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

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

Testing 2 Alternative Time Trade-Off Methods for Valuation of Children's Health States.

OBJECTIVES: Composite time trade-off (cTTO) values for EQ-5D-Y-3L health states tend to be high, raising concerns about sensitivity particularly for mild or moderate health states. We conceptualized and pilot tested 2 alternative time trade-off (TTO) variants: the caregiver TTO (CGTTO) and lag-time TTO (LTTO). METHODS: We collected CGTTO and LTTO data in China for 10 EQ-5D-Y-3L health states and compared the resulting values, respondent feedback, and task completion times with those from an EQ-5D-Y-3L valuation study using cTTO. We also examined how age and parental status of respondents could affect TTO values. RESULTS: A total of 304 participants were included in this study. Overall, cTTO showed statistically better results in all feedback questions. On a 5-point Likert scale where lower score means greater agreement, the mean (SD) feedback scores for cTTO, LTTO, and CGTTO were 1.18 (0.58), 1.45 (0.91), and 1.65 (1.02) for "easy to understand"; 1.45 (0.91), 1.94 (1.08), and 1.86 (1.24) for "easy to differentiate"; and 3.61 (1.29), 2.97 (1.33), and 3.02 (1.50) for "difficult to decide," respectively. The mean (SD) TTO values of all 10 states were 0.463 (0.494), 0.387 (0.555), and 0.123 (0.710) for cTTO, LTTO, and CGTTO, respectively. The effects of age and parental status on TTO values differed by the 3 methods. CONCLUSIONS: LTTO and CGTTO produce values with good characteristics and merit further investigation. Researchers need to be aware of the differences in design and values when using the TTO method to value children's health states.

Using the Online Elicitation of Personal Utility Functions Approach to Derive a Patient-Based 5-Level Version of EQ-5D Value Set: A Study in 122 Patients With Rheumatic Diseases From Germany.

OBJECTIVES: Traditional preference elicitation methods, such as discrete choice experiments or time trade-off, usually require large sample sizes. This can limit their applicability in patient populations, where recruiting enough participants can be challenging. The objective of this study was to test a new method, called the Online elicitation of Personal Utility Functions (OPUF) approach, to derive an EQ-5D-5L value set from a relatively small sample of patients with rheumatic diseases. METHODS: OPUF is a new type of online survey that implements compositional preference elicitation techniques. Central to the method are 3 valuation steps: (1) dimension weighting, (2) level rating, and (3) anchoring. An English demo version of the OPUF survey can be accessed at https://valorem.health/eq5d5l. From the responses, a personal EQ-5D-5L utility function can be constructed for each participant, and a group-level value set can be derived by aggregating model coefficients across participants. RESULTS: A total of 122 patients with rheumatic disease from Germany completed the OPUF survey. The survey was generally well received; most participants completed the survey in less than 20 minutes and were able to derive a full EQ-5D-5L value set. The precision of mean coefficients was high, despite the small sample size. CONCLUSIONS: Our findings demonstrate that OPUF can be used to derive an EQ-5D-5L value set from a relatively small sample of patients. Although the method is still under development, we think that it has the potential to be a valuable preference elicitation tool and to complement traditional methods in several areas.

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

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

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

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

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

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

An Intersectional Approach to Quantifying the Impact of Geographic Remoteness and Health Disparities on Quality-Adjusted Life Expectancy: Application to Australia.

OBJECTIVES: An intersectional multilevel analysis of individual heterogeneity and discriminatory accuracy (MAIHDA) is a novel method for exploring the interaction between sociodemographic characteristics that affect health outcomes. This study explores the interaction between geographic remoteness and socioeconomic status on health outcomes in Australia from an intersectional perspective. METHODS: Data from a cross-sectional survey were matched with data from the Australian Bureau of Statistics and the Australian Institute of Health and Welfare. To explore the effect of health-related quality of life on life expectancy, quality-adjusted life expectancy (QALE) was estimated through applying utility values derived from the EQ-5D-5L to life table data from the Australian Bureau of Statistics. The effect of geographic remoteness on QALE was quantified using multivariable linear regression. An intersectional MAIHDA was performed to explore differences in mean QALE across strata formed by intersections of age, sex, and Socioeconomic Indexes for Areas score. RESULTS: Based on multivariable linear modeling, QALE declined significantly with increasing remoteness (inner regional, -1.0 years [undiscounted]; remote/very remote, -3.3 years [undiscounted]) (P < .001). In contrast, life expectancy was only significantly different between participants in remote/very remote areas and major cities (ß-coefficient, -2.4; 95% CI -4.4 to -0.4; P = .016). No intersectional interaction effects between strata on QALE were found in the MAIHDA. CONCLUSIONS: QALE has considerable value as a metric for exploring disparities in health outcomes. Given that no intersectional interactions were identified, our findings support broad interventions that target the underlying social determinants of health appropriately reduce disparities versus interventions targeting intersectional interactions.

