Value in Health最新文献

{"title":"An Empirical Comparison of Statistical Methods for Estimating Treatment Effects on EQ-5D in Randomized Clinical Trials","authors":"Jiajun Yan MSc,&nbsp;Brittany Humphries PhD,&nbsp;Menglu Che PhD,&nbsp;Eleanor Pullenayegum PhD,&nbsp;Shun Fu Lee PhD,&nbsp;Feng Xie PhD","doi":"10.1016/j.jval.2026.04.009","DOIUrl":"10.1016/j.jval.2026.04.009","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aimed to empirically compare commonly used statistical models for estimating treatment effects using EuroQol 5-Dimension (EQ-5D) data from randomized clinical trials (RCTs).</div></div><div><h3>Methods</h3><div>We identified eligible RCTs through Vivli using National Clinical Trial numbers. Trials reporting EQ-5D at baseline and at least 2 follow-ups were included. Treatment effects on EQ-5D utilities or EQ Visual Analog Scale (VAS) were estimated as change from baseline both at the final visit and averaged across visits. We compared 4 commonly used models: linear mixed-effects model (LMM), generalized estimating equations (GEE), mixed-effects Tobit model, and mixed-effects Beta model, in terms of model diagnostics on distributional assumptions, agreements in statistical significance (<em>P</em> &lt; .05), and clinical relevance using the minimally important difference.</div></div><div><h3>Results</h3><div>Thirteen RCTs (<em>n</em> = 120-1841) were included. Baseline mean EQ-5D utilities ranged from 0.596 to 0.772, and EQ VAS scores from 44.5 to 75.5.</div><div>For EQ-5D utilities, all 4 models agreed on statistical significance at the final visit. For average effects, 1 comparison in each model differed in statistical significance. LMM and GEE showed 95.7%-100% agreement in clinical relevance, whereas Tobit and Beta showed 3-5 disagreements.</div><div>For EQ VAS, GEE and Tobit agreed on statistical significance in 95.7% of comparisons at the final visit; LMM showed 1 disagreement for average effects. All models agreed on clinical relevance, except for 1 LMM disagreement at the final visit.</div></div><div><h3>Conclusions</h3><div>Our analyses showed high agreement in statistical significance and clinical relevance across models. Considering model diagnostics, robustness, and practical usability, the LMM might be a reasonable and pragmatic option.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1636-1645"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147960286","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":"Evidence and Pricing: Bridging the Comparative Effectiveness Gap in Federal and State Drug Policy","authors":"Victoria W. Dayer PharmD, PhD,&nbsp;Jens Grueger PhD,&nbsp;Lucille Sun PharmD, MS,&nbsp;Blythe J. Adamson PhD, MPH,&nbsp;Douglas Barthold PhD,&nbsp;Kristi Martin MPA,&nbsp;T. Joseph Mattingly II PharmD, MBA, PhD,&nbsp;R. Brett McQueen PhD,&nbsp;Daniel A. Ollendorf PhD, MPH,&nbsp;Julia F. Slejko PhD,&nbsp;Stacey Kowal MSc,&nbsp;Carolina Reyes PhD,&nbsp;Sean D. Sullivan PhD,&nbsp;The CER-to-Policy Expert Collaborative","doi":"10.1016/j.jval.2026.04.014","DOIUrl":"10.1016/j.jval.2026.04.014","url":null,"abstract":"<div><h3>Objectives</h3><div>Federal and state drug pricing programs typically involve a review of comparative clinical evidence to understand the added benefit that interventions provide for the indicated and covered patient population. Contemporary (recent) comparative effectiveness research (CER) can be instrumental in illustrating benefit under conditions of current, real-world use to support these decisions.</div></div><div><h3>Methods</h3><div>We convened 7 subject-matter experts plus 2 expert moderators to discuss gaps, challenges, and opportunities around the use of contemporary CER for federal and state drug pricing programs. We synthesized and structured key recommendations for the use of CER for policy and pricing decision makers.</div></div><div><h3>Results</h3><div>Key gaps and challenges to using contemporary CER to support pricing decisions by the Centers for Medicare and Medicaid Services and state Prescription Drug Affordability Boards are centered around soliciting and generating fit-for-purpose evidence, improving transparency on how evidence is used in decision making, and mitigating resource constraints. Recommendations for policymakers include asking clear questions to solicit necessary data, considering a Population-Intervention-Comparator-Outcomes-Time framework, balancing usefulness with limitations, improving transparency on decision-making, and streamlining processes. Recommendations for manufacturers include leveraging resources, supporting scientific credibility, and tailoring evidence submissions to decision-maker realities.