Journal of Health Economics and Outcomes Research最新文献

{"title":"Retrospective Analysis of Burden of Illness of Congenital Pulmonary Valve Disease in a Large, Commercially Insured US Population.","authors":"Tariku J Beyene, Vincent J Willey, Malvika Venkataraman, Katherine Wong, Eric Anderson, Sophie C Hofferberth","doi":"10.36469/001c.156168","DOIUrl":"10.36469/001c.156168","url":null,"abstract":"<p><strong>Background: </strong>Babies born with congenital pulmonary valve disease (CPVD) face a lifetime burden of disease and significant medical expense beginning in their first year of life.</p><p><strong>Objectives: </strong>To assess costs and burden of care in patients with CPVD from birth through age 65.</p><p><strong>Methods: </strong>This retrospective study used administrative claims data to examine healthcare costs and utilization among commercially insured patients with ≥2 claims with CPVD diagnosis between January 1, 2006, and April 30, 2024; the earliest CPVD claim defined the index date. Per-patient-per-year costs were calculated by age category. Mean annual costs for each age group were multiplied by age category duration to estimate full age category period costs. Costs for each age category period were summed to estimate lifetime costs through 65 years of age. Sensitivity analyses were conducted in subgroups with continuous eligibility from birth through 60 months and 120 months.</p><p><strong>Results: </strong>Among eligible patients (N = 22 751), 53.2% experienced an average of 1.8 CPVD-related inpatient admissions during their first year: mean (standard deviation) cumulative admission days, 50.2 (74.2); with <math><mn>267</mn> <mrow><mo> </mo></mrow> <mn>945</mn> <mo>(</mo> <mn>737</mn> <mrow><mo> </mo></mrow> <mn>565</mn> <mo>;</mo> <mi>m</mi> <mi>e</mi> <mi>d</mi> <mi>i</mi> <mi>a</mi> <mi>n</mi> <mo>,</mo> <mn>6487</mn> <mo>[</mo> <mi>i</mi> <mi>n</mi> <mi>t</mi> <mi>e</mi> <mi>r</mi> <mi>q</mi> <mi>u</mi> <mi>a</mi> <mi>r</mi> <mi>t</mi> <mi>i</mi> <mi>l</mi> <mi>e</mi> <mi>r</mi> <mi>a</mi> <mi>n</mi> <mi>g</mi> <mi>e</mi> <mo>(</mo> <mi>I</mi> <mi>Q</mi> <mi>R</mi> <mo>)</mo> <mo>,</mo> <mn>0</mn> <mo>-</mo> <mn>238</mn> <mrow><mo> </mo></mrow> <mn>486</mn> <mo>]</mo> <mi>i</mi> <mi>n</mi> <mi>a</mi> <mi>s</mi> <mi>s</mi> <mi>o</mi> <mi>c</mi> <mi>i</mi> <mi>a</mi> <mi>t</mi> <mi>e</mi> <mi>d</mi> <mi>c</mi> <mi>o</mi> <mi>s</mi> <mi>t</mi> <mi>s</mi> <mo>.</mo> <mi>A</mi> <mi>m</mi> <mi>o</mi> <mi>n</mi> <mi>g</mi> <mi>t</mi> <mi>h</mi> <mi>o</mi> <mi>s</mi> <mi>e</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>i</mi> <mi>n</mi> <mi>p</mi> <mi>a</mi> <mi>t</mi> <mi>i</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>a</mi> <mi>d</mi> <mi>m</mi> <mi>i</mi> <mi>s</mi> <mi>s</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mi>s</mi> <mi>f</mi> <mi>o</mi> <mi>r</mi> <mi>C</mi> <mi>P</mi> <mi>V</mi> <mi>D</mi> <mo>-</mo> <mi>r</mi> <mi>e</mi> <mi>l</mi> <mi>a</mi> <mi>t</mi> <mi>e</mi> <mi>d</mi> <mi>p</mi> <mi>r</mi> <mi>o</mi> <mi>c</mi> <mi>e</mi> <mi>d</mi> <mi>u</mi> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mi>d</mi> <mi>u</mi> <mi>r</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>c</mi> <mi>h</mi> <mi>i</mi> <mi>l</mi> <mi>d</mi> <mi>h</mi> <mi>o</mi> <mi>o</mi> <mi>d</mi> <mo>,</mo> <mi>a</mi> <mi>v</mi> <mi>e</mi> <mi>r</mi> <mi>a</mi> <mi>g</mi> <mi>e</mi> <mi>a</mi> <mi>n</mi> <mi>n</mi> <mi>u</mi> <mi>a</mi> <mi>l</mi> <mi>c</mi> <mi>o<","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"56-65"},"PeriodicalIF":2.5,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13005609/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147498897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Fiscal Consequences for the Canadian Government of Efgartigimod in the Treatment of Generalized Myasthenia Gravis.","authors":"Ana Teresa Paquete, Mark P Connolly, Cynthia Qi, Hans Katzberg, Syed Raza, Charles Kassardjian, Zaeem A Siddiqi, Roger Kaprielian, Jason Locklin, Glenn A Phillips, Nikos Kotsopoulos","doi":"10.36469/001c.157709","DOIUrl":"10.36469/001c.157709","url":null,"abstract":"<p><p><b>Background:</b> Generalized myasthenia gravis (gMG) severely impacts activities of daily living. Productivity losses and the need for care can impact household finances and consequently government public accounts. This study adopts a governmental perspective framework to assess the fiscal consequences of treating gMG that is inadequately controlled by standard therapy, beyond healthcare costs. Savings in tax revenue