Use of Utility and Disability Weights in Economic Evaluation of Pediatric Vaccines.

OBJECTIVE: To describe how utility weights and disability weights have been used in the context of quality-adjusted life-years (QALYs) and disability-adjusted life-years (DALYs)-based cost-effectiveness analysis (CEA) of pediatric vaccines for infectious diseases and assess the comparability between weights. METHODS: A systematic review was conducted of CEAs of pediatric vaccines for 16 infectious diseases, published between January 2013 and December 2020 and using QALYs or DALYs as outcome measure. Data on values and sources of weights for the estimation of QALYs and DALYs were extracted from studies and compared across similar health states. Reporting was conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. RESULTS: Out of 2154 articles identified, 216 CEAs met our inclusion criteria. Of the included studies, 157 used utility weights and 59 used disability weights in their valuation of health states. In QALY studies, the source, background, who's preferences (adults'/children's) were applied and adjustments made to utility weights were poorly reported. In DALY studies, the Global Burden of Disease study was most often referenced. Valuation weights for similar health states varied within QALY studies and between DALY and QALY studies, but no systematic differences were identified. CONCLUSIONS: This review identified considerable gaps in the way valuation weights are used and reported on in CEA. The nonstandardized use of weights may lead to different conclusions about cost-effectiveness of vaccines and policy decisions.

A Qualitative Investigation to Develop an Adapted Version of the EQ-5D-Y-3L for Use in Children Aged 2-4 Years.

OBJECTIVES: Few preference-weighted health-related quality-of-life measures exist for children under 5 years of age. Young children are substantial consumers of healthcare services. This project aims to assess EQ-5D-Y-3L's appropriateness in children aged 2 to 4 years and to coproduce with parents a suitable adaptation. METHODS: Purposive sampling at the Murdoch Children's Research Institute and Royal Children's Hospital was used to recruit parents or carers of children aged 2 to 4 years in Australia. Online focus groups were conducted consisting of 13 parents of healthy children, and 6 parents of children with moderate to severe health conditions. Parents provided feedback on each dimension of the proxy EQ-5D-Y-3L. Recordings were transcribed and thematic analysis was conducted. Qualitative findings guided the design of adaptations to the instrument. The adaptations were piloted to obtain feedback and refined to improve language translatability and comparability with other EuroQol instruments. RESULTS: The adapted EQ-5D-Y-3L was considered generally acceptable by the parents. Parents provided a wide range of examples of how each domain related to their children, with varied examples provided across ages 2 to 4 years and health status. Additional or alternative wording was suggested by parents to improve the applicability of the instrument to this age group. One example of this was the change of the domain wording "walking about" to "movement"-ID5:"In this age group, movement is more important than walking." CONCLUSIONS: The adapted EQ-5D-Y-3L has improved relevance for 2-4-year olds and appears easy to complete. Further testing of the adapted instrument is required to evaluate acceptability, reliability, and validity.

Distinct developmental trajectories of health-related quality of life for boys and girls throughout childhood and adolescence; a national level longitudinal study.

BACKGROUND: To identify and describe distinct developmental trajectories of health-related quality of life (HRQoL) in a national level Australian population sample, overall and separately for boys and girls. METHODS: Data were from the Longitudinal Study of Australian Children (LSAC). Participants were children aged 4-5 years recruited in 2004 and followed through to age 16-17 years in 2016, and their caregivers. Group-based trajectory modelling was used to identify groups of children that follow qualitatively distinct developmental trajectories of HRQoL. RESULTS: Three distinct trajectories were identified for the total sample: (1) high-stable (52.2% of children); (2) middle-stable (38.0%); and (3) low-declining (9.8%). These trajectories differed for boys, who saw increasing HRQoL in the highest trajectory group; a middle-stable trajectory; and declining and rebounding HRQoL in the lowest trajectory group. In contrast, girls saw no increasing or rebounding trajectories; approximately half of girls had high-stable HRQoL and the remaining half had either steadily or rapidly declining HRQoL from age 4-5 to 16-17 years. CONCLUSIONS: Our results highlight the importance of considering the distinct trajectories for girls and boys and not relying on population mean levels of HRQoL for decision-making. The presence of developmentally distinct trajectories of HRQoL, and differences in the trajectories faced by boys and girls, should be considered when assessing the effectiveness of treatments and interventions impacting upon HRQoL throughout childhood and adolescence. Failure to account for these pre-existing trajectories may over- or under-estimate treatment effects.

