Exploring the Factors that Drive Clinical Negligence Claims: Stated Preferences of Those Who Have Experienced Unintended Harm.

BACKGROUND: Better understanding of the factors that influence patients to make a financial claim for compensation is required to inform policy decisions. This study aimed to assess the relative importance of factors that influence those who have experienced a patient safety incident (PSI) to make a claim for compensation. METHOD: Participants completed an online discrete choice experiment (DCE) involving 10 single profile tasks where they chose whether or not to file a claim. DCE data were modelled using logistic, mixed logit and latent class regressions; scenario analyses, external validity, and willingness to accept were also conducted. RESULTS: A total of 1029 participants in the United Kingdom responded to the survey. An appropriate apology and a satisfactory investigation reduced the likelihood of claiming. Respondents were more likely to claim if they could hold those responsible accountable, if the process was simple and straightforward, if the compensation amount was higher, if the likelihood of compensation was high or uncertain, if the time to receive a decision was quicker, and if they used the government compensation scheme. Men are more likely to claim for low impact PSIs. DISCUSSION AND CONCLUSIONS: The actions taken by the health service after a PSI, and people's perceptions about the probability of success and the size of potential reward, can influence whether a claim is made. Results show the importance of giving an appropriate apology and conducting a satisfactory investigation. This stresses the importance around how patients are treated after a PSI in influencing the clinical negligence claims that are made.

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.

Assessment of health state utilities associated with adult and pediatric acid sphingomyelinase deficiency (ASMD).

INTRODUCTION: Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health state utilities. Therefore, the purpose of this study was to estimate utilities associated with varying levels of severity of adult and pediatric ASMD type B. METHODS: Seven adult and seven child health state vignettes describing ASMD were developed based on published literature, clinical trial results, and interviews with clinicians, patients with ASMD, and parents of children with ASMD. The health states were valued in time trade-off interviews with adult general population respondents in the UK. RESULTS: Interviews were completed with 202 participants (50.0% female; mean age = 41.3 years). The health state representing ASMD without impairment had the highest mean utility for both the adult and child health states (0.92/0.94), and severe ASMD had the lowest mean utility (0.33/0.45). Every child health state had a significantly greater utility than the corresponding adult health state. Differences between adult/child paired states ranged from 0.02 to 0.13. Subgroup analyses explored the impact of parenting status on valuation of child health states. DISCUSSION: Greater severity of ASMD was associated with lower mean utility. Results have implications for valuation of pediatric health states. The resulting utilities may be useful in cost-utility modeling estimating the value of treatment for ASMD.

Understanding UK public views on normative decisions made to value health-related quality of life in children: A qualitative study.

Developing methodology for measuring and valuing child health-related quality of life (HRQoL) is a priority for health technology agencies. One aspect of this is normative decisions that are made in child HRQoL valuation. This qualitative study aimed to better understand adult public opinion on the normative questions of whose preferences to elicit (adults, children, or both) and from which perspective (who should be imagined living in impaired health), when valuing child HRQoL. Opinions of the adult UK public (N = 32) were solicited using online semi-structured focus groups, featuring a breadth of age, sex, ethnicities, and responsibility for children under 18 years. Participants were provided with bespoke informational material on health state valuation and were probed for their views. Arguments for and against different positions were discussed. Data was analysed using framework analysis. Participants demonstrated near-to-universal agreement that children should be involved in valuation in some form, yet this should differ depending on age or maturity. There was strong support for approaches combining involvement from children and adults (e.g., their parents), especially for younger children. There was little intuitive support for the 'taxpayer argument' for asking taxpaying adults. In the context of greater involvement of children in valuation, most participants supported using an 'own' perspective. Most participants thought that valuation study participants should know the exercise is about valuing child health states for ethical reasons. Informed views from the UK public on who should be asked and with what perspective when valuing child HRQoL appear to differ from normative positions previously advocated by some health economists, such as prioritising the preferences of taxpaying adults. In contrast, the results suggest including adults and children in valuation, with the proviso that the children are of an appropriate age and level of maturity, and that an own perspective is used wherever possible.

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.

Using Age-Specific Values for Pediatric HRQoL in Cost-Effectiveness Analysis: Is There a Problem to Be Solved? If So, How?

