Development of a new health-related quality of life measure for people with diabetes who experience hypoglycaemia: the Hypo-RESOLVE QoL.

AIMS/HYPOTHESIS: Valid and reliable patient-reported outcome measures are vital for assessing disease impact, responsiveness to healthcare and the cost-effectiveness of interventions. A recent review has questioned the ability of existing measures to assess hypoglycaemia-related impacts on health-related quality of life for people with diabetes. This mixed-methods project was designed to produce a novel health-related quality of life patient-reported outcome measure in hypoglycaemia: the Hypo-RESOLVE QoL. METHODS: Three studies were conducted with people with diabetes who experience hypoglycaemia. In Stage 1, a comprehensive health-related quality of life framework for hypoglycaemia was elicited from semi-structured interviews (N=31). In Stage 2, the content validity and acceptability of draft measure content were tested via three waves of cognitive debriefing interviews (N=70 people with diabetes; N=14 clinicians). In Stage 3, revised measure content was administered alongside existing generic and diabetes-related measures in a large cross-sectional observational survey to assess psychometric performance (N=1246). The final measure was developed using multiple evidence sources, incorporating stakeholder engagement. RESULTS: A novel conceptual model of hypoglycaemia-related health-related quality of life was generated, featuring 19 themes, organised by physical, social and psychological aspects. From a draft version of 76 items, a final 14-item measure was produced with satisfactory structural (χ2=472.27, df=74, p<0.001; comparative fit index =0.943; root mean square error of approximation =0.069) and convergent validity with related constructs (r=0.46-0.59), internal consistency (α=0.91) and test-retest reliability (intraclass correlation coefficient =0.87). CONCLUSIONS/INTERPRETATION: The Hypo-RESOLVE QoL is a rigorously developed patient-reported outcome measure assessing the health-related quality of life impacts of hypoglycaemia. The Hypo-RESOLVE QoL has demonstrable validity and reliability and has value for use in clinical decision-making and as a clinical trial endpoint. DATA AVAILABILITY: All data generated or analysed during this study are included in the published article and its online supplementary files ( https://doi.org/10.15131/shef. DATA: 23295284.v2 ).

Development and Valuation of a Preference-Weighted Measure in Age-Related Macular Degeneration From the Vision Impairment in Low Luminance Questionnaire: A MACUSTAR Report.

OBJECTIVES: This study generates VILL-UI (Vision Impairment in Low Luminance - Utility Index), a preference-weighted measure (PWM) derived from the VILL-33 measure for use in patients with age-related macular degeneration (AMD) and valued to generate United Kingdom and German preference weights. METHODS: A PWM consists of a classification system to describe health and utility values for every state described by the classification. The classification was derived using existing data collected as part of the MACUSTAR study, a low-interventional study on AMD, conducted at 20 clinical sites across Europe. Items were selected using psychometric and Rasch analyses, published criteria around PWM suitability, alongside instrument developer views and concept elicitation work that informed VILL-33 development. An online discrete choice experiment (DCE) with duration of the health state was conducted with the United Kingdom and German public. Responses were modeled to generate utility values for all possible health states. RESULTS: The classification system has 5 items across the 3 domains of VILL-33: reading and accessing information, mobility and safety, and emotional well-being. The DCE samples (United Kingdom: n = 1004, Germany: n = 1008) are broadly representative and demonstrate good understanding of the tasks. The final DCE analyses produce logically consistent and significant coefficients. CONCLUSIONS: This study enables responses to VILL-33 to be directly used to inform economic evaluation in AMD. The elicitation of preferences from both United Kingdom and Germany enables greater application of VILL-UI for economic evaluation throughout Europe. VILL-UI fills a gap in AMD in which generic preference-weighted measures typically lack sensitivity.

Producing a preference-based quality of LIFE measure to quantify the impact of HYPOGLYCAEMIA on people living with diabetes: A mixed-methods research protocol.

