Estimating Australian Population Utilities for Inherited Retinal Disease Using Time Trade-Off.

PURPOSE: Inherited retinal disease (IRD) causes progressive loss of visual function, degenerating towards complete blindness. Economic evaluation of gene therapies for rare forms of genetic IRDs have had to rely on health-related quality of life (HR-QoL) estimates from other diseases because there is limited data available for such a rare condition. This study aimed to estimate Australian societal-based utility values for IRD health states that can be used in cost-utility analyses (CUA) using a time trade-off (TTO) protocol adapted from a UK study. METHODS: The EuroQol Valuation Technology (EQVT) protocol composite TTO (cTTO) framework was followed, which includes worse-than-death (WTD) states and quality control (QC) measures. Preferences were collected from a general population sample of 110 Australian adult participants. Five health state vignettes from the UK study which had been validated with patients and clinicians were presented randomly to participants during videoconferencing (VC) interviews with one of four interviewers. Technical and protocol feasibility were assessed in a pilot of 10 interviews. QC measures were used to monitor interviewers' performance during the study. RESULTS: One participant withdrew consent. The final analysis was conducted on 109 respondents (including 4 non-traders). The average time to complete the interview was 44.2 minutes (SD 8.7). Participants reported mean visual analogue scale (VAS) scores between 63.15 for 'moderate impairment' and 17.98 for 'hand motion' to 'no light perception'. Mean health state utilities (HSU) varied between 0.76 (SD 0.26) in 'moderate impairment', and 0.20 (SD 0.58) in 'hand motion' to 'no light perception'. Of all HSU evaluations, 14% were considered WTD which most commonly occurred in the most severe visually impaired health state. CONCLUSION: This study provides valuable information on HSUs across a range of IRD health states from the Australian general population perspective. The utilities obtained in this study can be used as inputs into CUA of IRD therapies.

Creating an SF-6Dv2 social value set for New Zealand.

The SF-6D health descriptive system and its second version published in 2020, the SF-6Dv2, is used worldwide for valuing health-related quality of life (HRQoL) for economic evaluation and measuring patient-reported health outcomes. In this study, a valuation tool was developed and applied to create a social value set, comprising 18,750 health state values, for the SF-6Dv2 for New Zealand (NZ). This tool was adapted and extended from the one used to create a social value set for the EQ-5D-5L, a simpler health descriptive system with fewer dimensions and health states. The tool implements the PAPRIKA method, a type of adaptive discrete choice experiment, and a binary search algorithm to identify health states worse than dead and has extensive data quality controls to ensure the validity and reliability of the social value set derived from participants' personal value sets. The tool, accompanied by a short introductory video designed specifically for the SF-6Dv2, was distributed via an online survey to a large representative sample of adult New Zealanders in June-July 2022. The tool's data quality controls enabled participants who failed to understand or sincerely engage with the valuation tasks to be identified and excluded, resulting in the participants being pared down to a sub-sample of 2985 'high-quality' participants whose personal value sets were averaged for the social value set. These results, including participants' positive feedback, demonstrate the feasibility and acceptability of using the tool to value larger health descriptive systems such as the SF-6Dv2. Having successfully created an SF-6Dv2 social value set for NZ, the valuation tool can be readily applied to other countries, used to generate personal value sets for personalised medicine and adapted to create value sets for other health descriptive systems.

Putting a Dollar Value on Informal Care Time Provided to People Living With Dementia: A Discrete Choice Experiment.

OBJECTIVES: Informal care represents a significant cost driver in dementia but monetizing informal care hours to inform cost-of-illness or economic evaluation studies remains a challenge. This study aimed to use a discrete choice experiment to estimate the value of informal care time provided to people with dementia in Australia accounting for positive and negative impacts of caregiving. METHODS: Attributes and levels were derived from a literature review, interviews with carers, and advice received from an advisory group. Attributes included 4 positive and negative caregiving experiences, in addition to "hours of care provided" and the "monetary compensation from the government." A D-efficient design was constructed with 2 generic alternatives that represented hypothetical informal caregiving situations. The discrete choice experiment survey was administered online to a representative sample of the Australian general population and a group of informal carers of people with dementia. The willingness to accept estimates were calculated for the 2 samples separately using the mixed logit model in the willingness to pay space. RESULTS: Based on 700 respondents included in the analysis (n = 488 general public, n = 212 informal carers), the mean willingness to accept for an additional hour of informal care, corrected for the positive and negative impacts of informal care, was $21 (95% CI 18-23) for the general public and $20 (95% CI 16-25) for the informal carers sample. CONCLUSION: The estimates generated in this study can be used to inform future cost-of-illness studies and economic evaluations, ensuring that informal care time is considered in future policy and funding decisions.

