Value in Health最新文献

{"title":"Capturing the Additional Cardiovascular Benefits of SGLT2 Inhibitors and GLP-1 Receptor Agonists Beyond the Control of Traditional Risk Factors in People With Diabetes.","authors":"Shu Niu, Dawei Guan, Lizheng Shi, Vivian Fonseca, Mikael Svensson, Mohammed K Ali, Yan V Sun, Xin Hu, Chang Su, Carl Yang, Hui Shao","doi":"10.1016/j.jval.2025.01.015","DOIUrl":"10.1016/j.jval.2025.01.015","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to quantify the additional cardioprotective effects of sodium-glucose cotransporter 2 inhibitors (SGLT2is) and glucagon-like peptide-1 receptor agonists (GLP-1RAs) beyond the traditional risk factors control in individuals with type 2 diabetes. This helps calibrate the Building, Relating, Assessing, and Validating Outcomes (BRAVO) diabetes simulation model to capture the total cardiovascular benefits of new diabetes medications accurately.</p><p><strong>Methods: </strong>We extracted patient characteristics and treatment efficacy data from 4 cardiovascular outcome trials (CVOTs) of SGLT2is and 4 CVOTs of GLP-1RAs completed before May 2023. Using the BRAVO diabetes simulation model, we translated reductions in traditional risk factors (ie, glycated hemoglobin, systolic blood pressure, low-density lipids, and body mass index) from the newer drugs into risk reductions in cardiovascular outcomes (ie, myocardial infarction [MI], stroke, congestive heart failure [CHF], and mortality), assuming that the drug-associated risk reductions were only driven by traditional risk factors. Then, we compared the simulated risk-factor-driven risk reductions of cardiovascular outcomes with observed risk reductions from the trials and calculated drug-specific incremental benefits (DIB).</p><p><strong>Results: </strong>After accounting for the cardiovascular effects from traditional risk factors control, SGLT2is was associated with an additional 19% risk reduction in CHF (DIB: 0.81, 95% CI 0.72-0.90). Furthermore, the uncalibrated model predicted a risk reduction in stroke with SGLT2is, which was not observed in CVOTs. This discrepancy highlights the need for an SGLT2i-specific calibrator to align the simulation results with the observed outcomes. In contrast, no additional cardiovascular benefit was associated with GLP-1RAs after controlling for traditional risk factors.</p><p><strong>Conclusions: </strong>Our study revealed that SGLT2is could further reduce CHF risk beyond the control of traditional risk factors but may offer additional pathways to offset the overall benefits of traditional risk factor control in stroke risk. No additional cardiovascular benefits were observed for GLP-1RAs beyond traditional risk factor control. The BRAVO model calibration enhances cardiovascular outcome prediction with these newer antidiabetic therapies.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374785","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":"Understanding the Economic Value of Interventions Addressing Perinatal Mental Health Problems: A Literature Review and Methodological Considerations.","authors":"Annette Bauer, Alain Gregoire, Nazak Salehi, Jessica Weng, Martin Knapp","doi":"10.1016/j.jval.2025.01.016","DOIUrl":"10.1016/j.jval.2025.01.016","url":null,"abstract":"<p><strong>Objectives: </strong>Economic evaluations of mental health problems typically only include short-term measures from an individual healthcare perspective. In perinatal mental health, which spans generations, this is likely to lead to an underestimation of interventions' potential benefits. We sought to understand the spectrum of outcomes of perinatal mental health problems that have economic consequences and how they are captured in economic evaluations.</p><p><strong>Methods: </strong>We conducted a systematic search of the peer-reviewed literature to identify 2 types of evidence: (1) synthesized evidence (ie, systematic reviews and meta-analyses) or recent cohort studies that measured the outcomes of perinatal mental health problems and (2) economic evaluations. After presenting the evidence narratively, we derive an overview of different types of outcomes to include in economic evaluations.</p><p><strong>Results: </strong>Evidence on the many, wide-ranging adverse outcomes with short- and long-term economic consequences is rich, ranging from those that can be measured during the perinatal period (eg, mother's employment), those that require a longer-term follow-up period (eg, children's mental health service use), and those that can be used as predictors in modeling studies (eg, birth weight). Only a small subset of economic consequences and their predictors (eg, child maltreatment and poor attachment) are currently measured in economic evaluations. We make some recommendations on how more and new types of economic evaluations might start addressing the gap in knowledge.