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Objective Methods Results Significance Health state utilities are required to obtain quality adjusted life years, a common metric that informs clinical decision‐making at individual, group, and health policy levels. Health state utilities are different from health‐related quality of life, and their distribution across patients with epilepsy, as well as the factors that impact them, have not been studied in depth. We aimed to describe the distribution of health state utilities in people with epilepsy and the impact of different combinations of clinical and demographic factors on health state evaluation.We performed a retrospective analysis of patients' data prospectively collected in the Calgary Comprehensive Epilepsy Program registry. Patient‐reported health state utilities were measured using the 5‐level EuroQol 5‐Dimension scale (EQ‐5D‐5L) completed at their initial assessment. EQ‐5D‐5L index scores were derived via the time trade‐off approach based on Canadian norms, and their distribution across different health states and patient characteristics was obtained. The Tobit regression model was used to evaluate the determinants of EQ‐5D‐5L index scores.Of 1446 patients included in this analysis, 724 (50.5%) were female. The median (interquartile range) Canada‐normed EQ‐5D‐5L index score was .87 (.71–.91). Patients with significantly lower health utilities were more likely to be female (p = .008), to be older (p = .034), to be unmarried (p = .013), to have failed to achieve 1‐year seizure freedom (p < .001), to have no postsecondary education (p = .028), to be depressed (p < .001), to have antiseizure medication side effects (p = .001), to be unemployed (p < .001), and to be unable to drive (p < .001). A look‐up table of health utilities based on combinations of clinical–demographic characteristics was produced.Health utility estimates for combinations of different health states in people with epilepsy attending specialty clinics are now available. These can help guide clinical decision‐making in routine clinical practice, economic evaluations of treatment interventions, and health care policies. [ABSTRACT FROM AUTHOR]

Abstract Background Individuals with transfusion-dependent β-thalassemia (TDT) experience symptoms and functional impacts that reduce their health-related quality of life. However, EQ-5D-derived health utility index scores in TDT often indicate good HRQoL, suggesting the EQ-5D may not adequately capture the impact of TDT. This study explored the disease and treatment burden of TDT and examined the appropriateness of the EQ-5D-5L descriptive system (DS) in measuring HRQoL in TDT. Methods Adults with TDT in the United Kingdom, United States, and France completed a background questionnaire and EQ-5D-5L DS, followed by 60-minute semi-structured interviews on symptoms and HRQoL impacts of TDT (concept elicitation) and appropriateness of EQ-5D-5L DS (cognitive debrief). Transcribed interviews were analyzed using thematic and content analyses. The relationship between TDT symptoms and impacts were summarized in a conceptual model. EQ-5D-5L DS was mapped to concepts identified in the qualitative data to assess its capture of HRQoL concepts. Participants’ EQ-5D-5L DS scores were compared to their qualitative descriptions for each dimension to assess their concordance. Results Thirty participants in the United States (n = 14 [46.7%]), United Kingdom. (n = 12 [40.0%]), and France (n = 4 [13.3%]) completed the study (73.3% female; mean age = 28.4 years [standard deviation (SD) = 5.1]; mean annual red blood cell transfusion [RBCT] frequency = 18.4 [SD = 7.6]). Participants reported TDT symptoms and impacts on HRQoL, all fluctuating across the RBCT cycle. EQ-5D-5L DS did not fully capture 11 of 16 (68.8%) HRQoL concepts reported. Most participants (n = 20/27 [74.1%]) reported that EQ-5D-5L DS did not capture important aspects of living with TDT, and 42.9% (n = 12/28) reported negative/neutral overall impressions of EQ-5D-5L DS. The highest degree of discordance between participants’ qualitative data and EQ-5D-5L DS dimension scores was observed with mobility (42.3%) and self-care (34.6%), where the qualitative descriptions relating to these dimensions were worse than their quantitative scores. Conclusion Current findings suggest that EQ-5D-5L DS lacks content validity and the derived health utility index score may not fully represent the burden of disease in TDT.

Background: Individuals with transfusion-dependent β-thalassemia (TDT) experience symptoms and functional impacts that reduce their health-related quality of life. However, EQ-5D-derived health utility index scores in TDT often indicate good HRQoL, suggesting the EQ-5D may not adequately capture the impact of TDT. This study explored the disease and treatment burden of TDT and examined the appropriateness of the EQ-5D-5L descriptive system (DS) in measuring HRQoL in TDT. Methods: Adults with TDT in the United Kingdom, United States, and France completed a background questionnaire and EQ-5D-5L DS, followed by 60-minute semi-structured interviews on symptoms and HRQoL impacts of TDT (concept elicitation) and appropriateness of EQ-5D-5L DS (cognitive debrief). Transcribed interviews were analyzed using thematic and content analyses. The relationship between TDT symptoms and impacts were summarized in a conceptual model. EQ-5D-5L DS was mapped to concepts identified in the qualitative data to assess its capture of HRQoL concepts. Participants' EQ-5D-5L DS scores were compared to their qualitative descriptions for each dimension to assess their concordance. Results: Thirty participants in the United States (n = 14 [46.7%]), United Kingdom. (n = 12 [40.0%]), and France (n = 4 [13.3%]) completed the study (73.3% female; mean age = 28.4 years [standard deviation (SD) = 5.1]; mean annual red blood cell transfusion [RBCT] frequency = 18.4 [SD = 7.6]). Participants reported TDT symptoms and impacts on HRQoL, all fluctuating across the RBCT cycle. EQ-5D-5L DS did not fully capture 11 of 16 (68.8%) HRQoL concepts reported. Most participants (n = 20/27 [74.1%]) reported that EQ-5D-5L DS did not capture important aspects of living with TDT, and 42.9% (n = 12/28) reported negative/neutral overall impressions of EQ-5D-5L DS. The highest degree of discordance between participants' qualitative data and EQ-5D-5L DS dimension scores was observed with mobility (42.3%) and self-care (34.6%), where the qualitative descriptions relating to these dimensions were worse than their quantitative scores. Conclusion: Current findings suggest that EQ-5D-5L DS lacks content validity and the derived health utility index score may not fully represent the burden of disease in TDT. [ABSTRACT FROM AUTHOR]

Introduction: Utility values are used in health economic modeling analyses of type 2 diabetes (T2D) to quantify the effect of acute and long-term complications on quality of life (QoL). For accurate modeling projections, it is important that the utility values used are up to date, accurate and representative of the simulated model cohort. Methods: A literature review was performed to identify utility values for health states representing acute and chronic T2D-related complications including cardiovascular complications, stroke, renal disease, ophthalmic complications, neuropathy, diabetic foot, amputation and hypoglycemia. Searches were performed using the PubMed, Embase and Cochrane Library databases and limited to articles published since 2010. Supplementary searches were performed to identify data published at congresses in 2019–2023. Results: A total of 54 articles were identified that reported utility values for T2D-related complications. The most frequently used elicitation method/instrument was the EQ-5D (n = 42 studies) followed by the Short Form-6 dimensions (n = 6), time tradeoff (n = 5), the Health Utilities Index Mark 2 or Mark 3 (n = 2), 15D (n = 1), visual analog scale (n = 1) and standard gamble (n = 1). Stroke and amputation were consistently associated with the largest decrements in QoL. There is a lack of published data that distinguishes between severity of several complications including renal disease, retinopathy and neuropathy. Conclusions: Diabetes-related complications can have a profound impact on QoL; therefore, it is important that these are captured accurately and appropriately in health economic models. Recently published utility values for diabetes-related complications that can be used to inform health economic models are summarized here. [ABSTRACT FROM AUTHOR]

Objectives: Utility scores associated with preference-based health-related quality-of-life instruments such as the EQ-5D-3L are reported as point estimates. In this study, we develop methods for capturing the uncertainty associated with the valuation study of the UK EQ-5D-3L that arises from the variability inherent in the underlying data, which is tacitly ignored by point estimates. We derive a new tariff that properly accounts for this and assigns a specific closed-form distribution to the utility of each of the 243 health states of the EQ-5D-3L. Methods: Using the UK EQ-5D-3L valuation study, we used a Bayesian approach to obtain the posterior distributions of the derived utility scores. We constructed a hierarchical model that accounts for model misspecification and the responses of the survey participants to obtain Markov chain Monte Carlo (MCMC) samples from the posteriors. The posterior distributions were approximated by mixtures of normal distributions under the Kullback–Leibler (KL) divergence as the criterion for the assessment of the approximation. We considered the Broyden–Fletcher–Goldfarb–Shanno (BFGS) algorithm to estimate the parameters of the mixture distributions. Results: We derived an MCMC sample of total size 4,000 × 243. No evidence of nonconvergence was found. Our model was robust to changes in priors and starting values. The posterior utility distributions of the EQ-5D-3L states were summarized as 3-component mixtures of normal distributions, and the corresponding KL divergence values were low. Conclusions: Our method accounts for layers of uncertainty in valuation studies, which are otherwise ignored. Our techniques can be applied to other instruments and countries' populations. Highlights: Guidelines for health technology assessments typically require that uncertainty be accounted for in economic evaluations, but the parameter uncertainty of the regression model used in the valuation study of the health instrument is often tacitly ignored. We consider the UK valuation study of the EQ-5D-3L and construct a Bayesian model that accounts for layers of uncertainty that would otherwise be disregarded, and we derive closed-form utility distributions. The derived tariff can be used by researchers in economic evaluations, as it allows analysts to directly sample a utility value from its corresponding distribution, which reflects the associated uncertainty of the utility score. [ABSTRACT FROM AUTHOR]

