Journal of Medical Economics最新文献

{"title":"Budget impact analysis of cenobamate, a novel adjunctive therapy for the treatment of drug resistant focal onset seizures, from the Belgian healthcare payer perspective.","authors":"Kristl Vonck, Olivier Bodart, Sarah Weckhuysen, Ann Tanghe, Britt Callebaut, Caroline Verdonck, Lynn Van den Bergh, Benjamin Legros","doi":"10.1080/13696998.2025.2520182","DOIUrl":"10.1080/13696998.2025.2520182","url":null,"abstract":"<p><strong>Objective: </strong>Cenobamate has recently been introduced as new anti-seizure medication (ASM) for patients with focal onset seizures (FOS) who are insufficiently controlled despite the use of three previous ASMs. To date, few evaluations have addressed the budgetary impact for the healthcare payer of add-on ASMs in patients with drug-resistant epilepsy (DRE). This study aims to assess the budgetary implications for the Belgian health insurer, the National Institute for Health and Disability Insurance (NIHDI), if cenobamate were reimbursed for the adjuvant treatment of FOS in adults with DRE.</p><p><strong>Methods: </strong>A prevalence-based budget impact model (BIM) was developed from the perspective of the Belgian NIHDI, considering all direct healthcare costs over a 3-year time horizon. A standardized expert elicitation process with experienced epileptologists was conducted to collect data on Belgian clinical practice. Source data uncertainty impact was investigated through a one-way sensitivity analysis (OWSA).</p><p><strong>Results: </strong>Over a 3-year period, considering the cumulative drug costs of cenobamate, replacement of other third-generation ASMs, and savings generated at medical cost level, the introduction of cenobamate as adjunctive treatment for the target population was estimated to reduce the NIHDI budget by -€8,105,616. The robustness of these savings was confirmed through an OWSA.</p><p><strong>Conclusion: </strong>The savings at medical cost level fully offset the impact of cenobamate on the drug budget, leading to an overall healthcare budget saving of -€8,105,616 for NIHDI. This favourable outcome is largely due to cenobamate's high efficacy reflected in its high response rate and significant effect on reducing seizure frequency.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"990-1001"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144317125","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":"Using system dynamics modeling approach to strengthen health systems to combat cancer: a systematic literature review.","authors":"Xianwen Chen, Kuangyuan Cai, Yan Xue, Carolina Oi Lam Ung, Hao Hu, Mihajlo Jakovljevic","doi":"10.1080/13696998.2025.2450168","DOIUrl":"https://doi.org/10.1080/13696998.2025.2450168","url":null,"abstract":"<p><strong>Aim: </strong>Dynamic cancer control is a current health system priority, yet methods for achieving it are lacking. This study aims to review the application of system dynamics modeling (SDM) on cancer control and evaluate the research quality.</p><p><strong>Methods: </strong>Articles were searched in PubMed, Web of Science, and Scopus from the inception of the study to 15 November 2023. Inclusion criteria were English original studies focusing on cancer control with SDM methodology, including prevention, early detection, diagnosis and treatment, and palliative care. Exclusion criteria were non-original research, and studies lacking SDM focus. Analysis involved categorization of studies and extraction of relevant data to answer the research question, ensuring a comprehensive synthesis of the field. Quality assessment was used to evaluate the SDM for cancer control.</p><p><strong>Results: </strong>Sixteen studies were included in this systematic review predominantly from the United States (7, 43.75%), with a focus on breast cancer research (5, 31.25%). Studies were categorized by WHO cancer control modules, and some studies may contribute to multiple modules. The results showed that included studies comprised two focused on prevention (1.25%), ten on early detection (62.50%), six on diagnosis and treatment (37.50%), with none addressing palliative care. Seven studies presented a complete SDM process, among which nine developed causal loop diagrams for conceptual models, ten utilized stock-flow charts to develop computational models, and thirteen conducted simulations.</p><p><strong>Limitations: </strong>This review's macrofocus on SDM in cancer control missed detailed methodological analysis. The limited number of studies and lack of stage-specific intervention comparisons limit comprehensiveness. Detailed analysis of SDM construction was also not conducted, potentially overlooking nuances in cancer control strategies.