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Purpose: To identify utility-based patient-reported outcome measures (PROMs) for assessing health-related quality of life (HRQoL) in cardiac rehabilitation and secondary prevention programs (CR) and appraise existing evidence on their measurement properties. Secondly, to link their items to the International Classification of Functioning Disability and Health (ICF) and the International Consortium of Health Outcome Measures (ICHOM) domains for cardiovascular disease (CVD). Methods: Eight databases were searched. The review followed the COSMIN and JBI guidelines for measurement properties systematic reviews and PRISMA 2020 reporting guidelines. Non-experimental and observational empirical studies of patients ≥ 18 years of age with CVD undergoing CR and assessed quality of life (QoL) or HRQoL using utility-based PROMs or one accompanied by health state utilities were included. Results: Nine PROMs were identified with evidence on measurement properties for three measures: the German translations of SF-12, EQ-5D-5L, and MacNew heart disease HRQoL questionnaire. There was moderate quality evidence for responsiveness and hypothesis testing of the SF-12 and EQ-5D-5L, and high-quality evidence for responsiveness and hypothesis testing for the MacNew. All items of SF-12 and EQ-5D were linked to ICF categories, but four items of the MacNew were not classified or defined. All the PROM domains were mapped onto similar constructs from the ICHOM global sets. Conclusion: Three utility-based PROMs validated in CR were identified: the German versions of the EQ-5D and SF-12 and the MacNew questionnaire. These PROMs are linked to a breadth of ICF categories and all ICHOM global sets. Additional validation studies of PROMs in CR are required. [ABSTRACT FROM AUTHOR]

Purpose: In many countries, there are calls to address health inequalities experienced by Indigenous people. Preference-based measures (PBMs) provide a measurement of health-related quality of life and can support resource allocation decisions. This review aimed to identify, summarize, and appraise the literature reporting the use and performance of PBMs with Indigenous people. Methods: Eleven major databases were searched from inception to August 31, 2022. Records in English that (1) assessed any measurement property of PBMs, (2) directly elicited health preferences, (3) reported the development or translation of PBMs for Indigenous people, or (4) measured health-related quality of life (HRQL) using PBMs were included. Ethically engaged research with Indigenous people was considered as an element of methodological quality. Data was synthesized descriptively (PROSPERO ID: CRD42020205239). Results: Of 3139 records identified, 81 were eligible, describing psychometric evaluation (n = 4), preference elicitation (n = 4), development (n = 4), translation (n = 2), and HRQL measurement (n = 71). 31 reported ethically engaged research. Reports originated primarily from Australia (n = 38), New Zealand (n = 20), USA (n = 9) and Canada (n = 6). Nearly all (n = 73) reported indirect, multi-attribute PBMs, the most common of which was the EQ-5D (n = 50). Conclusion: A large number of recent publications from diverse disciplines report the use of PBMs with Indigenous people, despite little evidence on measurement properties in these populations. Understanding the measurement properties of PBMs with Indigenous people is important to better understand how these measures might, or might not, be used in policy and resource decisions affecting Indigenous people. (Funding: EuroQoL Research Foundation). [ABSTRACT FROM AUTHOR]

Abstract Background The PROMIS Preference score (PROPr) is a new health state utility (HSU) score that aims to comprehensively incorporate the biopsychosocial model of health and apply favorable psychometric properties from the descriptive PROMIS system to HSU measurements. However, minimal evidence concerning comparisons to the EQ-5D-3L and the PROPr’s capability to differentiate clinical severity are available. Therefore, the aim of this study was to compare the PROPr to the EQ-5D-3L in terms of scale agreement, ceiling/floor effects, distribution, construct validity, discriminatory power, and relative efficiency (RE) in terms of the Oswestry Disability Index (ODI) for patients with low back pain (LBP). Methods We used intra-class correlation coefficients (ICC) and Bland–Altman plots to compare the PROPr and EQ-5D-3L with regared to scale agreement in a cross-sectional routine sample of LBP patients. For distribution, we used the Pearson’s coefficient for skewness and for ceiling/floor effects, a 15%-top/bottom threshold. For convergent validity, we used Pearson’s correlation coefficients. For known-groups validity, we applied a linear regression with interaction terms (predictors sex, age, and ODI level) and an analysis of variance (ANOVA). For discriminatory power, we calculated the effect size (ES) using Cohen’s d and the ratio of the area under the receiver-operating characteristics curves (AUROC-ratio = AUROCPROPr/AUROCEQ-5D-3L). RE was measured using the ratio of F-values (RE = FPROPr/FEQ-5D-3L). Results Of 218 LBP patients, 50.0% were female and the mean age was 61.8 years. The mean PROPr (0.20, 95%CI: 0.18; 0.22) and EQ-5D-3L scores (0.55, 95%CI: 0.51; 0.58) showed low agreement (d = 0.35, p ESPROPr and RE 1 in lower ODI severity. AUROC-ratios did not show significant differences in terms of ODI severity. Conclusions All PROPr and EQ-5D-3L biopsychosocial dimensions of health showed impairment in LPB patients. The capability of EQ-5D-3L and PROPr to differentiate ODI levels depends on ODI severity. Joint application of both tools may provide additional information.

This study assessed the content validity of generic and condition-specific preference-based measures (PBMs) with patients treated for cancer, evaluated against 10 Consensus-Based Standards for the Selection of Health Measurement Instruments criteria for good content validity, to best inform measurement strategies regarding the use of PBMs in oncology development programs and real-world applications. Individual, semistructured interviews were conducted with patients who received drug treatment for cancer in the United Kingdom (n = 47) and the United States (n = 49). During the interview, patients completed 3 generic PBMs (EQ-5D-5L, EuroQol Health and Wellbeing measure–Short Form, Château Santé Base) and 2 condition-specific PBMs (Quality of Life Utility-Core 10 Dimension, Functional Assessment of Cancer Therapy Eight Dimension [FACT-8D]). Interviews were conducted via teleconference, audio recorded, and transcribed verbatim. Transcripts were coded using thematic and content analysis methods. Condition-specific measures were evaluated as having better relevancy than generic PBMs. Overall, the FACT-8D was evaluated as holding the best content validity in terms of relevancy, and the EuroQol Health and Wellbeing measure–Short Form received the most favorable evaluation of relevancy for generic PBMs. All measures demonstrated comparable comprehensiveness, with all suggested by patients to be missing concepts. The EQ-5D-5L was evaluated best in terms of comprehensibility. This was followed by the Quality of Life Utility-Core 10 Dimension and FACT-8D; both received similar evaluations. All measures were generally seen by patients as adequate in capturing appropriate aspects of health-related quality of life for measuring cancer outcomes, although together condition-specific measures were evaluated as having better relevancy than generic PBMs. Further health-related quality of life instrument development is encouraged, particularly with regard to the longer-term detrimental impacts of cancer and treatment side effects. Other developments could include new cancer-specific tools inclusive of conventional health items, treatment impacts, and psychological items. • Generic preference-based measures (PBMs) are recommended to assess patient-reported outcomes across interventions and to allow for comparative assessments across unrelated health conditions and interventions. Generic PBMs may exclude dimensions relevant to specific conditions, whereas condition-specific PBMs can contain items related to specific symptoms. Recent work has identified several concepts that are relevant to health-related quality of life and psychological wellbeing, which are not measured by existing outcome measures. • Patients who had received drug treatment for cancer in the United Kingdom and the United States evaluated 3 generic and 2 condition-specific PBMs during qualitative interviews. All measures demonstrated comparable comprehensiveness, missing concepts considered by patients as important to measure for their cancer and cancer treatment experiences. Language and format were frequently challenged, with patients suggesting changes to multiple items across all measures. Recall periods were frequently raised as problematic because of the substantial variation in cancer symptoms within a given day or week. • Given the burden of cancer symptoms and drug treatment effects, capturing all relevant outcomes of people with cancer is necessary to inform accurate decision making for cancer technologies. Modification of existing measures should be considered with regard to measurement gaps identified. New cancer-specific tools could be considered, inclusive of conventional health items, treatment impacts, and psychological items. These developments have a large potential for impact given the high proportions of oncology health technology assessment submissions. [ABSTRACT FROM AUTHOR]

