Value in Health最新文献

{"title":"Item-Level Convergent and Structural Validity of the EQ-HWB-9 in US Informal Caregivers Compared With ASCOT-Carer, CarerQoL, and EQ-5D-5L","authors":"Soumana C. Nasser PharmD,&nbsp;A. Simon Pickard PhD,&nbsp;Maja Kuharić PhD","doi":"10.1016/j.jval.2026.04.003","DOIUrl":"10.1016/j.jval.2026.04.003","url":null,"abstract":"<div><h3>Objectives</h3><div>To examine convergent validity and factorial structure of the EQ Health and Well-being 9-items (EQ-HWB-9) among US caregivers relative to caregiver-specific (Care-Related Quality of Life-7 Dimension [CarerQoL], Adult Social Care Outcomes Toolkit for Carers) and generic (EuroQol 5-Dimension 5-Level) measures.</div></div><div><h3>Methods</h3><div>Cross-sectional data from 504 informal caregivers were collected via online survey (2022-2023). Convergent validity was assessed using Spearman correlations with a priori hypotheses of strong correlations (<em>r</em>_s &gt; 0.50) between conceptually similar items. Exploratory factor analysis using unweighted least-squares extraction and oblique rotation with parallel analysis examined dimensional structure across all instruments.</div></div><div><h3>Results</h3><div>Convergent validity hypotheses were supported. Strong correlations emerged for physical dimensions (EQ-HWB mobility with EQ-5D mobility, <em>r</em>_s = 0.57; EQ-HWB daily activities with EQ-5D usual activities, <em>r</em>_s = 0.55) and emotional dimensions (EQ-HWB sadness/depression with EQ-5D anxiety/depression, <em>r</em>_s = 0.67). For caregiver-specific measures, strong correlations included EQ-HWB sadness/depression with CarerQoL mental health (<em>r</em>_s = 0.56) and EQ-HWB control with both ASCOT-Carer control (<em>r</em>_s = 0.51) and ASCOT-Carer occupation/spend time (<em>r</em>_s = 0.51). Weaker correlations between theoretically distinct constructs supported discriminant validity (<em>r</em>_s = 0.02-0.45). A 4-factor solution explained 100% of common variance with minimal cross-loading: social care-related quality of life (ASCOT-Carer items exclusively), mental/emotional well-being (primarily EQ-HWB and EQ-5D emotional items), physical functioning (EQ-5D and EQ-HWB physical items), and care-related burden (CarerQoL items). The 100% of common variance explained reflects the common factor model’s extraction of shared variance only, excluding unique variance.</div></div><div><h3>Conclusion</h3><div>EQ-HWB-9 demonstrated strong convergent validity with clear factorial distinction from caregiver-specific measures, capturing general health constructs complementary to unique care-related domains assessed by ASCOT-Carer and CarerQoL. These findings support EQ-HWB-9 as a valid measure of general health and well-being in caregiver populations, while indicating that comprehensive caregiver assessment may benefit from combining generic and caregiver-specific instruments depending on study objectives.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1706-1713"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147782316","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric Performance of Preference-Weighted Instruments in Older Adults: A Systematic Review","authors":"Lidia Engel PhD,&nbsp;Valeriia Sokolova MSc,&nbsp;Jan Faller MSc,&nbsp;Yared Belete Belay PhD,&nbsp;Pei Wang PhD,&nbsp;Jan Abel Olsen PhD,&nbsp;Brendan Mulhern PhD","doi":"10.1016/j.jval.2026.03.2234","DOIUrl":"10.1016/j.jval.2026.03.2234","url":null,"abstract":"<div><h3>Objectives</h3><div>Population aging is a major public health concern. Preference-weighted instruments are widely used to assess health-related quality of life and inform cost-effectiveness analyses. Given the range of available measures, it is important to identify those most suitable for older adults. This study systematically reviewed evidence on the psychometric properties of preference-weighted measures in older adults.</div></div><div><h3>Methods</h3><div>Four databases (Medline, Embase, PsycINFO, and CINAHL) were searched, supplemented by citation tracking. Peer-reviewed studies assessing the psychometric properties of preference-weighted measures in adults aged ≥60 were included. Two reviewers independently screened and extracted data. Methodological quality was assessed using the Consensus-Based Standards for the Selection of Health Measurement Instrument risk of bias checklist, and evidence was graded using a modified GRADE approach.