Value in Health最新文献

{"title":"Individualized Prediction of Patient-Defined Treatment Success to Support Patient-Centered Clinical Decision-Making.","authors":"Robbert M Wouters, Lisa Hoogendam, Dominic Furniss, Joy C MacDermid, Thomas J Hoogeboom","doi":"10.1016/j.jval.2026.09.3129","DOIUrl":"https://doi.org/10.1016/j.jval.2026.09.3129","url":null,"abstract":"<p><strong>Objective: </strong>Clinical prediction models often target generic thresholds for treatment success, such as the Minimal Important Change. The Personal Meaningful Gain was introduced as a personalized threshold, capturing the improvement an individual seeks to achieve in a self-chosen domain to become satisfied with the treatment results. This study developed and internally validated a treatment-conditional prediction model estimating the probability of achieving the PMG within 3 months in patients with common hand and wrist conditions.</p><p><strong>Methods: </strong>This study used real-world data from 25 specialized hand and wrist care clinics in the Netherlands. We included 6263 patients (training data: n=5010; test data: n=1253) treated for common hand and wrist conditions. The prediction target was the PMG achievement at 3 months post-treatment. Model performance was evaluated using discrimination (area under the curve [AUC]) and calibration. The final model was selected based on AUC, calibration, and the number of model features.</p><p><strong>Results: </strong>We selected a gradient boosting machine algorithm with thirteen predictors. The model had an AUC of 0.73 (95% CI: 0.70 to 0.76, indicating acceptable-to-good discrimination) and excellent calibration in the test set. Following a user-centered design, we built a Shiny app visualizing the probability of reaching the PMG for different treatment options (i.e., hand therapy ± injection, injection, or surgery) (https://doi.org/10.6084/m9.figshare.31084375).</p><p><strong>Conclusions: </strong>A treatment-conditional prediction model using a patient-defined threshold for treatment success can provide individualized estimates of the probability of achieving outcomes that matter to patients. This generalizable approach may enhance shared decision-making and expectation management in preference-sensitive care.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149224976","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":"Why Bayesian Nonparametric Models Should Be a First-Line Approach for Health State Valuations.","authors":"Samer A Kharroubi","doi":"10.1016/j.jval.2026.10.003","DOIUrl":"https://doi.org/10.1016/j.jval.2026.10.003","url":null,"abstract":"<p><p>Health state valuation is fundamental to the calculation of quality-adjusted life years and, consequently, to cost-effectiveness analysis. Parametric models have traditionally been used to estimate health state values; however, their reliance on prespecified functional forms may limit their ability to capture the complex characteristics of preference-based utility data, including nonlinearity, heterogeneity, skewness, and sparsity. In the present article, we argue that Bayesian nonparametric (BNP) methods should be considered a preferred or first-line approach for modeling health state valuations, particularly when data are limited or the underlying functional form is uncertain. Drawing on more than two decades of methodological and applied research, we discuss their flexibility in representing health state preferences, predictive performance, probabilistic quantification of uncertainty, and ability to borrow strength from existing valuation studies. These features provide a strong rationale for wider adoption and systematic evaluation of BNP methods, particularly in challenging valuation settings.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149224968","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":"Sleep and fatigue/energy bolt-ons for the EQ-5D-3L and EQ-5D-5L: a systematic review.","authors":"Clement Cheuk-Wai Ng, Meixia Liao, Stevanus Pangestu, Nan Luo, Brendan Mulhern, Aureliano Paolo Finch, Jan Abel Olsen, Tessa Peasgood, Eliza Lai-Yi Wong, Fanni Rencz","doi":"10.1016/j.jval.2026.09.3128","DOIUrl":"https://doi.org/10.1016/j.jval.2026.09.3128","url":null,"abstract":"<p><strong>Objectives: </strong>This study systematically reviewed the development, use, psychometrics, and valuation of sleep and fatigue/energy bolt-ons for the EQ-5D-3L and EQ-5D-5L.