Value in Health最新文献

{"title":"Synergizing Needs Assessments and Patient Preference Studies for Enhanced Patient-Centered Decision Making in Healthcare","authors":"Elise Schoefs MSc ,&nbsp;Alice Vanneste MSc ,&nbsp;Alessandra Blonda MSc ,&nbsp;Zilke Claessens MSc ,&nbsp;Khadidja Abdallah PhD ,&nbsp;Fábio Cardoso Borges MPharm ,&nbsp;Jolien Broekmans MSc ,&nbsp;Thomas Desmet MSc ,&nbsp;Teodora Lalova-Spinks PhD ,&nbsp;Phaedra Locquet MSc ,&nbsp;Janos Meszaros PhD ,&nbsp;Lauren Michiels MPharm ,&nbsp;Charlotte Verbeke MSc ,&nbsp;Io Wens MSc ,&nbsp;Isabelle Huys PhD","doi":"10.1016/j.jval.2025.04.2169","DOIUrl":"10.1016/j.jval.2025.04.2169","url":null,"abstract":"<div><h3>Objectives</h3><div>Over the past decades, criticism has grown regarding the supply-driven approach of medicinal product development. In response, patient-centered methods have been developed to inform decision making. This perspective article aims to reflect on two of these methods, which are needs assessments and patient preference studies (PPSs).</div></div><div><h3>Methods</h3><div>A reflection was conducted based on existing literature and our collaborative experiences, proposing a conceptual framework that synergistically combines needs assessments and PPSs.</div></div><div><h3>Results</h3><div>Needs assessments identify unmet health-related needs from the patient and/or societal perspective, whereas PPSs determine the trade-offs patients make among treatment options. Although both methods produce patient-based evidence, their differing scopes and purposes offer complementary benefits and limitations. Combining needs assessments with PPSs can result in significant advantages by providing a holistic and in-depth understanding of patients’ needs and preferences. In their conceptual framework, the authors advocate for a sequential approach: conducting a needs assessment to identify a broad spectrum of unmet health-related needs, followed by a PPS to capture nuanced preferences guiding patients’ priorities. Although this approach enhances accuracy and relevance, practical constraints and contextual considerations may hamper its application, necessitating careful consideration of the most suitable approach based on the specific research context.</div></div><div><h3>Conclusions</h3><div>This perspective article provides clarity on the effective use of both methods in navigating patient-centered research in healthcare. It emphasizes the need for well-designed, unbiased studies applying this conceptual framework to shift from a supply-driven to a needs- and preference-driven healthcare system, ensuring that innovations align more closely with patients’ true needs and preferences.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1253-1258"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144095226","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 Health-Related Quality of Life: Evidence on the Relationship Between and Comparative Performance of the EQ-5D-Y-5L and EQ-5D-5L","authors":"Nicole Reyes MPH ,&nbsp;Tianxin Pan PhD ,&nbsp;Renee Jones MPH ,&nbsp;Kim Dalziel PhD ,&nbsp;Nancy Devlin PhD ,&nbsp;Quality Of Life in Kids: Key evidence to strengthen decisions in Australia (QUOKKA) project team","doi":"10.1016/j.jval.2025.04.2161","DOIUrl":"10.1016/j.jval.2025.04.2161","url":null,"abstract":"<div><h3>Objectives</h3><div>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.</div></div><div><h3>Methods</h3><div>The Australian Pediatric Multi-Instrument Comparison 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 summarized using the level sum score. Acceptability, feasibility, ceiling effects, convergence, test-retest reliability, and known-group validity were assessed overall and in subgroups defined by special healthcare needs (SHCN), mental health concerns (MHC), and age (12-13, 14-16, and 17-18 years).</div></div><div><h3>Results</h3><div>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, whereas 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.</div></div><div><h3>Conclusions</h3><div>Although both instruments are valid for measuring HRQoL in adolescents aged 12 to 18 years, 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 involve transitions between age groups.