Value in Health最新文献

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EQ-5D-5L in Post-COVID-19 Condition. EQ-5D-5L在COVID-19后状态。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-21 DOI: 10.1016/j.jval.2026.03.2266
Hinpetch Daungsupawong, Viroj Wiwanitkit
{"title":"EQ-5D-5L in Post-COVID-19 Condition.","authors":"Hinpetch Daungsupawong, Viroj Wiwanitkit","doi":"10.1016/j.jval.2026.03.2266","DOIUrl":"10.1016/j.jval.2026.03.2266","url":null,"abstract":"","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148798425","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}
引用次数: 0
Cost-Effectiveness of Opportunistic Salpingectomy During Various Nongynecological Abdominal Surgeries for the Prevention of Tubo-Ovarian Carcinoma: Results From a Markov Modeling Study. 机会性输卵管切除术在各种非妇科腹部手术中预防输卵管卵巢癌的成本效益:来自马尔科夫模型研究的结果。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-19 DOI: 10.1016/j.jval.2026.07.011
Jolijn Schauwaert, Sabien J E Bosman, Charlotte Fisch, Jurgen M J Piek, Philip R de Reuver, Diederik M Somford, Joanne A de Hullu, Wietske Kievit, Rosella P M G Hermens
{"title":"Cost-Effectiveness of Opportunistic Salpingectomy During Various Nongynecological Abdominal Surgeries for the Prevention of Tubo-Ovarian Carcinoma: Results From a Markov Modeling Study.","authors":"Jolijn Schauwaert, Sabien J E Bosman, Charlotte Fisch, Jurgen M J Piek, Philip R de Reuver, Diederik M Somford, Joanne A de Hullu, Wietske Kievit, Rosella P M G Hermens","doi":"10.1016/j.jval.2026.07.011","DOIUrl":"10.1016/j.jval.2026.07.011","url":null,"abstract":"<p><strong>Objectives: </strong>Opportunistic salpingectomy (OS), the additional removal of the fallopian tubes during abdominal surgery, is a preventive strategy against tubo-ovarian carcinoma. Little is known about the economic impact of OS during nongynecological surgeries.</p><p><strong>Methods: </strong>Markov state transition models were created comparing the addition of OS in women aged 35 to 65 during cholecystectomy, bariatric surgery, nephrectomy for renal cell carcinoma, and colectomy for colorectal cancer to no addition of OS. Quality-adjusted life-years (QALYs) and healthcare costs were modeled over a lifetime horizon, and uncertainty was assessed with sensitivity analyses. A willingness-to-pay threshold of €20 000 was applied.</p><p><strong>Results: </strong>OS during cholecystectomy and bariatric surgery resulted in a QALY gain of 0.033 and 0.034, and a cost reduction of €12 and €7 per patient, respectively. The probability that OS is cost-effective exceeded 99%. OS during nephrectomy for renal cell carcinoma or colectomy for colorectal cancer yielded a QALY gain of 0.012 and 0.016 and a cost increase of €39 and €25 per patient, respectively. The probability that OS is cost-effective was 94.2% and 96.6%.</p><p><strong>Conclusion: </strong>Modeling OS during nongynecological surgeries for benign indications suggests that the procedure may be cost-effective, resulting in QALY gain and potential cost savings. For oncologic indications OS appears to be cost-effective, yielding a gain in QALYs, while also leading to an increase in costs. However, the results should be considered exploratory and interpreted with caution in light of the model assumptions.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148798345","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}
引用次数: 0
Super Learner G-Computation as a Model-Specification Sensitivity Analysis for Population-Adjusted Indirect Comparisons in Health Technology Assessment. 超级学习者g计算作为健康技术评估中人口调整间接比较的模型-规范敏感性分析。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-18 DOI: 10.1016/j.jval.2026.07.010
Ying Chen, Yun Bao, Yueting Chen, Yan Jin, Kaijie Yao, Wen Li, Yan Zhong, Bin Wu
{"title":"Super Learner G-Computation as a Model-Specification Sensitivity Analysis for Population-Adjusted Indirect Comparisons in Health Technology Assessment.","authors":"Ying Chen, Yun Bao, Yueting Chen, Yan Jin, Kaijie Yao, Wen Li, Yan Zhong, Bin Wu","doi":"10.1016/j.jval.2026.07.010","DOIUrl":"10.1016/j.jval.2026.07.010","url":null,"abstract":"<p><strong>Objectives: </strong>Parametric G-computation can align population-adjusted indirect comparisons (PAICs) with marginal estimands, but estimates may depend on unverifiable outcome-model specification. We evaluated Super Learner G-computation as a flexible outcome-modeling extension for anchored PAICs.</p><p><strong>Methods: </strong>We simulated 18 anchored A vs C and B vs C trial scenarios with index-trial individual patient data and comparator-trial aggregate data. Scenarios varied sample size, population overlap, and correct vs misspecified parametric outcome models. The target was the comparator-trial population; the estimand was the marginal A vs B log-odds ratio. We compared parametric G-computation, Super Learner G-computation, matching-adjusted indirect comparison, conventional mean-plug-in simulated treatment comparison, split-averaged Super Learner G-computation, and targeted maximum likelihood estimation. We used root mean squared error (RMSE) as the primary performance measure and empirical coverage to assess interval calibration.