Patient-reported outcomes in chronic hepatitis delta: An exploratory analysis of the Phase III MYR301 trial of bulevirtide.

BACKGROUND & AIMS: Once-daily treatment of chronic hepatitis delta (CHD) with bulevirtide is well tolerated and associated with significant reductions in HDV RNA in the blood and in biochemical liver disease activity. This study explored the effects of 48-week bulevirtide treatment on health-related quality of life (HRQoL) in patients with CHD. METHODS: In an open-label, randomised, Phase 3 trial, 150 patients with CHD and compensated liver disease were stratified by liver cirrhosis status and randomised 1:1:1 to no treatment (control), bulevirtide 2 mg/day, or bulevirtide 10 mg/day for 48 weeks. HRQoL was evaluated by the following patient-reported outcome (PRO) instruments at baseline, 24 weeks, and 48 weeks: EQ-5D-3L, Hepatitis Quality of Life Questionnaire (HQLQ), and Fatigue Severity Scale (FSS). RESULTS: Patient characteristics and HRQoL scores were balanced at baseline between the treatment (2 mg, n = 49; 10 mg, n = 50) and control (n = 51) groups. Patients receiving 2-mg bulevirtide reported significant improvements compared with controls on the HQLQ domains of role physical, hepatitis-specific limitations, and hepatitis-specific health distress. Numerically higher scores for general health, hepatitis-specific limitations, and hepatitis-specific health distress domains were reported by patients with cirrhosis who received bulevirtide vs control. FSS scores remained stable across treatment groups throughout. At week 48, patients in the 2-mg group showed greater mean improvement from baseline in health status compared with controls on the EQ-5D-3L visual analogue scale. CONCLUSION: PROs indicate that 48-week treatment with bulevirtide monotherapy may improve aspects of HRQoL in patients with CHD. IMPACT AND IMPLICATIONS: Bulevirtide 2 mg is the only approved treatment for patients with chronic hepatitis delta (CHD) in the EU. Patients with CHD have worse quality of life scores than those with chronic hepatitis B. Bulevirtide treatment for 48 weeks reduced HDV RNA and alanine aminotransferase levels and was well tolerated among patients with CHD. For the first time, this study shows that patients who received bulevirtide therapy for 48 weeks reported improvements in physical and hepatitis-related quality of life domains compared to those who did not receive therapy (control group). CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov Identifier, NCT03852719.

The short-term impact of Schistosoma mansoni infection on health-related quality of life: implications for current elimination policies.

The WHO aims to eliminate schistosomiasis as a public health problem by 2030. However, standard morbidity measures poorly correlate to infection intensities, hindering disease monitoring and evaluation. This is exacerbated by insufficient evidence on Schistosoma's impact on health-related quality of life (HRQoL). We conducted community-based cross-sectional surveys and parasitological examinations in moderate-to-high Schistosoma mansoni endemic communities in Uganda. We calculated parasitic infections and used EQ-5D instruments to estimate and compare HRQoL utilities in these populations. We further employed Tobit/linear regression models to predict HRQoL determinants. Two-thirds of the 560 participants were diagnosed with parasitic infection(s), 49% having S. mansoni. No significant negative association was observed between HRQoL and S. mansoni infection status/intensity. However, severity of pain urinating (ß = -0.106; s.e. = 0.043) and body swelling (ß = -0.326; s.e. = 0.005), increasing age (ß = -0.016; s.e. = 0.033), reduced socio-economic status (ß = 0.128; s.e. = 0.032), and being unemployed predicted lower HRQoL. Symptom severity and socio-economic status were better predictors of short-term HRQoL than current S. mansoni infection status/intensity. This is key to disentangling the link between infection(s) and short-term health outcomes, and highlights the complexity of correlating current infection(s) with long-term morbidity. Further evidence is needed on long-term schistosomiasis-associated HRQoL, health and economic outcomes to inform the case for upfront investments in schistosomiasis interventions.