</div></div><div><h3>Conclusions</h3><div>Incorporating contemporary CER is critical for informing policymakers on the value of interventions for drug pricing negotiations. Evaluating evidence, answering research questions, and inferring how evidence applies to different populations are core principles in health economics and health policy, which should be applied more rigorously and transparently in drug pricing programs.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1692-1697"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148035490","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":"Understanding Preference Heterogeneity in Digital Neurorehabilitation: Results of a Discrete Choice Experiment","authors":"Ann-Kathrin Fischer MSc,&nbsp;Andrew Sadler MSc,&nbsp;Thomas Kohlmann Prof Dr phil,&nbsp;Axel Mühlbacher Prof Dr rer oec","doi":"10.1016/j.jval.2026.03.2238","DOIUrl":"10.1016/j.jval.2026.03.2238","url":null,"abstract":"<div><h3>Objectives</h3><div>Increasing demand, rising costs, and limited access pose major challenges for stroke rehabilitation. Digital interventions offer solutions, although acceptance varies. Understanding heterogeneity in preferences and decision consistency is essential.</div></div><div><h3>Methods</h3><div>A discrete choice experiment (<em>N</em> = 1055 after data cleaning) assessed preferences for 7 attributes. Latent class analysis identified preference-based subgroups; a heteroscedastic conditional logit model examined scale heterogeneity.</div></div><div><h3>Results</h3><div>Three classes were identified. Class 1 (21.3%) prioritized low copayment (β_80€ = −3.355; β_0€ = 3.058; <em>P</em> &lt; .001) and showed limited digital readiness, placing less emphasis on technical features. Class 2 (32.2%) focused on therapy success, with large utility differences across levels (β₆₀% = −4.035; β_100% = 3.909; <em>P</em> &lt; .001) and higher digital affinity. Class 3 (46.5%) valued professional contact (β = 0.941; <em>P</em> &lt; .001), location flexibility (β = 0.632; <em>P</em> &lt; .001), and progress feedback (β = 0.558; <em>P</em> &lt; .001), reflecting differentiated engagement preferences. The heteroscedastic conditional logit model showed that satisfaction with health (γ = 0.114; <em>P</em> &lt; .001) and quality of life (γ = 0.043; <em>P</em> &lt; .001) were linked to greater consistency, whereas (very) good health status (γ = −0.177; <em>P</em> &lt; .001) and therapy experience (γ = −0.014; <em>P</em> &lt; .001) corresponded to increased variability.</div></div><div><h3>Conclusion</h3><div>Tailored interventions should reflect distinct preference types to foster acceptance. Cost-sensitive users may need simple formats; digitally engaged individuals benefit from flexible solutions; and those with inconsistent decisions may require clearer guidance or structured onboarding. Conducted in stroke rehabilitation, the study also captured broader rehabilitation experience.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1539-1550"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147676854","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":"Paid and Unpaid Productivity Losses Associated With Musculoskeletal Conditions: Longitudinal Evidence From Australia","authors":"Nanati Legese Alemu MSc, MPH,&nbsp;Anthony Niu PhD,&nbsp;Jennifer J. Watts MComm (Ec),&nbsp;Suzanne Robinson PhD,&nbsp;Gang Chen PhD,&nbsp;Lan Gao PhD","doi":"10.1016/j.jval.2026.04.007","DOIUrl":"10.1016/j.jval.2026.04.007","url":null,"abstract":"<div><h3>Objectives</h3><div>To examine the impact of musculoskeletal conditions on paid and unpaid productivity using longitudinal Australian data.</div></div><div><h3>Methods</h3><div>Data were drawn from 4 waves of the Household, Income, and Labor Dynamics in Australia Survey. Musculoskeletal conditions (MSCs) were identified from self-reported arthritis or osteoporosis, with prescription medication as an indicator of symptom severity. Functional limitation severity was classified using tertiles of the SF-36 Physical Functioning subscale. Correlated random-effects models estimated within- and between-person associations between MSCs and weekly paid, unpaid, and total productivity, adjusting for demographic, socioeconomic, and lifestyle covariates.