</p><p><strong>Results: </strong>In the correctly specified large-sample good-overlap scenario, parametric G-computation had the lowest RMSE (0.263). In the nonlinear small-sample poor-overlap scenario, Super Learner G-computation had RMSE 0.513 vs 0.811 for parametric G-computation, 0.901 for conventional simulated treatment comparison, and 1.153 for matching-adjusted indirect comparison. The split-averaged version reduced RMSE to 0.475. Across 9 nonlinear scenarios, flexible plug-in estimators had lower RMSE than parametric G-computation in 7, mainly under poor or moderate overlap, but coverage was lower in all 9. Targeted maximum likelihood estimation improved coverage in some settings but did not consistently reduce RMSE.</p><p><strong>Conclusions: </strong>Super Learner G-computation may be useful as an outcome-model specification sensitivity analysis for marginal PAIC estimands. Lower RMSE came with lower coverage; therefore, uncertainty intervals require caution.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148798422","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}
引用次数: 0
Mapping Treatment Effects on Generic Health-Related Quality-of-Life Scales From Disease-Specific Measures Using Bivariate Meta-Analytic Models and Summary Data. 使用双变量荟萃分析模型和汇总数据从疾病特异性测量中绘制治疗对一般健康相关生活质量量表的影响。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-18 DOI: 10.1016/j.jval.2026.07.008
Ayman S Sadek, Nicola J Cooper, Nicky J Welton, Sylwia Bujkiewicz
{"title":"Mapping Treatment Effects on Generic Health-Related Quality-of-Life Scales From Disease-Specific Measures Using Bivariate Meta-Analytic Models and Summary Data.","authors":"Ayman S Sadek, Nicola J Cooper, Nicky J Welton, Sylwia Bujkiewicz","doi":"10.1016/j.jval.2026.07.008","DOIUrl":"10.1016/j.jval.2026.07.008","url":null,"abstract":"<p><strong>Objectives: </strong>Missing treatment effects on generic measures of health-related quality of life (HRQOL) is a common limitation in economic evaluation. Mapping from disease-specific measures of HRQOL bridges this evidence gap. We aimed to estimate treatment effects on generic measures of HRQOL in ankylosing spondylitis (AnSp) and nonradiographic axial Spondylarthritis (nr-axSpA) and highlight the benefits for economic evaluation.</p><p><strong>Methods: </strong>Summary measures of HRQOL were extracted from 25 randomized control trials of treatments for AnSp and nr-axSpA. We identified correlated pairs of disease-specific and generic measures. We mapped disease-specific to generic measures using multivariate meta-analytic surrogate endpoint models. In cases which generic measures were missing, they were predicted from the disease-specific measures. We compared predictions from bivariate random-effects meta-analysis (BRMA) models, the fixed-effects model by Daniels-Hughes, and the BRMA in product-normal formulation (BRMA-PNF). Furthermore, we compared the pooled generic measures, including and excluding predictions of the missing treatment effects.</p><p><strong>Results: </strong>Prediction intervals for the generic measures-the 36-Item Short Form Survey Physical Component Summary and the Mental Component Summary-overlapped with 90% of the reported observed estimates. Estimates of the missing treatment effects were more precise with BRMA-PNF compared with the Daniels-Hughes model. Pooling reported and predicted treatment effects using BRMA-PNF reduced uncertainty by up to 38% compared with univariate models.</p><p><strong>Conclusion: </strong>Aggregate level mapping with BRMA-PNF model provided the best fit to data and gave more precise estimates of relative treatment effects on the generic measures of HRQOL for this case study in AnSp and nr-axSpA.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148798412","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}
引用次数: 0
Therapeutic Drug Monitoring Guided Vedolizumab Treatment in Ulcerative Colitis in Cyprus: A Cost-Effectiveness Analysis. 治疗药物监测引导维多单抗治疗溃疡性结肠炎在塞浦路斯:成本效益分析。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-18 DOI: 10.1016/j.jval.2026.07.009
Olga Pitsillidou, Panagiotis Petrou, Petya Milushewa, Maria Pitsillidou, Elli Filippou, Sean P Gavan