Experience-based health state valuation using the EQ VAS: a register-based study of the EQ-5D-3L among nine patient groups in Sweden.

BACKGROUND: The EQ VAS component of the EQ-5D questionnaire has been used to assess patients' valuation of their own health besides its use for self-reporting of overall health status. The objective of the present study was to identify patients' valuation of EQ-5D-3L health states using the EQ VAS in different patient groups over time and in comparison to the general population. METHODS: Data were obtained from patients from nine National Quality Registers (n = 172,070 patients) at baseline and at 1-year follow-up and compared with data from the general population (n = 41,761 participants). The correlation between EQ VAS scores and EQ-5D-3L index based on the Swedish experience-based VAS value set was assessed. Ordinary least squares (OLS) regression models were used to determine the association between EQ-5D-3L dimensions and EQ VAS valuation. RESULTS: EQ VAS scores showed consistency with severity of health states both at baseline and at 1-year follow-up in the nine selected EQ-5D-3L health states. The regression models showed mostly consistent decrements by severity levels in each dimension at both time points and similar to the general population. The dimension mainly associated with inconsistency was the self-care severity level three. Problems in the anxiety/depression dimension had the largest impact on overall health status in most of the patient groups and the general population. CONCLUSION: The study has demonstrated the important role EQ VAS can play in revealing patients' valuation of their health and showed the variation in valuation of EQ-5D-3L dimensions and levels of severity across different patient groups.

Health-related quality of life in patients with extremity bone sarcoma after surgical treatment: a systematic review.

PURPOSE: We conducted a systematic review of studies reporting on measurement of health-related quality of life (HRQoL), with a special focus on the use of the preference-weighted instruments, in patients with extremity bone sarcoma treated with limb-salvage surgery or amputation. METHODS: We searched MedLine, Embase, Cochrane Library and Web of Science for English-language studies reporting on HRQoL of patients with bone sarcoma from inception to 28 August 2023. All records found were independently reviewed by two reviewers. We used the Newcastle-Ottawa Scale (NOS) and the CONSORT 2010 checklist to assess the quality of the cohort and randomised studies, respectively. RESULTS: The search identified 1225 records, of which 16 studies were included for data extraction. Only one study used a preference-weighted instrument for measuring HRQoL in a small sample of patients (n = 28). Ten studies used the generic SF-36 questionnaire, but no preference-weighted HRQoL based on SF-6D was derived from the SF-36 scores. Most studies comparing HRQoL between amputation and limb-salvage surgery reported no significant differences. Twelve cohort studies scored six or more out of nine points based on the NOS. The only randomised study scored 54% on the CONSORT 2010 checklist. CONCLUSIONS: The approaches used to measure HRQoL were inconsistent and outcome scores varied substantially. Only one study used preference-weighted instruments for HRQoL measurement. Future research into the surgical treatment of extremity bone sarcoma should consider the use of preference-weighted instruments to measure HRQoL, which will therefore enable economic evaluation for the growing orthopaedic armamentarium of novel surgical interventions. REGISTRATION: This systematic review was registered with the PROSPERO International prospective register of systematic reviews (CRD42021282380).

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.

Understanding the measurement relationship between EQ-5D-5L, PROMIS-29 and PROPr.

PURPOSE: Many generic patient-reported instruments are available for the measurement of health outcomes, including EQ-5D-5L, and the Patient-Reported Outcome Measurement Information System (PROMIS). Assessing their measurement characteristics informs users about the consistency between, and limits of, evidence produced. The aim was to assess the measurement relationship between the EQ-5D-5L descriptive system and value sets, the PROMIS-29 and PROPr (PROMIS value set). METHODS: Data were extracted from a cross-sectional survey administering measures of quality of life online in Australia. Descriptive analysis, agreement and construct validity assessment methods were used to compare instruments at the item, domain and value set level. RESULTS: In total, 794 Australians completed the survey. Convergent validity analysis found that similar dimensions across instruments were highly correlated (> 0.50), but the PROMIS-29 assesses additional health concepts not explicitly covered by EQ-5D (sleep and fatigue). Known-group assessment found that EQ-5D-5L and PROPr were able to detect those with and without a condition (ES range 0.78-0.83) but PROPr could more precisely detect differing levels of self-reported health. Both instruments were sensitive to differences in levels of pain. DISCUSSION: There is some consistency in what the EQ-5D-5L, PROMIS-29 and PROPr measure. Differences between value set characteristics can be linked to differences what is measured and the valuation approaches used. This has implications for the use of each in assessing health outcomes, and the results can inform decisions about which instrument should be used in which context.