</p><p><strong>Methods: </strong>A systematic review was conducted based on key elements of the PRISMA 2020 guidelines. Searches on PubMed, Embase, and Web of Science, supplemented by citation tracking and manual Google Scholar and EuroQol conference searches, were used. Key study characteristics, bolt-on specifications, psychometric properties, and valuation evidence were summarized narratively.</p><p><strong>Results: </strong>Twenty-one publications from 19 unique studies conducted in 19 countries were published between 1991 and 2025. Sleep and fatigue/energy bolt-ons were used together in 13 studies. Twelve distinct sleep and 14 fatigue/energy bolt-on versions have been developed, mostly in five-level formats (sleep=67%; fatigue/energy=71%). The development of 3 sleep and 5 fatigue/energy bolt-ons was supported by qualitative evidence. Quantitative evidence from 13 sleep and 13 fatigue/energy bolt-on studies revealed bolt-ons commonly reduced ceiling effects although not across all conditions (absolute reduction: sleep=0.3-30.0%, fatigue/energy=1.9-30.9%), showed good convergent validity with conceptually related external measures or their items/domains, and improved EQ VAS explanatory power (sleep=0.7-10.0%, fatigue/energy=0.1%-3.7%). Findings on known-group validity were heterogeneous across clinically relevant known-groups. Evidence on test-retest reliability (n=1) and responsiveness (n=3) was limited. Valuation evidence (n=5) regarding the impact of bolt-ons on preferences was scarce and inconclusive.</p><p><strong>Conclusions: </strong>The development of sleep and fatigue/energy bolt-ons to date has lacked sufficient qualitative input, while some psychometric findings supported their added value to the EQ-5D. Future mixed-methods bolt-on development and refinement studies are needed for both constructs.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149207474","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":"Beyond Preference Heterogeneity: Toward Biobehavioral Stratification in Digital Neurorehabilitation.","authors":"Baharuddin Baharuddin, Syahiduz Zaman, Jordan Bakhriansyah, Afib Rulyansah, Prasanna Srinivasan Ramalingam","doi":"10.1016/j.jval.2026.07.015","DOIUrl":"https://doi.org/10.1016/j.jval.2026.07.015","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149207486","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 Telisotuzumab Vedotin-tllv for Previously Treated High c-Met Protein-Overexpressing Nonsquamous Non-Small Cell Lung Cancer in the United States.","authors":"Glen J Weiss","doi":"10.1016/j.jval.2026.10.002","DOIUrl":"https://doi.org/10.1016/j.jval.2026.10.002","url":null,"abstract":"<p><strong>Objectives: </strong>To estimate the incremental cost-effectiveness of telisotuzumab vedotin-tllv (teliso-V) versus docetaxel in previously treated high c-Met protein-overexpressing (≥50% of tumor cells with 3+ staining) advanced nonsquamous non-small cell lung cancer.</p><p><strong>Methods: </strong>A partitioned survival model with monthly cycles and a five-year horizon was built from the US healthcare sector perspective. Individual time-to-event data were reconstructed from published Kaplan-Meier curves of the c-Met High subgroup (N=78) of LUMINOSITY and the CONTACT-01 docetaxel arm (N=180). Because individual covariate data were unavailable, a naive (unadjusted) indirect comparison was conducted. Seven parametric distributions were fit independently by arm; their median and range were the primary summary. The analysis was preregistered.</p><p><strong>Results: </strong>The unadjusted overall survival hazard ratio favored teliso-V but was not statistically significant (HR 0.690; 95% CI 0.471-1.010). The median incremental cost-effectiveness ratio was approximately $2.0 million per QALY (range, $1.7-$2.2 million). Across 70,000 probabilistic iterations, cost-effectiveness was not achieved in any iteration at $50,000, $100,000, $150,000, or $200,000 per QALY. The threshold payment limit at $150,000 per QALY was approximately $1,209 per 100 mg (92% reduction).</p><p><strong>Conclusions: </strong>At the current Medicare Part B payment limit, telisotuzumab vedotin-tllv is unlikely to be cost-effective versus docetaxel in the US high c-Met protein-overexpressing population at conventional willingness-to-pay thresholds. The conclusion was robust to modeled survival extrapolation and parameter uncertainty. Because the comparison is unadjusted, comparative effectiveness remains uncertain pending randomized results.