Journal of Medical Economics最新文献

{"title":"Incorporating underreporting of epidemiological burden in COVID-19 models: a targeted literature review.","authors":"Ishveen Chopra, Jingyan Yang, Alon Yehoshua, Carlos Fernando Mendoza, Manuela Di Fusco","doi":"10.1080/13696998.2026.2613591","DOIUrl":"10.1080/13696998.2026.2613591","url":null,"abstract":"<p><strong>Background: </strong>Underreporting of infections, hospitalizations, and deaths can pose challenges to accurately estimating the true burden of COVID-19. Consequently, health burden assessments and economic evaluations may underestimate the public health impact of interventions such as vaccination.</p><p><strong>Methods: </strong>This targeted literature review summarized economic evaluations of COVID-19 that reported having adjusted for underreporting of epidemiological burden. Searches were performed in PubMed through 08/31/2025 with no geographic restrictions. Key study characteristics extracted: country, time period, population, parameters adjusted for underreporting, and the adjustment multipliers used. A high-level quality assessment of evidence was conducted, building on Drummond checklist and CHEERS. Given the qualitative nature of the question and the expected heterogeneity in study designs, the results were summarized qualitatively.</p><p><strong>Results: </strong>A total of 20 studies met the inclusion criteria. Of these, 14 (70%) reported numerical adjustment factors, and the remaining 30% did not report a numerical factor. The studies covered diverse geographic regions and time frames, with adjustments applied to parameters such as infections, hospitalizations, and mortality. The study quality was moderate to high. The multipliers used ranged widely across studies: 1 to 5 for mortality, 1 to 5 for hospitalizations, and 1 to 10 for infections, where a value higher than 1.0 reflects an adjustment factor for underreporting. The methodologies used to estimate underreporting varied, including comparisons to excess mortality data, Monte Carlo simulations, and validation against external datasets.</p><p><strong>Limitations: </strong>Most studies used pandemic time horizons.</p><p><strong>Conclusions: </strong>This review identified 14 modelling studies reporting numerical adjustment factors. The studies used diverse approaches and adjustment factors, reflecting variability in data availability and estimation methods. Recognizing and standardizing these adjustments is crucial for improving the accuracy and comparability of health economic analyses that inform policy decisions. Further research could refine underreporting estimates and assess their impact on economic model outcomes.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"193-212"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146018588","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":"Time to access matters: patient gains from faster CAR T-cell reimbursement in Europe.","authors":"Yan Zhi Tan, Emily Breislin, Matthew Woods, Matthew Madin-Warburton, Daniel Gladwell, Alexandra Zang, Sachin Vadgama, Brett Doble, Adriana Wiesinger, Oriol Sola-Morales","doi":"10.1080/13696998.2026.2626239","DOIUrl":"10.1080/13696998.2026.2626239","url":null,"abstract":"<p><strong>Background: </strong>Despite clinical efficacy and cost-effectiveness, time to reimbursement for chimeric antigen receptor (CAR) T-cell therapies varies greatly across Europe. We examined these differences, and quantified potential patient benefits with faster access.</p><p><strong>Methods: </strong>A targeted literature search collated reimbursement statuses for all approved CAR T-cell therapy indications by 1 October 2024. Time to reimbursement decision (TTRD), defined as time between marketing authorization and published reimbursement decision, was assessed. By benchmarking against the TTRD of the three fastest countries, patient impact, in the form of potential gains in lives, life-years (LYs) and long-term survivors, for the first three approved indications for CAR T-cell therapies were estimated. Each outcome was multiplied by population size, incidence, CAR T-cell eligibility, and market share to obtain population-level impact. Scenario analyses were conducted by examining different TTRD assumptions.