Purpose: Identify aspects of quality of life (QoL) important to Australian informal carers and explore how well the Adult Social Care Outcomes Toolkit for Carers, Care-related Quality of Life instrument and Carer Experience Scale capture these aspects in the Australian context. Methods: Online questionnaires were completed by Australian informal carers. Socio-demographics, open-ended questions: positive/negative aspects of caring and QoL aspects missing from the instruments, and ranking of the instrument domains was used to explore the content of the instruments. Instruments were scored using preference-weighted value sets (reported in another paper). Content analysis was used to analyse the open-ended responses. Chi-squared test looked at differences in domain importance. Descriptive analyses summarised all other information. Results: Eight themes were identified: Behaviour-mood of the care recipient, Caring responsibilities, Finances, Health, Own life, Perception of carers, Relationship with care recipient and Support. Many aspects of carer QoL mentioned as missing in the instruments appeared covered by the domains, of which all were reported as important. The highest ranked domain was relationship with the care recipient. The influence of the care recipient specific support, behaviour/mood and health on carer QoL appear absent in all instruments. Conclusion: The content of the three instruments appears relevant in an Australian setting. The influence of care recipient's health and well-being on carer QoL should be considered, along with spillover effects. A content and/or face validity analysis is required to confirm differences in item interpretation in Australian informal carers. [ABSTRACT FROM AUTHOR]

Background: The PROMIS Preference score (PROPr) is a new health state utility (HSU) score that aims to comprehensively incorporate the biopsychosocial model of health and apply favorable psychometric properties from the descriptive PROMIS system to HSU measurements. However, minimal evidence concerning comparisons to the EQ-5D-3L and the PROPr's capability to differentiate clinical severity are available. Therefore, the aim of this study was to compare the PROPr to the EQ-5D-3L in terms of scale agreement, ceiling/floor effects, distribution, construct validity, discriminatory power, and relative efficiency (RE) in terms of the Oswestry Disability Index (ODI) for patients with low back pain (LBP). Methods: We used intra-class correlation coefficients (ICC) and Bland–Altman plots to compare the PROPr and EQ-5D-3L with regared to scale agreement in a cross-sectional routine sample of LBP patients. For distribution, we used the Pearson's coefficient for skewness and for ceiling/floor effects, a 15%-top/bottom threshold. For convergent validity, we used Pearson's correlation coefficients. For known-groups validity, we applied a linear regression with interaction terms (predictors sex, age, and ODI level) and an analysis of variance (ANOVA). For discriminatory power, we calculated the effect size (ES) using Cohen's d and the ratio of the area under the receiver-operating characteristics curves (AUROC-ratio = AUROCPROPr/AUROCEQ-5D-3L). RE was measured using the ratio of F-values (RE = FPROPr/FEQ-5D-3L). Results: Of 218 LBP patients, 50.0% were female and the mean age was 61.8 years. The mean PROPr (0.20, 95%CI: 0.18; 0.22) and EQ-5D-3L scores (0.55, 95%CI: 0.51; 0.58) showed low agreement (d = 0.35, p < 0.001; ICC 0.27, 95%CI: -0.09; 0.59). The PROPr's distribution was positively skewed, whereas the EQ-5D-3L's was negative. Neither tool showed ceiling/floor effects, but all EQ-5D-3L dimensions did. Pearson correlation was r = 0.66 (95%CI: 0.58; 0.73). Differences were invariant to sex and age but not to ODI severity: ESEQ-5D-3L > ESPROPr and RE < 1 in higher ODI severity; ESEQ-5D-3L < ESPROPr and RE > 1 in lower ODI severity. AUROC-ratios did not show significant differences in terms of ODI severity. Conclusions: All PROPr and EQ-5D-3L biopsychosocial dimensions of health showed impairment in LPB patients. The capability of EQ-5D-3L and PROPr to differentiate ODI levels depends on ODI severity. Joint application of both tools may provide additional information. [ABSTRACT FROM AUTHOR]

This study aimed to develop a scoring function to calculate health utilities for health states described by the Health Utility for Glaucoma (HUG-5) based on the preferences of the general population in the United States. Preferences for HUG-5 health states were elicited using the standard gamble and visual analog scale through an online survey. Quota-based sampling was used to recruit a representative sample of the US general population in terms of age, sex, and race. A multiple attribute disutility function (MADUF) approach was adopted to derive scoring for the HUG-5. Model fit was assessed using mean absolute error associated with 5 HUG-5 marker health states that describe mild/moderate and severe glaucoma. Of 634 respondents completing the tasks, 416 were included in the estimation of the MADUF; 260 respondents (63%) considered worst possible HUG-5 health state better than death. The preferred scoring function generates the utilities ranging from 0.05 (worst HUG-5 health state) to 1 (best HUG-5 health state). The correlation between mean elicited and estimated values for marker states was strong (R 2 = 0.97) with mean absolute error = 0.11. The MADUF for HUG-5 is used to measure health utilities on the scale of perfect health and death, which can be used to estimate quality-adjusted life-years for economic evaluations of glaucoma interventions. [ABSTRACT FROM AUTHOR]

Abstract Background There is increasing interest in the validation of pediatric preference-based health-related quality of life measurement instruments. It is critical that children with various degrees of health-related quality of life (HRQoL) impact are included in validation studies. To inform patient sample selection for validation studies from a pragmatic perspective, this study explored HRQoL impairments between known-groups and HRQoL changes over time across 27 common chronic child health conditions and identified conditions with the largest impact on HRQoL. Methods The health dimensions of two common preference-based HRQoL measures, the EQ-5D-Y and CHU9D, were constructed using Pediatric Quality of Life Inventory items that overlap conceptually. Data was from the Longitudinal Study of Australian Children, a nationally representative sample with over 10,000 children at baseline. Seven waves of data were included for the analysis, with child age ranging from 2 to18 years. Impacts to specific health dimensions and overall HRQoL between those having a specific condition versus not were compared using linear mixed effects models. HRQoL changes over time were obtained by calculating the HRQoL differences between two consecutive time points, grouped by “Improved” and “Worsened” health status. Comparison among various health conditions and different age groups (2–4 years, 5–12 years and 13–18 years) were made. Results Conditions with the largest statistically significant total HRQoL impairments of having a specific condition compared with not having the condition were recurrent chest pain, autism, epilepsy, anxiety/depression, irritable bowel, recurrent back pain, recurrent abdominal pain, and attention deficit hyperactivity disorder (ADHD) for the total sample (2–18 years). Conditions with largest HRQoL improvement over time were anxiety/depression, ADHD, autism, bone/joint/muscle problem, recurrent abdominal pain, recurrent pain in other part, frequent headache, diarrhea and day-wetting. The dimensions included in EQ-5D-Y and CHU9D can generally reflect HRQoL differences and changes. The HRQoL impacts to specific health dimensions differed by condition in the expected direction. The conditions with largest HRQoL impacts differed by age group. Conclusions The conditions with largest HRQoL impact were identified. This information is likely to be valuable for recruiting patient samples when validating pediatric preference-based HRQoL instruments pragmatically.

Purpose: This study aimed to elicit willingness to pay (WTP) values for fluoride varnish application from participants using the publically-funded health services in Brazil and the UK, and to identify differences in the variables impacting these values. A secondary aim was to compare WTP values from parents for their own preventive treatment and their child's.Materials and Methods: This was a cross-sectional analysis of quantitative data collected from participants attending routine dental appointments. The clinics were hosted by the Pontifical Catholic University of Rio Grande do Sul (PUCRS), Brazil and Newcastle Dental Hospital, UK.Results: The mean WTP for Brazilian adults was R$60.37 (=£15.97). WTP was highly variable and factors affecting it were difficult to identify. UK parents valued fluoride varnish at mean values of £28.21 and £28.12 for themselves and their child, respectively. Regression modelling found those with higher incomes had higher WTP in both samples. In the UK, parental and child WTP increased when parents had higher self-perceived need for dental treatment, had experienced recent dental pain, or their child had received restorations in the last 2 years.Conclusions: WTP for fluoride varnish varied dramatically between individuals. In both countries, it was difficult to predict this variance, as factors which would likely impact upon on WTP had a limited effect and were sometimes counter-intuitive. WTP values for a parent and their child were not statistically significantly different. [ABSTRACT FROM AUTHOR]