</div></div><div><h3>Results</h3><div>A total of 106 studies were included, covering EQ-5D-3L (4), EQ-5D-5L (34), ICECAP-O (29), ASCOT (15), QOL-ACC (9), HUI3 (8), HUI2 (7), QWB (5), EQ-HWB-9 (5), WOOP (3), SF-6D (3), AQoL-8D (2), AQoL-4D (1), and 15D (1). EQ-5D versions showed acceptable performance on some criteria, although ceiling effects, limited interrater reliability, and inconsistent responsiveness were reported. ICECAP-O and ASCOT showed more consistent evidence, but both need further testing on responsiveness. Limited and mixed findings were available for other measures. Early evidence for QOL-ACC and WOOP was promising but incomplete. Although interrater reliability and responsiveness evidence remained limited across all measures, specific measures performed generally better than generic measures but had fewer studies.</div></div><div><h3>Conclusions</h3><div>Preference-weighted measures are widely used with older adults, but psychometric evidence remains incomplete because of limited assessment of responsiveness, few head-to-head comparisons, and lack of age-group-specific analyses.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1714-1724"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147655260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Smoothing and Modeling Approach on Quality-Adjusted Life Expectancy Estimates","authors":"Hosein Ameri PhD,&nbsp;Thomas G. Poder PhD","doi":"10.1016/j.jval.2026.03.2241","DOIUrl":"10.1016/j.jval.2026.03.2241","url":null,"abstract":"<div><h3>Objectives</h3><div>Quality-adjusted life expectancy (QALE) is a composite indicator integrating life expectancy and health utility values. Most studies have used the Sullivan method to calculate QALE, whereas Markov modeling offers a more flexible alternative simulating health transitions over time. The primary objective of this study was to estimate age- and sex-specific QALE for Quebec and to compare results across 4 methodological approaches: Sullivan versus Markov modeling, each with and without cubic polynomial fit of age-specific utilities.</div></div><div><h3>Methods</h3><div>We analyzed 4803 EQ-5D-5L records from 2016 to 2024 health surveys and pooled 2021 to 2023 life tables. Age-specific utilities were smoothed using cubic polynomial regression. Age- and sex-specific QALE norms were estimated using both Sullivan method and a stochastic 2-state Markov microsimulation, with Monte Carlo simulations applied to both methods to quantify uncertainty. Sensitivity analyses assessed the impact of reducing the Markov cycle length from 1 year to 0.5 year for the combined population, and differences between methods were evaluated using a 1-sample <em>t</em> test.</div></div><div><h3>Results</h3><div>Cubic polynomial regressions produced smooth age-utility curves with excellent fit for the combined population (R² = 0.86), males (R² = 0.96), and females (R² = 0.92). Smoothed utilities modestly reduced uncertainty, particularly at older ages. Total QALE estimates from the Sullivan and Markov approaches were highly consistent, with small absolute differences (0.28-0.32 QALE) and strong correlation (R² = 0.99) over the full remaining lifetime. The 1-sample <em>t</em> test showed that Markov QALE estimates were slightly higher than Sullivan estimates (mean difference 0.315 QALE, 95% CI 0.308-0.322; t = 85.47, <em>P</em> &lt; .001), though the absolute differences were minor relative to overall QALE. Sensitivity analyses demonstrated that reducing the Markov cycle length had minimal impact on QALE estimates (differences ≤0.29 QALE), confirming robustness.</div></div><div><h3>Conclusions</h3><div>These findings support the use of either method for population health assessment and health technology evaluation because both produce valid and reliable QALE estimates.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1646-1652"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147718175","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-Minimization Analysis of the Women’s Cancer Risk Identification—Quantitative Polymerase Chain Reaction Test for Endometrial Cancer Epigenetic Biomarker Compared With Transvaginal Ultrasound for Triaging Abnormal Uterine Bleeding","authors":"Tristan M. Snowsill PhD,&nbsp;Sara Sleigh PhD,&nbsp;James Barrett PhD,&nbsp;Martin Widschwendter MD","doi":"10.1016/j.jval.2026.03.2235","DOIUrl":"10.1016/j.jval.2026.03.2235","url":null,"abstract":"<div><h3>Objectives</h3><div>Abnormal uterine bleeding most often has benign cause, but symptomatic cancers need to be found and treated quickly. An objective DNA methylation test, the Women’s Cancer Risk Identification—quantitative polymerase chain reaction test for Endometrial Cancer (WID-qEC), has equivalent sensitivity to transvaginal ultrasound (TVUS) for endometrial cancer detection but better specificity and lower failure rates. We conducted a cost-minimization analysis of WID-qEC versus TVUS for initial evaluation of abnormal uterine bleeding in those aged 45 years or older in secondary care cancer exclusion services.