{"title":"Therapeutic Drug Monitoring Guided Vedolizumab Treatment in Ulcerative Colitis in Cyprus: A Cost-Effectiveness Analysis.","authors":"Olga Pitsillidou, Panagiotis Petrou, Petya Milushewa, Maria Pitsillidou, Elli Filippou, Sean P Gavan","doi":"10.1016/j.jval.2026.07.009","DOIUrl":"10.1016/j.jval.2026.07.009","url":null,"abstract":"<p><strong>Objectives: </strong>Vedolizumab (VDZ) is indicated for patients with ulcerative colitis who have not responded to antitumor necrosis factor therapies. Lack of response often leads to surgical intervention due to exhaustion of pharmacological options, with surgery associated with substantial costs and morbidity. Emerging evidence suggests that therapeutic drug monitoring (TDM)-guided VDZ dose optimization may improve clinical response and reduce the need for surgery. This study assessed the cost-effectiveness of TDM-guided VDZ optimization in Cyprus.</p><p><strong>Methods: </strong>An exploratory cost-utility analysis compared a TDM-guided VDZ strategy with standard VDZ care from the Cypriot healthcare payer perspective. A Markov model with a 10-year time horizon estimated costs and quality-adjusted life-years (QALYs). Decision uncertainty was explored through probabilistic/deterministic and expected value of perfect information analyses.</p><p><strong>Results: </strong>Compared with standard care, TDM-guided VDZ resulted in an incremental cost of €16 924 and an incremental gain of 0.16 QALYs. The resulting incremental cost-effectiveness ratio was €108 297 per QALY, exceeding the €90 000 per QALY threshold and indicating that TDM-guided VDZ was not cost-effective. Threshold analysis showed that reductions in VDZ acquisition costs would render the intervention cost-effective. Expected value of perfect information analysis suggested that additional evidence would be valuable in the Cypriot context.</p><p><strong>Conclusion: </strong>TDM-guided vedolizumab improved health outcomes but increased costs and was not cost-effective at current prices. However, threshold analyses indicate that managed entry agreements linking price to real-world outcomes may enable cost-effective adoption by addressing patient heterogeneity and uncertainty.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148798406","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}
引用次数: 0
Cost-Effectiveness of Epigenetic Drugs in Oncology: A Systematic Review of Economic Evaluations. 肿瘤表观遗传药物的成本效益:经济评估的系统综述。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-13 DOI: 10.1016/j.jval.2026.06.024
Lou-Anne Peretti, Isabelle Borget, Sophie Postel-Vinay, Christophe Willekens, Julia Bonastre, Arnaud Pagès
{"title":"Cost-Effectiveness of Epigenetic Drugs in Oncology: A Systematic Review of Economic Evaluations.","authors":"Lou-Anne Peretti, Isabelle Borget, Sophie Postel-Vinay, Christophe Willekens, Julia Bonastre, Arnaud Pagès","doi":"10.1016/j.jval.2026.06.024","DOIUrl":"10.1016/j.jval.2026.06.024","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to systematically review the cost-effectiveness analyses of epigenetic drugs, an emerging class of therapies in oncology.</p><p><strong>Methods: </strong>A systematic search was conducted in Medline, Scopus, and Embase databases for references published from January 2000 to November 2024, reporting an economic evaluation on an epigenetic drug regardless of the oncologic or onco-hematologic indication. Eligible studies were cost-effectiveness analysis or cost-utility analysis reporting outcomes in cost per quality-adjusted life-year (QALY) or cost per life-year gained (LYG). The reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist and the methodological quality with the ISPOR checklist. All costs were converted into 2023 US dollars using purchasing power parity.</p><p><strong>Results: </strong>Of the 541 articles, abstracts, posters, and letters identified, 19 articles were included, encompassing 32 comparisons across 7 epidrugs (azacitidine, decitabine, panobinostat, ivosidenib, tazemetostat, enasidenib, and vorinostat) in 6 distinct indications in oncology. Overall reporting quality was high, 16 of 19 studies meeting at least 70% of CHEERS recommendations and the relevance and credibility were sufficient. Differences in QALYs ranged from -7.71 to 2.6 QALY and cost differences from -$173 300 to $867 500. Base-case results for the incremental cost-effectiveness ratio in terms of cost per QALY ranged from -$129 848/QALY to $1 323 228/QALY.</p><p><strong>Conclusion: </strong>This systematic review offers a comprehensive synthesis of current economic evaluations of epidrugs in oncology. The wide variability in incremental cost-effectiveness ratios and methodological approaches underscores the need for more standardized and robust evaluations to clarify the economic value of epigenetic drugs.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148762478","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}
引用次数: 0
Measuring Cause-Specific Tolerance for Health Inequalities Using the Social Marginal Rate of Substitution. 利用社会边际替代率衡量卫生不平等的特定原因容忍度。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-10 DOI: 10.1016/j.jval.2026.07.007
Ignacio Abásolo, Aki Tsuchiya
