Preference-based Instruments Research Articles

Chinese utility weights for the EORTC cancer-specific utility instrument QLU-C10D.

The aim of this study is to provide Chinese utility weights for the European Organization for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (EORTC QLU-C10D) which is a preference-based cancer-specific utility instrument derived from the EORTC QLQ-C30. We conducted an online survey of the general population in China, with quota sampling for age and gender. Each respondent was asked to complete a discrete choice experimental survey consisting of 16 randomly selected choice sets. The conditional logit model and mixed logit model were used to analyze respondents' preferences, and the goodness of fit of the model was tested. A total of 2003 respondents were included in the analysis. Utility decrements within dimensions were typically monotonic. Monotonic inconsistency issues in the Fatigue, Sleep, and Nausea dimensions were normalized by monotonicity correction. Physical functioning, Pain, and Role functioning were associated with the greatest utility weights, with the smallest decrements being in Bowel problems and Emotional functioning. The utility value for the worst health state was 0.083, i.e. slightly higher than being dead. This study provides the first China-specific set of value for the QLU-C10D based on societal preferences of the Chinese adult general population. The value set can be used as a cancer-specific scoring system for economic evaluations of new oncology therapies and technologies in China.

Impact of secondary health conditions on the quality of life and wellbeing of Canadians living with spinal cord injury: A comparison of preference-weighted index scores derived from generic standardized instruments

Context/objectives To compare the assessment of the impact of secondary health conditions (SHCs) on the quality of life and wellbeing of Canadians living with spinal cord injury (SCI) using four preference-based outcome measures. Design Secondary analysis of data from a cross-sectional, online survey. Setting Community. Participants Community-dwelling adults (n = 364) living with traumatic or non-traumatic spinal cord injury at least one year post-injury (70% at least 10 years post-injury). Outcome measures A modified version of the Spinal Cord Injury Secondary Conditions Scale (SCI-SCS); three health-related instruments (EQ-5D-5L, Health Utilities Index Mark 3 (HUI3), and the Assessment of Quality of Life 8-dimension questionnaire (AQoL-8D)) and a capability wellbeing instrument (ICEpop CAPability measure for Adults (ICECAP-A)). Results Across unadjusted and controlled analyses (i.e. controlling for associations between index scores and sociodemographic and impairment characteristics), trends were observed that identified lower levels of quality of life/wellbeing with higher problem ratings for each of the SHCs. Despite the trends, there was considerable variation in mean index scores across instruments, with HUI3 scores the lowest of the health-related instruments and ICECAP-A scores the highest overall. Respiratory problems, depression/mood problems, pressure sores, and autonomic dysreflexia were associated with the lowest levels of quality of life and wellbeing. Conclusions Higher problem ratings for SHCs are negatively associated with scores derived from preference-based quality of life and wellbeing instruments. Variation in index scores across instruments – including across the health-related instruments alone – highlights the critical importance of assessing the relative merits of preference-based instruments when using (or considering using) these instruments/estimates in comparative effectiveness research and economic evaluation.

A novel approach for health state valuation: Multiple bounded dichotomous choice compared to the traditional standard gamble

Objectiveto assess the feasibility of a new stated preference approach, the multiple bounded dichotomous choice (MBDC), designed to generate value sets for preference-based measurement of health-related quality of life. MethodsMBDC and standard gamble (SG) tasks were completed to derive SF-6Dv2 value sets from a sample of the general population in Quebec, Canada. Participants were randomized between the two approaches: 6 health states were evaluated in SG and 11 health states in MBDC. Several models were used to estimate data in each approach, and the preferred models were chosen by using mean absolute error (MAE), logical consistency of parameters, and significance levels. Results of MBDC were compared with SG in terms of acceptability (self-reported difficulty and quality levels in answering, and completion time), consistency (monotonicity of model coefficients), accuracy (standard errors), dimensions coefficient magnitude, correlation between the value sets estimated, and the range of estimated values. The intra-class correlation coefficient (ICC) was computed to assess value sets' consistency. ResultsOut of 655 individuals who completed MBDC tasks and 828 who completed SG tasks, a total of 585 participants for MBDC and 714 for SG tasks were included for analysis. The preferred models for both approaches were GLS Tobit. No significant difference was observed in self-reported difficulties and qualities in answers among approaches, but MBDC had less excluded participants and was less prone to report difficulties in answering. Additionally, completion time in the MBDC group was significantly lower (99.80 vs 68.12 s). Most standard errors in the MBDC were lower than those in SG, and the number of non-significant parameters was also lower. The range of utility values generated by MBDC tended to be wider (−0.372 to 1) than those generated by the SG (−0.137 to 1) and the number of worse-than-dead states in MBDC (0.91%) was higher than for SG (0.08%). The Pain dimension was identified as the most significant, while the Vitality dimension showed the lowest significant decrement. Both approaches exhibited a tendency to overestimate severe health state values and underestimate better health state values. The correlation and ICC between the two value sets were 0.937 and 0.983, respectively. ConclusionBased on empirical evidence, it can be inferred that the MBDC method is not only feasible but also holds the potential to generate meaningful and well-informed preference data from respondents. This approach can be used to derive a value set for preference-based instrument.

