Value in Health最新文献

{"title":"Performance of the EQ-5D-5L With Skin Irritation and Self-Confidence Bolt-On Items in Patients With Urticaria.","authors":"Junchao Feng, Yichi Yin, Nan Luo, Jie Guo, Dongmei Wang, Zhuxin Mao, Zehui He, Chuanjian Lu, Shunping Li, Jingjie Yu","doi":"10.1016/j.jval.2025.02.010","DOIUrl":"10.1016/j.jval.2025.02.010","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to validate the EQ-5D-5L with the addition of skin irritation and self-confidence bolt-on items in Chinese patients with urticaria.</p><p><strong>Methods: </strong>The skin irritation and self-confidence bolt-on items were translated into Simplified Chinese and cognitively debriefed. A cross-sectional survey was subsequently conducted among patients diagnosed with urticaria in Guangdong Province, China. The questionnaire included the EQ-5D-5L with the 2 bolt-on items, along with the Dermatology Life Quality Index (DLQI) and Skindex-16. The study examined the ceiling effects, informativity, and known-group validity of the 2 bolt-on items. Multiple regression analysis was conducted to determine the explanatory power of the EQ-5D-5L and bolt-on items in predicting the EQ VAS.</p><p><strong>Results: </strong>A total of 475 patients completed the questionnaire and were included in the analysis. The inclusion of the 2 bolt-on items reduced the ceiling effect from 54.95% to 4.21%. The skin irritation item showed a greater correlation with DLQI item 1 and the symptoms dimension of the Skindex-16 (correlation coefficients: 0.39 and 0.31, respectively). The self-confidence item exhibited a moderate correlation with DLQI item 2 (r = 0.42) and the functioning dimension of the Skindex-16 (r = 0.33). Known-group validity analysis confirmed that the skin irritation item demonstrated satisfactory discriminatory power. Multiple regression analyses indicated that the inclusion of skin irritation enhanced the explanatory power for EQ VAS more effectively than self-confidence.</p><p><strong>Conclusions: </strong>The inclusion of 2 bolt-ons in the EQ-5D-5L enhanced its informativity, convergent validity, discriminatory power, known group validity, and explanatory power among Chinese patients with urticaria.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143587269","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Price Effects of Biosimilars in the United States.","authors":"Mireia Jofre-Bonet, Alistair McGuire, Victoria Dayer, Joshua A Roth, Sean D Sullivan","doi":"10.1016/j.jval.2025.02.008","DOIUrl":"10.1016/j.jval.2025.02.008","url":null,"abstract":"<p><strong>Objectives: </strong>This study evaluates the impact of biosimilar competition on the price of reference products and biosimilars in the US pharmaceutical market. We focus on average sales price (ASP) dynamics since 2015 in Medicare Part B because Medicare is a critical payer stakeholder in biosimilar price dynamics.</p><p><strong>Methods: </strong>Using Centers for Medicare and Medicaid Services data over 34 quarters beginning in 2015, we analyzed the price effects of biosimilar competition in 8 biologic product markets. First, we evaluated the effect of biosimilar competition on ASP. Second, we evaluated the effect of market concentration on ASP using the Herfindahl-Hirschman index. We used instrumental variable methods to address the endogeneity between competition and ASP. Across analyses, our outcome variable is price ratio-the ratio of ASP measured over time relative to the fixed ASP at study baseline.</p><p><strong>Results: </strong>Our results showed a 10% to 13% decrease in the originator price ratio for entry of each additional biosimilar competitor, and a 1.8% decrease in the originator price ratio with a 10% decrease in market concentration at the lowest Herfindahl-Hirschman index observed in study period. These findings highlight the statistically significant effect of biosimilar competition and market concentration on prices in high-cost biologics drug markets.</p><p><strong>Conclusions: </strong>Our findings demonstrate that the US biosimilars market has become highly competitive, and as a result, prices have declined significantly over the period 2015 to 2023. These price dynamics have generated substantial savings for Medicare; however, there is a potential threat to market sustainability if these price trend dynamics persist in the coming years.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573884","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early Cost-Effectiveness Analysis of Using Whole-Genome Sequencing for Patients With Castration-Resistant Prostate Cancer.","authors":"Jinjing Fu, Nora Franzen, Eline Aas, J C Koen van der Mijn, Pim J van Leeuwen, Valesca P Retel","doi":"10.1016/j.jval.2025.02.004","DOIUrl":"10.1016/j.jval.2025.02.004","url":null,"abstract":"<p><strong>Objectives: </strong>This study aims to assess the potential cost-effectiveness of using whole-genome sequencing (WGS)-guided systemic therapy in metastatic castrate-resistant prostate cancer compared with the European Association of Urology guideline recommended diagnostics from a Dutch societal perspective.