{"title":"Measuring Cause-Specific Tolerance for Health Inequalities Using the Social Marginal Rate of Substitution.","authors":"Ignacio Abásolo, Aki Tsuchiya","doi":"10.1016/j.jval.2026.07.007","DOIUrl":"10.1016/j.jval.2026.07.007","url":null,"abstract":"<p><strong>Objectives: </strong>We examine if tolerance for a fixed health inequality varies by whether it is caused by differences in household income, freely chosen lifestyles, or genetics. Distributional cost-effectiveness analysis (DCEA) uses equity weights to reflect societal preferences over improving total health and reducing health inequality, and cause-specific tolerance can ground DCEA in empirical preferences.</p><p><strong>Methods: </strong>Using benefit trade-off tasks in an unassisted online survey, respondents recruited from the Spanish public (n = 497) chose between hypothetical options that traded total health for reduced health inequality between 2 equally sized population halves. For a given inequality in healthy life-years, we use the social marginal rate of substitution (SMRS) as a direct measure of tolerance. We elicited indifference points (IPs), compared paired IP distributions nonparametrically, and estimated implied SMRS with individual fixed-effects regressions.</p><p><strong>Results: </strong>Most respondents traded total health for equality with a sizeable nontrading minority. Implied mean SMRS by cause were 3.60 (Income), 3.25 (Genetics), and 1.74 (Lifestyles). Income-Lifestyles and Genetics-Lifestyles differences were significant (P < .001); Income-Genetics difference was not (P = .512). Results were robust to scenario order and to recoding IPs for nontraders.</p><p><strong>Conclusions: </strong>Tolerance for a given health inequality depends on its perceived cause: tolerance was higher when inequality was caused by lifestyles than income or genetics, between which no significant difference was found. Thus, \"natural\" (genetics-caused) inequalities are not viewed as more legitimate than income-caused ones. Cause-specific SMRS can help inform the calibration of DCEA equity weights to capture not only the extent of inequality but also its cause.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148707681","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}
引用次数: 0
Swimming in the Sea of Real-World Evidence Guidance: Why More Guidance Documents Are Needed to Help Researchers Swim. 在真实世界证据的海洋中游泳(RWE)指导:为什么需要更多的指导文件来帮助研究人员游泳。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-06 DOI: 10.1016/j.jval.2026.07.006
Sandipan Bhattacharjee
{"title":"Swimming in the Sea of Real-World Evidence Guidance: Why More Guidance Documents Are Needed to Help Researchers Swim.","authors":"Sandipan Bhattacharjee","doi":"10.1016/j.jval.2026.07.006","DOIUrl":"10.1016/j.jval.2026.07.006","url":null,"abstract":"<p><p>Real-world evidence (RWE) plays an expanding role in regulatory, health technology assessment, and lifecycle decision making, prompting a rapid increase in guidance documents intended to support its generation and use. This commentary argues that additional guidance is not redundant; rather, it is necessary to sustain consistency, credibility, and confidence as RWE methods become more specialized and operationally complex. Recent advances, including pragmatic and registry-based trials, hybrid randomized-real-world designs, external control arms, artificial intelligence and machine learning applications, digital health data, synthetic controls, and data tokenization, have outpaced the scope of many existing frameworks. Although recent reporting initiatives and international harmonization efforts have improved transparency and reproducibility, important gaps remain in implementation-focused guidance on issues such as causal inference, dynamic borrowing, linkage validation, algorithm auditability, reproducibility, and distributed data environments. The need is further amplified by heterogeneity across regulatory and health technology assessment agencies, for which differing evidentiary expectations can create uncertainty and inefficiency, and by the limited availability of context-appropriate guidance for low- and middle-income countries, where structural data and infrastructure constraints may hinder RWE generation and use. Future progress should emphasize targeted, modular, and potentially \"living\" guidance that is updated as methods evolve, while also improving uptake of existing frameworks through clearer reporting expectations, education, and stakeholder collaboration. More guidance, when focused and implementation oriented, can better translate methodological principles into decision-grade RWE.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148685779","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}
引用次数: 0
Reframing NICE's Cost-Effectiveness Threshold: Technical Benchmark or Policy Signal? A Documentary Content Analysis. 重构NICE的成本效益门槛:技术基准还是政策信号?纪录片内容分析。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-06 DOI: 10.1016/j.jval.2026.07.004
Hesam Ghiasvand, Seyran Naghdi
