Journal of Health Economics and Outcomes Research最新文献

{"title":"Peripheral T-cell Lymphoma in Japan: Real-World Patient Characteristics, Treatment Patterns, Healthcare Resource Utilization, and Costs.","authors":"Charles Dharmani, Oluwatosin Fofah, Pingping Qu, Zoe Jiang, Jingjian Wang, Li Li, Yijing Tao, Lin Song","doi":"10.36469/001c.161191","DOIUrl":"10.36469/001c.161191","url":null,"abstract":"<p><strong>Background: </strong>Peripheral T-cell lymphomas (PTCLs) are a rare and heterogeneous group of non-Hodgkin lymphomas associated with aggressive clinical course and suboptimal outcomes. PTCL accounts for a higher proportion of non-Hodgkin lymphoma cases in Japan compared with Western countries; however, real-world evidence on treatment patterns, healthcare resource utilization (HRU), and costs in Japan is limited.</p><p><strong>Objectives: </strong>To describe real-world patient characteristics, treatment patterns, HRU, and costs among patients with PTCL in Japan.</p><p><strong>Methods: </strong>This retrospective observational study used administrative claims data from the Medical Data Vision database in Japan. Adult patients diagnosed with PTCL between September 2012 and August 2022 were identified. Patients were categorized as receiving nonsystemic therapy or systemic therapy and stratified by lines of therapy. Patient demographics, comorbidities, treatment regimens, time on treatment, HRU, and healthcare costs, measured per patient per month, were analyzed descriptively.</p><p><strong>Results: </strong>Among 910 patients identified, 71.3% received systemic treatment and 28.7% received nonsystemic therapy, most receiving corticosteroids only. Mean age at diagnosis was 69.8 years; 70% of patients were at least 65 years old. Key comorbidities included peptic ulcer disease, congestive heart failure, and diabetes. PTCL-not otherwise specified and angioimmunoblastic T-cell lymphoma accounted for over 75% of subtypes. First-line therapy was mainly cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) or CHOP-like regimens; later lines were more heterogeneous and of shorter duration. Mogamulizumab and brentuximab vedotin were used in 4.16% and 10.48% of patients, respectively. Stem cell transplantation was infrequent (6.3%). Median per patient per month PTCL-related total healthcare cost was <math><mn>9712</mn> <mo>;</mo> <mi>m</mi> <mi>e</mi> <mi>d</mi> <mi>i</mi> <mi>a</mi> <mi>n</mi> <mi>d</mi> <mi>r</mi> <mi>u</mi> <mi>g</mi> <mo>(</mo></math> 7056.9) and hospitalization costs ($5177.7) were the main contributors.</p><p><strong>Discussion: </strong>These findings demonstrate substantial clinical and economic burden among PTCL patients in Japan. Treatment heterogeneity and short duration in later lines of therapy highlight ongoing therapeutic challenges.</p><p><strong>Conclusions: </strong>High economic burden of PTCL treatment underscores the need for more effective, tolerable, and less costly therapeutic strategies for treating PTCL patients in Japan.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"161191"},"PeriodicalIF":2.5,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13264034/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251338","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":"Cognitive Behavioral Therapy in Early Intervention for Psychosis: A Cost-Effectiveness Analysis.","authors":"Maximilian Duckworth, Sofia Venturini, Andres Roman-Urrestarazu","doi":"10.36469/001c.162047","DOIUrl":"10.36469/001c.162047","url":null,"abstract":"<p><strong>Background: </strong>Early intervention for psychosis (EIP) is a multidisciplinary service that treats individuals experiencing early psychosis. Treatment often consists of pharmacotherapy and cognitive behavioral therapy for psychosis (CBTp). While EIP demonstrates clinical efficacy and cost-effectiveness as a comprehensive package, the incremental contribution of its individual components remains unclear. With mental health services facing sustained funding pressures, understanding which EIP elements offer value for money is crucial for resource allocation decisions.</p><p><strong>Objectives: </strong>To evaluate whether CBTp used in conjunction with pharmacotherapy in EIP is cost-effective compared with pharmacotherapy alone.</p><p><strong>Methods: </strong>A cost-effectiveness analysis was conducted using a cohort-based Markov model from the societal perspective over a 3-year time horizon. Model parameters were derived from a targeted literature review. The primary outcome was the incremental cost-effectiveness ratio (ICER), expressed as the cost per quality-adjusted life-year (QALY) gained. One-way sensitivity analysis identified influential parameters and probabilistic sensitivity analysis (PSA) quantified uncertainty around the ICER.