One and the same or different? An empirical comparison of aged care recipient and non-aged care recipient preferences for quality of aged care amongst older Australians

The Quality of Care Experience Aged Care Consumers (QCE-ACC) is a new preference-based instrument recently adopted by the Australian government nationally as a new quality indicator for aged care. This study employed a discrete choice experiment (DCE) approach to develop an aged care user-specific value set for the QCE-ACC instrument. This is crucial for establishing the relative importance of key QCE-ACC dimensions for informing quality assessment and economic evaluation in aged care. We further empirically compared the preferences of aged care recipients and non-aged care recipients amongst the older Australian population (65 years and above) for quality of care experience using the QCE-ACC. A total of 201 older people (age 74.2 ± 6.2; 59.7% female) receiving aged care services completed the DCE survey between August and September 2022. The comparison of relative importance indicated some divergence in the preferences between the aged care recipients and non-aged care recipients. Amongst aged care recipients, being treated with “Respect & Dignity” was the most important quality of care experience defining dimension, with “Health & Wellbeing” ranked second and “Skills & Training” (of staff) ranked third. However, within non-aged care recipients, “Skills Training” (of staff) was considered the most important quality of care dimension. Distinction in the QCE-ACC utility weights distributions and mean values were also observed, suggesting that aged care recipients may have different opinions about the quality of aged care compared to those who have not accessed aged care services. The findings shed light on the unique preferences of aged care recipients, indicating that aged care recipients and non-aged care recipients’ preferences for quality of aged care are not interchangeable. The value set developed in this study is specifically tailored for assessing the quality of aged care using the QCE-ACC instrument from the perspective of aged care users in Australia.

Mapping Kansas City cardiomyopathy, Seattle Angina, and minnesota living with heart failure to the MacNew-7D in patients with heart disease.

The Kansas City Cardiomyopathy Questionnaire (KCCQ), Seattle Angina Questionnaire (SAQ), and Minnesota Living with Heart Failure Questionnaire (MLHFQ) are widely used non-preference-based instruments that measure health-related quality of life (QOL) in people with heart disease. However, currently it is not possible to estimate quality-adjusted life-years (QALYs) for economic evaluation using these instruments as the summary scores produced are not preference-based. The MacNew-7D is a heart disease-specific preference-based instrument. This study provides different mapping algorithms for allocating utility scores to KCCQ, MLHFQ, and SAQ from MacNew-7D to calculate QALYs for economic evaluations. The study included 493 participants with heart failure or angina who completed the KCCQ, MLHFQ, SAQ, and MacNew-7D questionnaires. Regression techniques, namely, Gamma Generalized Linear Model (GLM), Bayesian GLM, Linear regression with stepwise selection and Random Forest were used to develop direct mapping algorithms. Cross-validation was employed due to the absence of an external validation dataset. The study followed the Mapping onto Preference-based measures reporting Standards checklist. The best models to predict MacNew-7D utility scores were determined using KCCQ, MLHFQ, and SAQ item and domain scores. Random Forest performed well for item scores for all questionnaires and domain score for KCCQ, while Bayesian GLM and Linear Regression were best for MLHFQ and SAQ domain scores. However, models tended to over-predict severe health states. The three cardiac-specific non-preference-based QOL instruments can be mapped onto MacNew-7D utilities with good predictive accuracy using both direct response mapping techniques. The reported mapping algorithms may facilitate estimation of health utility for economic evaluations that have used these QOL instruments.