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196798","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":"Ann-Kathrin Fischer, Axel Mühlbacher","doi":"10.1016/j.jval.2026.10.001","DOIUrl":"https://doi.org/10.1016/j.jval.2026.10.001","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196858","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 insulin regimens and insulin types for adults with type 1 diabetes: a systematic review.","authors":"Shiwa Yaghoubi Bina, Steve Njokuocha","doi":"10.1016/j.jval.2026.09.3127","DOIUrl":"https://doi.org/10.1016/j.jval.2026.09.3127","url":null,"abstract":"<p><strong>Objective: </strong>To assess the global cost-effectiveness of insulin regimens, comparing long- and short-acting insulin analogues to human insulins, in adults with T1D.</p><p><strong>Methods: </strong>Systematic review (CRD420250654279). We searched MEDLINE, Embase, Web of Science, WHO Global Index Medicus, and the HTA database on 17 March 2025. We included full economic evaluations comparing insulin regimens for adults with T1D. Incremental Cost Effectiveness Ratios (ICERs) and Incremental Cost Utility Ratios (ICURs) were converted and adjusted into a common currency (2024 US dollar) using purchasing power parity. The SBU and the CHEERS checklists were used for quality assessment.</p><p><strong>Results: </strong>We included 35 studies, covering 50 insulin comparisons across 19 countries, predominantly in Europe. Most studies were model-based (97%) and industry-funded (86%). Several studies indicated that analogue-based regimens may be cost-effective under study-specific assumptions (ICURs ranging from USD 4,550 to 10,720.28 per QALY). Among 35 comparisons of long-acting insulins, findings were mixed: ICERs ranged from USD 17 to 10,940 per 1% HbA1c reduction, and ICURs ranged from USD 102 to 124 million per QALY. Short-acting insulins were also cost-effective (ICERs ranging from USD 88 to 7,844 per 1% HbA1c annual reduction per patient; ICURs from USD 7,382 to 34,769 per QALY).</p><p><strong>Conclusions: </strong>Long- and short-acting insulin analogues are often reported to be cost-effective compared with human insulins in adults with T1D, but this conclusion rests on an evidence base with recurrent methodological limitations, wide statistical uncertainty and a high risk of sponsor-related bias and limited evidence from low- and lower-middle-income countries.</p><p><strong>Conclusion: </strong></p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149170146","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":"Health Utilities and Costs for Multiple Sclerosis.","authors":"Yankier Pijeira Perez, Carolyn A Young, Roger J Mills, Dyfrig A Hughes","doi":"10.1016/j.jval.2026.08.015","DOIUrl":"https://doi.org/10.1016/j.jval.2026.08.015","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS) is a chronic neurological condition that imposes a significant burden on patients and healthcare systems.</p><p><strong>Objectives: </strong>To estimate health utilities and costs of MS within the UK setting.</p><p><strong>Methods: </strong>Patients with MS recruited to the Trajectories of Outcomes in Neurological Conditions (TONiC-MS) study completed a survey that included the EuroQoL EQ-5D-5L, EQ VAS, and a resource use questionnaire for recording their use of MS-related primary, secondary and community care services, diagnostic tests and medicines in the previous 3 months. Data were analysed descriptively and using regression models.</p><p><strong>Results: </strong>Of 10,031 patients invited to participate, 5,913 (58.9%) returned their survey questionnaires. Overall, participants' mean (95% CI) utilities scores were 0.576 (0.568, 0.583). These were highest for those with relapsing remitting MS 0.667 (0.659, 0.675) and lowest in secondary progressive MS 0.385 (0.368, 0.402). Utility scores ranged from 0.727 (0.720, 0.734) for participants with 0≤EDSS≤4.0, to 0.125 (0.089,0.162) with 8.0≤EDSS≤9.5. Total, 3-month MS-related costs were £2807 (£2665, £2974), ranging from £809 (£563, £1188) for those within 8.0≤EDSS≤9.5 and primary progressive MS, and £9390 (£4601, £23101) for those with 7.0≤EDSS≤7.5 and rapidly evolving MS. Age, sex, MS types, EDSS scores, and some interactions between EDSS band scores and MS types explained variability in both health utilities and costs.</p><p><strong>Conclusions: </strong>The impact of MS on health utilities and costs differs by MS type, disability scores and their interaction. These data can be used in cost-effectiveness analyses to inform healthcare decision-making.