Ralph HB Benedict,1 Pamela Vo,2 Nicholas Adlard,2 Olwyn Grennan,3 Ashley Enstone,4 Daisy Bridge,4 Robin Wyn,4 Stanley L Cohan5 1Department of Neurology, Jacobs School of Medicine and Biomedical Sciences, Buffalo, NY, USA; 2Novartis Pharma AG, Basel, Switzerland; 3Novartis Ireland, Dublin, Ireland; 4Adelphi Values PROVE, Bollington, UK; 5Providence Brain and Spine Institute, Providence St. Joseph Health, Portland, OR, USACorrespondence: Pamela Vo, Email pamela.vo@novartis.comIntroduction: Cognitive impairment, especially relating to cognitive processing speed, is a major cause of disability in people with multiple sclerosis (MS). Utility values are quantitative estimates of the quality of life experienced in specific health states and are a key component of cost-effectiveness modelling. However, existing health state utility values in MS typically focus on physical ability and are generally derived using generic (not disease-specific) measures of quality of life. The objective of the current study was to generate health state utility values for levels of cognitive impairment. We used a direct utility elicitation approach called the time trade-off (TTO) methodology.Materials and Methods: Health state descriptions were created following interviews with healthcare professionals, patients, and caregivers in the United States (n=35), and with healthcare professionals in the UK (n=5). Three health states (mild, moderate, and severe impairment) were defined based upon a well-established and validated test for cognitive dysfunction called the Symbol Digit Modalities Test (SDMT) and described using qualitative interview findings. Next, interviews with members of the general public in the UK were conducted to estimate utility values for each health state using the TTO methodology. The procedure was based on the established Measurement and Valuation of Health (MVH) protocol, which generates values on a scale from 0.0 to 1.0.Results: Mean health state utility values were 0.77 ± 0.24 in “mild impairment” (SDMT 43-40), 0.57 ± 0.26 in “moderate impairment” (SDMT 39-32), and 0.34 ± 0.28 in “severe impairment” (SDMT ≤ 31).Discussion: Results indicate that the public perceives that health states of cognitive slowing (as observed in MS) are associated with a substantial reduction in affected individuals’ health-related quality of life, quantified using the TTO methodology. Future economic modeling should consider how utility impacts of both cognitive and physical disability can be appropriately incorporated.Keywords: cognitive impairment, processing speed, health state utility, health-related quality of life, multiple sclerosis, time trade-off

Background: The increasing interest in early identification of people at risk of developing dementia, has led to the development of numerous models aimed at estimating the likelihood of progression from mild cognitive impairment (MCI) to dementia. It is important to study both the need for and possible outcomes related with such prediction models, including the impact of risk predictions on perceived quality of life (QoL). Objective: This study aimed to quantify the impact that receiving a risk prediction on progression from MCI to dementia has on QoL. Methods: A Discrete Choice Experiment (DCE) and Time Trade Off (TTO) study were performed. Participants completed choice tasks related to dementia prognosis while imagining having MCI. We collected DCE data by an online survey, and TTO data via videoconferencing interviews. DCE data were analyzed using a mixed multinomial logit model and were anchored to a health state utility scale using mean observed TTO valuations. Results: 296 people participated in the DCE and 42 in the TTO. Moderate and high predicted dementia risks were associated with decrements in utility (–0.05 and –0.18 respectively), compared to no prognostic information. Low predicted risk was associated with an increase in utility (0.06), as well as the availability of medication or lifestyle interventions (0.05 and 0.13 respectively). Conclusions: This study shows a significant impact of dementia risk predictions on QoL and highlights the importance of caution when sharing information about expected MCI disease courses. [ABSTRACT FROM AUTHOR]

Purpose: Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) is a chronic condition with a constellation of symptoms presenting as severe and profound fatigue of ≥ 6 months not relieved by rest. ME/CFS affects health-related quality of life (HRQoL), which can be measured using multi-attribute health state utility (HSU) instruments. The aims of this study were to quantify HSUs for people living with ME/CFS, and to identify an instrument that is preferentially sensitive for ME/CFS. Methods: Cross-sectional national survey of people with ME/CFS using the AQoL-8D and EQ-5D-5L. Additional questions from the AQoL-8D were used as 'bolt-ons' to the EQ-5D-5L (i.e., EQ-5D-5L-Psychosocial). Disability and fatigue severity were assessed using the De Paul Symptom Questionnaire-Short Form (DSQ-SF). HSUs were generated using Australian tariffs. Mean HSUs were stratified for sociodemographic and clinical factors. Bland–Altman plots were used to compare the three HSU instruments. Results: For the 198 participants, mean HSUs (95% confidence intervals) were EQ-5D-5L: 0.46 (0.42–0.50); AQoL-8D: 0.43 (0.41–0.45); EQ-5D-5L-Psychosocial: 0.44 (0.42–0.46). HSUs were substantially lower than population norms: EQ-5D-5L: 0.89; AQoL-8D: 0.77. As disability and fatigue severity increased, HSUs decreased in all three instruments. Bland–Altman plots revealed interchangeability between the AQoL-8D and EQ-5D-5LPsychosocial. Floor and ceiling effects of 13.5% and 2.5% respectively were observed for the EQ-5D-5L instrument only. Conclusions: ME/CFS has a profound impact on HRQoL. The AQoL-8D and EQ-5D-5L-Psychosocial can be used interchangeably: the latter represents a reduced participant burden. [ABSTRACT FROM AUTHOR]

Abstract Background The PROMIS Preference score (PROPr) is a new health state utility (HSU) score that aims to comprehensively incorporate the biopsychosocial model of health and apply favorable psychometric properties from the descriptive PROMIS system to HSU measurements. However, minimal evidence concerning comparisons to the EQ-5D-3L and the PROPr’s capability to differentiate clinical severity are available. Therefore, the aim of this study was to compare the PROPr to the EQ-5D-3L in terms of scale agreement, ceiling/floor effects, distribution, construct validity, discriminatory power, and relative efficiency (RE) in terms of the Oswestry Disability Index (ODI) for patients with low back pain (LBP). Methods We used intra-class correlation coefficients (ICC) and Bland–Altman plots to compare the PROPr and EQ-5D-3L with regared to scale agreement in a cross-sectional routine sample of LBP patients. For distribution, we used the Pearson’s coefficient for skewness and for ceiling/floor effects, a 15%-top/bottom threshold. For convergent validity, we used Pearson’s correlation coefficients. For known-groups validity, we applied a linear regression with interaction terms (predictors sex, age, and ODI level) and an analysis of variance (ANOVA). For discriminatory power, we calculated the effect size (ES) using Cohen’s d and the ratio of the area under the receiver-operating characteristics curves (AUROC-ratio = AUROCPROPr/AUROCEQ-5D-3L). RE was measured using the ratio of F-values (RE = FPROPr/FEQ-5D-3L). Results Of 218 LBP patients, 50.0% were female and the mean age was 61.8 years. The mean PROPr (0.20, 95%CI: 0.18; 0.22) and EQ-5D-3L scores (0.55, 95%CI: 0.51; 0.58) showed low agreement (d = 0.35, p ESPROPr and RE 1 in lower ODI severity. AUROC-ratios did not show significant differences in terms of ODI severity. Conclusions All PROPr and EQ-5D-3L biopsychosocial dimensions of health showed impairment in LPB patients. The capability of EQ-5D-3L and PROPr to differentiate ODI levels depends on ODI severity. Joint application of both tools may provide additional information.

Pharmacoeconomic evaluation is the essential supporting information for the inclusion of rare disease drugs into medical insurance in China. The accurate measurement of the health state utility of rare diseas is of practical significance to the development of rare disease pharmacoeconomic evaluation. Based on the review of pharmacoeconomic evaluation requirements for rare diseases in some countries/regions, we systematically retrieved the published studies on the measurement of health state utility for 121 rare diseases in China and other countries and regions. We identified 17 591 papers in the initial review, and later selected 230 after screening. We also made a comprehensive analysis of the quality of literature, evaluation content and use of tools for measuring health state utility in rare diseases in China. Finally, we analyzed the challenges in measurement in terms of population, instruments use, and application of results and made recommendations based on analysis, hoping to provide reference for the development of rare disease health state utility measurement studies in China.

Abstract Background The increased prevalence of myopia creates and earlier age of onset has created public health concerns for the long-term eye health, vision impairment and carries with it a significant economic burden. The quality of the economic evaluation is dependent on the sensitivity and validity of the approaches. Nowadays, there are many approaches to measure patients’ health state utility (HSU). However, little is known regarding the performance of direct approach and indirect approach in people with myopia. This study is aimed to compare the psychometric properties of four HSU approaches among patients with myopia in mainland China, including two direct approaches (TTO and SG), the generic preference-based measures (PBM) (AQoL-7D) and the disease-specific PBM (VFQ-UI). Methods A convenience sampling framework was used to recruit patients with myopia who attended a large ophthalmic hospital in Jinan, China. Spearman’s rank correlations coefficient was used to assess concurrent validity. Known-group validity was analyzed by: (1) whether the patients wear corrective devices; (2) severity of myopia as low or moderate to high of the better eye; (3) duration of myopia as ≤ 10 years or > 10 years. Effect size (ES), relative efficiency (RE) statistic and the largest area under the receiver operating characteristic curve (AUC) were used to assess sensitivity. The intra-class correlation coefficient (ICC) and Bland–Altman plots were used to assess agreement. Results A valid sample size of 477 myopia patients was analyzed (median duration: 10 years). The mean HSU scores between TTO and SG were similar (0.95) and higher than AQoL-7D (0.89) and VFQ-UI (0.83). Overall, the VFQ-UI had the best performance based on the psychometric analysis. The agreement indicated that there was no pair of approaches that could be used interchangeably. Conclusions The VFQ-UI showed better psychometric properties than other three approaches for providing health state utility in Chinese myopia patients. Given the widespread use and its generic nature of the AQoL-7D, it could be used alongside with VFQ-UI to provide complementary health state utility from a generic and disease-specific perspective for economic evaluation. More evidence on the responsiveness of four health utility approaches in myopia patients is required.