An Analysis of 5-Level Version of EQ-5D Adjusting for Treatment Switching: The Case of Patients With Epidermal Growth Factor Receptor T790M-Positive Nonsmall Cell Lung Cancer Treated With Osimertinib.

OBJECTIVES: Treatment switching from control to treatment after disease progression is common in oncology trials. Analyses of survival data typically adjust for this bias, but such adjustments are rarely performed in analyses of patient-reported outcomes. This analysis aimed to examine the impact of adjusting for treatment switching on estimated treatment effects on 5-level version of EQ-5D (EQ-5D-5L) utilities and quality-adjusted life-years (QALYs). The AURA3 trial (NCT02151981) was a randomized controlled trial comparing osimertinib with platinum-based doublet chemotherapy (standard care) in patients with locally advanced or metastatic epidermal growth factor receptor mutant- and T790M-positive nonsmall cell lung cancer whose disease has progressed with previous epidermal growth factor receptor tyrosine kinase inhibitor therapy. METHODS: Descriptive analyses were used to compare treatment arms. The primary analysis used a 2-stage least squares instrumental variable regression to estimate treatment effect adjusting for treatment crossover. Time to deterioration, defined from baseline to minimally important deterioration in EQ-5D-5L utility, was assessed using a rank preserving structural failure time model. RESULTS: Intention-to-treat analysis of imputed data showed incremental QALYs for osimertinib of 0.23 at 60 weeks. Accounting for treatment switching increased this to 0.52 in the primary analysis and to 0.63 QALYs in sensitivity analysis at 150 weeks. Time to deterioration analysis showed longer health-related quality of life maintenance with osimertinib, of 12.76 weeks, although this was at the borderline of statistical significance (acceleration factor, ψ = -0.275; 95% confidence interval -0.50 to 0.00). CONCLUSIONS: This analysis demonstrates methods to adjust for treatment switching in the analysis of EQ-5D-5L from clinical trials. Failure to account for crossover substantially underestimated the QALY gain for osimertinib.

A Systematic Review of International Guidance for Self-Report and Proxy Completion of Child-Specific Utility Instruments.

OBJECTIVES: This study aimed to identify and summarize published guidance and recommendations for child self- and proxy assessment of existing child-specific instruments of health-related quality of life (HRQoL) that are accompanied by utilities. METHODS: A total of 9 databases plus websites of (1) health technology assessment and health economics outcomes research organizations and (2) instrument developers were systematically searched. Studies were included if they reported guidance for child self- and proxy assessment for child populations (0-18 years old). Three reviewers independently screened titles, abstracts, and full-text reviews against the inclusion criteria. Key features of the guidance identified were summarized. RESULTS: A total of 19 studies met the inclusion criteria. In general, journal articles provided little guidance on child self- and proxy assessment, with the majority focused on instrument development and psychometric performance more broadly. Instrument developers' websites provided more guidance for child self- and proxy reports with specific guidance found for the EQ-5D-Y and the Pediatric Quality of Life Inventory. This guidance included the minimum age for self-report and mode of administration; recommended proxy types, age range of child for whom proxy report can be completed, and target population; and recall period. Websites of leading organizations provided general guidance on HRQoL evaluation in children but lacked specific guidance about self- and proxy completion. CONCLUSIONS: EQ-5D-Y and Pediatric Quality of Life Inventory developers' websites provided the most comprehensive guidance for self-report and proxy report of their respective instruments. More evidence is required for developing best practice guidance on why, when, and how to use self- and proxy reports in assessing HRQoL in child populations.

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.

Estimating health system opportunity costs: the role of non-linearities and inefficiency.

