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.

The Validity of the EuroQol Health and Wellbeing Short Version (EQ-HWB-S) Instrument in Parents of Children With and Without Health Conditions.

BACKGROUND:  The EuroQol Health and Wellbeing Short Version (EQ-HWB-S) instrument has been developed to measure the health and wellbeing of care-recipients and their caregivers for use in economic evaluation.The EQ-HWB-S has nine items, and pilot UK preference weights have now been developed. OBJECTIVE: We aimed to investigate the validity of the instrument in parents of children with and without health conditions. METHODS: EQ-HWB-S data were sourced from an Australian paediatric multi-instrument comparison study. We analysed the baseline characteristics and response distribution of the EQ-HWB-S items. Assessment of known-group validity was conducted for EQ-HWB-S items, level sum-scores and preference-weighted scores, including partial effects. Known-group analyses included three child health variables and where caregivers reported coronavirus disease 2019 (COVID-19) had impacted their wellbeing. We included analyses across gender, controlled for child and parent demographic variables, and compared scores across child health conditions. RESULTS:  Item responses were distributed as expected, with higher skew for mobility and activities. Parents experienced high levels of exhaustion. We detected significant differences between groups for level sum-scores and preference-weighted scores, as hypothesised; all tests were significant (p < 0.001), with moderate effect sizes (effect sizes were slightly higher for female than male parents). The regression analysis identified significantly different EQ-HWB-S scores for child health samples compared with the general population after controlling for demographic variables. Differences were observed between child health conditions. CONCLUSION: The EQ-HWB-S is a useful instrument to measure parent quality of life for economic evaluation in this population. Data were limited to one time point; further research should investigate the instrument's sensitivity to change and test-retest reliability in this population.

Systematic Review of the Relative Social Value of Child and Adult Health.

OBJECTIVES: We aimed to synthesise knowledge on the relative social value of child and adult health. METHODS: Quantitative and qualitative studies that evaluated the willingness of the public to prioritise treatments for children over adults were included. A search to September 2023 was undertaken. Completeness of reporting was assessed using a checklist derived from Johnston et al. Findings were tabulated by study type (matching/person trade-off, discrete choice experiment, willingness to pay, opinion survey or qualitative). Evidence in favour of children was considered in total, by length or quality of life, methodology and respondent characteristics. RESULTS: Eighty-eight studies were included; willingness to pay (n = 9), matching/person trade-off (n = 12), discrete choice experiments (n = 29), opinion surveys (n = 22) and qualitative (n = 16), with one study simultaneously included as an opinion survey. From 88 studies, 81 results could be ascertained. Across all studies irrespective of method or other characteristics, 42 findings supported prioritising children, while 12 provided evidence favouring adults in preference to children. The remainder supported equal prioritisation or found diverse or unclear views. Of those studies considering prioritisation within the under 18 years of age group, nine findings favoured older children over younger children (including for life saving interventions), six favoured younger children and five found diverse views. CONCLUSIONS: The balance of evidence suggests the general public favours prioritising children over adults, but this view was not found across all studies. There are research gaps in understanding the public's views on the value of health gains to very young children and the motivation behind the public's views on the value of child relative to adult health gains. CLINICAL TRIAL REGISTRATION: The review is registered at PROSPERO number: CRD42021244593. There were two amendments to the protocol: (1) some additional search terms were added to the search strategy prior to screening to ensure coverage and (2) a more formal quality assessment was added to the process at the data extraction stage. This assessment had not been identified at the protocol writing stage.

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).

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.

Understanding the valuation of paediatric health-related quality of life: a qualitative study protocol.

INTRODUCTION: There is evidence from previous studies that adults value paediatric health-related quality of life (HRQoL) and adult HRQoL differently. Less is known about how adolescents value paediatric HRQoL and whether their valuation and decision-making processes differ from those of adults. Discrete choice experiments (DCEs) are widely used to develop value sets for measures of HRQoL, but there is still much to understand about whether and how the methods choices in the implementation of DCE valuation tasks, such as format, presentation and perspective, affect the decision-making process of participants. This paper describes the protocol for a qualitative study that aims to explore the decision-making process of adults and adolescents when completing DCE valuation tasks. The study will also explore the impact of methodological choices in the design of DCE studies (including decisions about format and presentation) on participants' thinking process. METHODS AND ANALYSIS: An interview protocol has been developed using DCE valuation tasks. Interviews will be conducted online via Zoom with both an adolescent and adult sample. In the interview, the participant will be asked to go through some DCE valuation tasks while 'thinking aloud'. After completion of the survey, participants will then be asked some predetermined questions in relation to various aspects of the DCE tasks. Interviews will be recorded and transcribed and analysed using a thematic analysis approach. ETHICS AND DISSEMINATION: Ethics approval for this study has been received for the adult sample (UTS ETH20-9632) as well as the youth sample (UTS ETH22-6970) from the University of Technology Sydney Human Research Ethics Committee. Results from this study will inform the methods to be used in development of value sets for use in the health technology assessment of paediatric interventions and treatments. Findings from this study will also be disseminated through national/international conferences and peer-reviewed journals.

