Value in Health最新文献

{"title":"Reasons for Distributional Preferences for Health Outcomes: A Mixed-Method Interview Study.","authors":"Kyoko Shimamoto, Aki Tsuchiya","doi":"10.1016/j.jval.2026.07.005","DOIUrl":"10.1016/j.jval.2026.07.005","url":null,"abstract":"<p><strong>Objectives: </strong>Policy makers need information on how members of the public value improving population health (efficiency) relative to reducing socioeconomic health inequality (equity). Quantitative evidence of the trade-off using benefit trade-off (BTO) is emerging, but not qualitative or mixed-method evidence. This mixed-method study aimed to elicit distributional preferences among the general population in Japan over socioeconomic inequality in lifetime health using BTO tasks and understand the underlying reasonings.</p><p><strong>Methods: </strong>Individual online interviews (n = 50) were conducted in 2022, comprising 2 main elements: (1) BTO tasks and (2) qualitative questions regarding the participant's understanding and reasonings behind their BTO task choices.</p><p><strong>Results: </strong>The median BTO response was inequality averse (prioritarian). The qualitative analysis found the relevance of nonhealth considerations such as prioritizing the health of the rich because they make more tax contributions, violating the symmetry assumption of the standard BTO, self-interested motivations despite the exercise using a social perspective, indecisive choices, and inconsistent reasonings across BTO subtasks.</p><p><strong>Conclusions: </strong>This is the first mixed-method interview study on BTO preferences of the public, involving an in-depth qualitative component. Some of the reasonings observed were similar to those in existing qualitative studies, albeit based on non-BTO methods. The findings also suggest heterogeneous reasonings by participant gender and geographic region. Some participants gave coherent explanations for behavior such as choosing a more unequal distribution in favor of the rich (\"inequality seeking\") or choosing a less unequal distribution even when nobody is better off (violation of monotonicity).</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":"148685802","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":"Economic Evaluations of Hypertension Interventions in Pregnancy Lack Standardization: Results of a Systematic Review.","authors":"Ling Jie Cheng, Lucy Abel, Svetlana Ratushnyak, Oliver Rivero-Arias, Richard J McManus, Lucy C Chappell, Helen E Campbell","doi":"10.1016/j.jval.2026.06.025","DOIUrl":"10.1016/j.jval.2026.06.025","url":null,"abstract":"<p><strong>Objectives: </strong>Hypertensive disorders of pregnancy (HDP), including preeclampsia, pose lifelong health risks for women and infants. Although HDP interventions are increasingly evaluated for cost-effectiveness, methodological challenges and heterogeneity between studies exist. This systematic review examines how economic evaluations have been conducted, focusing on methodology, outcome measurement, perspectives, time horizons, and cost-effectiveness conclusions.</p><p><strong>Methods: </strong>Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses 2020 guidelines, we searched MEDLINE, Embase, EconLit, and Web of Science from inception to December 31, 2024 for cost-effectiveness, cost-utility, cost-benefit, cost-consequence, and cost-minimization analyses of HDP interventions. Searching was updated on November 4, 2025. Data on costs, outcomes, and methodology were extracted. Reporting quality was evaluated using Consolidated Health Economic Evaluation Reporting Standards 2022.</p><p><strong>Results: </strong>Thirty-five included studies covered interventions from prevention to management. One-third of cost-effectiveness and cost-utility studies reported only women's outcomes. Among those capturing maternal and infant outcomes, more than half estimated cost-effectiveness using outcomes for only 1 party. The remainder generated results for mothers and infants but using different approaches. Long-term maternal cardiovascular risks and infant outcomes were rarely extrapolated. Most studies adopted healthcare perspectives. Screening, prevention, and diagnostic interventions were frequently cost-effective, whereas evidence for treatment and management strategies was mixed. Most studies reported cost per preeclampsia case averted; fewer applied generic measures, limiting comparability.</p><p><strong>Conclusions: </strong>Economic evaluations of HDP interventions face methodological challenges, particularly in capturing joint maternal and infant impacts. Furthermore, future studies should use adequate time horizons, wider perspectives, and generic health outcomes to ensure that cost-effectiveness evidence captures the full value of HDP interventions and informs policies optimizing maternal and neonatal health.