Value in Health最新文献

{"title":"A New and Improved Experimental Design for the Discrete Choice Experiment Module of the EuroQol Valuation Technology Protocol.","authors":"Marcel F Jonker, Bram Roudijk","doi":"10.1016/j.jval.2024.05.024","DOIUrl":"10.1016/j.jval.2024.05.024","url":null,"abstract":"<p><strong>Objectives: </strong>The EuroQol Valuation Technology (EQ-VT) protocol, which is used to value the EQ-5D-5L instrument, comprises a composite time trade-off and a discrete choice experiment (DCE) module. Despite significant limitations, the DCE module has not been updated since its inception in 2012. This study aimed to update the EQ-VT DCE design using state-of-the-art methods.</p><p><strong>Methods: </strong>DCE data from 19 EQ-5D-5L valuation studies were summarized using a Bayesian hierarchical meta-analysis model, which created the priors for our Bayesian efficient DCE design. This design comprised 20 subdesigns, each with 12 choice tasks, and included 2 levels that overlapped to reduce the complexity of the choice tasks. The relative efficiency and robustness of the new design were established by comparing the D-errors and minimal sample size requirements for the 19 within-sample and 7 out-of-sample countries with the previous DCE design.</p><p><strong>Results: </strong>The updated DCE design shows large reductions in the D-error: by 20% and 22% for the 19 within-sample and 7 out-of-sample countries, respectively. Sample size requirements were also reduced, resulting in an average reduction of 45% for both the within and out-of-sample countries.</p><p><strong>Conclusions: </strong>The updated DCE design outperforms the current EQ-VT design. Given its enhanced performance and reduced complexity, it is set to replace the existing DCE design in future EQ-5D-5L valuation studies using the EQ-VT protocol.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559792","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":"Mapping the Lequesne Functional Index Into the EQ-5D-5L Utility Index in Patients With Knee Osteoarthritis.","authors":"Nadia Dardenne, Anne-Françoise Donneau, Olivier Bruyère","doi":"10.1016/j.jval.2024.06.017","DOIUrl":"10.1016/j.jval.2024.06.017","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to map the Lequesne index onto the EuroQol 5 Dimension (EQ-5D-5L) utility index for patients with knee osteoarthritis.</p><p><strong>Methods: </strong>Baseline data from a previous randomized controlled trial were used; 461 patients were involved in the mapping development, and 230 in the validation phase. Various modeling techniques, including generalized linear models, tobit, and beta regression, were used. Factors such as age, sex, and body mass index were considered as covariates. Model selection was based on criteria such as Akaike and Bayesian information criteria, adjusted R², mean absolute error, and root mean squared error. Validation involved assessing the preselected models using mean absolute error, root mean squared error, and intraclass correlation coefficient. This study follows the Mapping Onto Preference-Based Measures Reporting Standards statement.</p><p><strong>Results: </strong>Five models were developed, with 2 incorporating age, sex, with or without body mass index along with the Lequesne index showing the best fit across regressions. Validation results were similar for the 3 regressions, with beta regression models exhibiting wider ranges closer to the validation data set. Intraclass correlation coefficient values were better for beta regression models. Both models tended to overpredict for lower EQ-5D-5L values and underpredict for better health status.</p><p><strong>Conclusion: </strong>These mapping functions, the first of their kind, effectively translate the Lequesne index to EQ-5D-5L values in patients with knee osteoarthritis. They demonstrate satisfactory fit and precision, providing valuable tools for clinicians and researchers, particularly in situations where generic preference-based health-related quality of life instruments are inaccessible for utility derivation in cost-effectiveness studies.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559813","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 Many Monte Carlo Samples are Needed for Probabilistic Cost-Effectiveness Analyses?","authors":"Reza Yaesoubi","doi":"10.1016/j.jval.2024.06.016","DOIUrl":"10.1016/j.jval.2024.06.016","url":null,"abstract":"<p><strong>Objectives: </strong>Probabilistic sensitivity analysis (PSA) is conducted to account for the uncertainty in cost and effect of decision options under consideration. PSA involves obtaining a large sample of input parameter values (N) to estimate the expected cost and effect of each alternative in the presence of parameter uncertainty. When the analysis involves using stochastic models (eg, individual-level models), the model is further replicated P times for each sampled parameter set. We study how N and P should be determined.