</div></div><div><h3>Results</h3><div>Overall, 36% of respondents reported an MSC in at least 1 wave. Individuals with MSCs worked 3.2 fewer paid hours per week (95% CI −3.9 to −2.4) but performed 3.1 more unpaid hours (95% CI 2.1 to 4.2), leaving total productivity largely unchanged. Respondents with MSCs but no medication worked 4.7 fewer paid hours (95% CI −5.8 to −3.5) and 4.7 more unpaid hours (95% CI 2.9 to 6.5), whereas those using medication worked 6.8 fewer paid hours (95% CI −7.8 to −5.9) and 3.0 more unpaid hours (95% CI 1.4 to 4.7). At lower physical functioning levels, paid (−12.8 hours) and total productivity (−13.2 hours) declined sharply. Paid-work losses were larger for men, consistent with significant sex differences in the correlated random-effects models.</div></div><div><h3>Conclusions</h3><div>MSCs substantially reduce paid work and partly redirect time into unpaid work. Policies that support labor-force retention—such as early management of functional decline—and recognition of the unpaid role are essential to address the broader productivity burden.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1551-1559"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147857183","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-Effectiveness of Artificial Intelligence in Breast Cancer Screening: An Ethical Perspective on a Complex Issue","authors":"Magnus Dustler PhD,&nbsp;Simon Rosenqvist PhD,&nbsp;Johan Brännmark PhD,&nbsp;Sophia Zackrisson MD, PhD,&nbsp;Andrew D.A. Maidment PhD","doi":"10.1016/j.jval.2026.03.2253","DOIUrl":"10.1016/j.jval.2026.03.2253","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1743-1744"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148200055","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 a Global Longitudinal Strain-Based Screening and Prevention Program for Pre-Heart Failure Compared With Standard of Care","authors":"Hannah Kentenich MSc,&nbsp;Sebastian Kelle MD,&nbsp;Rebecca E. Beyer MD,&nbsp;Maximilian L. Müller MD,&nbsp;Bjoern A. Remppis MD,&nbsp;Tim Friede PhD,&nbsp;Dusan Simic Dipl-Kfm,&nbsp;Stephanie Stock PhD,&nbsp;Arim Shukri Dipl-Math,&nbsp;Dirk Müller PhD","doi":"10.1016/j.jval.2026.04.002","DOIUrl":"10.1016/j.jval.2026.04.002","url":null,"abstract":"<div><h3>Objectives</h3><div>To prevent heart failure (HF), the global longitudinal strain (GLS) emerged as a promising systolic function parameter for targeted pre-HF screening and prevention measures. This analysis investigated the cost-effectiveness of a GLS-based screening and prevention program for pre-HF in a German at-risk population.</div></div><div><h3>Methods</h3><div>A patient-level simulation model was developed from the perspective of the German statutory health insurance. By comparing the lifetime costs and consequences of a GLS-based screening program with those of standard of care without screening, the incremental cost-effectiveness ratio (ICER) was calculated. Input parameters were obtained from HERZCHECK trial data and the literature. Results were presented as costs per quality-adjusted life-year (QALY) and costs per life-year (LY) gained. Deterministic and probabilistic sensitivity analyses examined the robustness of the results, and a model validation was performed. An option value was calculated to reflect the impact of the potential costs and benefits of future innovations.</div></div><div><h3>Results</h3><div>Compared with standard of care, the GLS-based screening program resulted in both additional costs (€7031) and benefits (0.10 QALYs/0.12 LY), with an ICER of €69 543/QALY and €59 552/LY. The probabilistic sensitivity analysis showed that the GLS-based screening program was cost-effective with a probability of 67% at a willingness-to-pay threshold of €100 000/QALY. While screening only men or patients aged ≥60 years decreased the ICER by 25% and 21%, respectively, considering the option value increased the ICER by 59%.</div></div><div><h3>Conclusion</h3><div>Our results indicate that a GLS-based screening program could be offered at an acceptable cost-effectiveness ratio. Specifying the target population may improve the cost-effectiveness.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1560-1570"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147782323","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":"Do Quality-Adjusted-Life-Year-Based Cost-Effectiveness Analyses Discriminate Against Disabled Patients? A Theoretical and Empirical Analysis","authors":"Yue Ma MSc,&nbsp;Peter J. Neumann ScD,&nbsp;David J. Vanness PhD,&nbsp;Hongchao Li PhD,&nbsp;Feng Xie PhD","doi":"10.1016/j.jval.2026.04.005","DOIUrl":"10.1016/j.jval.2026.04.005","url":null,"abstract":"<div><h3>Objectives</h3><div>Quality-adjusted life years (QALYs) have been widely used in cost-effectiveness analyses (CEAs). Nonetheless, critics argue that this measure may inherently discriminate against people with disabilities. This study aims to investigate whether the use of QALYs disadvantages disabled populations through combining theoretical analysis with examination of empirical evidence.