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1231-1240"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","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":"Mapping EQ-5D-5L Utilities in Dementia: Integrating Self and Proxy Reports","authors":"Hannah Hussain MPharm, MSc, PhD ,&nbsp;Anju Keetharuth PhD ,&nbsp;Allan Wailoo PhD ,&nbsp;Donna Rowen PhD","doi":"10.1016/j.jval.2025.05.009","DOIUrl":"10.1016/j.jval.2025.05.009","url":null,"abstract":"<div><h3>Objectives</h3><div>EQ-5D is widely used in dementia research, but cognitive impairments often necessitate proxy assessments, resulting in differences between self- and proxy-reported data. Some EQ-5D dimensions are better reported by people with dementia (PwD), particularly in the mild to moderate stages, whereas others are more accurately captured by proxies. This study evaluates whether a combined utility value, integrating both PwD and proxy reports, can be predicted when data from only 1 respondent type are available.</div></div><div><h3>Methods</h3><div>Data from 2 dementia studies, ACTIFCARE and EPIC, were used to develop mapping models aimed at predicting combined utility values. These models integrate dimension-specific EQ-5D-5L responses from both respondent types to enhance health-related quality-of-life (HRQoL) assessments. Response mapping with ordered probit models was used to predict EQ-5D-5L responses when only 1 respondent type’s data were available. Model performance was evaluated by comparing observed and predicted data across dementia severity stages.</div></div><div><h3>Results</h3><div>Combined utility values provided a more accurate reflection of HRQoL, showing greater sensitivity to health status changes. Proxy data proved to be more effective in predicting PwD responses for certain EQ-5D-5L dimensions, suggesting that proxy data collection may be particularly useful in specific situations. The mapping models performed well for mild-to-moderate dementia but were less accurate in severe dementia because of limited data.</div></div><div><h3>Conclusions</h3><div>Combined utility values improve HRQoL assessments, particularly in detecting subtle health changes in mild-to-moderate dementia. These models support their use in economic evaluations of dementia interventions, although challenges remain in severe dementia because of data limitations.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1204-1212"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144249827","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 Exploratory Study of Alternative Time Frames and Descriptors for EQ-5D-5L in Obstructive Airway Diseases Using Mixed Methods","authors":"","doi":"10.1016/j.jval.2024.12.001","DOIUrl":"10.1016/j.jval.2024.12.001","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1303-1304"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142903628","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 PhD ,&nbsp;Rachael L. Fleurence PhD, MSc","doi":"10.1016/j.jval.2025.04.2164","DOIUrl":"10.1016/j.jval.2025.04.2164","url":null,"abstract":"<div><div>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.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1141-1144"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","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":"The Burden of Mitochondrial Disease: Healthcare and Societal Costs","authors":"Deborah Schofield PhD ,&nbsp;Katherine Lim MHlthEcPol ,&nbsp;Owen Tan PhD ,&nbsp;Rupendra Shrestha PhD ,&nbsp;Sameen Haque PhD ,&nbsp;Karen Crawley MBBS ,&nbsp;Sarah West MBMSc ,&nbsp;Adam Percival ,&nbsp;Jayamala Parmar MScMedSci ,&nbsp;Joshua Kraindler MSc ,&nbsp;Carolyn M. Sue PhD","doi":"10.1016/j.jval.2025.05.016","DOIUrl":"10.1016/j.jval.2025.05.016","url":null,"abstract":"<div><h3>Objectives</h3><div>To estimate the burden of mitochondrial disease by measurement of healthcare, societal, and lifetime costs of mitochondrial disease.</div></div><div><h3>Methods</h3><div>We recruited patients aged 18 years or over with a clinical confirmation of mitochondrial disease and their carer from a mitochondrial disease referral center to complete a survey for our study. The survey responses and linked administrative data from the patients and carers were used in a microsimulation model to estimate the healthcare and societal costs of mitochondrial disease.