1","pages":"655-663"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144014522","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}

{"title":"A Swedish cost-utility analysis of ferric derisomaltose versus ferric carboxymaltose in the treatment of iron deficiency anemia in patients with inflammatory bowel disease.","authors":"Stefan C Lindgren, Hans Strid, Henrik Hjortswang, Bardh Manxhuka, Neesha Nanu, Richard F Pollock","doi":"10.1080/13696998.2025.2487359","DOIUrl":"10.1080/13696998.2025.2487359","url":null,"abstract":"<p><strong>Objectives: </strong>Iron deficiency anemia (IDA) is a common extraintestinal manifestation of inflammatory bowel disease (IBD), driven by impaired iron absorption, inflammation of intestinal mucosa and blood loss due to intestinal bleeding. Exogenous iron is indicated to correct iron deficiency, with intravenous iron preferred in patients with malabsorption or intolerance of oral iron, active bleeding, systemic inflammation, or a need for rapid iron replenishment. The objective was to assess the cost-utility of two high-dose, rapid-infusion iron formulations-ferric derisomaltose (FDI) and ferric carboxymaltose (FCM)-in the treatment of patients with IBD and IDA in Sweden.</p><p><strong>Methods: </strong>The analysis used a previously-published micro-simulation model. Phosphate monitoring was modeled based on the product labelling, while iron need and disease-related quality of life (QoL) were modeled based on data from the PHOSPHARE-IBD randomized controlled trial. Cost-utility was evaluated from the national healthcare payer perspective over a five-year time horizon. Sensitivity and scenario analyses were performed.</p><p><strong>Results: </strong>For each iron treatment course, patients treated with FDI required 0.41 fewer infusions than those treated with FCM. The reduced number of infusions resulted in savings of SEK 9,876 over five years from iron administration costs alone (SEK 44,216 with FCM versus SEK 34,340 with FDI). Phosphate monitoring in patients treated with FCM cost SEK 2,776 over five years versus no monitoring costs with FDI. Total cost savings with FDI were SEK 14,962. FDI also resulted in a 0.076 quality-adjusted life year (QALY) improvement versus FCM driven primarily by the QoL improvements reported in PHOSPHARE-IBD, and FDI was therefore the dominant intervention.</p><p><strong>Limitations: </strong>The analysis did not capture costs or outcomes associated with hypophosphatemic osteomalacia or fractures.</p><p><strong>Conclusion: </strong>Relative to FCM, fewer infusions of FDI were required, there was no need for phosphate monitoring, and disease-related QoL was improved, while overall costs were reduced.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"567-575"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753085","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}