</p><p><strong>Results: </strong>CBTp as an adjunct to pharmacotherapy resulted in a gain of 0.48 QALYs in the base case compared with pharmacotherapy alone. The corresponding ICER was £32 979 per QALY gained. This currently sits above the conventional upper willingness-to-pay threshold of £30 000 per QALY. One-way sensitivity analysis identified cost parameters and utility parameters for symptom-free status as the most influential variables on the ICER. PSA showed that CBTp had approximately a 50% probability of being cost-effective at the £30 000 threshold.</p><p><strong>Conclusions: </strong>Parameter uncertainty, particularly regarding costs and health utilities, drives the marginal cost-effectiveness case for CBTp. The intervention's proximity to conventional thresholds suggests targeted improvements in service delivery or patient selection could enhance value. CBTp could potentially be a cost-effective adjunct to pharmacotherapy in EIP; however, there is uncertainty around the results of this modeling. These findings support the targeted implementation of CBTp within EIP. Further high-quality long-term studies are recommended to strengthen the evidence base.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"162047"},"PeriodicalIF":2.5,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13264041/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251931","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":"Real-World Treatment Landscape of Individuals with Rett Syndrome Treated and Untreated with Trofinetide in the United States.","authors":"Nazia Rashid, Vinod K Yakkala, Safiuddin S Syed, Krithika Rajagopalan","doi":"10.36469/001c.162589","DOIUrl":"10.36469/001c.162589","url":null,"abstract":"<p><strong>Background: </strong>Trofinetide (TROF) became the first approved pharmacologic treatment for Rett syndrome (RTT) in the United States in March 2023; however, real-world evidence comparing TROF-treated and untreated individuals is limited.</p><p><strong>Objective: </strong>This study aimed to compare the baseline demographic and clinical profiles of those treated with TROF vs untreated in routine clinical practice.</p><p><strong>Methods: </strong>This retrospective cohort study used linked IQVIA Anonymized Patient Level Database medical claims and a specialty pharmacy database. Individuals with ≥1 medical claim for RTT between 01/01/2021 and 09/30/2024 (study period) were identified. Treated individuals had ≥1 TROF prescription during 04/01/2023 to 09/30/2023 (identification period), with first prescription set as index; untreated individuals had no TROF prescription. A risk-set sampling approach was used to assign proxy index dates to untreated individuals to improve comparability and reduce immortal time bias. Individuals with cerebrovascular disease or brain trauma before RTT diagnosis and those without 12 months pre-and post-index enrollment were excluded. Baseline characteristics were assessed during the 12 months pre-index.</p><p><strong>Results: </strong>Of 8047 individuals with RTT identified, 2950 met eligibility criteria; 766 (26.0%) were treated with TROF and 2184 (74.0%) remained untreated. Treated individuals were younger at index (15.4 vs 23.5 years), and a greater proportion were pediatric (≤17 years; 66.6% vs 38.3%). Females predominated in both groups, while males represented a smaller proportion of the treated (4.6% vs 10.8%). Treated individuals more frequently had documented nonspecific developmental delay (31.3% vs 21.6%), autism spectrum disorder (19.1% vs 15.7%), and core RTT-related neurodevelopmental features such as loss of acquired communication skills (23.8% vs 12.9%) and loss of acquired motor skills (11.1% vs 4.9%). Child neurology was the most common prescriber specialty in both groups and was more frequent among treated individuals (50.3% vs 26.8%). Overall comorbidity burden was broadly similar between groups.</p><p><strong>Conclusion: </strong>In this real-world analysis, only one-quarter of eligible individuals with RTT initiated TROF during the early post-approval period. TROF uptake appeared concentrated in younger, specialist-managed individuals with more clearly documented RTT-related features, while three-quarters remained untreated. These findings highlight the need to better understand treatment pathways and barriers to initiation in males and adults in routine RTT care.