</div></div><div><h3>Methods</h3><div>The EPI-SURE study was a single-center prospective evaluation of the comparative diagnostic accuracy of WID-qEC and TVUS. A decision-analytic model was constructed using individual-level EPI-SURE data and National Health Service reference costs. Data from participants in EPI-SURE were combined with assumptions about downstream testing and estimates of relevant costs to determine the costs associated with initial evaluation by WID-qEC or TVUS.</div></div><div><h3>Results</h3><div>Definitive evaluation was required for 57% of patients with initial evaluation by TVUS but only for 12% of patients with initial evaluation by WID-qEC. A breakeven price of £302 was estimated, with a very high probability (&gt;99%) of WID-qEC resulting in cost savings at an indicative price of £200. A sensitivity analysis using a higher threshold for WID-qEC generated greater savings (breakeven price £331). At a WID-qEC price of £150, the mean cost saving was £152 per patient.</div></div><div><h3>Conclusions</h3><div>Use of the WID-qEC as the first line triage test in patients aged 45 years or older undergoing urgent evaluation of abnormal uterine bleeding has the potential to reduce costs and unnecessary invasive, often painful, subsequent testing.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1599-1607"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147655267","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessment of Carbon Emission Impact of Semaglutide in Patients With Type 2 Diabetes in the United Kingdom Using an Innovative Modeling Approach","authors":"Niels Lund MBA,&nbsp;Andreas Rasche PhD,&nbsp;Matthew Taylor PhD,&nbsp;Barrie Chubb Msc,&nbsp;Weiwei Xu MD, PhD,&nbsp;Loveleen Taneja DNB,&nbsp;Anamaria-Vera Olivieri MSc,&nbsp;Yuvraj Sharma MSc","doi":"10.1016/j.jval.2026.03.2249","DOIUrl":"10.1016/j.jval.2026.03.2249","url":null,"abstract":"<div><h3>Objectives</h3><div>To evaluate the carbon footprint and clinical outcomes of once-weekly subcutaneous semaglutide in patients with type 2 diabetes mellitus (T2DM) in the United Kingdom.</div></div><div><h3>Methods</h3><div>Using the IQVIA Core Diabetes Model, environmental and clinical impacts were projected over a lifetime horizon from the National Health Services perspective. Data from the SUSTAIN-2 (patients with T2DM) and SUSTAIN-6 (patients with T2DM and established cardiovascular [CV] risk) trials were used to compare semaglutide with sitagliptin and placebo, respectively. Outcomes included life expectancy measured in life-years (LYs) and quality-adjusted life expectancy measured in quality-adjusted life-years (QALYs) gained, incidence of diabetes-related complications and adverse events, carbon emissions, and incremental carbon footprint effectiveness ratios.</div></div><div><h3>Results</h3><div>In patients with T2DM, semaglutide dominates sitagliptin, with higher discounted LYs (0.096) and QALYs (0.095) gains, a 6.9% reduction in diabetes-related complications and adverse events, and reduced lifetime carbon emissions by 9.3% (13,899 kg carbon dioxide equivalent emissions [CO₂e] versus 15,319 kg CO₂e). Similarly, in patients with T2DM and established CV risk, semaglutide showed higher discounted LYs (0.206) and QALYs (0.164), with reductions in all complications except heart failure-related hospitalizations versus placebo. Lifetime carbon emissions were 5.2% lower with semaglutide (14,546 kg CO₂e versus 15,341 kg CO₂e), again with a dominant carbon footprint effectiveness ratio. The reduction in CO₂ was mainly driven by reduced complications and treatment needs. Sensitivity analyses supported the robustness of the results.</div></div><div><h3>Conclusion</h3><div>Semaglutide offers environmental advantages over sitagliptin for patients with T2DM overall and over standard of care for those with established CV risk.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1626-1635"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147821350","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 Frameworks for Economic Evaluation of Digital Health Interventions","authors":"Carl Asche PhD,&nbsp;Rossella Di Bidino PhD,&nbsp;Anita Burrell MA, MBA,&nbsp;Annette Champion MBA,&nbsp;Zsombor Zrubka MD, PhD","doi":"10.1016/j.jval.2026.03.2252","DOIUrl":"10.1016/j.jval.2026.03.2252","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1740-1741"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148151507","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 Autologous Stem Cell Transplantation in Multiple Myeloma Patients Aged 65 to 69 Years (CAREMM-2110 Study)","authors":"Jung Yeon Lee PhD,&nbsp;Seungpil Jung PhD,&nbsp;Sung-Soo Park PhD,&nbsp;Seonggyu Byeon PhD,&nbsp;Seung-Hwan Shin PhD,&nbsp;Young-Woo Jeon PhD,&nbsp;Seung-Ah Yahng PhD,&nbsp;Suein Choi PhD,&nbsp;Jin Seok Kim PhD,&nbsp;Chang-Ki Min PhD","doi":"10.1016/j.jval.2026.05.013","DOIUrl":"10.1016/j.jval.2026.05.013","url":null,"abstract":"<div><h3>Objectives</h3><div>Autologous stem cell transplantation (ASCT) remains a cornerstone therapy for eligible patients with multiple myeloma (MM), but its use in older adults is debated because of concerns about toxicity and cost-effectiveness (CE). This study aimed to evaluate the CE of upfront ASCT compared with nontransplant therapy in newly diagnosed patients with MM aged 65 to 69 years within a single-payer healthcare system.