Value sets for the EQ-5D-Y-3L published to date appear to have distinctive characteristics compared with value sets for corresponding adult instruments: in many cases, the value for the worst health state is higher and there are fewer values < 0. The aim of this paper is to consider how and why values for child and adult health differ; and what the implications of that are for the use of EQ-5D-Y-3L values in economic evaluations to inform healthcare resource allocation decisions. We posit four potential explanations for the differences in values: (a) The wording of severity labels may mean the worst problems on the EQ-5D-Y-3L are descriptively less severe than those on the EQ-5D-5L; (b) Adults may genuinely consider that children are less badly affected than adults by descriptively similar health issues. That is, for any given health problem, adult respondents in valuation studies consider children's overall health-related quality of life (HRQoL) on average to be higher than that for adults; (c) Values are being sought by eliciting adults' stated preferences for HRQoL in another person, rather than in themselves (regardless of whether the 'other person' concerned is a child); and (d) The need to elicit preferences for child HRQoL that are anchored at dead = 0 invokes special considerations regarding children's survival. Existing evidence does not rule out the possibility that (c) and (d) exert an upward bias in values. We consider the implications of that for the interpretation and use of values for pediatric HRQoL. Alternative methods for valuing children's HRQoL in a manner that is not 'age specific' are possible and may help to avoid issues of non-comparability. Use of these methods would place the onus on health technology assessment bodies to reflect any special considerations regarding child quality-adjusted life-year gains.

Randomised comparison of online interviews versus face-to-face interviews to value health states.

INTRODUCTION: Health state valuation studies using composite time trade-off (cTTO) interviews have historically been conducted face-to-face. The COVID-19 pandemic forced disruptive innovation meaning a number of valuation studies conducted interviews via videoconference. These studies found online interviews feasible and acceptable; however, studies were not constructed to test the impact of online versus face-to-face interviews. This study builds on its sister study from the UK and aims to assess the acceptability and equivalence of in person face-to-face interviews with online interviews on cTTO valuation outcomes and on data quality. METHODS: Participants were recruited into a randomised equivalence study via an external research company. Consenting participants were randomly allocated to complete a cTTO interview face-to-face or online, using the same 10 EQ-5D-5L health states. Mean and distribution of the cTTO values, participant understanding, data quality, demographic characteristics, participant preference, participant engagement and participant feedback were all compared across interview mode. Statistical equivalence for cTTO values for each state was tested using two one-sided t-tests by mode. Finally, regression analysis was completed to assess the impacts of interview mode on cTTO value while controlling for demographic characteristics of the participants. RESULTS: Mean cTTO values were shown to be equivalent for mild health states and showed no significant difference for serious health states. The proportion of individuals who expressed an interest in the study but declined to arrange an interview after finding out their randomisation was significantly higher for the face-to-face (21.6%) than the online group (1.8%). No significant difference was found between groups for participant engagement, understanding or feedback nor for any indicators of data quality. CONCLUSION: Administrating interviews face to face or online did not appear to have a statistically significant impact on mean cTTO values. Offering both online and face-to-face interviews routinely allows all participants to select the most convenient option.

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.

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.

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.

Disease-specific assessment of Vision Impairment in Low Luminance in age-related macular degeneration - a MACUSTAR study report.

BACKGROUND/AIMS: To further validate the Vision Impairment in Low Luminance (VILL) questionnaire, which captures visual functioning and vision-related quality of life (VRQoL) under low luminance, low-contrast conditions relevant to age-related macular degeneration (AMD). METHODS: The VILL was translated from German into English (UK), Danish, Dutch, French, Italian and Portuguese. Rasch analysis was used to assess psychometric characteristics of 716 participants (65% female, mean age 72±7 years, 82% intermediate AMD) from the baseline visit of the MACUSTAR study. In a subset of participants (n=301), test-retest reliability (intraclass correlation coefficient (ICC) and coefficient of repeatability (CoR)) and construct validity were assessed. RESULTS: Four items were removed from the VILL with 37 items due to misfit. The resulting Vision Impairment in Low Luminance with 33 items (VILL-33) has three subscales with no disordered thresholds and no misfitting items. No differential item functioning and no multidimensionality were observed. Person reliability and person separation index were 0.91 and 3.27 for the Vision Impairment in Low Luminance Reading Subscale (VILL-R), 0.87 and 2.58 for the Vision Impairment in Low Luminance Mobility Subscale (VILL-M), and 0.78 and 1.90 for the Vision Impairment in Low Luminance Emotional Subscale (VILL-E). ICC and CoR were 0.92 and 1.9 for VILL-R, 0.93 and 1.8 for VILL-M and 0.82 and 5.0 for VILL-E. Reported VRQoL decreased with advanced AMD stage (p<0.0001) and was lower in the intermediate AMD group than in the no AMD group (p≤0.0053). CONCLUSION: The VILL is a psychometrically sound patient-reported outcome instrument, and the results further support its reliability and validity across all AMD stages. We recommend the shortened version of the questionnaire with three subscales (VILL-33) for future use. TRIAL REGISTRATION NUMBER: NCT03349801.