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.

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.

Value Insider Season 1 Episode 2: How to Measure Quality of Life and Utility? (QoL) [Podcast].

How do we measure quality of life and utility of interventions? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Nancy Devlin about incorporating quality of life in value assessment. Prof. Devlin is professor of health economics at the University of Melbourne. Her past roles include Director of Research at the Office of Health Economics (OHE) London, Professor of Health Economics at City University of London, and she has been director of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the Chair of the Board of the EuroQol Research Foundation, the international research organization which has developed and maintains the EQ-5D patient-reported outcome (PRO) instrument. Prof Devlin explains the value of the patient voice and how it can be measured and taken into account when considering the value of healthcare interventions.

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.

An Economic Model of Gambling Behaviour: A Two-Stage Approach.

Gambling can cause significant harms and these can result in a net negative utility from participation, although lower levels of participation have potential benefits and can yield positive net utility. It is therefore important to understand and distinguish between these two stages of gambling behaviour. Currently, economic models have had limited focus on explaining why someone would gamble despite it yielding a negative utility. Here, we present a two-stage model, motivated by empirical literature and intuitive assumptions, that improves on existing economic models by distinguishing between the likelihood of gambling participation and of gambling that yields a negative utility. The model's predictions are empirically testable, consistent with existing literature, and add new insights. The model's ability to distinguish between the two stages helps to inform interventions that aim to reduce the prevalence of gambling-related harm while avoiding the need for restrictive approaches that aim to eliminate gambling altogether.

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.

Supply-Side Cost-Effectiveness Thresholds: Questions for Evidence-Based Policy.

There is growing interest in cost-effectiveness thresholds as a tool to inform resource allocation decisions in health care. Studies from several countries have sought to estimate health system opportunity costs, which supply-side cost-effectiveness thresholds are intended to represent. In this paper, we consider the role of empirical estimates of supply-side thresholds in policy-making. Recent studies estimate the cost per unit of health based on average displacement or outcome elasticity. We distinguish the types of point estimates reported in empirical work, including marginal productivity, average displacement, and outcome elasticity. Using this classification, we summarise the limitations of current approaches to threshold estimation in terms of theory, methods, and data. We highlight the questions that arise from alternative interpretations of thresholds and provide recommendations to policymakers seeking to use a supply-side threshold where the evidence base is emerging or incomplete. We recommend that: (1) policymakers must clearly define the scope of the application of a threshold, and the theoretical basis for empirical estimates should be consistent with that scope; (2) a process for the assessment of new evidence and for determining changes in the threshold to be applied in policy-making should be created; (3) decision-making processes should retain flexibility in the application of a threshold; and (4) policymakers should provide support for decision-makers relating to the use of thresholds and the implementation of decisions stemming from their application.

Capturing the value of vaccination within health technology assessment and health economics: Literature review and novel conceptual framework.

BACKGROUND: Vaccination provides significant health gains to individuals and society and can potentially improve health equity, healthcare systems and national economies. Policy decisions, however, are rarely informed by comprehensive economic evaluations (EE) including vaccination's wide-ranging value. The objective of this analysis was to focus on health technology assessment systems to identify relevant value concepts in order to improve current EE of non-pandemic vaccines. METHODS: Following a literature review, a novel Value of Vaccination (VoV) framework was developed with experts in vaccine EE from developed countries with established health technology assessment systems. RESULTS: Forty-four studies presenting value frameworks or concepts applicable to vaccination were included. Eighteen unique value concepts relevant to EE were identified and defined. These were categorised within the VoV framework using three dimensions, moving from a narrow payer perspective to a more expansive and societal perspective. The dimensions were: (I) conventional payer perspective concepts (e.g., health gains in vaccinees, direct medical costs); (II) conventional societal perspective concepts (e.g., indirect health/economic gains to caregivers/households, productivity in vaccinees); and (III) novel societal concepts (e.g., financial risk protection, peace of mind, societal health gains, healthcare systems security, political stability, social equity and macroeconomic gains). While good quality evidence and methods are available to support concepts in Dimensions I and II, further work is needed to generate the required evidence for vaccination impact on Dimension III concepts. CONCLUSIONS: The devastating effect on nations of the COVID-19 pandemic has helped to highlight the potential far-reaching benefits that many vaccination programmes can offer. This VoV framework is particularly relevant to policy decisions considering EE, and the potential future expansion of non-pandemic vaccination value considerations. The framework helps to understand and compare current value considerations across countries and payer versus societal perspectives. It provides decision-makers with a transparent and logical path to broaden consideration of VoV in EE.