loss and benefit payments are considered. <b>Objectives:</b> To value the fiscal consequences of treating adults with acetylcholine receptor-antibody positive (AChR-Ab+) gMG with efgartigimod vs current treatments. The lifetime impact on people living with gMG and their caregivers is considered from the perspective of Canada's public accounts. <b>Methods:</b> A lifetime Markov cohort simulation following adults with gMG according to their Activities of Daily Living (MG-ADL) score was linked to labor and fiscal stages of both patients and care- givers. Based on the MyRealWorld MG study, MG-ADL scores defined the labor market characteristics of both individuals with gMG and their caregivers. National statistics data on sex- and age-specific labor outcomes were used to model patients with minimal symptoms. Benefit payments and tax revenue losses attributable to gMG were estimated and valued according to national official sources. Public healthcare costs were included. The difference between efgartigimod and current treatments was assessed by discounted lifetime incremental fiscal consequences. Sensitivity analyses were applied to the fiscal parameters. <b>Results:</b> Without active treatments, the lifetime fiscal burden associated with individuals with gMG and their caregivers was estimated at CAD <math><mn>1.24</mn> <mi>m</mi> <mi>i</mi> <mi>l</mi> <mi>l</mi> <mi>i</mi> <mi>o</mi> <mi>n</mi> <mi>i</mi> <mi>n</mi> <mi>g</mi> <mi>o</mi> <mi>v</mi> <mi>e</mi> <mi>r</mi> <mi>n</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>e</mi> <mi>x</mi> <mi>p</mi> <mi>e</mi> <mi>n</mi> <mi>d</mi> <mi>i</mi> <mi>t</mi> <mi>u</mi> <mi>r</mi> <mi>e</mi> <mi>s</mi> <mo>.</mo> <mi>C</mi> <mi>o</mi> <mi>m</mi> <mi>p</mi> <mi>a</mi> <mi>r</mi> <mi>e</mi> <mi>d</mi> <mi>w</mi> <mi>i</mi> <mi>t</mi> <mi>h</mi> <mi>t</mi> <mi>h</mi> <mi>e</mi> <mi>c</mi> <mi>u</mi> <mi>r</mi> <mi>r</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>w</mi> <mi>e</mi> <mi>i</mi> <mi>g</mi> <mi>h</mi> <mi>t</mi> <mi>e</mi> <mi>d</mi> <mi>b</mi> <mi>u</mi> <mi>n</mi> <mi>d</mi> <mi>l</mi> <mi>e</mi> <mi>o</mi> <mi>f</mi> <mi>t</mi> <mi>r</mi> <mi>e</mi> <mi>a</mi> <mi>t</mi> <mi>m</mi> <mi>e</mi> <mi>n</mi> <mi>t</mi> <mi>s</mi> <mi>i</mi> <mi>n</mi> <mi>C</mi> <mi>a</mi> <mi>n</mi> <mi>a</mi> <mi>d</mi> <mi>a</mi> <mo>,</mo> <mi>e</mi> <mi>f</mi> <mi>g</mi> <mi>a</mi> <mi>r</mi> <mi>t</mi> <mi>i</mi> <mi>g</mi> <mi>i</mi> <mi>m</mi> <mi>o</mi> <mi>d</mi> <mi>w</mi> <mi>a</mi> <mi>s</mi> <mi>e</mi> <mi>s</mi> <mi>t</mi> <mi>i</mi> <mi>m</mi> <mi>a</mi> <mi>t</mi> <mi>e</mi> <m","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"85-92"},"PeriodicalIF":2.5,"publicationDate":"2026-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12986971/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147468272","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Modeling Financial Insolvency and Income Loss Insurance in Head and Neck Cancer.","authors":"Ana V Araujo, Murray J Bartho, Garren M I Low, Ryan J Li","doi":"10.36469/001c.155597","DOIUrl":"10.36469/001c.155597","url":null,"abstract":"<p><strong>Background: </strong>Head and neck cancer (HNC) carries high morbidity, and its treatment can be functionally devastating, impacting a patient's ability to work. While most patients have medical insurance benefits, studies on the impact of HNC on overall household finances have been limited.</p><p><strong>Objectives: </strong>This study explored the effect of HNC treatment on household finances and the feasibility of catastrophic income loss insurance.</p><p><strong>Methods: </strong>This cross-sectional study was based on a population-level survey of American adults. Participants, aged 35 to 64 years, were respondents to the US Federal Reserve 2023 Survey on Household Economics and Decisionmaking (SHED).</p><p><strong>Results: </strong>With total income loss, 16% of simulated HNC patients were insolvent after 3 months, rising to 49% at 6 months. With a 50% loss in income, 3% of patients were insolvent at 3 months, increasing to 5% at 6 months. If savings were liquid, 0.5% of patients were insolvent at 3 months, rising to 1.3% at 6 months.</p><p><strong>Discussion: </strong>Our findings underscore the substantial financial vulnerability faced by patients undergoing treatment for HNC. Even in a simulated model based on national economic data, nearly half of patients experiencing total income loss were insolvent by 6 months. Given the intensive and prolonged nature of HNC treatment, these financial challenges may compound physical and psychosocial stressors, affecting overall recovery and quality of life.