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.

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.

Health utilities of patients with epilepsy in a Canadian population.

OBJECTIVE: 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. METHODS: 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. RESULTS: 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. SIGNIFICANCE: 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.

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.

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 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.

Using the Online Elicitation of Personal Utility Functions Approach to Derive a Patient-Based 5-Level Version of EQ-5D Value Set: A Study in 122 Patients With Rheumatic Diseases From Germany.

OBJECTIVES: Traditional preference elicitation methods, such as discrete choice experiments or time trade-off, usually require large sample sizes. This can limit their applicability in patient populations, where recruiting enough participants can be challenging. The objective of this study was to test a new method, called the Online elicitation of Personal Utility Functions (OPUF) approach, to derive an EQ-5D-5L value set from a relatively small sample of patients with rheumatic diseases. METHODS: OPUF is a new type of online survey that implements compositional preference elicitation techniques. Central to the method are 3 valuation steps: (1) dimension weighting, (2) level rating, and (3) anchoring. An English demo version of the OPUF survey can be accessed at https://valorem.health/eq5d5l. From the responses, a personal EQ-5D-5L utility function can be constructed for each participant, and a group-level value set can be derived by aggregating model coefficients across participants. RESULTS: A total of 122 patients with rheumatic disease from Germany completed the OPUF survey. The survey was generally well received; most participants completed the survey in less than 20 minutes and were able to derive a full EQ-5D-5L value set. The precision of mean coefficients was high, despite the small sample size. CONCLUSIONS: Our findings demonstrate that OPUF can be used to derive an EQ-5D-5L value set from a relatively small sample of patients. Although the method is still under development, we think that it has the potential to be a valuable preference elicitation tool and to complement traditional methods in several areas.

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.

Health-Related Quality of Life of People Who Inject Drugs: The Enhancing Treatment of Hepatitis C in Opioid Substitution Settings Engage Study.

OBJECTIVES: There is limited research on health-related quality of life (HRQoL) among people who inject drugs (PWID). We evaluated the HRQoL and associated factors among a cohort of PWID in Australia. METHODS: Participants were enrolled in an observational cohort study (the Enhancing Treatment of Hepatitis C in Opioid Substitution Settings Engage Study) from May 2018 to September 2019 (wave 1) and November 2019 to June 2021 (wave 2). Participants completed the EQ-5D-5L survey at enrolment. Two-part models were used to assess the association of clinical and socioeconomic characteristics with EQ-5D-5L scores. RESULTS: Among 2395 participants (median age, 43 years; 66% male), 65% reported injecting drug use in the past month, 20% had current hepatitis C virus (HCV) infection, and 68% had no/mild liver fibrosis (F0/F1). Overall, the mean EQ-5D-5L and EQ-visual analog scale scores were 0.78 and 57, respectively. In adjusted analysis, factors associated with significantly lower EQ-5D-5L scores include older ages, female (marginal effect = -0.03, P = .014), being homeless (marginal effect = -0.04, P = .040), and polysubstance use (marginal effect = -0.05, P < .001). Factors associated with significantly higher EQ-5D-5L scores were being Aboriginal/Torres Strait Islander (marginal effect = 0.03, P = .021) and recent injecting drug use in the past 12 months. Current HCV infection and liver fibrosis stage were not associated with reduced HRQoL among the study participants. CONCLUSIONS: PWID experienced a lower HRQoL compared with the general population. Further research is needed to understand HRQoL in this population to facilitate the development of multifaceted care models for PWID beyond HCV cure and inform health economic analyses for identifying optimal health strategies for PWID.

This Dead or That Dead: Framing Effects in the Evaluation of Health States.

OBJECTIVES: Current approaches to health state valuation rely on credible classification of states as either "better than dead" or "worse than dead" (WTD). We investigate how such evaluations of health states are affected by the framing in pairwise comparison tasks. METHODS: We conducted an online survey with 361 participants to compare the propensity to evaluate a state as WTD under 6 frames: (A) using a regular time trade-off (TTO) choice task, (B) using a lead-time TTO choice task, (C) excluding the immediacy of death, (D) avoiding reference to the process of dying, (E) focusing on longevity, and (F) focusing on improvements in quality of life. Each participant evaluated 9 EQ-5D-5L health states using 3 frames. The frames were compared using several statistical approaches to confirm robustness to indirect comparisons or respondent heterogeneity and inattentiveness. RESULTS: The odds of a state being evaluated as WTD, compared with frame A, increase 2.7-fold and 1.5-fold in frame B and E, respectively, and decrease >5-fold in frame F. Frames C and D do not differ significantly from frame A. CONCLUSIONS: Different framings for questions about whether a state is WTD or better than dead, even if theoretically equivalent, yield substantially different results. Notably, whether a state is evaluated as WTD differs greatly between the regular TTO and lead-time TTO choice tasks and when either final outcomes or improvements over time are considered.

