Value in Health最新文献

{"title":"Use of Real-World Data to Support Economic Evaluations of Tumor-Agnostic Therapies - Challenges Remain.","authors":"Kate Young, Grant McCarthy, Raquel Aguiar-Ibáñez, Dave Gelb, Wenjun Zhong, Roman Groisberg, David Fogelman, Mayur Amonkar","doi":"10.1016/j.jval.2024.11.016","DOIUrl":"https://doi.org/10.1016/j.jval.2024.11.016","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144042301","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 Taxonomy of Generative AI in HEOR: Concepts, Emerging Applications, and Advanced Tools - An ISPOR Working Group Report.","authors":"Rachael L Fleurence, Xiaoyan Wang, Jiang Bian, Mitchell K Higashi, Turgay Ayer, Hua Xu, Dalia Dawoud, Jagpreet Chhatwal","doi":"10.1016/j.jval.2025.04.2167","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2167","url":null,"abstract":"<p><strong>Objective: </strong>This article presents a taxonomy of generative artificial intelligence (AI) for health economics and outcomes research (HEOR), explores emerging applications, outlines methods to improve the accuracy and reliability of AI-generated outputs and describes current limitations.</p><p><strong>Methods: </strong>Foundational generative AI concepts are defined, and current HEOR applications are highlighted, including for systematic literature reviews, health economic modeling, real-world evidence generation, and dossier development. Techniques such as prompt engineering (e.g., zero-shot, few-shot, chain-of-thought, persona pattern prompting), retrieval-augmented generation, model fine-tuning, and domain-specific models, and use of agents are introduced to enhance AI performance. Limitations associated with the use of generative AI foundation models are described.</p><p><strong>Results: </strong>Generative AI demonstrates significant potential in HEOR, offering enhanced efficiency, productivity, and innovative solutions to complex challenges. While foundation models show promise in automating complex tasks, challenges persist in scientific accuracy and reproducibility, bias and fairness and operational deployment. Strategies to address these issues and improve AI accuracy are discussed.</p><p><strong>Conclusion: </strong>Generative AI has the potential to transform HEOR by improving efficiency and accuracy across diverse applications. However, realizing this potential requires building HEOR expertise and addressing the limitations of current AI technologies. Ongoing research and innovation will be key to shaping AI's future role in our field.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144052015","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 budget impact analysis of AI to support the review of radiographic examinations for suspected fractures in NHS emergency departments (ED).","authors":"Lucy Gregory, Trishal Boodhna, Mathew Storey, Susan Shelmerdine, Alex Novak, David Lowe, Hugh Harvey","doi":"10.1016/j.jval.2025.04.2165","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2165","url":null,"abstract":"<p><strong>Objective: </strong>To develop an early budget impact analysis of and inform future research on the national adoption of a commercially available AI application to support clinicians reviewing radiographs for suspected fractures across NHS emergency departments in England.</p><p><strong>Methods: </strong>A decision tree framework was coded to assess a change in outcomes for suspected fractures in adults when AI fracture detection was integrated into clinical workflow over a 1-year time horizon. Standard of care was the comparator scenario and the ground truth reference cases were characterised by radiology report findings. The effect of AI on assisting ED clinicians when detecting fractures was sourced from US literature. Data on resource use conditioned on the correct identification of a fracture in the ED was extracted from a London NHS trust. Sensitivity analysis was conducted to account for the influence of parameter uncertainty on results.</p><p><strong>Results: </strong>In one year, an estimated 658,564 radiographs were performed in emergency departments across England for suspected wrist, ankle or hip fractures. The number of patients returning to the ED with a missed fracture was reduced by 21,674 cases and a reduction of 20, 916 unnecessary referrals to fracture clinics. The cost of current practice was estimated at £66,646,542 and £63,012,150 with the integration of AI. Overall, generating a return on investment of £3,634,392 to the NHS.</p><p><strong>Conclusion: </strong>The adoption of AI in EDs across England has the potential to generate cost savings. However, additional evidence on radiograph review accuracy and subsequent resource use is required to further demonstrate this.