</p><p><strong>Conclusion: </strong>The model suggested a need for more substantial income loss protection programs. Financial hardship applies to other cancer types and merits further study into the household financial impact of HNC and other cancers.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"80-84"},"PeriodicalIF":2.5,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12962255/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147377452","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Environmental Impact of Pentaspline Pulsed Field Ablation-Global Warming or Arctic Front?","authors":"Peter Calvert, Vishal Luther, Dhiraj Gupta","doi":"10.36469/001c.157672","DOIUrl":"10.36469/001c.157672","url":null,"abstract":"","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"79"},"PeriodicalIF":2.5,"publicationDate":"2026-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12944332/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147325991","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Antiretroviral Treatment Switch Among Treatment-Experienced People with HIV.","authors":"Benjamin Chastek, Uche Mordi, Mary J Christoph, Lisa B Le, Travis Lim, Sunil Majethia, Cassidy Trom, Joshua Cohen","doi":"10.36469/001c.156180","DOIUrl":"10.36469/001c.156180","url":null,"abstract":"<p><strong>Background: </strong>HIV requires lifelong continuous antiretroviral therapy (ART) to prevent morbidity and mortality and to reduce onward transmission. People with HIV (PWH) may switch ART regimens for various clinical and nonclinical reasons. Identifying patterns of ART switching can inform shared decision-making and optimize regimen selection among treatment-experienced PWH.</p><p><strong>Objectives: </strong>To describe and compare treatment switching among PWH by regimen in a large US claims database, including a subset of PWH enrolled in Medicare Advantage.</p><p><strong>Methods: </strong>A retrospective analysis of closed US medical and pharmacy claims data was conducted in treatment-experienced PWH with commercial insurance or Medicare Advantage with Part D coverage from the Optum Research Database. PWH who switched to an ART regimen of interest (bictegravir/emtricitabine/tenofovir alafenamide [B/F/TAF], dolutegravir/lamivudine, dolutegravir/abacavir/lamivudine, dolutegravir + emtricitabine/tenofovir alafenamide, dolutegravir + emtricitabine/tenofovir disoproxil fumarate, and cabotegravir + rilpivirine) between July 1, 2017, and November 30, 2023, were included. Baseline characteristics were balanced across regimens in the overall study population using inverse probability treatment weighting. Time to switch/add-on was estimated using Kaplan-Meier analyses. Adjusted hazard ratios for the entire follow-up period were calculated using multivariable Cox proportional hazards models. Outcomes were assessed in the overall population and in the subset enrolled in Medicare Advantage.</p><p><strong>Results: </strong>Overall, 14 826 treatment-experienced PWH were included in the study. The proportion of PWH without switch/add-on after the first 12 months of follow-up was greater for those who were taking B/F/TAF compared with those taking the other regimens of interest (all <i>P</i> < .001). The risk of switch/add-on was significantly lower for those who were taking B/F/TAF vs those taking the other regimens of interest (all <i>P</i> ≤ .001). Findings were similar for the subset of Medicare Advantage enrollees.</p><p><strong>Discussion: </strong>Due to more effective treatments, PWH have increased life expectancy, and a growing proportion are qualifying for Medicare coverage. This analysis showed that PWH taking B/F/TAF, both in the overall population and the Medicare Advantage subset, maintained their initial treatment regimen longer and had a reduced risk of treatment switch/add-on compared with the other regimens of interest.</p><p><strong>Conclusions: </strong>These findings suggest that B/F/TAF has a lower likelihood of treatment switch/add-on for treatment-experienced PWH than other current ART regimens.