</p><p><strong>Conclusions: </strong>To inform decisions about reducing the costs of perinatal mental health problems, economic evaluations that provide knowledge of the ability of interventions to reduce short- and long-term economic consequences are urgently needed.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374195","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":"How Do Individuals Value Worse-Than-Dead EQ-5D-5L Health States in Composite Time Trade-Off Tasks? A Qualitative Study","authors":"Meixia Liao MPA ,&nbsp;Zhihao Yang PhD ,&nbsp;Milad Karimi PhD ,&nbsp;Kim Rand PhD ,&nbsp;Nan Luo PhD","doi":"10.1016/j.jval.2025.01.010","DOIUrl":"10.1016/j.jval.2025.01.010","url":null,"abstract":"<div><h3>Objectives</h3><div>Limited knowledge exists regarding individuals’ thought processes when valuing health states using composite time trade-off (cTTO) tasks, particularly for health states considered worse than dead (WTD). This study aimed to explore the reasons for WTD perception, the strategies respondents used to value WTD states, and the challenges they faced during cTTO tasks.</div></div><div><h3>Methods</h3><div>This qualitative investigation was embedded within a methodological EQ-5D-5L valuation study involving the general public in Singapore, where respondents completed 7 cTTO and 7 novel TTO tasks. Semistructured interviews were conducted with respondents who assigned WTD values to 1 or more health states in the cTTO tasks. Interviews were recorded, transcribed verbatim, and analyzed using framework analysis.</div></div><div><h3>Results</h3><div>Nineteen respondents were interviewed (age range: 21-71 years old). Respondents identified a range of interconnected factors leading to WTD valuation, including health state and its duration, personal beliefs and circumstances, and consequences of poor health. Although respondents usually considered these factors thoroughly and were capable of complex trading-off between life years and health, some respondents resorted to heuristic strategies to simplify the valuation process, including attribute nonattendance, affect heuristic, and elimination-by-aspects strategies. Respondents encountered various difficulties when they completed the cTTO tasks, including identifying the indifference point, transitioning from conventional TTO to lead-time TTO, and interpreting and imagining health states.</div></div><div><h3>Conclusions</h3><div>This study investigated how individuals value WTD health states in cTTO tasks. It suggested that cTTO needs improvement and provided insights into how to improve the design of valuation tasks for severe health states.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 4","pages":"Pages 622-632"},"PeriodicalIF":4.9,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374883","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":"Managing Uncomplicated Singleton Breech Pregnancy Using the OptiBreech Collaborative Care Pathway: Early Economic Model and Value of Information Analysis.","authors":"Siddesh S Shetty, Shawn Walker, Julia Fox-Rushby","doi":"10.1016/j.jval.2025.01.013","DOIUrl":"10.1016/j.jval.2025.01.013","url":null,"abstract":"<p><strong>Objectives: </strong>Management of breech presentation is a subject of ongoing clinical debate. The cost-effectiveness of improving safe vaginal breech birth is unknown. This study examines potential cost-effectiveness of OptiBreech collaborative care and assesses value of undertaking further research.</p><p><strong>Methods: </strong>A decision tree was used to evaluate the potential cost-effectiveness of OptiBreech care versus standard care for a hypothetical cohort of women with confirmed singleton breech pregnancy after 36 + 0 weeks gestation and babies born thereafter for 1 year. Probabilities, costs, and outcomes were obtained from literature and OptiBreech pilot trial. Uncertainty and value of information were analyzed to prioritize future research. The main outcomes were incremental cost-utility and cost-effectiveness ratios, net benefits, cost-effectiveness acceptability curve, and expected value of perfect and perfect partial information.</p><p><strong>Results: </strong>Using preexisting evidence, OptiBreech care is less effective but sufficiently less costly, ie, cost-effective compared with standard care. Influential parameters include cephalic birth rate after external cephalic version, training costs, and vaginal birth rate after opting for vaginal birth. Emerging evidence for OptiBreech care significantly improved the cost-effectiveness ratio. The expected population value of perfect information was £31.5 million, with utilities identified as key research priority.