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"162589"},"PeriodicalIF":2.5,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13264036/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251395","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":"Validation of the Medicare-Enhanced Laboratory and Demographics (MELD™) Dataset: A Comprehensive Psychometric, Epidemiologic, and Predictive-Utility Assessment of a 60-Million-Patient Real-World Evidence Resource.","authors":"Onur Baser","doi":"10.36469/001c.162896","DOIUrl":"10.36469/001c.162896","url":null,"abstract":"<p><strong>Background: </strong>Real-world evidence studies increasingly rely on integrated data resources that combine Medicare fee-for-service claims with electronic medical records (EMRs), laboratory results, and patient-reported outcomes. The Medicare-Enhanced Laboratory and Demographics (MELD™) dataset, developed by Columbia Data Analytics, spans 2020 through 2024 and contains 60 million unique patients, roughly 1 billion visits, and more than 241 000 clinicians. Its data-quality properties must be characterized before regulatory-grade use.</p><p><strong>Objective: </strong>To provide a preregistered, multidomain validation of MELD, quantifying internal consistency, concurrent validity against Centers for Medicare & Medicaid Services (CMS) benchmarks, construct validity, record-linkage quality, missing-data mechanisms, temporal stability, and predictive validity across 20 indication cohorts.</p><p><strong>Methods: </strong>An analytic cohort of 32 118 604 patients with at least 12 months of continuous Medicare enrollment was derived. Internal consistency used Cronbach's α and Kuder-Richardson KR-20. Concurrent validity compared MELD with CMS benchmarks using bias, mean absolute percentage error, Lin's concordance correlation coefficient, and Bland-Altman limits of agreement. Construct validity used exploratory and confirmatory factor analysis. Record linkage used Fellegi-Sunter probabilistic matching. Predictive validity was assessed for 12-month mortality and 30-day readmission using the area under the receiver operating characteristic curve (AUROC), Brier score, and calibration slope/intercept.</p><p><strong>Results: </strong>Internal consistency was high (Cronbach's α, 0.87; KR-20, 0.84). MELD replicated CMS prevalence with mean bias, -1.32 per 1000; Lin's concordance correlation coefficient, 0.993 (95% confidence interval, 0.986-0.997); and mean absolute percentage error, 3.1%. The 3-factor confirmatory model achieved excellent fit (comparative fit index, 0.962; root mean square error of approximation, 0.041; standardized root mean square residual, 0.036). Fellegi-Sunter linkage produced a 94.7% match rate with false-match probability <0.3%. Weighted κ between International Classification of Diseases, Tenth Revision claims and EMR problem lists ranged 0.66 to 0.91. AUROC was 0.821 for mortality and 0.783 for readmission, with calibration slopes 0.97 and 0.94.</p><p><strong>Conclusions: </strong>MELD demonstrated strong psychometric, epidemiologic, and predictive properties, supporting use in health economics and outcomes research for Medicare-aged and adjacent populations. Limitations include residual missingness of race/ethnicity (26%) and EMR fragmentation outside networked provider groups.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"226-235"},"PeriodicalIF":2.5,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13264042/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251804","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":"Patterns in Attribute Selection and Development Reporting in Patient Preference Studies Between 2007-2024: A Systematic Literature Review.","authors":"Siu Hing Lo, Rebekah Hall, Joy Wong, Gin Nie Chua","doi":"10.36469/001c.162119","DOIUrl":"10.36469/001c.162119","url":null,"abstract":"<p><strong>Background: </strong>Discrete choice experiments (DCE) are commonly used for understanding patient preferences. However, their validity relies upon appropriate attribute selection and development. The present review aimed to identify reporting patterns and gaps to inform future reporting of patient preference studies.</p><p><strong>Methods: </strong>Ovid (MEDLINE and EMBASE) was used to search terms for \"patients,\" \"discrete choice experiments,\" and \"attribute selection.\" Two independent reviewers screened studies against PICOS eligibility criteria, focusing on formative attribute development methods and reporting quality in patient preference DCEs up to 2024. Only full-text journal publications detailing attribute selection and development in patient preference DCEs were included. A coding form was developed and used to capture reporting on formative research used for attribute selection or development. Data synthesis employed a narrative approach following PRISMA guidelines.</p><p><strong>Results: </strong>Six categories of formative methods were identified across 28 studies: patient qualitative concept elicitation (n = 27; 96%), literature reviews (n = 21; 75%), expert consultation (n = 21; 75%), quantitative prioritization (n = 17, 61%), quantitative pilot DCE surveys (n = 13; 46%), and qualitative cognitive debriefing ( n =7; 25%). Most studies stated objectives (>85%), but methodological transparency decreased substantially for other elements, including rationale for choice of method, sample characteristics, sampling methods, data collection procedures data analysis, and results for formative research methods involving primary data collection. Most studies reported attribute lists following formative research (>75% across methods), but across formative methods fewer than half overall reported how formative findings informed attribute selection, level selection, or wording decisions. While 96% of studies included patients as research participants, only 7% reported engaging patients as research partners.