</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":"148685824","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":"Psychometric Evaluation of Self- and Proxy-Reported EQ-5D-Y-3L and EQ-5D-Y-5L in Children With Attention-Deficit/Hyperactivity Disorder.","authors":"Richard Huan Xu, Chenxi Yang, Zhuxin Mao, Xiao Liang, Fanni Rencz","doi":"10.1016/j.jval.2026.07.003","DOIUrl":"10.1016/j.jval.2026.07.003","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to assess the psychometric properties of 2 versions of EuroQol 5-Dimension Youth version (EQ-5D-Y) (3 level [Y-3L] and 5 level [Y-5L]) in children with attention-deficit/hyperactivity disorder (ADHD) and to compare their performance across self- and proxy-reported responses.</p><p><strong>Methods: </strong>Overall, 188 child-caregiver dyads (mean age 9 years with a clinical ADHD diagnosis) completed a cross-sectional survey, followed by a 2-week assessment to examine test-retest reliability. Outcome measures included the Y-3L and Y-5L, Child Health utility 9D, Child Behavior Checklist, and the Swanson, Nolan, and Pelham Rating Scale for ADHD severity. Ceiling, informativity, convergent and known-group validity, patient-proxy agreement, and test-retest reliability were assessed.</p><p><strong>Results: </strong>Self-reported Y-3L (48%) and Y-5L (28%) had higher ceilings than proxy versions (32% and 25%), and the Y-5L showed higher absolute informativity for both self- and proxy-reports. Self-reports correlated more strongly with the child-completed Child Health utility 9D, whereas proxy reports were more aligned with Child Behavior Checklist. Self-report version showed good known-groups validity: the Y-3L discriminated 11 of 14 groups, and the Y-5L discriminated 13 of 14. The proxy versions showed slightly weaker discrimination. Item-level test-retest reliability and patient-proxy agreement were good (Gwet's agreement coefficient [AC] 0.71-0.98 and 0.70-0.97), but scale-level reliability and agreement were lower.</p><p><strong>Conclusion: </strong>In Chinese children with ADHD, the Y-3L appears more suitable for self-report, whereas the Y-5L may be better for proxy report, depending on the population and study aims. Further research is needed to identify the optimal version across age groups and clinical conditions.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":6.2,"publicationDate":"2026-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148679891","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":"Informing the US Medicare Drug Price Negotiation for Trelegy Ellipta and Breo Ellipta: Evaluating the Impact of Real-World Evidence","authors":"Marina Richardson PhD,&nbsp;Abigail C. Wright PhD,&nbsp;Shahariar Mohammed Fahim PhD,&nbsp;Jeffrey A. Tice MD,&nbsp;Kangho Suh PharmD, PhD,&nbsp;Josh J. Carlson PhD,&nbsp;David M. Rind MD","doi":"10.1016/j.jval.2026.03.003","DOIUrl":"10.1016/j.jval.2026.03.003","url":null,"abstract":"<div><h3>Objectives</h3><div>The Institute for Clinical and Economic Review (ICER) conducted a special assessment of Trelegy Ellipta and Breo Ellipta for chronic obstructive pulmonary disease to inform the Centers for Medicare and Medicaid Services (CMS) drug price negotiation process. In this manuscript, we examine the type, quality, and impact of the observational evidence included.</div></div><div><h3>Methods</h3><div>We described the type of observational evidence used in ICER’s assessment with a primary focus on adherence and persistence data. Risk of bias was assessed using the ROBINS-I.</div></div><div><h3>Results</h3><div>Observational data included in ICER’s review primarily informed the assessment of differential adherence and persistence between single and multiple inhaler products. The majority of studies were real-world evidence (RWE), utilizing claims or electronic health record data, and were deemed to be at moderate or severe risk of bias. RWE reported that Trelegy Ellipta and Breo Ellipta were associated with greater adherence and persistence compared with generic multiple-inhaler or multiple-daily-dosing alternatives. Persistence data were critical for differentiating between products in the cost-effectiveness analysis; however, persistence was based on prescription refills which is an imperfect measure of treatment continuation.</div></div><div><h3>Conclusions</h3><div>RWE played a critical role in evaluating the comparative clinical and cost-effectiveness of Trelegy Ellipta and Breo Ellipta to inform Centers for Medicare and Medicaid Services drug price negotiations. Differences in persistence informed differential pricing to generic multiple-inhaler or multiple-daily-dosing alternatives, demonstrating how data collected outside of clinical trials can inform value assessments. However, RWE limitations and the lack of evidence linking adherence or persistence to clinical outcomes highlight the need for future research.