Health-related quality of life assessment in health economic analyses involving type 2 diabetes.

AIM: Incorporating health-related quality of life (HRQoL) measures into health economic analyses can help to provide evidence to inform decisions about how to improve patient outcomes in the most cost-effective manner. The aim of this narrative review was to assess which HRQoL instruments have been used in economic evaluations of type 2 diabetes management including in Indigenous communities. METHOD: MEDLINE (Ovid), Embase (Ovid) and Cochrane were searched from inception to June 2022. Studies included patients with type 2 diabetes; economic evaluations, derived scores from direct questioning of individuals; and were in English. Records were assessed for bias using the JBI critical appraisal tools. RESULTS: A total of 3737 records were identified, with 22 publications meeting the criteria for inclusion. Across those 22 articles, nine HRQoL instruments had been utilised. Generic tools were most frequently used to measure HRQoL, including EQ-5D (-3 L and -5 L) (n = 10, 38%); SF-12 (n = 5, 19%); and SF-36 (n = 4, 15%). Two tools addressing the specific stressors faced by people with type 2 diabetes were utilised: Problem Areas In Diabetes tool (n = 1, 4%) and Diabetes Distress Scale (n = 1, 4%). Two publications reported whether the study population included Indigenous peoples. CONCLUSION: A wide range of HRQoL instruments are used in economic evaluations of type 2 diabetes management, with the most frequent being varying forms of the EQ-5D. Few economic evaluations noted whether Indigenous peoples were featured in the study population. More research into HRQoL in people living with type 2 diabetes is urgently needed to improve evidence on effectiveness and cost-effectiveness of interventions.

Capturing the Value of Vaccination within Health Technology Assessment and Health Economics-Practical Considerations for Expanding Valuation by Including Key Concepts.

Following the development of a value of vaccination (VoV) framework for health technology assessment/cost-effectiveness analysis (HTA/CEA), and identification of three vaccination benefits for near-term inclusion in HTA/CEA, this final paper provides decision makers with methods and examples to consider benefits of health systems strengthening (HSS), equity, and macroeconomic gains. Expert working groups, targeted literature reviews, and case studies were used. Opportunity cost methods were applied for HSS benefits of rotavirus vaccination. Vaccination, with HSS benefits included, reduced the incremental cost-effectiveness ratio (ICER) by 1.4-50.5% (to GBP 11,552-GBP 23,016) depending on alternative conditions considered. Distributional CEA was applied for health equity benefits of meningococcal vaccination. Nearly 80% of prevented cases were among the three most deprived groups. Vaccination, with equity benefits included, reduced the ICER by 22-56% (to GBP 7014-GBP 12,460), depending on equity parameters. Macroeconomic models may inform HTA deliberative processes (e.g., disease impact on the labour force and the wider economy), or macroeconomic outcomes may be assessed for individuals in CEAs (e.g., impact on non-health consumption, leisure time, and income). These case studies show how to assess broader vaccination benefits in current HTA/CEA, providing decision makers with more accurate and complete VoV assessments. More work is needed to refine inputs and methods, especially for macroeconomic gains.

Is anchoring at 'dead' a theoretical requirement for health state valuation?

Values that accompany generic health measures are typically anchored at 1 = full health and 0 = dead. Some health states may then be considered 'worse than dead' (WTD) and assigned negative values, which causes fundamental measurement problems. In this paper, we challenge the assumption that anchoring values at 'dead = 0' is necessary for quality-adjusted life year (QALY) estimation. We summarise the role of 'dead' in health state valuation and consider three critical questions: (i) whether the measurement properties of health state values require 'dead'; (ii) whether 'dead' needs to be valued relative to health states; and (iii) whether values for states WTD are meaningful or useful. We conclude that anchoring 0 at dead is not a requirement of health status measurement or cost-effectiveness analysis. This results from reframing QALYs as the relevant unit of measurement and reframing values as being derived from QALYs rather than the reverse.

Rationale, conceptual issues, and resultant protocol for a mixed methods Person Trade Off (PTO) and qualitative study to estimate and understand the relative value of gains in health for children and young people compared to adults.