Purpose: Measuring health-related quality of life (HRQoL) in ovarian cancer patients is critical to understand the impact of disease and treatment. Preference-based HRQoL measures, called health state utilities, are used specifically in health economic evaluations. Real-world patient-reported data on HRQoL and health state utilities over the long-term course of ovarian cancer are limited. This study aims to determine HRQoL and health state utilities in different health states of ovarian cancer. Methods: This cross-sectional, multicenter study included patients with stage III-IV ovarian cancer in six health states: at diagnosis, during chemotherapy, after cytoreductive surgery (CRS), after chemotherapy, in remission, and at first recurrence. HRQoL was measured using the European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire C30, and the ovarian cancer-specific module OV28. Health state utilities were assessed using the EuroQol five-dimension five-level (EQ-5D-5L) questionnaire. Descriptive analyses were performed for each health state. Results: Two hundred thirty-two patients participated, resulting in 319 questionnaires. Median age was 66 years. The lowest HRQoL was observed during chemotherapy and shortly after CRS. Physical and role functioning were most affected and the highest symptom prevalence was observed in the fatigue, nausea, pain, dyspnea, gastrointestinal, neuropathy, attitude, and sexuality domains. Patients in remission had the best HRQoL. Mean utility values ranged from 0.709 (±0.253) at diagnosis to 0.804 (±0.185) after chemotherapy. Conclusions: This study provides clinicians with a valuable resource to aid in patient counseling and clinical decision-making. The utilities, in particular, are crucial for researchers conducting economic analyses to inform policy decisions.

Background: In valuation studies of the EQ-5D-5L instrument, the composite time tradeoff method (cTTO) is often used to elicit preferences. In cTTO, some health states are considered worse than dead (WTD) and are assigned negative utility values. However, these negative values correlate poorly with state severity, which suggests that cTTO is insufficiently sensitive. A recent threshold explanation has been offered to account for the lack of correlation: because the severity threshold beyond which a state is considered WTD differs between respondents, the correlation should be studied for individual respondents clustered by the number of WTD states. The results obtained in such a threshold approach were interpreted to disprove the insensitivity of the cTTO method. Aim: To scrutinize the threshold explanation and test whether it indeed refutes the insensitivity of cTTO. Methods: The study uses data from the EQ-5D-5L Polish valuation study, which includes cTTO responses from 1,510 participants, each of whom evaluated 10 EQ-5D-5L states. The correlation analysis and threshold approach are repeated to confirm the results from previous studies. The data are then modified in 2 contrasting ways. First, negative utilities are randomly reshuffled to test whether the threshold approach can capture cTTO insensitivity. Second, individual-level regressions are used to simulate negative values to ensure they correlate with severity at the individual respondent level, verifying whether the overall severity-utility correlation should be observed. Results: First, reshuffling negative utilities does not change the results of the threshold approach. Hence, the threshold explanation fails to prove cTTO sensitivity. Second, when sensitivity was introduced on an individual level, a significant overall correlation between severity and negative utility arose. Conclusion: cTTO is insensitive to severity for WTD states. Highlights: For the composite time tradeoff method, the utility values of health states worse than dead correlate poorly with state severity, which suggests that cTTO has insufficient sensitivity. Recently, a so-called threshold explanation was offered for the lack of correlation. I show why the threshold explanation fails and why the composite time tradeoff is indeed insensitive for worse-than-dead states. [ABSTRACT FROM AUTHOR]

Background: The PROMIS Preference score (PROPr) is a new health state utility (HSU) score that aims to comprehensively incorporate the biopsychosocial model of health and apply favorable psychometric properties from the descriptive PROMIS system to HSU measurements. However, minimal evidence concerning comparisons to the EQ-5D-3L and the PROPr's capability to differentiate clinical severity are available. Therefore, the aim of this study was to compare the PROPr to the EQ-5D-3L in terms of scale agreement, ceiling/floor effects, distribution, construct validity, discriminatory power, and relative efficiency (RE) in terms of the Oswestry Disability Index (ODI) for patients with low back pain (LBP). Methods: We used intra-class correlation coefficients (ICC) and Bland–Altman plots to compare the PROPr and EQ-5D-3L with regared to scale agreement in a cross-sectional routine sample of LBP patients. For distribution, we used the Pearson's coefficient for skewness and for ceiling/floor effects, a 15%-top/bottom threshold. For convergent validity, we used Pearson's correlation coefficients. For known-groups validity, we applied a linear regression with interaction terms (predictors sex, age, and ODI level) and an analysis of variance (ANOVA). For discriminatory power, we calculated the effect size (ES) using Cohen's d and the ratio of the area under the receiver-operating characteristics curves (AUROC-ratio = AUROCPROPr/AUROCEQ-5D-3L). RE was measured using the ratio of F-values (RE = FPROPr/FEQ-5D-3L). Results: Of 218 LBP patients, 50.0% were female and the mean age was 61.8 years. The mean PROPr (0.20, 95%CI: 0.18; 0.22) and EQ-5D-3L scores (0.55, 95%CI: 0.51; 0.58) showed low agreement (d = 0.35, p < 0.001; ICC 0.27, 95%CI: -0.09; 0.59). The PROPr's distribution was positively skewed, whereas the EQ-5D-3L's was negative. Neither tool showed ceiling/floor effects, but all EQ-5D-3L dimensions did. Pearson correlation was r = 0.66 (95%CI: 0.58; 0.73). Differences were invariant to sex and age but not to ODI severity: ESEQ-5D-3L > ESPROPr and RE < 1 in higher ODI severity; ESEQ-5D-3L < ESPROPr and RE > 1 in lower ODI severity. AUROC-ratios did not show significant differences in terms of ODI severity. Conclusions: All PROPr and EQ-5D-3L biopsychosocial dimensions of health showed impairment in LPB patients. The capability of EQ-5D-3L and PROPr to differentiate ODI levels depends on ODI severity. Joint application of both tools may provide additional information. [ABSTRACT FROM AUTHOR]

Background: The increased prevalence of myopia creates and earlier age of onset has created public health concerns for the long-term eye health, vision impairment and carries with it a significant economic burden. The quality of the economic evaluation is dependent on the sensitivity and validity of the approaches. Nowadays, there are many approaches to measure patients' health state utility (HSU). However, little is known regarding the performance of direct approach and indirect approach in people with myopia. This study is aimed to compare the psychometric properties of four HSU approaches among patients with myopia in mainland China, including two direct approaches (TTO and SG), the generic preference-based measures (PBM) (AQoL-7D) and the disease-specific PBM (VFQ-UI). Methods: A convenience sampling framework was used to recruit patients with myopia who attended a large ophthalmic hospital in Jinan, China. Spearman's rank correlations coefficient was used to assess concurrent validity. Known-group validity was analyzed by: (1) whether the patients wear corrective devices; (2) severity of myopia as low or moderate to high of the better eye; (3) duration of myopia as ≤ 10 years or > 10 years. Effect size (ES), relative efficiency (RE) statistic and the largest area under the receiver operating characteristic curve (AUC) were used to assess sensitivity. The intra-class correlation coefficient (ICC) and Bland–Altman plots were used to assess agreement. Results: A valid sample size of 477 myopia patients was analyzed (median duration: 10 years). The mean HSU scores between TTO and SG were similar (0.95) and higher than AQoL-7D (0.89) and VFQ-UI (0.83). Overall, the VFQ-UI had the best performance based on the psychometric analysis. The agreement indicated that there was no pair of approaches that could be used interchangeably. Conclusions: The VFQ-UI showed better psychometric properties than other three approaches for providing health state utility in Chinese myopia patients. Given the widespread use and its generic nature of the AQoL-7D, it could be used alongside with VFQ-UI to provide complementary health state utility from a generic and disease-specific perspective for economic evaluation. More evidence on the responsiveness of four health utility approaches in myopia patients is required. [ABSTRACT FROM AUTHOR]

Background/Purpose: Health state utilities (HSU) are a subjective measure of an individual's health-related quality of life (HRQoL), adjusted by societal or patient relative preference weights for living in different states of health-related quality of life (HRQoL), derived from patient-reported responses to multi-attribute utility instrument (MAUI), and can be used as inputs for cost-utility analyses and in clinical assessment. This research assessed associations of diet with subsequent HSU in a large international cohort of people living with multiple sclerosis (MS), a progressive autoimmune condition of the central nervous system. Methods: HSUs were generated from responses to Short-Form Six-Dimension (SF-6D) MAUI, and quality-of-the-diet by Diet Habits Questionnaire (DHQ). Cross-sectional, and short- and long-term prospective associations of DHQ with HSU evaluated by linear regression at 2.5- and 5-years. Pooled prospective associations between DHQ and HSU evaluated using linear and quantile regression. Analyses adjusted for relevant demographic and clinical covariates. Results: Among 839 participants, baseline DHQ scores showed short- and long-term associations with subsequent HSU, each 10-unit increase in total DHQ score associated with 0.008–0.012 higher HSU (out of 1.00). These associations were dose-dependent, those in the top two quartiles of baseline DHQ scores having 0.01–0.03 higher HSU at follow-up, 0.03 being the threshold for a minimally clinically important difference. Fat, fiber, and fruit/vegetable DHQ subscores were most strongly and consistently associated with better HSU outcomes. However, baseline meat and dairy consumption were associated with 0.01–0.02 lower HSU at subsequent follow-up. Conclusions: A higher quality-of-the-diet showed robust prospective relationships with higher HSUs 2.5- and 5-years later, substantiating previous cross-sectional relationships in this cohort. Subject to replication, these results suggest interventions to improve the quality-of-the-diet may be effective to improve HRQoL in people living with MS. [ABSTRACT FROM AUTHOR]