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Pages 1360-1367"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147491696","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":"Development of the Medical Device Economic Evaluation Methodological Quality (DEEM-Q) Assessment Checklist","authors":"Ilke Akpinar MD,&nbsp;Jeff Round PhD,&nbsp;Mike Paulden PhD","doi":"10.1016/j.jval.2026.03.002","DOIUrl":"10.1016/j.jval.2026.03.002","url":null,"abstract":"<div><h3>Objectives</h3><div>Economic evaluations increasingly inform adoption and reimbursement decisions for medical devices; yet, existing quality assessment tools were largely developed for pharmaceuticals and may not adequately capture device-specific methodological challenges. The objective of the medical Device Economic Evaluation Methodological Quality assessment study was to develop a consensus-based instrument for assessing the methodological quality of economic evaluations of medical devices.</div></div><div><h3>Methods</h3><div>Candidate items were identified through a systematic review of existing methodological quality assessment tools and economic evaluation guidelines, including those from Canada’s Drug Agency. A 2-round modified e-Delphi study was conducted with 40 health economists experienced in Canadian medical device evaluation (response rates: 100% and 90% for rounds 1 and 2, respectively). Using a 9-point importance scale, items were retained if ≥70% of participants rated them as critical (scores 7-9) and ≤15% rated them as not important (scores 1-3). The resulting checklist underwent pilot testing for clarity, applicability, and consistency.</div></div><div><h3>Results</h3><div>The initial 49 items were refined to 39, covering general economic evaluation domains (decision problem, comparators, perspective, time horizon, modeling, analysis, and uncertainty) and device-specific considerations (evidence gaps, learning curve, organizational impacts, incremental innovation, and diversity). Pilot testing confirmed clarity and consistent application.</div></div><div><h3>Conclusions</h3><div>The Device Economic Evaluation Methodological Quality assessment represents the first consensus-based checklist for assessing the methodological quality of medical device economic evaluations. It provides a practical tool for researchers, reviewers, and decision makers. A companion explanation and elaboration document has been developed to support consistent interpretation and application across decision contexts.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Pages 1348-1359"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147491660","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":"Retraction notice to Integrating Generative AI Into Evidence Synthesis: Opportunities, Safeguards, and Methodological Refinements","authors":"Weihao Cheng BEng,&nbsp;Hangyu Zhu BEng,&nbsp;Yuanyuan Lian BEng","doi":"10.1016/j.jval.2026.06.008","DOIUrl":"10.1016/j.jval.2026.06.008","url":null,"abstract":"<div><div>This article has been retracted: please see Elsevier Policy on Article Withdrawal (<span><span>https://www.elsevier.com/about/policies/article-withdrawal</span><svg><path></path></svg></span>).</div><div>This letter has been retracted at the request of the Editors-in-Chief.</div><div>The undisclosed use of artificial intelligence in the preparation of this work represents a clear violation of the journal’s editorial policies and publishing ethics standards.</div><div>Apologies to the readers of the journal that this was not detected during the submission process.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Page 1537"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148631606","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":"Bayesian Indirect Comparison and Cost-Effectiveness of Sacituzumab Govitecan Versus Sacituzumab Tirumotecan in Metastatic Triple-Negative Breast Cancer","authors":"Yamin Shu MSc,&nbsp;Ying Tang MD,&nbsp;Yiling Ding PhD,&nbsp;Wenting Zhang MD,&nbsp;Qilin Zhang MD","doi":"10.1016/j.jval.2026.02.004","DOIUrl":"10.1016/j.jval.2026.02.004","url":null,"abstract":"<div><h3>Objectives</h3><div>Sacituzumab govitecan (SG) and sacituzumab tirumotecan (sac-TMT) are 2 approved antibody-drug conjugates for metastatic triple-negative breast cancer but lack direct comparison. Given their similar indications and high costs, an indirect cost-effectiveness analysis is essential to inform pricing and reimbursement decisions from the perspective of the Chinese healthcare system.