Value in Health最新文献

{"title":"Unlocking Patient Preferences: The Potential of Stated-Preference Methods in Clinical Decision Making.","authors":"Semra Ozdemir, Jorien Veldwijk, Janine van Til, Ilene L Hollin, Deborah A Marshall, Shelby D Reed","doi":"10.1016/j.jval.2025.08.016","DOIUrl":"10.1016/j.jval.2025.08.016","url":null,"abstract":"<p><p>Stated-preference (SP) methods are gaining attention as tools to support patient-centered clinical decision making by quantifying individual preferences through structured tradeoffs. These methods may improve shared decision making by helping patients and clinicians better understand the relative importance of treatment attributes and the trade-offs patients are willing to make. However, integrating SP methods into clinical settings poses practical challenges, including concerns about complexity, patient burden, and relevance of hypothetical scenarios. Successful application will require thoughtful design, attention to accessibility, and collaboration across disciplines. Despite current limitations, SP methods hold promise for enhancing the quality and alignment of medical decisions with patient values.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145056030","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 head-to-head comparison of the psychometric properties of the EQ-5D-Y-3L, EQ-5D-Y-5L, and CHU9D in children with asthma.","authors":"Rachel Lee-Yin Tan, Jian Yi Soh, Elizabeth Huiwen Tham, Anne Eng Neo Goh, Zai Ru Cheng, Michael Herdman, Nan Luo","doi":"10.1016/j.jval.2025.08.017","DOIUrl":"https://doi.org/10.1016/j.jval.2025.08.017","url":null,"abstract":"<p><strong>Objectives: </strong>There is limited evidence on the relative performance of generic preference-weighted health-related quality of life instruments designed for paediatric patients. This study aimed to compare the psychometric properties of EQ-5D-Y-3L (Y-3L), EQ-5D-Y-5L (Y-5L), and CHU9D in patients with asthma.</p><p><strong>Methods: </strong>Patients diagnosed with asthma, aged between 8-18 years old completed the Y-3L, Y-5L, CHU9D, and the PROMIS Asthma Impact Scale Short Form (PAIS-SF). Patients' asthma control was assessed using the Global Initiative for Asthma (GINA) classification. Patients were followed up with a single item on change in health, Y-3L, Y-5L, and CHU9D in a subsequent clinic visit. Ceiling effects of the instruments were compared. Convergent validity and known-groups validity were assessed using hypothesis testing. Responsiveness was assessed using mean change scores and Standardized Effect Size (SES).</p><p><strong>Results: </strong>A total of 164 paediatric patients (mean age:11.9; female: 31.7%) participated in the study, with 93 patients completing the follow-up survey. The ceiling effects of Y-3L, Y-5L, and CHU9D, were 54.9%, 53.7%, and 19.5%, respectively. Almost all convergent and known-group validity hypotheses tested for the three instruments, based on PAIS-SF and GINA, were met. Y-5L best discriminated between relevant known groups, while CHU9D showed better responsiveness (SES= 0.43 in children reporting improvement), compared to Y-3L (SES= 0.16), and Y-5L (SES= 0.17).</p><p><strong>Conclusion: </strong>While the EQ-5D-Y instruments demonstrated stronger construct validity, their responsiveness appeared to be limited and inferior to the CHU9D in the study sample. Further research in more clinically diverse asthma populations is needed to inform instrument selection.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145055922","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":"Methodological Insights from Health Valuation Studies in Low- and Middle-Income Countries: A Scoping Review.","authors":"Thomas Gadsden, Janine Verstraete, Audrey Moyo, Stanley Carries, Nokwanda Sithole, Eugene Lee Davids, Donela Besada, Blake Angell, Stephen Jan, Darshini Govindasamy","doi":"10.1016/j.jval.2025.08.014","DOIUrl":"https://doi.org/10.1016/j.jval.2025.08.014","url":null,"abstract":"<p><strong>Objectives: </strong>Valuation studies generate utility values for health states using stated preference methods. These studies are complex and resource intensive, posing implementation challenges in low- and middle-income countries (LMICs). This review aimed to examine how valuation studies in LMICs have navigated these challenges.</p><p><strong>Methods: </strong>A scoping review was conducted, with database (MEDLINE, EMBASE and CINAHL) and grey literature searches performed between April and June 2024. Inclusion was limited to valuation studies in LMICs using generic or disease-specific instruments and stated-preference techniques with adult respondents and published in English. Results were narratively synthesized.