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144034533","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 Value of Hope in Cancer Care: Risk Preference and Heterogeneity in Cancer Patients and the General Public.","authors":"Jihyung Hong, Eun-Young Bae, Shuye Yu","doi":"10.1016/j.jval.2025.04.2166","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2166","url":null,"abstract":"<p><strong>Objectives: </strong>To quantify the value of hope in cancer care and examine age as a potential modifier, comparing patients and the general public.</p><p><strong>Methods: </strong>A discrete choice experiment was conducted in 2024 with 426 cancer patients diagnosed within the past five years and 2,048 general public members without prior cancer experience in South Korea. Choice options comprised treatment effects, health status, and out-of-pocket costs. Treatment effects included a fixed 2-year survival option and four risk options offering 10% chances of extended survival (5 or 10 years) paired with 10% chances of minimum survival (3 months or 1 year), with varying survival durations for the remaining 80% to yield the same expected survival. Each participant completed 10 choice tasks, repeated for hypothetical ages of 5 and 70. Mixed logit and latent class analyses (LCA) were performed.</p><p><strong>Results: </strong>Both groups, particularly cancer patients, positively valued treatments with 10% chances of 10-year and 1-year survival over certainty. The monetary value of hope for this option was $12,445 for patients, about double that for the general public ($5,985). These values increased to $51,103 and $35,609, respectively, for age 5, but were much lower for age 70. Conversely, the certainty option was preferred over treatments with 10% chances of 5-year and 3-month survival. LCA revealed substantial preference heterogeneity.</p><p><strong>Conclusions: </strong>The value of hope represents a meaningful aspect of treatment preferences, especially in pediatric contexts. However, substantial preference heterogeneity poses challenges to effectively incorporating 'hope' into health technology assessments, particularly in publicly funded healthcare systems.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036196","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":"Exemption system for economic evaluation data submission for pharmaceuticals in South Korea: Achievements and Challenges.","authors":"So-Jung Hwang, Namhee Kim, Eunsol Seo, Yeji Kim, Tae-Jin Lee","doi":"10.1016/j.jval.2025.04.2163","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2163","url":null,"abstract":"<p><strong>Background: </strong>Since the implementation of the positive listing system in South Korea in 2007, new and improved drugs have been reimbursed based on economic evaluation. However, concerns have arisen about reduced accessibility to some drugs. To address this, an exemption system for economic evaluation data submission (EEE) was introduced in 2015 to improve access to drugs with uncertainties in evidence generation.</p><p><strong>Objectives: </strong>This study aims to assess the institutional and budgetary impact of EEE.</p><p><strong>Methods: </strong>A descriptive analysis was conducted of the status of EEE-listed drugs. The institutional impact was assessed by examining changes in listing rates, time to listing and cost-effectiveness assessment schemes. The budgetary impact was analyzed by comparing EEE-listed drugs with those listed under other schemes.</p><p><strong>Results: </strong>The EEE system has positively influenced accessibility, as seen in increased listing rates. By September 2023, 33 ingredients had been listed under the EEE, including 23 for cancer and 6 for rare diseases, with 20 available controlled clinical data. The implementation of the EEE has significantly weakened the role of economic evaluations, especially for cancer and rare disease treatments. The cost per person for EEE-listed drugs were 2.13 to 8.52 times higher, and the number of patients treated with EEE follow-on drugs was substantially larger than those under other schemes.</p><p><strong>Discussion: </strong>Given the negative impact of the EEE on deriving appropriate drug values through economic evaluations, there is an imminent need for improved cost management. This can be achieved by implementing a reliable cost-effectiveness evaluation framework that addresses various uncertainties.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144000043","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":"Understanding the transition between age-specific measures of HRQoL: Evidence on the relationship and performance between EQ-5D-Y-5L and adult EQ-5D-5L.","authors":"Nicole Reyes, Tianxin Pan, Renee Jones, Kim Dalziel, Nancy Devlin","doi":"10.1016/j.jval.2025.04.2161","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2161","url":null,"abstract":"<p><strong>Objectives: </strong>The EQ-5D-5L is widely used to measure adults' health-related quality of life (HRQoL). The EQ-5D-Y-5L is a corresponding measure adapted for children/adolescents, in principle allowing HRQoL to be measured consistently from childhood to adulthood. However, little is known about how their measurement properties compare. This study investigated the relationship between EQ-5D-Y-5L and EQ-5D-5L in adolescents and compared their psychometric performance.