Reliability of the quality of life-aged care consumers (QOL-ACC) and EQ-5D-5L among older people using aged care services at home

PurposeThe Quality of Life-Aged Care Consumers (QOL-ACC), a valid preference-based instrument, has been rolled out in Australia as part of the National Quality Indicator (QI) program since April 2023 to monitor and benchmark the quality of life of aged care recipients. As the QOL-ACC is being used to collect quality of life data longitudinally as one of the key aged care QI indicators, it is imperative to establish the reliability of the QOL-ACC in aged care settings. Therefore, we aimed to assess the reliability of the QOL-ACC and compare its performance with the EQ-5D-5L.MethodsHome care recipients completed a survey including the QOL-ACC, EQ-5D-5L and two global items for health and quality of life at baseline (T1) and 2 weeks later (T2). Using T1 and T2 data, the Gwet’s AC2 and intra-class correlation coefficient (ICC) were estimated for the dimension levels and overall scores agreements respectively. The standard error of measurement (SEM) and the smallest detectable change (SDC) were also calculated. Sensitivity analyses were conducted for respondents who did not change their response to global item of quality of life and health between T1 and T2.ResultsOf the 83 respondents who completed T1 and T2 surveys, 78 respondents (mean ± SD age, 73.6 ± 5.3 years; 56.4% females) reported either no or one level change in their health and/or quality of life between T1 and T2. Gwet’s AC2 ranged from 0.46 to 0.63 for the QOL-ACC dimensions which were comparable to the EQ-5D-5L dimensions (Gwet’s AC2 ranged from 0.52 to 0.77). The ICC for the QOL-ACC (0.85; 95% CI, 0.77–0.90) was comparable to the EQ-5D-5L (0.83; 95% CI, 0.74–0.88). The SEM for the QOL-ACC (0.08) was slightly smaller than for the EQ-5D-5L (0.11). The SDC for the QOL-ACC and the EQ-5D-5L for individual subjects were 0.22 and 0.30 respectively. Sensitivity analyses stratified by quality of life and health status confirmed the base case results.ConclusionsThe QOL-ACC demonstrated a good test-retest reliability similar to the EQ-5D-5L, supporting its repeated use in aged care settings. Further studies will provide evidence of responsiveness of the QOL-ACC to aged care-specific interventions in aged care settings.

Further Evidence of Feasibility, Validity, and Reliability of Quality of Life-Aged Care Consumers: Evidence From Home-Based Care Settings

ObjectivesThe Quality of Life-Aged Care Consumers (QOL-ACC) is an aged-care-specific preference-based instrument currently being rolled out in residential care across Australia as part of the aged care Quality Indicator program. This study aimed to provide a comprehensive assessment of the feasibility, reliability, and construct validity of the QOL-ACC in a large national sample of older adults receiving aged care services at home. MethodsOlder adults receiving in-home aged care services completed a survey including the QOL-ACC, Quality of Care Experience-ACC, adult social care outcome tool, EQ-5D-5L, and 2 global single item measures of health and quality of life. Feasibility was assessed by missing responses (≤5%) and ceiling/floor effects (≤15%). Construct validity was assessed by exploring the relationship between the QOL-ACC and other instruments (convergent validity) and its ability to discriminate varying levels of self-rated health and quality of life (known-group validity). ResultsA total of 802 respondents (mean age, 74.5 ± 6.3 years; 56.0% females) completed the survey. The QOL-ACC had no missing responses, no floor effects, and very low ceiling effect (3.5%) and demonstrated moderate correlation with adult social care outcome tool (r = 0.59, P < .001), EQ-5D-5L (r = 0.65, P < .001), EQ-VAS (r = 0.53, P < .001), and a lower correlation with the QCE-ACC (r = 0.41, P < .001). Respondents with poor self-rated health and quality of life had significantly lower preference-weighted scores on the QOL-ACC. ConclusionsThe QOL-ACC demonstrated adequate feasibility, reliability, and construct validity in a large population of older people accessing government-subsidized aged care services at home. Further studies will explore the responsiveness of the QOL-ACC to aged-care-specific interventions both in home and residential aged care settings.

Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L

BackgroundThe progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, few health state utility data exist; published utilities tend to be derived from small samples for a limited number of health states and are often based on caregiver-reported patient health status. This study estimated utility values for varied clinical and functional health states in DMD, based on patient-reported health status.MethodsIndividuals with DMD in the US aged 12–40 years completed the EQ-5D (5-level) and Health Utilities Index (HUI) preference-based instruments. Based on responses to a clinical questionnaire, participants self-classified into functional health states according to level of lower and upper limb function, use of respiratory support, and presence of cardiomyopathy. Mean [standard deviation (SD)] utility and EQ-5D visual analogue scale (VAS) scores were estimated according to health state; and median (interquartile range) attribute levels calculated to understand which domains of health are most severely affected in DMD.ResultsOf 63 males with DMD, mean (SD) age was 19.8 (6.1) years and 11 (17.5%) were ambulatory. Mean (SD) utility values were 0.92 (0.08; HUI2), 0.84 (0.20; HUI3), and 0.84 (0.13; EQ-5D) for ambulatory patients without cardiomyopathy (n = 10). For non-ambulatory patients with moderately impaired upper limb function, night and daytime ventilation without cardiomyopathy, mean (SD) utilities were 0.49 (0.07) for the HUI2, 0.16 (0.15) for the HUI3 and 025 (0.14) for the EQ-5D. Mean (SD) VAS scores for the same health states were 91 (9) and 83 (21), respectively. In addition to impairments in mobility/ambulation, and self-care, attributes like usual activities and pain also showed notable effects of DMD.ConclusionsIn DMD, although a relationship between disease progression and HRQoL is observed, there is large variability in utility within functional health states, and across instruments. Utility values for less severe non-ambulatory health states described by level of upper limb function are novel. These utility values, derived based on direct patient feedback rather than from caregiver report, are relevant to individuals of varying functional statuses and augment scarce DMD-specific utility data.

UnitedStates Value Set for the Functional Assessment of Cancer Therapy-General Eight Dimensions (FACT-8D), a Cancer-Specific Preference-Based Quality of Life Instrument.

To develop a value set reflecting the United States (US) general population's preferences for health states described by the Functional Assessment of Cancer Therapy (FACT) eight-dimensions preference-based multi-attribute utility instrument (FACT-8D), derived from the FACT-General cancer-specific health-related quality-of-life (HRQL) questionnaire. A US online panel was quota-sampled to achieve a general population sample representative by sex, age (≥ 18 years), race and ethnicity. A discrete choice experiment (DCE) was used to value health states. The valuation task involved choosing between pairs of health states (choice-sets) described by varying levels of the FACT-8D HRQL dimensions and survival (life-years). The DCE included 100 choice-sets; each respondent was randomly allocated 16 choice-sets. Data were analysed using conditional logit regression parameterized to fit the quality-adjusted life-year framework, weighted for sociodemographic variables that were non-representative of the US general population. Preference weights were calculated as the ratio of HRQL-level coefficients to the survival coefficient. 2562 panel members opted in, 2462 (96%) completed at least one choice-set and 2357 (92%) completed 16 choice-sets. Pain and nausea were associated with the largest utility weights, work and sleep had more moderate utility weights, and sadness, worry and support had the smallest utility weights. Within dimensions, more severe HRQL levels were generally associated with larger weights. A preference-weighting algorithm to estimate US utilities from responses to the FACT-General questionnaire was generated. The worst health state's value was -0.33. This value set provides US population utilities for health states defined by the FACT-8D for use in evaluating oncology treatments.

PCR198 Should We be Mapping from Sleep-Specific to Generic Preference-Based Quality-of-Life Instruments? Unravelling the Debate with a Multi-Instrument Mapping Study of 6 Sleep-Specific and 4 Preference-Based Instruments

PCR198 Should We be Mapping from Sleep-Specific to Generic Preference-Based Quality-of-Life Instruments? Unravelling the Debate with a Multi-Instrument Mapping Study of 6 Sleep-Specific and 4 Preference-Based Instruments

Measurement of Health-Related Quality of Life in Individuals With Rare Diseases in China: Nation-Wide Online Survey.