</div></div><div><h3>Results</h3><div>In total, 92.5% recruited agreed to participate in our study. We estimated total annual average costs at AU$ 112 721 per household. Of the total annual average costs, a large proportion were societal costs (92%). 4%, 10%, and 86% of societal costs were borne by the Commonwealth government, state government, and private out of pocket, respectively. In total, 8% of the total annual average costs were healthcare, with 61%, 9%, and 30% of the annual average health costs borne by the Commonwealth government, state government, and private out of pocket, respectively. We estimated the total lifetime cost of mitochondrial disease at AU$ 7.6 million per household and the national annual cost is estimated at AU$ 8.6 billion.</div></div><div><h3>Conclusions</h3><div>Mitochondrial disease is an expensive condition with the majority of the costs comprising societal costs, with substantial costs borne privately out of pocket. The findings from this study can be used in other studies, such as cost-effectiveness analysis, to examine the benefits of interventions to treat or prevent mitochondrial disease.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1169-1175"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258960","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":"Psychometric Properties of the Modified EQ Health and Wellbeing and its Shorter Version in a General Chinese Population: A Comparative Analysis With EQ-5D-5L, SF-6Dv2, ICEpop CAPability measure for Adults, Short Version of Warwick-Edinburgh Mental Wellbeing Scale, and World Health Organization Well-Being Index 5-Item","authors":"Richard H. Xu PhD ,&nbsp;Fanni Rencz PhD","doi":"10.1016/j.jval.2025.04.2151","DOIUrl":"10.1016/j.jval.2025.04.2151","url":null,"abstract":"<div><h3>Objectives</h3><div>The Modified EQ Health and Wellbeing (EQ-HWB) and its shorter version (EQ-HWB-S) are new health and well-being measures nearing finalization. Since the 2022 version, the development group has proposed revisions to the item order, response levels, and wording. This study aimed to assess the psychometric properties of the modified Simplified Chinese EQ-HWB and EQ-HWB-S and compare their performance with other measures.</div></div><div><h3>Methods</h3><div>The data were collected through a web-based survey of the Chinese general population (<em>n</em> = 1053). Outcome measures included the modified EQ-HWB, EQ-5D-5L, SF-6Dv2, ICEpop CAPability measure for Adults, short version of Warwick-Edinburgh Mental Wellbeing Scale (SWEMWBS), and World Health Organization Well-Being Index 5-Item. We examined psychometric properties, such as distributional characteristics (ceiling and floor), convergent (correlations), known-group (effect sizes), and structural validity (principal component analysis).</div></div><div><h3>Results</h3><div>At the instrument level, neither the modified EQ-HWB nor EQ-HWB-S showed ceiling effect. The response distribution of the 3 items (enjoyable activities, excluded by others, and negative feelings about oneself) modified from positively to negatively framed was in the range of the other items. The EQ-HWB “getting around inside or outside,” “day-to-day activities,” and “enjoyable activities” items showed only moderate correlations with the corresponding EQ-5D-5L and SF-6Dv2 dimensions (rho = 0.31-0.36). The EQ-HWB items mainly loaded on separate factors from the ICEpop CAPability measure for Adults, short version of Warwick-Edinburgh Mental Wellbeing Scale, and World Health Organization Well-Being Index 5-Item items. The EQ-HWB effectively differentiated between sociodemographic and health-related known groups with larger effect sizes than the EQ-HWB-S.</div></div><div><h3>Conclusions</h3><div>The modified EQ-HWB and EQ-HWB-S demonstrated mixed psychometric performance. Several modified items showed improved distributional characteristics compared with earlier findings with the original EQ-HWB. However, item-level correlations were somewhat weaker than those reported in previous studies using the original version.