BACKGROUND: Economic evaluation of healthcare typically assumes that an identical health gain to different patients has the same social value. There is some evidence that the public may give greater value to gains for children and young people, although this evidence is not always consistent. We present a mixed methods study protocol where we aim to explore public preferences regarding health gains to children and young people relative to adults, in an Australian setting. METHODS: This study is a Person Trade Off (PTO) choice experiment that incorporates qualitative components. Within the PTO questions, respondents will be asked to choose between treating different groups of patients that may differ in terms of patient characteristics and group size. PTO questions will be included in an online survey to explore respondent views on the relative value of health gains to different age groups in terms of extending life and improving different aspects of quality of life. The survey will also contain attitudinal questions to help understand the impact of question style upon reported preferences. Additionally, the study will test the impact of forcing respondents to express a preference between two groups compared with allowing them to report that the two groups are equivalent. One-to-one 'think aloud', semi-structured interviews will be conducted to explore a sub-sample of respondents' motivations and views in more detail. Focus groups will be conducted with members of the public to discuss the study findings and explore their views on the role of public preferences in health care prioritisation based on patient age. DISCUSSION: Our planned study will provide valuable information to healthcare decision makers in Australia who may need to decide whether to pay more for health gains for children and young people compared with adults. Additionally, the methodological test of forcing respondent choice or allowing them to express equivalence will contribute towards developing best practice methods in PTO studies. The rationale for and advantages of the study approach and potential limitations are discussed in the protocol.

Comparing Generic Paediatric Health-Related Quality-of-Life Instruments: A Dimensionality Assessment Using Factor Analysis.

BACKGROUND: Widely used generic instruments to measure paediatric health-related quality of life (HRQoL) include the EQ-5D-Y-5L, Child Health Utility 9 Dimension (CHU-9D), Paediatric Quality of Life Inventory (PedsQL) and Health Utilities Index (HUI). There are similarities and differences in the content of these instruments, but there is little empirical evidence on how the items they contain relate to each other, and to an overarching model of HRQoL derived from their content. OBJECTIVE: This study aimed to explore the dimensionality of the instruments using exploratory factor analysis (EFA). METHODS: Data from the Australian Paediatric Multi-Instrument Comparison (P-MIC) Study were used. EQ-5D-Y-5L, CHU-9D, PedsQL and HUI data were collected via proxy or child self-report data. EFA was used to investigate the underlying domain structure and measurement relationship. Items from the four instruments were pooled and domain models were identified for self- and proxy-reported data. The number of factors was determined based on eigenvalues greater than 1. A correlation cut-off of 0.32 was used to determine item loading on a given factor, with cross-loading also considered. Oblique rotation was used. RESULTS: Results suggest a six-factor structure for the proxy-reported data, including emotional functioning, pain, daily activities, physical functioning, school functioning, and senses, while the self-report data revealed a similar seven-factor structure, with social functioning emerging as an additional factor. CONCLUSION: We provide evidence of differences and similarities between paediatric HRQoL instruments and the aspects of health being measured by these instruments. The results identified slight differences between self- and proxy-reported data in the relationships among items within the resulting domains.

A Comparison of the Psychometric Properties of the EQ-5D-Y-3L and EQ-5D-Y-5L Using Paediatric Multi-Instrument Comparison (P-MIC) Study Data.

BACKGROUND: The EQ-5D-Y is a generic health-related quality of life (HRQoL) measure suitable for children and adolescents. There are two versions of the instrument, which differ in response levels: the three- (Y-3L) and five-level (Y-5L) versions. The Y-5L has been developed to improve the psychometric performance of the Y-3L. OBJECTIVE: This study aims to assess the psychometric properties of Y-5L and to compare the performance of Y-5L with Y-3L in separate self- and proxy-reported samples. METHODS: Both versions of the instrument were administered to children/adolescents (self-report) or caregivers (proxy-report) of Australian children aged 5-18 years. The data were gathered as part of the Australian Paediatric Multi-Instrument Comparison (P-MIC) study. The Y-5L and Y-3L were evaluated in terms of ceiling effects, criterion validity, inconsistency, informativity, and test-retest reliability in both proxy and self-complete populations. RESULTS: Overall, 5945 respondents completed both the Y-3L and Y-5L, with 2083 proxy and 3862 self-completed responses. Ceiling effects were lower in the Y-5L than the Y-3L. Items from the same domains show the strongest correlation for both samples. Regarding informativity, the Y-5L demonstrated more discriminatory power, indicated by having a higher Shannon diversity index in all domains of the Y-5L compared with the Y-3L. Test-retest showed fair agreement between responses at baseline and follow-up. CONCLUSION: The Y-5L has demonstrated moderate validity and fair reliability for use in measuring HRQoL in children and adolescents, both when self-reported by children or proxy reported. Overall, Y-5L is a dependable and valid extension from the Y-3L.