</p><p><strong>Conclusion: </strong>SDM in cancer control is underutilized, focusing mainly on early detection and treatment. Inconsistencies suggest a need for standardized SDM approaches. Future research should expand SDM's application and integrate it into cancer control strategies.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"28 1","pages":"168-185"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142950177","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":"Real-world perioperative treatment patterns and economic burden of recurrence in early-stage HER2-negative breast cancer: a SEER-Medicare study.","authors":"Jagadeswara Rao Earla, Kalé Kponee-Shovein, Allison W Kurian, Malena Mahendran, Yan Song, Qi Hua, Annalise Hilts, Yezhou Sun, Kim M Hirshfield, Jaime A Mejia","doi":"10.1080/13696998.2024.2439228","DOIUrl":"10.1080/13696998.2024.2439228","url":null,"abstract":"<p><strong>Aim: </strong>This study aimed to describe treatment patterns and quantify the economic impact of recurrence in early-stage human epidermal growth factor receptor 2 (HER2)-negative breast cancer (BC).</p><p><strong>Materials & methods: </strong>Medicare beneficiaries with stages I-III HER2-negative BC and lumpectomy or partial/total mastectomy were identified from SEER-Medicare data (2010-2019). Perioperative therapies were reported in the neoadjuvant and adjuvant setting. Locoregional recurrence and distant metastasis were identified using a claims-based algorithm developed with clinical input and consisting of a diagnosis-based and treatment-based indicator. All-cause and BC-related healthcare resource utilization (HRU) per-patient-month and monthly healthcare costs were estimated from the recurrence date for patients with recurrence and from an imputed index date for patients without recurrence using frequency matching. HRU and costs were compared between groups stratified by hormone receptor-positive (HR+) or triple negative BC (TNBC) using multivariable regression models.</p><p><strong>Results: </strong>Of 28,655 patients, 8.5% experienced recurrence, 90.4% had HR+ disease, and 5.6% received neoadjuvant therapy. Relative to patients without recurrence, patients with recurrence had more advanced disease (stage II/III: 73.7% vs. 34.0%) and higher-grade tumors (Grade 3/4: 40.6% vs. 18.0%) at diagnosis. Recurrence in HR+/HER2-negative BC and TNBC was associated with higher rates of all-cause hospitalizations (incidence rate ratio [IRR]: 2.84 and 3.65), emergency department (ED) visits (IRR: 1.75 and 2.00), and outpatient visits (IRR: 1.46 and 1.55; all <i>p</i> < 0.001). Similarly, recurrence was associated with higher rates of BC-related HRU, particularly for ED visits in HR+/HER2-negative BC (IRR: 4.24; <i>p</i> < 0.001) and hospitalizations in TNBC (IRR: 11.71; <i>p</i> < 0.001). Patients with HR+/HER2-negative BC and TNBC recurrence incurred higher monthly all-cause (cost difference [CD]: $3988 and $4651) and BC-related healthcare costs (CD: $3743 and $5819).</p><p><strong>Conclusions: </strong>Our findings highlight the considerable economic burden of recurrence in early-stage HER2-negative BC and underscore the unmet need for optimization of therapies that reduce recurrence in this population.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"54-69"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142794908","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":"Analyzing factors influencing hospitalization costs for five common cancers in China using neural network models.","authors":"Hong Jiang, Sinuo Ren, Shengbo Zhang, Xudan Luo, Rui He, Shuai Fei Wang, Jian Dong Yan, Shan Zhou, Chengliang Yin, Ying Xiao, Zhihuan Li","doi":"10.1080/13696998.2025.2494459","DOIUrl":"https://doi.org/10.1080/13696998.2025.2494459","url":null,"abstract":"<p><strong>Background: </strong>Malignant tumors are a major global health crisis, causing 25% of deaths in China, with lung, liver, thyroid, breast, and colon cancers being the most common. Understanding the factors influencing hospitalization costs for these cancers is crucial for public health and economics. This study aimed to identify key cost factors and develop a neural network model for predicting hospitalization costs, thereby providing tools to ease the financial burden on patients and healthcare systems.