Value in Health最新文献

{"title":"Psychometric Evaluation of the Proxy-Reported Pediatric Quality of Life Inventory Generic Core Scales Across the Childhood Lifespan in Australian Children and Adolescents With Specified Health Conditions.","authors":"Rakhee Raghunandan, Kirsten Howard, Sarah C Smith, Anagha Killedar, Erin Cvejic, Martin Howell, Stavros Petrou, Emily Lancsar, Germaine Wong, Jonathan C Craig, Alison Hayes","doi":"10.1016/j.jval.2025.03.018","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.018","url":null,"abstract":"<p><strong>Objectives: </strong>Current generic childhood health-related quality-of-life instruments lack comprehensive psychometric evidence across all ages. The Pediatric Quality-of-Life Inventory v4.0 Generic Core Scales (PedsQL GCS) covers ages 2 to 18 years old, but evidence on its psychometric properties is limited to restricted age groups. This study aimed to evaluate the proxy-reported PedsQL GCS across the entire childhood lifespan.</p><p><strong>Methods: </strong>The study used data from the Longitudinal Study of Australian Children for children aged 2 to 17 years with 1 of 6 health conditions: high weight status, eczema, attention deficit hyperactivity disorder, vision problems, hearing problems, and learning difficulty. Psychometric properties of the proxy-reported PedsQL GCS were assessed in early childhood, middle childhood, and adolescence against established criteria.</p><p><strong>Results: </strong>In analyses of 9317 children with 50 934 total observations, the PedsQL GCS demonstrated good acceptability across the childhood lifespan, except for high rates of missing data in 2 to 9 year olds (range = 12%-30%). Strong internal consistency was evident across health conditions and age (α range = 0.72-0.93; item-total correlations range = 0.28-0.80). Known group validity was strong with differentiation between children with/without the condition across all ages, except for eczema. Responsiveness was variable with inconsistencies mainly in early childhood.</p><p><strong>Conclusions: </strong>This study adds to the PedsQL psychometric evidence base, finding that the proxy-reported PedsQL GCS demonstrated robust reliability and known group validity, good acceptability, and mixed responsiveness in Australian children with health conditions across age. We propose the PedsQL GCS as a robust instrument to take forward for valuation to directly generate utility values for use in economic evaluations.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143988056","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":"Identifying the Influential Dynamic Inputs in Cost-Effectiveness Analyses.","authors":"Melanie D Whittington, Joshua T Cohen, Peter J Neumann, Tyler D Wagner, Jonathan D Campbell","doi":"10.1016/j.jval.2025.03.016","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.016","url":null,"abstract":"<p><strong>Objectives: </strong>Compare cost-effectiveness estimates calculated using a static approach to pricing over time with an approach that accounts for price changes over time (a dynamic approach) and identify which dynamic inputs have the greatest influence on cost-effectiveness.</p><p><strong>Methods: </strong>A panel of economic modeling experts identified cost-effectiveness analysis model inputs that change over time, possible values for each dynamic input, and approaches for incorporating dynamic inputs into an economic model. Informed by the advisory panel, static and dynamic cost-effectiveness estimates were calculated for 4 cases: (1) a chronically administered treatment for a chronic condition, (2) a chronically administered treatment for a catastrophic condition, (3) a 1-time treatment for a chronic condition, and (4) a 1-time treatment for a catastrophic condition.</p><p><strong>Results: </strong>The static cost-effectiveness estimate was less favorable than the dynamic estimate for a chronically administered drug by between 82% (case 1) and 62% (case 2), and for a 1-time drug by between 34% (case 3) and 27% (case 4). For chronically administered treatments, the post-loss-of-exclusivity price had the greatest impact on cost-effectiveness. For 1-time treatments, the age of individuals at baseline and the discount rate had a greater impact on cost-effectiveness than the drug's assumed price changes.</p><p><strong>Conclusions: </strong>Compared with a static approach, a dynamic approach can result in substantially different cost-effectiveness estimates, especially for treatments administered over time. To incorporate dynamics, research should prioritize estimating price changes after loss of exclusivity.