The performance of the EQ-HWB-S as a measure of quality-of-life of caregivers in families that have experienced adverse events.

PURPOSE: The recently developed EQ Health and Wellbeing Instrument (EQ-HWB) is a broad, generic measure of quality-of-life designed to be suitable for caregivers. The aim of this study was to investigate performance and validity of the 9-item version (EQ-HWB-S) for caregivers where families had experienced adverse-life-events. METHODS: Using survey data from caregivers of children aged 0-8 years attending a community-health centre in 2021-2022, the general performance, feasibility, convergent and known-group validity, responsiveness-to-change, and test-retest reliability of the EQ-HWB-S was assessed. Twelve semi-structured interviews were conducted with survey respondents to assess acceptability and content validity. RESULTS: The sample included  234 caregivers at baseline (81% female, mean age 36-years, 38% Australian-born) and 190 at 6-months follow-up. Most EQ-HWB-S item responses were evenly spread, except for 'Mobility'. The instrument showed good convergent validity with psychological distress (Kessler 6 (K6)) and personal-wellbeing (PWI-A) scales. EQ-HWB-S level sum-scores and preference-weighted scores were significantly different in all known-group analyses, in expected directions, and the instrument was responsive to change. For test-retest reliability, Intraclass Correlation Coefficients were excellent and individual item Kappa scores were moderate. The instrument was well received by interviewees who found the questions clear and relevant. The items were appropriate for parents experiencing adversity and carers of children with additional needs. CONCLUSION: The EQ-HWB-S appeared valid, responsive to change, feasible, and well accepted by caregivers. By demonstrating the validity of the EQ-HWB-S in this hard-to-reach population of caregivers in families experiencing adverse events, this study adds to existing international evidence supporting its use.

A happiness approach to valuing health states for children.

Preference weights are widely used to score generic health states into utility indexes for estimation of quality adjusted life years (QALYs) and to aid health care funding decisions. To date, health state utilities are predominantly derived using stated preference methods based on decision utility. This paper tests an alternative and generates preference weights using experienced utility for children based on the Child Health Utility 9D (CHU9D) descriptive system. We estimate the relative values of the CHU9D health states with regard to experienced utility, where experienced utility is approximated by self-reported happiness. A nationally-representative longitudinal survey was used including 6090 Australian children aged 12-17 years surveyed over 2014-2018. The derived weights were then applied to calculate the utility decrements for a few common child health conditions. We found that the estimated utility decrements are largely similar to those estimated using the published CHU9D Australian adolescent weights based on decision utility, except for pain and depression. A smaller utility decrement for pain and a larger utility decrement for depression were indicated by experienced utility. We contribute to the literature by showing that using experienced utility methods to generate preference weights for health states is possible, and we discuss some important methodological challenges for future studies such as the impracticability of anchoring to 'dead' when utilizing experienced utility.

The Ceiling Effects of EQ-5D-3L and 5L in General Population Health Surveys: A Systematic Review and Meta-Analysis.

OBJECTIVES: This review aims to examine the ceiling effects of EQ-5D-3L (3L) and EQ-5D-5L (5L) in general adult populations and identify the factors influencing these effects. METHODS: We searched 8 databases for observational studies published in English from inception to 24 July 2023. Ceiling effects were calculated by dividing the number of participants reporting full health at dimension or profile level by the total sample size. Subgroup analysis and meta-regression using the metafor package in R software were performed. RESULTS: We identified 94 studies from 70 articles, including 4 543 647 adults across 37 countries. The global pooled proportion of individuals reporting full health ("11111") was 56% (95% CI 51%-62%) for 3L and 49% (95% CI 44%-54%) for 5L. The self-care dimension showed the highest ceiling effects (3L: 97%; 5L: 94%), whereas pain/discomfort had the lowest (3L: 69%; 5L: 60%). The ceiling effects in East/South-East Asia were higher than in Europe by 25% (95% CI 18%-32%) in 3L and 9% (95% CI -2%-20%) in 5L. Adjusting for mean age and proportion of males, significant regional differences persisted in the overall profile level of 3L, in all 3L dimensions (except for self-care), and 5L dimensions (except for pain/discomfort and anxiety/depression). CONCLUSIONS: This review highlights significant ceiling effects in the EQ-5D, especially in Asian populations. The 5L version exhibited fewer ceiling effects than the 3L, indicating its superiority for general population surveys. Further research is crucial to understand the disparities in self-reported health outcomes between Asians and other populations.