</p><p><strong>Results: </strong>Thirty-six studies from 22 LMICs were included. Studies were conducted across low (n=2), lower-middle (n=11), and upper-middle income countries (n=9). Half were published since 2020. Thirty-three studies developed nationally representative value sets, two of which were based on patient preferences. Two pilot studies and one cancer-specific value set were also included. The EQ-5D-5L was used most (n=16), followed by the EQ-5D-3L (n=14), SF-6D (n=4), CQ-11D (n=1) and EORTC-8D (n=1). Methodological adaptions included 'lite' protocols, portable tools, and crosswalk methodology. Comprehension aids were reported in 11 studies; five included illiterate participants, and seven were conducted in multiple languages.</p><p><strong>Conclusion: </strong>Valuation studies are increasing rapidly in LMICs and there is growing experimentation to reduce resource demands and enhance inclusivity. While this is promising, the resource demands of valuation studies still limit their implementation in low-income settings. As a result, countries may still find it more cost-efficient to adapt value sets from neighbouring countries rather than develop their own.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145001137","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":"Author Reply.","authors":"Jagpreet Chhatwal, Rachael L Fleurence","doi":"10.1016/j.jval.2025.08.013","DOIUrl":"10.1016/j.jval.2025.08.013","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145001090","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 Impact of the Inflation Reduction Act's Drug Price Negotiation Program on Incentives for Clinical Development of New Drugs.","authors":"Robert J Nordyke, Julie A Patterson, James Motyka, Benjamin March, Noam Y Kirson, Genia Long","doi":"10.1016/j.jval.2025.08.011","DOIUrl":"10.1016/j.jval.2025.08.011","url":null,"abstract":"<p><strong>Objectives: </strong>We explore the impact of the Drug Price Negotiation Program (DPNP) on investment incentives for drug research and development (R&D).</p><p><strong>Methods: </strong>We modeled 5 hypothetical scenarios of future drugs selected for the DPNP. Scenarios were defined by disease area, post-approval development strategy, and potential responses to the DPNP, such as delaying, resequencing, or canceling additional indications. We estimated risk-adjusted net present values and internal rates of return at 3 points during development of initial indications: beginning of phase 1, phase 2 (accelerated approval) or 3 (others), and FDA approval. Small molecules and biologics were modeled.</p><p><strong>Results: </strong>The DPNP had a substantial impact on estimated investment returns across disease areas and post-approval development strategies for the modeled drugs that were assumed to be selected for the program. At the start of phase 1 for the initial indication, DPNP reduced the modeled discounted value of projects, and the estimated net present value reductions ranged from 22% to 95% for small molecules and 14% to 45% for biologics. Estimated internal rates of return were also reduced, by 5% to 14% for small molecules and 3% to 7% for biologics. No modeled response restored investment incentives to pre-DPNP levels; however, some modeled responses improved estimated returns relative to post-DPNP.</p><p><strong>Conclusions: </strong>Examples illustrated how the DPNP may negatively affect R&D investment incentives for small molecules and biologics that were likely to be selected. Although short-term observed effects may be limited, over the longer term, DPNP-induced reductions in investment incentives may redirect funding away from programs with the largest expected impacts, potentially leading to far-reaching implications for drug development.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144993343","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":"Clinical Outcomes and Measures for Vaginal Relaxation Syndrome: A Systematic Review.","authors":"Hongqin Chen, Jian Meng, Qiao Li, Yajing Wang, Yueyue Chen, Xiaoyu Niu, Dongmei Wei","doi":"10.1016/j.jval.2025.08.012","DOIUrl":"10.1016/j.jval.2025.08.012","url":null,"abstract":"<p><strong>Objectives: </strong>This study systematically investigates the application of clinical outcomes and measures reported in the management of vaginal relaxation syndrome (VRS). It further analyzes and compares variations in clinical outcomes and measures across different interventions in VRS, with a focus on assessing their generalizability and applicability. The findings aim to inform the design of high-quality clinical trials and provide a foundation for developing a Core Outcome Set (COS) and a Core Outcome Measurement Set (COMS).