Élisabeth Mercier, John R Fullarton, Bosco A Paes, Ian P Keary, Barry S Rodgers-Gray, Nisha Thampi, Robert Delatolla
{"title":"Cost-effectiveness of wastewater and environmental monitoring of respiratory syncytial virus to guide universal infant immunoprophylaxis in Canada.","authors":"Élisabeth Mercier, John R Fullarton, Bosco A Paes, Ian P Keary, Barry S Rodgers-Gray, Nisha Thampi, Robert Delatolla","doi":"10.1080/13696998.2025.2473810","DOIUrl":"10.1080/13696998.2025.2473810","url":null,"abstract":"<p><strong>Aims: </strong>To compare the cost-effectiveness of wastewater and environmental monitoring (WEM) <i>versus</i> clinical surveillance (CS)-guided respiratory syncytial virus (RSV) prophylaxis programs in Canada.</p><p><strong>Materials and methods: </strong>A cost-utility model was developed comprising two identical decision trees for RSV-WEM and RSV-CS. Within each tree, children could conservatively receive nirsevimab prophylaxis (71% coverage) or not at the start of the RSV season and subsequently experience an RSV-related hospitalization, medically-attended, non-hospitalized RSV-infection, or be uninfected/non-medically attended. All children could experience respiratory morbidity up to age 18 years, with higher rates following RSV-related hospitalization. All prophylaxis and RSV-related costs were identical for RSV-WEM and RSV-CS. No costs were assumed for RSV-CS; whereas a cost of CAD$12.31 per infant (infrastructure: CAD$4.07 plus sampling: CAD$8.24) was assumed if a new RSV-WEM system was initiated, with all infrastructure costs included in year 1. Predicated on data from the 2022-23 Ontario RSV season, RSV-WEM was assumed to provide a 15.1% benefit for earlier initiation of the prophylaxis program <i>versus</i> RSV-CS. Outcomes were modelled over an 18-year time horizon (1.5% discounting).</p><p><strong>Results: </strong>RSV-WEM dominated (lower costs and higher utilities) RSV-CS and remained unaltered in all scenario analyses. Scenarios included: amortization of RSV-WEM infrastructure costs over 5 years; using existing WEM infrastructure for RSV detection; 25% reduction in extra cases identified by RSV-WEM; 50%-90% prophylaxis coverage based on real-world data; and 25% increase in the cost of RSV-WEM.</p><p><strong>Conclusions: </strong>The integration of RSV-WEM appears a highly cost-effective strategy (<i>vs</i> RSV-CS exclusively) to guide the earlier launch of RSV seasonal prophylaxis in Canada.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"354-362"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143523709","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}
Bernadette Pöllinger, Amin Haiderali, Min Huang, Burcu Akyol Ersoy, Ahmed H Abdelaziz, Loay Kassem, Gihan Hamdy Elsisi
{"title":"The cost-effectiveness of treatment for high-risk, early-stage, triple-negative breast cancer in Egypt: an analysis of neoadjuvant pembrolizumab plus chemotherapy followed by adjuvant single-agent pembrolizumab.","authors":"Bernadette Pöllinger, Amin Haiderali, Min Huang, Burcu Akyol Ersoy, Ahmed H Abdelaziz, Loay Kassem, Gihan Hamdy Elsisi","doi":"10.1080/13696998.2024.2441073","DOIUrl":"10.1080/13696998.2024.2441073","url":null,"abstract":"<p><strong>Objective: </strong>The cost-effectiveness of neoadjuvant pembrolizumab + chemotherapy followed by adjuvant pembrolizumab compared to neoadjuvant chemotherapy plus placebo followed by adjuvant placebo was assessed in high-risk, early-stage, triple-negative breast cancer patients from an Egyptian societal perspective over a lifetime horizon.</p><p><strong>Methods: </strong>A 4-state Markov cohort model was developed to compare the cost-effectiveness of pembrolizumab + chemotherapy/pembrolizumab vs chemotherapy alone for the treatment of high-risk, early-stage, triple-negative breast cancer. The model simulated the clinical course of high-risk, early-stage, triple-negative breast cancer across four health states: event-free survival, locoregional recurrence, distant metastasis, and death. Clinical inputs for the simulation were derived from modeling of efficacy and safety data collected in the KEYNOTE-522 trial. Direct medical costs and indirect costs were reported in 2022 Egyptian pounds (EGP) and converted to US dollars ($). Probabilistic and deterministic sensitivity analyses were conducted to assess the robustness of model results.