</p><p><strong>Results: </strong>Across 12 identified indications for CAR T-cell therapy, France, Germany, and Switzerland had the fastest TTRD, compared to Denmark, the Netherlands, and Ireland which had the slowest. Among an estimated annual incident population of 6,594 across 3 L + diffuse large B-cell lymphoma (DLBCL), mantle cell lymphoma (MCL), and pediatric acute lymphoblastic leukemia (ALL), 1,199 lives, 1,222 long-term survivorships, and 37,229 LYs, could potentially be gained by ensuring time to access across these countries was equivalent to the fastest three assessors in these 3L + DLBCL, MCL, and pediatric ALL.</p><p><strong>Conclusion: </strong>Access to CAR T-cell therapies varied widely across European countries. Faster access to these therapies can result in significant health gains. Ensuring prompt and equitable access to innovative therapies requires proactive alignments between manufacturers and HTA bodies, possibly through mechanisms such alternative pricing models and risk-sharing agreements.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"481-497"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146220053","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":"Health and productivity benefits of anti-PD-(L)1 agents for early-stage cancer treatment in Hungary.","authors":"Daniel Ladino, Karl Patterson, Máté Várnai, Éva Balogh, Vivek Khurana, Raquel Aguiar-Ibáñez","doi":"10.1080/13696998.2026.2626240","DOIUrl":"10.1080/13696998.2026.2626240","url":null,"abstract":"<p><strong>Aim: </strong>Anti-PD-(L)1 agents, inhibitors of programmed cell death protein 1 (PD-1) or its ligand (PD-L1), are established therapies that improve cancer management as well as the disease and societal burden of specific metastatic and early-stage cancers. The aim of the study was to determine the impact of adopting anti-PD-(L)1 agents for the treatment of all eligible patients with early-stage cancers versus reserving anti-PD-(L)1 agents for patients with metastatic disease alone in Hungary.</p><p><strong>Methods: </strong>This study evaluated two scenarios, one where anti-PD-(L)1 agents were used to treat all eligible early-stage disease case<i>s</i> (ESD scenario) of melanoma (stage IIB-C and III), renal cell carcinoma (RCC), and triple-negative breast cancer (TNBC) versus a reference scenario where anti-PD-(L)1 agents were only used to treat metastatic disease cases in Hungary (2024-2033). A Markov-modeling approach estimated the health outcomes and productivity losses from each scenario from a societal perspective. Outcomes included recurrence-/event-/disease-free life-years, total life-years, quality-adjusted life-years (QALYs), productive years (patients and caregivers), recurrences/events, active treatments for metastatic disease, and deaths. The cumulative health and productivity impact of ESD treatment with anti-PD-(L)1 agents in Hungary was the difference in health and productivity outcomes between the ESD and reference scenarios for the time horizon of the model.</p><p><strong>Results: </strong>ESD treatment with anti-PD-(L)1 agents was estimated to increase recurrence-/event-/disease-free life-years (+13.8%), total life-years (+3.7%), and QALYs (+4.7%), as well as productive work years for patients (+39.6%) and caregivers (+27.6%). Concurrently, there would be fewer recurrences/events (-31.0%), active treatments for metastatic disease (-34.0%), post-recurrence deaths (-30.3%), and total deaths (-23.1%).</p><p><strong>Conclusion: </strong>Investing in anti-PD-(L)1 agents for early-stage disease may not only increase the life expectancy and QALYs for patients in Hungary but also increase productive work years for both patients and caregivers in Hungary. In addition, it may also help to reduce metastatic disease treatments and cancer-related deaths.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"379-392"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146213448","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":"Delta price cost-effectiveness analysis of PCV21 vs PCV20 use in adults aged ≥65 years in Switzerland.","authors":"Muloongo Simuzingili, Andrea Favre-Bulle, Thomas Mutschler, Zinan Yi, Eleana Tsoumani, Kwame Owusu-Edusei","doi":"10.1080/13696998.2026.2622857","DOIUrl":"10.1080/13696998.2026.2622857","url":null,"abstract":"<p><strong>Introduction: </strong>This study evaluated the health and economic impacts of the 21-valent pneumococcal conjugate vaccine (PCV21) compared to the 20-valent pneumococcal conjugate vaccine (PCV20) in Swiss adults aged 65 years and older using the delta-price approach.