Exploring the Use of Pictorial Approaches in the Development of Paediatric Patient-Reported Outcome Instruments: A Systematic Review.

INTRODUCTION: Children may find self-reporting health-related quality of life (HRQoL) using patient-reported outcome measures (PROMs) presented in text-based formats difficult, particularly younger children and children with developmental delays or chronic illness. In such cases, pictorial PROMs (where pictorial representations are used alongside or to replace text) may offer a valid alternative. AIM: This systematic literature review focused on identifying and describing paediatric PROMs that incorporate pictorial approaches, providing children with more effective means to express their HRQoL. METHODS: Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines were followed. Seven electronic databases were searched from inception to 1 March 2022. There were no country restrictions applied to the search; all English-language studies were considered for inclusion in the review. Characteristics and development methods of the identified pictorial PROMs were evaluated against context-specific good practice guidelines published by The Professional Society for Health Economics and Outcomes Research (ISPOR). RESULTS: A total of 22 paediatric pictorial PROMs, comprising 28 unique versions, were identified. These PROMs were predominantly developed in the USA and the UK, targeting children aged 3-18 years. Likert scales with pictorial anchors, particularly happy-sad faces, were commonly used for response options, appearing in 15 (54%) of the PROMs. Various graphic methods, such as happy-sad faces, cartoons, and thermometers, were adapted to specific content domains. These PROMs covered a wide range of domains, including physical and emotional health and social functioning. Emphasis was placed on content validity, including active child participation in developing pictorial elements. Notably, children's participation was sought during the development of the pictorial elements for 13 (46%) of the PROMs. Various development methods were employed, with 43% of paediatric PROMs using literature reviews, 43% using focus groups, and 32% involving expert consultation. Interviews emerged as the primary method, being employed in 61% of the studies. Additionally, three measures specifically addressed cross-cultural considerations. CONCLUSION: Paediatric pictorial PROMs offer child-friendly tools for assessing HRQoL for application with children who find reading and understanding text-based PROMs challenging. There is some evidence that pictorial PROMs facilitate self-report in this population and improve measurement properties compared to text-only PROMs. Further research is needed to develop, validate, and test paediatric pictorial PROMs, with an emphasis on including children from the inception in the co-design process.

Comparing the Psychometric Performance of Generic Paediatric Health-Related Quality of Life Instruments in Children and Adolescents with ADHD, Anxiety and/or Depression.

OBJECTIVE: The aim of this study was to examine the validity, reliability and responsiveness of common generic paediatric health-related quality of life (HRQoL) instruments in children and adolescents with mental health challenges. METHODS: Participants were a subset of the Australian Paediatric Multi-Instrument Comparison (P-MIC) study and comprised 1013 children aged 4-18 years with attention-deficit/hyperactivity disorder (ADHD) (n = 533), or anxiety and/or depression (n = 480). Participants completed an online survey including a range of generic paediatric HRQoL instruments (PedsQL, EQ-5D-Y-3L, EQ-5D-Y-5L, CHU9D) and mental health symptom measures (SDQ, SWAN, RCADS-25). A subset of participants also completed the HUI3 and AQoL-6D. The psychometric performance of each HRQoL instrument was assessed regarding acceptability/feasibility; floor/ceiling effects; convergent validity; known-group validity; responsiveness and test-retest reliability. RESULTS: The PedsQL, CHU9D, EQ-5D-Y-3L and EQ-5D-Y-5L showed similarly good performance for acceptability/feasibility, known-group validity and convergent validity. The CHU9D and PedsQL showed no floor or ceiling effects and fair-good test-retest reliability. Test-retest reliability was lower for the EQ-5D-Y-3L and EQ-5D-Y-5L. The EQ-5D-Y-3L showed the highest ceiling effects, but was the top performing instrument alongside the CHU9D on responsiveness to improvements in health status, followed by the PedsQL. The AQoL-6D and HUI3 showed good acceptability/feasibility, no floor or ceiling effects, and good convergent validity, yet poorer performance on known-group validity. Responsiveness and test-retest reliability were not able to be assessed for these two instruments. In subgroup analyses, performance was similar for all instruments for acceptability/feasibility, known-group and convergent validity, however, relative strengths and weaknesses for each instrument were noted for ceiling effects, responsiveness and test-retest reliability. In sensitivity analyses using utility scores, performance regarding known-group and convergent validity worsened slightly for the EQ-5D-Y-3L and CHU9D, though improved slightly for the HUI3 and AQoL-6D. CONCLUSIONS: While each instrument showed strong performance in some areas, careful consideration of the choice of instrument is advised, as this may differ dependent on the intended use of the instrument, and the age, gender and type of mental health condition of the population in which the instrument is being used. TRIAL REGISTRATION: ANZCTR-ACTRN12621000657820.