</div></div><div><h3>Methods</h3><div>For the theoretical analysis, we constructed 5 scenarios to compare incremental QALYs between disabled and nondisabled patients reflecting different treatment effects on health-related quality of life or survival. We then searched the Tufts CEA Registry to identify published CEAs for selected chronic diseases including rheumatoid arthritis, type 2 diabetes, and chronic kidney disease to match published evidence with these scenarios.</div></div><div><h3>Results</h3><div>Across 5 scenarios, the differences in incremental QALYs between disabled and nondisabled groups were driven by relative changes in utility and life-years, rather than indicating a systematic bias. In 9 of 11 published CEAs, the disabled subgroup achieved greater incremental QALY, lower (more favorable) incremental cost-effectiveness ratios, or dominant results.</div></div><div><h3>Conclusions</h3><div>These findings suggest that, within the theoretical scenarios and empirical contexts examined, QALY-based CEAs do not appear to inherently disadvantage patients with disabilities.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1580-1589"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147821403","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":"A Cost-Utility Analysis of LISTEN: Low-Intensity Mental Health Support via Telehealth-Enabled Network for Diabetes Distress","authors":"Joahna Kevin Perez MHEcon,&nbsp;Mary Lou Chatterton PhD,&nbsp;Harish Raj Narandaran DrPH,&nbsp;Matthew P. Hamilton MSc,&nbsp;Edith Holloway PhD,&nbsp;Jane Speight PhD,&nbsp;Christel Hendrieckx,&nbsp;Cathrine Mihalopoulos PhD","doi":"10.1016/j.jval.2026.03.2250","DOIUrl":"10.1016/j.jval.2026.03.2250","url":null,"abstract":"<div><h3>Objectives</h3><div>This study assessed the cost-effectiveness of Low-Intensity mental health Support via a Telehealth Enabled Network (LISTEN) for adults experiencing diabetes distress, facilitated by diabetes health professionals.</div></div><div><h3>Methods</h3><div>A within-trial cost-utility analysis included 428 participants randomized to either LISTEN (diabetes-specific problem-solving therapy) or usual care (web-based diabetes distress and generic mental health resources). Quality-adjusted life-years (QALYs) were derived using the Assessment of Quality of Life-4D. Costs were measured from health sector and societal perspectives using a self-reported resource use questionnaire. Incremental cost-effectiveness ratios were calculated and nonparametric bootstrapping estimated 95% confidence intervals. Costs were presented in Australian dollars (A$) for the 2021-2022 financial year.</div></div><div><h3>Results</h3><div>The mean direct cost of delivering LISTEN was A$262 per person. Over 6 months, the LISTEN group had higher total QALYs (0.024; 95% confidence intervals [CI] 0.007 to 0.041). Compared with usual care, mean health sector cost (−A$3271; 95% CI −A$5134 to A$1008) and societal cost (−A$3588; 95% CI −A$5913 to A$860) were lower, although these differences were not statistically significant. At a A$50 000 per QALY willingness-to-pay threshold, LISTEN was dominant with a 94% probability of being cost-effective from both perspectives. Findings should be interpreted cautiously given the substantial missing data.</div></div><div><h3>Conclusion</h3><div>LISTEN significantly improves health-related quality of life of adults with diabetes distress and is likely to be cost-effective. Point estimates show lower mean costs alongside QALY gains, but cost differences were not statistically significant.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1590-1598"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147960303","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":"Validation and Clinical Meaningfulness of the 18-item National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy Lymphoma Symptom Index in Patients With Lymphoma","authors":"Kimberly A. Webster MA,&nbsp;Anne R. Zola MSc,&nbsp;Maria Corona MS,&nbsp;Elijah A. Patten MA,&nbsp;Reem Karmali MD,&nbsp;Leo Gordon MD,&nbsp;Shuo Ma MD, PhD,&nbsp;Jane N. Winter MD,&nbsp;Alexander Upton MSc,&nbsp;John Devin Peipert PhD","doi":"10.1016/j.jval.2026.03.2244","DOIUrl":"10.1016/j.jval.2026.03.2244","url":null,"abstract":"<div><h3>Objectives</h3><div>To further evaluate the psychometric properties of the National Comprehensive Cancer Network/Functional Assessment of Cancer Therapy Lymphoma Symptom Index-18 (NFLymSI-18), including estimates of meaningful within-patient change thresholds, to support its ability to reliably and validly measure clinical- and patient-relevant outcomes in diverse research settings.