</p><p><strong>Method: </strong>A published state-transition Markov model was used to track health and economic outcomes of invasive pneumococcal disease (IPD), inpatient non-bacteremic pneumococcal pneumonia (NBPP), and post-meningitis sequelae (PMS). Estimated quality-adjusted life years (QALYs)and cost outcomes were discounted at 3%. All costs were presented in 2024 Swiss Franc (CHF). Price premiums were estimated as the difference between the prices of PCV21 and PCV20, and the maximum premiums at which PCV21 remains cost-saving and cost-effective (at a willingness-to-pay threshold of CHF 40,000) were reported. A scenario analysis was conducted to include a pneumococcal polysaccharide vaccine 23-valent (PPSV23) and 13-valent pneumococcal conjugate vaccine (PCV13) vaccine-experienced population. Sensitivity analysis identified the input categories that were most influential on the price premiums.</p><p><strong>Results: </strong>Overall, PCV21 averted more cases of IPD, NBPP, PMS and associated deaths, and saved more direct costs than PCV20. Compared to PCV20, PCV21 was cost-saving until a price premium of CHF 25.10 and cost-effective up to a price premium of CHF 88.01. In the scenario analysis, PCV21 was cost-saving up to a price premium of CHF 25.38, and cost-effective up to a price premium of CHF 88.68. Vaccine effectiveness and disease costs were the most influential inputs on the price premiums.</p><p><strong>Conclusion: </strong>PCV21 provides greater health and economic benefits than PCV20 and is cost-effective over a range of price premiums.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"334-344"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146213527","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 treatment switching and healthcare costs of onabotulinumtoxinA and calcitonin gene-related peptide monoclonal antibodies in Medicare patients with chronic migraine: a retrospective claims analysis.","authors":"Yanan Dong, Ziling Mao, Annaliza Dominguez, Tae Jin Park","doi":"10.1080/13696998.2026.2630604","DOIUrl":"10.1080/13696998.2026.2630604","url":null,"abstract":"<p><strong>Aims: </strong>Evaluate treatment switching and healthcare costs of onabotulinumtoxinA (onabotA) compared to calcitonin gene-related peptide (CGRP) monoclonal antibodies (mAbs) for the preventive treatment of chronic migraine (CM).</p><p><strong>Materials and methods: </strong>Adult patients with CM who initiated onabotA or a CGRP mAb between 1 October 2021 and 31 December 2023 were identified from the Optum de-identified Clinformatics Data Mart database. Index date was the first recorded treatment claim and patients must have had continuous Medicare coverage 12-months pre- and post-index period. Treatment switching, defined as ≥1 claim for a different branded migraine preventive treatment in the 12 months post-index period, was evaluated. Two additional treatment switch definitions were evaluated. All-cause healthcare resource utilization and costs were evaluated over the 12-month follow-up. Multivariable logistic regression adjusted for baseline characteristic differences when comparing odds of switching between onabotA and CGRP mAbs. Patient information on CM severity was not available in the database and not reported.</p><p><strong>Results: </strong>Of 887 patients identified, 367 initiated onabotA and 520 a CGRP mAb as index treatment. After 12 months of follow-up, 8.7% of onabotA users and 18.3% of CGRP mAb users initiated a different branded migraine preventive treatment. After adjusting for differences in baseline characteristics, CGRP mAb users had 134% higher odds of switching treatment during the 12-month follow-up compared to onabotA users (OR, 2.34; 95% CI: 1.49, 3.67, <i>p</i> < 0.001), or 9.3% absolute risk difference. Results for additional treatment switch definitions were consistent. OnabotA and CGRP mAb users had comparable all-cause healthcare costs during the 12-month follow-up.</p><p><strong>Limitations: </strong>Outcomes could only be adjusted for known and observed confounders, which could introduce bias between comparators.</p><p><strong>Conclusions: </strong>Patients with CM on a CGRP mAb were significantly more likely to switch to a different branded migraine preventive treatment within 12 months of treatment initiation compared to those on onabotA. Total costs were comparable between treatments.