</p><p><strong>Methods: </strong>We show that PSA could be structured such that P can be an arbitrary number (say, P=1). To determine N, we derive a formula based on Chebyshev's inequality such that the error in estimating the incremental cost-effectiveness ratio (ICER) of alternatives (or equivalently, the willingness-to-pay value at which the optimal decision option changes) is within a desired level of accuracy. We described 2 methods to confirm, visually and quantitatively, that the N informed by this method results in ICER estimates within the specified level of accuracy.</p><p><strong>Results: </strong>When N is arbitrarily selected, the estimated ICERs could be substantially different from the true ICER (even as P increases), which could lead to misleading conclusions. Using a simple resource allocation model, we demonstrate that the proposed approach can minimize the potential for this error.</p><p><strong>Conclusions: </strong>The number of parameter samples in probabilistic cost-effectiveness analyses should not be arbitrarily selected. We describe 3 methods to ensure that enough parameter samples are used in probabilistic cost-effectiveness analyses.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559836","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":"Uncertainty in Long-Term Relative Effectiveness of Medicines in Health Technology Assessment.","authors":"Jan-Willem Versteeg, Rick Vreman, Aukje Mantel-Teeuwisse, Wim Goettsch","doi":"10.1016/j.jval.2024.05.023","DOIUrl":"10.1016/j.jval.2024.05.023","url":null,"abstract":"<p><strong>Objectives: </strong>Uncertainty regarding the long-term relative effectiveness is an important factor in health technology assessment (HTA) of medicines. This study investigated how different HTA bodies address this uncertainty in their assessments.</p><p><strong>Methods: </strong>A total of 49 HTA reports from 6 national HTA bodies, assessing 9 medicines for spinal muscular atrophy, cystic fibrosis, and hypercholesterolemia, were included. In these reports, 81 relative effectiveness assessments and 45 cost-effectiveness assessments were performed on an indication level. We collected information on included trials, assessment outcomes, uncertainty regarding the long-term effectiveness, proposed managed entry agreements, and reassessments.</p><p><strong>Results: </strong>Uncertainty regarding the long-term effectiveness was an important consideration in almost all cost-effectiveness assessments (91%) and three-quarters of relative effectiveness assessments (74%), despite differences in methodologies among HTA bodies. There were considerable differences in the amount and type of long-term effectiveness data included by HTA bodies due to timing and inclusion criteria. In total 23 managed entry agreements were proposed of which 14 were linked to uncertainty regarding the long-term effectiveness. In addition, 13 reassessments were performed of which 4 led to an increase in patient access because of more available long-term effectiveness data.</p><p><strong>Conclusions: </strong>Uncertainty regarding the long-term effectiveness is an important challenge for HTA bodies. There are large differences in the acceptance of evidence among HTA bodies, which leads to heterogeneity in the inclusion of available long-term effectiveness data for decision making. In cases with large uncertainty regarding the long-term effectiveness, outcome-based agreements and reassessments are used by HTA bodies, but differently between HTA bodies and indications.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141545247","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":"Hepatitis C Elimination in Rwanda: Progress, Feasibility, and Economic Evaluation","authors":"Huaiyang Zhong PhD ,&nbsp;Alec Aaron MPH ,&nbsp;Lindsey Hiebert MPH ,&nbsp;Janvier Serumondo MD, MPH ,&nbsp;Yueran Zhuo PhD ,&nbsp;Madeline Adee MPH ,&nbsp;Gallican N. Rwibasira MD, MPH ,&nbsp;John W. Ward MD ,&nbsp;Jagpreet Chhatwal PhD","doi":"10.1016/j.jval.2024.03.005","DOIUrl":"10.1016/j.jval.2024.03.005","url":null,"abstract":"<div><h3>Objectives</h3><p>In 2018, Rwanda launched a national program to eliminate the hepatitis C virus (HCV). We aim to assess the impact of the program to date and identify strategies to achieve the World Health Organization’s HCV elimination goals by 2030.