The Validity of the EuroQol Health and Wellbeing Short Version (EQ-HWB-S) Instrument in Parents of Children With and Without Health Conditions.

BACKGROUND:  The EuroQol Health and Wellbeing Short Version (EQ-HWB-S) instrument has been developed to measure the health and wellbeing of care-recipients and their caregivers for use in economic evaluation.The EQ-HWB-S has nine items, and pilot UK preference weights have now been developed. OBJECTIVE: We aimed to investigate the validity of the instrument in parents of children with and without health conditions. METHODS: EQ-HWB-S data were sourced from an Australian paediatric multi-instrument comparison study. We analysed the baseline characteristics and response distribution of the EQ-HWB-S items. Assessment of known-group validity was conducted for EQ-HWB-S items, level sum-scores and preference-weighted scores, including partial effects. Known-group analyses included three child health variables and where caregivers reported coronavirus disease 2019 (COVID-19) had impacted their wellbeing. We included analyses across gender, controlled for child and parent demographic variables, and compared scores across child health conditions. RESULTS:  Item responses were distributed as expected, with higher skew for mobility and activities. Parents experienced high levels of exhaustion. We detected significant differences between groups for level sum-scores and preference-weighted scores, as hypothesised; all tests were significant (p < 0.001), with moderate effect sizes (effect sizes were slightly higher for female than male parents). The regression analysis identified significantly different EQ-HWB-S scores for child health samples compared with the general population after controlling for demographic variables. Differences were observed between child health conditions. CONCLUSION: The EQ-HWB-S is a useful instrument to measure parent quality of life for economic evaluation in this population. Data were limited to one time point; further research should investigate the instrument's sensitivity to change and test-retest reliability in this population.

Psychometric Performance Comparison of the Adapted versus Original Versions of the EQ-5D-Y-3L and -Y-5L in Proxy Respondents for 2- to 4-Year-Olds.

INTRODUCTION: Few preference-weighted instruments are available to measure health-related quality of life in young children (2-4 years of age). The EQ-5D-Y-3L and EQ-5D-Y-5L were recently modified for this purpose. OBJECTIVE: The aim of this study was to test the psychometric properties of these adapted versions for use with parent proxies of children aged 2-4 years and to compare their performance with the original versions. It was hypothesised that the adapted instrument wording would result in improved psychometric performance. METHODS: Survey data of children aged 2-4 years were obtained from the Australian Paediatric Multi-Instrument Comparison study. Distributional and psychometric properties tested included feasibility, convergence, distribution of level scores, ceiling effects, known-group validity (Cohen's D effect sizes for prespecified groups defined by the presence/absence of special healthcare needs [SHCNs]), test-retest reliability (intraclass correlation coefficients [ICCs]), and responsiveness (standardised response mean [SRM] effect sizes for changes in health). Level sum scores were used to provide summary outcomes. Supplementary analysis using utility scores (from the Swedish EQ-5D-Y-3L value set) were conducted for the adapted and original EQ-5D-Y-3L, and no value sets were available for the EQ-5D-Y-5L. RESULTS: A total of 842 parents of children aged 2-4 years completed the survey. All instruments were easy to complete. There was strong convergence between the adapted and original EQ-5D-Y-3L and EQ-5D-Y-5L. The adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L showed more responses in the severe levels of the five EQ-5D-Y dimensions, particularly in the usual activity and mobility dimensions (EQ-5D-Y-5L: mobility level 1: adapted n = 478 [83%], original n = 253 [94%]; mobility level 4/5: adapted n = 17 [2.9%], original n = 4 [1.5%)]). The difference in the distribution of responses was more evident in children with SHCNs. Assessment of known-group validity showed a greater effect size for the adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L compared with the original instruments (EQ-5D-Y-5L: adapted Cohen's D = 1.01, original Cohen's D = 0.83) between children with and without SHCNs. The adapted EQ-5D-Y-3L and adapted EQ-5D-Y-5L showed improved reliability at 4-week follow-up, with improved ICCs (EQ-5D-Y-5L: adapted ICC = 0.83, original ICC = 0.44). The responsiveness of all instruments moved in the hypothesised direction for better or worse health at follow-up. Probability of superiority analysis showed little/no differences between the adapted and original EQ-5D-Y-3L and EQ-5D-Y-5L. Supplementary psychometric analysis of the adapted and original EQ-5D-Y-3L using utilities showed comparable findings with analyses using level sum scores. CONCLUSIONS: The findings suggest improved psychometric performance of the adapted version of the EQ-5D-Y-3L and EQ-5D-Y-5L in children aged 2-4 years compared with the original versions.