Health utility assessments in individuals undergoing diagnostic and surveillance colonoscopy: improved discrimination with a cancer-specific scale.

PURPOSE: To compare the sensitivity and discriminant validity of generic and cancer-specific measures for assessing health-related quality of life (HRQoL) for individuals undergoing diagnostic or surveillance colonoscopy for colorectal cancer. METHODS: HRQoL was assessed using EQ-5D-5L (generic), and EORTC QLQ-C30 (cancer-specific) scales, 14 days after (baseline) and one-year following colonoscopy (follow-up). Utility scores were calculated by mapping EORTC-QLQ-C30 onto QLU-C10D. Differences between participants with different indications for colonoscopy (positive faecal occult blood test (FOBT), surveillance, or symptoms) and colonoscopy findings (no polyps, polyps, or cancer) were tested using Wilcoxon-Mann-Whitney and Kruskal-Wallis H tests. Sensitivity was assessed by calculating the ceiling effects (proportion reporting the best possible level). RESULTS: 246 adults completed the survey, including those undergoing colonoscopy for symptoms (n = 87), positive FOBT (n = 92) or surveillance (n = 67). Those with symptoms had the lowest HRQoL at both baseline and follow-up, with differences observed within the HRQoL domains/areas of role function, appetite loss and bowel function on the QLU-C10D. No differences were found in HRQoL when stratified by findings at colonoscopy with both measures or when comparing baseline and follow-up responses. Participants reporting full health with EQ-5D-5L (21% at baseline and 16% at follow-up) still had problems on the QLU-C10D, with fatigue and sleep at baseline and with role function and fatigue at follow-up. CONCLUSION: Patients undergoing colonoscopy for symptoms had lower HRQoL compared to surveillance or positive FOBT. The cancer-specific QLU-C10D was more sensitive and had greater discriminant ability between patients undergoing colonoscopy for different indications.

Patient-reported quality of life and working status outcomes in ambulatory patients with idiopathic inflammatory myopathy.

OBJECTIVE: To investigate the health-related quality of life (HR-QoL), work productivity and activity impairment and associated factors among patients with idiopathic inflammatory myopathy (IIM). METHODS: This was an observational, cross-sectional study. The 189 ambulatory patients with IIM were recruited from May 2019 to May 2022. HR-QoL was measured by the European Quality of Life 5-Dimension (EQ-5D) questionnaire. The Work Productivity and Activity Impairment (WPAI) questionnaire was used to evaluate work productivity and activity impairment. The IIM-related parameters were assessed by the 8-item Manual Muscle Test (MMT-8), Myositis Disease Activity Assessment visual analogue scale (MYOACT), Myositis Damage Index (MDI), Disease Activity Score (DAS) and Physician/Patient Global Assessment (PhGA/PtGA). Quantile regression and ordinal logistic regression were performed to identify the factors, considering EQ-5D or WPAI scores as dependent variables, respectively. RESULTS: Of the 189 IIM patients enrolled, 60% had DM, 13% had PM and 27% had clinical amyopathic DM. The median EQ-5D score was 1.00 (95% CI 0.73, 1.00), 28% were employed and 45% of overall work was impaired due to health problems. EQ-5D values were positively associated with MMT-8 and negatively with MYOACT, DAS, MDI-global and PhGA/PtGA. For the WPAI, activity impairment was associated with a lower MMT-8 score, older onset age and higher PhGA only in 25th-75th percentile. Increased PtGA was associated with increased activity and overall working productivity impairment in most quantiles (P<0.05). CONCLUSION: Multiple disease characteristics were associated with reduced HR-QoL or working productivity impairment in patients with IIM, especially for PtGA.

How do patient reported outcome measures affect treatment intensification and patient satisfaction in the management of psoriatic arthritis? A cross sectional study of 503 patients.