Development of algorithms for estimating the Child Health Utility 9D from Caregiver Priorities and Child Health Index of Life with Disability.

PURPOSE: The primary aim was to determine Child Health Utility 9D (CHU9D) utilities from the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) for non-ambulatory children with cerebral palsy (CP). METHODS: One hundred and eight surveys completed by Australian parents/caregivers of children with CP were analysed. Spearman's coefficients were used to investigate the correlations between the two instruments. Ordinary least square, robust MM-estimator, and generalised linear models (GLM) with four combinations of families and links were developed to estimate CHU9D utilities from either the CPCHILD total score or CPCHILD domains scores. Internal validation was performed using 5-fold cross-validation and random sampling validation. The best performing algorithms were identified based on mean absolute error (MAE), concordance correlation coefficient (CCC), and the difference between predicted and observed means of CHU9D. RESULTS: Moderate correlations (ρ 0.4-0.6) were observed between domains of the CHU9D and CPCHILD instruments. The best performing algorithm when considering the CPCHILD total score was a generalised linear regression (GLM) Gamma family and logit link (MAE = 0.156, CCC = 0.508). Additionally, the GLM Gamma family logit link using CPCHILD comfort and emotion, quality of life, and health domain scores also performed well (MAE = 0.152, CCC = 0.552). CONCLUSION: This study established algorithms for estimating CHU9D utilities from CPCHILD scores for non-ambulatory children with CP. The determined algorithms can be valuable for estimating quality-adjusted life years for cost-utility analysis when only the CPCHILD instrument is available. However, further studies with larger sample sizes and external validation are recommended to validate these findings.

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.

Understanding caregiver burden from multiple perspectives: dyadic agreement between caregiver and care recipient.

PURPOSE: Caregiver burden (CB) is typically self-assessed by caregivers. However, an emerging concept is assessment of CB by the recipients of care, i.e., the patient. The specific objectives are (1) to assess the level of agreement between care recipients' and caregivers' view on CB, across financial, physical, emotional, and social domains; (2) to explore two care recipient perspectives: their self-perceived burden (CR-SPB), and their interpretation of the caregiver's view (Proxy-CB). METHODS: Data were collected from 504 caregiver-care recipient dyads in the U.S. using an online Qualtrics panel. The survey assessed caregiver burden using CarerQol and newly developed items. The level of agreement between responses was quantified using weighted kappa (κ) coefficients for individual items and intraclass correlation coefficients (ICC) for index/summary scores. RESULTS: The average age of caregivers was 49.2 years, and 62.7 years for care recipients. Dyads most commonly consisted of spouses/partners (34.5%); 68.3% lived together. Proxy-CB aligned more closely with caregiver's view, with moderate to substantial agreement across CB domains (from κ = 0.48 for emotional to κ = 0.66 for financial). In the same perspective, the CarerQol-7D Index showed moderate agreement (ICC = 0.58) and the summary score of CB items substantial agreement (ICC = 0.76). Care recipients generally overestimated  CB in the Proxy-CB perspective, while they underestimated it in the CR-SPB perspective. CONCLUSION: Results demonstrate there is a difference between perspectives. Strong agreement in Proxy-CB perspective suggests that care recipients can potentially substitute for caregivers depending on the domain. CR-SPB agrees less with caregivers and may provide complementary information.

Comparison of the EQ-HWB and EQ-HWB-S With Other Preference-Based Measures Among United States Informal Caregivers.