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.

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.

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.

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.

Valuing child and adolescent health: a qualitative study on different perspectives and priorities taken by the adult general public.

BACKGROUND: Quantitative health preference research has shown that different "perspectives", defined here as who is imagined to be experiencing particular health states, impact stated preferences. This qualitative project aimed to elucidate this phenomenon, within the context of adults' valuation of child and adolescent health states. METHODS: Six focus groups with 30 members of the UK adult public were conducted between December 2019 and February 2020 and analysed using framework analysis. Each focus group had two stages. First, participants individually completed time trade-off tasks and a pairwise task (mirroring a discrete choice experiment without duration) for two EQ-5D-Y health states, assuming a series of perspectives: (a) themselves at current age; (b) another adult; (c) 10-year old child; (d) themselves as a 10-year old child. Second, a semi-structured discussion explored their responses. RESULTS: Participants' views were often heterogeneous, with some common themes. Qualitatively, participants expressed a different willingness to trade-off life years for a 10-year old child versus themselves or another adult, and this differed by the health profile and child imagined. The same health states were often viewed as having a different impact on utility for a 10-year old child than adults. Imagining a 10-year old child is difficult and there is variation in who is imagined. Participants found answering based on their own-adult perspective most acceptable. There were no strong preferences for prioritising child health over working-age adults' health. CONCLUSIONS: If an adult sample is used to value child- and adolescent-specific health states it is important to consider the perspective employed. Members of the adult public provide different responses when different perspectives are used due to differences in the perceived impact of the same health states. If adults are asked to imagine a child, we recommend that sampling is representative for parental status, since this can affect preferences.

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.

What is the best approach to adopt for identifying the domains for a new measure of health, social care and carer-related quality of life to measure quality-adjusted life years? Application to the development of the EQ-HWB?

Economic evaluation combines costs and benefits to support decision-making when assessing new interventions using preference-based measures to measure and value benefits in health or health-related quality of life. These health-focused instruments have limited ability to capture wider impacts on informal carers or outcomes in other sectors such as social care. Sector-specific instruments can be used but this is problematic when the impact of an intervention straddles different sectors.An alternative approach is to develop a generic preference-based measure that is sufficiently broad to capture important cross-sector outcomes. We consider the options for the selection of domains for a cross-sector generic measure including how to identify domains, who should provide information on the domains and how this should be framed. Beyond domain identification, considerations of criteria and stakeholder needs are also identified.This paper sets out the case for an approach that relies on the voice of patients, social care users and informal carers as the main source of domains and describes how the approach was operationalised in the 'Extending the QALY' project which developed the new measure, the EQ-HWB (EQ health and wellbeing instrument). We conclude by discussing the strengths and limitations of this approach. The new measure should be sufficiently generic to be used to consistently evaluate health and social care interventions, yet also sensitive enough to pick up important changes in quality of life in patients, social care users and carers.

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.

A Review of the Methods Used to Generate Utility Values in NICE Technology Assessments for Children and Adolescents.

OBJECTIVE: This review summarizes and critically examines methods used to generate utilities for child and adolescent health states in previous National Institute for Health and Care Excellence (NICE) technology assessments (TA) and highly specialized technology (HST) evaluations. METHODS: We identified all NICE TA and HST evaluations in which the licensed indication for the technology included people younger than 18 and included in the review all evaluations using a cost-utility analysis. RESULTS: The review includes 40 TA and HST evaluations. Most assessments generated utility values with the EQ-5D scored using the adult version of the EQ-5D either exclusively (n = 16) or alongside other utility measures and direct elicitation methods of patient own utility (n = 17), although 7 did not use the EQ-5D. Eight assessments used both the EQ-5D child- and adolescent-specific preference-based measures: Health Utilities Index Mark 2 (n = 6), child- and adolescent-specific preference-based measure for atopic dermatitis (n = 1), and youth version of the EQ-5D (EQ-5D-Y) valued using the adult EQ-5D value set (n = 1) or generated using mapping and valued using the adult EQ-5D value set (n = 2). Some cost-utility analyses used age adjustment (utility subtractions, weights, and published mapping formulae) from the adult EQ-5D UK population norms to reflect the general population or disease-free health for children and adolescents (n = 9), and 1 assessment assumed full health (utility value of 1). CONCLUSION: The review found limited use of child and adolescent population-specific measures to generate health state utility values for children and adolescents in NICE technology assessments. Often assessments involve the use of an adult-specific measure to reflect the health of children.