</div></div><div><h3>Methods</h3><div>A partitioned survival model using Bayesian network meta-analysis and flexible parametric modeling estimated long-term survival, quality-adjusted life-years (QALYs), and costs. Participants were derived from the ASCENT and OptiTROP-Breast01 randomized controlled trials. Key outcomes included time-varying hazard ratio, life-years, costs, incremental cost-effectiveness ratio (ICER), incremental net health benefit, and incremental net monetary benefit. Multiple sensitivity and scenario analyses tested model robustness.</div></div><div><h3>Results</h3><div>In the base-case analysis, SG yielded 0.095 additional QALYs compared with sac-TMT at an incremental cost of $68 266, resulting in an ICER of $719 726 per QALY and an incremental net monetary benefit of −$64 400. Sensitivity analyses identified SG price and model time horizon as key drivers of ICER. In all plausible scenarios, SG remained not cost-effective versus sac-TMT. Probabilistic results showed a 0% probability of cost-effectiveness at a willingness-to-pay threshold of $40 763 per QALY. Scenario analysis confirmed the robustness of findings across assumptions on time horizon, utility, wastage, and discounting.</div></div><div><h3>Conclusions</h3><div>SG showed slightly greater health gains but much higher costs than sac-TMT, leading to poor cost-effectiveness at current prices. Given the high substitutability between antibody-drug conjugates in metastatic triple-negative breast cancer, indirect comparisons with economic modeling can help guide payer decisions and pricing negotiations when head-to-head data are lacking.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Pages 1385-1394"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147310618","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":"Valuation of EQ-5D-5L With 2 Bolt-On Items: Further Evaluation of the Scaling Factor Model","authors":"Zhihao Yang PhD,&nbsp;Kim Rand PhD,&nbsp;Aureliano Finch PhD,&nbsp;Nan Luo PhD","doi":"10.1016/j.jval.2026.02.012","DOIUrl":"10.1016/j.jval.2026.02.012","url":null,"abstract":"<div><h3>Objectives</h3><div>The scaling factor model (SFM) uses parameters of existing EQ-5D value sets to estimate value sets for new EQ-5D descriptive systems expanded with bolt-ons. This study aimed to compare the performance of SFM and the standard modeling approach (ie, “standalone” model) for modeling the general public’s preferences for EQ-5D-5L health states expanded with bolt-on.</div></div><div><h3>Methods</h3><div>In a composite time trade-off (cTTO) valuation study, we selected EQ-5D-5L and bolt-on health states using an orthogonal array design. We randomized 597 respondents to valuing EQ-5D-5L health states (arm 1), EQ-5D-5L states with vision bolt-on (arm 2), or EQ-5D-5L states with cognition bolt-on (arm 3). We modeled the cTTO data derived from arm 1 and used the estimated coefficients to model the cTTO data derived from arm 2 and arm 3 separately using SFM. Both additive and cross-attribute level effects model specifications were used in the analysis. Using a cross-validation method, we evaluated the predictive accuracy of both SFM and standalone models.</div></div><div><h3>Results</h3><div>SFM showed better predictive accuracy than standalone model in the cross-validation analysis. For EQ-5D states with vision bolt-on, the mean absolute errors were 0.049 and 0.064 for SFM and 0.183 and 0.085 for standalone model; for cognition, the mean absolute errors were 0.051 and 0.047 for SFM and 0.152 and 0.063 for standalone model.</div></div><div><h3>Conclusions</h3><div>This study suggests that the SFM could be a viable approach to utilizing existing EQ-5D value sets to estimate value sets for new, bolt-on enhanced EQ-5D health-state descriptive systems.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Pages 1443-1451"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147445007","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":"Real-World Evidence in the First Round of the United States Inflation Reduction Act Drug Price Negotiations: A Citation Analysis of the Centers for Medicare and Medicaid Services’ Maximum Fair Price Explanation Documents","authors":"Debra Winberg PhD, MSc, MA,&nbsp;Liping Du,&nbsp;Lizheng Shi PhD, MsPharm, MA","doi":"10.1016/j.jval.2026.03.006","DOIUrl":"10.1016/j.jval.2026.03.006","url":null,"abstract":"<div><h3>Objectives</h3><div>To quantify and characterize the use of real-world evidence (RWE) in the Centers for Medicare and Medicaid Services (CMS) in the CMS drug pricing negotiations to assess the transparency and methodological consistency of RWE incorporation.