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"563-573"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147271163","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 lorlatinib as first-line treatment for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer in Italy.","authors":"Ilaria Valentini, Filippo Rumi, Eugenio Di Brino, Alessandra Bearz, Giulia Pasello, Giuseppe Novelli, Roberto Di Virgilio, Michele Basile","doi":"10.1080/13696998.2026.2658451","DOIUrl":"10.1080/13696998.2026.2658451","url":null,"abstract":"<p><strong>Background: </strong>Lorlatinib is a third-generation anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor (TKI) with high central nervous system penetration and activity against resistance mutations. Although its clinical efficacy as first-line treatment for ALK-positive advanced non-small cell lung cancer (NSCLC) has been demonstrated, its economic value within the Italian healthcare system remains to be fully established. This study evaluated the cost-effectiveness of lorlatinib compared with alectinib as first-line therapy in Italy.</p><p><strong>Methods: </strong>A partitioned survival model with three health states (progression-free, progressed disease, and death) was developed from the Italian National Health Service and societal perspectives over a 30-year time horizon. Clinical efficacy inputs were derived from the CROWN trial and indirect treatment comparisons using network meta-analysis. Costs (2026 euros) included drug acquisition, subsequent treatments, healthcare resource use, adverse events, end-of-life care, and societal costs. Health outcomes were expressed as life-years (LYs) and quality-adjusted life-years (QALYs). Deterministic, probabilistic, and scenario analyses were conducted to assess uncertainty.</p><p><strong>Results: </strong>In the base case, lorlatinib was dominant over alectinib, providing higher benefits (+2.01 LYs; +1.66 QALYs) at lower costs (-€19,210 per patient). This finding was robust across pricing scenarios. Probabilistic sensitivity analysis showed an almost 98,40% probability of cost-effectiveness at Italian willingness-to-pay thresholds.</p><p><strong>Conclusions: </strong>From both NHS and societal perspectives, lorlatinib represents a cost-saving and clinically superior first-line treatment option compared with alectinib for patients with ALK-positive advanced NSCLC in Italy.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1372-1386"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147773718","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 ferumoxtran-enhanced macrophage-specific-MRI and PSMA-PET/CT versus ePLND for nodal staging in primary prostate cancer: a decision analysis based on updated phase-3 trial data.","authors":"Patrik Zamecnik, Jürgen Feuerstein, Michael Berghahn, Jelle Barentsz","doi":"10.1080/13696998.2026.2699645","DOIUrl":"https://doi.org/10.1080/13696998.2026.2699645","url":null,"abstract":"<p><strong>Background and objective: </strong>Accurate nodal staging in intermediate- to high-risk prostate cancer (PCa) is crucial for treatment decisions. While extended pelvic lymph node dissection (ePLND) is the standard, it is invasive and has a low diagnostic yield. A 2019 analysis suggested that non-invasive imaging such as PSMA-PET/CT and ferumoxtran-enhanced macrophage-MRI (m-MRI) is cost-effective, but at the possible expense of a small QALY loss compared to ePLND, based on limited evidence. Recent Phase 3 trials have provided new data, prompting a reevaluation. Therefore, the aim of this article is to update a model with recent trial data to assess the cost-effectiveness of m-MRI and PSMA-PET/CT versus ePLND in Germany.</p><p><strong>Material and methods: </strong>We adapted a Markov model to simulate lifetime outcomes for men with intermediate- to high-risk PCa from a German insurer's perspective, with costs updated to 2025 level. Diagnostic accuracy data were derived from recent multicenter trials. Costs and QALYs were calculated using a 3% discount rate. Sensitivity analyses tested uncertainties. A practical interactive online-tool is provided for clinical decision-making and research purposes, which can incorporate various input data (https://macrophage-mri.app).