An Investigation of Inter-Rater and Intra-Proxy Agreement in Measuring Quality of Life of Children in the Community Using the EQ-5D-Y-3L.

BACKGROUND: Self-reporting of health-related quality of life (HRQoL) in children is not always feasible. To date, proxy perspectives (Proxy versions 1 and 2) using the EQ-5D-Y-3L have not been explored for its impact on agreement with child self-report. Proxy version 1 requires the proxy to consider their own view of the child's HRQoL (proxy-proxy), while with Proxy version 2, the proxy is asked to respond as they believe their child would self-report their HRQoL (proxy-child). This study compared the inter-rater and intra-proxy agreement (overall and dimension level) using the EQ-5D-Y-3L self, proxy-proxy, and proxy-child reports. METHODS: A community-based sample of child (aged 6-12 years) and parent dyads were invited to participate in a semi-structured interview. The child self-completed the EQ-5D-Y-3L independently of the parent who completed the EQ-5D-Y-3L from proxy-proxy and proxy-child perspectives. Agreement was determined using Concordance Correlation Coefficients (CCCs) for the overall (preference-weighted) HRQoL, while agreement at the dimension level was evaluated using Gwet's agreement coefficient (AC1). To assess the differences between the self and the two proxy reports, the Wilcoxon matched-pair signed-rank test was used. RESULTS: This study involved 85 child-parent dyads. The agreement between self and proxy overall HRQoL was low (fair) with both proxy-proxy (CCC = 0.28) and proxy-child (CCC = 0.26) reports. The largest discrepancy in the child-proxy agreement at dimension level with both the proxy versions was observed for 'feeling worried, sad or unhappy'. Within this dimension, the proxy-child perspective resulted in a stronger agreement (AC1 = 0.7, good) with child self-report compared with the traditional proxy-proxy perspective (AC1 = 0.58, moderate). Although the preference-weighted HRQoL was consistent across both the proxy perspectives, a significant difference was observed in the EQ VAS scores (p = 0.02). CONCLUSIONS: This study demonstrates that choice of proxy perspective may have an impact on the problems reported on HRQoL dimensions and EQ VAS scores. However, in this community-based sample of generally healthy children, no significant difference was observed in the inter-rater agreement for child-self and proxy preference-weighted EQ-5D-Y-3L values based on proxy perspectives. While this suggests that preference-weighted data are not sensitive to the choice of perspective, these findings may differ for different HRQoL instruments and for alternative value sets with different properties.

Psychometric Properties of Child Health Utility 9D (CHU9D) Proxy Version Administered to Parents and Caregivers of Children Aged 2-4 Years Compared with Pediatric Quality of Life Inventory™ (PedsQL).