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"73-78"},"PeriodicalIF":2.5,"publicationDate":"2026-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12940528/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147325736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Paradox of Life Extension with Traditional Cost-Effectiveness Analysis: A Case Study with Duchenne Muscular Dystrophy.","authors":"Lauren Sedita, Alexa Klimchak, Eleanor Perfetto, Katherine Gooch, Daniel C Malone","doi":"10.36469/001c.156118","DOIUrl":"10.36469/001c.156118","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Traditional quality-adjusted life-year (QALY)-based cost-effectiveness analyses (CEAs) may inadvertently penalize treatments extending survival in populations with disabilities. Duchenne muscular dystrophy (DMD) is a rare, progressive disease diagnosed during childhood with significant morbidity, premature mortality, and considerable healthcare costs. The impact of delaying mortality on CEAs for DMD is unclear.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To evaluate the impact of delaying nonfatal progression and/or mortality using a QALY-based CEA for three hypothetical DMD treatments, assessing if treatment value increases by delaying morbidity and/or mortality.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A previously published, five-state QALY-based cost-effectiveness model for analyzing treatments in an early ambulatory DMD population was replicated, validated, and adapted to include an early nonambulatory (ENA) population. Maximum annual treatment price was determined for three hypothetical treatments individually compared for each population with standard of care (SoC): (A) delays nonfatal progression and mortality; (B) delays nonfatal progression; or (C) delays mortality. A 10-year delay was assessed for all three. Willingness-to-pay (WTP) thresholds ranged from &lt;math&gt;&lt;mn&gt;50&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;000&lt;/mn&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt;&lt;/math&gt; 200 000/QALY.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;In both populations, QALYs gained vs SoC were highest for the hypothetical treatment delaying both nonfatal progression and mortality. Maximum annual treatment price was highest for the hypothetical treatment delaying nonfatal progression only (both populations and all WTP thresholds). Delaying mortality alone consistently had a negative annual valuation (not cost-effective even at &lt;math&gt;&lt;mn&gt;0&lt;/mn&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt;&lt;/math&gt; -8685 to &lt;math&gt;&lt;mo&gt;-&lt;/mo&gt; &lt;mn&gt;2148&lt;/mn&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mi&gt;E&lt;/mi&gt; &lt;mi&gt;A&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt;&lt;/math&gt; -13 253 to $-3278 (ENA population). For all treatments, valuation for the ENA population was consistently lower.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Discussion: &lt;/strong&gt;These model results illustrate that delaying both nonfatal progression and mortality is less valuable in a traditional CEA than delaying nonfatal progression alone, even when the additional survival is at the patient's best possible health and lowest costs. These results emphasize the significant shortcomings of QALY-based CEAs for assessing the value of potential life-extending treatments for progressive, disabling diseases, like DMD.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusions: &lt;/strong&gt;These results suggest that within a traditional CEA, delaying mortality decreases a DMD treatment's value","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"56-62"},"PeriodicalIF":2.5,"publicationDate":"2026-02-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12922800/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147271229","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness Analysis of Trastuzumab-Emtansine as Adjuvant Therapy for HER2-Positive Early Breast Cancer with Residual Invasive Disease in Colombia.","authors":"Daniel Samacá-Samacá, Diego Ballén, Milton Lombana, Melissa Díaz-Puentes, Sergio Augusto Cáceres-Maldonado, Juliana Saavedra, Laura Prieto-Pinto","doi":"10.36469/001c.156056","DOIUrl":"10.36469/001c.156056","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Patients with HER2-positive (HER2+) breast cancer (BC) who have residual invasive disease after neoadjuvant therapy remain at a significantly increased risk of recurrence. Updated results from the KATHERINE trial demonstrated the clinical benefit of trastuzumab-emtansine (T-DM1), showing a significant improvement in event-free survival. However, in budget-constrained settings, assessing the cost-effectiveness of TDM-1 is essential to guide sustainable adoption.