</p><p><strong>Results: </strong>Compared with chemotherapy alone, pembrolizumab + chemotherapy/pembrolizumab led to expected gains of 2.92 life years and 2.25 quality-adjusted life years, respectively, while increasing overall treatment costs by EGP 491,695 ($102,436). Incremental costs per year gained were EGP 218,285 ($45,476) per quality-adjusted life year and EGP 168,223 ($35,046) per life year, both of which were lower than the 2022 Egyptian cost-effectiveness threshold of EGP 398,439 ($83,008). The findings of sensitivity analyses indicated that the model was robust across a range of inputs and assumptions.</p><p><strong>Conclusions: </strong>In Egypt, pembrolizumab + chemotherapy/pembrolizumab is a cost-effective treatment for high-risk, early-stage, triple-negative breast cancer when considering health-related quality-of-life and years of life gained.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"105-113"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142813613","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}
Nannan Li, Marian Majoie, Silvia Evers, Kim Rijkers, Felix Gubler, Rob Rouhl, Richard Lazeron, Pim Klarenbeek, Vicki Laskier-Owens, Mickaël Hiligsmann
{"title":"Budget impact analysis of cenobamate for epilepsy patients with drug-resistant focal onset seizures in the Netherlands.","authors":"Nannan Li, Marian Majoie, Silvia Evers, Kim Rijkers, Felix Gubler, Rob Rouhl, Richard Lazeron, Pim Klarenbeek, Vicki Laskier-Owens, Mickaël Hiligsmann","doi":"10.1080/13696998.2024.2443338","DOIUrl":"10.1080/13696998.2024.2443338","url":null,"abstract":"<p><strong>Objective: </strong>The objective of this study was to explore the financial consequences of adopting cenobamate as a treatment alternative in epilepsy patients with drug-resistant focal onset seizures (FOS) from a societal perspective in the Netherlands.</p><p><strong>Methods: </strong>A previous budget impact model with a 5-year time horizon was adapted to the Dutch setting accounting for the eligible population, real-world market shares, treatment effectiveness and resource use in two scenarios: cenobamate with constant market share versus cenobamate with linearly increased market share up to 20%. Clinical inputs included treatment response, seizure reduction and adverse events. Costs consisted of drugs, medical and non-medical costs. One-way sensitivity analysis and scenario analysis were conducted to test the robustness of our results.</p><p><strong>Results: </strong>14,723 patients were eligible for cenobamate in 2022. Although cenobamate adds a gross budget impact of €12,686,30, the displacement of other drugs yields a total impact on the drug budget of €3,722,596 over 5 years. Adopting cenobamate resulted in a medical cost savings of €13,499,498 due to less resource use, and non-medical cost savings of €22,144,054 due to reduced productivity losses. Overall, savings generated at medical and non-medical cost level offset the gross drug budget impact of cenobamate, resulting in a saving of €31,920,955 over 5 years. Results were robust in the sensitivity/scenario analyses.</p><p><strong>Conclusion: </strong>Treatment with cenobamate is associated with both medical and non-medical cost savings, which offset the increase in drug budget and result in a significant potential budget saving. The higher the market share of cenobamate, the larger the budget savings. We acknowledge several limitations; Complex scenarios such as drug interactions, stopping/switching drugs, and multiple drug use were not taken into account. The long-term efficacy and safety of cenobamate and its comparators remains uncertain. Future real-world data are needed to confirm our findings.