Value in Health最新文献

{"title":"Evaluating the cost-effectiveness of a conservative approach to oxygen therapy for invasively ventilated adults in intensive care: the UK-ROX trial.","authors":"Zhengnan Zhu, Daniel Martin, Alex Cowden, Doug Gould, Michael P W Grocott, Tasnin Shahid, David Harrison, Kathryn M Rowan, Paul Mouncey, Zia Sadique","doi":"10.1016/j.jval.2026.03.2267","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2267","url":null,"abstract":"<p><strong>Objectives: </strong>There is a lack of economic evaluation evidence on the optimal oxygen dosing level for mechanically ventilated critically ill patients. This study reports the health economic outcomes of a large-scale randomized controlled trial (RCT), UK-ROX, and conducts a full cost-effectiveness analysis of conservative versus usual oxygen therapy.</p><p><strong>Methods: </strong>All-cause mortality, health related quality of life (HrQoL), quality-adjusted life-years (QALYs), costs, and incremental net monetary benefit (INB) were assessed at 90 days post-randomization. HrQoL was derived from information collected via an EQ-5D-5L questionnaire using an appropriate mapping function. Total healthcare costs include resource use that occurred in adult ICUs, general wards, and follow-up health services. The cost effectiveness analysis (CEA) used regression methods to report the cost-effectiveness and summarised the uncertainties around the incremental cost-effectiveness results.</p><p><strong>Results: </strong>After adjustment for prespecified baseline variables, the estimated incremental costs of conservative oxygen therapy versus usual oxygen therapy were -£968 (95% CI -£2279 to £342). The adjusted mean differences in EQ-5D utility score and life-years were 0.004 (95% CI -0.016 to 0.025) and -0.003 (95% CI -0.006 to 0.001), and incremental QALYs were 0.001 (95% CI -0.002 to 0.003). As a result, the mean INB of conservative oxygen therapy was £979 (95% CI -£334 to £2292).</p><p><strong>Conclusions: </strong>Conservative and usual oxygen therapy resulted in similar QALYs, and while conservative therapy appeared cost-saving with positive INBs, both estimates have uncertainty.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149115454","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":"Evaluating Treatment Sequences in Major Depressive Disorder Using an Open-Source Health Economic Model Based on Stakeholder and Patient Engagement","authors":"Alice Kate Cummings Joyner MS,&nbsp;Richard H. Chapman PhD,&nbsp;Yuan-Yuan Michelle Cheng MS,&nbsp;Richard Xie PhD,&nbsp;Larragem Parsley-Raines MS,&nbsp;Jennifer Bright MPA,&nbsp;Warren Stevens PhD","doi":"10.1016/j.jval.2026.05.005","DOIUrl":"10.1016/j.jval.2026.05.005","url":null,"abstract":"<div><h3>Objectives</h3><div>This study developed a patient-level discrete event simulation model to assess value of treatment sequences for newly diagnosed adult patients with major depressive disorder.</div></div><div><h3>Methods</h3><div>Model specifications were informed by a continual stakeholder and patient engagement approach, literature, and key findings from claims analysis. Key model outputs included time to response, remission rates, net monetary benefit, and cost-effectiveness. This open-source model attempts to capture the patient journey by modeling treatment pathways (vs individual treatments), periods with no treatment, and patient heterogeneity.</div></div><div><h3>Results</h3><div>The base-case analysis evaluates key outcomes from 5 treatment pathways over 5 years. Results show that all sequences of solo or combination treatments are superior to no treatment in terms of total quality-adjusted life-years (QALYs) accrued and net monetary benefit. Treatment with selective serotonin reuptake inhibitors (SSRI) across all 4 lines of therapy dominated no treatment. Treatment with SSRI as the first line, followed by serotonin-norepinephrine reuptake inhibitors (SNRI) as the second line, SNRI plus atypical antidepressant as the third line, and SNRI plus antipsychotic as the fourth line resulted in higher costs but also higher QALYs relative to 4 lines of SSRI, with an incremental cost-effectiveness ratio of approximately $28 000/QALY. In contrast, SSRI first-line treatment followed by SSRI plus intensive psychotherapy for second through fourth lines were not found to be cost-effective because of small QALY gains and high cost owing to the number of psychotherapy sessions modeled here.