Value in Health最新文献

{"title":"Author Reply.","authors":"Xujun Gu, Julia Slejko, Eberechukwu Onukwugha","doi":"10.1016/j.jval.2026.03.2246","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2246","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147782300","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 GLS-based screening and prevention program for pre-heart failure compared to standard of care.","authors":"Hannah Kentenich, Sebastian Kelle, Rebecca E Beyer, Maximilian L Müller, Bjoern A Remppis, Tim Friede, Dusan Simic, Stephanie Stock, Arim Shukri, Dirk Müller","doi":"10.1016/j.jval.2026.04.002","DOIUrl":"https://doi.org/10.1016/j.jval.2026.04.002","url":null,"abstract":"<p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>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 to those of standard of care (SoC) 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 (PSA) examined the robustness of the results, and a model validation was performed. An option value (OV) was calculated to reflect the impact of the potential costs and benefits of future innovations.</p><p><strong>Results: </strong>Compared to SoC, the GLS-based screening program resulted in both additional costs (€7,031) and benefits (0.10 QALYs/0.12 LY), with an ICER of €69,543/QALY and €59,552/LY. The PSA 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 OV increased the ICER by 59%.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-21","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The use of baseline risk in cost-effectiveness modelling of competing interventions.","authors":"Tasnim Hamza, Konstantina Chalkou, Fabio Pellegrini, Johannes Lorscheider, Jens Kuhle, Niklaus Meier, Mark Pletscher, Matthias Egger, Georgia Salanti","doi":"10.1016/j.jval.2026.03.2245","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2245","url":null,"abstract":"<p><strong>Objectives: </strong>Network meta-analysis (NMA) with individual participant data can estimate how treatment effects change with patient characteristics. Yet cost-effectiveness analyses typically use population-average effects. We introduce a framework that incorporates NMA-derived heterogeneous treatment effects into cost-effectiveness analysis using a risk-modelling approach.</p><p><strong>Methods: </strong>We first derived a baseline risk score for each patient using a prognostic model. This risk score was then used as effect modifier in a network meta-regression to estimate risk-specific treatment effects. These effects were incorporated into the cost-effectiveness model to estimate the incremental cost-effectiveness ratios (ICERs) and net monetary benefits (NMBs) as functions of the baseline risk score. We demonstrated the approach using data from observational and randomized studies in relapsing-remitting multiple sclerosis, comparing dimethyl fumarate, glatiramer acetate, and placebo.</p><p><strong>Results: </strong>Risk-dependent treatment effects from the prediction-NMR framework led to substantial variation in cost-effectiveness across the baseline risk distribution. When these treatment effects were incorporated into the cost-effectiveness model, ICERs increased steadily across baseline risk quintiles, from 48,811 CHF/QALY in the lowest-risk group to 212,870 CHF/QALY in the highest. Dimethyl fumarate has a higher NMB up to a baseline risk of 55%, after which glatiramer acetate becomes the preferred option.</p><p><strong>Conclusions: </strong>Our findings show that integrating baseline risk modelling with NMA and cost-effectiveness analysis provides more informative decision-making than relying on average effects. Treatment value can vary substantially across the risk spectrum, indicating that optimal therapy selection is strongly dependent on individual patient risk.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147782397","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":"Validation and Clinical Meaningfulness of the 18-item National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy (NCCN-FACT) Lymphoma Symptom Index (NFLymSI-18) in Patients with Lymphoma.","authors":"Kimberly A Webster, Anne R Zola, Maria Corona, Elijah A Patten, Reem Karmali, Leo Gordon, Shuo Ma, Jane N Winter, Alexander Upton, John Devin Peipert","doi":"10.1016/j.jval.2026.03.2244","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2244","url":null,"abstract":"<p><strong>Objectives: </strong>To further evaluate the psychometric properties of the National Comprehensive Cancer Network-Functional Assessment of Cancer Therapy (NCCN-FACT) 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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>Analyses indicated that the NFLymSI-18 demonstrated good internal consistency reliability (Cronbach's alpha >0.70) for all three subscales (with two >0.80), and excellent (>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 5-6 points for the NFLymSI-18 Total score and 2-4 points for the Disease-Related Symptoms - Physical (DRS-P) score.</p><p><strong>Conclusion: </strong>The NFLymSI-18 is reliable, valid, and responsive, and includes suggested values for individual patient change when planning lymphoma clinical trials.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-17","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":"Evaluating Policy Options for New Treatments Under Uncertain External Evidence.","authors":"Yizhi Liang, Boshen Jiao","doi":"10.1016/j.jval.2026.03.2240","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2240","url":null,"abstract":"<p><strong>Background: </strong>Health policymakers in many jurisdictions must decide whether to adopt new medical treatments based on clinical evidence generated abroad, despite uncertainty about the applicability of those results to local populations. This creates a fundamental trade-off: enabling early access to promising therapies versus delaying adoption until locally relevant evidence becomes available.