BACKGROUND: Assessment of patient-reported outcome measures (PROMs), including quality of life (QoL), is essential in diabetes research and care. However, a recent review concluded that current hypoglycaemia-specific PROMs have limited evidence of validity, reliability and responsiveness for assessing the impact of hypoglycaemia on QoL in people living with diabetes. None of the PROMs identified could be used directly to inform the cost-effectiveness of treatments and interventions. There is a need for a new hypoglycaemia-specific QoL PROM, which can be used directly to inform economic evaluations. AIMS: This project has three aims: (a) To develop draft PROM content for measuring the impact of hypoglycaemia on QoL in adults with diabetes. (b) To refine the draft content using cognitive debriefing interviews and psychometrics. This will result in a condition-specific PROM that can be used to quantify the impact of hypoglycaemia upon QoL. (c) To generate a preference-based measure (PBM) that will enable utility values to be calculated for economic evaluation. METHODS: A mixed-methods, three-stage design is used: (a) Qualitative interviews will inform the draft PROM content. (b) Cognitive debriefing interview data will be used to refine the draft PROM content. The PROM will be administered in a large-scale survey to enable psychometric validation. Final item selection for the PROM will be informed by psychometric performance, translatability assessment and input from stakeholder groups. (c) A classification system will be generated, comprising a reduced number of items from the PROM. A valuation survey will be conducted to derive a value set for the PBM.

Valuing the EQ Health and Wellbeing Short Using Time Trade-Off and a Discrete Choice Experiment: A Feasibility Study.

OBJECTIVES: The EQ Health and Wellbeing Short (EQ-HWB-S) is a new generic measure that covers health and wellbeing developed for use in economic evaluation in health and social care. The aim was to test the feasibility of using composite time trade-off (cTTO) and a discrete choice experiment (DCE) based on an international protocol to derive utilities for the EQ-HWB-S and to generate a pilot value set. METHODS: A representative UK general population was recruited. Online videoconference interviews were undertaken where cTTO and DCE tasks were administered using EuroQol Portable Valuation Technology. Quality control (QC) was used to assess interviewers' performance. Data were modeled using Tobit, probit, and hybrid models. Feasibility was assessed based on the distribution of data, participants, and reports of understanding from the interviewer, QC and modeling results. RESULTS: cTTO and DCE data were available for 520 participants. Demographic characteristics were broadly representative of the UK general population. Interviewers met QC requirements. cTTO values ranged between -1 to 1 with increasing disutility associated with more severe states. Participants understood the tasks and the EQ-HWB-S states; and the interviewers reported high levels of understanding and engagement. The hybrid Tobit heteroscedastic model was selected for the pilot value set with values ranging from -0.384 to 1. Pain, mobility, daily activities, and sad/depressed had the largest disutilities, followed by loneliness, anxiety, exhaustion, control, and cognition in the selected model. CONCLUSIONS: EQ-HWB-S can be valued using cTTO and DCE. Further methodological work is recommended to develop a valuation protocol specific to the EQ-HWB-S.

UK Valuation of EQ-5D-5L, a Generic Measure of Health-Related Quality of Life: A Study Protocol.

OBJECTIVES: A high-quality and widely accepted UK EQ-5D-5L value set is urgently required to enable the latest version of EQ-5D scored using recent UK public preferences to inform policy including health technology assessments submitted to the National Institute for Health and Care Excellence. This article outlines the study protocol for the generation of a new EQ-5D-5L UK value set. METHODS: Twelve hundred interviews will be undertaken using the composite time trade-off elicitation technique for 102 health states (86 from the international EQ-5D-5L valuation protocol, plus 16 with best predictive performance in an extended design used in the Native American EQ-5D-5L valuation). The sample will be UK adults (age ≥18 years) proportionately representative across England, Wales, Scotland, and Northern Ireland, representative for age, sex, ethnicity, and socioeconomic group, with inclusion of participants with/without health problems. Participants will choose to be interviewed via videoconference (by Zoom) or in-person in a central venue. Data quality will be rigorously assessed. RESULTS: The value set will be generated using tobit random effects and heteroscedastic tobit models (with censoring at -1) using all data, excluding time trade-off values highlighted by participants as ones they would reconsider and data from interviewers failing protocol compliance. Quality and acceptance will be achieved by public involvement, regular Steering Group meetings, independent assessment of data quality at 4 time points, and final endorsement of data and analyses. CONCLUSION: This study will produce a UK value set for the EQ-5D-5L for use in prospective and retrospective data sets containing EQ-5D-5L data.

Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised.