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"114-123"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142837298","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":"2024 in review: high impact articles from the <i>Journal of Medical Economics</i>.","authors":"Ivo Abraham, Mike Gregg","doi":"10.1080/13696998.2025.2455859","DOIUrl":"https://doi.org/10.1080/13696998.2025.2455859","url":null,"abstract":"","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"28 1","pages":"221-223"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143052794","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":"Patterns of responses in time trade-off studies: a latent class analysis of health states worse than dead.","authors":"Adam B Smith, Stuart Mealing, Andria Joseph","doi":"10.1080/13696998.2025.2485627","DOIUrl":"10.1080/13696998.2025.2485627","url":null,"abstract":"<p><strong>Aim: </strong>The study was to characterize participant responses in a time trade-off (TTO) task involving states worse than dead (WTD) to determine patterns of congruence and incongruence.</p><p><strong>Methods: </strong>The online TTO task involved 4 hypothetical health states describing a rare paediatric condition. Participants completed the task from a parental perspective with a 10-year time horizon. Mean health utilities were derived for the health states (HS). Congruence patterns were defined <i>a priori</i> as \"perfect\", i.e. completely logical sequence of health state utilities, \"incongruent\", reversal of the most and least severe HS utilities, and \"mixed\" congruence. A multinomial regression and latent class analysis (LCA) were applied to elucidate congruence patterns.</p><p><strong>Results: </strong>A total of 322 participants completed the TTO (49% females; average age 43 years). Perfect congruence response patterns were observed in only 11.5% (37) participants; 99 participants (30.8%) had incongruent response patterns and 186 (57.8%) a mixture. Two (most severe) HS were rated WTD by the \"mixed\", but only one HS was rated WTD by the \"perfect\" congruence group, who rated the other 3 HS better than dead. The multinomial regression identified age and gender as potential factors influencing congruence patterns; the most impactful factor was the number of stated rated WTD. The LCA was not able to identify a single class of perfectly congruent responders.</p><p><strong>Limitations: </strong>This online TTO did not allow further exploration of individual choices through post-task participant interviews.</p><p><strong>Conclusions: </strong>Facets of TTO tasks such as states WTD may introduce an additional cognitive burden on participants and impact on congruence. However, incongruence was observed even within ostensibly perfectly congruent responses; conversely incongruent response patterns showed some internal coherence. Removing the latter in favour of the former may therefore also introduce bias. A balance may need to be struck between sacrificing perfect congruence and the inclusion of some incongruence to achieve greater representativeness of health state utilities.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"508-516"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143719851","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}
Vamsi Kota, David Wei, Daisy Yang, Hela Romdhani, Dominick Latremouille-Viau, Annie Guérin, Kejal Jadhav
{"title":"Non-optimal treatment with first tyrosine kinase inhibitor and associated economic burden in chronic myeloid leukemia.","authors":"Vamsi Kota, David Wei, Daisy Yang, Hela Romdhani, Dominick Latremouille-Viau, Annie Guérin, Kejal Jadhav","doi":"10.1080/13696998.2025.2506265","DOIUrl":"10.1080/13696998.2025.2506265","url":null,"abstract":"<p><strong>Aims: </strong>Although adenosine triphosphate (ATP)-competitive tyrosine kinase inhibitors (TKI) approved by the Food and Drug Administration have demonstrated effectiveness in treating chronic myeloid leukemia (CML), patients often experience intolerance or resistance, leading to non-optimal treatment (NOPT). This study assessed the treatment patterns, as well as NOPT and its associated economic burden, in newly diagnosed patients with CML receiving first TKI treatment.</p><p><strong>Methods: </strong>This retrospective study identified adult patients with ≥2 CML diagnoses who initiated first TKI treatment (imatinib, dasatinib, nilotinib, or bosutinib) in 2012 or later from United States administrative health claims databases (01 January 2007-30 June 2022). Treatment sequence and time to treatment discontinuation/switch were assessed. NOPT, identified based on treatment modification/adherence criteria, and its associated incremental healthcare resource utilization and medical costs were evaluated.