Mapping algorithms are developed using data sets containing patient responses to a preference-based questionnaire and another health-related quality-of-life questionnaire. When data sets include repeated measurements from the same individuals over time, the assumption of observations' independence, required by standard models, is violated, and standard errors are underestimated. This review aimed to identify how studies deal with methodological challenges of repeated measurements, provide an overview of practice to date, and potential implications for future work. We conducted a systematic literature search of MEDLINE, Cumulative Index to Nursing and Allied Health Literature, specialized databases, and previous systematic reviews. A data template was used to extract, among others, start and target instruments if the data set(s) used for estimation and validation had repeated measurements per patient, used regression techniques, and which (if any) adjustments were made for repeated measurements. We identified 278 publications developing at least 1 mapping algorithm. Of the 278 publications, 121 used a data set with repeated measurements, among which 92 used multiple time points for estimation, and 39 selected specific time points to have 1 observation per participant. A total of 36 studies did not account for repeated measurements. An adjustment was conducted using cluster-robust standard errors (21), random-effects models (30), generalized estimating equations (7), and other methods (7). The inconsistent use of methods to account for interdependent observations in the literature indicates that mapping guidelines should include recommendations on how to deal with repeated measurements, and journals should update their guidelines accordingly. • Authors often use data sets with repeated measurements per individual over time to develop their mapping algorithms. Nevertheless, our review suggests that many authors do not account for the interdependence of data or do not clearly describe the methods used. • This updated systematic review of mapping articles found that, when data sets with repeated measurements are used, authors often only use 1 time point in the estimation data set and another time point for its validation, hence ignoring that health states with different levels of severity may be present only at a specific time point. • When multiple time points are used, often the methodological implications are not explicitly considered; this means that variance-covariance matrices from these models—often used in probabilistic sensitivity analyses—are not accurate. Mapping guidelines should expand on how to deal with repeated measurements, and journals should make sure that these updated guidelines are followed. [ABSTRACT FROM AUTHOR]

Background: There is increasing interest in the validation of pediatric preference-based health-related quality of life measurement instruments. It is critical that children with various degrees of health-related quality of life (HRQoL) impact are included in validation studies. To inform patient sample selection for validation studies from a pragmatic perspective, this study explored HRQoL impairments between known-groups and HRQoL changes over time across 27 common chronic child health conditions and identified conditions with the largest impact on HRQoL. Methods: The health dimensions of two common preference-based HRQoL measures, the EQ-5D-Y and CHU9D, were constructed using Pediatric Quality of Life Inventory items that overlap conceptually. Data was from the Longitudinal Study of Australian Children, a nationally representative sample with over 10,000 children at baseline. Seven waves of data were included for the analysis, with child age ranging from 2 to18 years. Impacts to specific health dimensions and overall HRQoL between those having a specific condition versus not were compared using linear mixed effects models. HRQoL changes over time were obtained by calculating the HRQoL differences between two consecutive time points, grouped by "Improved" and "Worsened" health status. Comparison among various health conditions and different age groups (2–4 years, 5–12 years and 13–18 years) were made. Results: Conditions with the largest statistically significant total HRQoL impairments of having a specific condition compared with not having the condition were recurrent chest pain, autism, epilepsy, anxiety/depression, irritable bowel, recurrent back pain, recurrent abdominal pain, and attention deficit hyperactivity disorder (ADHD) for the total sample (2–18 years). Conditions with largest HRQoL improvement over time were anxiety/depression, ADHD, autism, bone/joint/muscle problem, recurrent abdominal pain, recurrent pain in other part, frequent headache, diarrhea and day-wetting. The dimensions included in EQ-5D-Y and CHU9D can generally reflect HRQoL differences and changes. The HRQoL impacts to specific health dimensions differed by condition in the expected direction. The conditions with largest HRQoL impacts differed by age group. Conclusions: The conditions with largest HRQoL impact were identified. This information is likely to be valuable for recruiting patient samples when validating pediatric preference-based HRQoL instruments pragmatically. [ABSTRACT FROM AUTHOR]

Objectives: Preference‐based quality‐of‐life measures (PBMs) have been developed in many clinical areas to aid estimation of more accurate utility values for economic evaluations. Existing oral health‐related quality‐of‐life (OHRQoL) instruments are non‐PBM and hence, cannot be used to generate utility values. The objective of this study was to develop a classification system for a new PBM (dental caries utility index‐DCUI) for the most prevalent childhood oral health condition: dental caries. Methods: Possible domains and items to be included in the classification system were identified based on the reviewing available pediatric non‐PBM OHRQoL instruments, studies eliciting utility values for oral health outcomes and clinical dentistry textbooks and based on the findings, a draft classification system was developed. To refine the draft classification system, semi‐structured interviews were conducted among a convenience sample of 15 12–17‐year‐old adolescents who had experience with dental caries. The classification system was further refined and validated by a group of dental experts, using a modified Delphi technique. Results: The classification system comprised five items (pain/discomfort, difficulty in eating food/drinking, worried, ability to participate in activities, and appearance) and each item had a four‐level response scale. Conclusion: The classification system developed herein is considered an amenable tool for the subsequent development of a new PBM for dental caries. Once the scoring algorithm is completed, the classification system can be used to incorporate economic evaluations of dental caries health interventions. [ABSTRACT FROM AUTHOR]

Objective: The objective of this study was to evaluate the construct validity of 2 health utility instruments—the EuroQoL–5 Dimension (EQ-5D) and the Health Utilities Index–Mark 3 (HUI-3)—and to compare them with disease-specific measures in patients with head and neck cancer. Study Design: Prospective cross-sectional analysis. Setting: Princess Margaret Cancer Centre. Methods: Patients were administered the EQ-5D, HUI-3, the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) and its head and neck cancer module (EORTC QLQ-H&N35), and the University of Washington Quality of Life Questionnaire (UWQoL). Several a priori expected relations were examined. The correlative and discriminative properties of the various instruments were examined. Results: A total of 209 patients completed the 4 questionnaires. A significant ceiling effect was observed among EQ-5D responses (23% reported a maximum score of 1). The EQ-5D (rho = 0.79) and HUI-3 (rho = 0.60) had a strong correlation with the social-emotional domain of the UWQoL. The EQ-5D had a moderate correlation with the physical domain of the UWQoL (rho = 0.42), whereas the HUI-3 had a weak correlation (rho = 0.29). The EQ-5D and HUI-3 were able to distinguish among levels of health severity measured on the EORTC QLQ-C30 though not the QLQ-H&N35. Comparatively, the UWQoL was able to distinguish levels of disease severity on the EORTC QLQ-C30 and QLQ-H&N35. Conclusion: The results of this study demonstrate that disease-specific domains from head and neck quality-of-life instruments are not strongly correlated with the EQ-5D and HUI-3. Consideration should be put toward development of a disease-specific preference-based measure for health economic evaluation. Level of evidence: 4. [ABSTRACT FROM AUTHOR]

Abstract Background Generic preference-based measures (GPBMs) are health-related quality of life (HRQoL) measures commonly used to evaluate the cost-utility of interventions in healthcare. However, the degree to which the content of GPBMs reflect the HRQoL of individuals with chronic obstructive pulmonary disease (COPD) has not yet been assessed. The purpose of this study was to examine the content and convergent validity of GPBMs in people with COPD. Methods COPD patients were recruited from healthcare centers in Ontario, Canada. The Patient-Generated Index (PGI) (an individualized HRQoL measure) and the RAND-36 (to obtain SF-6D scores; a GPBM) were administered. Life areas nominated with the PGI were coded using the International Classification of Functioning Disability and Health and mapped onto GPBMs. Results We included 60 participants with a mean age of 70 and FEV1% predicted of 43. The mean PGI score was 34.55/100 and the top three overarching areas that emerged were: ‘mobility’ (25.93%), ‘recreation and leisure’ (25.19%) and ‘domestic life’ (19.26%). Mapping of the nominated areas revealed that the Quality of Well-Being scale covered the highest number of areas (84.62%), Health Utilities Indices covered the least (15.38% and 30.77%) and other GPBMs covered between 46 and 62%. A correlation of 0.32 was calculated between the SF-6D and the PGI. Conclusions The majority of GPBMs covered approximately half of the areas reported as being important to individuals with COPD. When areas relevant to COPD are not captured, HRQoL scores generated by these measures may inaccurately reflect patients’ values and affect cost-effectiveness decisions.