</p><p><strong>Methods: </strong>A systematic search of PubMed, Embase, Web of Science, and Cochrane databases (up to January 2025) identified clinical studies on VRS treatment. Two researchers independently screened studies, extracted data, and analyzed clinical outcomes, measurement tools, and assessment time points.</p><p><strong>Results: </strong>A total of 74 studies (15 randomized controlled trials, 59 observational studies, and 4866 patients) reported 113 outcomes using 85 measurement tools. The most frequently used were patient-reported outcome measures (Female Sexual Function Index, Vaginal Laxity Questionnaire, VAS, and PISQ-12), followed by clinician-reported outcome measures (VHI and biopsy). Most follow-ups lasted under 6 months. Surgical treatments uniquely assessed recurrence, aesthetic satisfaction, and partners' sexual satisfaction, whereas nonsurgical approaches focused on overall efficacy, tolerability, and patients satisfaction with vaginal tightness.</p><p><strong>Conclusions: </strong>Clinical outcomes and measures for VRS are overly complex, particularly PROs. Future research should focus on optimizing PROs by developing highly feasible, practical PROMs. Delphi surveys and expert consensus could establish a comprehensive VRS-specific Core Outcome Set and Core Outcome Measurement Set, alongside standardized assessment time points, improving research consistency and evidence-based management of VRS.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144993314","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":"How Do Different Health States Impact Preferences for Social-Care-Related Quality of Life: An Exploration of EQ-5D-5L and Adult Social Care Outcomes Toolkit Using a Vignette Approach.","authors":"Peiwen Jiang, Deborah J Street, Rosalie Viney, Alice Yu, Brendan Mulhern","doi":"10.1016/j.jval.2025.08.010","DOIUrl":"10.1016/j.jval.2025.08.010","url":null,"abstract":"<p><strong>Objectives: </strong>Social-care-related quality-of-life instruments, such as the Adult Social Care Outcomes Toolkit (ASCOT), are important for valuing interventions, particularly when they include utility weights. Utility weights may vary by population and health conditions; yet, most value sets are developed without considering contexts. This study aims to explore this issue by assessing whether the valuation of ASCOT social states differs in the context of poor physical or mental health versus full health.</p><p><strong>Methods: </strong>An online discrete choice experiment was conducted with a representative sample of 654 respondents in Australia. Respondents completed choice tasks choosing between ASCOT social care states, while imagining living in a poor health (EQ-5D-5L) state for half of the tasks and in full health for the other half. Respondents were evenly assigned to 2 study arms (poor physical or mental health). Data were analyzed using multinomial and mixed logit models, with interaction terms assessing the impact of context.</p><p><strong>Results: </strong>In the mental health arm, the poor health context had no statistically significant impact on ASCOT estimates compared with the full health context. In the physical health arm, there were significant differences in the coefficients of \"control\" (level 4), \"safety\" (level 2), and \"accommodation\" (level 4). However, in all cases, \"control,\" \"safety,\" and \"cleanliness\" accounted for more than 50% of the relative attribute importance.</p><p><strong>Conclusions: </strong>This study found limited influence of health context on valuation of social-care-related quality-of-life dimensions, providing evidence supporting development of ASCOT value sets independent of context.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144993317","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":"Patient-Reported Outcomes (PROs) Now and into the Future: Multi-Stakeholder Perspectives on Optimizing PRO Evidence Use in Decision-Making across the US Healthcare Continuum.","authors":"Sharan Randhawa, Adam Gater, Roy Bentley","doi":"10.1016/j.jval.2025.08.008","DOIUrl":"https://doi.org/10.1016/j.jval.2025.08.008","url":null,"abstract":"<p><strong>Objectives: </strong>Patient-reported outcome measures (PROMs) are tools intended to capture directly from patients how they feel and/or function. They can provide important evidence to support regulatory approval, product labeling, payer reimbursement, and clinical care decisions. However, patient-reported outcome (PRO) evidence remains underutilized in regulatory, payer, and overall healthcare decision-making. This initiative aimed to understand stakeholder perspectives regarding current use of PRO evidence across the US healthcare continuum and opportunities to further advance its use in healthcare-related decision-making.