OBJECTIVE: This study examines the psychometric properties of the Child Health Utility 9D (CHU9D) proxy version administered to parents/caregivers of 2-4-year-old Australian children compared with Pediatric Quality of Life Inventory™ version 4.0 (PedsQL). METHODS: Data collected in 2021/2022 from parents/caregivers of 2-4-year-olds from the Australian pediatric multi-instrument comparison study were used. Feasibility, ceiling/floor effects, test-retest reliability, convergent validity, known-group validity, and responsiveness were assessed. RESULTS: A total of 842 caregivers completed the survey at baseline, with 513 completing the follow-up survey. The CHU9D did not demonstrate ceiling effects in the sample with special health care needs, with only 6% of respondents reporting best levels for all nine dimensions. CHU9D correlated with PedsQL moderately-to-strongly between comparable items (correlation coefficients 0.34-0.70). CHU9D was able to differentiate between groups with known health differences with moderate-to-large effect sizes (Cohen's d 0.58-2.03). Moderate test-retest reliability was found for CHU9D in those reporting no health change at a 2-day follow-up (ICC 0.52). A standard response mean (SRM) of 0.25-0.44 was found for children with changes in general health and a SRM of 0.72-0.82 for children who reported worsened health when developing new illnesses, indicating small-to-large responsiveness according to different definitions of health changes. Compared with PedsQL, CHU9D had similar known-group validity and responsiveness and slightly poorer test-retest reliability. CONCLUSION: The CHU9D was found to be valid and reliable to measure health-related quality-of-life in children aged 2-4 years, although with relatively low test-retest reliability in some dimensions. Further development and validation work is warranted.

Exploring health preference heterogeneity in the UK: Using the online elicitation of personal utility functions approach to construct EQ-5D-5L value functions on societal, group and individual level.

A new method has recently been developed for valuing health states, called 'Online elicitation of Personal Utility Functions' (OPUF). In contrast to established methods, such as time trade-off or discrete choice experiments, OPUF does not require hundreds of respondents, but allows estimating utility functions for small groups and even at the individual level. In this study, we used OPUF to elicit EQ-5D-5L health state preferences from a (not representative) sample of the UK general population, and then compared utility functions on the societal-, group-, and individual level. A demo version of the survey is available at: https://eq5d5l.me. Data from 874 respondents were included in the analysis. For each respondent, we constructed a personal EQ-5D-5L value set. These personal value sets predicted respondents' choices in three hold-out discrete choice tasks with an accuracy of 78%. Overall, preferences varied greatly between individuals. However, PERMANOVA analysis showed that demographic characteristics explained only a small proportion of the variability between subgroups. While OPUF is still under development, it has important strengths: it can be used to construct value sets for patient reported outcome instruments such as EQ-5D-5L, while also allowing examination of underlying preferences in an unprecedented level of detail. In the future, OPUF could be used to complement existing methods, allowing valuation studies in smaller samples, and providing more detailed insights into the heterogeneity of preferences across subgroups.

The RETRIEVE Checklist for Studies Reporting the Elicitation of Stated Preferences for Child Health-Related Quality of Life.

BACKGROUND: Recent systematic reviews show varying methods for eliciting, modelling, and reporting preference-based values for child health-related quality-of-life (HRQoL) outcomes, thus producing value sets with different characteristics. Reporting in many of the reviewed studies was found to be incomplete and inconsistent, making them difficult to assess. Checklists can help to improve standards of reporting; however, existing checklists do not address methodological issues for valuing child HRQoL. Existing checklists also focus on reporting methods and processes used in developing HRQoL values, with less focus on reporting of the values' key characteristics and properties. We aimed to develop a checklist for studies generating values for child HRQoL, including for disease-specific states and value sets for generic child HRQoL instruments. DEVELOPMENT: A conceptual model provided a structure for grouping items into five modules. Potential items were sourced from an adult HRQoL checklist review, with additional items specific to children developed using recent reviews. Checklist items were reduced by eliminating duplication and overlap, then refined for relevance and clarity via an iterative process. Long and short checklist versions were produced for different user needs. The resulting long RETRIEVE contains 83 items, with modules for reporting methods (A-D) and characteristics of values (E), for researchers planning and reporting child health valuation studies. The short RETRIEVE contains 14 items for decision makers or researchers choosing value sets. CONCLUSION: Applying the RETRIEVE checklists to relevant studies suggests feasibility. RETRIEVE has the potential to improve completeness in the reporting of preference-based values for child HRQOL outcomes and to improve assessment of preference-based value sets.

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.