Paul O’Brien,1 Ashley Enstone,2 Daisy Bridge,2 Robin Wyn,2 Judit Banhazi3 1Novartis Ireland, Dublin, Ireland; 2Adelphi Values PROVE, Bollington, UK; 3Novartis Pharma AG, Basel, SwitzerlandCorrespondence: Paul O’Brien, Email paul-2.obrien@novartis.comIntroduction: Retinitis pigmentosa (RP) is an inherited retinal pathology associated with “night blindness” and progressive loss of peripheral vision, in some cases leading to complete blindness. Health state utility values are required for activities such as modelling disease burden or the cost-effectiveness of new interventions. The current study aimed to generate utility values for health states of varying levels of functional vision in RP, with members of the general public in the UK.Methods: Five health states were defined according to standard clinical measures of visual ability. Health state descriptions were developed following interviews with patients with RP in the UK (n=5). Further interviews were conducted for confirmation with healthcare professionals with specific experience of managing patients with RP in the UK (n=2). Interviews with members of the general public in the UK were conducted to value health states. A time trade-off (TTO) process based on the established Measurement and Valuation of Health (MVH) protocol was used. Due to the ongoing COVID-19 pandemic, all interviews were web-enabled and conducted 1:1 by a trained moderator.Results: In total, n=110 TTO interviews were conducted with members of the UK general public. Mean TTO utility values followed the logical and expected order, with increasing visual impairment leading to decreased utility. Mean values varied between 0.78 ± 0.20 (“moderate impairment”), and 0.33 ± 0.26 (“hand motion” to “no light perception”). Supplementary visual analogue scale (VAS) scores also followed the logical and expected order: mean VAS values varied between 47.95 ± 15.38 (“moderate impairment”) and 17.22 ± 12.49 in (“hand motion” to “no light perception”).Discussion: These data suggest that individuals living with RP have substantially impaired quality of life. Utility values for RP have been elicited here using a method and sample that is suitable for economic modelling and health technology assessment purposes.Keywords: retinitis pigmentosa, inherited retinal disease, health state utility, health-related quality of life, time trade-off, vignette study

Background: Hair loss causes significant psychosocial distress to patients. Health utility measurements offer an objective, quantitative assessment of health-related quality of life (QOL). Methods: We performed a prospective cohort study on patients with hair loss between January 1, 2018 and December 31, 2020. Patient metrics were compared with layperson perception of alopecia, prospectively collected between August 1 and December 31, 2017. Health utility measures were quantified using the visual analog scale (VAS), standard gamble (SG), and time trade-off (TTO) in quality-adjusted life-years (QALYs) and relative to the minimal clinically important difference (MCID). Bonferroni correction to the significance threshold was performed. Results: Thirty-one patients with alopecia were compared with 237 laypeople. Patient metrics for female hair loss were all significantly lower than laypeople measures (VAS QALYs 0.65 ± 0.21 vs. 0.83 ± 0.18, p = 0.0001). Mean SG QALYs were lower for patients in the male alopecia state (0.86 ± 0.23 vs. 0.96 ± 0.12, p = 0.0278). Post-hair transplant improvement in TTO was significantly greater for patients (+ 0.08 ± 0.12 vs. + 0.02 ± 0.09, p = 0.0070) and significantly more often exceeded the MCID (45.2% vs. 16.9%, p = 0.0006). Conclusions: Alopecia negatively impacts QOL, and the true patient experience is more taxing than what is perceived by laypeople. Hair transplantation improves QOL more for male patients than common perception. Level of Evidence IV: This journal requires that authors assign a level of evidence to each article. For a full description of these Evidence-Based Medicine ratings, please refer to the Table of Contents or the online Instructions to Authors www.springer.com/00266. [ABSTRACT FROM AUTHOR]

Simple Summary: Patient-reported outcomes play an important role in clinical trials and health economic evaluation. In addition to health-related quality of life, there has been increasing recognition of measuring wider wellbeing that goes beyond health. This study aims to understand the sensitivity and comparability of commonly used preference-based health-related quality of life and subjective wellbeing measures in patients with cancer. This study further explored the life domain importance of cancer patients. The findings from this study shed light on the choice of patient-reported outcome measures in clinical studies as well as the prioritized aspects to improve the overall life satisfaction of cancer patients. Different wellbeing measures have been used among cancer patients. This study aimed to first investigate the sensitivity of health state utility (HSU), capability, and subjective wellbeing (SWB) instruments in cancer. A cancer-specific instrument (QLQ-C30) was included and transferred onto the cancer-specific HSU scores. Furthermore, it examined the relative importance of key life domains explaining overall life satisfaction. Data were drawn from the Multi-instrument Comparison survey. Linear regression was used to explore the extent to which the QLQ-C30 sub-scales explain HSU and SWB. Kernel-based Regularized Least Squares (KRLS), a machine learning method, was used to explore the life domain importance of cancer patients. As expected, the QLQ-C30 sub-scales explained the vast majority of the variance in its derived cancer-specific HSU (R2 = 0.96), followed by generic HSU instruments (R2 of 0.65–0.73) and SWB and capability instruments (R2 of 0.33–0.48). The cancer-specific measure was more closely correlated with generic HSU than SWB measures, owing to the construction of these instruments. In addition to health, life achievements, relationships, the standard of living, and future security all play an important role in explaining the overall life satisfaction of cancer patients. [ABSTRACT FROM AUTHOR]

Purpose: People living with HIV (PLHIV) have reported challenges associated with daily oral antiretroviral therapy (ART), including missed doses, negative psychological impact, and difficulty remaining discreet while at home or traveling. Recently approved long-acting injectable (LAI) ART may help eliminate these concerns. The purpose of this study was to examine patient preferences and estimate health state utilities associated with oral and LAI treatment for ART. Methods: Four health state vignettes were developed based on published literature, clinician interviews, and a pilot study. All vignettes included the same description of HIV, but differed in treatment regimens: (A) single daily oral tablet, (B) two daily oral tablets, (C) injections once monthly, and (D) injections every two months. PLHIV in the UK reported their preferences and valued the health states in time trade-off utility interviews. Results: The sample included 201 PLHIV (83.1% male; mean age = 44.9y). The health states frequently selected as most preferable were D (n = 119; 59.2%) and A (n = 75; 37.3%). Utility differences among health states were relatively small, which is typical for treatment process utilities (mean utilities: A, 0.908; B, 0.905; C, 0.900; D, 0.910). Statistically significant differences in utility were found for one vs. two tablets and injections every month vs. every two months (p < 0.001). Participants' quotations highlight the wide range of reasons for treatment process preferences. Conclusions: Current results indicate that many PLHIV would prefer LAI ART. The reported utilities may be useful in economic modeling comparing oral vs. LAI ART. [ABSTRACT FROM AUTHOR]

This study aimed to demonstrate how to estimate the value of health gain after patients with a multisystem disease achieve a condition-specific composite response endpoint. Data from patients treated in routine practice with an exemplar multisystem disease (systemic lupus erythematosus) were extracted from a national register (British Isles Lupus Assessment Group Biologics Register). Two bespoke composite response endpoints (Major Clinical Response and Improvement) were developed in advance of this study. Difference-in-differences regression compared health utility values (3-level version of EQ-5D; UK tariff) over 6 months for responders and nonresponders. Bootstrapped regression estimated the incremental quality-adjusted life-years (QALYs), probability of QALY gain after achieving the response criteria, and population monetary benefit of response. Within the sample (n = 171), 18.2% achieved Major Clinical Response and 49.1% achieved Improvement at 6 months. Incremental health utility values were 0.0923 for Major Clinical Response and 0.0454 for Improvement. Expected incremental QALY gain at 6 months was 0.020 for Major Clinical Response and 0.012 for Improvement. Probability of QALY gain after achieving the response criteria was 77.6% for Major Clinical Response and 72.7% for Improvement. Population monetary benefit of response was £1 106 458 for Major Clinical Response and £649 134 for Improvement. Bespoke composite response endpoints are becoming more common to measure treatment response for multisystem diseases in trials and observational studies. Health technology assessment agencies face a growing challenge to establish whether these endpoints correspond with improved health gain. Health utility values can generate this evidence to enhance the usefulness of composite response endpoints for health technology assessment, decision making, and economic evaluation. • Condition-specific composite response endpoints are used to estimate treatment efficacy and effectiveness for diseases that affect more than one organ system simultaneously. • The health gain associated with a composite response endpoint can be valued by reference to the subsequent improvement in health utility values. • Evidence of this health gain will help health technology assessment agencies compare the relative benefits of alternative endpoints, developers of endpoints to select the most valuable response criteria, inform trial design by guiding analysts to select an endpoint corresponding with the greatest health gain, and improve the usefulness of published studies for decision making. [ABSTRACT FROM AUTHOR]

To obtain appropriate health state utility values for cost-effectiveness analyses of new Mycobacterium avium complex pulmonary disease (MAC-PD) treatments. The impact of MAC-PD severity and symptoms on quality of life (QoL) also were quantified. A questionnaire describing four health states, MAC-positive severe, MAC-positive moderate, MAC-positive mild, and MAC-negative, was developed based on St. George's Respiratory Questionnaire (SGRQ) Symptom and Activity scores from the CONVERT trial. The time trade-off (TTO) method with ping-pong titration procedure was used to estimate health state utilities. Regression analyses assessed the impacts of covariates. Of 319 Japanese adults (49.8% female, mean age 44.8 years), mean (95% CI) health state utility scores (MAC-positive severe, MAC-positive moderate, MAC-positive mild, and MAC-negative) were 0.252 (0.194–0.310), 0.535 (0.488–0.582), 0.816 (0.793–0.839), and 0.881 (0.866–0.896), respectively. MAC-negative state utility scores were significantly higher than MAC-positive severe (mean difference [95% CI], 0.629 [0.574–0.684]), MAC-positive moderate (0.346 [0.304–0.389]), and MAC-positive mild (0.065 [0.048–0.082]) scores (p <.001 each). Most participants would trade survival duration to avoid MAC-positive states (97.5% to avoid MAC-positive severe; 88.7% MAC-positive moderate; 61.4% MAC-positive mild). Regression analyses to investigate the impact of background characteristics showed similar utility differences between health states when not adjusted for covariates. Some participant demographics differed from the general population; however, this did not impact utility differences among health states as regression analyses adjusting for demographics did not affect these differences. Similar investigations are needed among patients with MAC-PD and in other countries. This study evaluating the impact of MAC-PD on utilities using the TTO method demonstrates that differences in utilities are dependent on the severity of respiratory symptoms and their impacts on daily activities and QoL. These results could contribute to a better quantification of the value of MAC-PD treatments and improve assessments of cost-effectiveness. [ABSTRACT FROM AUTHOR]