Preference Elicitation Techniques Used in Valuing Children's Health-Related Quality-of-Life: A Systematic Review.

BACKGROUND AND OBJECTIVES: Valuing children's health states for use in economic evaluations is globally relevant and is of particular relevance in jurisdictions where a cost-utility analysis is the preferred form of analysis for decision making. Despite this, the challenges with valuing child health mean that there are many remaining questions for debate about the approach to elicitation of values. The aim of this paper was to identify and describe the methods used to value children's health states and the specific issues that arise in the use of these methods. METHODS: We conducted a systematic search of electronic databases to identify studies published in English since 1990 that used preference elicitation methods to value child and adolescent (under 18 years of age) health states. Eligibility criteria comprised valuation studies concerning both child-specific patient-reported outcome measures and child health states defined in other ways, and methodological studies of valuation approaches that may or may not have yielded a value set algorithm. RESULTS: A total of 77 eligible studies were identified from which data on country setting, aims, condition (general population or clinically specific), sample size, age of respondents, the perspective that participants were asked to adopt, source of values (respondents who completed the preference elicitation tasks) and methods questions asked were extracted. Extracted data were classified and evaluated using narrative synthesis methods. The studies were classified into three groups: (1) studies comparing elicitation methods (n = 30); (2) studies comparing perspectives (n = 23); and (3) studies where no comparisons were presented (n = 26); selected studies could fall into more than one group. Overall, the studies varied considerably both in methods used and in reporting. The preference elicitation tasks included time trade-off, standard gamble, visual analogue scaling, rating/ranking, discrete choice experiments, best-worst scaling and willingness to pay elicited through a contingent valuation. Perspectives included adults' considering the health states from their own perspective, adults taking the perspective of a child (own, other, hypothetical) and a child/adolescent taking their own or the perspective of another child. There was some evidence that children gave lower values for comparable health states than did adults that adopted their own perspective or adult/parents that adopted the perspective of children. CONCLUSIONS: Differences in reporting limited the conclusions that can be formed about which methods are most suitable for eliciting preferences for children's health and the influence of differing perspectives and values. Difficulties encountered in drawing conclusions from the data (such as lack of consensus and poor reporting making it difficult for users to choose and interpret available values) suggest that reporting guidelines are required to improve the consistency and quality of reporting of studies that value children's health using preference-based techniques.

Capturing the value of vaccination within health technology assessment and health economics: Country analysis and priority value concepts.

BACKGROUND: A value of vaccination framework for economic evaluation (EE) identified unique value concepts for the broad benefits vaccination provides to individuals, society, healthcare systems and national economies. The objectives of this paper were to work with experts in developed countries to objectively identify three priority concepts to extend current EE. METHODS: The previously developed classification of value concepts in vaccination distinguished 18 concepts, categorised as conventional payer and societal perspective concepts and novel broader societal concepts. Their inclusion in current EE guidelines was assessed. Experts identified eight criteria relevant to decision-making and measurement feasibility, which were weighted and used to score each concept. The relative ranking of concepts by importance and the gaps in guidelines were used to identify three priority concepts on which to focus immediate efforts to extend EE. RESULTS: The EE guidelines review highlighted differences across countries and between guidelines and practice. Conventional payer perspective concepts (e.g., individual and societal health gains and medical costs) were generally included, while gaps were evident for conventional societal perspective concepts (e.g., family/caregiver health and economic gains). Few novel broader societal benefits were considered, and only in ad hoc cases. The top-three concepts for near-term consideration: macroeconomic gains (e.g., benefiting the economy, tourism), social equity and ethics (e.g., equal distribution of health outcomes, reduced health/financial equity gaps) and health systems strengthening, resilience and security (e.g., efficiency gains, reduced disruption, increased capacity). CONCLUSIONS: Gaps, inconsistencies and limited assessment of vaccination value in EE can lead to differences in policy and vaccination access. The three priority concepts identified provide a feasible approach for capturing VoV more broadly in the near-term. Robust methods for measuring and valuing these concepts in future assessments will help strengthen the evidence used to inform decisions, improving access to vaccines that are demonstrably good value for money from society's point of view.