OBJECTIVES: The ASSIST study investigated prescribing in routine psoriatic arthritis (PsA) care and whether the patient reported outcome: PsA Impact of Disease questionnaire (PsAID-12), impacted treatment. This study also assessed a range of patient and clinician factors and their relationship to PsAID-12 scoring and treatment modification. METHODS: Patients with PsA were selected across the UK and Europe between July 2021-March 2022. Patients completed the PsAID questionnaire, with the results shared with their physician. Patient characteristics, disease activity, current treatment methods, treatment strategies, medication changes and patient satisfaction scores were recorded. RESULTS: 503 patients recruited. 36.2% had changes made to treatment, 88.8% of this had treatment escalation. Overall, the mean PsAID-12 score was higher for patients with treatment escalation; the PsAID-12 score was associated with odds of treatment escalation (OR: 1.58; p< 0.0001). However, most clinicians reported PsAID-12 did not impact their decision to escalate treatment, instead supporting treatment reduction decisions. Physician's assessment of disease activity had the most statistically significant effect on likelihood of treatment escalation, (OR = 2.68, per 1-point score increase). Escalation was more likely in patients not treated with biologic therapies. Additional factors associated with treatment escalation included: patient characteristics, physician characteristics, disease activity and disease impact. CONCLUSION: This study highlights multiple factors impacting treatment decision making for individuals with PsA. PsAID-12 scoring correlates with multiple measures of disease severity and odds of treatment escalation. However, most clinicians reported the PsAID-12 did not influence treatment escalation decisions. PsAID scoring could be used to increase confidence in treatment de-escalation.

Association of social support and health status outcomes in peripheral artery disease.

OBJECTIVE: A critical goal in the care of patients with peripheral artery disease (PAD) is to optimize their health status; that is, their symptoms, function, and quality of life. Social support has been proposed to be a predictor of disease-specific health status in patients with PAD. However, the prevalence of low perceived social support, the association with health status outcomes, and the interaction with other biopsychosocial variables, is unknown. Our aim was to assess the association of baseline perceived social support with health status at 12 months in patients with PAD. METHODS: The Patient-Centered Outcomes Related Treatment Practices in Peripheral Arterial Disease: Investigating Trajectories (PORTRAIT) registry, which enrolled patients with PAD in the United States, the Netherlands, and Australia from 2011 to 2015, was used. Perceived social support was assessed at baseline with the Enhancing Recovery in Coronary Heart Disease Patients (ENRICHD) Social Support Inventory (ESSI), and disease-specific (Peripheral Artery Disease Questionnaire [PAQ]) and generic health status (Euro-Quality of Life Visual Analog Scale [VAS] and EQ-5D-3L Index) questionnaires were assessed at baseline and 12 months. Low social support was defined as a score of ≤3 on two items and an ESSI score of ≤18. A hierarchical mixed level linear regression model adjusting for biopsychosocial variables was used to assess the association between low perceived social support and the ESSI score with health status at 12 months. RESULTS: A total of 949 patients were included (mean age, 67.64 ± 9.32 years; 37.9% female), with low social support being present in 18.2%. Patients with low social support were more likely to not be married or to be living alone (50.0% vs 77.5%; P < .001); have more financial constraints; have more depressive, stress, and anxiety symptoms; and have lower disease-specific and generic health status at baseline and at 12 months. In the unadjusted model, low social support was associated with a -7.02 (95% confidence interval [CI], -10.97 to -3.07) point reduction in the PAQ, -7.43 (95% CI, -10.33 to -4.54) in the VAS, and -0.06 (95% CI, -0.09 to -0.03) in the EQ-5D-3L Index. Adjusting for biopsychosocial factors minimally attenuated these associations (PAQ: -6.52; 95% CI, -10.55 to -2.49; P = .002; VAS: -5.39; 95% CI, 8.36 to -2.42; P < .001; EQ-5D-3L Index: -0.04; 95% CI, -0.07 to 0.01; P = .022). The ESSI per-point score was associated with a decrease of 0.51 (95% CI, 0.18-0.85; P = .003) in PAQ and 0.46 (95% CI, 0.12-0.61; P = .004) in the VAS. CONCLUSIONS: Among patients with PAD, low social support was frequent and associated with a lower health status at 1 year independent of other biopsychosocial variables. Improving social support could improve health status and outcomes in PAD.