</p><p><strong>Results: </strong>Nearly a quarter of patients experienced NOPT, as indicated by early treatment modifications or low treatment adherence. NOPT was associated with significant incremental healthcare resource utilization (80% more inpatient admissions; twice as many inpatient days; 30% more outpatient visits) and medical costs (adjusted mean cost difference = $13,551 per-patient-per-year).</p><p><strong>Limitations: </strong>Given the lack of information on reasons of treatment modification in health claims data, NOPT was identified based on indicators of management of intolerance and resistance to TKI.</p><p><strong>Conclusion: </strong>These findings highlight that unmet clinical needs and significant economic burden still persist in this population and that patients with CML may benefit from using therapeutic options with better efficacy and tolerability profiles as first treatment.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"743-752"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143996173","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}
Christina Qian, Karissa M Johnston, Maria Tinajero, M Lauren Voss, Austin Nam, Mackenzie A Hamilton
{"title":"Characterizing the clinical and economic burden of COVID-19 among individuals with immunocompromising conditions in Ontario, Canada - a matched, population-based observational study.","authors":"Christina Qian, Karissa M Johnston, Maria Tinajero, M Lauren Voss, Austin Nam, Mackenzie A Hamilton","doi":"10.1080/13696998.2025.2482372","DOIUrl":"10.1080/13696998.2025.2482372","url":null,"abstract":"<p><strong>Aims: </strong>COVID-19 continues to be associated with substantial burden among immunocompromised patients (IC). This study aimed to describe and compare outcomes during and following COVID-19 hospitalizations among IC and non-IC patients.</p><p><strong>Methods: </strong>Patients hospitalized with COVID-19 (January 2020-March 2023) were identified in Ontario health administrative claims databases. All eligible IC (≥1 of solid organ or stem cell transplant; hematological malignancy; rheumatoid arthritis; multiple sclerosis; or primary immunodeficiency) were matched (1:4) to eligible non-IC. Clinical, resource, and costburden were assessed during and post-hospitalization. Multivariate regression models were used to estimate relative risks (RRi), rates (RRa), and corresponding 95% confidence intervals (CIs), adjusting for neighborhood deprivation, long-term care residency, baseline comorbidities, and COVID-19 vaccination status.</p><p><strong>Results: </strong>9,283 IC hospitalized (mean age 68.7 years; 52.1% female) were matched to 37,127 non-IC. During index hospitalization, IC had greater risks of intensive care unit admission (RRi = 1.06 [1.01-1.12]), ventilation (RRi = 1.27 [1.19-1.36]), and all-cause mortality (RRi = 1.34 [1.27-1.41]) compared to non-IC. Within 30-days post-discharge, IC had greater rates of all-cause readmission (RRa = 1.33 [1.26-1.40]), emergency departments admission (RRa = 1.13 [1.08-1.18]), home oxygen use (RRi = 1.35 [1.15-1.58]), and COVID-19-related rehabilitation (RRa = 1.52 [1.22-1.89]), resulting in 21% (16%-25%) and 51% (45%-58%) greater costs in hospital and post-discharge, respectively. All-cause mortality remained approximately 5% higher for IC vs. non-IC at 30- and 60-days post-discharge (<i>p</i> < .001). Resource use remained elevated among IC with 57% (50%-64%) greater costs within 180 days post-discharge.</p><p><strong>Limitations: </strong>Unmeasured confounding remains; hospital prescription data were not available such that treatments for COVID-19 were not captured. Attribution of post-discharge resource use and costs to COVID-19 was subject to greater uncertainty further from the index hospitalization.</p><p><strong>Conclusion: </strong>IC experienced more severe COVID-19 hospitalization outcomes compared to non-IC. COVID-mitigating policies and prophylactic treatments are needed to protect immunocompromised populations.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"479-493"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143753160","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}
Olivia M Dong, Na'ngono Manga, Yue Zhong, Yuanhui Zhang, Taryn Krause, Hannah Hancock, James Brockbank, Jud Griffin, William L Herring, Sorrel Wolowacz