</div></div><div><h3>Conclusions</h3><div>Understanding drivers of effective treatment across diverse patient journeys is crucial to optimizing care. This model offers insights into relative value of treatment strategies aligned with real-world experiences, supporting more informed decision making and improved outcomes.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1805-1813"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148176955","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":"Longitudinal Modeling and Simulation of Growth Hormone Efficacy in Children With Idiopathic Short Stature","authors":"Hyojin Cho PharmD, MS,&nbsp;Hyeseon Jeon BPharm, MS,&nbsp;Chaeri Kang PharmD, MS,&nbsp;Jihyun Jeon PhD,&nbsp;Woojin Jung BPharm, PhD,&nbsp;Hwi-yeol Yun PhD,&nbsp;Mi Ran Lee PharmD, MS,&nbsp;Soyoung Lee PharmD, PhD,&nbsp;Yoo-Mi Kim MD, PhD,&nbsp;Jung-woo Chae PharmD, PhD","doi":"10.1016/j.jval.2026.05.006","DOIUrl":"10.1016/j.jval.2026.05.006","url":null,"abstract":"<div><h3>Objectives</h3><div>Growth hormone (GH) has been widely used to treat idiopathic short stature (ISS), defined as a height that falls below the third percentile or 2 standard deviations below the mean for age and sex. This study aimed to evaluate the long-term effects of GH on relative growth response in children with ISS using a nonlinear mixed-effects (NLME) model for longitudinal data and to support personalized medicine through predictive modeling approach.</div></div><div><h3>Methods</h3><div>A total of 91 prepubertal patients with ISS were included in this study. The height percentile adjusted for age and sex was used as the outcome to reflect physiological growth, and the cumulative GH dose (mg/kg) was used as the exposure metric. A Gompertz-based NLME model was fitted to describe the dose-response relationship, and stepwise covariate modeling identified significant baseline predictors. The model performance was assessed using visual predictive checks and goodness-of-fit plots.</div></div><div><h3>Results</h3><div>The final model estimated a baseline height percentile of 0.63 ± 0.90 and an upper growth limit of 17.2 ± 11.1. Body mass index, insulin-like growth factor-binding protein 3, and paternal height were significant covariates influencing the total growth.</div></div><div><h3>Conclusion</h3><div>This study demonstrated the utility of NLME modeling for quantifying the treatment response to GH in patients with ISS. The developed model was integrated into a web-based tool, allowing clinicians to predict individualized growth trajectories and optimize GH treatment strategies in real-world practice.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1760-1768"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148537032","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":"Developing Meaningful Score Differences for the Bayley-4 and Vineland-3 in Angelman Syndrome Using a Delphi Panel","authors":"Sonya Powers PhD,&nbsp;Katherine N. Anderson BA,&nbsp;Wen-Hann Tan BMBS,&nbsp;Julian Tillmann PhD,&nbsp;Mark Daniel PhD,&nbsp;Audrey Thurm PhD,&nbsp;Cristan Farmer PhD,&nbsp;Susanne Clinch PhD,&nbsp;Anne C. Wheeler PhD,&nbsp;Anjali Sadhwani PhD","doi":"10.1016/j.jval.2025.11.021","DOIUrl":"10.1016/j.jval.2025.11.021","url":null,"abstract":"<div><h3>Objectives</h3><div>This study aimed to develop within-patient meaningful score differences (MSDs) on the Bayley Scales of Infant Development, Fourth Edition (Bayley-4), and the Vineland Adaptive Behavior Scales, Third Edition (Vineland-3), for individuals with Angelman syndrome (AS).</div></div><div><h3>Methods</h3><div>A Delphi method, involving a panel of 19 caregivers of individuals with AS, was used to establish MSDs for Bayley-4 and Vineland-3 growth scale values. MSD was defined as the smallest change that would noticeably affect the daily functioning of an individual with AS or family quality of life in a way that was important to the individual with AS or their family. For each subscale of the Bayley-4 and Vineland-3, the panel was presented with 2 to 4 vignettes describing varying levels of baseline functioning and asked to select an MSD from a range of potential values. An iterative process involving 3 rounds of ratings and 2 rounds of discussion was used to build consensus. The median caregiver rating from round 3 was retained as the final recommended MSD value for each vignette.</div></div><div><h3>Results</h3><div>Final MSD ratings for the 5 subscales of Bayley-4 and 10 subscales of the Vineland-3 had an agreement rate of 70% or higher. MSD thresholds for each subscale were not single cutoffs, but rather reflected a range of MSD values dependent on the level of baseline functioning.