OBJECTIVES: Cystic fibrosis (CF) limits survival and negatively affects health-related quality of life (HRQOL). Cost-effectiveness analysis (CEA) may be used to make reimbursement decisions for new CF treatments; nevertheless, generic utility measures used in CEA, such as EQ-5D, are insensitive to meaningful changes in lung function and HRQOL in CF. Here we develop a new, CF disease-specific, preference-based utility measure based on the adolescent/adult version of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely used, CF-specific, patient-reported measure of HRQOL. METHODS: Blinded CFQ-R data from 4 clinical trials (NCT02347657, NCT02392234, NCT01807923, and NCT01807949) were used to identify discriminating items for a classification system using psychometric (eg, factor and Rasch) analyses. Thirty-two health states were selected for a time trade-off (TTO) exercise with a representative sample of the UK general population. TTO utilities were used to estimate a preference-based scoring algorithm by regression analysis (tobit models with robust standard errors clustered on participants with censoring at -1). RESULTS: A classification system with 8 dimensions (CFQ-R-8 dimensions; physical functioning, vitality, emotion, role functioning, breathing difficulty, cough, abdominal pain, and body image) was generated. TTO was completed by 400 participants (mean age, 47.3 years; 49.8% female). Among the regression models evaluated, the tobit heteroscedastic-ordered model was preferred, with a predicted utility range from 0.236 to 1, no logical inconsistencies, and a mean absolute error of 0.032. CONCLUSION: The CFQ-R-8 dimensions is the first disease-specific, preference-based scoring algorithm for CF, enabling estimation of disease-specific utilities for CEA based on the well-validated and widely used CFQ-R.

Hypothetical versus experienced health state valuation: a qualitative study of adult general public views and preferences.

OBJECTIVES: Responses from hypothetical and experienced valuation tasks of health-related quality of life differ, yet there is limited understanding of why these differences exist, what members of the public think about them, and acceptable resolutions. This study explores public understanding of, opinions on, and potential solutions to differences between hypothetical versus experienced responses, in the context of allocating health resources. METHODS: Six focus groups with 30 members of the UK adult public were conducted, transcribed verbatim, and analysed using framework analysis. Participants self-completed the EQ-5D-5L, before reporting the expected consequences of being in two hypothetical EQ-5D-5L health states for ten years. Second, participants were presented with prior results on the same task from a public (hypothetical) and patient (experienced) sample. Third, a semi-structured discussion explored participants': (1) understanding, (2) opinions, and (3) potential resolutions. RESULTS: Twenty themes emerged, clustered by the three discussion points. Most participants found imagining the health states difficult without experience, with those aligned to mental health harder to understand. Participants were surprised that health resource allocation was based on hypothetical responses. They viewed experienced responses as more accurate, but noted potential biases. Participants were in favour of better informing, but not influencing the public. Other solutions included incorporating other perspectives (e.g., carers) or combining/weighting responses. CONCLUSION: Members of the UK public appear intuitively not to support using potentially uninformed public values to hypothetical health states in the context of health resource allocation. Acceptable solutions involve recruiting people with greater experience, including other/combinations of views, or better informing respondents.

Developing an Australian utility value set for MacNew-7D health states.

BACKGROUND: A new preference-based measure (MacNew-7D) has recently been developed to allow condition-specific data to be used to capture the quality of life in health economic evaluations in cardiology; however, a general population value set has not yet been developed. This study developed a population utility value set for the MacNew-7D heart disease-specific instrument. METHODS: The discrete choice experiments (DCE) technique was chosen as the preference-elicitation method. The DCE asked respondents to compare two options and to state their preferences. The survey was conducted using an online panel of respondents, with quota sampling using age groups, sex and jurisdictions to achieve representativeness of the Australian population. The total design consisted of 200 choice sets, of which each respondent answered eight. Additionally, each respondent answered two quality control choice sets. The best-fitting models were selected on the basis of consistency, parsimony, and goodness of fit. RESULTS: In total, 1903 respondents were included in the analyses. The MacNew-7D utility value set ranged from -0.4456 to 1.000 for health states defined by the classification system. The best-fitting model retained all levels for five dimensions and collapsed one adjacent level for the other two dimensions. Findings were robust to sensitivity analyses related to the inclusion or exclusion of dominancy and repeat tasks. CONCLUSION: Findings indicated that the MacNew-7D utility value set is likely suitable for estimating quality-adjusted life years derived from the MacNew heart disease health-related quality-of-life questionnaire. This value set was derived from an Australian population-based sample and may not be generalisable to dissimilar populations.

Should We Consider Including a Value for "Hope" as an Additional Benefit Within Health Technology Assessment?