OBJECTIVES: Several measures have been used or developed to capture the health and well-being of caregivers, including the EQ Health and Well-being (EQ-HWB) and its short form, EQ-HWB-S. This study aimed to evaluate the psychometric properties and construct validity of the EQ-HWB/EQ-HWB-S in a US caregiver population. METHODS: A cross-sectional survey was conducted involving 504 caregivers. Eligible participants were 18+ years old, provided unpaid care to a relative/friend aged 18+ in the past 6 months, and spent on average of at least 1 hour per week caregiving. Survey included the following measures: EQ-HWB, Adult Social Care Outcomes Toolkit for Carers-Carer, CarerQol, and EQ-5D-5L. Psychometric properties were assessed using response distributions, floor/ceiling effects, Spearman's correlation for convergent validity, and effect sizes (ES) for known-group validity based on caregiving situations and intensity. RESULTS: The average age of caregivers was 49.2 (SD = 15.4), with 57.5% being female. More than half (54.4%) reported high caregiving intensity, and 68.3% lived with the care recipient. The EQ-HWB-S index showed a strong positive correlation with the EQ-5D-5L (rs = 0.72), Adult Social Care Outcomes Toolkit for Carers (rs = 0.54), and CarerQol (rs = 0.54) indices. Notably, the EQ-HWB-S index showed the largest ES among measures in differentiating caregiving scenarios with a large ES for caregiver's general health (d = 1.00) and small ES for caregiving intensity (d = 0.39). CONCLUSIONS: Results support construct validity of EQ-HWB and EQ-HWB-S as measures for assessing health and well-being of adult informal caregivers in comparison with other validated instruments. Differing levels of known-group validity across anchors emphasize the importance of selecting appropriate measures for caregivers, depending on research question and/or intervention aims.

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.

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.

What matters most to people with metastatic uveal melanoma? A qualitative study to inform future measurement of health-related quality of life.

Metastatic uveal melanoma (mUM) is a rare cancer with poor prognosis, but novel treatments are emerging. Currently, there are no mUM-specific health-related quality of life (HRQL) questionnaires available for clinical research. We aimed to explore how mUM and its treatment affect HRQL and assess the content validity of existing questionnaires. Participants were patients with mUM and healthcare professionals involved in their care. Qualitative data were collected using semi-structured interviews and focus groups. Data collection and analysis used an integrative approach involving inductive questions/coding to elicit new concepts and deductive questions/coding based on domains of existing HRQL questionnaires. Initial interviews/focus groups focussed on HRQL questionnaires designed for patients with uveal melanoma or liver metastases. As new concepts were elicited, domains and items from other questionnaires were subsequently added. Seventeen patients and 16 clinicians participated. HRQL concerns assessed by uveal melanoma-specific questionnaires were largely resolved by the time of metastasis. The Functional Assessment of Cancer Therapy - Immunotherapy Module (FACT-ICM) adequately captured most immunotherapy-related side effects during initial treatment cycles. However, most patients emphasised emotional impacts over physical ones, focussing on the existential threat posed by disease amidst uncertainty about treatment accessibility and effectiveness. Patients were also concerned with treatment burden, including time commitment, travel, need for hospitalisation, and expenses. The relative importance of HRQL issues varied over time and across treatment modalities, with no single questionnaire being sufficient. Pending further development and psychometric testing, clinical researchers may need to take a modular approach to measuring the HRQL impacts of mUM.

Exploring the measurement of health related quality of life and broader instruments: A dimensionality analysis.