</p><p><strong>Conclusions: </strong>The findings revealed inconsistent reporting of formative methods for attribute selection and development. Reporting of formative method details and results was mixed, with particularly low levels of reporting for how formative research results informed attribute selection, level selection, and wording. Furthermore, few studies engaged patients as research partners, suggesting another key area for development in the field of patient preference research. Improved reporting standards are needed to support methodological clarity and strengthen the validity of patient preference research. Patient engagement in the development of studies may further strengthen the patient relevance of patient preference studies.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"162119"},"PeriodicalIF":2.5,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13264046/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251031","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":"A Cost-per-Responder Analysis of Ritlecitinib vs Baricitinib in Severe Alopecia Areata.","authors":"Ashley S Cha-Silva, Kate H Zhang, Christopher N Graham, Samantha K Kurosky, Helen Tran, Ernest H Law, Eingun J Song","doi":"10.36469/001c.159914","DOIUrl":"10.36469/001c.159914","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Ritlecitinib, a JAK3/TEC family kinase inhibitor, and baricitinib, a JAK1/2 inhibitor, are approved for treating severe alopecia areata (AA).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;Develop a cost-per-responder analysis for ritlecitinib and baricitinib for treating severe AA to estimate the budget impact based on clinical efficacy, available dosages, and pricing structure.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;A decision tree evaluated cost implications of once-daily ritlecitinib 50 mg, or baricitinib 2 mg and 4 mg treatment over 1 year from a US perspective. Drug costs were estimated using wholesale acquisition costs. Distribution across treatment and dosing options were based on real-world evidence. We defined shorter-term response as Severity of Alopecia Tool (SALT) score relative change from baseline ≥30% (SALT Δ≥30%; SALT&lt;sub&gt;30&lt;/sub&gt;) at Weeks 18 and 24, and longer-term response as absolute SALT score ≤20 at Weeks 36 and 52. Patients initiating baricitinib 2 mg with no shorter-term response could uptitrate to 4 mg or discontinue therapy. Decision probabilities were derived from real-world evidence and clinical trial data.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;At Week 24, 52.10% of ritlecitinib initiators and 36.28% of baricitinib initiators achieved SALT Δ ≥30%; at Week 52, 40.26% and 30.63% of ritlecitinib and baricitinib initiators, respectively, achieved SALT ≤20. Baricitinib had a higher cost per responder than ritlecitinib at Weeks 24 ( &lt;math&gt;&lt;mn&gt;54&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;887&lt;/mn&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt;&lt;/math&gt; 45 577) and 52 ( &lt;math&gt;&lt;mn&gt;107&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;217&lt;/mn&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt;&lt;/math&gt; 94 834). Cost-equivalence was reached at Week 52 ( &lt;math&gt;&lt;mn&gt;38&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;180&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;w&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mn&gt;42.72&lt;/mn&gt;&lt;/math&gt; 1532) and higher at Week 52 (difference, &lt;math&gt;&lt;mn&gt;10&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;728&lt;/mn&gt; &lt;mo&gt;)&lt;/mo&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;o&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;b&lt;/mi&gt; &lt;mi&gt;a&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;i&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mn&gt;2&lt;/mn&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;g&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;e&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;mi&gt;l&lt;/mi&gt; &lt;mi&gt;o&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;a&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;W&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;k&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mn&gt;24&lt;/mn&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mn&gt;52&lt;/mn&gt; &lt;mo&gt;(&lt;/mo&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;f&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;n&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;/math&gt; 20 001 and -$60 073) than only baricitinib 4 mg use. The lower cost per responder for ritlecitinib 50 mg vs baricitinib 2 or 4 mg at Weeks 24 and 52, and greater early efficacy and fewer discontinuations is consistent with the results of a prior indirect treatment comparison that supported favorable outcomes with ritlecitinib 50 mg vs baricitinib 2 mg.