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. [ABSTRACT FROM AUTHOR]

Objectives: To identify the themes to inform the content of a new generic measure, the EQ-HWB (EQ Health and Wellbeing), that can be used in economic evaluation across health, social care, and public health, based on the views of users and beneficiaries of these services including informal carers.Methods: A qualitative review was undertaken. Systematic and citation searches were undertaken focusing on qualitative evidence of the impact on quality of life from reviews for selected health conditions, informal carers, social care users, and primary qualitative work used in the development of selected measures. A subset of studies was included in the review. Framework analysis and synthesis were undertaken based on a conceptual model.Results: A total of 42 reviews and 24 primary studies were selected for inclusion in the review. Extraction and synthesis resulted in 7 high-level themes (with subthemes): (1) feelings and emotions (sadness, anxiety, hope, frustration, safety, guilt/shame); (2) cognition (concentration, memory, confusion, thinking clearly); (3) self-identity (dignity/respect, self-esteem); (4) "coping, autonomy, and control" relationships; (5) social connections (loneliness, social engagement, stigma, support, friendship, belonging, burden); (6) physical sensations (pain, discomfort, sleep, fatigue); and (7) activity (self-care, meaningful activities, mobility, communication, hearing, vision). Apart from physical sensations, most of the other themes and subthemes were relevant across both health and social care, including for informal carers.Conclusions: The findings from this broad review identified themes that go beyond health and that are relevant to patients, informal carers, and social care users. The themes and subthemes informed the domains for the EQ-HWB. [ABSTRACT FROM AUTHOR]

Abstract Background Assessing the cost-effectiveness of interventions for people with dementia, based on cost per quality-adjusted life years (QALYs) gained, requires that the measures used to derive QALYs are preference-based whilst also being valid, feasible to use, comprehensible and acceptable for people with dementia. The aim of this study was to assess the content and face validity of six preference-based measures (PBMs) within the context of dementia. Methods Qualitative focus groups and interviews were conducted with community-dwelling individuals with mild dementia and carers of people with dementia. After exploring participants’ understanding of ‘quality of life’ (QoL), six PBMs were assessed for content and face validity: two measures assessing health-related QoL (EQ-5D-5L and AQoL-8D); two covering broader aspects of capability wellbeing and social care-related QoL (ICECAP-O and ASCOT); and two dementia-specific QoL measures (DEMQOL-U and AD-5D). A random mix of one health-related QoL measure, one wellbeing measure, and one dementia-specific measure was explored in each session. All sessions were audiotaped and transcribed verbatim. Data were analysed thematically. Results Nine individuals with mild dementia and 17 carers of people with dementia participated across 4 focus groups and 10 interviews. Participants perceived 9 broad QoL domains as relevant to them: Activity, Autonomy, Cognition, Communication, Coping, Emotions, End-of-Life, Physical Functioning, and Relationships. These domains had limited overlap with the content of the six PBMs. Assessment of face validity was summarized into eight themes: (1) ambiguous questions, (2) double –barrelled questions, (3) difficult/abstract questions, (4) judgemental/confronting questions, (5) lack of relevance and comprehensiveness, (6) response options, (7) layout/format and (8) proxy-response. There was no clear preference for one of the six measures explored; participants identified advantages and disadvantages across all measures. Although particularly designed for individuals with dementia, dementia-specific QoL measures were not always favoured over non-specific measures. Conclusion Given the shortcomings of PBMs identified in this study, further empirical comparative analyses are necessary to guide the selection of PBMs for future dementia research.

Background: Assessing health-related quality of life in dementia poses challenges due to patients' cognitive impairment. It is unknown if the newly introduced EQ-5D five-level version (EQ-5D-5L) is superior to the 3-level version (EQ-5D-3L) in this cognitively impaired population group.Objective: To assess the psychometric properties of the EQ-5D-5L in comparison to the EQ-5D-3L in patients living with dementia (PwD).Methods: The EQ-5D-3L and EQ-5D-5L were assessed via interviews with n = 78 PwD at baseline and three and six months after, resulting in 131 assessments. The EQ-5D-3L and EQ-5D-5L were evaluated in terms of acceptability, agreement, ceiling effects, redistribution properties and inconsistency, informativity as well as convergent and discriminative validity.Results: Mean index scores were higher for the EQ-5D-5L than the EQ-5D-3L (0.70 versus 0.64). Missing values occurred more frequently in the EQ-5D-5L than the EQ-5D-3L (8%versus 3%). Agreement between both measures was acceptable but poor in PwD with moderate to severe cognitive impairment. The index value's relative ceiling effect decreased from EQ-5D-3L to EQ-5D-5L by 17%. Inconsistency was moderate to high (13%). Absolute and relative informativity increased in the EQ-5D-5L compared to the 3L. The EQ-5D-5L demonstrated a lower discriminative ability and convergent validity, especially in PwD with moderate to severe cognitive deficits.Conclusion: The EQ-5D-5L was not superior as a self-rating instrument due to a lower acceptability and discriminative ability and a high inconsistency, especially in moderate to severe dementia. The EQ-5D-3L had slightly better psychometric properties and should preferably be used as a self-rating instrument in economic evaluations in dementia. [ABSTRACT FROM AUTHOR]

PurposeDocumenting the impact of different types of cancer on daily functioning and well-being is important for understanding burden relative to other chronic medical conditions. This study examined the impact of 10 different cancers and 13 other chronic medical conditions on health-related quality of life.MethodsHealth-related quality of life data were gathered on the Medicare Health Outcomes Survey (MHOS) between 1998 and 2002. Cancer information was ascertained using the National Cancer Institute's surveillance, epidemiology, and end results program and linked to MHOS data.ResultsThe average SF-6D score was 0.73 (SD = 0.14). Depressive symptoms had the largest unique association with the SF-6D, followed by arthritis of the hip, chronic obstructive pulmonary disease/asthma, stroke, and sciatica. In addition, the majority of cancer types were significantly associated with the SF-6D score, with significant negative weights ranging from -0.01 to -0.02 on the 0-1 health utility scale. Distant stage of cancer was associated with large decrements in the SF-6D ranging from -0.04 (prostate) to -0.08 (female breast).ConclusionA large number of chronic conditions, including cancer, are associated uniquely with decrements in health utility. The cumulative effects of comorbid conditions have substantial impact on daily functioning and well-being of Medicare beneficiaries.

BackgroundProfile instruments are frequently used to assess health-related quality of life and other patient-reported outcomes. However, preference-based measures are required for health-economic cost-utility evaluations.ResultsAlthough regression-based approaches are commonly used to map from profile measures to preference measures, we show that this results in biased estimates because of regression to the mean.ConclusionsLinking (scale-aligning) is proposed as an alternative.

Abstract Background The WHOQOL-BREF is a widely used questionnaire for measuring quality of life. It is important to establish the measurement equivalence of various language versions of WHOQOL-BREF so that scores from different language versions may be pooled together. The primary aim of this article was to evaluate the measurement equivalence of the English, Chinese and Malay versions of the WHOQOL-BREF. Methods We analysed data from the previously published, cross-sectional, WONDERS study and used linear regression models to adjust for potential confounding variables. Based on equivalence clinical trial methods, measurement equivalence was assessed by comparing 90% confidence interval (CI) of differences in scores across language versions with a predefined equivalence margin of 0.3 SD. Equivalence was achieved if the 90% CI fell within 0.3 SD. Data from 1203 participants, aged above 21 years, were analysed. Results Participants who completed the different language versions of WHOQOL-BREF expectedly differed in age, ethnicity, highest education level, marital status, smoking status and Body Mass Index (BMI). The English and Malay language versions were definitely equivalent for all domains. The English and Chinese language versions were definitely equivalent for physical and environmental domains but inconclusive for psychological and social domains. Likewise, for Chinese and Malay versions. Conclusion The English, Chinese and Malay language versions of the WHOQOL-BREF questionnaire may be considered equivalent, with evidence being more robust for some domains than the others. Given the large number of people who speak/ read Chinese and Malay, this study has widespread relevance.