</p></div><div><h3>Methods</h3><p>We developed a microsimulation model to simulate Rwanda’s HCV epidemic from 2015 through 2050 and evaluated temporal trends in HCV infection, prevalence, mortality, and the total cost of care for scenarios that could achieve HCV elimination by 2030.</p></div><div><h3>Results</h3><p>Between 2018 and 2022, over 7 million people were screened for HCV, and 60 000 were treated. The study projected that Rwanda could achieve HCV elimination as early as 2027. A feasible strategy of an annual screening rate of 15% and a treatment rate of 100% would achieve all World Health Organization elimination goals by 2028, requiring screening an additional 4 million people and treating 23 900 patients by 2030. The elimination strategy costs $25 million for screening and diagnosis and $21 million for treatment from 2015 to 2050. The national program would avert 4900 hepatocellular carcinoma cases and 6700 HCV-related deaths and save the health system $25.33 million from 2015 to 2050.</p></div><div><h3>Conclusions</h3><p>Rwanda is poised to become one of the first countries in the world to eliminate HCV. Rwanda’s program serves as a blueprint for other countries in the African region. By rapid screening and treatment scale-up (eg, by leveraging HIV platforms) and by drug price negotiations, HCV elimination is not only feasible but can be cost-saving in low-income settings.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140140810","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":"Challenges of Incorporating Life Cycle Drug Pricing in Cost-Effectiveness Models: A Review of Methods and Modeling Suggestions","authors":"Mathilde Puls MPH ,&nbsp;James Horscroft PhD ,&nbsp;Benjamin Kearns PhD ,&nbsp;Daniel Gladwell PhD ,&nbsp;Edward Church MSc ,&nbsp;Kasper Johannesen PhD ,&nbsp;Bill Malcolm MSc ,&nbsp;John Borrill MSc","doi":"10.1016/j.jval.2024.03.006","DOIUrl":"10.1016/j.jval.2024.03.006","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aimed to conduct a review of existing methods used to incorporate life cycle drug pricing (LCDP) in cost-effectiveness analyses (CEAs), identify common methodological challenges, and suggest modeling approaches for prospectively implementing LCDP in CEA.</p></div><div><h3>Methods</h3><p>Two complementary searches were conducted in PubMed, combined with hand searching and reference mining, to identify English language full-text articles that explored (1) how drug prices change over time and (2) methods used to apply dynamic pricing in cost-effectiveness models (CEMs). Relevant articles were reviewed, and authors discussed the common methodological practices used in the literature and their associated challenges on prospectively implementing LCDP in CEMs. For each key challenge identified, we provide modeling suggestions to address the issue.</p></div><div><h3>Results</h3><p>We screened 1200 studies based on title and abstract; 117 were reviewed for eligibility, and 47 individual studies were included across both searches. Variations in prices over a product’s life cycle are complex and multifactorial, and models applying LCDP in CEA varied in their methodology. We identified 4 key challenges to modeling LCDP in CEA, including how to model price trends before and after loss of exclusivity, how to capture the effect of price changes on future patient cohorts, and how to report results.</p></div><div><h3>Conclusion</h3><p>Accurately quantifying the impact of LCDP requires careful consideration of multiple aspects pertaining to both the evolution of drug prices and how to reflect these in CEA. Although uncertainties remain, our findings can aid implementation and evaluation of LCDP in economic evaluations.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524001219/pdfft?md5=a1a4a85a51a5f3ce2a5d34d747d40188&pid=1-s2.0-S1098301524001219-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140185706","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":"Flexible Approaches Based on Multistate Models and Microsimulation to Perform Real-World Cost-Effectiveness Analyses: An Application to Proprotein Convertase Subtilisin-Kexin Type 9 Inhibitors","authors":"Caterina Gregorio PhD ,&nbsp;Federico Rea PhD ,&nbsp;Francesca Ieva PhD ,&nbsp;Arjuna Scagnetto MS ,&nbsp;Carla Indennidate MD ,&nbsp;Chiara Cappelletto MD ,&nbsp;Andrea Di Lenarda MD ,&nbsp;Giulia Barbati PhD","doi":"10.1016/j.jval.2024.03.008","DOIUrl":"10.1016/j.jval.2024.03.008","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aims to show the application of flexible statistical methods in real-world cost-effectiveness analyses applied in the cardiovascular field, focusing specifically on the use of proprotein convertase subtilisin-kexin type 9 inhibitors for hyperlipidemia.