</p><p><strong>Results: </strong>Both imaging options were dominant over ePLND (€37,855; 18.11 QALYs). PSMA-PET/CT was €7,869 cheaper and gained 0.800 QALYs; m-MRI was €9,985 cheaper and gained 0.990 QALYs. m-MRI was superior, saving €2,116 and gaining 0.19 QALYs over PSMA-PET/CT. The probabilistic analysis showed that m-MRI was optimal over 95% of the time at an €80,000/QALY threshold; the probability of PSMA-PET/CT being optimal was less than 5%.</p><p><strong>Conclusions: </strong>An imaging-first approach outperforms routine ePLND, with m-MRI as a cost-effective option. PSMA-PET/CT's low sensitivity limits its usefulness, though it is still cheaper than ePLND. These results support including m-MRI in guidelines for initial staging.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1974-1983"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148471137","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":"Impact of screening all high-risk patients at hospital admission for methicillin-resistant <i>Staphylococcus aureus</i> in Spain: a cost-consequence analysis.","authors":"Vicens Diaz-Brito, Filipa de Aragão, Carmen Aguilar-Romero, Maria Gonzalez-Cuevas, Inmaculada Salcedo","doi":"10.1080/13696998.2026.2700422","DOIUrl":"https://doi.org/10.1080/13696998.2026.2700422","url":null,"abstract":"<p><strong>Aim: </strong>Screening for methicillin-resistant <i>Staphylococcus aureus</i> (MRSA) colonization by culture-based or molecular-based methods in hospitals identifies patients for decolonization, mitigating transmission. This study aimed at quantifying the impact of expanding MRSA screening at hospital admission in Spain to all patients at high risk of being colonized with MRSA (HRP), using on-demand molecular PCR instead of culture.</p><p><strong>Methods: </strong>In the base case, HRP were those admitted to coronary, burn, or hemodialysis units, hematology or oncology medical departments, intensive care units (ICU) or specified surgical departments. A decision-tree model was developed to compare MRSA screening strategies: S1 was preventive contact precautions (PrevCP) + screening with on-demand molecular PCR (Xpert MRSA NxGⁱ) applied to all HRP; S2 was PrevCP + culture-based screening applied to all HRP; S3 was current clinical practice in Spain of PrevCP + culture-based screening for ICU-admitted patients only. A 60-day time horizon was considered, and the hospital perspective was adopted. Model input values were obtained from publicly available data and expert opinion.</p><p><strong>Results: </strong>In a hospital with 20,000 annual admissions, S1 versus S2 was estimated to generate 7.4 more MRSA-positive tests, 4.3 more successful decolonizations, and 0.8 fewer MRSA-related infections. The increase in total testing costs (+61,447.4€) was more than compensated by savings in excess cost due to PrevCP (-792,211.0€) and infection-related costs (-18,271.2€). Estimated total cost savings with S1 versus S2 were 748,971.2€ (-438.8€/HRP tested). Screening with S1 versus S3 increased the number of HRP tested (1,524.0), identified 113.3 more MRSA-positive tests, with 12.6 fewer MRSA-related infections, and 3.4 fewer deaths. Estimated total cost savings with S1 versus S3 were 272,118.5€ (-3,278.0€/HRP tested).</p><p><strong>Conclusion: </strong>Screening all HRP with Xpert MRSA NxG in Spain compared with culture and current clinical practice could generate savings from a hospital perspective and prevent infections, nosocomial colonization, and mortality.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"1998-2009"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148471177","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":"Clinical and healthcare burden of myotonic dystrophy type 1 (DM1) in the United States: a claims-based study.","authors":"Johanna I Hamel, Mihail Samnaliev, Lauren Do, Diane Ito, Yi Pan, Ashish Dugar, Aaron Novack","doi":"10.1080/13696998.2026.2633923","DOIUrl":"10.1080/13696998.2026.2633923","url":null,"abstract":"<p><strong>Objectives: </strong>Myotonic dystrophy type 1 (DM1) is a multi-systemic disease affecting skeletal and smooth muscle, the eye, brain, and heart. No disease-modifying therapies are available. Prior studies on clinical care and economic burden combined DM1 and DM type 2. This study describes the US DM1 population aged ≥12 years, assessed organ system involvement over time, estimated healthcare resource utilization (HCRU), associated costs, and identified predictors of high-cost status.