</p><p><strong>Conclusions: </strong>Planned vaginal birth for singleton breech pregnancy may be cost-effective despite a potential loss in health outcomes. Emerging data on the effectiveness of OptiBreech care significantly increased the cost-effectiveness likelihood. Conclusions did not change for litigation cost assumptions or for sole neonatal perspective. Further research on health utilities would provide a valuable reduction in decision uncertainty.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":" ","pages":""},"PeriodicalIF":4.9,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374887","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 Standardized Measurement and Valuation Scale of Genomic Utility for Policy Decisions: The GUV Scale","authors":"Ilias Goranitis PhD ,&nbsp;Daniel Sheen MSc ,&nbsp;Zoe Fehlberg MPH ,&nbsp;Andrew J. Mallett PhD ,&nbsp;Stephanie Best PhD ,&nbsp;Zornitza Stark PhD","doi":"10.1016/j.jval.2024.11.014","DOIUrl":"10.1016/j.jval.2024.11.014","url":null,"abstract":"<div><h3>Objectives</h3><div>The multifaceted ways in which genomics can be valuable to clinicians, patients, families, and society are important for informing prioritization decisions by policy makers. This study aims to develop a standardized, cumulative, and preference-weighted genomic utility valuation (GUV) on a scale of 0% to 100%.</div></div><div><h3>Methods</h3><div>A multicriteria decision analysis was conducted with experts involved in policy, clinical, research, and consumer advocacy leadership in Australia for the valuation of policy priority indicators of genomic utility. The use of the GUV scale to support policy decisions is illustrated through a stylized example, and benchmark scoring thresholds of genomic utility were identified by mapping evidence from real-world health technology assessments leading to the public reimbursement of genomic testing in Australia onto the GUV scale.</div></div><div><h3>Results</h3><div>In total, 33 (73%) invited experts participated in the study. Clinical utility had the highest priority, followed by societal, diagnostic, economic, and family utilities. Improving health outcomes had the highest preference value (29.5%), followed by improving equity (22.6%), Having high diagnostic yield (22.2%), improving symptom management (15.5%), being cost saving (14.3%), having average diagnostic yield (13.1%), enabling access to clinical trials (12.3%), and enabling reproductive family planning (11.5%). Genomic testing scores from real-world health technology assessments ranged from 46% for syndromic and nonsyndromic intellectual disability to about 60% for mitochondrial conditions and genetic kidney diseases.</div></div><div><h3>Conclusions</h3><div>Comparisons of genomic utility across different clinical contexts may seem difficult because of the multiple criteria required to be weighted to support policy decisions. This comparison is now facilitated in a standardized manner with the GUV scale.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 184-190"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898556","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":"Economic Evaluations of Interventions Addressing Inequalities in Cancer Care: A Systematic Review","authors":"Bedasa Taye Merga MPH ,&nbsp;Nikki McCaffrey PhD ,&nbsp;Suzanne Robinson PhD ,&nbsp;Ebisa Turi MPH ,&nbsp;Anita Lal PhD","doi":"10.1016/j.jval.2024.09.010","DOIUrl":"10.1016/j.jval.2024.09.010","url":null,"abstract":"<div><h3>Objectives</h3><div>Although substantial evidence exists on the costs and benefits of cancer care and screening programs for the general population, economic evidence of interventions addressing inequalities is less well known. This systematic review summarized economic evaluations of interventions addressing inequalities in cancer screening and care to inform decision makers on the value for money of such interventions.</div></div><div><h3>Methods</h3><div>Embase, MEDLINE, Cochrane Library, EconLit, and Scopus databases were searched for studies published from database inception to October 27, 2023. Studies were eligible for inclusion if they were economic evaluations of interventions to improve or address inequalities in cancer care among disadvantaged population groups. Study characteristics and cost-effectiveness results (US dollars 2023) were summarized. Study quality was assessed by 2 authors using the Drummond checklist.</div></div><div><h3>Results</h3><div>The searches yielded 2937 records, with 30 meeting the eligibility criteria for data extraction. In most of the studies (n = 27, 90%), interventions were considered cost-effective in addressing inequalities in cancer care and screening among disadvantaged populations. Notably, 60% of the studies were rated as high quality, 33.3% as good, and 6.7% as fair quality.