</p><p><strong>Methods: </strong>A decision analytic model combining a decision tree and partitioned survival models was developed to link diagnostic results with subsequent biomarker-guided treatments. Two diagnostic strategies, WGS and guideline-recommended practice-the genomic testing for breast cancer gene 1/2 (BRCA1/2) and deficient mismatch repair, were simulated to compare the health outcome and cost. Treatment effectiveness was estimated through survival analysis using published trial data. Sensitivity and scenario analyses were conducted to examine result robustness and to identify conditions under which WGS may be cost-effective.</p><p><strong>Results: </strong>WGS identified an additional 21% of patients eligible for personalized therapy (PD-1/PDL-1 inhibitors and olaparib), resulting in an incremental increase in cost (€14 260) and quality-adjusted life years (QALY = 0.05). These results yielded an incremental cost-effectiveness ratio of €289 625 per QALY gained. WGS would become cost-effective if the cost of biomarker-guided therapies decreases by 62% and when identifying a proportion of 23% more patients with actional targets.</p><p><strong>Conclusions: </strong>Our findings suggest that future treatments with improved efficacy and reduced cost could potentially make the WGS strategy cost-effective. Its unaccounted potential value to identify prognostic biomarkers, diagnostic alternatives, and patient heterogeneity should be addressed in future research and considered for optimal implementation. New reimbursement options are needed considering the high prices of biomarker-guided therapies that drive the incremental cost-effectiveness ratio.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Systematic Review and Meta-Analysis of Health-State Utility Values for Infectious Diseases With Pandemic Potential and Associated Vaccine Adverse Reactions.","authors":"Taito Kitano, Daniel A Salmon, Matthew Z Dudley, Ian J Saldanha, David A Thompson, Lilly Engineer","doi":"10.1016/j.jval.2025.02.007","DOIUrl":"10.1016/j.jval.2025.02.007","url":null,"abstract":"<p><strong>Objectives: </strong>The COVID-19 pandemic and its vaccine deployment have illustrated the importance of estimating the disease burden of the pandemic and vaccine adverse reactions in a comparable fashion. The objective was to evaluate health-state utility value (HSUV) scores for infectious diseases with pandemic potential and associated vaccine adverse reactions.</p><p><strong>Methods: </strong>We included studies from which we could extract a single HSUV associated with priority infectious diseases with pandemic potential or associated vaccine adverse reactions. We assessed risk of bias using the criteria recommended by National Institute for Health and Care Excellence. We conducted random-effects meta-analyses.</p><p><strong>Results: </strong>We included 39 studies, and data synthesis was conducted for COVID-19, influenza, and dengue for infectious diseases and myocarditis and narcolepsy for vaccine adverse reactions. Response rates varied (2%-98%), whereas follow-up rates ranged from 68% to 100%. Twenty-four studies did not report how they handled missing data. Compared with control groups, COVID-19 (acute phase) and influenza (acute phase) had reduced EQ-5D scores: -0.11 (95% CI -0.14, -0.07) in COVID-19 and -0.50 (95% CI -0.60, -0.41) in influenza. For associated vaccine adverse reactions, the mean EQ-5D score for myocarditis and PedsQL score for narcolepsy were 0.88 (95% CI 0.76, 1.00) and 64.0 (95% CI 59.4, 68.7), respectively. No apparent asymmetry was observed in funnel plots.</p><p><strong>Conclusions: </strong>This study provided HSUV scores for some infectious diseases with pandemic potential and associated vaccine adverse reactions. These results can be used as HSUV indicators for future health technology and cost-effectiveness assessments.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic Evaluations of Treatment of Depressive Disorders in Adolescents: A Scoping Review.","authors":"Claire de Oliveira, Joyce Mason, Bahar Amani, Terri Rodak, Peter Szatmari, Jo Henderson, Darren B Courtney","doi":"10.1016/j.jval.2025.02.005","DOIUrl":"10.1016/j.jval.2025.02.005","url":null,"abstract":"<p><strong>Objectives: </strong>Depressive disorders in adolescents are common and impairing. The objective of this review was to ascertain the existing literature on economic evaluations of treatments for adolescent depression.</p><p><strong>Methods: </strong>We searched MEDLINE, Embase, PsyclNFO, EconLit, and the International Health Technology Assessment Database from inception to May 2024 and the National Health Services Economic Evaluation Database from inception to December 2014. We included publications containing economic evaluations of clinical trials or model-based studies, which tested the treatment of depression in adolescents, regardless of jurisdiction. We extracted data, assessed the quality of reporting and methodology of all studies using the Consolidated Health Economic Evaluation Reporting Standards and Quality of Health Economic Studies checklists, respectively, and used a narrative approach to synthesize findings by treatment.