Objective: This study aimed to gain insight into decision-making strategies individuals used when evaluating pairs of SF-6Dv2 health states in discrete choice experiments (DCEs).Methods: This qualitative, cross-sectional, noninterventional study asked participants to use a think-aloud approach to compare SF-6Dv2 health states in DCEs. Thematic analysis focused on comprehension and cognitive strategies used to compare health states and make decisions.Results: Participants (N = 40) used 3 main strategies when completing DCEs: (1) trading, (2) reinterpretation, and (3) relying on previous experience. Trading was the most common strategy, used by everyone at least once, and involved prioritizing key attributes, such as preferring a health state with significant depression but no bodily pain. Reinterpretation was used by 17 participants and involved reconstructing health states by changing underlying assumptions (eg, rationalizing selecting a health state with significant pain because they could take pain medications). Finally, some (n = 13) relied on previous experience when making decisions on some choice tasks. Participants with experience dealing with pain, for instance, prioritized health states with the least impact in this dimension.Conclusions: Qualitatively evaluating the decision-making strategies used in DCEs allows researchers to evaluate whether the tasks and attributes are interpreted accurately. The findings from this study add to the understanding of the generation of SF-6Dv2 health utility weights and the validity of these weights (e.g., reinterpreting health states could undermine the validity of DCEs and utility weights), and the overall usefulness of the SF-6Dv2. The methodology described in this study can and should be carried forth in valuing other health utility measures, not just the SF-6Dv2. [ABSTRACT FROM AUTHOR]

Willingness‐to‐pay (WTP) and time‐trade‐off (TTO) are widely used as measures of disease burden. However, the sensitivity of the metrics has not been compared in psoriasis. This article aims to report WTP and TTO in Chinese psoriasis patients, and to compare the WTPcure (USD), WTPcontrol (%), and TTO regarding the sensitivity in measuring the burden of psoriasis. Patients were recruited from a community‐based cohort, a tertiary hospital, and an online forum. WTP was elicited for two scenarios: WTP for controlling the disease and WTP for curing the disease. WTPcontrol was elicited by asking about the percentage of monthly income that the participant patients would like to pay to fully control the clinical symptoms of psoriasis and WTPcure was elicited as absolute money for curing the disease. The evaluation of WTP and TTO were referred to the Psoriasis Area and Severity Index (PASI) as the indicator of clinical severity, and the Dermatology Life Quality Index (DLQI) as the measure of impaired quality‐of‐life. Data distribution, concurrent validity, and capability of discriminating moderate‐to‐severe psoriasis (receiver operating characteristic curve, ROC) were compared among the indices. A total of 479 patients were enrolled from the cohort (88), hospital (175), and web (216). WTPcontrol was significantly higher in patients with PASI>10 than those with PASI<10, while TTO was significantly higher in patients with DLQI>10 than those with DLQI<10. WTP control showed a positive association with PASI in mild psoriasis (r = 0.36, p < 0.001), while TTO showed an inverse correlation to DLQI in different severity subgroups, respectively (r = −0.20, p = 0.001). ROC analysis showed consistent results that WTP control was superior in discriminating patients with PASI>10, while WTP control was better in discriminating DLQI>10. WTPcure demonstrated no significant results. WTPcontrol is superior in detecting mild psoriasis compared to TTO, while TTO is more sensitive in measuring the impaired QoL. [ABSTRACT FROM AUTHOR]

Purpose: There is a paucity of empirically estimated health state utility (HSU) values to estimate health-related quality of life among individuals with sickle cell disease (SCD). This study aims to map the Pediatric Quality of Life Inventory generic core scales (PedsQL GCS) to HSUs for children and adolescents with SCD in the United States, using published algorithms, and to assess the construct validity of these HSUs against SCD-specific PedsQL scores. Methods: We used the published mapping algorithms identified in four published articles, in which the PedsQL GCS was mapped to either the EuroQol-5 Dimension 3-Level, Youth Version or the Child Health Utility 9-Dimension to obtain HSUs. We employed the algorithms to calculate HSUs for a sample of children and adolescents from the Sickle Cell Clinical Research and Intervention Program. To assess the construct validity of the mapped HSUs in SCD patients, we computed Spearman's correlation coefficient comparing the HSUs with the PedsQL SCD total score and separately with each PedsQL SCD dimension-specific score. Results: The mean mapped HSU across published algorithms was 0.792 (95% CI: 0.782–0.801). It was significantly higher among children aged 5–12 years than children aged 13–17 years. The Spearman's correlation coefficient for HSUs versus PedsQL SCD total scores was 0.64 (95% CI: 0.57–0.71). Correlations ranged from 0.40 (95% CI: 0.32–0.48) to 0.60 (95% CI: 0.54–0.66) for HSUs versus PedsQL SCD dimension-specific scores. Conclusions: The existing mapping algorithms show acceptable construct validity in children and adolescents with SCD. Additional algorithms are needed for adults and for specific SCD comorbidities. [ABSTRACT FROM AUTHOR]

Objectives: Sickle cell disease (SCD) is a complex, chronic condition that impairs health-related quality of life of affected individuals and their caregivers. As curative therapies emerge, comprehensive cost-effectiveness models will inform their value. These models will require descriptions of health states and their corresponding utility values that accurately reflect health-related quality of life over the disease trajectory. The objectives of this systematic review were to develop a catalog of health state utility (HSU) values for SCD, identify research gaps, and provide future directions for preference elicitation.Methods: Records were identified through searches of PubMed and Embase, Tufts Medical Center Cost-Effectiveness Analysis Registry, reference lists of relevant articles, and consultation with SCD experts (2008-2020). We removed duplicate records and excluded ineligible studies. For included studies, we summarized the study characteristics, methods used for eliciting HSUs, and HSU values.Results: Five studies empirically elicited utilities using indirect methods (EQ-5D) (n = 3) and Short Form-6 Dimension (n = 2); these represent health states associated with general SCD (n = 1), SCD complications (n = 2), and SCD treatments (n = 3). Additionally, we extracted HSUs from 7 quality-adjusted life-years-based outcome research studies. The HSU among patients with general SCD without specifying complications ranged from 0.64 to 0.887. Only 36% of the HSUs used in the quality-adjusted life-year-based outcomes research studies were derived from individuals with SCD. No study estimated HSUs in caregivers.Conclusions: There is a dearth of literature of HSUs for use in SCD models. Future empirical studies should elicit a comprehensive set of HSUs from individuals with SCD and their caregivers. [ABSTRACT FROM AUTHOR]

Overall, 13% of parent-proxy respondents reported that COVID-19 pandemic and self-isolation/quarantine measures had prevented their children from receiving medical care related to FA "quite a lot", "very much" or "extremely." A short simple tool to measure the impact of food allergy on patients in routine clinical practice; the Food Allergy Quality of Life Questionnaire, Parent Form 10 (FAQLQ-PF10). COVID-19, food allergy, health state utility, life satisfaction, pediatrics, quality-of-life Keywords: COVID-19; food allergy; health state utility; life satisfaction; pediatrics; quality-of-life EN COVID-19 food allergy health state utility life satisfaction pediatrics quality-of-life 162 166 5 01/05/22 20220101 NES 220101 Key Messages We surveyed parents of children and adolescents with food allergy before and during the pandemic. [Extracted from the article]

Background We aimed to obtain a set of health state utility scores of patients with esophageal cancer (EC) and precancerous lesions in China, and to explore the influencing factors of health‐related quality of life (HRQoL). Methods A hospital‐based multicenter cross‐sectional study was conducted. From 2013 to 2014, patients with EC or precancerous lesions were enrolled. HRQoL was assessed using a European quality of life‐5 dimension (EQ‐5D‐3L) instrument. Multivariable linear regression analysis was performed to explore the influencing factors of the EQ‐5D utility scores. Results A total of 2090 EC patients and 156 precancer patients were included in the study. The dimension of pain/discomfort had the highest rate of self‐reported problems, 60.5% in EC and 51.3% in precancer patients. The mean visual analog scale (VAS) score for EC and precancer patients were 68.4 ± 0.7 and 64.5 ± 3.1, respectively. The EQ‐5D utility scores for EC and precancer patients were estimated as 0.748 ± 0.009 and 0.852 ± 0.022, and the scores of EC at stage I, stage II, stage III, and stage IV were 0.693 ± 0.031, 0.747 ± 0.014, 0.762 ± 0.015, and 0.750 ± 0.023, respectively. According to the multivariable analyses, the factors of region, occupation, household income in 2012, health care insurance type, pathological type, type of therapy, and time points of the survey were statistically associated with the EQ‐5D utility scores of EC patients. Conclusions There were remarkable decrements of utility scores among esophageal cancer patients, compared with precancer patients. The specific utility scores of EC would support further cost‐utility analysis in populations in China.