Factors Influencing Health-Related Quality of Life of Patients with Spinocerebellar Ataxia.

BACKGROUND: Little is known about the progression of health-related quality of life (HRQoL) and predicting factors in spinocerebellar ataxia (SCA). Such knowledge is crucial to identify modifiable factors promoting everyday life with SCA and attenuating HRQoL decline. OBJECTIVES: This study is to assess HRQoL progression and identify factors affecting SCA patients' HRQoL. METHODS: Longitudinal data (three-year follow-up) of 310 SCA patients of the European SCA3/Machado-Joseph-Disease Initiative (ESMI) (2016-2022) and 525 SCA patients (SCA1, SCA2, SCA3 or SCA6) of the EUROSCA natural history study cohort (2006-2015) were assessed. Both large cohort studies share standardized assessments of clinical measures, SARA, INAS, PHQ-9, and HRQoL (EQ-5D-3L). The association between HRQoL and clinical measures was assessed by Spearman Correlation (rs). Multivariable panel regression models were performed to evaluate the impact of patients' socio-demographics, age of onset, SCA type and body mass index (BMI), and clinical measures on HRQoL progression. RESULTS: HRQoL significantly decreased over one (- 0.014, p = 0.095), two (- 0.028, p = 0.003), and three years (- 0.032, p = 0.002). Ataxia severity and mental health strongly correlated with HRQoL (rsSARA = - 0.589; rsPHQ-9 = - 0.507). HRQoL more intensively declined in male (ß = - 0.024, p = 0.038) patients with an earlier age of onset (ß = 0.002, p = 0.058). Higher progression of ataxia severity (ß = - 0.010, p ≤ 0.001), mental health problems (ß = - 0.012, p < 0.001), and higher BMI (ß = - 0.003, p = 0.029) caused more severe decline of patients' HRQoL over time. DISCUSSION: In absence of curative treatments, stronger focus on mental health and weight influence could help clinical evaluation and accompany treatment improving SCA patients' HRQoL, especially in male patients with early disease onset.

Impact of multiple sclerosis comorbidities on quality of life and job activity.

BACKGROUND: Multiple sclerosis (MS) is frequently accompanied by comorbid conditions. OBJECTIVES: This study aimed to determine the prevalence of key comorbid conditions in people with multiple sclerosis (pwMS) and assess their impact on quality of life and work-related activities. METHODS: A cross-sectional study involving 755 pwMS from two third-level Italian MS centers was conducted. Comorbidities were identified from medical records, and quality of life was assessed using the EQ-5D-3L questionnaire. Work-related challenges were evaluated using the Multiple Sclerosis Questionnaire for Job Difficulties (MSQ-Job). RESULTS: 53.8% of pwMS had at least one comorbidity. Hypertension, depression, and anxiety were the most prevalent. Comorbidity presence was associated with reduced quality of life scores in almost all EQ-5D-3L domains and greater job difficulties in all but one MSQ-Job domain. CONCLUSION: Comorbidities in pwMS are prevalent and have a profound influence on quality of life and work-related activities. This comprehensive study offers new insights into the role of comorbidities in MS within the Italian context, emphasizing the need for a multidisciplinary approach in MS management. Further research is crucial to deepen our understanding of these findings in the broader Italian MS community.

Health utilities in adults with hemophilia A: A retrospective cohort study.