Rare diseases (RDs) affect millions of people worldwide, and these diseases can severely impact the health-related quality of life (HRQoL) of those affected. Despite this, there is a lack of research measuring HRQoL using the EQ-5D-5L, which is one of the most widely used generic preference-based instruments to measure HRQoL in populations living with RDs. This study aimed to measure HRQoL using the EQ-5D-5L in a large number of patients with various types of RDs in China, and to examine the relationship between respondents' socioeconomic characteristics and preference-based health utility scores. The data used in this study were obtained from a web-based survey conducted in China. The survey aimed to explore and understand the health and socioeconomic status of patients with RDs in China. We recruited registered and eligible members, including patients or their primary caregivers, from 33 RD patient associations to complete the questionnaires via their internal social networks. HRQoL was measured using the EQ-5D-5L utility score, which was calculated based on an established Chinese value set. Utility scores have been presented based on demographics and disease-related information. Univariate linear regression analysis was used to assess the differences in the EQ-5D-5L utility scores between subgroups. A total of 12,502 respondents completed the questionnaire and provided valid responses, including 6919 self-completed respondents and 5583 proxy-completed respondents. Data from 10,102 participants over the age of 12 years were elicited for analysis. Among patients with RDs, 65.3% (6599/10,102), 47.5% (4799/10,102), 47.0% (4746/10,102), 24.8% (2506/10,102), and 18.4% (1855/10,102) reported no problems for "self-care," "usual activities," "mobility," "pain/discomfort," and "anxiety/depression," respectively. A full health state was reported by 6.0% (413/6902) and 9.2% (295/3200) of self- and proxy-completed patients, respectively. Among self-completed patients, 69.9% (4826/6902) and 50.4% (3478/6902) reported no problems for "self-care" and "usual activities," respectively, whereas only 17.7% (1223/6902) reported problems for "anxiety/depression." Proxy-completed respondents showed a higher proportion of reporting extreme problems than self-completed respondents in all 5 dimensions. The mean utility scores reported by self- and proxy-completed respondents were 0.691 and 0.590, respectively. Different types of caregivers reported different utility scores, and among them, proxy-completed (mother) respondents reported the highest mean utility score. The establishment of a normative profile for RD patients can facilitate patients' adaptation and assess the effectiveness of interventions to improve the HRQoL and well-being of this population. Differences between self- and proxy-completed HRQoL assessed by the EQ-5D-5L have been identified in this study. This finding highlights the importance of incorporating perspectives from both patients and their proxies in clinical practice. Further development of the patient cohort is necessary to assess long-term changes in HRQoL in the RD population.

Health-related Quality of Life in 10 years Long-term Survivors of Chronic Kidney Disease: A From-J Study

ObjectiveThe Chronic Kidney Disease (CKD) practice facilitation program in the Frontier of Renal Outcome Modifications in Japan (FROM-J) study reduced cardiovascular disease (CVD) events in patients with CKD. 10-year long-term survivors with CKD lived with serious complications, including end-stage kidney disease (ESKD) and CVD. This study aimed to measure health-related quality of life (HRQOL) in 10-year long-term CKD survivors and examine the predictors and determinants of clinical indices for measured QOL scores. MethodsThe EQ-5D-5L, a generic preference-based instrument, was administered to 1,473 CKD survivors enrolled in the FROM-J study. The 10th-year data collection was performed by either primary care physicians or participants who filled out questionnaires from October 2018 to March 31, 2019. ResultsThe response rate was 38.2% (423/1,473). The mean QOL score was 0.893 (95% confidence interval (CI), 0.880–0.906), and the median QOL score was 1.000 (interquartile range (IQR), 0.806–1.000). The mean QOL score in participants with renal replacement therapy was 0.824 (95% CI, 0.767–0.881), and the median was 0.828 (IQR, 0.755–1.000). The mean QOL score in participants with CVD was 0.877 (95% CI, 0.811–0.943), and the median was 1.000 (IQR, 0.723–1.000). The mean QOL score in participants with 50% decline in eGFR was 0.893 (95% CI, 0.860–0.926), and the median was 0.889 (IQR, 0.825–1.000). The decrease in QOL scores with baseline CKD stages was significant according to the Jonckheere–Terpstra test for trend (P = 0.002). Baseline age, systolic blood pressure, and history of hyperuricemia were significant predictors of 10th-year QOL scores. ConclusionWe suggest that CKD complications negatively affect the QOL scores in 10-year long-term survivors with CKD. CKD guideline-based practices, prevention of ESKD/CVD and management of hypertension, diabetes and hyperuricemia, might contribute to future HRQOL in patients with CKD.

Comparison of the Early Childhood Oral Health Impact Scale (ECOHIS-4D) and Child Health Utility Index (CHU-9D) in children with oral diseases.

Accurate assessment of child oral health is important for guiding economic evaluations and informing healthcare decision-making. Early Childhood Oral Health Impact Scale (ECOHIS-4D) is a preference-based instrument that measures the oral health-related quality of life of young children. The aim of this study was to compare the utility scores of ECOHIS-4D and Child Health Utility Index (CHU-9D), against an oral health indicator to evaluate which utility score corresponds better with the oral health indicator. The ECOHIS-4D and CHU-9D were applied to 314 parent/child dyads from preschools in a primary healthcare setting in Perth, Western Australia. Four parameters were used to assess which instrument corresponds better with the oral health indicator (decayed, missing and filled teeth score-dmft score): (i) discrimination, the ability to discriminate between different clinical severity groups, (ii) external responsiveness, how much the utility values relate to the changes in dmft scores, (iii) correlation, the association between the two instruments and the related dimensions and (iv) differences in the utility values across the two instruments. Most participants (81%) were 2-6 years old, and nearly 50% had a dmft score <3. ECOHIS-4D demonstrated a superior ability to differentiate between dmft severity groups and respond to changes in dmft scores. A significant weak correlation was observed between dmft and ECOHIS-4D (-0.26, 95%, CI -0.36 to -0.15) compared to a non-significant very poor correlation between dmft and CHU-9D (0.01, 95% CI -0.12 to 0.10). The utility scores of the two instruments had relatively good agreement towards good health and weak agreement towards poor health. ECOHIS-4D, the oral health-specific instrument, is more sensitive in assessing children's oral health-related quality of life than the generic CHU-9D. Thus, ECOHIS-4D is more appropriate for utility estimates in economic evaluations of oral health-related interventions and resource allocation decision-making.