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To evaluate the cost-effectiveness analysis of adjuvant T-DM1 compared with trastuzumab in HER2+ early BC patients and residual disease, following neoadjuvant treatment, from the Colombian Health System perspective.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We conducted a Markov model with a lifetime horizon to evaluate the cost-effectiveness of T-DM1 using the updated results of the KATHERINE trial (8.4-year follow-up). The model comprises 6 health states: patients with residual invasive disease on adjuvant treatment, non-metastatic recurrence, remission after non-metastatic recurrence, metastatic disease (first-line [1LmBC] and subsequent lines [2LmBC]), and death. Direct medical costs were included. The primary outcome was quality-adjusted life-years, with both costs and effects discounted annually at a 5% rate. Model assumptions were based on current guidelines and validated by local experts. Sensitivity and scenario analysis were performed to confirm the reliability of the results. Prices are shown in 2024 US dollars (4071.35 Colombian pesos = US $1).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Under a willingness-to-pay threshold of 86% of the 2024 gross domestic product per capita (US &lt;math&gt;&lt;mn&gt;6831&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;T&lt;/mi&gt; &lt;mo&gt;-&lt;/mo&gt; &lt;mi&gt;D&lt;/mi&gt; &lt;mi&gt;M&lt;/mi&gt; &lt;mn&gt;1&lt;/mn&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;z&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mo&gt;.&lt;/mo&gt; &lt;mi&gt;C&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mo&gt;&gt;&lt;/mo&gt; &lt;mn&gt;50&lt;/mn&gt;&lt;/math&gt; -30 510) and 2LmBC (US $-6317). The probabilistic sensitivity analysis confirmed T-DM1 dominance in 81.2% of the 1000 Monte Carlo simulations, demonstrating consistency across settings.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;T-DM1 is a cost-effective and dominant strategy for","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"58-65"},"PeriodicalIF":2.5,"publicationDate":"2026-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12919760/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147271172","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Trial Interviews to Explore Glycogen Storage Disease Type Ia Patient Experiences Following Gene Therapy.","authors":"Diane M Turner-Bowker, Jessica Butler, Shayna Egan, David A Weinstein, David F Rodriguez-Buritica, Ayesha Ahmad, María-Luz Couce, Rebecca Riba-Wolman, John J Mitchell, Christina Theodore-Oklota","doi":"10.36469/001c.155666","DOIUrl":"10.36469/001c.155666","url":null,"abstract":"<p><strong>Background: </strong>Glycogen storage disease type Ia (GSDIa) is a rare, inherited, autosomal recessive deficiency of glucose-6-phosphatase (G6Pase), an enzyme necessary in glycogenolysis and gluconeogenesis. To maintain normal blood glucose levels and ensure survival, individuals living with GSDIa must frequently consume complex carbohydrates (eg, uncooked cornstarch). Dietary management can result in chronic complications and significant patient burden. DTX401 (pariglasgene brecaparvovec) is an investigational adeno-associated virus serotype 8 vector (AAV8)-based gene therapy designed to restore endogenous glucose production.</p><p><strong>Objectives: </strong>Patient experience interviews were conducted as part of an open-label, phase 1/2 dose-escalation trial (NCT03517085) evaluating the safety and efficacy of DTX401 in adults ≥18 years with GSDIa.</p><p><strong>Methods: </strong>Telephone interviews were conducted at Weeks 24, 52, and 104, using a semistructured interview guide. Qualitative interview data were audio recorded, transcribed, coded, and analyzed.</p><p><strong>Results: </strong>Most (86%; n = 6/7) reported overall symptom improvement and reduced burden following DTX401 treatment. Three (43%) reported no negative outcomes following gene therapy; 4 (57%) mentioned at least one negative change attributed to instances of blood sugar instability, lifestyle, or diet adjustments. Satisfaction fluctuated across timepoints; however, most were somewhat satisfied/very satisfied with gene therapy at Weeks 24 (80%), 52 (86%), and 104 (86%). No participants reported being very dissatisfied.