</div></div><div><h3>Methods</h3><div>Using nationwide claims data from the Korean Health Insurance Review and Assessment Service (HIRA database, 2009-2024; study enrollment period, 2017-2024), we conducted a CE analysis of 735 patients, of whom 458 received bortezomib-thalidomide-dexamethasone induction followed by ASCT and 277 received nontransplant therapy. A state-transition semi-Markov model-estimated life-years, quality-adjusted life-years (QALYs), and direct medical costs from a healthcare system perspective. Inverse probability of treatment weighting reduced confounding bias, and deterministic and probabilistic sensitivity analyses assessed parameter uncertainty.</div></div><div><h3>Results</h3><div>ASCT yielded superior overall survival (4-year: 67.8% vs. 55.0%; HR 0.62, <em>P</em> = .001) and longer time to next treatment (29.8 vs 19.7 months, <em>P</em> &lt; .001), with 0.71 additional QALYs at an incremental cost of $21 378. The incremental CE ratio was $30 251 per QALY, remaining below Korea’s willingness-to-pay threshold of $37 453. Probabilistic sensitivity analysis demonstrated a 70.3% likelihood of CE at this threshold.</div></div><div><h3>Conclusion</h3><div>Upfront ASCT is a cost-effective consolidation strategy for appropriately selected patients with MM aged 65 to 69 years within Korea’s single-payer healthcare system, supporting fitness-based rather than age-based transplant eligibility criteria.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1571-1579"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148303312","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":"Assessing the Agreement Between Self- and Proxy-Reported Responses for Measuring Health-Related Quality of Life in People With Dementia Using the Alzheimer’s Disease 5 Dimensions Instrument","authors":"Syed A. Keramat PhD,&nbsp;Tiet-Hanh Dao-Tran PhD,&nbsp;Kim-Huong Nguyen PhD,&nbsp;Alyssa Welch PhD,&nbsp;Tracy Comans PhD","doi":"10.1016/j.jval.2026.04.011","DOIUrl":"10.1016/j.jval.2026.04.011","url":null,"abstract":"<div><h3>Objectives</h3><div>The level of agreement between utility values derived from self- and proxy reports, assessed using the preference-based health-related quality-of-life (HRQoL) instrument, the Alzheimer’s Disease 5 Dimensions (AD-5D), remains unclear. We aimed to investigate the agreement between self- and proxy-reported HRQoL, measured using the AD-5D, a dementia-specific, preference-based HRQoL instrument.</div></div><div><h3>Methods</h3><div>The data comprise 77 Australian dyads of people with dementia and their caregivers. The agreement between AD-5D utility values derived from self- and proxy reports was analyzed using a Bland-Altman plot. The ordinary least squares regression technique was used to identify factors associated with the AD-5D utility value sets and to assess agreement between the resulting AD-5D utility values derived from self- and proxy reports.</div></div><div><h3>Results</h3><div>The mean AD-5D utility value derived from self-reports (0.667) was higher than the value derived from proxy reports (0.523). The Bland-Altman plot shows that 7.79% of the differences in AD-5D utility values fell outside the limits of agreement. The regression results indicated that the AD-5D utility value derived from self-reports for people with dementia aged 80 years or older was, on average, 0.20 points lower (β = 0.20, standard error = 0.10) than that for people with dementia aged less than 70 years.</div></div><div><h3>Conclusions</h3><div>Utility values derived from self-reports were higher than those derived from caregiver proxy reports. Although proxy reporting is a necessary alternative when self-reporting is not feasible, these perspectives are not interchangeable. Future economic evaluations should incorporate sensitivity analyses to account for this systematic “proxy gap.”