Mapping the Schizophrenia Quality of Life Scale to EQ-5D, HUI3 and SF-6D utility scores in patients with schizophrenia

Objectives The current study aimed to map the disease-specific Schizophrenia Quality of Life Scale (SQLS) onto the three- and five-level EuroQol five-dimension (EQ-5D-3L and EQ-5D-5L), Healthy Utility Index Mark 3 (HUI3) and Short Form six-dimensional (SF-6D) preference-based instruments to inform future cost-utility analyses for treatment in patients with schizophrenia. Methods Data from 251 outpatients with schizophrenia spectrum disorders was included for analysis. Ordinary least square (OLS), Tobit and beta regression mixture models (betamix) were employed to estimate the utility scores. Model selection of 66 regression models (regression methods x six model specifications) was determined by goodness of fit and predictive indices. Distribution of the original data to the distributions of the data generated using the preferred estimated models were then compared. Results EQ-5D-3L and EQ-5D-5L were best predicted by the OLS model, including SQLS domain scores, domain scores-squared, age, and gender as explanatory predictors. The models produced the best performance index and resemble more closely to the observed EQ-5D data. HUI3 and SF-6D were best predicted by the OLS and Tobit model respectively. Conclusion The current study developed mapping models for converting SQLS scores into generic utility scores, which can be used in economics evaluation in schizophrenia patients.

How do older adults receiving aged care services understand and respond to the EQ-5D-5L? A think-aloud study in residential care

PurposeThe EQ-5D-5L is a preference-based instrument for measuring and valuing health-related quality of life (HRQoL). The EQ-5D-5L has been used extensively in economic evaluation, including in aged care. However, older adults’ understanding of the EQ-5D-5L has not been comprehensively investigated to date. This research aimed to assess older adults’ understanding of the EQ-5D-5L using a think-aloud protocol with two cognition groups: no cognitive impairment and mild/moderate cognitive impairment.MethodsParticipants’ cognition was assessed using the Standardised Mini-Mental State Examination (SMMSE). Face-to face interviews were conducted with concurrent and retrospective think-aloud encouraged through verbal probing. Audio recordings were transcribed, and qualitative analysis, informed by the Tourangeau four-stage Response Model (comprehension, retrieval, decision process, response process) was conducted in NVivo.ResultsIn total, 46 older adults (age 65 +) were recruited from 10 residential care facilities across South Australia (n = 25 no cognitive impairment, n = 21 mild/moderate cognitive impairment). Comprehension, retrieval, judgement and response mapping issues were common across all cognition levels and EQ-5D-5L dimensions. The two dimensions resulting in the most response issues were usual activities and personal care.ConclusionOlder adults may bring a different understanding to the EQ-5D-5L descriptive system than that expected given testing with general population samples. Dimension descriptors that are more relevant to this population may facilitate responses that better align with the underlying EQ-5D-5L concept model.

Using Rasch Analysis to Assess and Improve the Measurement Properties of a Questionnaire With Few Items: The York Binaural Hearing-Related Quality of Life (YBHRQL) Questionnaire.