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"136-140"},"PeriodicalIF":2.5,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13264044/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251972","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 Clinical and Economic Value of Incorporating Monocyte Distribution Width Testing in the Detection of Occult Sepsis.","authors":"Lopamudra Das, Melissa Naiman, Jeff Radcliff, Mary Anne Sullivan Lofstrom, Shawn Schwartz, Matia Saeedian, William Ngantung, Sana Mirza","doi":"10.36469/001c.162383","DOIUrl":"10.36469/001c.162383","url":null,"abstract":"<p><strong>Background: </strong>Sepsis is a leading cause of morbidity, mortality, and increased healthcare costs. Early recognition is critical, yet occult sepsis often evades standard screening, delaying care and worsening outcomes. Monocyte distribution width (MDW), a novel blood biomarker automatically reported with a routine complete blood cell count (CBC), has emerged as a promising tool for early detection of occult sepsis in adult patients in the emergency department (ED).</p><p><strong>Objectives: </strong>To evaluate the clinical and economic impact of incorporating MDW into standard ED sepsis screening vs standard of care (SoC).</p><p><strong>Methods: </strong>This study utilized a cost-consequence model to evaluate the clinical and economic impact of incorporating MDW into standard ED sepsis screening compared with standard screening (SoC) alone. Adults presenting to the ED and meeting sepsis criteria within 24 hours of CBC collection were stratified by sepsis suspicion level (no, low, high) and MDW values (normal, elevated). Outcomes, including time-to-antibiotics, hospital and intensive care unit (ICU) length of stay (LOS), ICU admission, mortality, and readmissions, were estimated using trial data and regression models from published literature. Costs were assessed from a US provider perspective in 2024 US dollars.</p><p><strong>Results: </strong>In the base case, SoC + MDW testing was associated with lower costs per patient than SoC ( <math><mn>36</mn> <mrow><mo> </mo></mrow> <mn>340</mn> <mi>v</mi> <mi>s</mi></math> 37 703; savings of $1363). Savings were driven by reduced ward and ICU LOS, particularly among patients with no or low suspicion of sepsis (eg, ICU LOS 0.30 vs 0.44 days; ward LOS 2.22 vs 2.75 days). Mortality was also reduced (0.03 vs 0.06 deaths per patient). While ED LOS increased in the MDW arm, inpatient savings offset these costs. Scenario analyses confirmed greater savings when unadjusted time-to-antibiotic data were applied. These findings are consistent with prior economic evaluations of early sepsis diagnostic tools and support the role of MDW in reducing downstream resource utilization, particularly among patients with occult presentations.</p><p><strong>Conclusions: </strong>MDW was found to be a low-cost tool that can strengthen sepsis screening by flagging cases earlier, especially in occult sepsis. Because it is reported with a routine CBC, MDW can be readily implemented and may improve outcomes while reducing costs.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"218-225"},"PeriodicalIF":2.5,"publicationDate":"2026-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13259569/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148251756","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":"Clinical Characteristics as Predictors of Healthcare Resource Utilization in Immunoglobulin A Nephropathy: A Retrospective Database Study.","authors":"Nancy Cannizzaro, Hema K Gandhi, Qiaoling Chen, Sasikiran Nunna, Mohit Mathur, John J Sim","doi":"10.36469/001c.158847","DOIUrl":"10.36469/001c.158847","url":null,"abstract":"<p><strong>Background: </strong>Progression rates in immunoglobulin A nephropathy (IgAN) are variable, making future healthcare needs in this patient population difficult to anticipate.</p><p><strong>Objectives: </strong>We sought to determine whether baseline patient characteristics at biopsy diagnosis are predictive of subsequent healthcare resource utilization (HCRU) in individuals affected by IgAN.