{"title":"Use of patient-reported outcome measures in cost-effectiveness models informing health technology assessments of non-oncology treatments: a review.","authors":"Olivia M Dong, Na'ngono Manga, Yue Zhong, Yuanhui Zhang, Taryn Krause, Hannah Hancock, James Brockbank, Jud Griffin, William L Herring, Sorrel Wolowacz","doi":"10.1080/13696998.2025.2522010","DOIUrl":"10.1080/13696998.2025.2522010","url":null,"abstract":"<p><strong>Objective: </strong>Including patient-reported outcome measures (PROMs) in cost-effectiveness models (CEMs) represents an important opportunity to reflect patients' perspectives in health technology assessments (HTAs). Beyond utility measures, little is known about how PROMs are used in CEMs and their acceptance by reviewers. We elucidated how PROMs were incorporated into CEMs submitted to the UK National Institute for Health and Care Excellence (NICE) for non-oncology treatments and identified feedback from NICE and external assessment groups (EAGs).</p><p><strong>Methods: </strong>NICE HTAs for non-oncology treatments from January 2016 to August 2022 which used a PROM (other than a generic utility measure) as a key element in the CEM were reviewed. Information on how PROMs were used, rationale for their use, and feedback from NICE and EAGs were summarized.</p><p><strong>Results: </strong>Of 428 HTAs identified, 27 (6.3%) were included in our review. PROMs were included in the CEMs <i>via</i> treatment-effect parameters (<i>n</i> = 25/27, 92.6%), model structure (<i>n</i> = 25/27, 92.6%), treatment-stopping rules (<i>n</i> = 21/27, 77.8%), and/or condition-specific utility measures (<i>n</i> = 18/27, 66.7%). Of the 27 HTAs, 22 (81.5%) provided justification for using a PROM in the CEM, most frequently citing the PROM validation. NICE and/or the EAG raised concerns in 20 HTAs (74.1%) regarding the use of PROMs, most commonly regarding the data source, statistical methods, and implementation of the PROM in clinical practice.</p><p><strong>Limitations: </strong>This review did not focus on ways the patient voice may be reflected in the NICE process beyond incorporation of PROMs in CEMs. Generalizability of the findings to other HTA settings may be limited.</p><p><strong>Conclusions: </strong>NICE accepted the use of PROMs in CEMs where high-quality data relevant to the target population and appropriate statistical methods were used. Few CEMs used PROMs other than utility measures. Further research is needed to understand the value of PROMs in CEMs for HTAs.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1045-1060"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144600710","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}
John J Volpi, Scott E Kasner, Tjeerd Looman, Giorgia Tiozzo, Timon Louwsma, Ryan J Imhoff, Erik J Landaas
{"title":"Projected clinical and economic benefits of improved patent foramen ovale testing among cryptogenic stroke patients in the United States.","authors":"John J Volpi, Scott E Kasner, Tjeerd Looman, Giorgia Tiozzo, Timon Louwsma, Ryan J Imhoff, Erik J Landaas","doi":"10.1080/13696998.2025.2535236","DOIUrl":"10.1080/13696998.2025.2535236","url":null,"abstract":"<p><strong>Background: </strong>Accurate determination of stroke etiology is essential for effective secondary stroke prevention, yet 25% to 40% of ischemic strokes are classified as cryptogenic. Patent foramen ovale (PFO), a common finding in cryptogenic stroke, elevates the risk of strokes. However, underuse of diagnostic practices may lead to underdiagnosis of PFO, missing opportunities for guideline-recommended PFO closures and preventable recurrent strokes. This study estimates the value of improving testing for PFO among patients with cryptogenic stroke in the US.</p><p><strong>Methods: </strong>A cost-effectiveness analysis was conducted, employing a hybrid model including a decision tree and a Markov model to assess health outcomes and economic impacts from a US payor perspective over a life-time horizon. The model compared two PFO testing scenarios: the Current Diagnostic Scenario (54% testing) and Optimal PFO Diagnostics (100% testing). The decision tree evaluated diagnostic pathways for PFO (TTE, TEE, TCD), while the Markov model simulated patient progression through various health states (recurrent ischemic stroke, TIA, and death). Cost-effectiveness was determined using the incremental cost-effectiveness ratio (ICER) with a willingness-to-pay threshold of $75,000 per Quality-Adjusted Life Year (QALY).