</p><p><strong>Methods: </strong>Linked open claims and EHR data (Clarivate) from 1 January 2015 to 25 August 2023 were analyzed. Eligible individuals had a DM1 diagnosis (index), ≥6 months pre-index data, and were aged ≥12 years; congenital DM was excluded. Organ system involvement rates up to 7 years post-index were estimated using the Kaplan-Meier method. Annual HCRU rates and costs to payers (2023 USD) were summarized by setting, type of care and specialty and compared by organ involvement. Predictors of high-cost status were evaluated using multivariable logistic regression.</p><p><strong>Results: </strong>The cohort included 1,343 people with DM1. Over 7 years, most experienced neurological, gastrointestinal, cardiac, pulmonary, and/or musculoskeletal system involvement. Annually post-index, approximately 24% of patients had emergency room visits, and 15% had inpatient visits. Common specialty visits included rehabilitation (13%), cardiology (13%), and neurology (11%). Frequent device use included noninvasive positive-pressure ventilation (12%), assistive devices (7%), and oxygen therapy (4%). Mean annual total cost of care was $20,687 (SD = $122,082), primarily driven by inpatient and outpatient hospital visits. Cardiac and pulmonary complications were significant predictors of high-cost status.</p><p><strong>Conclusion: </strong>DM1 imposes significant clinical and economic burden, marked by extensive organ involvement and variable healthcare costs. As any patient may progress to costly cardiac or pulmonary complications, early proactive management is critical. The complexity and heterogeneity of DM1 highlight ongoing unmet needs and the importance of developing effective treatments.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"641-653"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147321705","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":"Employment and productivity losses from cervical spinal cord injury: a 7-year nationwide cohort study in Taiwan, 2012-2018.","authors":"Wei-Ming Wang, Jung-Der Wang, Fuhmei Wang, Wei-Chih Lien","doi":"10.1080/13696998.2026.2637390","DOIUrl":"10.1080/13696998.2026.2637390","url":null,"abstract":"<p><strong>Objective: </strong>To investigate the extent to which employment and productivity losses could be prevented by mitigating cervical spinal cord injury (SCI) and concurrent traumatic brain injury (TBI) in Taiwan.</p><p><strong>Design: </strong>Using Taiwan's National Health Insurance Database, we identified individuals with cervical SCI and concurrent TBI, with the 7-year data collection period (2012-2018) and a general population cohort as the reference. Survival and employment status were extrapolated to lifetime and age using rolling extrapolation with restricted cubic spline regression and generalized linear models.</p><p><strong>Results: </strong>This study included 2,852 patients with cervical SCI and 367 with concurrent TBI. In cervical SCI, loss-of-life expectancy, loss-of-lifetime employment duration (LED), and loss-of-lifetime insured salary (LIS; a proxy for productivity) were 19.3 years, 7.5 years, and $122,998, respectively. The relative loss-of-LED of motor-vehicle-related cervical SCI with concurrent TBI was significantly higher than that without TBI (72.7% vs. 64.2%). The relative loss-of-LED in middle-aged cervical SCI was significantly higher than loss-of-LIS (58.2% vs. 63.5%), suggesting that total productivity was more severely affected than working time.</p><p><strong>Conclusion: </strong>This 7-year nationwide cohort provides the first lifetime employment and productivity loss estimates for cervical SCI, demonstrating that concurrent TBI and younger age amplify economic burden. The findings shift the policy focus from generic SCI prevention to specific, high-yield interventions: mandatory TBI screening, age-tailored vocational programs, and productivity-targeted workplace accommodations. These evidence-based figures enable precise cost-benefit analyses for preventive measures (e.g. enhanced helmet legislation, road safety) and rehabilitation investments, offering actionable data for Taiwan's health and labor policymakers.</p>","PeriodicalId":16229,"journal":{"name":"Journal of Medical Economics","volume":"29 1","pages":"819-834"},"PeriodicalIF":3.1,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147458118","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}