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.

Systematic Review of Conceptual, Age, Measurement and Valuation Considerations for Generic Multidimensional Childhood Patient-Reported Outcome Measures.

BACKGROUND AND AIMS: Patient-reported outcome measures (PROMs) for children (aged ≤ 18 years) present methodological challenges. PROMs can be categorised by their diverse underlying conceptual bases, including functional, disability and health (FDH) status; quality of life (QoL); and health-related quality of life (HRQoL). Some PROMs are designed to be accompanied by preference weights. PROMs should account for childhood developmental differences by incorporating age-appropriate health/QoL domains, guidance on respondent type(s) and design. This systematic review aims to identify generic multidimensional childhood PROMs and synthesise their characteristics by conceptual basis, target age, measurement considerations, and the preference-based value sets that accompany them. METHODS: The study protocol was registered in the Prospective Register of Systematic Reviews (CRD42021230833), and reporting followed Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. We conducted systematic database searches for generic multidimensional childhood PROMs covering the period 2012-2020, which we combined with published PROMs identified by an earlier systematic review that covered the period 1992-2011. A second systematic database search identified preference-based value sets for generic multidimensional PROMs. The PROMs were categorised by conceptual basis (FDH status, QoL and HRQoL) and by target age (namely infants and pre-schoolers aged < 5 years, pre-adolescents aged 5-11, adolescents aged 12-18 and multi-age group coverage). Descriptive statistics assessed how PROM characteristics (domain coverage, respondent type and design) varied by conceptual basis and age categories. Involvement of children in PROM development and testing was assessed to understand content validity. Characteristics of value sets available for the childhood generic multidimensional PROMs were identified and compared. RESULTS: We identified 89 PROMs, including 110 versions: 52 FDH, 29 QoL, 12 HRQoL, nine QoL-FDH and eight HRQoL-FDH measures; 20 targeted infants and pre-schoolers, 29 pre-adolescents, 24 adolescents and 37 for multiple age groups. Domain coverage demonstrated development trajectories from observable FDH aspects in infancy through to personal independence and relationships during adolescence. PROMs targeting younger children relied more on informant report, were shorter and had fewer ordinal scale points. One-third of PROMs were developed following qualitative research or surveys with children or parents for concept elicitation. There were 21 preference-based value sets developed by 19 studies of ten generic multidimensional childhood PROMs: seven were based on adolescents' stated preferences, seven were from adults from the perspective of or on behalf of the child, and seven were from adults adopting an adult's perspective. Diverse preference elicitation methods were used to elicit values. Practices with respect to anchoring values on the utility scale also varied considerably. The range and distribution of values reflect these differences, resulting in value sets with notably different properties. CONCLUSION: Identification and categorisation of generic multidimensional childhood PROMs and value sets by this review can aid the development, selection and interpretation of appropriate measures for clinical and population research and cost-effectiveness-based decision-making.

How are Child-Specific Utility Instruments Used in Decision Making in Australia? A Review of Pharmaceutical Benefits Advisory Committee Public Summary Documents.

Measuring and valuing health-related quality of life (HRQOL) in children can be challenging but is an important component for providing decision makers with accurate information to fund new interventions, including medicines and vaccines for public subsidy. We review funding submissions of medicines made to the Pharmaceutical Benefits Advisory Committee contained in public summary documents to examine the use of child-specific HRQOL measures in decision making in Australia. A sample frame of medicines used by children was derived from four sources. Public summary documents relating to these medicines were searched in the Pharmaceutical Benefits Advisory Committee web resources for whether they related to children (aged under 18 years) and contained HRQOL information and/or cost-utility analyses. Data about the use of utilities in decision making were extracted and analysed. Of the 1889 public summary documents available, 62 public summary documents (29 medicines) contained information pertaining to children and utilities. Of these, four public summary documents included child-specific HRQOL measures, 16 included adult HRQOL measures, 11 included direct elicitation and the HRQOL source was not defined in 31 documents. Excluding documents using child-specific HRQOL measures, we considered that in 85% of medicines, decision making uncertainty might have been reduced by using child-specific HRQOL measures. Despite the growing literature on economic analysis in paediatric populations, the use of child-specific HRQOL measures in submissions to the Pharmaceutical Benefits Advisory Committee was minimal. Submissions involved inconsistent approaches, use of adult measures and weights, and substantial gaps in evidence. We recommend the consistent use of child-specific measures to improve the evidence base for decisions about medicines for children in Australia.