Direct and Indirect Mapping of Assessment of Quality of Life - 6 Dimensions (AQoL-6D) Onto EQ-5D-5L Utilities Using Data From a Multicenter, Cross-Sectional Study of Malaysians With Chronic Heart Failure.

OBJECTIVES: The Assessment of Quality of Life - 6 Dimensions (AQoL-6D), a generic preference-based measure, is an appealing alternative to EQ-5D-5L for assessing health status in patients with chronic heart failure (HF), given its expanded scope. However, without a Malaysian value set, the AQoL-6D cannot generate health state utility values (HSUVs) to support local economic evaluations. This study intended to develop algorithms for predicting EQ-5D-5L HSUVs from AQoL-6D in an HF population. METHODS: Cross-sectional data from a multicenter cohort of 419 HF outpatients were used. Both direct and indirect mapping approaches were attempted using 5 sets of explanatory variables and 8 models (ordinary least squares, Tobit, censored least absolute deviations, generalized linear model, 2-part model [TPM], beta regression-based model, adjusted limited dependent variable mixture model, and multinomial ordinal regression [MLOGIT]). The models' predictive performance was assessed through 10-fold cross-validated mean absolute error [MAE] and root mean squared error [RMSE]). Potential prediction bias was also examined graphically. The best-performing models, with the lowest RMSE and no bias, were then identified. RESULTS: Among the models evaluated, TPM, which included age, sex, and 5 AQoL-6D dimension scores as predictors, appears to be the best-performing model for directly predicting EQ-5D-5L HSUVs from AQoL-6D. TPM yielded the lowest MAE (0.0802) and RMSE (0.1116), and demonstrated predictive accuracy for HSUVs >0.2 without significant bias. A MLOGIT model developed for response mapping had suboptimal predictive accuracy. CONCLUSIONS: This study developed potentially useful mapping algorithms for generating Malaysian EQ-5D-5L HSUVs from AQoL-6D responses among patients with HF when direct EQ-5D-5L data are unavailable.

Estimating a Minimal Important Difference for the EQ-5D-5L Utility Index in Dialysis Patients.

OBJECTIVES: The EQ-5D-5L is a commonly used health-related quality of life instrument for evaluating interventions in patients receiving dialysis; however, the minimal important difference (MID) that constitutes a meaningful treatment effect for this population has not been established. This study aims to estimate the MID for the EQ-5D-5L utility index in dialysis patients. METHODS: 6-monthly EQ-5D-5L measurements were collected from adult dialysis patients between April 2017 and November 2020 at a renal network in Sydney, Australia. EQ-VAS and Integrated Palliative care Outcome Scale Renal symptom burden scores were collected simultaneously and used as anchors. MID estimates for the EQ-5D-5L utility index were derived using anchor-based and distribution-based methods. RESULTS: A total of 352 patients with ≥1 EQ-5D-5L observation were included, constituting 1127 observations. Mean EQ-5D-5L utility index at baseline was 0.719 (SD ± 0.267), and mean EQ-5D-5L utility decreased over time by -0.017 per year (95% CI -0.029 to -0.006, P = .004). Using cross-sectional anchor-based methods, MID estimates ranged from 0.073 to 0.107. Using longitudinal anchor-based methods, MID for improvement and deterioration ranged from 0.046 to 0.079 and -0.111 to -0.048, respectively. Using receiver operating characteristic curves, MID for improvement and deterioration ranged from 0.037 to 0.122 and -0.074 to -0.063, respectively. MID estimates from distribution-based methods were consistent with anchor-based estimates. CONCLUSIONS: Anchor-based and distribution-based approaches provided EQ-5D-5L utility index MID estimates ranging from 0.034 to 0.134. These estimates can inform the target difference or "effect size" for clinical trial design among dialysis populations.

Psychometric Properties of the EQ-5D-Y-5L for Children With Intellectual Disability.