Background: The Food Allergy Quality‐of‐Life Questionnaire‐Parent Form (FAQLQ‐PF) is widely used to assess food allergy–specific health‐related quality of life (FAQL), but cannot be used directly in cost‐utility analyses, which require health state utility (HSU) scores. Currently, limited evidence is available regarding the HSU of food‐allergic children/adolescents. This study aimed to develop mapping algorithms from the FAQLQ‐PF onto HSU scores generated by generic, preference‐based, health‐related quality‐of‐life (HRQL) instruments. Methods: Caregivers of children aged 7 to 17 years with a clinician diagnosis of IgE‐mediated food allergy, recruited via Allergy & Anaphylaxis Australia, completed an online FAQLQ‐PF questionnaire and proxy generic preference‐based pediatric instruments (Assessment of Quality of Life [AQoL]‐6D and Child Health Utility 9D [CHU9D]). Optimal statistical methods were based on series of goodness‐of‐fit statistics. Results: Mean FAQLQ‐PF total score, AQoL‐6D, and CHU9D utility scores of 238 food‐allergic children/adolescents were 3.49 (SD: 1.41), 0.78 (SD: 0.22), and 0.74 (SD: 0.22), respectively. The Spearman correlation coefficients of FAQLQ‐PF with AQoL‐6D and CHU9D were rho = −0.56 and rho = −0.45, respectively. Optimal mapping algorithms were generated from selected FAQLQ‐PF items, mapped onto AQoL‐6D or CHU9D utility scores, with AQoL‐6D demonstrating better performance. Conclusions: This study generated mapping algorithms to help facilitate the use of FAQLQ‐PF for cost‐utility analyses, which are essential for health economic evaluation. External validation of the reported mapping algorithms is warranted. [ABSTRACT FROM AUTHOR]

Objectives: Cancer diagnoses at later stages are associated with a decrease in health-related quality of life (HRQOL). Health state utility values (HSUVs) reflect preference-based HRQOL and can vary based on cancer type, stage, treatment, and disease progression. Detecting and treating cancer at earlier stages may lead to improved HRQOL, which is important for value assessments. We describe published HSUVs by cancer type and stage. Methods: A systematic review was conducted using Embase, MEDLINE®, EconLit, and gray literature to identify studies published from January 1999 to September 2019 that reported HSUVs by cancer type and stage. Disutility values were calculated from differences in reported HSUVs across cancer stages. Results: From 13,872 publications, 27 were eligible for evidence synthesis. The most frequent cancer types were breast (n = 9), lung (n = 5), colorectal (n = 4), and cervical cancer (n = 3). Mean HSUVs decreased with increased cancer stage, with consistently lower values seen in stage IV or later-stage cancer across studies (e.g., − 0.74, − 0.44, and − 0.51 for breast, colorectal, and cervical cancer, respectively). Disutility values were highest between later-stage (metastatic or stage IV) cancers compared to earlier-stage (localized or stage I–III) cancers. Conclusions: This study provides a summary of HSUVs across different cancer types and stages that can inform economic evaluations. Despite the large variation in HSUVs overall, a consistent decline in HSUVs can be seen in the later stages, including stage IV. These findings indicate substantial impairment on individuals' quality of life and suggest value in early detection and intervention. [ABSTRACT FROM AUTHOR]

Objectives: Health state utilities (HSUs) are an input metric for estimating quality-adjusted life-years (QALY) in cost–utility analyses. Currently, there is a paucity of data on association of knee symptoms with HSUs for middle-aged populations. We aimed to describe the association of knee symptoms and change in knee symptoms with SF-6D HSUs and described the distribution of HSUs against knee symptoms' severity. Methods: Participants (36–49-years) were selected from the third follow-up (completed 2019) of Australian Childhood Determinants of Adult Health study. SF-6D HSUs were generated from the participant-reported SF-12. Association between participant-reported WOMAC knee symptoms' severity, change in knee symptoms over 6–9 years, and HSUs were evaluated using linear regression models. Results: For the cross-sectional analysis, 1,567 participants were included; mean age 43.5 years, female 54%, BMI ± SD 27.18 ± 5.31 kg/m2. Mean ± SD HSUs for normal, moderate, and severe WOMAC scores were 0.820 ± 0.120, 0.800 ± 0.120, and 0.740 ± 0.130, respectively. A significant association was observed between worsening knee symptoms and HSUs in univariable and multivariable analyses after adjustment (age and sex). HSU decrement for normal-to-severe total-WOMAC and WOMAC-pain was − 0.080 (95% CI − 0.100 to − 0.060, p < 0.01) and − 0.067 (− 0.085 to − 0.048, p < 0.01), exceeding the mean minimal clinically important difference (0.04). Increase in knee pain over 6–9 years was associated with a significant reduction in HSU. Conclusion: In a middle-aged population-based sample, there was an independent negative association between worse knee symptoms and SF-6D HSUs. Our findings may be used by decision-makers to define more realistic and conservative baseline and ongoing HSU values when assessing QALY changes associated with osteoarthritis interventions. [ABSTRACT FROM AUTHOR]

Health economics uses quality adjusted life years (QALYs) to help healthcare decision makers. However, unlike life expectancy for which age‐ and sex‐dependent national life tables are available, no general population norms exist to use as a benchmark against which to compare observed or modeled projections of QALYs in sub‐populations or patients. We developed a 2‐state Markov model to generate QALY population norms for the USA, UK, China and Australia. Annual age‐ and sex‐specific probabilities of all‐cause mortality were taken from life tables combined with general population country‐specific age‐ and sex‐specific health state utilities for the EQ‐5D‐3L (all countries); and SF‐6D (Australia) multi‐attribute utility instruments (MAUI). To validate our QALY benchmark model we found that the model closely predicted population life expectancies. Using EQ‐5D‐3L, undiscounted QALYs for males/females aged 18 years ranged 54.62/58.90 (USA), 55.55/60.21 (China), 57.11/60.16 (Australia), and 58.01/61.43 (UK) years. SF‐6D benchmark QALYs for Australia were consistently lower than those generated from the EQ‐5D‐3L. The gap in undiscounted QALYs between the UK (highest) and the USA (lowest) was 2.53 QALYs in women and 3.39 QALYs in men aged 18 years. Our model's QALY population norms can be used for internal validation of future health economic models for the country‐specific value sets for the instruments that we adopted, and when quantifying burden of disease in terms of QALYs lost due to illness compared to the general population. We have created a publicly available repository to continuously include QALY benchmarks that use country‐specific value sets for other MAUIs and life expectancies. [ABSTRACT FROM AUTHOR]

Abstract Background Health state utility (HSU) is a core component of QALYs and cost-effectiveness analysis, although HSU is rarely estimated among a representative sample of patients. We explored the feasibility of assessing HSU in head and neck cancer from the French National Hospital Discharge database. Methods An exhaustive sample of 53,258 incident adult patients with a first diagnosis of head and neck cancer was identified in 2010–2012. We used a cross-sectional approach to define five health states over two periods: three "cancer stages at initial treatment" (early, locally advanced or metastatic stage); a "relapse state" and otherwise a "relapse-free state" in the follow-up of patients initially treated at early or locally advanced stage. In patients admitted in post-acute care, a two-parameter graded response model (Item Response Theory) was estimated from all 144,012 records of six Activities of Daily Living (ADLs) and the latent health state scale underlying ADLs was calibrated with the French EQ-5D-3 L social value set. Following linear interpolation between all assessments of the patient, daily estimates of utility in post-acute care were averaged by health state, patient and month of follow-up. Finally, HSU was estimated by health state and month of follow-up for the whole patient population after controlling for survivorship and selection in post-acute care. Results Head and neck cancer was generally associated with poor HSU estimates in a real-life setting. As compared to “distant metastasis at initial treatment”, mean HSU was higher in other health states, although numerical differences were small (0.45 versus around 0.54). It was primarily explained by the negative effects on HSU of an older age (38.4% aged ≥70 years in “early stage at initial treatment”) and comorbidities (> 50% in other health states). HSU estimates significantly improved over time in the “relapse-free state” (from 8 to 12 months of follow-up). Conclusions HSU estimates in head and neck cancer were primarily driven by age at diagnosis, comorbidities, and time to assessment of cancer survivors. This feasibility study highlights the potential of estimating HSU within and across severe conditions in a systematic way at the national level.

Aim: Evaluate health-related quality of life (HRQoL) and health utility impact of single-agent selinexor in heavily pretreated patients with relapsed/refractory diffuse large B-cell lymphoma. Patients & methods: Functional Assessment of Cancer Therapy (FACT) - Lymphoma and EuroQoL five-dimensions five-levels data collected in the single-arm Phase IIb trial SADAL (NCT02227251) were analyzed with mixed-effects models. Results: Treatment responders maintained higher FACT - Lymphoma (p ≤ 0.05), FACT - General (p < 0.05) and EuroQoL five-dimensions five-levels index scores (p < 0.001) beginning in cycle 3. The estimated difference in health state utilities for treatment response and progressive disease was both statistically significant and clinically meaningful (mean difference: 0.07; p = 0.001). Conclusion: In patients with relapsed/refractory diffuse large B-cell lymphoma, objective response to selinexor was associated with HRQoL maintenance, reduction in disease-related HRQoL decrements and higher health utilities. [ABSTRACT FROM AUTHOR]