</p><p><strong>Methods: </strong>Data on hospitalization costs for 30,893 cancer patients from secondary or higher-level hospitals in Zhuhai, Guangdong Province, between 2017 and 2022, were analyzed. Neural network classification and feature importance analysis were used to determine the main factors influencing costs and to develop predictive models. Model performance was evaluated using the area under the receiver operating characteristic curve (AUROC), with a 95% confidence interval (CI) calculated for the AUROC value.</p><p><strong>Results: </strong>The key factors influencing hospitalization costs for lung cancer are metastasis and malignant solid tumor (MST), with correlation coefficients of 0.126 and 0.086, respectively, both showing statistical significance (<i>p</i> < 0.05). For colon cancer, the key factors influencing hospitalization costs are mortality and coronary disease (CD), with correlation coefficients of 0.092 and 0.090, respectively, both demonstrating statistical significance (<i>p</i> < 0.05). The AUROC value for the lung cancer model is 0.9078 (95% CI = 0.8975-0.9186), and the AUROC value for the colon cancer model is 0.9017 (95% CI = 0.8848-0.9196).</p><p><strong>Conclusion: </strong>This study confirmed the strong clinical applicability of the neural network predictive model in analyzing hospitalization costs for lung and colon cancer and revealed the factors that influence hospitalization costs for these cancers.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"28 1","pages":"615-624"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143999910","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 of using cariprazine as the first versus subsequent adjunctive therapy for major depressive disorder.","authors":"Prakash S Masand, Anita H Clayton, Mousam Parikh, François Laliberté, Guillaume Germain, Malena Mahendran, Cristina Martinez, Nadia Nabulsi","doi":"10.1080/13696998.2025.2457872","DOIUrl":"10.1080/13696998.2025.2457872","url":null,"abstract":"<p><strong>Aim: </strong>Inadequate response to antidepressant therapy (ADT) is common in major depressive disorder (MDD); atypical antipsychotic (AA) adjunctive therapy may be effective for these patients. This study aimed to compare healthcare resource utilization (HRU) and costs between patients initiating the AA cariprazine as their first adjunctive therapy vs those initiating cariprazine subsequently.</p><p><strong>Methods: </strong>The Merative MarketScan Commercial Database (January 1, 2015, to June 30, 2021) was used to identify US adults with MDD and ≥1 pharmacy claim for cariprazine adjunctive to ADT in 2018 or after. Rates of mental health (MH)‑related and all‑cause HRU per patient-year (PPY) and mean healthcare costs per-patient-per-year (PPPY) were assessed after patients first initiated adjunctive therapy. HRU and costs were compared between cohorts using rate ratios (RRs) and mean cost differences, respectively, estimated from multivariable regression models.</p><p><strong>Results: </strong>Of 838 patients receiving cariprazine, 44.7% initiated cariprazine as their first adjunctive therapy to ADT, and 55.3% initiated it subsequently. Those initiating cariprazine first had significantly lower rates of MH‑related hospitalizations (RR [95% confidence interval] = 0.55 [0.30, 0.90], <i>p</i> = .020) and outpatient (OP) visits (0.67 [0.57, 0.82], <i>p</i> < .001) PPY than those initiating cariprazine subsequently. Moreover, patients initiating cariprazine as their first adjunctive therapy had lower annual total MH‑related healthcare costs (mean cost difference [95% confidence interval] -$2,182 [-$4,206, -$69], <i>p</i> = .040), driven primarily by lower OP visit costs (-$1,511 [-$2,330, -$615], <i>p</i> < .001). Similar trends were observed for all-cause HRU and costs.</p><p><strong>Limitations: </strong>This was a retrospective analysis of secondary data with limited follow-up. Claims were a proxy for cariprazine use.</p><p><strong>Conclusions: </strong>Results from this real‑world study of commercially insured US adults suggest that initiating cariprazine as the first adjunctive therapy rather than a subsequent therapy could help mitigate the considerable economic burden of MDD for appropriate patients.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":" ","pages":"235-244"},"PeriodicalIF":2.9,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143023832","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 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}

{"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}

{"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}