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1241-1252"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143983512","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 PhD, MPSA ,&nbsp;James Buchanan PhD ,&nbsp;Ilias Goranitis PhD ,&nbsp;Maarten J. IJzerman PhD ,&nbsp;Tara A. Lavelle PhD ,&nbsp;Deborah A. Marshall PhD ,&nbsp;Dean A. Regier PhD, MA ,&nbsp;Wendy J. Ungar PhD ,&nbsp;Deirdre Weymann MA ,&nbsp;Sarah Wordsworth PhD ,&nbsp;Kathryn A. Phillips PhD ,&nbsp;Jeroen P. Jansen PhD","doi":"10.1016/j.jval.2025.04.2162","DOIUrl":"10.1016/j.jval.2025.04.2162","url":null,"abstract":"<div><h3>Objectives</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1213-1220"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","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 Comprehensive View of the Methods Used to Measure the Societal Impact of Healthcare Interventions: A Systematic Review","authors":"Ahmed H. Seddik PhD ,&nbsp;Jeroen Paulissen MSc ,&nbsp;Ramesh Marapin MSc ,&nbsp;Sebastiaan Fuhler MSc ,&nbsp;Sukhvinder Johal PhD ,&nbsp;Mats Rosenlund PhD, MPH ,&nbsp;Kyle Dunton MSc ,&nbsp;Maarten Postma PhD ,&nbsp;Roel Freriks PhD","doi":"10.1016/j.jval.2025.02.009","DOIUrl":"10.1016/j.jval.2025.02.009","url":null,"abstract":"<div><h3>Objectives</h3><div>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.</div></div><div><h3>Methods</h3><div>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.</div></div><div><h3>Results</h3><div>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.</div></div><div><h3>Conclusions</h3><div>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.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1288-1299"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","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":"Addressing Subgroup-Specific Treatment Effects to Avoid Inefficient Care: The Conflict Between Statistical and Health Economic Analyses","authors":"Sopany Saing PhD ,&nbsp;Gerjon Hannink PhD ,&nbsp;H. Amarens Geuzinge PhD ,&nbsp;Hendrik Koffijberg PhD","doi":"10.1016/j.jval.2025.05.004","DOIUrl":"10.1016/j.jval.2025.05.004","url":null,"abstract":"<div><h3>Objectives</h3><div>When patients are classified into subgroups based on previously identified heterogeneity, this heterogeneity may affect the cost-effectiveness of health interventions. Whether this heterogeneity is reflected or ignored in cost-effectiveness analysis may influence reimbursement decisions. This is illustrated using a simulation study of a hypothetical treatment to prevent disease progression.</div></div><div><h3>Methods</h3><div>With the decision analysis in R for technologies in health Sick-Sicker Markov model, we analyzed the cost-effectiveness of Treatment versus standard of care in a population comprising group 1 (G1) and group 2 (G2). We compared 3 strategies for informing reimbursement decisions: (1) ignore evidence on subgroup differences (ignore subgroup evidence), (2) test for subgroup differences in trial data at hand (statistically guided), and (3) use all evidence on subgroup differences (all evidence). This simulation study varied total sample size, G2 proportion, treatment effectiveness, and baseline mortality risk. For each scenario, the net health benefit and reimbursement decision (ie, reimburse in both subgroups, G1 only, G2 only, or no reimbursement) was determined per strategy.</div></div><div><h3>Results</h3><div>The statistically guided strategy led to subgroups being ignored except for the largest total sample sizes. At a willingness-to-pay threshold of €50 000/quality-adjusted life years gained, the statistically guided strategy resulted in an incremental net health benefit of −1.00 and 0.49 when compared with the strategies of ignoring subgroup evidence and for incorporating, respectively.</div></div><div><h3>Conclusions</h3><div>When subgroup heterogeneity is known, ignoring subgroups or taking a statistically guided approach will result in suboptimal reimbursement decisions and thus fail to optimize societal benefits. Therefore, subgroup-specific cost-effectiveness analyses should be informed by all available evidence of subgroup differences.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 8","pages":"Pages 1145-1152"},"PeriodicalIF":6.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143668","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}