Health technology assessment (HTA) typically uses average health-related quality of life gain as its main measure of benefit used in economic evaluation. Nevertheless, there have been calls to consider novel aspects of benefit including the "value of hope," defined as a patients' potential preferences for a wider distribution of treatment benefit with a positive skew, in the hope that they will be one of the lucky ones. The value of hope may also derive from feeling hopeful as a positive mental state, which may be missing from current measures of health-related quality of life. The value attributed to feeling hopeful could be related to, or additional to, the value derived from possible risk-seeking preferences. Here, we reflect upon the strength of the case for the inclusion of the "value of hope" taking a critical look at the commonly referenced evidence for including the "value of hope" as risk-seeking preferences. We also draw attention to other conceptions of hope-as an emotion, a cognitive process, or a combination of both-and reflect upon the potential of including these broader notions of hope into HTA. The case for the inclusion of the "value of hope" based on risk-seeking preferences is weak. We suggest research questions that could give further evidence on whether hope is an important missing value from HTA.

A Discrete Choice Experiment to Elicit General Population Preferences Around the Factors Influencing the Choice to Make Clinical Negligence Claims.

OBJECTIVES: This article determines public stated preferences around different factors that influence the choice to make clinical negligence claims against a national healthcare system. METHODS: A large online survey was conducted using a discrete choice experiment (DCE) with the UK general population (N = 1013). DCE tasks involved a single profile and participants chose whether to make a claim for compensation (yes/no) after one of 3 randomly allocated patient safety incident (PSI) "scenarios" of different severities (mild, moderate, severe). DCE attributes described the actions of the healthcare system after a PSI and characteristics of the clinical negligence claims process. The data were modeled separately for each scenario (mild, moderate, severe) using logistic regression. Marginal effects and the probability of making a claim in a baseline case were estimated. RESULTS: Probability of choosing to claim was reduced by receipt of an apology, investigation and prevention of recurrence of the PSI, and longer time until claim decision and increased by an easy and straightforward claims process and high chance of compensation and for the mild scenario higher compensation amounts. Marginal effects and baseline case probabilities differed by scenario severity. CONCLUSIONS: The results suggest the actions of the healthcare system after a PSI and characteristics of the claims process have a larger impact on the probability of making a claim for milder PSIs. For more severe PSIs, a larger probability of making a claim was observed, and the choice was less influenced by the actions of the healthcare system after the PSI and characteristics of the claims process.

Does Changing the Age of a Child to be Considered in 3-Level Version of EQ-5D-Y Discrete Choice Experiment-Based Valuation Studies Affect Health Preferences?

OBJECTIVES: There has been some debate about the choice of perspective and the age of the child considered when completing preference elicitation tasks in the 3-level version of EQ-5D-Y (EQ-5D-Y-3L) valuation protocol. This study aimed to clarify the impact on latent scale EQ-5D-Y-3L values of varying the age of the child experiencing the health state considered by respondents completing the discrete choice experiment (DCE) tasks of the protocol. METHODS: We conducted an online DCE with a representative sample of 1000 adults in the United Kingdom and 1000 adults in the United States. Respondents selected the health state they prefer from a series of DCE paired EQ-5D-Y-3L health state comparisons using their own perspective and that of a hypothetical child from the following age groups: "5-7 years old," "8-10 years old," "11-13 years old," and "14-15 years old." Data analysis was conducted using separate multinomial logit models for each perspective and country. We also estimated combined models including data from each possible pair of perspectives and used interactions between EQ-5D-Y-3L levels and perspective to determine whether any differences were statistically significant. RESULTS: No statistically significant differences in coefficients between perspectives were found in the United States. In the United Kingdom, there were differences between the own perspective and the 5 to 7 years old perspective (looking after myself level 3) and between the 5 to 7 years old perspective and the 8 to 10 years old perspective (usual activities level 3). CONCLUSIONS: Our results suggest that there is minimal impact on latent scale values when using different ages of the hypothetical child in the current EQ-5D-Y-3L valuation protocol.

The EQ-HWB: Overview of the Development of a Measure of Health and Wellbeing and Key Results.