</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1663-1671"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147960328","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Can Large Language Models Predict Patient Treatment Choices? A Discrete Choice Experiment Framework","authors":"Tina Cheng MPH,&nbsp;Juan Marcos Gonzalez PhD,&nbsp;Matthew M. Engelhard MD, PhD,&nbsp;Shelby D. Reed PhD,&nbsp;Semra Ozdemir PhD","doi":"10.1016/j.jval.2026.04.006","DOIUrl":"10.1016/j.jval.2026.04.006","url":null,"abstract":"<div><h3>Objectives</h3><div>This study evaluated the viability of large language models, specifically GPT-4, in predicting patient health preference-consistent choices using a discrete choice experiment framework.</div></div><div><h3>Methods</h3><div>Synthetic data were generated from real discrete choice experiment responses by patients with a history of cancer. Analytical data included 50 synthetic patients, each answering 48 2-alternative treatment choice questions varying in expected survival, chance of long-term survival, health limitations, and out-of-pocket cost. GPT-4’s predictive performance was assessed across 4 experiments. In experiments 1 and 2, GPT-4 predicted 20 hold-out questions (ie, new choice questions) leveraging 28 fixed (experiment 1) or randomly selected (experiment 2) sample questions. Experiment 3 varied the number of sample questions to examine prediction accuracy and prediction confidence. Experiment 4 evaluated how characteristics of the hold-out questions influenced prediction accuracy.</div></div><div><h3>Results</h3><div>GPT-4 achieved an average prediction accuracy of 70.5% (95% confidence interval [CI]: 68.3%-72.7%) in experiment 1 and 69.9% (95% CI: 66.9%-72.9%) in experiment 2, with greater variability when sample questions were randomized. Experiment 3 revealed a learning curve, in which accuracy improved from 53% with 5 sample questions to 64% with 10, after which performance plateaued. Experiment 4 showed higher prediction accuracy for questions with more salient attribute differences.</div></div><div><h3>Conclusions</h3><div>GPT-4 demonstrated the ability to infer patient preferences from limited samples, achieving accuracy levels comparable to surrogate decision makers. Its performance remained consistent across randomized input sequences and improved as the number of sample questions increased, eventually reaching a plateau where additional training yielded diminishing returns.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1698-1705"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147843329","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":"Critical Comments by the European Medicines Agency on Patient-Reported Outcomes in Regulatory Submissions (2020 to 2023)","authors":"Courtney Levy BSc,&nbsp;Sarah Sauchelli PhD,&nbsp;Jessica L. Abel MPH,&nbsp;Mia Wraight BS,&nbsp;Dorothee Oberdhan PhD,&nbsp;Lynda Doward MRes,&nbsp;Ari Gnanasakthy MSc, MBA","doi":"10.1016/j.jval.2026.04.017","DOIUrl":"10.1016/j.jval.2026.04.017","url":null,"abstract":"<div><h3>Objectives</h3><div>Inclusion of patient-reported outcome (PRO) data in Summaries of Product Characteristics (SmPCs) and package leaflets published by the European Medicines Agency (EMA) is critical to communicate patient-reported value of new medicines. This study aimed to identify key issues in the incorporation and interpretation of PROs in clinical trials that may hinder approval of PRO-related language in SmPCs/package leaflets.</div></div><div><h3>Methods</h3><div>Indications of new medicines recommended for approval by the EMA between 2020 and 2023 were identified. The Public Assessment Reports, SmPCs, and package leaflets were systematically reviewed. Information pertaining to inclusion of PROs in confirmatory trial(s) and PRO-related reviewer comments were extracted and categorized.</div></div><div><h3>Results</h3><div>Of 167 indications examined, 58 (35%) included PRO-related statements of treatment effect in the SmPC/package leaflet. Critical comments by EMA reviewers focused on study design and PRO data robustness, concerns about statistical analysis, data quality, and PRO measure appropriateness. Such critiques did not always lead to exclusion of PROs from the SmPC, particularly when PRO endpoints were multiplicity-controlled. PRO data collected in unblinded trials—sometimes unavoidable—were often deemed acceptable if multiplicity-controlled.</div></div><div><h3>Conclusions</h3><div>Review findings emphasize that thoughtful selection of PRO measures, their implementation in trial protocol design, and appropriate statistical analysis planning can strengthen the credibility of PRO results in confirmatory trials, which is critical for supporting the approval of PRO-based value messaging. Insights generated may be used by marketing authorization applicants to support a favorable assessment of PRO data by the EMA.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1672-1682"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148319337","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}