Preference-based measures allow patients to report their level of health, and the responses are then scored using preference weights from a representative general population sample for use in cost utility analysis. The development process of new preference-based measures should ensure that valid items are selected to reflect the constructs of interest included in the measure and that are suitable for use in preference-elicitation exercises. Existing criteria on patient-reported outcome measures (PROMs) development were reviewed, and additional considerations were taken into account in order to generate criteria to support development of new preference-based measures. Criteria covering 22 different aspects related to item selection for preference-based measures are presented. These include criteria related to how items are phrased to ensure accurate completion, the coverage of items in terms of range of domains as well as focus on current outcomes and whether items are suitable for valuation. The criteria are aimed at supporting the development of new preference-based measures with discussion to ensure that even where there is conflict between criteria, issues have been considered at the item selection stage. This would minimize problems at valuation stage by harmonizing established criteria and expanding lists to reflect the unique characteristics of preference-based measures. [ABSTRACT FROM AUTHOR]

Background: Generic preference-based measures (GPBMs) are health-related quality of life (HRQoL) measures commonly used to evaluate the cost-utility of interventions in healthcare. However, the degree to which the content of GPBMs reflect the HRQoL of individuals with chronic obstructive pulmonary disease (COPD) has not yet been assessed. The purpose of this study was to examine the content and convergent validity of GPBMs in people with COPD.Methods: COPD patients were recruited from healthcare centers in Ontario, Canada. The Patient-Generated Index (PGI) (an individualized HRQoL measure) and the RAND-36 (to obtain SF-6D scores; a GPBM) were administered. Life areas nominated with the PGI were coded using the International Classification of Functioning Disability and Health and mapped onto GPBMs.Results: We included 60 participants with a mean age of 70 and FEV1% predicted of 43. The mean PGI score was 34.55/100 and the top three overarching areas that emerged were: 'mobility' (25.93%), 'recreation and leisure' (25.19%) and 'domestic life' (19.26%). Mapping of the nominated areas revealed that the Quality of Well-Being scale covered the highest number of areas (84.62%), Health Utilities Indices covered the least (15.38% and 30.77%) and other GPBMs covered between 46 and 62%. A correlation of 0.32 was calculated between the SF-6D and the PGI.Conclusions: The majority of GPBMs covered approximately half of the areas reported as being important to individuals with COPD. When areas relevant to COPD are not captured, HRQoL scores generated by these measures may inaccurately reflect patients' values and affect cost-effectiveness decisions. [ABSTRACT FROM AUTHOR]

Abstract Background Economic evaluation normally requires information to be collected on outcome improvement using utility values. This is often not collected during the treatment of substance use disorders making cost-effectiveness evaluations of therapy difficult. One potential solution is the use of mapping to generate utility values from clinical measures. This study develops and evaluates mapping algorithms that could be used to predict the EuroQol-5D (EQ-5D-5 L) and the ICEpop CAPability measure for Adults (ICECAP-A) from the three commonly used clinical measures; the CORE-OM, the LDQ and the TOP measures. Methods Models were estimated using pilot trial data of heroin users in opiate substitution treatment. In the trial the EQ-5D-5 L, ICECAP-A, CORE-OM, LDQ and TOP were administered at baseline, three and twelve month time intervals. Mapping was conducted using estimation and validation datasets. The normal estimation dataset, which comprised of baseline sample data, used ordinary least squares (OLS) and tobit regression methods. Data from the baseline and three month time periods were combined to create a pooled estimation dataset. Cluster and mixed regression methods were used to map from this dataset. Predictive accuracy of the models was assessed using the root mean square error (RMSE) and the mean absolute error (MAE). Algorithms were validated using sample data from the follow-up time periods. Results Mapping algorithms can be used to predict the ICECAP-A and the EQ-5D-5 L in the context of opiate dependence. Although both measures can be predicted, the ICECAP-A was better predicted by the clinical measures. There were no advantages of pooling the data. There were 6 chosen mapping algorithms, which had MAE scores ranging from 0.100 to 0.138 and RMSE scores ranging from 0.134 to 0.178. Conclusion It is possible to predict the scores of the ICECAP-A and the EQ-5D-5 L with the use of mapping. In the context of opiate dependence, these algorithms provide the possibility of generating utility values from clinical measures and thus enabling economic evaluation of alternative therapy options. Trial registration ISRCTN22608399. Date of registration: 27/04/2012. Date of first randomisation: 14/08/2012.

Objectives: To compare the psychometric properties of the Adult Social Care Outcomes Toolkit for carers (ASCOT-Carer), the Carer Experience Scale (CES), and the Care-related Quality of Life (CarerQol) to inform the choice of instrument in future studies.Methods: Data were derived from a 2018 online survey of informal carers in Australia. Reliability was assessed via internal consistency (Cronbach alpha, α) and test-retest reliability (intraclass correlation coefficient, ICC) for respondents who self-reported no change in their quality of life as a carer over 2 weeks. Convergent validity was evaluated via predetermined hypotheses about associations (Spearman's rank correlation) with existing, validated measures. Discriminative validity was assessed based on the ability of the carer-related scores to distinguish between different informal care situations (Mann-Whitney U, Kruskal-Wallis one-way analysis of variance).Results: Data from 500 carers were analyzed. The ASCOT-Carer demonstrated a higher degree of internal consistency, possibly due to a unidimensional structure, and test-retest reliability than the CarerQol and CES (α = 0.87, 0.65, 0.59; ICC, 0.87, 0.67, 0.81, respectively). All 3 instruments exhibited convergent validity and detected statistically significant associations between carer-related scores and different informal care situations, except for the CarerQol-7D and sole carer status.Conclusions: The ASCOT-Carer, CarerQol, and CES performed reasonably well psychometrically; the ASCOT-Carer exhibited the best psychometric properties overall in this sample of Australian informal carers. Findings should be used in conjunction with consideration of research goals, carer population, targeted carer-related constructs, and prevailing perspectives on the economic evaluation to inform choice of instrument in future studies. [ABSTRACT FROM AUTHOR]

Background. To incorporate the spillover effects experienced by carers providing informal care in health policy decisions, new carer-related preference-based measures have been developed for use in economic evaluation, which include the Adult Social Care Outcomes Toolkit for Carers (ASCOT-Carer), Carer Experience Scale (CES), and Care-Related Quality of Life (CarerQoL). The aim of this study was to investigate the extent to which these 3 instruments measure complementary or overlapping constructs. Methods. Data were derived from an online survey undertaken with carers residing in Australia. An exploratory factor analysis was conducted to ascertain the underlying latent constructs of the 3 measures. Results. Data from 351 informal carers yielded a 5-factor model describing general quality of life outside caring, problems due to caring, fulfilment from caring, social support with caring, and relationship with the care recipient. Most of the ASCOT-Carer and the CarerQol items loaded onto the first and second factors, respectively. The greatest overlap was observed between CarerQol and CES items loading onto the other 3 shared common factors. Limitations. Online data collection resulted in inconsistent responses, which had to be removed to yield logical data. A convenience sampling approach may have compromised the generalizability of study findings. Conclusion. Although some overlap was observed, the 3 carer-related preference-based measures seem to tap into different constructs of carer-related quality of life and caring experiences and cannot be used interchangeably. [ABSTRACT FROM AUTHOR]

Background: We sought to develop mapping functions that use EORTC responses to approximate health utility (HU) scores for patients with head and neck cancer (HNC). Methods: In total, 209 outpatients with HNC completed the EORTC QLQ‐C30 & QLQ‐H&N35 (EORTC), EQ‐5D‐5L and the HUI‐3. Results of the EORTC were mapped onto both EQ‐5D‐5L and HUI‐3 scores using ordinary least squares regression and two‐part models. Results: The OLS model mapping EORTC onto the EQ‐5D‐5L performed best (adjusted R2 =.75, 10‐fold cross‐validation RMSE = 0.064, MAE 0.050). The HUI‐3 model mapping onto EORTC through OLS was more limited (adjusted R2 =.5746, 10‐fold cross cross‐validation RMSE = 0.168, MAE 0.080). The EQ‐5D‐5L model was able to discriminate between certain clinical indices of disease severity on subgroup analysis. Conclusion: The EORTC to EQ‐5D‐5L mapping algorithm has good predictive validity and may enable researchers to translate EORTC scores into HU scores for head and neck patients with cancer. [ABSTRACT FROM AUTHOR]