</p><p><strong>Results: </strong>In a simulated cohort of 1,000 patients, increasing the diagnostic testing rate for PFO from 54% to 100% is expected to prevent 63 recurrent strokes, resulting in 23 life years saved and 286 QALYs gained. This led to cost-savings of $1.9 million for payors, indicating a dominant economic position (ICER = -$6,770/QALY). The model estimated screening four patients would lead to identifying and closing one PFO, while screening seven would prevent one recurrent stroke. Thorough sensitivity analyses confirmed the robustness of these findings.</p><p><strong>Conclusions: </strong>Improving PFO diagnostic testing among patients with cryptogenic stroke is projected to result in improved health outcomes for patients, while yielding cost-savings, underscoring the importance of adhering to PFO diagnostic guidelines.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1137-1150"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144642711","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}
Marie Wislez, Andromachi Giannopoulou, Isabelle Borget, Colin Lindsay, Sacha I Rothschild, Bastien Vincent, Joseph Milce, Ihtisham Sultan, Björn Stollenwerk, Miklos Pless
{"title":"Clinical characteristics, healthcare resource use, and survival outcomes among patients with advanced NSCLC tested for KRAS mutations in France, the United Kingdom, and Switzerland.","authors":"Marie Wislez, Andromachi Giannopoulou, Isabelle Borget, Colin Lindsay, Sacha I Rothschild, Bastien Vincent, Joseph Milce, Ihtisham Sultan, Björn Stollenwerk, Miklos Pless","doi":"10.1080/13696998.2025.2535225","DOIUrl":"10.1080/13696998.2025.2535225","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate real-world patient characteristics, healthcare resource use (HRU), and clinical outcomes among patients with advanced or metastatic (A/M) non-small cell lung cancer (NSCLC) stratified by KRAS mutation status (KRAS G12C, KRAS non-G12C, and KRAS wild-type[WT]).</p><p><strong>Methods: </strong>This retrospective chart review included adults with A/M NSCLC and known KRAS status who received second- or third-line non-targeted therapy (index therapy) in France, the UK, or Switzerland. Patient characteristics, HRU, and key clinical outcomes-including time to treatment discontinuation (TTD), progression-free survival (PFS), and overall survival (OS)-were analyzed using the Kaplan-Meier method and log-rank methods. Exploratory multivariate Cox models adjusted for clinical covariates.</p><p><strong>Results: </strong>The study included 211 patients (France: 192, UK: 13, Switzerland: 6), with 53.1% having KRAS G12C, 21.8% KRAS non-G12C, and 25.1% KRAS WT NSCLC. Median age was 66 years; 62.1% were male, and 95.8% were current/former smokers. Baseline characteristics were comparable across KRAS subgroups. HRU was high, including 125 unplanned healthcare provider visits, primarily to general practitioners (42.4%) and specialists (24.0%). Hospitalization was frequent (70.1% of patients), with 40.8% experiencing unplanned admissions, largely due to disease complications (54.2%) and grade 3/4 adverse events (24.4%). Median TTD, PFS, and OS were comparable across KRAS subgroups for second-line (4.4-4.7 months, 5.3-6.3 months, and 11.2-15.0 months) and third-line (3.2-4.1 months, 3.4-5.2 months, and 5.1-9.2 months) therapy. Multivariate analysis showed that KRAS status, performance status, histology, and comorbidities were not significantly associated with survival outcomes.</p><p><strong>Conclusions: </strong>Patients with advanced NSCLC, regardless of KRAS mutation status, experience a substantial disease burden, frequent hospitalizations, and poor clinical outcomes. These findings highlight the urgent need for more effective treatment options for advanced NSCLC, including therapies tailored to KRAS-mutated disease.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"1151-1167"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144674993","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}