OBJECTIVES: Existing measures for estimating quality-adjusted life-years are mostly limited to health-related quality of life. This article presents an overview of the development the EQ-HWB (EQ Health and Wellbeing), which is a measure that encompasses health and wellbeing. METHODS: Stages: (1) Establishing domains through reviews of the qualitative literature informed by a conceptual framework. (2) Generation and selection of items to cover the domains. (3) Face validation of these items through qualitative interviews with 168 patients, social care users, general population, and carers across 6 countries (Argentina, Australia, China, Germany, United Kingdom, United States). (4) Extensive psychometric testing of candidate items (using classical, factor analysis, and item response theory methods) on > 4000 respondents in the 6 countries. Stakeholders were consulted throughout. RESULTS: A total of 32 subdomains grouped into 7 high-level domains were identified from the qualitative literature and 97 items generated to cover them. Face validation eliminated 36 items, modified 14, and added 3. Psychometric testing of 64 items found little difference in missing data or problems with response distribution, the conceptual model was confirmed except in China, and most items performed well in the item response theory in all countries. Evidence was presented to stakeholders in 2 rounds of consultation to inform the final selection of items for the EQ-HWB (25-item) and the short version of EQ-HWB (9-items). CONCLUSIONS: EQ-HWB measures have been developed internationally for evaluating interventions in health, public health, and social care including the impact on patients, social care users, and carers.

Convergent validity of EQ-5D with core outcomes in dementia: a systematic review.

OBJECTIVES: To explore through a systematic review, the convergent validity of EQ-5D (EQ-5D-3L and EQ-5D-5L (total score and dimensions)) with core outcomes in dementia and investigate how this may be impacted by rater-type; with the aim of informing researchers when choosing measures to use in dementia trials. METHODS: To identify articles relevant to the convergent validity of EQ-5D with core dementia outcomes, three databases were electronically searched to September 2022. Studies were considered eligible for inclusion within the review if they included individual level data from people with dementia of any type, collected self and/or proxy reported EQ-5D and collected at least one core dementia outcome measure. Relevant data such as study sample size, stage of dementia and administration of EQ-5D was extracted, and a narrative synthesis was adopted. RESULTS: The search strategy retrieved 271 unique records, of which 30 met the inclusion criteria for the review. Twelve different core outcome measures were used to capture dementia outcomes: cognition, function, and behaviour/mood across the studies. Most studies used EQ-5D-3L (n = 27). Evidence related to the relationship between EQ-5D and measures of function and behaviour/mood was the most robust, with unanimous directions of associations, and more statistically significant findings. EQ-5D dimensions exhibited associations with corresponding clinical outcomes, whereby relationships were stronger with proxy-EQ-5D (than self-report). CONCLUSION: Measuring health-rated quality of life in dementia populations is a complex issue, particularly when considering balancing the challenges associated with both self and proxy report. Published evidence indicates that EQ-5D shows evidence of convergent validity with the key dementia outcomes, therefore capturing these relevant dementia outcomes. The degree of associations with clinical measures was stronger when considering proxy-reported EQ-5D and differed by EQ-5D dimension type. This review has revealed that, despite the limited targeted psychometric evidence pool and reliance on clinical and observational studies, EQ-5D exhibits convergent validity with other dementia outcome measures.

Assessing the psychometric performance of EQ-5D-5L in dementia: a systematic review.

BACKGROUND: EQ-5D is widely used for valuing changes in quality of life for economic evaluation of interventions for people with dementia. There are concerns about EQ-5D-3L in terms of content validity, poor inter-rater agreement and reliability in the presence of cognitive impairment, but there is also evidence to support its use with this population. An evidence gap remains regarding the psychometric properties of EQ-5D-5L. OBJECTIVES: To report psychometric evidence around EQ-5D-5L in people with dementia. METHODS: A systematic review identified primary studies reporting psychometric properties of EQ-5D-5L in people with dementia. Searches were completed up to November 2020. Study selection, data extraction and quality assessment were undertaken independently by at least 2 researchers. RESULTS: Evidence was extracted from 20 articles from 14 unique studies covering a range of dementia severity. Evidence of known group validity from 5 of 7 studies indicated that EQ-5D-5L distinguishes severity of disease measured by cognitive impairment, depression, level of dependence and pain. Convergent validity (9 studies) showed statistically significant correlations of weak and moderate strengths, between EQ-5D-5L scores and scores on other key measures. Statistically significant change was observed in only one of 6 papers that allowed this property to be examined. All seven studies showed a lack of inter-rater reliability between self and proxy reports with the former reporting higher EQ-5D-5L scores than those provided by proxies. Five of ten studies found EQ-5D-5L to be acceptable, assessed by whether the measure could be completed by the PwD and/or by the amount of missing data. As dementia severity increased, the feasibility of self-completing EQ-5D-5L decreased. Three papers reported on ceiling effects, two found some evidence in support of ceiling effects, and one did not. CONCLUSIONS: EQ-5D-5L seems to capture the health of people with dementia on the basis of known-group validity and convergent validity, but evidence is inconclusive regarding the responsiveness of EQ-5D-5L. As disease progresses, the ability to self-complete EQ-5D-5L is diminished.