BACKGROUND: Comprehensively measuring the outcomes of interventions and policy programmes impacting both health and broader areas of quality of life (QoL) is important for decision-making within and across sectors. Increasingly, broad QoL measures are being developed to capture outcomes beyond health-related quality of life (HRQoL). Jointly exploring the dimensionality of diverse instruments can improve our understanding about their evaluative space and how they conceptually build on each other. This study explored the measurement relationship between five broader QoL measures and the most widely used HRQoL measure, the EQ-5D. METHODS: Participants from the Dutch general population (n = 1002) completed six instruments (n = 126 items) in December of 2020. The measurement relationship was explored using qualitative and quantitative dimensionality assessment methods. This included a content analysis and exploratory factor analyses which were used to develop a confirmatory factor model of the broader QoL dimensions. Correlations between the identified dimensions and self-reported overall health and wellbeing were also explored. RESULTS: The final CFA model exhibited acceptable/good fit and described 12 QoL dimensions: 'psychological symptoms', 'social relations', 'physical functioning', 'emotional resilience', 'pain', 'cognition', 'financial needs', 'discrimination', 'outlook on life/growth', 'access to public services', 'living environment', and 'control over life'. All dimensions were positively correlated to self-reported health and wellbeing, but the magnitudes in associations varied considerably (e.g., 'pain' had the strongest correlation with overall health but a weak correlation with wellbeing). CONCLUSIONS: This study contributes to a broader understanding of QoL by exploring the dimensionality and relationships among various QoL measures. A number of the dimensions identified are HRQoL-focused, with others covering broader constructs. Our findings offer insights for the development of comprehensive instruments, or use of instrument suites that capture multidimensional aspects of QoL. Further research should explore the relevance and feasibility/appropriateness of measuring the identified dimensions in different settings and populations.

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.

Scoring the EQ-HWB-S: can we do it without value sets? A non-parametric item response theory analysis.

BACKGROUND: Only one pilot value set (UK) is currently available for the EQ Health and Wellbeing Instrument short version (EQ-HWB-S). As an alternative to preference-weighted scoring, we examined whether a level summary score (LSS) is appropriate for the EQ-HWB-S using Mokken scaling analyses. METHODS: Data from patients, carers and the general population collected during the developmental phase of the EQ-HWB-S in Australia, US and UK were used, noting 3 of 9 items have since undergone revision. EQ-HWB-S data fit was examined using R package Mokken scaling's monotone homogeneity model, utilizing the automated item selection procedure (AISP) as well as Loevinger's scaling coefficients for items and the scale (HS). Manifest monotonicity was assessed by examining whether the cumulative probability for responses at or above each response level did not decrease across the summary score. RESULTS: EQ-HWB-S data were available for 3340 respondents: US = 903, Australia = 514 and UK = 1923. Mean age was 50 ± 18 and 1841 (55%) were female. AISP placed all 9 items of the EQ-HWB-S on a single scale when the lower bound was set to < 0.448. Strong scalability (HS = 0.561) was found for the EQ-HWB-S as a single scale. Stronger scales were formed by separating the psychosocial items (n = 6, HS = 0.683) and physical sensation items (n = 3, HS = 0.713). No violations of monotonicity were found except for the items mobility and daily activities for the subgroups with long-term conditions and UK subjects, respectively. DISCUSSION: As EQ-HWB-S items formed a strong scale and subscales based on Mokken analysis, LSS is a promising weighting-free approach to scoring.

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.

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.

Eliciting a value set for the Swedish Capability-Adjusted Life Years instrument (CALY-SWE).

PURPOSE: Our aim was to elicit a value set for Capability-Adjusted Life Years Sweden (CALY-SWE); a capability-grounded quality of life instrument intended for use in economic evaluations of social interventions with broad consequences beyond health. METHODS: Building on methods commonly used in the quality-adjusted life years EQ-5D context, we collected time-trade off (TTO) and discrete choice experiment (DCE) data through an online survey from a general population sample of 1697 Swedish participants. We assessed data quality using a score based on the severity of inconsistencies. For generating the value set, we compared different model features, including hybrid modeling of DCE and TTO versus TTO data only, censoring of TTO answers, varying intercept, and accommodating for heteroskedasticity. We also assessed the models' DCE logit fidelity to measure agreement with potentially less-biased DCE data. To anchor the best capability state to 1 on the 0 to 1 scale, we included a multiplicative scaling factor. RESULTS: We excluded 20% of the TTO answers of participants with the largest inconsistencies to improve data quality. A hybrid model with an anchor scale and censoring was chosen to generate the value set; models with heteroskedasticity considerations or individually varying intercepts did not offer substantial improvement. The lowest capability weight was 0.114. Health, social relations, and finance and housing attributes contributed the largest capability gains, followed by occupation, security, and political and civil rights. CONCLUSION: We elicited a value set for CALY-SWE for use in economic evaluations of interventions with broad social consequences.

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.