{"title":"Reframing NICE's Cost-Effectiveness Threshold: Technical Benchmark or Policy Signal? A Documentary Content Analysis.","authors":"Hesam Ghiasvand, Seyran Naghdi","doi":"10.1016/j.jval.2026.07.004","DOIUrl":"10.1016/j.jval.2026.07.004","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to examine how governmental, industry, academic, consultancy, journalistic, and international health technology assessment (HTA) stakeholders framed the cost-effectiveness threshold (CET) uplift announced for the National Institute for Health and Care Excellence (NICE) in December 2025 during the early phase of policy debate.</p><p><strong>Methods: </strong>A qualitative documentary content analysis was conducted following the READ framework (Ready your materials, Extract data, Analyse data, and Distil your findings) for systematic document analysis in health policy research. Twenty-seven publicly available documents published between October 2025 and March 2026 were purposively sampled across government, NICE, industry, academia, consultancy, journalism, civil society, and international HTA actors. Data were analyzed through iterative coding and thematic synthesis.</p><p><strong>Results: </strong>The 2025 to 2026 debate extended beyond technical discussions of cost-effectiveness and revealed 5 interrelated themes. First, government documents explicitly reframed the CET as an instrument of industrial policy, economic growth, and pharmaceutical competitiveness. Second, substantial disagreement emerged regarding HTA governance and ministerial involvement in threshold setting. Third, stakeholders advanced competing interpretations of the consequences of the reform, with government and industry emphasizing innovation access, whereas academic and public-interest sources highlighted opportunity costs, service displacement, and population-health impacts. Fourth, many documents situated the reform within broader geopolitical and pharmaceutical-policy dynamics, including trade negotiations, investment incentives, and potential international spillovers. Finally, stakeholders expressed contrasting views regarding transparency, consultation, and procedural legitimacy.</p><p><strong>Conclusions: </strong>The NICE threshold uplift was framed as a politically mediated reform with implications for industrial strategy, HTA governance, healthcare resource allocation, and public trust. The findings demonstrate how CETs can become sites of broader political, economic, and institutional contestation, extending beyond their conventional role within health-economic evaluation.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148685752","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}
引用次数: 0
Swimming in the Sea of Real-World Evidence Guidance: Why More Guidance Will Not Help Researchers Swim. 在RWE指南的海洋中游泳:为什么更多的指导不会帮助研究人员游泳。
IF 6.2 2区 医学
Value in Health Pub Date : 2026-08-06 DOI: 10.1016/j.jval.2026.06.026
Ashley Jaksa
{"title":"Swimming in the Sea of Real-World Evidence Guidance: Why More Guidance Will Not Help Researchers Swim.","authors":"Ashley Jaksa","doi":"10.1016/j.jval.2026.06.026","DOIUrl":"10.1016/j.jval.2026.06.026","url":null,"abstract":"<p><p>The global use of real-world data and real-world evidence (RWE) in regulatory and health technology assessment decisions has led to a crucial need for alignment on what constitutes high-quality RWE. Regulators, health technology assessment bodies, and professional societies have issued an abundance of guidance documents, resulting in a state of guidance oversaturation. The existence of multiple initiatives to track this guidance is itself indicative of the challenge researchers face in interpreting and aligning with decision-maker expectations. Although the proliferation of guidance forces researchers into exhaustive \"compare and contrast\" exercises, multiple publications have documented substantial alignment on core principles, including study design, data quality, the importance of fit-for-purpose data, and methodological rigor. For example, the fundamental requirements for real-world data quality are similar between the Food and Drug Administration and European Medicines Agency. This convergence suggests that additional, redundant guidance is not the answer to addressing remaining gaps. Instead of producing new, high-level guidance, future efforts should pivot toward 2 critical areas. First, global harmonization of existing guidance is necessary to reduce the resource burden on sponsors and prevent evidentiary fragmentation across jurisdictions. Second, a transparent, annotated repository of diverse case studies must be developed to build operational transparency in RWE use and decision making. This repository should capture why specific data and methodological decisions were made, how the evidence was evaluated, and how it was ultimately used in decision making. By shifting the focus from guidance to transparent, precedent-based learning and harmonized standards, the industry can establish a more predictable and efficient pathway for decision-grade RWE integration.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148685774","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}
引用次数: 0
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