</p><p><strong>Methods: </strong>The P-MIC Study includes a sample of 591 adolescents (aged 12-18) who completed both EQ-5D-5L and EQ-5D-Y-5L. Responses were compared descriptively and HRQoL summarised using the level sum score. Acceptability, feasibility, ceiling effects, convergence, test-retest reliability, and known-group validity were assessed overall and in sub-groups defined by special health care needs (SHCN), mental health concerns (MHC) and age (12-13, 14-16 and 17-18 years).</p><p><strong>Results: </strong>Ceiling effects were lower for EQ-5D-Y-5L than EQ-5D-5L. The EQ-5D-Y-5L better differentiated between adolescents with and without SHCN and MHC than EQ-5D-5L, while EQ-5D-5L showed better test-retest reliability in adolescents with SHCN and MHC. We found strong correlations between dimensions anticipated to be correlated. EQ-5D-Y-5L identified a higher incidence of self-reported HRQoL problems than EQ-5D-5L both overall and particularly in mental health.</p><p><strong>Conclusions: </strong>While both instruments are valid for measuring HRQoL in adolescents 12-18 years old, EQ-5D-Y-5L had some psychometric advantages. The instruments are closely related, but differences in their descriptive systems produce differences in self-reported HRQoL. Results highlight potential discontinuities in HRQoL measured using age-specific instruments which may be important for their use in economic models that involves transitions between age groups.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048555","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":"Realizing the Economic Potential of Meditation and Mindfulness Practices in Mental Health Care.","authors":"Jagpreet Chhatwal, Rachael L Fleurence","doi":"10.1016/j.jval.2025.04.2164","DOIUrl":"10.1016/j.jval.2025.04.2164","url":null,"abstract":"<p><p>This commentary explores the economic potential of integrating meditation and mindfulness practices into mental health care, highlighting their cost-effectiveness in alleviating mental illness. Despite growing evidence of their benefits in reducing anxiety, depression, and stress, these practices remain underutilized. Economic evaluations are limited because of insufficient funding, heterogeneous methodologies, and a lack of standardized interventions and outcome measures, compounded by a scarcity of robust randomized controlled trials. The article purports that increased investment in rigorous, long-term studies, the creation of standardized protocols, and the incorporation of digital tools to enhance adherence will help increase their uptake in practice. Addressing these barriers could elevate meditation and mindfulness from supplementary treatments to mainstream mental health interventions, thereby reducing healthcare costs and improving accessibility.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144034535","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":"Distributional Cost-Effectiveness Analysis in Genomic Medicine: Considerations for Addressing Health Equity.","authors":"Hadley Stevens Smith, James Buchanan, Ilias Goranitis, Maarten J IJzerman, Tara A Lavelle, Deborah A Marshall, Dean A Regier, Wendy J Ungar, Deirdre Weymann, Sarah Wordsworth, Kathryn A Phillips, Jeroen P Jansen","doi":"10.1016/j.jval.2025.04.2162","DOIUrl":"10.1016/j.jval.2025.04.2162","url":null,"abstract":"<p><strong>Objectives: </strong>Distributional cost-effectiveness analysis (DCEA) supports equitable resource allocation by quantifying equity-efficiency trade-offs. DCEA may be particularly useful to understand equity impacts in the context of genomic medicine, a rapidly growing clinical area that has prompted concerns about its potential to exacerbate health inequities by differentially benefitting some population groups over others because of disparities in research inclusion and access to specialty care. This article critically examines the application of DCEA in the context of genomic medicine.</p><p><strong>Methods: </strong>We articulate steps for distributional impact assessment in the context of genomic medicine by adapting an existing conceptual framework for understanding the causal pathway between a healthcare intervention and the distribution of costs and effects among social groups, the inequality staircase. We discuss related data equity considerations and evidence requirements specific to genomic medicine interventions.</p><p><strong>Results: </strong>The need for and receipt of a genomic medicine intervention, as well as an intervention's short-term and long-term effects, may vary across equity-relevant subgroups. Research to enhance the relevance of DCEA in genomic medicine should avoid conflation of biological and social factors, empower populations that are underrepresented in genomics research, accurately assess variation in outcomes across equity-relevant subgroups, and develop methods for incorporation of nonhealth outcomes within a DCEA framework.