</p><p><strong>Methods: </strong>This was a longitudinal, retrospective cohort study of members enrolled in Kaiser Permanente Southern California, a racially, ethnically, and socioeconomically diverse population. We extracted data on members diagnosed with primary IgAN based on kidney biopsies performed from 2000 to 2021. Statistical associations between indicators of disease severity at baseline and subsequent HCRU were evaluated. The baseline variables assessed were chronic kidney disease (CKD) stage, urine protein creatinine ratio (UPCR), and the individual burden of comorbidities expressed as Elixhauser Comorbidity Index. The HCRU outcomes were emergency department visits, inpatient visits, outpatient visits, radiology, laboratory, and medication dispensations within 2 years after biopsy, all expressed as utilization per patient per month (PPPM).</p><p><strong>Results: </strong>A total of 612 adults with primary IgAN were identified. Worse baseline CKD stage and UPCR exhibited statistically significant positive correlations with the PPPM for aggregate HCRU. Patients in CKD stage G4 had an overall PPPM of 2.94, which was significantly (P < .01) greater than for each milder CKD category (1.56-2.04). Patients with UPCR >2 g/g had an overall PPPM (2.38), which was significantly (P < .01) greater than each milder UPCR category (1.70-1.84). Aggregate PPPM showed a numerically increasing trend from the least (<0.5 g/g) to most severe UPCR category (>2 g/g). CKD stage, UPCR, and comorbidity score all correlated significantly with individual HCRU outcomes.</p><p><strong>Discussion: </strong>Easily assessed patient baseline variables including CKD stage, proteinuria, and comorbidities are predictive of HCRU in primary IgAN. Our findings are consistent with recent IgAN management guidelines designating proteinuria ≥0.5 g/g as indicative of elevated progression risk.</p><p><strong>Conclusions: </strong>Patient clinical characteristics can be used to estimate future HCRU in IgAN, facilitating cost-benefit analysis. The accurate estimation of anticipated HCRU is becoming increasingly important in IgAN as new, disease-specific therapies become available for this long-term, progressive condition.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"211-217"},"PeriodicalIF":2.5,"publicationDate":"2026-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13252823/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148225877","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":"Real-World Health Care Resource Utilization and Economic Burden Among Patients with Idiopathic Pulmonary Fibrosis in Commercially Insured and Medicare Advantage Populations in the United States.","authors":"Joseph Yang, Rebecca Fee, Andrea Steffens, Lisa Le, Bonnie H Bui, Bijan J Borah","doi":"10.36469/001c.161503","DOIUrl":"10.36469/001c.161503","url":null,"abstract":"<p><p><b>Background:</b> The economic burden associated with idiopathic pulmonary fibrosis (IPF) is not well understood, especially since the approval of antifibrotic medications to treat IPF in 2014. <b>Objective:</b> This study assessed healthcare resource utilization (HCRU) and costs for patients with and without IPF among insured patients in the United States. <b>Methods:</b> This retrospective study used claims data from the Optum Research Database from 01 January 2016 through 31 December 2023. The IPF cohort included patients with at least 2 claims for IPF within 365 days. A comparator cohort was exact matched to the IPF cohort in a 4:1 ratio on demographic characteristics. Follow-up all-cause and respiratory-related HCRU and costs were collected. Outcomes were presented as weighted per-patient per-month (wPPPM) to account for variable follow-up. Descriptive results were reported by cohort, overall, and stratified by insurance type. All-cause and respiratory-related wPPPM costs were modeled using generalized linear models and time to all-cause and respiratory-related inpatient visits were modeled using Cox proportional hazards regression models, adjusted for baseline comorbidities. <b>Results:</b> A total of 7582 patients were included in the IPF cohort; 10.4% were commercially insured and 89.6% insured by Medicare Advantage Prescription Drug (MAPD) plans. The comparator cohort included 30 328 demographically matched controls. Overall, mean all-cause wPPPM counts of HCRU during follow-up were significantly higher in the IPF cohort than the comparator cohort for all categories (all P < .001). Similarly, mean (SD) follow-up all-cause wPPPM total healthcare costs were higher for the IPF cohort than the comparator cohort ( <math><mn>4667</mn> <mo>[</mo></math> 8252] vs <math><mn>1196</mn> <mo>[</mo></math> 2114], P < .001). After adjustment, the cost ratio for all-cause total healthcare costs between the IPF and comparator cohorts was 3.4 (95% confidence interval [CI], 3.10-3.64), and patients in the IPF cohort were 2.1 times (95% CI, 1.97-2.15) more likely to be hospitalized for all causes than the comparator cohort. For both HCRU and costs, similar patterns were observed in both the commercial and MAPD populations. Conclusion: Patients with IPF incurred substantially higher HCRU and costs than the demographically matched comparator cohort. Study results suggest the need for treatment options to improve management of IPF and reduce costly HCRU.