</div></div><div><h3>Conclusions</h3><div>The Delphi panel method incorporates the caregiver perspective to provide preliminary estimates of what constitutes meaningful within-person change on the Bayley-4 and Vineland-3 in individuals with AS with various levels of baseline functioning.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1781-1790"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145918497","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":"Introducing Risk Stratification Reduces Patient Burden and Improves Cost-Effectiveness of Hepatocellular Carcinoma Surveillance for People With Metabolic Dysfunction-Associated Steatotic Liver Disease: A Modeling Study","authors":"Joachim Worthington PhD,&nbsp;Anna Kelly,&nbsp;Emily He MPH, PhD,&nbsp;Jacob George MBBS, PhD,&nbsp;Leon A. Adams MBBS, PhD,&nbsp;Eleonora Feletto PhD","doi":"10.1016/j.jval.2026.06.018","DOIUrl":"10.1016/j.jval.2026.06.018","url":null,"abstract":"<div><h3>Objectives</h3><div>As the burden of hepatocellular carcinoma (HCC, the most common form of liver cancer) associated with metabolic dysfunction-associated steatotic liver disease (MASLD/metabolic-associated fatty liver disease) increases, there is a renewed focus on early detection via routine surveillance. However, a third of Australians have metabolic dysfunction-associated steatotic liver disease/metabolic-associated fatty liver disease because of high overweight/obesity rates, with varying liver disease and HCC risk. With such a large at-risk population, it is crucial that interventions are well targeted for those at highest risk to remain cost-effective. Affordable biomarker testing can identify those at highest risk, focusing efforts on those most likely to benefit.</div></div><div><h3>Methods</h3><div>We used the Policy1-Liver model of liver disease and cancer to evaluate the health impacts and cost-effectiveness of routine HCC surveillance. We assessed the benefits of risk stratification using FIB-4 biomarker liver disease score. Outcomes including HCC incidence and mortality, quality-adjusted life-years, costs, and cost-effectiveness were estimated.</div></div><div><h3>Results</h3><div>Routine surveillance reduced HCC mortality by 19% to 27% compared with no surveillance, but without risk stratification, it was not cost-effective for patients with early-stage liver fibrosis or no steatohepatitis. Incorporating FIB-4 risk stratification for inclusion in surveillance maintained health benefits while reducing costs and patient burden, with a cost-effectiveness under $30 000/quality-adjusted life-year at all cutoffs above 1.10. For example, routine surveillance for all individuals with a baseline FIB-4 above 2.67 had an incremental cost-effectiveness of $15 055/quality-adjusted life-year saved.</div></div><div><h3>Conclusion</h3><div>Risk stratification can improve the efficiency of routine HCC surveillance by focusing efforts on those at highest risk. As the burden of MASLD continues to grow, targeted intervention can be guided by predictive modeling.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1825-1835"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148449595","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":"Estimating EQ-5D-5L Quality-Adjusted Life-Year Weights From 12-item General Health Questionnaire Mental Health Data: A Large Sample Exploration of 4 Mapping Methods","authors":"Zhuxin Mao PhD,&nbsp;Joke Bilcke PhD,&nbsp;Koen Pepermans PhD,&nbsp;Philippe Beutels PhD","doi":"10.1016/j.jval.2026.05.001","DOIUrl":"10.1016/j.jval.2026.05.001","url":null,"abstract":"<div><h3>Objectives</h3><div>Mental health measures are common in population surveys but cannot be directly used for utility-based economic evaluations. This study explores methods to estimate health utility associated with mental health.</div></div><div><h3>Methods</h3><div>We used data from 12 701 respondents participating in a Belgian population survey. We compared direct methods (linear and inflated beta regression) that could directly generate utility values, with indirect methods that first estimate EuroQol 5-Dimension 5-Level instrument (EQ-5D-5L) dimension responses using non-parametric or ordered logistic regression before generating utilities. Regression models used 12-item General Health Questionnaire (GHQ-12) responses as independent variables, controlling for sociodemographic factors. Model performance was assessed using root mean-squared error and mean absolute errors. We also calculated error statistics stratified by utility quantiles.