INTRODUCTION: Haemophilia A negatively affects a patient's quality of life. There is a limited amount of health utility data (a measure of health-related quality of life) available for patients with haemophilia A. This information is crucial for cost-effectiveness analysis for haemophilia A treatment. OBJECTIVES: The goal of this project is to elicit the health utilities and factors impacting utility values for haemophilia A patients in Canada. METHODS: This is a population-based, cross-sectional, retrospective study of health utilities in patients with haemophilia A using Patient Report Outcomes Burdens and Experiences (PROBE) components from the Canadian Bleeding Disorders Registry (CBDR). A review of the mean utilities for three severity states, defined by clotting factor VIII level, was completed. A multiple linear regression analysis was completed to examine the determinants of health utilities including age, treatment type, chronic pain status, number of limited joints, and bleed rate. RESULTS: The average utility values (and standard deviations) for patients with haemophilia A in Canada are .79(.17), .76(.20), and .77(.19) for patients with severe, moderate, and mild haemophilia. The regression showed chronic pain status and the number of additional comorbidities as major significant factors (p-value < .001) in haemophilia A utility. Haemophilia severity was shown to be a major factor with smaller p-value (p-value < .05). CONCLUSIONS: Haemophilia A patients have lower utility than the general population. Chronic pain was shown to be a significant, major factor in health-related quality of life. Our study is essential for valuing health outcomes in haemophilia A-related cost-effectiveness analysis.

Valuation of EQ-5D-5L in the Kingdom of Saudi Arabia: A National Representative Study.

OBJECTIVES: To estimate a Saudi-specific value set for the EQ-5D-5L questionnaire using the EuroQol Valuation Technology program and the EuroQol Group's standard protocol. METHODS: Participants were quota-sampled from the Saudi adult population based on residency location, age group, gender, education level, and employment status. The participants were guided through the completion of composite time trade-off (cTTO) and discrete choice experiment (DCE) tasks by trained interviewers using EuroQol Valuation Technology software. Quality control (QC) measures were used to ensure good data quality. Random intercept and Tobit models analyzed the cTTO data, as well as models correcting for heteroskedasticity. DCE data were analyzed using conditional logit models, whereas hybrid models were used to analyze the cTTO and DCE data jointly. To evaluate model performance, prediction accuracy, logical consistency, significance level, and goodness of fit were used. RESULTS: The valuation study included a representative sample of the Saudi population (N = 1000). The hybrid heteroskedastic model without a constant was chosen as the preferred model for generating the value set. The predicted values ranged from -0.683 for the worst health state ("55555") to 1 for the full health state ("11111"). Pain and discomfort had the largest impact on health-state preference values, whereas usual activities had the least. CONCLUSION: The value set for the Kingdom of Saudi Arabia is the first value set for the EQ-5D-5L for any country in the Middle East. The value set can be used in Saudi health system economic evaluations and decision making.

Testing 2 Alternative Time Trade-Off Methods for Valuation of Children's Health States.

OBJECTIVES: Composite time trade-off (cTTO) values for EQ-5D-Y-3L health states tend to be high, raising concerns about sensitivity particularly for mild or moderate health states. We conceptualized and pilot tested 2 alternative time trade-off (TTO) variants: the caregiver TTO (CGTTO) and lag-time TTO (LTTO). METHODS: We collected CGTTO and LTTO data in China for 10 EQ-5D-Y-3L health states and compared the resulting values, respondent feedback, and task completion times with those from an EQ-5D-Y-3L valuation study using cTTO. We also examined how age and parental status of respondents could affect TTO values. RESULTS: A total of 304 participants were included in this study. Overall, cTTO showed statistically better results in all feedback questions. On a 5-point Likert scale where lower score means greater agreement, the mean (SD) feedback scores for cTTO, LTTO, and CGTTO were 1.18 (0.58), 1.45 (0.91), and 1.65 (1.02) for "easy to understand"; 1.45 (0.91), 1.94 (1.08), and 1.86 (1.24) for "easy to differentiate"; and 3.61 (1.29), 2.97 (1.33), and 3.02 (1.50) for "difficult to decide," respectively. The mean (SD) TTO values of all 10 states were 0.463 (0.494), 0.387 (0.555), and 0.123 (0.710) for cTTO, LTTO, and CGTTO, respectively. The effects of age and parental status on TTO values differed by the 3 methods. CONCLUSIONS: LTTO and CGTTO produce values with good characteristics and merit further investigation. Researchers need to be aware of the differences in design and values when using the TTO method to value children's health states.