</p><p><strong>Results: </strong>Among 1381 records, we found 10 eligible studies. Two studies reported that cognitive behavioral therapy (CBT) was cost-effective when compared with treatment as usual. Findings were mixed when combined CBT and selective serotonin reuptake inhibitors were compared with selective serotonin reuptake inhibitors alone because results were dependent on the applied willingness-to-pay thresholds (ie, the maximum price a decision maker is willing to pay for a given treatment). Overall, the quality of reporting and methodology was fairly high, although there were areas in which the studies could be improved upon.</p><p><strong>Conclusions: </strong>Eight studies indicate that CBT, alone or in combination with other treatments, can be cost-effective for treating adolescent depression. Future economic evaluations should consider characterizing distributional effects, describing how uncertainty about analytic judgments, inputs, or projections affect study findings, undertaking equity subanalyses, and engaging with patients and others affected by the study.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573868","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Comprehensive View of the Methods Used to Measure the Societal Impact of Healthcare Interventions: A Systematic Review.","authors":"Ahmed H Seddik, Jeroen Paulissen, Ramesh Marapin, Sebastiaan Fuhler, Sukhvinder Johal, Mats Rosenlund, Kyle Dunton, Maarten Postma, Roel Freriks","doi":"10.1016/j.jval.2025.02.009","DOIUrl":"10.1016/j.jval.2025.02.009","url":null,"abstract":"<p><strong>Objectives: </strong>This study summarizes the concepts that have been used to date to demonstrate societal impact of healthcare interventions and establish an analytical framework to comprehensively organize and describe the different elements of societal impact.</p><p><strong>Methods: </strong>We conducted a systematic review following Cochrane and Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. We searched MEDLINE and Embase, gray literature from ISPOR and Value Balancing Alliance websites, and the reference lists of the identified systematic reviews. Eligible studies assessed the societal impact and described a specific approach. For each included study, we extracted relevant characteristics, such as disease area, methodology, and societal impact metrics.</p><p><strong>Results: </strong>From a pool of 12 525 studies, 159 were included. Studies broadly examined the societal impact of an intervention at the patients, caregivers, healthcare organizations, and the societal level. Within those categories, we identified a total of 42 different concepts of societal value. We presented a frequency count of those concepts in a hierarchical framework and analyzed the studies qualitatively. We also assessed the literature against the ISPOR value flower.</p><p><strong>Conclusions: </strong>Our findings reveal recurrent themes and highlight the demand for methodological harmonization. While addressing the limitations of the studied literature from a decision-making perspective, we propose a consensus-driven path forward. Through our research, we emphasize the importance of refining the existing methods, the need for developing a standardized reporting format for future studies, and the importance of developing concise definitions for subjective concepts for societal impact of healthcare interventions.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573755","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Health Economics of Alzheimer’s Disease and Related Dementias","authors":"Thomas Rapp PhD ,&nbsp;Pei-Jung Lin PhD","doi":"10.1016/j.jval.2025.02.002","DOIUrl":"10.1016/j.jval.2025.02.002","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 4","pages":"Pages 495-496"},"PeriodicalIF":4.9,"publicationDate":"2025-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143433681","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An Acquired Taste: Latent Class Analysis to Compare Adolescent and Adult Preferences for EQ-5D-Y-3L Health States.","authors":"Jonathan L Nazari, Juan M Ramos-Goñi, Ning Yan Gu, A Simon Pickard","doi":"10.1016/j.jval.2025.01.020","DOIUrl":"10.1016/j.jval.2025.01.020","url":null,"abstract":"<p><strong>Objectives: </strong>US stakeholders advised including adolescents in the valuation study for the EQ-5D-Y-3L, a step toward greater acknowledgment of children in informing societal values. This study aimed to assess the relative contribution of adolescent and adult preferences to a model when combined.</p><p><strong>Methods: </strong>Discrete choice experiment data were collected from an online sample of 1529 adults and 714 adolescents (ages 11-17). Each respondent completed 15 discrete choice experiment tasks, which were analyzed using latent class models representing varying number of preference classes. Within the best-fitting model, the contribution of each class was determined by the scale-adjusted class share (SACS), combining the class's proportion of respondents (class share) and the magnitude of coefficients (within-class scale). We estimated the contribution of adolescent and adult respondents to SACS for each class, with lower SACS representing less contribution to the combined model.</p><p><strong>Results: </strong>The best-fitting model described 6 classes. Adults had higher contribution to all except 1 class, accounting for 78.7% of the total contribution. After adjusting for the unequal sample size of adolescent and adult respondents, adults contributed approximately 65.0%, and adolescents contributed 35.0% of the weights toward a combined model.