</div></div><div><h3>Results</h3><div>For overall model performance, root mean-squared error values were identical for the linear and ordered logistic models (0.142), higher for the beta model (0.148), and highest for the nonparametric approach (0.155). Ordered logistic models performed the best in mean absolute errors, followed by linear, beta, and nonparametric approaches. Stratified analyses revealed heterogeneity in prediction accuracy across the utility quantiles. Models showed poorer performance in the lowest utility quantiles. Indirect mapping is conceptually more robust because it aligns with the dimensional structure of EQ-5D-5L and minimizes variations associated with the use of different value sets.</div></div><div><h3>Conclusion</h3><div>This study provides up-to-date algorithms for mapping mental health data to health utility. The mapping models can be applied using GHQ-12 responses and basic sociodemographic variables to generate utility values for use in economic evaluations in which utility data are not available.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1888-1895"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148017653","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 Monetary Value of Health and Health Behaviors in Japan: A Well-Being Valuation Approach","authors":"Kenjiro Kawaguchi MD, MPH, PhD,&nbsp;Hiroki Takeuchi PhD,&nbsp;Atsushi Nakagomi MD, PhD,&nbsp;Kazushige Ide PhD,&nbsp;Katsunori Kondo MD, PhD","doi":"10.1016/j.jval.2026.05.008","DOIUrl":"10.1016/j.jval.2026.05.008","url":null,"abstract":"<div><h3>Objectives</h3><div>To estimate monetary values of health and health behavior outcomes and 1 well-being-adjusted life-year (WELLBY) in Japan using the well-being valuation approach for cross-sector policy evaluation and social return on investment analysis.</div></div><div><h3>Methods</h3><div>We analyzed data from a nationwide Internet-based survey conducted between February and March 2025, including 14 736 adults aged 20 to 79 years. One WELLBY was defined as a 1-point change in life satisfaction on a 0 to 10 scale sustained for 1 year. Using life satisfaction as the primary well-being measure, we estimated the income-subjective well-being gradient using 2-stage least squares with parental education as an instrumental variable. We examined associations between subjective well-being and 19 outcomes across mental health, social health, health behaviors, health literacy, and women’s health. We calculated compensating surplus values in Japanese yen anchored to the median equivalized household income.</div></div><div><h3>Results</h3><div>The instrumental variables were strong, and the coefficient of log income on life satisfaction was 2.27. Of the 19 outcomes examined, 18 were significantly associated with subjective well-being (<em>P</em> &lt; .001); cognitive restraint (TFEQ-CR) was the sole nonsignificant outcome. Monetary values per 1-point increase included ¥120 000 for the Lubben Social Network Scale-6 and −¥226 000 for the Patient Health Questionnaire-9. A 1-point increase in loneliness corresponded to a welfare loss of −¥513 000. The estimated value of 1 WELLBY was approximately ¥1.51 million (∼US$10 000).</div></div><div><h3>Conclusions</h3><div>This study provides Japan’s first yen-denominated set of monetary values for health-related outcomes and WELLBY, enabling cost-benefit analysis and social return on investment across sectors.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1878-1887"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148240110","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":"Interpreting the Economic Value of Docetaxel, Platinum, and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma: The Importance of Decision Context, Utility Timing, and Treatment Burden","authors":"Runji Zhou MB,&nbsp;Yuanyuan Peng MB,&nbsp;Qiming Liu MB,&nbsp;Jiaxuan Zhou MB","doi":"10.1016/j.jval.2026.03.2265","DOIUrl":"10.1016/j.jval.2026.03.2265","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1930-1931"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148319397","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 Remote Cognitive-Behavioral-Based Therapy for Chronic Pain Among People With High-Impact Chronic Pain","authors":"John F. Dickerson PhD,&nbsp;Maureen O’Keeffe-Rosetti MS,&nbsp;Meghan Mayhew MPH,&nbsp;Andrea J. Cook PhD,&nbsp;Robert D. Wellman MS,&nbsp;Francis J. Keefe PhD,&nbsp;Christine Rini PhD,&nbsp;Ashli A. Owen-Smith PhD, SM,&nbsp;Michael Von Korff ScD,&nbsp;Lynn L. DeBar PhD","doi":"10.1016/j.jval.2026.05.010","DOIUrl":"10.1016/j.jval.2026.05.010","url":null,"abstract":"<div><h3>Objectives</h3><div>Assess cost-effectiveness from the health plan perspective comparing 2 remote cognitive-behavioral-therapy-for-chronic-pain-based interventions (1) pain TRAINER online program and (2) health coach-led telehealth program relative to usual care (UC) and each other.