A Head-to-Head Comparison of EQ-HWB and EQ-5D-5L in Patients, Carers, and General Public in China.

OBJECTIVES: This study aimed to understand the psychometric properties of EQ Health and Wellbeing (EQ-HWB) and to examine its relationship with EQ-5D-5L in a sample covering patients, carers, and general public. METHODS: A cross-sectional study was conducted in Guizhou Province, China. The acceptability, convergent validity (using Spearman correlation coefficients), internal structure (using exploratory factor analysis), and known-group validity of EQ-HWB, EQ-HWB-Short (EQ-HWB-S), and EQ-5D-5L were reported and compared. RESULTS: A total of 323 participants completed the survey, including 106 patients, 101 carers, and 116 individuals from the general public. Approximately 7.4% of participants had at least 1 missing response. In the EQ-HWB and EQ-5D-5L items related to activities, there were more level 1 responses. The correlations between EQ-HWB and EQ-5D-5L items ranged from low to high, confirming the convergent validity of similar aspects between the 2 instruments. Notably, EQ-HWB measures 2 additional factors compared with EQ-5D-5L or EQ-HWB-S, both of which share 3 common factors. When the patient group was included, EQ-5D-5L had the largest effect size, but it failed to differentiate between the groups of general public and carers. Both EQ-HWB and EQ-HWB-S demonstrated better known-group validity results when carers were included. CONCLUSIONS: EQ-HWB measures a broader quality of life construct that goes beyond health measured by EQ-5D-5L. By encompassing a broader scope, the impact of healthcare interventions may become diluted, given that other factors can influence well-being outcomes as significantly as health conditions do.

The Measurement Properties of the EQ-HWB and the EQ-HWB-S in Italian Population: A Comparative Study With EQ-5D-5L.

OBJECTIVES: The EQ Health and Well-being, EQ-HWB (25-item) and the EQ-HWB-S (9-item), are new generic measures of health and well-being. The purpose of this study was to examine the measurement properties of the EQ-HWB and EQ-HWB-S measures in relation to the EQ-5D-5L among the Italian general population. METHODS: A cross-sectional survey was conducted from October 2020 to February 2021, followed by secondary analysis of collected data from Italian adults. This analysis included response pattern distributions, correlation strength, and known-group comparison. Known-group comparison was assessed using effect sizes (ES) across health conditions, caregiver status, and social care usage. The EQ-HWB-S index-based score was based on the UK pilot value set, whereas the Italian value set was used for scoring the EQ-5D-5L index. RESULTS: Out of the 1182 participants, 461 reported having a chronic condition, 185 identified as caregivers, and 42 were social care users. EQ-HWB items (7.5%) showed fewer ceiling effects than EQ-5D-5L items (34.7%). Strong correlations (rs > 0.5) were found between overlapping EQ-HWB and EQ-5D-5L items. EQ-HWB-S and EQ-5D-5L index scores demonstrated similar discrimination based on symptomatic chronic conditions (ES d = 0.68 vs d = 0.71), but EQ-HWB-S had slightly higher ES for social care users (ES d = 0.84 vs d = 0.74). CONCLUSIONS: Initial evidence supports the validity of EQ-HWB/EQ-HWB-S because outcome measures in the Italian population. EQ-HWB-S performed comparably to EQ-5D-5L among patients and was better in differentiating social care users. A slight decrease in discriminative properties for caregivers was observed when transitioning from EQ-HWB to EQ-HWB-S.