</p><p><strong>Methods: </strong>Three roundtable meetings were convened with an expert panel representing patients/patient advocacy groups, clinicians, PRO experts, payers/purchasers, and policymakers. Meetings were audio-recorded and transcripts were reviewed to identify key discussion points and themes via thematic analysis methods. Group members reviewed, discussed, and commented on findings as they emerged.</p><p><strong>Results: </strong>The themes that emerged on maximizing use of PRO evidence were: 1. Educating stakeholders on its value; 2. Ensuring standardization of PROM development methods, including maximizing patient engagement; 3. Addressing PROM implementation considerations in clinical trials and practice; 4. Effectively communicating PRO evidence to stakeholders; and 5. Encouraging PRO evidence integration into policy and shared clinical decision-making. A five-component framework resulted.</p><p><strong>Conclusions: </strong>PRO evidence is fundamental to patient-focused drug development and clinical care. Collaboration among all stakeholders making decisions related to treatment approval, access, and use is critical to realizing the value of PRO evidence, alongside greater stakeholder transparency regarding how PRO evidence is used to inform decisions. The five-component framework, co-developed by a multistakeholder expert panel, can help to support and enhance these collaborations.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144970729","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":"Pharmaceutical Pricing Evidence From a Healthcare System With Multiple Cost-per-Quality-Adjusted Life-Year Thresholds.","authors":"Anton P H Klockhoff, Jonathan Siverskog, Martin Henriksson","doi":"10.1016/j.jval.2025.07.027","DOIUrl":"10.1016/j.jval.2025.07.027","url":null,"abstract":"<p><strong>Objectives: </strong>To study drug pricing in a reimbursement system accepting a higher cost per quality-adjusted life-year for drugs targeting conditions of higher severity. We investigate whether higher incremental cost-effectiveness ratios (ICERs) are observed for conditions of higher severity, how close to the highest acceptable cost-per-quality-adjusted life-year threshold drug prices are set, and whether the thresholds are applied as price ceilings. Furthermore, we explore factors other than severity that might affect pricing.</p><p><strong>Methods: </strong>Disease severity and ICERs were extracted from publicly available reimbursement decisions made between 2017 and 2024 by the Swedish Dental and Pharmaceutical Benefits Agency. Linear regression assessed whether ICERs increase significantly with severity and other covariates and whether the same covariates explain deviations from the highest acceptable ICERs. We also assessed whether higher severity was associated with a higher likelihood of having confidential rebates in place.</p><p><strong>Results: </strong>A total of 84 decisions reported ICERs and disease severity. ICERs increased significantly with severity (P < .01). The average ICER was 30.6% lower than the relevant threshold, and there was no evidence of pricing above the thresholds. Very high severity was associated with a 27.4% higher probability than moderate severity of the ICER including confidential rebates (P < .01). ICERs including rebates were associated with 19.1 percentage points smaller deviations from the threshold (P < .01).</p><p><strong>Conclusions: </strong>Prices tend to be set below the reimbursement system's thresholds and these appear to be ceilings, but decision makers should anticipate that drug companies will adjust prices in response to reimbursement policy, at least to some degree.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144970649","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":"Reevaluating Artificial Intelligence-Enabled Emergency Care in the National Health Service: Economic Value, Diagnostic Realities, and Ethical Challenges.","authors":"Boju Wang, Zekai Yu","doi":"10.1016/j.jval.2025.06.023","DOIUrl":"10.1016/j.jval.2025.06.023","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144970696","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}