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048425","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 Argument-Based Approach to Validity Applied to Clinical Outcome Assessments: Some History and Notable Features.","authors":"Kevin P Weinfurt","doi":"10.1016/j.jval.2025.03.012","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.012","url":null,"abstract":"<p><strong>Objectives: </strong>Developing and evaluating clinical outcome assessments (COAs) requires a framework for understanding validity. The validity framework reflected in the most recent draft guidance from the US Food and Drug Administration is the argument-based approach. In this approach, a researcher should state how they would like to interpret or use scores from some measure, identify key assumptions that need to be true for the proposed interpretation/use to be justified, and evaluate evidence for or against those key assumptions. If the collection of assumptions, known as the rationale, has convincing evidence, then a decision is made that the proposed interpretation or use of scores is valid. In this article, I briefly review how this approach to validity that has been developed within educational and psychological testing has recently made its way into COAs. I then discuss several notable features of the argument-based approach that have implications for how COAs are developed and evaluated.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144055394","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":"Potential Impact of EQ-5D-5L Mapping Function Recently Recommended by the National Institute for Health and Care Excellence on Utility Values and Decision Making.","authors":"Johan Maervoet, Rito Bergemann","doi":"10.1016/j.jval.2025.03.015","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.015","url":null,"abstract":"<p><strong>Objectives: </strong>In the absence of an accepted local EQ-5D-5L tariff, the National Institute for Health and Care Excellence (NICE) requires EQ-5D-5L responses to be mapped to the long-standing EQ-5D-3L UK value set. Their 2022 Manual recommends using the mapping function recently developed by the NICE Decision Support Unit (DSU), instead of the van Hout Crosswalk that was previously endorsed. Our aim was to compare utility values derived using these 2 mapping methods and assess the potential impact on decision making.</p><p><strong>Methods: </strong>For all 3125 unique EQ-5D-5L health states, utility values for both mapping functions were obtained and compared using numerical analysis and data visualization. Simulations in synthetic patient populations were conducted to evaluate the potential impact of the mapping functions on utility values derived from EQ-5D-5L clinical trial data.</p><p><strong>Results: </strong>For 1898 (61%) health states spread across the severity spectrum, the DSU mapping function generates a higher utility value than the van Hout Crosswalk. The mean difference was 0.062 (±0.139 SD), ranging from +0.619 to -0.198 for individual health states. The simulations suggest that mean utility values estimated with the DSU mapping function may lie higher than those obtained with the van Hout Crosswalk.</p><p><strong>Conclusions: </strong>Not only the choice of EQ-5D instrument, but also the mapping approach can have an impact on utility values. The DSU mapping function may shift UK utility values further upward, potentially affecting NICE decision making. The use of various combinations of instruments, mapping functions, and tariffs may soon make it challenging to bring together historical 3L evidence and a growing body of 5L-based evidence.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036483","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":"Cost-Effectiveness of Enfortumab Vedotin and Pembrolizumab for First-Line Metastatic Urothelial Cancer in the United States.","authors":"Tanvi V Chiddarwar, Hawre Jalal, Fernando Alarid-Escudero, David Garibay, Praveen Kumar, Krishna Roy Chowdhury, Bruce L Jacobs, Paul Mathew, John B Wong, Karen M Kuntz","doi":"10.1016/j.jval.2025.03.010","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.010","url":null,"abstract":"<p><strong>Objectives: </strong>The EV-302 trial demonstrated that the combination of enfortumab vedotin and pembrolizumab (EV+P) significantly improved progression-free and overall survival in patients with metastatic urothelial cancer. However, the economic value of this regimen remains uncertain. Our study aims to evaluate the cost-effectiveness of EV+P versus platinum-based chemotherapy as first-line treatment for metastatic urothelial cancer from the US payer perspective.</p><p><strong>Methods: </strong>We developed a cohort state-transition model to simulate the lifetime costs and effectiveness of a cohort of 69-year-olds with metastatic urothelial cancer, resembling those in the EV-302 trial. Costs and utilities were derived from published literature to calculate the quality-adjusted life years (QALYs) and the incremental cost-effectiveness ratio for EV+P relative to chemotherapy. We conducted deterministic and probabilistic sensitivity analyses to identify factors influencing the cost-effectiveness.</p><p><strong>Results: </strong>In the base case, the chemotherapy strategy costs $80 874, yielding 1.26 QALYs and 1.69 life years. The EV+P strategy costs $752 637, yielding 2.54 QALYs and 3.31 life years. This resulted in an incremental cost-effectiveness ratio of $525 239/QALY and $414 927/life year. To achieve cost-effectiveness at a $150 000/QALY threshold, the price of the combination therapy would need to be reduced by 76%. A probabilistic sensitivity analysis indicated that EV+P was cost-effective in 0% of the simulations.