Development of a preference-based heart disease-specific health state classification system using MacNew heart disease-related quality of life instrument.

PURPOSE: The MacNew Heart Disease Health-Related Quality of Life Instrument (MacNew) is a validated, clinically sensitive, 27-item disease-specific questionnaire. This study aimed to develop a new heart disease-specific classification system for the MacNew amenable for use in health state valuation. METHODS: Patients with heart disease attending outpatient clinics and inpatient wards in Brisbane, Australia, completed MacNew. The development of the new disease-specific classification system included three stages. First, a principal component analysis (PCA) established dimensionality. Second, Rasch analysis was used to select items for each dimension. Third, Rasch analysis was used to explore response-level reduction. In addition, clinician and patient judgement informed item selection. RESULTS: Participants included 685 patients (acute coronary 6%, stable coronary 41%, chronic heart failure 20%). The PCA identified 4 dimensions (restriction, emotion, perception of others, and symptoms). The restriction dimension was divided into physical and social dimensions. One item was selected from each to be included in the classification system. Three items from the emotional dimension and two symptom items were also selected. The final classification system had seven dimensions with four severity levels in each: physical restriction; excluded from doing things with other people; worn out or low in energy; frustrated, impatient or angry; unsure and lacking in self-confidence; shortness of breath; and chest pain. CONCLUSION: This study generated a brief heart disease-specific classification system, consisting of seven dimensions with four severity levels in each. The classification system is amenable to valuation to enable the generation of utility value sets to be developed for use in economic evaluation.

Transforming challenges into opportunities: conducting health preference research during the COVID-19 pandemic and beyond.

The disruptions to health research during the COVID-19 pandemic are being recognized globally, and there is a growing need for understanding the pandemic's impact on the health and health preferences of patients, caregivers, and the general public. Ongoing and planned health preference research (HPR) has been affected due to problems associated with recruitment, data collection, and data interpretation. While there are no "one size fits all" solutions, this commentary summarizes the key challenges in HPR within the context of the pandemic and offers pragmatic solutions and directions for future research. We recommend recruitment of a diverse, typically under-represented population in HPR using online, quota-based crowdsourcing platforms, and community partnerships. We foresee emerging evidence on remote, and telephone-based HPR modes of administration, with further studies on the shifts in preferences related to health and healthcare services as a result of the pandemic. We believe that the recalibration of HPR, due to what one would hope is an impermanent change, will permanently change how we conduct HPR in the future.

Deriving a Preference-Based Measure for People With Duchenne Muscular Dystrophy From the DMD-QoL.

OBJECTIVES: This study generates a preference-based measure for capturing the quality of life of people with Duchenne muscular dystrophy (DMD) from a new measure of quality of life, DMD-QoL. METHODS: A health state classification system was derived from the DMD-QoL based on psychometric performance of items, factor analysis, and item response theory analysis. Preferences for health states described by the classification system were elicited using an online discrete choice experiment survey with life years as an additional attribute, from members of the UK general population (n = 1043). Discrete choice experiment data was modeled using a conditional fixed-effects logit model and utility estimates were directly anchored on the 1 to 0 full health-dead scale. RESULTS: The health state classification system has 8 dimensions: mobility, difficulty using hands, difficulty breathing, pain, tiredness, worry, participation, and feeling good about yourself. The standard model had mostly statistically significant coefficients and reflected the instrument's monotonic structure. However, 2 dimensions had inconsistent coefficients (where utility increased as health worsened) and a consistent model was estimated that merged adjacent inconsistent severity levels. The best state defined by the classification system has a value of 1 and the worst state has a value of -0.559. CONCLUSION: The modeled results enable DMD-QoL-8D utility values to be generated using DMD-QoL or DMD-QoL-8D data to generate QALYs for people with DMD. QALYs can then be used to inform economic models of the cost-effectiveness of interventions in DMD. Future research comparing the psychometric performance of DMD-QoL-8D to existing generic preference-based measures, including EQ-5D-5L, is recommended.