</div></div><div><h3>Methods</h3><div>Data from a longitudinal multicenter clinical trial in patients with indolent B cell lymphoma and a single-institution observational study of patients with Hodgkin’s and non-Hodgkin’s lymphoma were examined using inferential statistics. Available data were analyzed separately and then used collectively to summarize evidence of NFLymSI-18 reliability and validity. Effect sizes for known-groups comparisons and responsiveness analyses were calculated to determine meaningful score thresholds.</div></div><div><h3>Results</h3><div>Analyses indicated that the NFLymSI-18 demonstrated good internal consistency reliability (Cronbach’s alpha &gt; 0.70) for all 3 subscales (with 2 of them &gt; 0.80), and excellent (&gt; 0.90) for the NFLymSI-18 Total in the observational study. Stability (test-retest reliability) exceeded preset thresholds for good reliability for total score and all but one subscale. Known-groups analysis confirmed construct validity, often with moderate-to-large effect sizes, particularly between categories of performance status ratings in the observational study (effect size between −0.33 to −1.06). Responsiveness to changes in clinical status was significant, indicating a sensitivity to within-patient clinical changes. Clinically meaningful individual patient change thresholds were estimated to be 5 to 6 points for the NFLymSI-18 Total score and 2-4 points for the Disease-Related Symptoms-Physical score.</div></div><div><h3>Conclusion</h3><div>The NFLymSI-18 is reliable, valid, and responsive and includes suggested values for individual patient change when planning lymphoma clinical trials.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1683-1691"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147723711","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":"Validity, Reliability, and Responsiveness of the EQ-5D in Hematological Cancers: A Systematic Review of Measurement Properties","authors":"Aleksandra Dudzisz MSc,&nbsp;Grzegorz W. Basak MD,&nbsp;Emilian Snarski MD,&nbsp;Dominik Golicki MD","doi":"10.1016/j.jval.2026.03.2239","DOIUrl":"10.1016/j.jval.2026.03.2239","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aimed to conduct a systematic review of the published evidence regarding the measurement properties of the EuroQoL 5-Dimension (EQ-5D) (3 level [EQ-5D-3L] and 5 level [EQ-5D-5L]) in adult patients with hematological cancers.</div></div><div><h3>Methods</h3><div>A comprehensive literature search was carried out utilizing the MEDLINE, EMBASE, and Web of Science (up to October 2025). Full-text English articles focusing on original research concerning the measurement properties of the EQ-5D, including construct validity, test-retest reliability, or responsiveness, were included. Studies involving experimental versions of the EQ-5D were excluded. The quality assessment utilized the COSMIN Risk of Bias checklist, and data synthesis followed the COSMIN methodology.</div></div><div><h3>Results</h3><div>A total of 16 studies from 31 countries, presenting 996 individual tests (96% met results) of the EQ-5D measurement properties were included. Sufficient construct validity for EQ-5D-5L index, EQ-5D-5L dimensions, EQ-5D-3L index, EQ-5D-3L dimensions, and EQ visual analog scale (VAS) was supported by high certainty. Evidence regarding test-retest reliability was limited to EQ-5D-5L index and EQ VAS, had moderate and low certainty, and indicated sufficient overall rating of measurement properties. Responsiveness was assessed in 104 tests (100 for EQ-5D-5L index, 2 for EQ-5D-3L index, 2 for EQ VAS). Most tests demonstrated the expected directional change (97%; 101/104).</div></div><div><h3>Conclusion</h3><div>This systematic review provides an overview of the measurement properties of the EQ-5D as a health-related quality-of-life measure in hematological cancers. The available evidence suggests that both EQ-5D-3L and EQ-5D-5L have sufficient construct validity and may be used to assess health-related quality of life in this patient population. Evidence for reliability and responsiveness remains limited and warrants further investigation.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 9","pages":"Pages 1653-1662"},"PeriodicalIF":6.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147692575","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}