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149128588","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 Value of Digital Health Interventions: A Systematic Review of Evaluation Frameworks and Approaches, Key Dimensions and Criteria.","authors":"Ann-Kathrin Fischer, Axel Mühlbacher","doi":"10.1016/j.jval.2026.09.3115","DOIUrl":"https://doi.org/10.1016/j.jval.2026.09.3115","url":null,"abstract":"<p><strong>Objectives: </strong>Digital Health Interventions (DHIs) are systematically developed digital solutions that address key healthcare challenges such as accessibility, efficiency, and quality of care. These interventions optimize healthcare processes and benefit multiple stakeholders. Despite their increasing significance, the evaluation of DHIs remains complex due to their multidimensional impacts and value contribution. This review aims to identify and synthesize value assessment frameworks (VAFs) and further evaluation approaches for DHIs, focusing on value dimensions, value criteria, and methodological diversity.</p><p><strong>Method: </strong>This systematic review was reported in accordance with the PRISMA 2020 statement. Literature was retrieved from PubMed, Web of Science, and Google Scholar. Eligibility criteria were defined using the PICO framework. Two independent reviewers screened and selected studies. A qualitative synthesis categorized approaches and extracted value dimensions.</p><p><strong>Results: </strong>A total of 2,061 records were screened, with 97 studies meeting inclusion criteria. The review identified a diverse set of evaluation approaches, including checklists, scorecards, and multidimensional VAFs. While checklists and scorecards provided structured decision-making tools, comprehensive frameworks incorporated multiple value dimensions of DHIs. Key value dimensions included impact on subject (e.g., clinical benefit), impact on interaction (e.g., usability), impact on system (e.g., interoperability), and impact on society (e.g., public perception).</p><p><strong>Conclusion: </strong>The review identified key dimensions and associated value criteria across existing frameworks and approaches, providing a structured overview of how DHIs are assessed in current practice. These findings highlight the importance of multidimensional evaluation and may support the development of more structured approaches for assessing DHIs, thereby enhancing comparability and informing value-based decision-making.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149115439","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":"Reconciling Conflicting Evidence on Peri-Immune Checkpoint Inhibitor COVID-19 mRNA Vaccination in Lung Cancer: A Time-Aligned Target Trial Emulation-Informed Landmark Study.","authors":"Shih-Sen Lin, Ju-Fang Liu, Pei-Sung Hsu, Hung-Chi Lai, Wen-Pin Cheng","doi":"10.1016/j.jval.2026.09.3126","DOIUrl":"https://doi.org/10.1016/j.jval.2026.09.3126","url":null,"abstract":"<p><strong>Objectives: </strong>Recent reports suggest that peri-immune checkpoint inhibitor (ICI) COVID-19 mRNA vaccination may improve survival in lung cancer, but time-related and calendar-time biases may inflate real-world estimates. We evaluated vaccination timing and outcomes using a target trial emulation-informed, time-aligned design.</p><p><strong>Methods: </strong>This retrospective cohort study used deidentified electronic health record data from the TriNetX Global Collaborative Network. Time zero was first ICI administration. New users initiating ICI therapy from September 2, 2020, through December 31, 2025, were classified by mRNA vaccination within -100 to +100 days as peri-ICI, pre-only, post-only, or none. Follow-up began at a prespecified day-100 landmark. Cohorts were balanced using 1:1 propensity score matching. Endpoints were overall survival (OS) and time to next systemic treatment or death (TTNT-D).</p><p><strong>Results: </strong>After matching, 2,286 patients were included per group (4,572 total). Peri-ICI mRNA vaccination was not associated with OS (hazard ratio [HR] 1.005, 95% confidence interval [CI] 0.913-1.106) or TTNT-D (HR 1.059, 95% CI 0.956-1.174). The pre-only versus post-only OS estimate was near null (HR 0.920, 95% CI 0.804-1.053), with concordant patterns across prespecified sensitivity analyses.</p><p><strong>Conclusions: </strong>Peri-ICI COVID-19 mRNA vaccination was not associated with improved outcomes in patients with lung cancer. These findings clarify discrepant reports and underscore the importance of rigorous time alignment when evaluating time-dependent exposures.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149115430","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}