</p><p><strong>Conclusions: </strong>Adolescents showed diminished, disproportionate representation in a combined model, due in part to more indifferent, less informative preferences for EQ-5D-Y-3L health states compared with adults. Latent class analysis showcases one approach to estimate and weight contributions from intentionally sampled subgroups in a combined model.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426296","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Qini Curves for Potential Impact Assessment of Risk Predictive Models Informing Intervention Policies.","authors":"Pierpaolo Palumbo","doi":"10.1016/j.jval.2025.01.024","DOIUrl":"10.1016/j.jval.2025.01.024","url":null,"abstract":"<p><strong>Objectives: </strong>Predictive models in medicine help make decisions about which individual to treat with a given therapeutic or preventive intervention. Before being tested in large field studies and recommended for clinical adoption, it is important to evaluate not only their statistical accuracy but also the impact they may have when used to inform health intervention policies. We aim to provide simple methods for the potential impact assessment of health intervention policies based on predictive models.</p><p><strong>Methods: </strong>We propose an analytic framework based on Qini curves wherein prediction-based policies are analyzed on 2 impact endpoints: (1) the fraction of the population that would be selected for the intervention (coverage) and (2) the effect on the clinical outcomes of interest (disutility). The drivers of values are the disease prevalence, the predictive performance of the model, and the effectiveness of the intervention.</p><p><strong>Results: </strong>We present simple formulas for calculating coverage and disutility from either observational or randomized controlled data. We illustrate possible value measures arising from geometrical properties on the Qini plane: delta coverage and disutility, number needed to treat, and integrated difference between Qini curves. We show the applicability of the Qini analysis by providing examples about the prevention of falls in older adults and prevention of secondary cardiovascular events with pioglitazone.</p><p><strong>Conclusions: </strong>Coverage and disutility capture key value components of prediction-based policies. The method can be used for comparing models or tuning risk thresholds for managing trade-offs between conflicting objectives (eg, clinical benefits, side effects, and healthcare resources).</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426307","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Economic Evaluation of Internet-Based Psychological Interventions: A Scoping Review of Methodological Choices.","authors":"Amira J Hariz, Karine Chevreul, Laure Daval, Mathilde Husson, Corinne Alberti, Morgane Michel","doi":"10.1016/j.jval.2025.01.023","DOIUrl":"10.1016/j.jval.2025.01.023","url":null,"abstract":"<p><strong>Objectives: </strong>Internet-based psychological interventions hold promise for cost-effectiveness; yet, their evaluation lacks standardization, potentially leading to methodological discrepancies and inconclusive results. This study aims to conduct a scoping review of economic methods used when evaluating these interventions.</p><p><strong>Methods: </strong>Articles published between January 2015 and December 2020 were retrieved from PubMed, Embase, Cochrane Library, CINAHL, ECONLIT, and PsychINFO. Two reviewers independently screened titles, abstracts, and full texts of relevant publications and extracted prespecified data. Outcomes related to intervention characteristics, comparators, perspective, time horizon, costs, benefits, economic endpoints, and uncertainty analysis methods were retrieved and synthetized narratively.</p><p><strong>Results: </strong>We identified 703 references and included 85. Seventy-four of them included a cost-utility analysis and 58 a cost-effectiveness analysis, with 47 carrying out both. The comparator was treatment as usual in 52 studies (61.2%) but varied widely across studies. A societal perspective was adopted in 60 studies, supplemented by a healthcare perspective in half. Time horizon was 1 year or less in 68 articles (80.0%). Intervention costs (71/85 studies) predominantly covered delivery costs (45/71 studies), whereas development and promotional costs were infrequently considered (respectively, 14 and 5/71 studies). Interventions' reach, opportunity costs, user engagement, and equity issues were rarely addressed. Key factors influencing cost-effectiveness included perspective, time horizon, costs included, and methods for handling missing data.</p><p><strong>Conclusions: </strong>Assessment of cost-effectiveness in internet-based psychological interventions shows variability, potentially affecting efficiency evidence. Conventional methods are often favored overlooking digital tools' specificities. Tailored guidelines for such evaluations could be helpful for standardized and reliable evidence.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426303","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}