</p><p><strong>Discussion: </strong>Following DTX401 treatment, most participants reported substantial reduction in cornstarch intake and corresponding improvements in symptoms, physical function, diet management, emotional function, self-perception, social function, sleep quality, work performance, and overall health. Few negative changes were reported. While some results regarding met expectations were mixed, most indicated they would still want gene therapy even if they had to continue cornstarch and if they had continued diet restrictions, and most reported satisfaction with treatment. While the study had limitations, interview results suggest that DTX401 helps to address aspects of the condition and treatment that patients have identified as burdensome.</p><p><strong>Conclusions: </strong>Most interviewees in this open-label trial of investigational DTX401 described positive experiences, including substantial reduction in burden and improved health-related quality of life following treatment throughout the trial. To optimize patient outcomes and experience with gene therapy, guidance on and close monitoring of dietary changes during implementation should be provided.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"39-47"},"PeriodicalIF":2.3,"publicationDate":"2026-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12906305/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146201905","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Effectiveness of Continuously Diffused Oxygen Therapy Compared with Negative-Pressure Wound Therapy.","authors":"Matthew Mercurio, Lawrence A Lavery, Animesh Agarwal, Alisha Oropallo","doi":"10.36469/001c.155760","DOIUrl":"10.36469/001c.155760","url":null,"abstract":"<p><strong>Background: </strong>Continuous diffusion of oxygen (CDO) to wounds has demonstrated better effectiveness in healing wounds than negative-pressure wound therapy (NPWT). However, there is limited evidence regarding the cost-effectiveness of CDO therapy and its comparison with NPWT.</p><p><strong>Objectives: </strong>The purpose of this analysis is to report on the cost-effectiveness of CDO and NPWT using published clinical results as well as real-world clinical outcomes and cost information. The objectives included analyzing the cost-effectiveness of CDO therapy across multiple wound types and anatomical locations, testing the data for robustness, and comparing the cost-effectiveness using results from controlled clinical studies for CDO and NPWT.</p><p><strong>Methods: </strong>A prospective patients database using real-world clinical results of 764 patients treated using CDO therapy in a broad range of clinical practices across a wide range of wound types and wound locations was analyzed. The clinical data were combined with real world billing data to draw statistically valid cost comparisons. Using 3 methodologies, the cost savings were demonstrated across 2 healthcare systems.</p><p><strong>Results: </strong>In the US, the average cost savings of CDO was US $14 238 vs NPWT to heal a wound. Kaplan-Meier analysis showed that CDO use in clinical practice had 79.2% full closure in 112 days compared with NPWT, which has 43.2% full closure in the same timeframe for similar wound sizes and severity.</p><p><strong>Discussion: </strong>There are two primary reasons for the significant cost savings. First, CDO therapy is much easier to apply and maintain than traditional NPWT, as CDO dressings can easily be changed by patients at home without the assistance of a nurse. The second reason is the substantially higher efficacy of CDO therapy.</p><p><strong>Conclusion: </strong>CDO is highly efficacious in clinical practice and cost-effective compared with NPWT and other therapies such as moist wound therapy and hyperbaric oxygen.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"30-38"},"PeriodicalIF":2.3,"publicationDate":"2026-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12900533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146201973","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Erratum: Cost-Effectiveness of an Absorbable Antibacterial Envelope for Infection Control in Cardiac Implantable Electronic Device Procedures in Spain.","authors":"Tomás Datino, Daniela Afonso, Elana Greaves, Simon Eggington, Julen Monje, María Álvarez Orozco, Claudia Wolff, Stuart Mealing, Arístides de Alarcón","doi":"10.36469/001c.156166","DOIUrl":"10.36469/001c.156166","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.36469/jheor.2026.154974.].</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"20-29"},"PeriodicalIF":2.3,"publicationDate":"2026-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12883256/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146149895","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}