</p></div><div><h3>Methods</h3><p>The proposed method allowed us to use an electronic health database to emulate a target trial for cost-effectiveness analysis using multistate modeling and microsimulation. We formally established the study design and provided precise definitions of the causal measures of interest while also outlining the assumptions necessary for accurately estimating these measures using the available data. Additionally, we thoroughly considered goodness-of-fit assessments and sensitivity analyses of the decision model, which are crucial to capture the complexity of individuals’ healthcare pathway and to enhance the validity of this type of health economic models.</p></div><div><h3>Results</h3><p>In the disease model, the Markov assumption was found to be inadequate, and a “time-reset” timescale was implemented together with the use of a time-dependent variable to incorporate past hospitalization history. Furthermore, the microsimulation decision model demonstrated a satisfying goodness of fit, as evidenced by the consistent results obtained in the short-term horizon compared with a nonmodel-based approach. Notably, proprotein convertase subtilisin-kexin type 9 inhibitors revealed their favorable cost-effectiveness only in the long-term follow-up, with a minimum willingness to pay of 39 000 Euro/life years gained.</p></div><div><h3>Conclusions</h3><p>The approach demonstrated its significant utility in several ways. Unlike nonmodel-based or alternative model-based methods, it enabled to (1) investigate long-term cost-effectiveness comprehensively, (2) use an appropriate disease model that aligns with the specific problem under study, and (3) conduct subgroup-specific cost-effectiveness analyses to gain more targeted insights.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524001232/pdfft?md5=f9fb5965542cc82a7d9d549e2fdf6fab&pid=1-s2.0-S1098301524001232-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140319343","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":"Values Beyond “Health” in Budget-Constrained Healthcare Systems","authors":"Charles E. Phelps PhD","doi":"10.1016/j.jval.2024.02.005","DOIUrl":"10.1016/j.jval.2024.02.005","url":null,"abstract":"<div><h3>Objectives</h3><p>Most current methods to value healthcare treatments only incorporate measures such as quality-adjusted life-years, combining gains in health-related quality of life and life expectancy in specific ways. Failure of these methods to recognize other dimensions of value has led to calls for methods to include additional values that are associated with the healthcare treatments but not captured directly by quality-adjusted life-years. This article seeks to provide methodologically sound ways to incorporate additional health-related outcomes, focusing on budget-constrained healthcare systems, in which using standard welfare economics methods are often eschewed.</p></div><div><h3>Methods</h3><p>The analysis develops standard extra-welfarist approaches to maximizing aggregate health, subject to fixed-budget constraints, using Lagrange multiplier methods. Then, additional valuable health-related outcomes, eg, reduced caregiver burden, real option value, and market- and non-market productivity are introduced. The article also introduces a social welfare function approach to illuminate how disability, disease severity and other equity-related issues can be incorporated into complete welfare measures.</p></div><div><h3>Results</h3><p>Resulting analysis, fully developed in an <span>Appendix</span> in <span>Supplemental Materials</span> found at <span>https://doi.org/10.1016/j.jval.2024.02.005</span><svg><path></path></svg> and summarized in the main text, show that understanding how average and marginal healthcare costs increase with output and how health augments “additional values” provides ways to assess willingness to pay for them in these fixed-budget situations.</p></div><div><h3>Conclusions</h3><p>In budget-constrained healthcare systems, only from actual budget allocations can values both of health itself and “additional values” be inferred. These methods, combined with methodologically sound social welfare functions, demonstrate how to move from “health” to “welfare” in measuring the value of increased healthcare use.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524000755/pdfft?md5=1af48aa44aab1ef4692cb9ef226eebd6&pid=1-s2.0-S1098301524000755-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139917523","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":"Subjective Well-Being Population Norms and Inequalities in Hungary: A Large Cross-Sectional, Internet-Based Survey","authors":"Thao T.P. Nguyen MD, MSc ,&nbsp;Fanni Rencz MD, PhD ,&nbsp;Valentin Brodszky MD, PhD","doi":"10.1016/j.jval.2024.04.005","DOIUrl":"10.1016/j.jval.2024.04.005","url":null,"abstract":"<div><h3>Objectives</h3><p>This study aimed to provide subjective well-being (SWB) population norms in Hungary and explore the contribution of explanatory factors of SWB inequality among the Hungarian adult general population.