</div></div><div><h3>Methods</h3><div>Cost-effectiveness analysis includes <em>N</em> = 1725 individuals with high-impact chronic pain from 3 Kaiser Permanente sites of RESOLVE randomized clinical trial. Costs were calculated for all healthcare and pain-related services. Clinical outcomes at 1-year included: quality-adjusted life-years (QALY) and minimal clinically important difference (MCID) in pain severity score (≥30% improvement).</div></div><div><h3>Results</h3><div>Both cognitive-behavioral-therapy-for-chronic-pain-based interventions had significantly higher QALYs (painTRAINER: Adj Diff. = 0.035, <em>P</em> &lt; .001; health coach: Adj Diff. = 0.041, <em>P</em> &lt; .001) and proportion achieving MCID (painTRAINER: Adj Diff. = 14.1%, <em>P</em> &lt; .001; health coach: Adj Diff. = 12.2%, <em>P</em> &lt; .001) than UC. painTRAINER was dominant relative to UC (ie, less expensive and more effective) for both QALYs and MCIDs. The incremental cost-effectiveness ratio for health coach vs UC was $19 009 per QALY and $6267 per MCID. The incremental cost-effectiveness ratio for health coach vs painTRAINER was $244 446 per QALY. Net benefit regression identified painTRAINER as the cost-effective choice across all willingness-to-pay levels. Sensitivity analyses, excluding overly influential outliers, demonstrated consistent results, and that health coach vs painTRAINER was cost-effective above willingness-to-pay of $120 000 per QALY.</div></div><div><h3>Conclusion</h3><div>Analyses showed that both the painTRAINER and health coach programs are highly cost-effective relative to UC. Compared with each other, painTRAINER is more cost-effective than the health coach program.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1814-1824"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148253473","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":"Caregiver Meaningful Score Differences and Meaningful Score Regions for the Observer-Reported Communication Ability Measure for Individuals with Angelman Syndrome","authors":"Abigail Rader MS,&nbsp;Li Lin MS,&nbsp;Anjali Sadhwani PhD,&nbsp;Nicole Lucas,&nbsp;Harrison N. Jones PhD,&nbsp;Allyson Berent DVM, Dipl. ACVIM,&nbsp;Batsheva Friedman MA,&nbsp;Kelly Gordon MS, CCC-SLP,&nbsp;Wen-Hann Tan,&nbsp;Bryce B. Reeve PhD","doi":"10.1016/j.jval.2026.02.002","DOIUrl":"10.1016/j.jval.2026.02.002","url":null,"abstract":"<div><h3>Objectives</h3><div>This study presents a novel method for estimating meaningful score differences (MSDs) and meaningful score regions (MSRs) for the Observer-Reported Communication Ability (ORCA) Measure for individuals with Angelman syndrome (AS).</div></div><div><h3>Methods</h3><div>The AS Natural History Study collected longitudinal data on an individual’s communication abilities on the ORCA Measure, Vineland Adaptive Behavior Scales-3, and the Bayley Scales of Infant and Toddler Development-4. In a separate Delphi-based study, caregivers provided MSDs and MSRs for the Vineland-3 and Bayley-4 scores. Leveraging the strong correlations between ORCA and these measures, mixed models with random intercepts were used to estimate ORCA MSDs and MSRs based on the previously derived Vineland-3/Bayley-4 MSDs and MSRs.</div></div><div><h3>Results</h3><div>The ORCA Measure was completed by 192 caregivers, with 113 also completing the Bayley-4 and 159 completing the Vineland-3. ORCA T-score MSRs for low, intermediate, and high communication ability were estimated for expressive, receptive, and pragmatic communication domains using the Vineland-3 and Bayley-4 previously derived MSDs and MSRs. ORCA MSDs varied depending on the MSR and ranged from 0.38-4.55 (based on Vineland-3) and 1.79-4.62 (based on Bayley-4).</div></div><div><h3>Conclusions</h3><div>We present a novel anchor-based methodological approach for deriving MSD and MSR thresholds for clinical outcome assessments. Although these MSD and MSR estimates for the ORCA measure are preliminary, our methods offer a strategy for establishing meaningful change thresholds in rare disease populations in which traditional longitudinal methods may be challenging to implement.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 10","pages":"Pages 1791-1799"},"PeriodicalIF":6.2,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147309913","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}