</div></div><div><h3>Methods</h3><div>We extracted and reviewed all 1822 unique citations across the 10 MFP explanation documents available on the CMS’s website. For each citation, we identified whether the study constituted RWE, the data source (eg, claims, electronic health records registry/clinical cohort), study design, and whether external health technology assessments were cited. We calculated drug-specific and overall RWE proportions and summarized the methodological features of RWE studies.</div></div><div><h3>Results</h3><div>RWE was cited for all 10 drugs, but its use varied substantially. The share of RWE ranged from 13.8% (Farxiga) to 41.5% (Xarelto). Across all drugs, nearly half of RWE studies used claims/administrative data (47.0%), followed by electronic health record data (17.1%) and registry/clinical cohort data (17.1%). Most RWE studies employed cohort or longitudinal designs (82.1%). Three drugs’ citation lists included external health technology assessments HTAs (by the Institute for Clinical and Economic Review or the National Institute for Health and Care Excellence). Despite the broad inclusion of RWE, the maximum fair price documents did not specify how RWE influenced negotiated prices or how CMS evaluated the quality or relevance of these studies.</div></div><div><h3>Conclusion</h3><div>These findings underscore the need for greater transparency and consistent methodological standards for evaluating RWE, aligning Medicare’s process more closely with international health technology assessment practices.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Pages 1343-1347"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147515069","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":"Fast Science, Slow Evaluation: What Makes Health Technology Assessment of Genetic and Genomic Technologies So Complicated? A Systematic Review of Methodological Challenges and Identified Suggestions","authors":"Valentina Baccolini MD, MPH,&nbsp;Leonardo Maria Siena MD,&nbsp;Claudia Isonne MD,&nbsp;Antonio Sciurti MD,&nbsp;Carolina Marzuillo BSc,&nbsp;Corrado De Vito MD, PhD,&nbsp;Giuseppe La Torre MD, PhD,&nbsp;Marcello Arca MD,&nbsp;Giuseppe Migliara MD, PhD,&nbsp;Paolo Villari MD, MPH","doi":"10.1016/j.jval.2026.03.004","DOIUrl":"10.1016/j.jval.2026.03.004","url":null,"abstract":"<div><h3>Objectives</h3><div>The implementation of new applications into healthcare systems and practices frequently relies on health technology assessment (HTA). Given their complexity, HTA of genetic and genomic technologies (GGTs) faces multiple challenges; yet, no comprehensive synthesis exists. We systematically reviewed and summarized HTA challenges for GGTs, overall and by HTA domain.</div></div><div><h3>Methods</h3><div>Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, we searched 3 databases (OSF protocol h4qg2). Eligible studies primarily discussed HTA challenges/barriers for GGTs across any HTA domain. Issues were classified into 9 EUnetHTA’s Core Model domains plus a “framework” category. Results were synthesized narratively.</div></div><div><h3>Results</h3><div>Twenty records published between 2007 and 2025 were included: heterogeneous in structure, most discussed multiple HTA domains, and some reported suggestions. Economic (<em>n</em> = 12), clinical (<em>n</em> = 9), and social (<em>n</em> = 8) considerations predominated, whereas safety and organizational aspects were underrepresented. Across domains, we identified a persistent mismatch between the rapid evolution of GGTs and the slower generation of evidence, along with insufficient standardization and validation of key procedures that hinder cross-study comparability. Other major issues included defining clear healthcare pathways, valuing genomic testing, and strengthening regulatory frameworks to protect patient rights, data integrity, and equity of access. Suggested remedies were adopting living/rapid HTA, defining core outcome sets, harnessing real-world data sources, standardizing guidelines, engaging stakeholders, and enhancing national commitment.</div></div><div><h3>Conclusions</h3><div>Fast-moving science and inadequate methods hamper HTA of GGTs. Adopting flexible HTA methods, standardized outcomes and procedures, and multistakeholder engagement, supported by national infrastructures, can shift HTA toward a more agile, resource-enabled system that better supports timely, generalizable, and equitable decision making in genomics.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"29 8","pages":"Pages 1504-1514"},"PeriodicalIF":6.2,"publicationDate":"2026-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147499851","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}