Objective: In thyroid eye disease (TED), inflammation and expansion of orbital muscle and periorbital fat result in diplopia and proptosis, severely impacting patient quality of life (QOL). The reported health state utility (HSU) scores, which are QOL measures, allow quantification of TED impact and improvement with therapies; however, no current QOL instrument has been validated with HSU scores for TED. Here, we used the disease-specific Graves Ophthalmopathy Quality of Life (GO-QOL) questionnaire and HSU scores to validate QOL impact., Methods: The GO-QOL scores from patients in 2 randomized, masked, placebo-controlled teprotumumab trials (N=171) were compared with 6 HSU values based on severity of proptosis/diplopia in those studies. Patient GO-QOL and HSU scores were compared at baseline and after 6-month treatment via regression analyses. GO-QOL and HSU scores were correlated for validation and quantification of QOL impact by severity state and to estimate quality-adjusted life year improvement., Results: GO-QOL scores were correlated with TED severity, indicating that worse severity was associated with lower (worse) GO-QOL scores. Less severe health states were represented by higher (better) GO-QOL scores. Importantly, GO-QOL scores were positively correlated with utility scores of the 6 health states, allowing for conversion of the GO-QOL scores to utility scores. A positive (improved) 0.013 utility change was found for each 1-point (positive) improvement in GO-QOL score produced by teprotumumab versus placebo., Conclusion: Patients with moderate-to-severe active TED health states demonstrate increasing TED severity associated with declining utility values and worsening GO-QOL scores. These results indicate that the GO-QOL scores can be used to bridge to the HSU scores for benefit quantification., Competing Interests: Disclosure T.J. Smith was issued US patents covering the use of IGF-1 receptor inhibitors in TED which are held by UCLA and the Lundquist Institute and is a consultant for Amgen (formerly Horizon), Viridian, Minghui and Lundbeck. K. Cockerham is a consultant for Amgen Inc (formerly Horizon) and Viridian. N. Barretto and R.J. Holt are employees and hold stock in Amgen (formerly Horizon). S. Taylor is a former employee and stockholder of Horizon. A. Hirst, L. Oliver, A. Enstone, and G. Brandolini are employees of Adelphi Values PROVE (Adelphi Values received budgetary compensation from Horizon [now Amgen] for conduct of the study)., (Copyright © 2024 AACE. Published by Elsevier Inc. All rights reserved.)

Abstract Background The increasing emphasis on patient-reported outcomes in health care decision making has prompted greater rigor in the evidence to support the instruments used. Acceptability and content validity are important properties of any measure to ensure it assesses the relevant aspects of the target concept. The purpose of this study was to evaluate the acceptability and content validity of the EQ-5D 5-Level (EQ-5D-5L) to assess the impact of asthma on patients’ lives. Methods Qualitative interviews were conducted with 40 adults with asthma in the United Kingdom. The first 25 interviews used cognitive-debriefing methods to assess the relevance and acceptability of the EQ-5D-5L and two asthma-specific measures for comparison: an asthma-specific, preference-based measure (the Asthma Quality of Life Utility Index–5 Dimensions) and an Asthma Symptom Diary. The final 15 interviews combined concept elicitation to identify patient-perceived asthma impact, and cognitive debriefing to assess relevance and acceptability of the EQ-5D-5L and the Asthma Symptom Diary. Cognitive-debriefing feedback on the content of the measures was collated and summarized descriptively. The concept-elicitation data were analyzed thematically. Results Participants were aged 20 to 57 years and 62.5% were female. Although some participants expressed positive opinions on aspects of the EQ-5D-5L, only the usual activities dimension was consistently considered relevant to participants’ asthma experiences. The mobility and self-care dimensions prompted strong negative reactions from some participants. Variations in interpretation of the mobility dimension and difficulties with multiple concepts in the pain/discomfort and anxiety/depression dimensions also were noted. Concepts reported by participants as missing included environmental triggers, asthma symptoms, emotions, and sleep. The EQ-5D-5L was the least preferred measure to describe the impact of asthma on participants’ lives. Participants reported shortness of breath and impact on activities as especially salient issues. Conclusions The content of the EQ-5D-5L was poorly aligned with the patient-perceived impact of asthma, and the measure failed to meet basic standards for acceptability and content validity as a measure to assess the impact of asthma from the patient perspective. The shortcomings identified raise concerns regarding the appropriateness of the EQ-5D in asthma and further evaluation is warranted.

Objective: To evaluate the health‐related quality of life (HRQoL) and health state utility scores of gastric cancer patients in daily life at different clinical stages after treatment, and to explore influencing factors associated with HRQoL. Methods: Gastric cancer patients discharged from hospitals and healthy controls identified by screening were recruited. The three‐level EQ‐5D was employed to assess HRQoL and was scored using two Chinese‐specific tariffs published in 2014 and 2018. Results: A total of 1,399 patients and 2,179 healthy controls were recruited. The likelihood of reporting problems in the five dimensions for patients was 4.0–23.8 times higher than controls. Based on the 2014/2018 tariff, the mean EQ‐5D utility score was 0.321/0.163 lower than controls, and the mean utility scores of each patient subgroup were 0.077/0.039 (high‐grade intraepithelial neoplasia/carcinoma in situ), 0.254/0.121 (Stage I), 0.249/0.123 (Stage II), 0.353/0.182 (Stage III) and 0.591/0.309 (Stage IV) lower than controls (all statistically significant). Age, occupation, duration of illness, other chronic disease status and therapeutic regimen had a significant impact upon different aspects of HRQoL in patients. Conclusions: Gastric cancer significantly impaired patients' HRQoL in daily life after treatment. More advanced cancer stages were associated with larger decrements on health state utility. [ABSTRACT FROM AUTHOR]

Objectives: To explore the acceptability, consistency, and accuracy of eliciting health state utility values using discrete choice experiment (DCE) and DCE with life duration dimension (DCETTO) as compared with conventional time trade-off (TTO) by using the SF-6Dv2.Methods: During face-to-face interviews, a representative sample of the general population in Tianjin, China, completed 8 TTO tasks and 10 DCE/DCETTO tasks, with the order of TTO and DCE/DCETTO being randomized. The fixed-effect model and conditional logit models were used for TTO and DCEs data estimation, respectively. Acceptability was assessed by self-reported difficulties in understanding/answering. Consistency was observed by the monotonicity of model coefficients. Accuracy was evaluated by investigating differences between observed and predicted TTO values using intraclass correlation coefficient, mean absolute difference, and root mean square difference.Results: A total of 503 respondents (53.7% male; range, 18-86 years) were included, with comparable characteristics between respondents who completed DCE (N = 252) and DCETTO (N = 251). No significant difference was observed in self-reported difficulties among 3 approaches. The monotonicity of coefficients could not be achieved for 2 DCE approaches, even when combining the inconsistent levels. The health state utility values generated by DCE were generally higher than those generated by TTO, whereas DCETTO was lower than TTO. The TTO had a better prediction accuracy than the DCEs.Conclusions: Two DCE approaches are feasible for eliciting health state utility values; however, they are not considered to be easier to understand/answer than TTO. There are systematic differences in the health state utility values generated by 3 approaches. The issue of non-monotonicity from 2 DCE approaches remains a concern. [ABSTRACT FROM AUTHOR]

Background: We aimed to obtain a set of health state utility scores of patients with esophageal cancer (EC) and precancerous lesions in China, and to explore the influencing factors of health‐related quality of life (HRQoL). Methods: A hospital‐based multicenter cross‐sectional study was conducted. From 2013 to 2014, patients with EC or precancerous lesions were enrolled. HRQoL was assessed using a European quality of life‐5 dimension (EQ‐5D‐3L) instrument. Multivariable linear regression analysis was performed to explore the influencing factors of the EQ‐5D utility scores. Results: A total of 2090 EC patients and 156 precancer patients were included in the study. The dimension of pain/discomfort had the highest rate of self‐reported problems, 60.5% in EC and 51.3% in precancer patients. The mean visual analog scale (VAS) score for EC and precancer patients were 68.4 ± 0.7 and 64.5 ± 3.1, respectively. The EQ‐5D utility scores for EC and precancer patients were estimated as 0.748 ± 0.009 and 0.852 ± 0.022, and the scores of EC at stage I, stage II, stage III, and stage IV were 0.693 ± 0.031, 0.747 ± 0.014, 0.762 ± 0.015, and 0.750 ± 0.023, respectively. According to the multivariable analyses, the factors of region, occupation, household income in 2012, health care insurance type, pathological type, type of therapy, and time points of the survey were statistically associated with the EQ‐5D utility scores of EC patients. Conclusions: There were remarkable decrements of utility scores among esophageal cancer patients, compared with precancer patients. The specific utility scores of EC would support further cost‐utility analysis in populations in China. [ABSTRACT FROM AUTHOR]

Abstract Background Health-related quality of life (HRQoL) and health state utility value (HSUV) measurements are vital components of healthcare clinical and economic evaluations. Accurate measurement of HSUV and HRQoL require validated instruments. The 12-item Short-Form Health Survey (SF-12) is one of few instruments that can evaluate both HRQoL and HSUV, but its validity has not been assessed in people living with HIV/AIDS (PLWHA) in east Africa, where the burden of HIV is high. Methods This cross-sectional study used baseline data from a randomized trial involving PLWHA in Kenya. Data included responses from a translated and adapted SF-12 survey as well as key demographic and clinical data. Construct validity of the survey was examined by testing the SF-12’s ability to distinguish between groups known in advance to have differences in their health based on their disease severity. We classified disease severity based on established definitions from the US Center for Disease Control (CDC) and WHO, as well as a previously studied viral load threshold. T-tests and ANOVA were used to test for differences in HRQoL and HSUV scores. Area under the receive operator curve (AUC) was used to test the discriminative ability of the HRQoL and HSUV instruments. Results Differences in physical component scores met the minimum clinically important difference among participants with more advanced HIV when defined by CD4 count (4.3 units) and WHO criteria (compared to stage 1, stages 2, 3 and 4 were 2.0, 7.2 and 9.8 units lower respectively). Mental score differences met the minimum clinically important difference between WHO stage 1 and stage 4 patients (4.4). Differences in the HSUV were statistically lower in more advanced HIV by all three definitions of severity. The AUC showed poor to weak discriminatory ability in most analyses, but had fair discriminatory ability between WHO clinical stage 1 and clinical stage 4 individuals (AUC = 0.71). Conclusion Our findings suggest that the Kiswahili translated and adapted version of the SF-12 could be used as an assessment tool for physical health, mental health and HSUV for Kiswahili-speaking PLHWA. Trial registration Clinical trials.gov identifier: NCT00830622 . Registered 26 January 2009.

