Health utility values of breast cancer treatments and the impact of varying quality of life assumptions on cost-effectiveness.

In breast cancer research, utility assumptions are outdated and inconsistent which may affect the results of quality adjusted life year (QALY) calculations and thereby cost-effectiveness analyses (CEAs). Four hundred sixty four female patients with breast cancer treated at Erasmus MC, the Netherlands, completed EQ-5D-5L questionnaires from diagnosis throughout their treatment. Average utilities were calculated stratified by age and treatment. These utilities were applied in CEAs analysing 920 breast cancer screening policies differing in eligible ages and screening interval simulated by the MISCAN-Breast microsimulation model, using a willingness-to-pay threshold of €20,000. The CEAs included varying sets on normative, breast cancer treatment and screening and follow-up utilities. Efficiency frontiers were compared to assess the impact of the utility sets. The calculated average patient utilities were reduced at breast cancer diagnosis and 6 months after surgery and increased toward normative utilities 12 months after surgery. When using normative utility values of 1 in CEAs, QALYs were overestimated compared to using average gender and age-specific values. Only small differences in QALYs gained were seen when varying treatment utilities in CEAs. The CEAs varying screening and follow-up utilities showed only small changes in QALYs gained and the efficiency frontier. Throughout all variations in utility sets, the optimal strategy remained robust; biennial for ages 40-76 years and occasionally biennial 40-74 years. In sum, we recommend to use gender and age stratified normative utilities in CEAs, and patient-based breast cancer utilities stratified by age and treatment or disease stage. Furthermore, despite varying utilities, the optimal screening scenario seems very robust.

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

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

Direct and Indirect Mapping of Assessment of Quality of Life - 6 Dimensions (AQoL-6D) Onto EQ-5D-5L Utilities Using Data From a Multicenter, Cross-Sectional Study of Malaysians With Chronic Heart Failure.

OBJECTIVES: The Assessment of Quality of Life - 6 Dimensions (AQoL-6D), a generic preference-based measure, is an appealing alternative to EQ-5D-5L for assessing health status in patients with chronic heart failure (HF), given its expanded scope. However, without a Malaysian value set, the AQoL-6D cannot generate health state utility values (HSUVs) to support local economic evaluations. This study intended to develop algorithms for predicting EQ-5D-5L HSUVs from AQoL-6D in an HF population. METHODS: Cross-sectional data from a multicenter cohort of 419 HF outpatients were used. Both direct and indirect mapping approaches were attempted using 5 sets of explanatory variables and 8 models (ordinary least squares, Tobit, censored least absolute deviations, generalized linear model, 2-part model [TPM], beta regression-based model, adjusted limited dependent variable mixture model, and multinomial ordinal regression [MLOGIT]). The models' predictive performance was assessed through 10-fold cross-validated mean absolute error [MAE] and root mean squared error [RMSE]). Potential prediction bias was also examined graphically. The best-performing models, with the lowest RMSE and no bias, were then identified. RESULTS: Among the models evaluated, TPM, which included age, sex, and 5 AQoL-6D dimension scores as predictors, appears to be the best-performing model for directly predicting EQ-5D-5L HSUVs from AQoL-6D. TPM yielded the lowest MAE (0.0802) and RMSE (0.1116), and demonstrated predictive accuracy for HSUVs >0.2 without significant bias. A MLOGIT model developed for response mapping had suboptimal predictive accuracy. CONCLUSIONS: This study developed potentially useful mapping algorithms for generating Malaysian EQ-5D-5L HSUVs from AQoL-6D responses among patients with HF when direct EQ-5D-5L data are unavailable.

Health state utility values of type 2 diabetes mellitus and related complications: a systematic review and meta-regression.

BACKGROUND: This study aimed to synthesize and quantitatively examine Health State Utility Values (HSUVs) for Type 2 Diabetes Mellitus (T2DM) and its complications, providing a robust meta-regression framework for selecting appropriate HSUV estimates. METHOD: We conducted a systematic review to extract HSUVs for T2DM and its complications, encompassing various influencing factors. Relevant literature was sourced from a review spanning 2000-2020, supplemented by literature from PubMed, Embase, and the Web of Science (up to March 2024). Multivariate meta-regression was performed to evaluate the impact of measurement tools, tariffs, health status, and clinical and demographic variables on HSUVs. RESULTS: Our search yielded 118 studies, contributing 1044 HSUVs. The HSUVs for T2DM with complications varied, from 0.65 for cerebrovascular disease to 0.77 for neuropathy. The EQ-5D-3L emerged as the most frequently employed valuation method. HSUV differences across instruments were observed; 15-D had the highest (0.89), while HUI-3 had the lowest (0.70) values. Regression analysis elucidated the significant effects of instrument and tariff choice on HSUVs. Complication-related utility decrement, especially in diabetic foot, was quantified. Age <70 was linked to increased HSUVs, while longer illness duration, hypertension, overweight and obesity correlated with reduced HSUVs. CONCLUSION: Accurate HSUVs are vital for the optimization of T2DM management strategies. This study provided a comprehensive data pool for HSUVs selection, and quantified the influence of various factors on HSUVs, informing analysts and policymakers in understanding the utility variations associated with T2DM and its complications.