Objectives: In recent years, discrete choice experiments (DCEs) have become frequently used to generate utility values, but there are a diverse range of approaches to do this. The primary focus of this systematic review is to summarise the methods used for the design and analysis of DCEs when estimating utility values in both generic and condition-specific preference-based measures. Methods: Published literature using DCEs to estimate utility values from preference-based instruments were identified from MEDLINE, Embase, Cochrane Library and CINAHL using PRISMA guidelines. To assess the different DCE methods, standardised information was extracted from the articles including the DCE design method, the number of choice sets, the number of DCE pairs per person, randomisation of questions, analysis method, logical consistency tests and techniques for anchoring utilities. The CREATE checklist was used to assess the quality of the studies. Results: A total of 38 studies with samples from the general population, students and patients were included. Values for health states described using generic multi attribute instruments (MAUIs) (especially the EQ-5D) were the most commonly explored using DCEs. The studies showed considerable methodology and design diversity (number of alternatives, attributes, sample size, choice task presentation and analysis). Despite these differences, the quality of articles reporting the methods used for the DCE was generally high. Conclusion: DCEs are an important approach to measure utility values for both generic and condition-specific instruments. However, a gold standard method cannot yet be recommended. [ABSTRACT FROM AUTHOR]

Background: Assessing the cost-effectiveness of interventions for people with dementia, based on cost per quality-adjusted life years (QALYs) gained, requires that the measures used to derive QALYs are preference-based whilst also being valid, feasible to use, comprehensible and acceptable for people with dementia. The aim of this study was to assess the content and face validity of six preference-based measures (PBMs) within the context of dementia.Methods: Qualitative focus groups and interviews were conducted with community-dwelling individuals with mild dementia and carers of people with dementia. After exploring participants' understanding of 'quality of life' (QoL), six PBMs were assessed for content and face validity: two measures assessing health-related QoL (EQ-5D-5L and AQoL-8D); two covering broader aspects of capability wellbeing and social care-related QoL (ICECAP-O and ASCOT); and two dementia-specific QoL measures (DEMQOL-U and AD-5D). A random mix of one health-related QoL measure, one wellbeing measure, and one dementia-specific measure was explored in each session. All sessions were audiotaped and transcribed verbatim. Data were analysed thematically.Results: Nine individuals with mild dementia and 17 carers of people with dementia participated across 4 focus groups and 10 interviews. Participants perceived 9 broad QoL domains as relevant to them: Activity, Autonomy, Cognition, Communication, Coping, Emotions, End-of-Life, Physical Functioning, and Relationships. These domains had limited overlap with the content of the six PBMs. Assessment of face validity was summarized into eight themes: (1) ambiguous questions, (2) double -barrelled questions, (3) difficult/abstract questions, (4) judgemental/confronting questions, (5) lack of relevance and comprehensiveness, (6) response options, (7) layout/format and (8) proxy-response. There was no clear preference for one of the six measures explored; participants identified advantages and disadvantages across all measures. Although particularly designed for individuals with dementia, dementia-specific QoL measures were not always favoured over non-specific measures.Conclusion: Given the shortcomings of PBMs identified in this study, further empirical comparative analyses are necessary to guide the selection of PBMs for future dementia research. [ABSTRACT FROM AUTHOR]

Objectives: To assess the acceptability and validity of the 3 levels of the EQ-5D (EQ-5D-3L) compared with the Quality of Life in Alzheimer's Diseases (QoL-AD) in patients living with dementia.Methods: The analysis was based on 560 dyads of persons with dementia and their caregivers of the multicenter observational study of dementia care networks in Germany (DemNet-D). Health-related quality of life was assessed by face-to-face interviews using the EQ-5D-3L (self-rating) and the QoL-AD (self- and proxy-rating). The number of missing values, the score ranges (observed vs possible range) and the floor and ceiling effects were used to assess the acceptability. We used one-way analyses of variance and multivariate linear regression models to evaluate the discriminative ability. The convergent validity was assessed using Spearman's correlation coefficient (rs) and multivariate regression models.Results: The EQ-5D index had a higher response rate (89% vs 84%) and a comparable floor (>1%) but a higher ceiling effect (18% vs >1%) compared with the QoL-AD. Both measures can significantly differentiate between different stages of general health, instrumental activities of daily living, and depression. The EQ-5D index and the visual analog scale self-rating scores strongly correlated with the QoL-AD self-rating (rs = 0.644 and 0.553, respectively) but not with the proxy-rating score (rs = 0.314 and rs = 0.170, respectively), which was confirmed by multivariate regression analyses.Conclusion: The results satisfy acceptability, discriminative ability, and convergent validity for moderately cognitively and functionally impaired patients living with dementia. The EQ-5D-3L performed comparably with the QoL-AD, and could, therefore, be used in economic evaluations in dementia. The differences between self- and proxy-ratings should be evaluated and considered in the interpretation of health-related quality of life scores. [ABSTRACT FROM AUTHOR]

Background: There is no mechanism to predict health utility (HU) values from the University of Washington Quality of Life Questionnaire (UWQoL) scores. We sought to develop a mapping algorithm capable of using UWQoL data to approximate HU scores. Methods: Outpatients with head and neck cancer completed the UWQoL, EQ‐5D, and the Health Utilities Index‐Mark 3 (HUI‐3). Results of the UWQoL were mapped onto both EQ‐5D and HUI‐3 scores using ordinary least‐squares regression models. Two‐part models were explored. The predictive power of the model was assessed using 10‐fold cross‐validation. Results: A total of 209 patients were recruited. The reduced model converting UWQoL data into EQ‐5D scores performed best (adjusted R2 = 0.628, root mean square error = 0.076). Both models demonstrated construct validity by discriminating between clinical indices of disease severity. Conclusions: The abovementioned algorithms enable researchers to perform health economic evaluations with existing UWQoL data in cases where prospectively collected HU values are not available. [ABSTRACT FROM AUTHOR]

Background: Dementia-specific and proxy-completed preference-based measures have been proposed for use in intervention studies involving people living in residential care, in instances where generic, self-reported preference-based measures have been deemed inappropriate.Objective: This study was conducted to investigate the construct validity, criterion validity, and responsiveness of DEMQOL-Proxy-U and of self- and proxy-completed EQ-5D-5L.Methods: The analysis used a 3-wave, individual-level data set of 1004 people living with dementia in residential care that included self-completed EQ-5D-5L and formal-carer and informal-carer proxy-completed EQ-5D-5L and DEMQOL-Proxy-U utility values, in addition to other nonutility cognitive measures (Functional Assessment Staging [FAST], Clinical Dementia Rating [CDR], Cohen-Mansfield Agitation Inventory [CMAI]) and health-related quality of life (HRQOL) measures (nursing home version of the Quality of Life with Alzheimer's disease scale [QOL-AD-NH], Quality of Life in Late-Stage Dementia [QUALID] scale). Construct validity, criterion validity, and responsiveness were assessed using correlation, Bland-Altman plots, and panel data regression models.Results: Self-completed EQ-5D-5L failed to reflect clinically important differences and changes in FAST, CDR, and CMAI but did capture the resident's own view of HRQOL (QOL-AD-NH). As dementia severity increased, collection of EQ-5D-5L-proxy and DEMQOL-Proxy-U data was more feasible than collection of self-completed EQ-5D-5L. These formal-carer and informal-carer proxy measures also better reflected changes in FAST, CDR, and CMAI but did not capture the resident's own view of HRQOL (QOL-AD-NH), despite adequately capturing the proxy's own view of the resident's HRQOL (QUALID). This indicates discrepancies between a proxy's view and resident's view of the impact that tangible declines in health, cognition, or functional abilities have on HRQOL. The EQ-5D-5L-proxy and DEMQOL-Proxy-U were generally poor substitutes. Regardless of which proxy completed it, the EQ-5D-5L-proxy was typically more responsive than the DEMQOL-Proxy-U to changes in CDR, FAST, and CMAI, indicating that use of the DEMQOL-Proxy-U is not always justified.Conclusion: Disparities in the measurement properties of different utility measures mean that choices about how to measure utility in trials could affect economic evaluation outcomes and hence how resources are allocated for dementia care. [ABSTRACT FROM AUTHOR]