</p>","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"200-209"},"PeriodicalIF":2.5,"publicationDate":"2026-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13238604/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148199188","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 Impact of a Novel Pre-transplant Risk Assessment Tool for Early Acute Rejection in Kidney Transplant Patients.","authors":"Jacie T Cooper, John E Schneider","doi":"10.36469/001c.146282","DOIUrl":"10.36469/001c.146282","url":null,"abstract":"&lt;p&gt;&lt;p&gt;&lt;b&gt;Background:&lt;/b&gt; Accurately predicting outcomes and stratifying risks remains a challenge in kidney transplant patients. The Pre-Transplant Risk Assessment (PTRA) predicts risk of early acute rejection (EAR) in deceased donor transplant recipients, enabling clinicians to tailor immunosuppression strategies using individual immune response risk. &lt;b&gt;Objectives:&lt;/b&gt; We developed a cost-impact analysis of adding PTRA to the current standard of care to predict the risk of EAR compared with the current protocol-driven immunosuppression in standard-risk kidney transplant patients. &lt;b&gt;Methods:&lt;/b&gt; A decision tree model evaluated 90-day and 2-year outcomes post kidney transplantation. PTRA testing stratified patients as either high or low risk for EAR. Reduced maintenance immunosuppression protocols were modeled in low-risk PTRA patients compared with standard protocol, reducing adverse drug event readmissions, cytomegalovirus infections, graft loss, and deaths with a functioning graft. An increased maintenance immunosuppressive regimen was modelled for high-risk PTRA patients, reducing acute rejections and graft loss. &lt;b&gt;Results:&lt;/b&gt; The standard-risk population cohort consisted of 71.4% low-risk PTRA and 28.6% high-risk PTRA patients. The model generated per-patient cost savings of &lt;math&gt;&lt;mn&gt;358&lt;/mn&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mn&gt;90&lt;/mn&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;p&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;t&lt;/mi&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;s&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;l&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;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;mi&gt;a&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt;&lt;/math&gt; 13 234 per-patient over 2 years. If PTRA were used in all 14 468 patients falling into the standard risk immunosuppression protocol, &lt;math&gt;&lt;mn&gt;191&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;475&lt;/mn&gt; &lt;mrow&gt;&lt;mo&gt; &lt;/mo&gt;&lt;/mrow&gt; &lt;mn&gt;915&lt;/mn&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;d&lt;/mi&gt; &lt;mi&gt;b&lt;/mi&gt; &lt;mi&gt;e&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;e&lt;/mi&gt; &lt;mi&gt;d&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;mn&gt;2&lt;/mn&gt; &lt;mi&gt;y&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;a&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;h&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;v&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mo&gt;.&lt;/mo&gt; &lt;mi&gt;I&lt;/mi&gt; &lt;mi&gt;f&lt;/mi&gt; &lt;mi&gt;P&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;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;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;i&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;a&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;n&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;m&lt;/mi&gt; &lt;mi&gt;m&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;n&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;u&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;e&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mi&gt;s&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;p&lt;/mi&gt; &lt;mi&gt;r&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;t&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;c&lt;/mi&gt; &lt;mi&gt;o&lt;/mi&gt; &lt;mi&gt;l&lt;/mi&gt; &lt;mi&gt;s&lt;/mi&gt; &lt;mo&gt;,&lt;/mo&gt; &lt;mi&gt;p&lt;/mi&gt; &lt;m","PeriodicalId":16012,"journal":{"name":"Journal of Health Economics and Outcomes Research","volume":"13 1","pages":"146282"},"PeriodicalIF":2.5,"publicationDate":"2026-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13218370/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148138279","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}