Estimating a Preference-Based Index for Mental Health From the Recovering Quality of Life Measure: Valuation of Recovering Quality of Life Utility Index.

BACKGROUND: There are increasing concerns about the appropriateness of generic preference-based measures to capture health benefits in the area of mental health. OBJECTIVES: The aim of this study is to estimate preference weights for a new measure, Recovering Quality of Life (ReQoL-10), to better capture the benefits of mental healthcare. METHODS: Psychometric analyses of a larger sample of mental health service users (n = 4266) using confirmatory factor analyses and item response theory were used to derive a health state classification system and inform the selection of health states for utility assessment. A valuation survey with members of the UK public representative in terms of age, sex, and region was conducted using face-to-face interviewer administered time-trade-off with props. A series of regression models were fitted to the data and the best performing model selected for the scoring algorithm. RESULTS: The ReQoL-Utility Index (UI) classification system comprises 6 mental health items and 1 physical health item. Sixty-four health states were valued by 305 participants. The preferred model was a random effects model, with significant and consistent coefficients and best model fit. Estimated utilities modeled for all health states ranged from -0.195 (state worse than dead) to 1 (best possible state). CONCLUSIONS: The development of the ReQoL-UI is based on a novel application of item response theory methods for generating the classification system and selecting health states for valuation. Conventional time-trade-off was used to elicit utility values that are modeled to enable the generation of QALYs for use in cost-utility analysis of mental health interventions.

Vignette-Based Utilities: Usefulness, Limitations, and Methodological Recommendations.

Health technology assessment agencies often prefer that utilities used to calculate quality-adjusted life years in cost-utility analyses (CUAs) are derived using standardized methods, such as generic preference-based measures completed by patients in clinical trials. However, there are situations when no standardized approach is feasible or appropriate for a specific medical condition or treatment that must be represented in a CUA. When this occurs, vignette-based methods are often used to estimate utilities. A vignette (sometimes called a "scenario," "health state description," "health state vignette," or "health state") is a description of a health state that is valued in a preference elicitation task to obtain a utility estimate. This method is sometimes the only feasible way to estimate utilities representing a concept that is important for a CUA. Consequently, vignette-based studies continue to be conducted and published, with the resulting utilities used in economic models to inform decision making about healthcare resource allocation. Despite the potential impact of vignette-based utilities on medical decision making, there is no published guidance or review of this methodology. This article provides recommendations for researchers, health technology assessment reviewers, and policymakers who may be deciding whether to use vignette-based methods, designing a vignette study, using vignette-based utilities in a CUA, or evaluating a CUA that includes vignette-based utilities. Recommendations are provided on: (A) when to use vignette-based utilities, (B) methods for developing vignettes, (C) valuing vignettes, (D) use of vignette-based utilities in models, and (E) limitations of vignette methods.

A Review of the Psychometric Performance of Selected Child and Adolescent Preference-Based Measures Used to Produce Utilities for Child and Adolescent Health.

OBJECTIVE: This review examined the psychometric performance of 4 generic child- and adolescent-specific preference-based measures that can be used to produce utilities for child and adolescent health. METHODS: A systematic search was undertaken to identify studies reporting the psychometric performance of the Child Health Utility (CHU9D), EQ-5D-Y (3L or 5L), and Health Utilities Index Mark 2 (HUI2) or Mark 3 (HUI3) in children and/or adolescents. Data were extracted to assess known-group validity, convergent validity, responsiveness, reliability, acceptability, and feasibility. Data were extracted separately for the dimensions and utility index where this was reported. RESULTS: The review included 76 studies (CHU9D n = 12, EQ-5D-Y-3L n = 20, HUI2 n = 26,HUI3 n = 43), which varied considerably across conditions and sample size. EQ-5D-Y-3L had the largest amount of evidence of good psychometric performance in proportion to the number of studies examining performance. The majority of the evidence related to EQ-5D-Y-3L was based on dimensions. CHU9D was assessed in fewer studies, but the majority of studies found evidence of good psychometric performance. Evidence for HUI2 and HUI3 was more mixed, but the studies were more limited in sample size and statistical power, which was likely to have affected performance. CONCLUSIONS: The heterogeneity of published studies means that the evidence is based on studies across a range of countries, populations and conditions, using different study designs, different languages, different value sets and different statistical techniques. Evidence for CHU9D in particular is based on a limited number of studies. The findings raise concerns about the comparability of self-report and proxy-report responses to generate utility values for children and adolescents.