</p><p><strong>Conclusions: </strong>Best practice-aligned applications of DCEA may facilitate transparent discussions of health equity in coverage and implementation decisions. This article provides guidance to researchers on the use of DCEA in genomic medicine and other clinical areas with similarly complex considerations around equity.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144001398","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 Comparative Study of Health and Well-Being Measures in Ireland Using EQ Health and Well-Being (EQ-HWB) and its Short Version, EQ-5D-5L, and ICEpop Capability Measure for Adults (ICECAP-A).","authors":"Irina Kinchin, Lidia Engel, Fanni Rencz","doi":"10.1016/j.jval.2025.04.2160","DOIUrl":"10.1016/j.jval.2025.04.2160","url":null,"abstract":"<p><strong>Objectives: </strong>The EQ Health and Wellbeing (EQ-HWB) is a new experimental measure of health and well-being, having been validated in an increasing number of countries and languages. This study aims to examine the psychometric properties of the EQ-HWB and its short version (EQ-HWB-S) in Ireland and compare them with the EQ-5D-5L and ICEpop Capability Measure for Adults (ICECAP-A).</p><p><strong>Methods: </strong>A secondary analysis of cross-sectional data from a 2023 Irish general population survey (n = 1220). The survey included the EQ-HWB, EQ-5D-5L, ICECAP-A, and socioeconomic and health-related questions. EQ-HWB-S responses were derived from the EQ-HWB. The UK or Irish value sets were used for each measure to compute index values. Ceiling effects, convergent, divergent validity, and known-group validity were assessed and exploratory factor analysis was conducted.</p><p><strong>Results: </strong>Mean index values were 0.755, 0.760 to 0.830, and 0.807 for the EQ-HWB-S, EQ-5D-5L, and ICECAP-A, respectively. Ceiling effects were 3%, 7%, 23%, and 15% for EQ-HWB, EQ-HWB-S, EQ-5D-5L, and ICECAP-A. EQ-HWB-S index values correlated strongly with EQ-5D-5L (r = 0.740-0.759) and ICECAP-A (r = 0.604) index values. All measures demonstrated good known-group validity for health-related variables, with EQ-5D-5L performing best overall. Factor analysis identified 5 factors: psychosocial health, pain and discomfort, sensory and physical functioning, capability well-being, and positive psychological states. Feeling settled and secure was the only ICECAP-A item that loaded onto the same factor (psychosocial health) as any of the EQ-HWB items.</p><p><strong>Conclusions: </strong>To our knowledge, this is the first study to compare the measurement performance of EQ-HWB and EQ-HWB-S with ICECAP-A. The EQ-HWB and EQ-HWB-S showed good psychometric performance in an Irish population sample. Limited overlap was observed between EQ-HWB and ICECAP-A well-being concepts.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144038876","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-Utility Analysis of Durvalumab & Tremelimumab Versus Best Supportive Care in Refractory Metastatic Colorectal Cancer.","authors":"Monish Ahluwalia, Ambica Parmar, Jonathan M Loree, Christopher J O'Callaghan, Dongsheng Tu, Kelvin Kw Chan","doi":"10.1016/j.jval.2025.04.2159","DOIUrl":"https://doi.org/10.1016/j.jval.2025.04.2159","url":null,"abstract":"<p><strong>Objectives: </strong>To determine the cost-effectiveness of combined durvalumab and tremelimumab in patients with metastatic colorectal cancer in the intention-to-treat (ITT) and biomarker-enriched populations using direct CCTG CO.26 phase II trial data.</p><p><strong>Methods: </strong>A 4-state microsimulation model was used to evaluate the expected health outcomes in quality-adjusted life-years (QALYs) and costs (2023 CAD) over a lifetime horizon (5 years) from the Canadian public-payer perspective. Direct phase II CCTG CO.26 trial data informed model inputs, including OS Kaplan-Meier (KM) curves, progression-free survival KM curves, and adverse event rates. Health state utilities and costs of therapy, hospitalization, end-of-life care, sequencing panels, and physician care were obtained from published literature and Canadian costing databases. The incremental cost-utility ratios (ICURs) for the ITT and biomarker-enriched populations were determined.</p><p><strong>Results: </strong>In the ITT population, expected QALYs for the treatment and BSC arms were 0.47 and 0.33 (incremental (Δ)0.14) respectively, and expected costs were $56,743 and $17,177 (Δ$39,566) for an ICUR of $277,661/QALY. In the pTMB >28 subgroup, expected QALYs were 0.43 and 0.21 (Δ0.21) and expected costs were $58,498 and $16,941 (Δ$41,557) for an ICUR of $193,945/QALY.</p><p><strong>Conclusions: </strong>Combined durvalumab and tremelimumab is not cost-effective in refractory mCRC per conventional cost-effectiveness thresholds. Cost-effectiveness is more favourable in the high pTMB subgroup, but costs of screening and cutoffs used must be considered.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036093","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}