</div></div><div><h3>Conclusions</h3><div>This systematic review identified cost-effective strategies addressing inequalities in cancer screening and care that have the potential to be replicated in other locations. The interventions were mainly focused on screening programs, and few addressed equity gaps around risk reduction and diagnostic and treatment outcomes. This underscores the need for targeted approaches to address inequalities in under-researched priority population groups along the cancer care continuum.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 306-318"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401460","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 and Quality Assessment of Cardiovascular Disease–Specific Health-Related Quality-of-Life Instruments: Part II Psychometric Properties","authors":"Xue Li PhD ,&nbsp;Rui Li MSc ,&nbsp;Meixuan Li MSc ,&nbsp;Xu Hui MSc ,&nbsp;Jing Li MSc ,&nbsp;Liang Yao PhD ,&nbsp;Harriette Van Spall MD, MPH ,&nbsp;Kun Zhao PhD ,&nbsp;Qiang Fu PhD ,&nbsp;Feng Xie PhD","doi":"10.1016/j.jval.2024.08.011","DOIUrl":"10.1016/j.jval.2024.08.011","url":null,"abstract":"<div><h3>Objectives</h3><div>Health-related quality-of-life instruments for cardiovascular diseases (CVDs) have been commonly used to measure important patient-reported outcomes in clinical trials and practices. This study aimed to systematically identify and evaluate the psychometric properties of CVD-specific health-related quality-of-life instruments.</div></div><div><h3>Methods</h3><div>We searched cumulative index to nursing and allied health literature, Embase, and PubMed from inception to January 20, 2022. Studies that reported psychometric properties of CVD-specific instruments were included. Two reviewers independently assessed the methodological quality using the Consensus-based Standards for the Selection of Health Measurement Instruments methods for evaluating measurement properties and quality of evidence. Seven psychometric properties, including structural validity, internal consistency, test-retest reliability, convergent validity, divergent validity, discriminative validity, and responsiveness, were evaluated.</div></div><div><h3>Results</h3><div>We identified 142 studies reporting psychometric properties of 40 instruments. Five (12.5%) instruments demonstrated measurement properties with sufficient or inconsistent ratings; 16 (40.0%) instruments did not report any responsiveness evidence. Of the 40 instruments, 15 (37.5%) instruments were rated sufficient with high quality of evidence on internal consistency; 4 (10.0%) on structural validity, convergent validity and divergent validity; and 3 (7.5%) on discriminative validity.</div></div><div><h3>Conclusions</h3><div>When measuring patient-reported outcomes in clinical trials or routine practice, it is important to choose instruments with established psychometric properties.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 294-305"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142569713","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":"An Application of the Checklist for Health Economic Quality Evaluations in a Systematic Review Setting","authors":"Stijntje W. Dijk MD, MSc ,&nbsp;Skander Essafi MD ,&nbsp;Myriam G.M. Hunink MD, PhD","doi":"10.1016/j.jval.2024.10.3853","DOIUrl":"10.1016/j.jval.2024.10.3853","url":null,"abstract":"<div><h3>Objective</h3><div>Quality assessment tools serve an important role in evaluating economic evaluations. This article showcases the first application of the Checklist for Health Economic Quality Evaluations (CHEQUE) tool in a systematic review setting and offers descriptive reflections on its use.</div></div><div><h3>Methods</h3><div>We applied CHEQUE to 21 diverse economic evaluations in a systematic review of medical education. We visualized weighted CHEQUE scores, calculate correlations between methods and reporting sections, and provided all data and R code for reuse in future applications. Finally, we provided a detailed overview of our judgments and alternative considerations of the checklist items, and suggestions for further development.</div></div><div><h3>Results</h3><div>Scores ranged from 18% to 94% depending on the applied weighting method, with a positive correlation between the method and reporting quality. CHEQUE enables systematic and standardized assessment but may benefit from refinement in scoring clarity and burden reduction.</div></div><div><h3>Conclusion</h3><div>Our study provides insights for future guidance on applying and developing CHEQUE or similar tools in economic evaluation quality assessment.