</p><p><strong>Conclusions: </strong>Although EV+P therapy is effective, it is not cost-effective at its current price as a first-line therapy in the United States at a cost-effectiveness threshold of $150 000/QALY. A substantial reduction in its drug cost is required to be cost-effective at commonly accepted willingness-to-pay thresholds.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144050523","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":"Utility Studies in Rare Diseases: A Systematic Literature Review.","authors":"Benjamin Ruban-Fell, Sari D Wright, Adib Abdullah, Amy Smith, Sheela Upadhyaya, Rebecca van Pelt, Annabel G M Griffiths","doi":"10.1016/j.jval.2025.02.018","DOIUrl":"10.1016/j.jval.2025.02.018","url":null,"abstract":"<p><strong>Objectives: </strong>There are challenges associated with generating health-state utility values for rare diseases, leading to a potential lack of standardization in the methods used. This systematic literature review characterized the approaches used to generate utility data in rare diseases.</p><p><strong>Methods: </strong>Searches of MEDLINE/Embase, health technology assessment and cost-effectiveness databases were conducted, supplemented by grey literature searches. Due to the large volume of evidence identified, articles were prioritized for full-text review by applying a 2020 date limit.</p><p><strong>Results: </strong>Ninety-seven articles (assessing 56 rare conditions) were included. Nineteen unique health-related quality-of-life tools were identified, 14 of which were generic. Indirect valuation methods were more common than direct (80 vs 43 instances). Among the indirect methods, the preference-based EQ-5D questionnaire was most reported (55 instances), followed by the non-preference-based short-form questionnaires (8 instances). Five disease-specific, non-preference-based questionnaires were reported. Mapping algorithms were used for preference-based and non-preference-based measures, typically mapped to EQ-5D, although challenges with mapping disease-specific tools to preference-based measures were noted. Vignettes were used in 29 articles; however, incomplete reporting on the development process limited the quality assessment.</p><p><strong>Conclusions: </strong>Generic, preference-based measures were commonly used to generate utility data in rare diseases among the identified studies, facilitating comparison but potentially limiting sensitivity of results. Development of appropriate and valid disease-specific measures and more transparent/consistent reporting of vignette development, would help ensure that all aspects of health-related quality-of-life impacted by rare diseases are suitably captured, to allow reliable demonstration of the value of treatments to support future reimbursement.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017389","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":"Costs and Health Outcomes in Economic Evaluations of Person-Centered Care: A Systematic Review.","authors":"Hadeel Elhassan, Salma Pardhan, Rasika Hewage, Benjamin P Harvey, Hanna Gyllensten","doi":"10.1016/j.jval.2025.03.013","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.013","url":null,"abstract":"<p><strong>Objectives: </strong>The aim was to explore the current evidence on the costs and cost-effectiveness of person-centered care (PCC). This includes exploring how results vary by populations, settings, and PCC operationalization.</p><p><strong>Methods: </strong>PubMed, Scopus, CINAHL, and gray literature were systematically searched using terms related to PCC and economic evaluations. Selection of studies, extraction of data, and assessment of potential bias using a published checklist were conducted by 2 independent reviewers. Articles were included if indicating PCC as a partnership between carer and patient and reporting costs. Synthesis without meta-analysis was performed using categories based on PCC being either (1) cost-effective, (2) cost saving with negative health outcomes, (3) more costly but effective, or (4) more costly and less effective than usual care, respectively. Classification also considered adherence to the Gothenburg model for PCC cornerstones.</p><p><strong>Results: </strong>Of 2802 articles identified, 44 articles (representing 40 studies) met the specified inclusion criteria. Studies were from the period 1986 to 2021, mostly from high-income countries, and targeting patients above 60 years with long-term condition or multiple comorbidities. Most studies found PCC to be dominating usual care (n = 26), cost-effective (n = 1), or cost saving (n = 3). Only 4 studies found PCC to be dominated by usual care. Limiting the analysis to the cornerstones listed in the Gothenburg model gave similar but limited results.</p><p><strong>Conclusions: </strong>Although most studies found PCC to be cost-effective or cost saving, these findings have limited applicability for system-wide decision making because of significant variations in study methodologies and patient populations.