BACKGROUND: Empirical estimates of health system opportunity costs have been suggested as a basis for the cost-effectiveness threshold to use in Health Technology Assessment. Econometric methods have been used to estimate these in several countries based on data on spending and mortality. This study examines empirical evidence on four issues: non-linearity of the relationship between spending and mortality; the inclusion of outcomes other than mortality; variation in the efficiency with which expenditures generate health outcomes; and the relationship among efficiency, mortality rates and outcome elasticities. METHODS: Quantile Regression is used to examine non-linearities in the relationship between mortality and health expenditures along the mortality distribution. Data Envelopment Analysis extends the approach, using multiple measures of health outcomes to measure efficiency. These are applied to health expenditure data from 151 geographical units (Primary Care Trusts) of the National Health Service in England, across eight different clinical areas (Programme Budget Categories), for 3 fiscal years from 2010/11 to 2012/13. RESULTS: The results suggest differences in efficiency levels across geographical units and clinical areas as to how health resources generate outcomes, which indicates the capacity to adjust to a decrease in health expenditure without affecting health outcomes. Moreover, efficient units have lower absolute levels of mortality elasticity to health expenditure than inefficient ones. CONCLUSIONS: The policy of adopting thresholds based on estimates of a single system-wide cost-effectiveness threshold assumes a relationship between expenditure and health outcomes that generates an opportunity cost estimate which applies to the whole system. Our evidence of variations in that relationship and therefore in opportunity costs suggests that adopting a single threshold may exacerbate the efficiency and equity concerns that such thresholds are designed to counter. In most health care systems, many decisions about provision are not made centrally. Our analytical approach to understanding variability in opportunity cost can help policy makers target efficiency improvements and set realistic targets for local and clinical area health improvements from increased expenditure.

The relationship between physical and mental health multimorbidity and children's health-related quality of life.

PURPOSE: To examine the relationships between physical health problems, and borderline or clinical levels of mental health symptoms and children's health-related quality of life (HRQoL). METHODS: Data were from the Longitudinal Study of Australian Children (2004-2018). Parents reported on their child's HRQoL (PedsQL), physical health problems and mental health symptoms (Strengths and Difficulties Questionnaire, SDQ). A pooled cross-sectional analysis using linear regressions examined the relationships between physical health and clinical/borderline mental health symptoms, individually and when multi-morbid, and children's HRQoL, and whether these relationships vary by a range of child, family and social factors. RESULTS: The sample comprised 47,567 observations of children aged 4-17 years. Borderline and clinical levels of mental health symptoms were associated with significantly lower HRQoL, equal to more than two-times (10.5 points) and more than three-times (16.8 points) the clinically meaningful difference, respectively. This was a larger difference than that associated with physical health problems (4.4 points). We found a significant interaction effect between physical health problems and clinical mental health symptoms which was associated with even poorer HRQoL after accounting for the individual relationships of both problems. Mental health problems were associated with poorer HRQoL for older versus younger children; and the interaction effect was significant for boys but not girls. CONCLUSION: Findings highlight the importance of identifying and addressing mental health symptoms in children of all ages, even if these problems do not meet formal clinical criteria. Particular attention should be paid to the mental health and HRQoL of children with physical-mental multimorbidity, who are at risk of disproportionately poorer HRQoL.

Evidence on the relationship between PROMIS-29 and EQ-5D: a literature review.

PURPOSE: EQ-5D and PROMIS-29 are both concise, generic measures of patient-reported outcomes accompanied by preference weights that allow the estimation of quality-adjusted life years (QALYs). Both instruments are candidates for use in economic evaluation. However, they have different features in terms of the domains selected to measure respondents' self-perceived health and the characteristics of (and methods used to obtain) the preference weights. It is important to understand the relationship between the instruments and the implications of choosing either for the evidence used in decision-making. This literature review aimed to synthesise existing evidence on the relationship between PROMIS-29 (and measures based on it, such as PROMIS-29+2) and EQ-5D (both EQ-5D-3L and EQ-5D-5L). METHODS: A literature review was conducted in PubMed and Web of Science to identify studies investigating the relationship between PROMIS-29 and EQ-5D-based instruments. RESULTS: The literature search identified 95 unique studies, of which nine studies met the inclusion criteria, i.e. compared both instruments. Six studies examined the relationship between PROMIS-29 and EQ-5D-5L. Three main types of relationship have been examined in the nine studies: (a) comparing PROMIS-29 and EQ-5D as descriptive systems; (b) mapping PROMIS-29 domains to EQ-5D utilities; and (c) comparing and transforming PROMIS-29 utilities to EQ-5D utilities. CONCLUSION: This review has highlighted the lack of evidence regarding the relationship between PROMIS-29 and EQ-5D. The impact of choosing either instrument on the evidence used in cost-effectiveness analysis is currently unclear. Further research is needed to understand the relationship between the two instruments.