Health state utility values in patients with Ankylosing Spondylitis: a systematic review and meta-analysis.

INTRODUCTION: Ankylosing spondylitis (AS) is a chronic condition that requires lifelong treatment and results in a serious disease burden. Health state utility values (HSUVs) are a valuable tool for quantifying this burden and conducting cost-utility analysis. OBJECTIVE: We conducted a systematic review and meta-analysis to obtain estimates of HSUVs in patients with AS, explored potential sources of heterogeneity, and compared pooled patient HSUVs with population norms. METHOD: We searched PubMed, Embase, Web of science, Cochrane database and Scopus until July, 2023 to obtain eligible studies. The methodological quality of the included studies was assessed using the ROBINS-I checklist. RESULTS: Forty-two publications involving 11,354 participants were included in this systematic review. The most commonly used instrument is the EQ-5D (38 studies). The estimated HSUVs for patients with AS from all available studies was pooled as 0.62 (95% CI 0.59 to 0.65). The pooled mean utility estimates from the random effects meta-analysis for SF-6D, EQ-5D-3L, EQ-5D-5L, and HUI3 were 0.65 (95% CI 0.62,0.68), 0.63 (95% CI 0.59,0.66), 0.60 (95% CI 0.42,0.79), and 0.48 (95% CI 0.43,0.53), respectively. For the EQ-5D-3L we conducted stratified meta-analyses and meta-regression based on key subgroups. The pooled estimates of EQ-5D-3L were lower for patients published before 2010, with high disease activity, long duration of disease, and in developed countries. CONCLUSION: Pooled estimates of HSUVs for people with AS were substantially lower than population norms. These estimates provide robust evidence that can inform the economic evaluation of new therapies for individuals with AS.

Estimation of health utility values for alopecia areata.

PURPOSE: Alopecia areata (AA) is an autoimmune-mediated inflammatory dermatological disease characterised by non-scarring hair loss affecting the scalp and sometimes other hair-bearing sites. This study aimed to elicit health state utility values (HSUVs) from the UK general population for AA using time trade off (TTO) interviews. METHODS: Vignette descriptions of health states defined by the extent of hair loss were developed (as well as one describing caregiver burden). These were developed using data from standardised patient reported outcome (PRO) measures, a literature review and qualitative interviews. Health states were defined based on the severity of alopecia tool (SALT), which assesses extensiveness of scalp hair loss. HSUVs were then elicited for each health state in TTO interviews with the UK public. RESULTS: One caregiver and five patient health states were developed based on the literature review findings, clinical trial PRO (Hospital Anxiety and Depression Scale and Alopecia Areata Patient Priority Outcomes Questionnaire) data and qualitative interviews with patients (N = 11), clinical experts (N = 4) and caregivers of adolescents with AA (N = 10). These data showed a more severe impact among patients with more extensive hair loss. One hundred and twenty participants evaluated the vignettes in TTO interviews. Patient HSUVs ranged from 0.502 for the most extensive hair loss health state (SALT 50-100 + eyebrow and eyelash loss) to 0.919 (SALT 0-10) for the mildest health state. The caregiver HSUV was 0.882. CONCLUSION: Quantitative and qualitative data sources were used to develop and validate vignettes describing different AA health states. Patient and caregiver HSUVs demonstrate a large impact associated with AA, especially for states defined by more extensive hair loss.

Health-related quality of life and utility values among patients with anxiety and/or depression in a low-income tertiary care setting: a cross-sectional analysis.