Liu Liu,1,2 Shunping Li,1,2 Min Wang,3 Gang Chen4 1School of Health Care Management, Shandong University, Jinan, China; 2Key Laboratory of Health Economics and Policy Research, NHFPC (Shandong University), Jinan, China; 3Qingdao Municipal Hospital, Qingdao, China; 4Flinders Centre for Innovation in Cancer, School of Medicine, Flinders University, Adelaide, SA, Australia Objective: The objective of this study was to compare the differences in the five-level EuroQol-5 dimensions (EQ-5D-5L) health state utility scores derived from Chinese, Japanese, Korean, and UK tariffs.Methods: Six hundred and twenty-one breast cancer patients were invited for a face-to-face interview in Qingdao Municipal Hospital, China. EQ-5D-5L was scored using tariffs from China, Japan, Korea, and the UK. The null hypothesis of normal distribution of the EQ-5D-5L utility score was tested by the Shapiro–Wilk test. Nonparametric Friedman test and Wilcoxon signed-rank test were used to determine the difference among the four tariffs. The intraclass correlation coefficients (ICCs) and Bland–Altman plots were used to study the agreement among the four EQ-5D-5L scores. Known-groups validity was studied using a regression framework.Results: There were 608 participants in the final analysis, with a mean ± standard deviation (SD) age of 48.0±9.6 years. EQ-5D-5L utility scores were non-normally distributed. The means (median) ± SD of EQ-5D-5L utilities derived from Chinese, Japanese, Korean, and UK tariffs were 0.828 (0.879) ±0.184, 0.802 (0.823) ±0.164, 0.831 (0.829) ±0.137, and 0.838 (0.866) ±0.154, respectively. Among pairwise comparisons, the difference of median EQ-5D-5L utility scores was only insignificant between Chinese and UK tariffs. Excellent agreements (with ICCs >0.9) were found among the four tariffs albeit the limits of agreement between each pair of tariffs were wide. Known-groups validity was supported. Conclusion: Although four country-specific EQ-5D-5L tariffs have shown an overall high level of correlation and agreement, none of them could be regarded as interchangeable. The higher correlation and agreement between Chinese and UK tariffs may be due to the similar functions that were used in the tariff development. In the absence of Chinese-specific tariff, the UK tariff is the second-best option to be applied in the Chinese population. Results of this study further contribute to the explanation of variations among country-specific tariffs. Keywords: breast cancer, EQ-5D-5L, multi-attribute utility instrument, preference weights, value sets, health state utility, China

Background/Objective: Past studies have found a correlation between the Dermatology Life Quality Index (DLQI) and the Psoriasis Area and Severity Index (PASI) for Caucasian psoriasis patients. This study examined the relationships between health state utility and willingness to pay (WTP) and the DLQI, as well as between the PASI and the DLQI, health-related utilities, and WTP in Taiwanese patients. Methods: A total of 480 psoriasis patients from five hospitals across Taiwan were interviewed between August 2009 and February 2010. Health state utilities were elicited using the time trade-off (TTO) method, a visual analog scale (VAS), and the EuroQOL-five dimensions (EQ-5D). WTP for a cure was elicited by double-bounded binary-choice questions followed by a bidding game. Psoriasis severity was graded as mild (PASI

• This study provides vital evidence regarding time-varying benefits of PAC services to HRQoL of stroke patients. • Multiple predictors of HRQoL among stroke patients receiving PAC services were identified in this study. • The benefits of the PAC program to health state utilities were significantly greater in men, in those without the prior history of stroke admissions, in those with hemorrhagic strokes, and in those reporting extreme problems in any dimensions of EQ-5D questionnaire. This study aims to evaluate health-related quality of life (HRQoL) of middle-aged and older stroke patients receiving the stroke post care (PAC) program and to identify possible predictors. This is a retrospective cohort study. Demographic characteristics and functional outcomes (modified Rankin Scale, Barthel Index, Mini-Mental State Examination, and the Concise Chinese Aphasia Test) were collected at enrollment. EQ-5D HRQoL questionnaires were administered at the beginning and the end of PAC, and health state utilities were compared. The EQ-5D utilities of stroke patients aged 75–84 years and 85 years or above were estimated to be 0.091 and 0.159 lower than those aged less than 50 years. A decrease of the utility by 0.075 was observed among patients with the prior history of stroke. The EQ-5D utilities of patients having Barthel Index of 21–40, 41–60, and 61–100 were 0.1432, 0.1568, and 0.1387 higher than those having Barthel Index of 0–20, respectively. For patients reporting extreme problems in self-care or any dimension of EQ-5D questionnaires prior to PAC, increases in utilities by 0.0733 and 0.2875 were noted. The EQ-5D utility of PAC service duration rose by 0.0733 per one incremental day. This study provides vital evidence regarding time-varying benefits of PAC services to HRQoL of stroke patients and to identify multiple predictors of HRQoL among stroke patients receiving PAC services. This study thus could serve as good reference to enhance quality of PAC services among stroke patients. [ABSTRACT FROM AUTHOR]

Cost-effectiveness models that present results in terms of cost per quality-adjusted life-year for health technologies are used to inform policy decisions in many parts of the world. Health state utilities (HSUs) are required to calculate the quality-adjusted life-years. Even when clinical studies assessing the effectiveness of health technologies collect data on HSUs to populate a cost-effectiveness model, which rarely happens, analysts typically need to identify at least some additional HSUs from alternative sources. When possible, HSUs are identified by a systematic review of the literature, but, again, this rarely happens. In 2014, ISPOR established a Good Practices for Outcome Research Task Force to address the use of HSUs in cost-effectiveness models. This task force report provides recommendations for researchers who identify, review, and synthesize HSUs for use in cost-effectiveness models; analysts who use the results in models; and reviewers who critically appraise the suitability and validity of the HSUs selected for use in models. The associated Minimum Reporting Standards of Systematic Review of Utilities for Cost-Effectiveness checklist created by the task force provides criteria to judge the appropriateness of the HSUs selected for use in cost-effectiveness models and is suitable for use in different international settings. [ABSTRACT FROM AUTHOR]

Purpose: Measuring health-related quality of life (HRQoL) in ovarian cancer patients is critical to understand the impact of disease and treatment. Preference-based HRQoL measures, called health state utilities, are used specifically in health economic evaluations. Real-world patient-reported data on HRQoL and health state utilities over the long-term course of ovarian cancer are limited. This study aims to determine HRQoL and health state utilities in different health states of ovarian cancer., Methods: This cross-sectional, multicenter study included patients with stage III-IV ovarian cancer in six health states: at diagnosis, during chemotherapy, after cytoreductive surgery (CRS), after chemotherapy, in remission, and at first recurrence. HRQoL was measured using the European Organization for Research and Treatment of Cancer Core Quality of Life Questionnaire C30, and the ovarian cancer-specific module OV28. Health state utilities were assessed using the EuroQol five-dimension five-level (EQ-5D-5L) questionnaire. Descriptive analyses were performed for each health state., Results: Two hundred thirty-two patients participated, resulting in 319 questionnaires. Median age was 66 years. The lowest HRQoL was observed during chemotherapy and shortly after CRS. Physical and role functioning were most affected and the highest symptom prevalence was observed in the fatigue, nausea, pain, dyspnea, gastrointestinal, neuropathy, attitude, and sexuality domains. Patients in remission had the best HRQoL. Mean utility values ranged from 0.709 (±0.253) at diagnosis to 0.804 (±0.185) after chemotherapy., Conclusions: This study provides clinicians with a valuable resource to aid in patient counseling and clinical decision-making. The utilities, in particular, are crucial for researchers conducting economic analyses to inform policy decisions., Competing Interests: The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: GS Sonke reports receiving institutional research support from Agendia, Biovica, AstraZeneca, Merck, Novartis, Roche and Seagen outside the scope of this study. The other authors have stated explicitly that there are no conflicts of interest in connection with this article., (© 2023 The Authors.)

Purpose: To investigate the validity of direct and indirect health state utility (HSU) and subjective well-being measures in psoriasis vulgaris patients.Methods: A convenience sampling framework was used to successively recruit patients with psoriasis vulgaris from the outpatient clinics of a tertiary hospital in Changsha, Central South China. Participants completed time trade-off (TTO), standard gamble (SG), the five-level EQ-5D (EQ-5D-5L), the WHO-5 well-being index, and the psoriasis disability index (PDI). The concurrent and known-groups validity of HSUs and well-being index in psoriasis patients were firstly studied. The agreements among HSUs and the relationship between HSU and well-being measures were further explored.Results: A valid sample of 343 patients was analyzed. Mean HSU and well-being scores elicited from the EQ-5D-5L/TTO/SG and WHO-5 were 0.90/0.85/0.88 and 13.69, respectively. The Spearman correlation (concurrent validity) was the strongest between PDI and WHO-5 (r = 0.45), followed by with EQ-5D-5L (0.38), SG (r = 0.20), and the TTO (r = 0.18). The pairwise intraclass correlation coefficients among the three HSU measures were < 0.30. The known-groups validity was evident in all measures except for the SG. Exploratory factor analysis further suggests a complementary relationship between the EQ-5D-5L and WHO-5.Conclusions: There is a poor agreement between direct and indirect methods on measuring HSU with psoriasis vulgaris. Results from this study recommend that the EQ-5D-5L is the most preferred method to elicit HSU from psoriasis vulgaris patients in mainland China. It is important to further analyze the subjective well-being in addition to the HSU to fully understand the impact of psoriasis. [ABSTRACT FROM AUTHOR]