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048347","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":"Research Method, Conduct, and Reporting Considerations for Improving the Quality of Non-Hypothesis-Evaluating Treatment Effectiveness Analyses Using Real-World Data: An ISPOR Special Interest Group Report.","authors":"Eric Barrette, William H Crown, Melinda Hanisch, Rafael Alfonso-Cristancho, Sunny Sheth, Samuel C Z Gautier, Andy Surinach, Mark Cziraky, Jon D Morrow, Ami R Buikema","doi":"10.1016/j.jval.2025.02.017","DOIUrl":"https://doi.org/10.1016/j.jval.2025.02.017","url":null,"abstract":"<p><strong>Objectives: </strong>Numerous real-world evidence guidance documents have been published by many regulatory agencies, health technology assessment agencies, and academic groups. These guidances are largely focused on generating unbiased treatment effect estimates from real-world data (RWD) for use in approval and coverage decisions. One of the most prominently cited documents, is a joint ISPOR/ISPE Task Force guidance on the use of RWD for Hypothesis-Evaluating Treatment Effectiveness (HETE) Studies. However, there are a variety of uses of RWD that existing guidance does not explicitly address. Our goal was to assess existing guidance for its relevance to a range of non-HETE analyses.</p><p><strong>Methods: </strong>In this article, we delineate categories of non-HETE applications of RWD. This range of applications includes various types of non-HETE RWD analyses, as well as the use of RWD as inputs into other types of analyses, such as health economic modeling or simulation studies that are not typically considered RWD studies. We then map existing guidance documents to the categories of non-HETE studies to help identify existing resources for analysts interested in conducting these types of analyses.</p><p><strong>Results: </strong>We identified 13 published guidance documents for detailed review, including documents that outline best practices, provide checklists, or are structured templates. They were selected for their prominence, recency, specific reference to fit-for-purpose real-world evidence generation, or their relevance to specific analysis categories.</p><p><strong>Conclusions: </strong>We conclude that existing guidance documents, although they were largely developed for HETE studies, are highly useful for the broad range of non-HETE RWD analyses as well.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144016823","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":"First-Line Alectinib, Brigatinib, and Lorlatinib for Advanced Anaplastic Lymphoma Kinase-Positive Non-Small Cell Lung Cancer: A Cost-Effectiveness Analysis.","authors":"Rahul Mudumba, Jorge J Nieva, William V Padula","doi":"10.1016/j.jval.2025.03.014","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.014","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate the cost-effectiveness of alectinib, brigatinib, and lorlatinib as first-line therapies for anaplastic lymphoma kinase-positive advanced non-small cell lung cancer from a US healthcare sector perspective.</p><p><strong>Methods: </strong>We developed a 4-state partitioned survival model using progression-free survival, intracranial progression-free survival, and overall survival data from the ALEX, ALTA-1L, and CROWN clinical trials and published network meta-analyses. This model simulated patient transitions through progression-free, central-nervous-system-related progressed disease, non- central nervous system progressed disease, and death states over a 5-year horizon. Costs (2024 USD) included drug acquisition based on median of Department of Veteran Affairs and wholesale acquisition cost prices, healthcare utilization, and adverse events, all sourced from published literature. Quality-adjusted life years (QALYs) were derived using health utilities bootstrapped from these trials and adjusted for adverse events. We performed sensitivity and scenario analyses to evaluate uncertainty and explore various pricing and efficacy specifications.</p><p><strong>Results: </strong>Over a 5-year horizon, alectinib cost $1 105 814 for 2.85 QALYs gained, brigatinib cost $1 059 283 for 2.66 QALYs gained, and lorlatinib cost $1 163 519 for 2.88 QALYs gained. Incremental cost-effectiveness ratios for alectinib and lorlatinib versus brigatinib were $245 536/QALY and $481 386/QALY, respectively. Probabilistic sensitivity analysis indicated that at a willingness-to-pay threshold of $150 000 per QALY, brigatinib had a 54% chance of being the cost-effective option, with alectinib at 36% and lorlatinib at 10%.</p><p><strong>Conclusions: </strong>Although our model slightly favors brigatinib at a $150 000/QALY willingness-to-pay threshold, substantial uncertainty precludes definitive cost-effectiveness conclusions among the 3 first-line therapies.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048422","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 Factual Inaccuracy Noted in the Conclusions Specifically Regarding the Novelty of the Author's Work in the Middle East.","authors":"Samar Farid","doi":"10.1016/j.jval.2025.01.026","DOIUrl":"https://doi.org/10.1016/j.jval.2025.01.026","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144016815","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}