</p><p><strong>Methods: </strong>We evaluate four policy options under uncertain external clinical evidence: delayed adoption pending a local confirmatory trial, conditional adoption with a standard trial requirement, immediate adoption without a trial, and immediate rejection. To compare these strategies, we extend a Bayesian value-of-information framework using a power prior. The power-prior parameter determines the weight assigned to external evidence based on its applicability to the local population. The framework distinguishes pre- and post-confirmation periods and accounts for potential delays in trial completion when conditional approval is granted. We illustrate the approach using a lung cancer therapy trialed exclusively in China and considered for use in the United States.</p><p><strong>Results: </strong>The case study shows that optimal policy depends jointly on uncertainty about external evidence applicability, trial duration, delays in trial completion, and price. High uncertainty favors delayed adoption, while longer trial durations make conditional adoption more attractive by increasing the cost of waiting. Longer delays in trial completion can shift the optimal policy toward delayed adoption. Price reductions shift the policy frontier toward earlier access.</p><p><strong>Conclusions: </strong>Policies for treatments supported by external evidence should be tailored to the evidentiary context, accounting for applicability uncertainty, trial timelines, delays in trial completion, and pricing.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147718189","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":"Impact of Smoothing and Modeling Approach on Quality-Adjusted Life Expectancy Estimates.","authors":"Hosein Ameri, Thomas G Poder","doi":"10.1016/j.jval.2026.03.2241","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2241","url":null,"abstract":"<p><strong>Background: </strong>Quality-adjusted life expectancy (QALE) is a composite indicator integrating life expectancy and health utility. Most studies have used the Sullivan method to calculate QALE, while 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 four methodological approaches: Sullivan versus Markov modeling, each with and without cubic polynomial fit of age-specific utilities.</p><p><strong>Methods: </strong>We analyzed 4,803 EQ-5D-5L records from 2016-2024 health surveys and pooled 2021-2023 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 two-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 one-sample t-test.</p><p><strong>Results: </strong>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 one-sample t-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, p < 0.001), 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.</p><p><strong>Conclusions: </strong>These findings support the use of either method for population health assessment and health technology evaluation, as both produce valid and reliable QALE estimates.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-16","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A systematic review of quantitative health preference methods to support value clarification and shared-decision making: A report of an ISPOR special interest group.","authors":"Janine van Til, Holly L Peay, Ahmed Osman, Angie Botto-van Bemden, Caitlin Thomas, Carina Oedingen, Caroline Vass, Elise Schoefs, Laurie Batchelder, Semra Ozdemir, Christine Poulos","doi":"10.1016/j.jval.2026.03.2243","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2243","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to explore the design and outcomes of preference-based value clarification methods (Pb-VCM) used in the context of supporting patients' clinical decision making to understand whether guidance is used in Pb-VCM development and which Pb-VCM is most effective.</p><p><strong>Methods: </strong>In April of 2023, PubMed, Scopus, and Web of Science were searched for studies reporting the development and/or evaluation of Pb-VCM without restrictions on publication date. Two reviewers independently extracted data including: context and clinical decisions, study methods, sample, Pb-VCM design and development, type of preference information, and measurement of outcomes, such as feasibility/acceptability and effectiveness. Data were synthesized using descriptive and narrative analyses.</p><p><strong>Results: </strong>Of 3,207 abstracts screened, 50 studies were included: 28 descriptive, 9 observational, and 13 experimental. The most common Pb-VCM approaches were adaptive conjoint analysis (n=16), analytic hierarchy process (n=9), and simple ranking (n=6). Most studies (n=37) did not report using preference- or decision aid-specific guidelines to support development. Personalized preference information was provided to participants in 45 studies. Most studies reported positive findings related to feasibility and participant experience, though complexity was identified as a challenge. The most-frequently assessed outcomes were decisional conflict and value congruence. Evidence on effectiveness was limited and mixed.</p><p><strong>Conclusions: </strong>Although individual studies report positive findings, variation in design and reporting of existing studies hinders drawing of general conclusions regarding whether Pb-VCM improve decision-making processes and outcomes. Future research should focus on developing guidelines, for which the data-extraction form developed for this study could serve as starting point.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147718028","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":"Julia F Slejko, Salomé Ricci, PhD Susan dosReis, Richard Cookson, Stacey Kowal","doi":"10.1016/j.jval.2026.03.2242","DOIUrl":"https://doi.org/10.1016/j.jval.2026.03.2242","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147718036","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":"Eliciting Societal Preferences for Health Equity: Reflections on Health Inequality Aversion in the United States.","authors":"Jiayi Chen","doi":"10.1016/j.jval.2026.01.032","DOIUrl":"10.1016/j.jval.2026.01.032","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147718083","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, Grzegorz W Basak, Emilian Snarski, Dominik Golicki","doi":"10.1016/j.jval.2026.03.2239","DOIUrl":"10.1016/j.jval.2026.03.2239","url":null,"abstract":"<p><strong>Objectives: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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).</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.0,"publicationDate":"2026-04-13","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}