</p></div><div><h3>Methods</h3><p>The data originated from a large representative internet-based cross-sectional survey in Hungary, which was conducted in 2020. We applied validated multi-item instruments for measuring SWB, namely Satisfaction With Life Scale (SWLS) and World Health Organization-Five Well-Being Index (WHO-5). Multiple linear regressions were used to examine the relationship between demographic-socioeconomic-health status and both well-being instruments. The concentration index (CI) was used to measure the degree of income-related inequality in well-being.</p></div><div><h3>Results</h3><p>A total of 2001 respondents were enrolled with the means ± SD WHO-5 scores and SWLS scores of 0.51 ± 0.21 and 0.51 ± 0.23, respectively. Higher household income, higher educational level, better general health status, and absence of chronic morbidity were significant positive predictors for both WHO-5 and SWLS scores. The CI of WHO-5 scores was lower than that of SWLS scores in the total sample (0.0480 vs 0.0861) and in subgroups by gender (male, 0.0584 vs 0.1035; female, 0.0302 vs 0.0726). The positive CI values implied a slight pro-rich SWB inequality in this population. The regression analyses showed a positive association of SWB with having a higher household income and a better general health status.</p></div><div><h3>Conclusions</h3><p>This is the first representative study in Hungary to compare population norm of 2 well-being instruments and analyze well-being inequality. Slight pro-rich inequality was found consistently with both SWB measures. Our findings support the need for health and social policies that effectively tackle inequalities in Hungary.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140788923","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":"Testing the Psychometric Properties of 9 Bolt-Ons for the EQ-5D-5L in a General Population Sample","authors":"Fanni Rencz PhD ,&nbsp;Mathieu F. Janssen PhD","doi":"10.1016/j.jval.2024.03.2195","DOIUrl":"10.1016/j.jval.2024.03.2195","url":null,"abstract":"<div><h3>Objectives</h3><p>We aimed to assess the psychometric performance and added value of 9 existing bolt-ons (breathing problems, cognition, hearing, self-confidence, skin irritation, sleep, social relationships, tiredness, and vision) for the EQ-5D-5L in a general population sample.</p></div><div><h3>Methods</h3><p>The EQ-5D-5L, 9 bolt-ons, SF-6Dv1, Patient-Reported Outcomes Measurement Information System (PROMIS)-29+2, PROMIS Global Health, and Satisfaction with Life Scale were completed in an online cross-sectional survey among a general adult population sample in Hungary (n = 1587). The following psychometric properties were tested for the EQ-5D-5L + bolt-on(s): ceiling, divergent and convergent validity, structural validity, known-group validity, and explanatory power.</p></div><div><h3>Results</h3><p>Adding sleep (30%), tiredness (24%), or vision (21%) substantially reduced the ceiling of the EQ-5D-5L (41%). Cognition, sleep, social relationships, and tiredness correlated with corresponding PROMIS and SF-6D items (r_s = ǀ0.32ǀ − ǀ0.73ǀ). All bolt-ons, except cognition and self-confidence, loaded on a different factor from the EQ-5D-5L dimensions. Breathing, hearing, skin irritation, and vision significantly improved known-group validity in relevant health condition groups. The sleep bolt-on improved known-group validity in 9 of 13 chronic health conditions. Tiredness had the largest impact on explaining EQ VAS score variance in 8 of 13 conditions. Hearing and vision improved the ability of the EQ-5D-5L to capture declining health with age, whereas self-confidence and social relationships were valuable for mental health assessment.</p></div><div><h3>Conclusions</h3><p>This study established the validity of multiple bolt-ons for the EQ-5D-5L and highlights the usefulness of including relevant bolt-ons in population-based and patient surveys. Our findings inform the further development of these bolt-ons and the bolt-on item selection for clinical studies.</p></div>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1098301524023106/pdfft?md5=b2dc0c53c2eb70f2d7b462fbba7ada94&pid=1-s2.0-S1098301524023106-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140784589","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}