Background: Preference-based measures of health-related quality of life (HRQL) are used as primary or secondary endpoints in multiple sclerosis (MS) research. Objective: The purpose of this paper was to evaluate the structural, convergent, and known-groups validity of the preference-based multiple sclerosis index (PBMSI) of HRQL in people with MS. Methods: Participants were recruited from three MS clinics in Montreal. Structural validity was assessed using polychoric correlation coefficients and factor analysis. To assess convergent validity, hypotheses were formulated about the strength of correlations between the PBMSI and other HRQL measures. Known-groups validity was assessed against different measures of disability. Results: The average age of the sample was 46 and 77% were women. Factor analysis supported the structural validity of the PBMSI; the items collectively were measuring one underlying construct. The PBMSI showed convergent validity against generic measures of HRQL, and known-groups validity between persons with different levels of disability. Conclusion: The results of this study support the construct validity of the PBMSI as an outcome measure of HRQL in MS. The PBMSI overcomes limitations observed with currently used HRQL measures in MS and may be used to contrast different interventions for people with MS. [ABSTRACT FROM AUTHOR]

Background. Researchers often justify excluding some responses in studies eliciting valuations of health states as not representing respondents' true preferences. Here, we examine the effects of applying 8 common exclusion criteria on societal utility estimates. Setting. An online survey of a US nationally representative sample (N = 1164) used the standard gamble method to elicit preferences for health states defined by 7 health domains from the Patient-Reported Outcomes Measurement Information System (PROMIS®). Methods. We estimate the impacts of applying 8 commonly used exclusion criteria on mean utility values for each domain, using beta regression, a form of analysis suited to double-bounded scales, such as utility. Results. Exclusion criteria have varied effects on the utility functions for the different PROMIS health domains. As a result, applying those criteria would have varied effects on the value of treatments (and side effects) that change health status on those domains. Limitations. Although our method could be applied to any health utility judgments, the present estimates reflect the features of the study that produced them. Those features include the selected health domains, standard gamble method, and an online format that excluded some groups (e.g., visually impaired and illiterate individuals). We also examined only a subset of all possible exclusion criteria, selected to represent the space of possibilities, as characterized in a companion article. Conclusions. Exclusion criteria can affect estimates of the societal utility of health states. We use those effects, in conjunction with the results of the companion article, to make suggestions for selecting exclusion criteria in future studies. [ABSTRACT FROM AUTHOR]

Background. In a systematic review, Engel et al. found large variation in the exclusion criteria used to remove responses held not to represent genuine preferences in health state valuation studies. We offer an empirical approach to characterizing the similarities and differences among such criteria. Setting. Our analyses use data from an online survey that elicited preferences for health states defined by domains from the Patient-Reported Outcomes Measurement Information System (PROMIS®), with a U.S. nationally representative sample (N = 1164). Methods. We use multidimensional scaling to investigate how 10 commonly used exclusion criteria classify participants and their responses. Results. We find that the effects of exclusion criteria do not always match the reasons advanced for applying them. For example, excluding very high and very low values has been justified as removing aberrant responses. However, people who give very high and very low values prove to be systematically different in ways suggesting that such responses may reflect different processes. Conclusions. Exclusion criteria intended to remove low-quality responses from health state valuation studies may actually remove deliberate but unusual ones. A companion article examines the effects of the exclusion criteria on societal utility estimates. [ABSTRACT FROM AUTHOR]

Purpose: There is concern that some generic preference-based measures (GPMs) of health-related quality of life may be insensitive to interventions that improve hearing. Establishing where sensitivity arises could contribute to the design of improved measures. Accordingly, we compared the sensitivity of four widely used GPMs to a clinically effective treatment-cochlear implantation-which restores material degrees of hearing to adults with little or no functional hearing.Methods: Participants (N = 147) received implants in any of 13 hospitals in the UK. One month before implantation and 9 months after, they completed the HUI2, HUI3, EQ5D3L, and SF-6D questionnaires, together with the EuroQoL visual-analogue scale as a direct measure of health, a performance test of speech reception, and a self-report measure of annoyance due to tinnitus.Results: Implantation was associated with a large improvement in speech reception and a small improvement in tinnitus. HUI2 and HUI3 were sensitive to the improvement in speech reception through their Sensation and Hearing dimensions; EQ5D3L was sensitive to the improvement in tinnitus through its Anxiety/Depression dimension; SF-6D was sensitive to neither. Participants reported no overall improvement in health. Variation in health was associated with variation in tinnitus, not variation in speech reception.Conclusions: None of the four GPMs was sensitive to the improvements in both speech reception and tinnitus that were associated with cochlear implantation. To capture fully the benefits of interventions for auditory disorders, developments of current GPMs would need to be sensitive to both the health-related and non-health-related aspects of auditory dysfunction. [ABSTRACT FROM AUTHOR]

The EuroQol 5-dimension (EQ-5D) is a standard instrument that is widely used for measuring health-related quality of life and quality-adjusted life years in economic evaluation of healthcare interventions. To estimate a preference valuation set for EQ-5D 3-level (3L) health states from the perspective of the general population in the capital of Iran. Eight hundred seventy adults aged ≥18 years were interviewed in Tehran (Iran's capital) from July to November 2013. The participants were selected by a stratified random sampling method and were interviewed face-to-face at their usual residence. Forty-two health states were selected and valued from the 243 states derived from the EQ-5D-3L instrument. Each respondent valued 11 health states using the time trade-off method. Generalized least squares regression with random effect was used to predict values for health states. The analysis was performed for 846 respondents. The final model yielded the best fit for the time trade-off value at the individual level with an overall R2 of 0.45 and a mean absolute error of 0.214. The mean values for the 42 health states ranged from 0.934 for state 11121 to -0.142 for state 33333. This study provided for the first time a value set for calculating quality-adjusted life years from the EQ-5D instrument in Iran. The Iranian EQ-5D-3L value set slightly differs from the value sets of the UK and the United States. [ABSTRACT FROM AUTHOR]

Objective. To compare and contrast EQ-5D-5L (5L) and EQ-5D-3L (3L) health state values derived from a common sample. Methods. Data from the 2017 US EQ-5D valuation study were analyzed. Value sets were estimated with random-effects linear regression based on composite time trade-off (cTTO) valuations for 3L and 5L health states with 2 approaches to model specification: main effects only and additional N3/N45 terms. Properties of the descriptive system and value set characteristics were compared by examining distributions of predicted index scores, ceiling effects, and single-level transition values from adjacent corner health states. Mean transition values were calculated for all predicted 3L and 5L health states and plotted against baseline index scores. Results. A total of 1062 respondents were included in the analysis. The observed mean cTTO values for the worst possible 3L and 5L health states were −0.423 and −0.343, respectively. The range of scale was larger with the 3L, compared to the 5L, for both main effects and N term models. Values for the mildest 5L health states (range, 0.857−0.924) were similar to 11111 for the 3L. Parameter estimates for matched dimension levels differed by <|0.07| except for the most severe level of Mobility. For the main effects model, 3L mean transition values were greater for more severe baseline 3L index scores, whereas 5L mean transition values remained constant irrespective of the baseline index score. Conclusions. Compared to the 3L, the 5L exhibited a lower ceiling effect and improved measurement properties. There was a larger range of scale for the 3L compared to 5L; however, this difference was driven by differences in preference for the most severe level of problems in Mobility. [ABSTRACT FROM AUTHOR]

Objective: This article presents the development of the Dutch value set for the Child Health Utility 9D, a pediatric preference-based measure of quality of life that can be used to generate quality-adjusted life-years.Methods: A large online survey was conducted using a discrete choice experiment including a duration attribute with adult members of the Netherlands general population (N = 1276) who were representative in terms of age, gender, marital status, employment, education, and region. Respondents were asked which of two health states they prefer, where each health state was described using the nine dimensions of the Child Health Utility 9D (worried, sad, pain, tired, annoyed, school work/homework, sleep, daily routine, able to join in activities) and duration. The data were modeled using conditional logit with robust standard errors to produce utility values for every health state described by the Child Health Utility 9D.Results: The majority of the dimension level coefficients were monotonic, leading to a decrease in utility as severity increases. There was, however, evidence of some logical inconsistencies, particularly for the school work/homework dimension. The value set produced was based on the ordered model and ranges from -0.568 for the worst state to 1 for the best state.Conclusion: The valuation of the Child Health Utility 9D using online discrete choice experiment with duration with adult members of the Dutch general population was feasible and produced a valid model for use in cost utility analysis. Normative questions are raised around the valuation of pediatric preference-based measures, including the appropriate perspective for imagining hypothetical pediatric health states. [ABSTRACT FROM AUTHOR]

