Value in Health最新文献

{"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 The validity framework reflected in the most recent draft guidance from the US Food and Drug Administration is the argument-based approach.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":"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":"Health Impact and Cost-Effectiveness of Testing and Treatment of Mycobacterium Tuberculosis Infection Among Asian and Hispanic Persons With Diagnosed Diabetes in the United States.","authors":"Nicole A Swartwood, Maryam B Haddad, Suzanne M Marks, Garrett R Beeler Asay, Charles R Horsburgh, Ted Cohen, Nicolas A Menzies","doi":"10.1016/j.jval.2025.03.009","DOIUrl":"https://doi.org/10.1016/j.jval.2025.03.009","url":null,"abstract":"<p><strong>Objectives: </strong>To evaluate the cost-effectiveness of testing and treatment for Mycobacterium tuberculosis (Mtb) infection among Asian and Hispanic persons with diagnosed diabetes in the United States.</p><p><strong>Methods: </strong>We estimated population size and Mtb infection prevalence for Asian and Hispanic persons aged ≥15 years with diagnosed, nongestational diabetes, by age and US-born status. We assumed a 1-time test for Mtb infection intervention, with positive-testing persons offered treatment. Using a deterministic, transmission-dynamic model of incident tuberculosis (TB) in the United States, we estimated costs, TB cases and deaths averted, and quality-adjusted life years gained under the intervention compared with no intervention. We estimated incremental cost-effectiveness ratios (ICERs), calculated as costs per quality-adjusted life years gained, from a TB health services perspective, including diagnosis and treatment for TB infection and disease. We also assessed health services and societal perspectives. We estimated 95% uncertainty intervals via probabilistic sensitivity analysis.</p><p><strong>Results: </strong>TB cases averted per 100 000 persons tested ranged from 7.5 (95% uncertainty interval: 6.9-8.1) among US-born Hispanic persons to 238.9 (225.2-254.3) among non-US-born Asian persons. TB deaths averted per 100 000 persons tested ranged from 1.3 (1.2-1.4) among US-born Hispanic persons to 53.7 (51.4-56.1) among non-US-born Asian persons. ICERs for US-born Asian and Hispanic populations were $856 671 ($533 506-$1 234 032) and $1 081 646 ($673 142-$1 551 264), respectively. ICERs for non-US-born Asian and Hispanic populations were lower: $66 664 ($41 456-$93 625) and $68 749 ($43 136-$97 044), respectively. ICERs were 2% to 19% higher under a societal perspective.</p><p><strong>Conclusions: </strong>Although the intervention produced health benefits for all populations assessed, health benefits were greater-and ICERs more favorable-for non-US-born Asian and Hispanic populations with diagnosed diabetes.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144042300","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":"Reviewer Acknowledgment","authors":"","doi":"10.1016/j.jval.2025.02.003","DOIUrl":"10.1016/j.jval.2025.02.003","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 4","pages":"Pages 489-494"},"PeriodicalIF":4.9,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143704377","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 Role of Mental Health in Healthcare Choices: A Discrete Choice Experiment Examining Preferences for Primary Care.","authors":"Jemimah Ride, Emily Lancsar, Ingrid Ozols","doi":"10.1016/j.jval.2025.03.001","DOIUrl":"10.1016/j.jval.2025.03.001","url":null,"abstract":"<p><strong>Objectives: </strong>This study examines the role of mental health in consumer healthcare choices, using a discrete choice experiment to analyze choices regarding routine primary care visits in Australia. It captures mental health through 3 variables: self-reported current mental health condition and clinically validated measures of depression and anxiety symptoms, the Patient Health Questionnaire-9 capturing depression, and the Generalized Anxiety Disorder 7-item scale capturing anxiety.</p><p><strong>Methods: </strong>Data were collected during November and December 2021 from a sample (N = 568) representative of the Australian population in age, gender, and location. Participants made hypothetical choices between in-person and telehealth alternatives or a no-visit alternative. Alternatives were described in terms of general practitioner familiarity, out-of-pocket cost, wait time, waiting area size, mask requirements, and modality of telehealth.</p><p><strong>Results: </strong>The results suggest that symptoms of depression and anxiety could affect healthcare choices with opposite direction of effect on uptake and distinct from the presence of a mental health condition.</p><p><strong>Conclusions: </strong>These findings support the need for more careful consideration of the role of mental health in the analysis of discrete choice experiments, particularly in healthcare, including a better understanding of the mechanisms and time-varying nature of any effect.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143693571","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}