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 250-259"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142750640","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":"Reporting Uncertainty Around Health-State Values: A Standard Method and Worked Example","authors":"Nancy J. Devlin PhD ,&nbsp;Giselle Abangma MSc ,&nbsp;Andrew Lloyd DPhil ,&nbsp;David Parkin DPhil ,&nbsp;Andrew Briggs DPhil","doi":"10.1016/j.jval.2024.11.010","DOIUrl":"10.1016/j.jval.2024.11.010","url":null,"abstract":"<div><h3>Objectives</h3><div>Articles reporting value sets typically only report the standard errors (SEs) around each estimated coefficient in value set models. This is important information but does not help those building cost-effectiveness models, who need to know the uncertainty around the values of health states to conduct sensitivity analyses. This report’s aim is to demonstrate how SEs around health-related quality of life values can be calculated, using the example of the UK EQ-5D-3L value set.</div></div><div><h3>Methods</h3><div>We show how information from a model’s variance/covariance matrix can be used to estimate SEs for every health-state value, whether it is part of the modeling data set or not. Data from the Measurement and Valuation of Health study were used to replicate the original UK value set and the variance/covariance matrix and to produce SEs around the values for all 243 EQ-5D-3L states.</div></div><div><h3>Results</h3><div>The range of the SEs is small compared with the range of the health-state values but is conditional on a correct model specification and may be sensitive to alternative specifications.</div></div><div><h3>Conclusions</h3><div>Reporting these SEs should become routine practice in reporting value sets, to ensure that users are provided with information on parameter uncertainty. These SEs only capture one specific aspect of the sources of uncertainty around health-related quality of life values but represent a first step toward a more complete account of uncertainty in the preference weights used to estimate quality-adjusted life-years.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 191-196"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142855266","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 Cost-Effectiveness of Frontline Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia: In Pursuit of Treatment-Free Remission and Dose Reduction","authors":"Sanne J.J.P.M. Metsemakers MSc ,&nbsp;Rosella P.M.G. Hermens PhD ,&nbsp;Geneviève I.C.G. Ector PhD ,&nbsp;Nicole M.A. Blijlevens PhD ,&nbsp;Tim M. Govers PhD","doi":"10.1016/j.jval.2024.12.005","DOIUrl":"10.1016/j.jval.2024.12.005","url":null,"abstract":"<div><h3>Objectives</h3><div>The management of chronic myeloid leukemia (CML) now includes dose reduction (DR) and treatment-free remission (TFR). Evaluating the cost-effectiveness of lifelong-prescribed expensive tyrosine kinase inhibitors (TKIs) for CML is crucial. Prior cost-effectiveness evaluations state that imatinib is the favorable frontline TKI. Some of these evaluations address TFR, but not DR, nor aging and second-generation (2G)-TKIs upcoming patent expirations. This study evaluates the cost-effectiveness of frontline TKIs for CML patients including these factors.</div></div><div><h3>Methods</h3><div>This Markov model evaluates the cost-effectiveness of frontline TKIs for newly diagnosed patients with CML using 17 health states. Transition probabilities, costs, and utilities were derived from literature data. Incremental cost-effectiveness ratios were calculated. Sensitivity analysis and model validation were conducted.</div></div><div><h3>Results</h3><div>Nilotinib is most effective (20.13 quality-adjusted life-years [QALYs]) and imatinib is least effective (17.25 QALYs) for the model including TFR and DR. Imatinib was favored over dasatinib (89.80%), nilotinib (62.70%), and bosutinib (78.40%), at a willingness-to-pay threshold of €80 000 per QALY. Without TFR and DR, fewer QALYs were generated. For patients at the age of 70 years, imatinib has a high probability of being more cost-effective than dasatinib, nilotinib, and bosutinib. With 50% 2GTKI cost reductions, nilotinib is considered more cost-effective compared with imatinib (98.40%), dasatinib (94.80%), and bosutinib (68.90%).</div></div><div><h3>Conclusions</h3><div>The findings indicate that 2GTKIs are more effective in generating QALYs, including for older (age &gt;70 years) patients. Given the current TKI prices, imatinib remains cost-effective. Including DR and TFR in CML management generates more QALYs. Cost reductions from expected 2GTKIs patent expirations will greatly increase their cost-effectiveness. Results may inform 2GTKIs cost discussions after patent expiration, potentially broadening global availability. The findings also emphasize the importance of aiming for TFR and DR in CML management.</div></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":"28 2","pages":"Pages 224-232"},"PeriodicalIF":4.9,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142898558","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}