Objective. Self-management is becoming increasingly important in diabetes but is neglected in conventional preference-based measures. The objective of this paper was to generate health state utility values for a novel classification system measuring the quality-of-life impact of self-management for diabetes, which can be used to generate quality-adjusted life years (QALYs). Methods. A large online survey was conducted using a discrete choice experiment (DCE), with duration as an additional attribute, on members of the UK general population (n = 1,493) to elicit values for health (social limitations, mood, vitality, hypoglycaemia) and non-health (stress, hassle, control, support) aspects of self-management in diabetes. The data were modelled using a conditional fixed-effects logit model and utility estimates were anchored on the one to zero (full health to dead) scale. Results. The model produced significant and consistent coefficients, with one logical inconsistency and 3 insignificant coefficients for the milder levels of some attributes. The anchored utilities ranged from 1 for the best state to −0.029 for the worst state (meaning worse than dead) defined by the classification system. Conclusion. The results presented here can potentially be used to generate utility values capturing the day to day impact of interventions in diabetes on both health and self-management. These utility values can potentially be used to generate QALYs for economic models of the cost-effectiveness of interventions in diabetes. [ABSTRACT FROM AUTHOR]

Background: No current guidance is available in the UK on the choice of preference-based measure (PBM) that should be used in obtaining health-related quality of life from children. The aim of this study is to review the current usage of PBMs for obtaining health state utility values in child and adolescent populations, and to obtain information on patient and parent-proxy respondent preferences in completing PBMs in the UK.Methods: A literature review was conducted to determine which instrument is most frequently used for child-based economic evaluations and whether child or proxy responses are used. Instruments were compared on dimensions, severity levels, elicitation and valuation methods, availability of value sets and validation studies, and the range of utility values generated. Additionally, a series of focus groups of parents and young people (11-20 years) were convened to determine patient and proxy preferences.Results: Five PBMs suitable for child populations were identified, although only the Health Utilities Index 2 (HUI2) and Child Heath Utility 9D (CHU-9D) have UK value sets. 45 papers used PBMs in this population, but many used non-child-specific PBMs. Most respondents were parent proxies, even in adolescent populations. Reported missing data ranged from 0.5 to 49.3%. The focus groups reported their experiences with the EQ-5D-Y and CHU-9D. Both the young persons' group and parent/proxy groups felt that the CHU-9D was more comprehensive but may be harder for a proxy to complete. Some younger children had difficulty understanding the CHU-9D questions, but the young persons' group nonetheless preferred responding directly.Conclusion: The use of PBMs in child populations is increasing, but many studies use PBMs that do not have appropriate value sets. Parent proxies are the most common respondents, but the focus group responses suggest it would be preferred, and may be more informative, for older children to self-report or for child-parent dyads to respond. [ABSTRACT FROM AUTHOR]

Background: Several utility-based instruments have been applied in cost-utility analysis to assess health state values for people with dementia. Nevertheless, concerns and uncertainty regarding their performance for people with dementia have been raised.Objectives: To assess the performance of available utility-based instruments for people with dementia by comparing their psychometric properties and to explore factors that cause variations in the reported health state values generated from those instruments by conducting meta-regression analyses.Methods: A literature search was conducted and psychometric properties were synthesized to demonstrate the overall performance of each instrument. When available, health state values and variables such as the type of instrument and cognitive impairment levels were extracted from each article. A meta-regression analysis was undertaken and available covariates were included in the models.Results: A total of 64 studies providing preference-based values were identified and included. The EuroQol five-dimension questionnaire demonstrated the best combination of feasibility, reliability, and validity. Meta-regression analyses suggested that significant differences exist between instruments, type of respondents, and mode of administration and the variations in estimated utility values had influences on incremental quality-adjusted life-year calculation.Conclusions: This review finds that the EuroQol five-dimension questionnaire is the most valid utility-based instrument for people with dementia, but should be replaced by others under certain circumstances. Although no utility estimates were reported in the article, the meta-regression analyses that examined variations in utility estimates produced by different instruments impact on cost-utility analysis, potentially altering the decision-making process in some circumstances. [ABSTRACT FROM AUTHOR]

Background. Existing methods to link preference-based and profile-based health-related quality of life (HRQoL) questionnaires have their limitations. Hence, we developed a new mapping method (the mean rank method, MRM) and applied it to map the World Health Organization Quality of Life Questionnaire (WHOQOL-BREF) to the EuroQoL 5 Dimensions 5 Levels (EQ-5D-5L). We then compared the new MRM with current methods; i.e., regression-mapped (OLS method) and equipercentile method (EPM). Methods. Singapore residents, aged ≥21 y, were recruited from the general population and 2 outpatient clinics in acute care hospitals. Performance of the MRM was evaluated using both simulation and split-sample validation (n = 658 in training and n = 657 in validation samples). Using the training sample, we derived 3 sets of mapped EQ-5D-5L utilities based on MRM, OLS method and EPM. Using simulation and the validation sample, we compared the performance of the mapping methods in terms of distribution parameters, mean utility by strata, association with health covariates, and prediction errors at the individual level, among others. Results. The WHOQOL-BREF Physical Health domain is the only domain significantly associated with EQ-5D-5L utilities. Simulation showed that MRM more accurately reproduced the variance and percentiles of the distribution of the observed utilities than did the OLS method or EPM. OLS method tended to underestimate the mean utility of good health states, overestimate the mean utility of poor health states, and underestimate the association with covariates. An analysis of validation sample gave similar results. Conclusion. In scenarios similar to the mapping of WHOQOL-BREF to the EQ-5D-5L, the MRM outperformed the OLS method and EPM in important—though not all—aspects. The simplicity and reproducibility of the MRM makes it an attractive alternative to current methods. [ABSTRACT FROM AUTHOR]

Dental caries has significant negative impacts on the lives of children and young people. Whilst the impacts on children’s oral health-related quality of life (OHRQoL) have been increasingly investigated, the effect on children’s overall wellbeing remains largely unknown. Data were obtained from a survey conducted across four cities in the Netherlands. Children and their parents completed a series of questionnaires, which included Dutch versions of a caries-specific pediatric measure of OHRQoL (CARIES-QC-NL) and a generic pediatric health utility measure (CHU9D-NL). The participating children underwent dental examinations to determine their caries status. A total of 486 11-year-old children participated in the study, of which 184 had caries experience (38%). The mean number of decayed, missing and filled teeth (DMFT) was 0.71. The CARIES-QC-NL was found to have statistically significant correlations with the DMFT and CHU9D-NL. There were no statistically significant correlations between the CHU9D and the clinical variables. The CARIES-QC-NL had acceptable internal consistency and construct validity in this population despite the low prevalence of active caries. A relationship was demonstrated between OHRQoL and generic wellbeing in this population. Despite this, the CHU9D did not show any correlation with the clinical data, which may limit its application in studies of the impact of dental caries.

Objectives: To describe the use of a novel approach in health valuation of a discrete-choice experiment (DCE) including a cost attribute to value a recently developed classification system for measuring the quality-of-life impact (both health and treatment experience) of self-management for diabetes.Methods: A large online survey was conducted using DCE with cost on UK respondents from the general population (n = 1497) and individuals with diabetes (n = 405). The data were modeled using a conditional logit model with robust standard errors. The marginal rate of substitution was used to generate willingness-to-pay (WTP) estimates for every state defined by the classification system. Robustness of results was assessed by including interaction effects for household income.Results: There were some logical inconsistencies and insignificant coefficients for the milder levels of some attributes. There were some differences in the rank ordering of different attributes for the general population and diabetic patients. The WTP to avoid the most severe state was £1118.53 per month for the general population and £2356.02 per month for the diabetic patient population. The results were largely robust.Conclusions: Health and self-management can be valued in a single classification system using DCE with cost. The marginal rate of substitution for key attributes can be used to inform cost-benefit analysis of self-management interventions in diabetes using results from clinical studies in which this new classification system has been applied. The method shows promise, but found large WTP estimates exceeding the cost levels used in the survey. [ABSTRACT FROM AUTHOR]