OBJECTIVES: The EQ-5D-Y-5L is a generic preference-based measure of health-related quality of life for children. This study aimed to describe the distributional properties, test-retest reliability, and convergent validity of the EQ-5D-Y-5L in children with intellectual disability (ID). METHODS: Caregivers of children with ID (aged 4 to 18 years) completed an online survey, including a proxy-report EQ-5D-Y-5L, the Quality-of-life Inventory-Disability, and disability-appropriate measures corresponding to the EQ-5D dimensions: mobility, self-care (SC), usual activities (UA), pain/discomfort (PD), and worry/sadness/unhappiness. Twenty-one participants repeated the EQ-5D-Y-5L a few weeks later. Test-retest reliability was computed using weighted kappa and intraclass correlation coefficients, and convergent validity using Spearman's and Pearson's correlation coefficients. RESULTS: Caregivers of 234 children completed the survey, with <1% missing values. Only 1.7% reported "no problems" on all dimensions (11111). The dimensions with the lowest percentage of "no problems" were SC and UA (both 8%). Test-retest reliability coefficients were fair to substantial for 4 dimensions (weighted kappa .30 to .79) but low for PD and overall health, as measured by the visual analog scale (EQ-VAS). Convergent validity was strong (Spearman's correlation .65 to .87) for mobility, SC, and PD; moderate to strong for worry/sadness/unhappiness (.47 to .60) and the EQ-VAS (Pearson's correlation .49); and weak to moderate for UA (.21 to .52). CONCLUSIONS: Convergent validity was generally good; test-retest reliability varied. Children with ID had lower scores on SC and UA than other populations, and their EQ-VAS could fluctuate greatly, indicating poorer and less stable health-related quality of life.

How Well Does the EQ-5D-Y-5L Describe Children With Intellectual Disability?: "There's a Lot More to My Child Than That She Can't Wash or Dress Herself."

OBJECTIVES: The EQ-5D-5L is a generic health utility instrument for measuring health-related quality of life (HRQoL), with self-report and proxy report versions for children (EQ-5D-Y-5L). Children with intellectual disability (ID) are a heterogeneous population whose impairments and comorbidities place them at risk of poor HRQoL. This study aimed to describe the content validity and suitability for children with ID of a proxy report version of the EQ-5D-Y-5L as seen by their caregivers. METHODS: A proxy report EQ-5D-Y-5L was administered to caregivers of children with ID. Using cognitive think-aloud interviewing, participants were encouraged to provide the reasoning for their choices, assess the questions' relevance, comprehensibility, and comprehensiveness, and comment on the tool's strengths and weaknesses. Qualitative content analysis used both directed (deductive) and conventional (inductive) methods. RESULTS: There were 28 interviews with 30 caregivers of children with ID (aged 8-22 years, 17 boys, with autism spectrum disorder, cerebral palsy, Down syndrome, and rare genetic disorders). The EQ-5D-Y-5L was considered clear, concise, and largely relevant, but insufficiently comprehensive for this population. Interviewees sought clarification of the definition of HRQoL, whether it included unchanging impairments (vs fluctuating health states), and what basis of comparison to use (child or peer). Many interviewees suggested inclusion of questions for other domains, including communication and social engagement, equipment and human supports required, and a wider range of mental health questions. CONCLUSIONS: The study suggests that further work is required to ensure accurate responses to the EQ-5D-Y-5L from caregivers of children with ID and to describe these children adequately.

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.

The EQ-5D-5L valuation study for Trinidad and Tobago.

PURPOSE: The 2016 EQ-5D-3L value set for Trinidad and Tobago (T&T) allows for the calculation of EQ-5D-5L values via the crosswalk algorithm. The 2016 value set was based on methods predating the EQ-VT protocol, now considered the gold standard for developing EQ-5D value sets. Furthermore, direct elicitation of EQ-5D-5L is preferred over crosswalked values. This study aimed to produce an EQ-5D-5L value set for T&T. METHODS: A representative sample (age, sex, geography) of adults each completed 10 composite Time Trade-Off (cTTO) tasks and 12 Discrete Choice Experiment (DCE) tasks in face-to-face interviews. The cTTO data were analyzed using a Tobit model that corrects for heteroskedasticity. DCE data were analyzed using a mixed logit model. The cTTO and DCE data were combined in hybrid models. RESULTS: One thousand and seventy-nine adults completed the valuation interviews. Among the modelling approaches that were explored, the hybrid heteroskedastic Tobit model produced all internally consistent, statistically significant coefficients, and performed best in terms of out-of-sample predictivity for single states. Compared to the existing EQ-5D-5L crosswalk set, the new value set had a higher number of negative values (236 or 7.6% versus 21 or 0.7%). The mean absolute difference was 0.157 and the correlation coefficient between the two sets was 0.879. CONCLUSION: This study provides a value set for the EQ-5D-5L for T&T using the EQ-VT protocol. We recommend this value set for QALY computations relating to T&T.