The York Binaural Hearing-Related Quality of Life questionnaire is a condition-specific preference-based instrument sensitive to advantages of binaural over monaural hearing. Respondents use 5-point scales to report the difficulty experienced with three dimensions of listening that are easier or more successful when hearing is binaural: understanding speech in spatially separated noise, localizing sources of sound in azimuth, and the associated effort and fatigue. Previously, a preference value was estimated for each combination of dimension and level so that a value of binaural utility could be assigned to a respondent to inform analyses of cost effectiveness. The present objective was to determine whether the questionnaire conforms with the Rasch model sufficiently well for estimates of the binaural abilities of respondents to be obtained on an interval scale to inform parametric analyses of clinical effectiveness. Data were obtained from unilateral cochlear implantees (N = 418; 209 ≤62 years; 209 ≥63 years) and members of the public (N = 325; 207 ≤62 years; 118 ≥63 years). A subset of implantees (N = 118) responded at test and retest. Responses were fitted to the partial credit model using the Extended Rasch Modeling package. Conformity with the model was evaluated in six ways: the ordering of response categories ( Monotonicity ) was assessed with plots of response probability against ability; differential item functioning ( DIF ) was assessed by analyses of variance of standardized response residuals; alignment of participants' abilities with item difficulties ( Targeting ) was assessed with person-item maps; fit to the model ( Fit ) was assessed by comparing the means and variabilities of observed and expected responses, and by comparing observed values with analyses of simulated datasets; the hypothesis that item difficulties and participants' abilities were measured on a single underlying scale ( Unidimensionality ) was assessed with principal components analyses of standardized response residuals. Values of fit statistics were toward the lower end of the acceptable range. Comparisons with analyses of simulated datasets showed that low values were primarily the result of the structural limitation of including only three items. Modal values of the probabilities of response categories were ordered monotonically, but some response thresholds were disordered because of under-use of one category. Pooling categories to correct disordered thresholds resulted in estimates of ability that were less discriminatory of differences within and between groups, and showed less reproducibility between test and retest, than did the original estimates. Neither source-related DIF nor gender-related DIF arose. Uniform age-related DIF arose for the speech-in-noise item and could be managed by resolving the item. The resulting estimates of ability and difficulty were well targeted and unidimensional. The York Binaural Hearing-Related Quality of Life questionnaire, with three items each with five response categories, conforms with the Rasch model sufficiently well to yield practically useful measures of the abilities of participants. The trait measured by the questionnaire aligns with the ability to benefit from binaural hearing. More discriminatory measurement of this ability would be achieved with more items. Nonetheless, the questionnaire possesses the virtue that responses to the same three questions can be scored in different ways to inform parametric analyses of both cost-effectiveness and clinical effectiveness.

Mapping the World Health Organization Disability Assessment Schedule (WHODAS 2.0) onto SF-6D Using Swedish General Population Data.

Mapping algorithms can be used for estimating quality-adjusted life years (QALYs) when studies apply non-preference-based instruments. In this study, we estimate a regression-based algorithm for mapping between the World Health Organization Disability Assessment Schedule (WHODAS 2.0) and the preference-based instrument SF-6D to obtain preference estimates usable in health economic evaluations. This was done separately for the working and non-working populations, as WHODAS 2.0discriminates between these groups when estimating scores. Using a dataset including 2258 participants from the general Swedish population, we estimated the statistical relationship between SF-6D and WHODAS 2.0. We applied three regression methods, i.e., ordinary least squares (OLS), generalized linear models (GLM), and Tobit, in mapping onto SF-6D from WHODAS2.0 at the overall-score and domain levels. Root mean squared error (RMSE) and mean absolute error (MAE) were used for validation of the models; R2 was used to assess model fit. The best-performing models for both the working and non-working populations were GLM models with RMSE ranging from 0.084 to 0.088, MAE ranging from 0.068 to 0.071, and R2 ranging from 0.503 to 0.608. When mapping from the WHODAS2.0 overall score, the preferred model also included sex for both the working and non-working populations. When mapping from the WHODAS2.0 domain level, the preferred model for the working population included the domains mobility, household activities, work/study activities, and sex. For the non-working population, the domain-level model included the domains mobility, household activities, participation, and education. It is possible to apply the derived mapping algorithms for health economic evaluations in studies using WHODAS 2.0. As conceptual overlap is incomplete, we recommend using the domain-based algorithms over the overall score. Different algorithms must be applied depending on whether the population is working or non-working, due to the characteristics of WHODAS 2.0.

China Health Related Outcomes Measures (CHROME): development of a descriptive system to support cardiovascular disease specific preference-based measure for the Chinese population.