Testing the Psychometric Properties of 9 Bolt-Ons for the EQ-5D-5L in a General Population Sample.

OBJECTIVES: 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. METHODS: 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. RESULTS: 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 (rs = 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. CONCLUSIONS: 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.

EQ-5D-5L Population Norms for China Derived From a National Health Survey.

OBJECTIVES: To develop the EQ-5D-5L (5L) population norms for China and to assess the relationship between various factors and 5L data. METHODS: This study used data derived from the Psychology and Behavior Investigation of Chinese Residents, a national sample survey of 21 909 representative participants aged 12 years and above. Participants' health-related quality of life (HRQoL) was measured by the 5L. Their socioeconomic characteristics, behavioral factors, and health conditions were also obtained from the survey. Norm scores were generated and compared for different socioeconomic variables. Multiple linear and logistic regressions were used to assess the relationships of the 3 kinds of variables with the 5L utility, visual analog scale (VAS) scores and 5L health problems. RESULTS: The mean (SD) age of participants was 39.4 (18.9) years, and 50.0% of them were female. The mean (SD) utility and VAS scores were 0.940 (0.138) and 73.4 (21.6), respectively. Participants reported considerably more problems in anxiety/depression (26.2%) and pain/discomfort (22.2%) dimensions. The gender difference in HRQoL is attenuated. The participants older than 75 years suffered from a sharp decline in HRQoL; the participants in Shanghai and Tibet provinces reported lower utility and VAS scores and more health problems. Those who were younger, with better socioeconomic status and healthier lifestyles, and without diseases tended to report higher utility and VAS scores and fewer health problems. CONCLUSIONS: This study derived the 5L population norms for China based on a representative population sample.

EQ-5D-5L Population Scores in Mainland China: Results From a Nationally Representative Survey 2021.

OBJECTIVES: There is a lack of monitoring changes in the population scores of the most recent version, EQ-5D-5L, in mainland China. This study aimed to address this knowledge gap by assessing the EQ-5D-5L scores in mainland China using a nationally representative sample. METHODS: Data were extracted from the 2021 Survey of Health Index of Chinese Families, which covered 31 provinces/autonomous regions/municipalities in mainland China. The survey used a multistage quota sampling strategy encompassing 120 prefecture-level cities. Quotas were allocated to each prefecture-level city in accordance with the 2020 China Population Census. This approach resulted in a final sample of 11 030 eligible questionnaires. The utility index (UI) and EuroQol Visual Analog Scale (EQ VAS) scores were reported for the entire sample (age-gender-urban/rural weighted) and by the characteristics of the study participants. RESULTS: The study participants had a weighted mean UI of 0.939 (SD 0.135) and EQ VAS score of 80.19 (SD 18.39). The most commonly reported problem was anxiety/depression (26.37%), whereas self-care was the least reported problem (6.18%). Those who were male, were younger, lived without chronic conditions and disabilities, had higher levels of education, earned higher monthly household income, and were covered by basic medical insurance for urban employees had higher scores in both the UI and EQ VAS. CONCLUSION: This study revealed slightly lower UI scores despite a much higher drop in EQ VAS scores whereas China maintained minimum cases of COVID-19 in 2021 compared with the population norms recorded in 2019. Further studies are warranted to unveil the full impacts of COVID-19 outbreaks.

Valuation of Multi-Dimensional Health States With a Bolt-On: Is There a Shortcut?