Preference-based measures have been increasingly recommended to measure health outcomes for economic evaluation. However, none of existing cardiovascular disease (CVD)-specific health-related quality of life (HRQoL) instruments are preference-based. This study aimed to develop the descriptive system of preference-based HRQoL instrument for Chinese patients with CVDs under the Initiative of China Health Related Outcomes Measures (CHROME). Qualitative face-to-face interviews were conducted with Chinese patients with CVDs. Content analysis was employed to generate candidate items for the instrument. Then expert consultation and cognitive debriefing interviews were conducted to guide further selection and revision of the items. We interviewed 127 CVD patients with 67.7% being male and 63.8% living in the urban area. A hierarchical code book comprised of four themes, 20 categories, 62 sub-categories, and 207 codes, was developed. Candidate items were selected based on the criteria set by the Consensus-based Standards for the selection of health Measurement Instruments (COSMIN) methodology and ISPOR PRO guidance. An online survey and meeting with an expert advisory panel (n = 15) followed by cognitive debriefing interviews with 20 patients and 13 physicians were conducted to further select and revise the candidate items. The descriptive system of CHROME-CVD consists of 14 items, namely frequency and severity of chest pain, chest tightness, palpitation, shortness of breath, dizziness, fatigue, appetite, sleeping, mobility, daily activities, depression, worry, and social relationship. Four or five level responses were selected based on cognitive debriefing results to each item. The current study developed the descriptive system (items and response options) of CHROME-CVD, the future CVD-specific preference-based HRQoL instrument for Chinese CVD patients.

Derivation of a UK preference-based value set for the Short Warwick-Edinburgh Mental Well-being Scale (SWEMWBS) to allow estimation of Mental Well-being Adjusted Life Years (MWALYs)

BackgroundThe Mental Well-being Adjusted Life Year (MWALY) is an alternative outcome measure to the quality-adjusted life year (QALY) in economic evaluations of interventions aimed at improving mental well-being. However, there is a lack of preference-based mental well-being instruments for capturing population mental well-being preferences. ObjectivesTo derive a UK preference-based value set for the Short Warwick-Edinburgh Mental Well-being Scale (SWEMWBS). Methods225 participants that were interviewed between December 2020 and August 2021 completed 10 composite time trade-off (C-TTO) and 10 discrete choice experiment (DCE) interviewer-administered exercises. Heteroskedastic Tobit and conditional logit models were used to model C-TTO and DCE responses respectively. The DCE utility values were rescaled to a C-TTO comparable scale through anchoring and mapping. An inverse variance weighting hybrid model (IVWHM) was used to derive weighted-average coefficients from the modelled C-TTO and DCE coefficients. Model performance was assessed using statistical diagnostics. ResultsThe valuation responses confirmed the feasibility and face validity of the C-TTO and DCE techniques. Apart from the main effects models, statistically significant associations were estimated between the predicted C-TTO value and participants’ SWEMWBS scores, gender, ethnicities, education levels, and the interaction terms between age and useful feeling. The IVWHM was the most optimal model with the fewest logically inconsistent coefficients and the lowest pooled standard errors. The utility values generated by the rescaled DCE models and the IVWHM were generally higher than those of the C-TTO model. The predictive ability of the two DCE rescaling methods was similar according to the mean absolute deviation and root mean square deviation statistics. ConclusionsThis study has produced the first preference-based value set for a measure of mental well-being. The IVWHM provided a desirable blend of both C-TTO and DCE models. The value set derived by this hybrid approach can be used for cost-utility analyses of mental well-being interventions.

Sensitivity to change of generic preference-based instruments (EQ-5D-3L, EQ-5D-5L, and HUI3) in the context of treatment for people with prescription-type opioid use disorder in Canada

PurposeUsing data from a randomized controlled trial for treatment of prescription-type opioid use disorder in Canada, this study examines sensitivity to change in three preference-based instruments [EQ-5D-3L, EQ-5D-5L, and the Health Utilities Index Mark 3 (HUI3)] and explores an oft-overlooked consideration when working with contemporaneous responses for similar questions—data quality.MethodsAnalyses focused on the relative abilities of three instruments to capture change in health status. Distributional methods were used to categorize individuals as ‘improved’ or ‘not improved’ for eight anchors (seven clinical, one generic). Sensitivity to change was assessed using area under the ROC (receiver operating characteristics) curve (AUC) analysis and comparisons of mean change scores for three time periods. A ‘strict’ data quality criteria, defined a priori, was applied. Analyses were replicated using ‘soft’ and ‘no’ criteria.ResultsData from 160 individuals were used in the analysis; 30% had at least one data quality violation at baseline. Despite mean index scores being significantly lower for the HUI3 compared with EQ-5D instruments at each time point, the magnitudes of change scores were similar. No instrument demonstrated superior sensitivity to change. While six of the 10 highest AUC estimates were for the HUI3, ‘moderate’ classifications of discriminative ability were identified in 12 (of 22) analyses for each EQ-5D instrument, compared with eight for the HUI3.ConclusionNegligible differences were observed between the EQ-5D-3L, EQ-5D-5L, and HUI3 regarding the ability to measure change. The prevalence of data quality violations—which differed by ethnicity—requires further investigation.