OBJECTIVES: To use the EQ-5D questionnaire with bolt-on dimensions in economic evaluation studies, new value sets are needed. In this study, we explored the feasibility of a new approach called the scaling factor model, which estimates bolt-on value sets using estimated EQ-5D dimensional weights. METHODS: We designed a 2-arm study, inviting university students to value health states with and without bolt-on items using the composite time trade-off method. We selected 25 health states from an orthogonal array and added the 5 mildest EQ-5D states in the design. In arm 1, EQ-5D without self-care and standard EQ-5D states were valued, and in arm 2, standard EQ-5D states and EQ-5D with vision were valued. By arm, we compared the mean observed values of health states with and without bolt-on item. Next, by arm, we estimated value sets for the EQ-5D with bolt-on states using both standard model and scaling factor model. Model performances were compared in terms of prediction accuracy and correlation with likelihood-based mean values. RESULTS: Adding a five-level bolt-on to EQ-5D resulted in statistically lower values. This effect was consistent across 2 arms and bolt-on items. The scaling factor models outperformed the standard models in all statistics. CONCLUSIONS: The scaling factor model offers a methodologically viable and low-cost option for producing value sets for EQ-5D supplemented with bolt-on items. Future studies should further test this method using other bolt-on items and more relevant study populations.

A New and Improved Experimental Design for the Discrete Choice Experiment Module of the EuroQol Valuation Technology Protocol.

OBJECTIVES: 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. METHODS: 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. RESULTS: 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. CONCLUSIONS: 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.

Testing "Pits" Time Trade-Off: Can Data Quality be Improved by Removing Death From Valuation of Health States?

OBJECTIVES: Composite time trade-off (cTTO) often exhibits issues such as a value clustering at -1, potentially due to suboptimal valuation task design. We developed a novel time trade-off which uses the "pits" state as an anchor state and enables a unified time trade-off procedure for all health states (referred to as "pTTO"). This study aimed to test the pTTO for valuation of EQ-5D-5L health states. METHODS: A total of 120 members of the general public were invited to value 3 pairs of dominant/dominated EQ-5D-5L states using both cTTO and pTTO and the "pits" state using cTTO. We compared the pTTO with cTTO in terms of feasibility (interviewer-rated task-completing difficulty), acceptability (participant-reported task experience), value distribution, logical consistency, and discriminatory ability (using the standardized response mean, an effect size measure, derived from 3 pairwise comparisons of states with dominant/dominated relationship). RESULTS: The pTTO demonstrated similar feasibility to cTTO but slightly lower acceptability. The pTTO values exhibited a smoother and more continuous distribution compared with cTTO values. Relative to the cTTO, the pTTO showed higher individual-level logical consistency rate for the pairs comprising severe states (stringent criterion: 53.04% vs 17.39%; less stringent criterion: 82.61% vs 78.26%). The standardized response mean value based on pTTO values were higher than those based on cTTO values for the pair comprising severe states (0.388 vs -0.140). CONCLUSIONS: The pTTO appeared to be feasible and acceptable to value EQ-5D-5L states. The pTTO values were less clustered, more logically consistent, and discriminative compared with the cTTO values for severe health states.

A Feasible Estimation of a "Corrected" EQ-5D Social Tariff.

OBJECTIVES: To demonstrate the feasibility of estimating a social tariff free of utility curvature and probability weighting biases and to test transferability between riskless and risky contexts. METHODS: Valuations for a selection of EQ-5D-3L health states were collected from a large and representative sample (N = 1676) of the Spanish general population through computer-assisted personal interviewing. Two elicitation methods were used: the traditional time trade-off (TTO) and a novel risky-TTO procedure. Both methods are equivalent for better than death states, which allowed us to test transferability of utilities across riskless and risky contexts. Corrective procedures applied are based on rank-dependent utility theory, identifying parameter estimates at the individual level. All corrections are health-state specific, which is a unique feature of our corrective approach. RESULTS: Two corrected value sets for the EQ-5D-3L system are estimated, highlighting the feasibility of developing national tariffs under nonexpected utility theories, such as rank-dependent utility. Furthermore, transferability was not supported for at least half of the health states valued by our sample. CONCLUSIONS: It is feasible to estimate a social tariff by using interviewing techniques, sample sizes, and sample representativeness equivalent to prior studies designed to generate national value sets for the EQ-5D. Utilities obtained in distinct contexts may not be interchangeable. Our findings caution against routinely taking transferability of utility for granted.