OBJECTIVES: To describe the health-related quality of life (HRQoL), estimate the associated health state utility values (HSUVs) and explore factors associated with HRQoL of patients with anxiety and/or depression in a resource-limited hospital setting. METHODS: A cross-sectional survey involving 462 participants was conducted in a hospital setting. The Amharic version of the EQ-5D-5 L assessed HRQoL, while the GAD-7 and PHQ-9 measured severity of anxiety and depression symptoms respectively. HSUVs were analysed based on clinical and demographic profiles; mean differences were compared using t-tests and one-way ANOVA; Scheffe's post hoc comparisons and effect sizes (Cohen's d statistic) were used to assess the magnitude of group differences. Factors associated with HRQoL were explored using regression analysis. RESULTS: The mean HSUV was 0.87 (SD = 0.17) and the EQ VAS was 71.4 (SD = 19.1). Patients with both anxiety and depression scored significantly lower (HSUV = 0.83 [0.16], EQ VAS = 64.4 [17.9]) compared to those with either anxiety only (HSUV = 0.88 [0.17], EQ VAS = 75.3 [17.9]) or depression only (HSUV = 0.89 [0.18], EQ VAS 74.4 [19.7]). Males had slightly higher mean scores than females, while those aged 18-35 years demonstrated the highest scores on both the EQ-5D-5 L and EQ VAS. Older age (ß=-0.002), higher PHQ-9 scores (ß=-0.008) and comorbid hypertension (ß=-0.07) associated with lower HSUVs. Lower EQ VAS scores were associated with being female (ß=-4.4), having comorbid hypertension (ß=-7.4) and higher PHQ-9 scores (ß=-0.86), while a positive association was found with having 'more than enough' income (ß = 11.8). CONCLUSIONS: Older age, severity or co-diagnosis of anxiety or depression and comorbid conditions were associated with lower HRQoL, highlighting the need for better interventions to improve the HRQoL of patients with anxiety and depression in Ethiopia.

Examining health-related quality of life in ambulatory adult patients with chronic heart failure: insights from Malaysia using EQ-5D-5L.

PURPOSE: Health-related quality-of-life (HRQoL) data for the chronic heart failure (HF) population in Malaysia are lacking. Using EQ-5D-5L, this study intended to describe their HRQoL, identify predictors of worse HRQoL, and derive EQ-5D-5L index scores for use in economic evaluations. METHODS: A cross-sectional survey was conducted between April and September 2023 to collect EQ-5D-5L, sociodemographic, and clinical data from outpatients with HF across seven public specialist hospitals in Malaysia. Multivariable logistic and linear regression models were used to identify independent predictors of reported problems in the EQ-5D-5L dimensions, and predictors of index scores and EQ-VAS, respectively. RESULTS: EQ-5D-5L data from 424 outpatients of multi-ethnic background (mean age: 57.1 years, 23.8% female, mean left ventricular ejection fraction: 35.7%, 89.7% NYHA class I-II) were collected using either Malay, English, or Chinese, achieving a 99.8% completion rate. Nearly half of the respondents reported issues in the Mobility, Usual Activities, and Pain/Discomfort dimensions. Mean EQ-5D-5L index was 0.820, lower than the general population, and significantly lower with NYHA class III-IV (0.747) versus NYHA class I (0.846) and NYHA class II (0.805). Besides NYHA class, independent predictors of worse HRQoL included Indian ethnicity, living alone, lower education, unemployment due to ill-health, and proxy-reported HRQoL, largely aligning with existing literature. CONCLUSION: Community-dwelling Malaysians with HF reported poorer HRQoL compared to the general population. The observed disparities in HRQoL among HF patients may be linked to specific patient characteristics, suggesting potential areas for targeted interventions. HRQoL assessment using EQ-5D-5L proves feasible and should be considered for routine implementation in local clinics.

Mapping of Family Reported Outcome Measure (FROM-16) scores to EQ-5D: algorithm to calculate utility values.

OBJECTIVE: Although decision scientists and health economists encourage inclusion of family member/informal carer utility in health economic evaluation, there is a lack of suitable utility measures comparable to patient utility measures such those based on the EQ-5D. This study aims to predict EQ-5D-3L utility values from Family Reported Outcome Measure (FROM-16) scores, to allow the use of FROM-16 data in health economic evaluation when EQ-5D data is not available. METHODS: Data from 4228 family members/partners of patients recruited to an online cross-sectional study through 58 UK-based patient support groups, three research support platforms and Welsh social services departments were randomly divided five times into two groups, to derive and test a mapping model. Split-half cross-validation was employed, resulting in a total of ten multinomial logistic regression models. The Monte Carlo simulation procedure was used to generate predicted EQ-5D-3L responses, and utility scores were calculated and compared against observed values. Mean error and mean absolute error were calculated for all ten validation models. The final model algorithm was derived using the entire sample. RESULTS: The model was highly predictive, and its repeated fitting using multinomial logistic regression demonstrated a stable model. The mean differences between predicted and observed health utility estimates ranged from 0.005 to 0.029 across the ten modelling exercises, with an average overall difference of 0.015 (a 2.2% overestimate, not of clinical importance). CONCLUSIONS: The algorithm developed will enable researchers and decision scientists to calculate EQ-5D health utility estimates from FROM-16 scores, thus allowing the inclusion of the family impact of disease in health economic evaluation of medical interventions when EQ-5D data is not available.

Longitudinal associations between Latine parents' support and their adolescents' science motivation and STEM career expectations.

INTRODUCTION: Parents' science support and adolescents' motivational beliefs are associated with adolescents' expectations for their future occupations; however, these associations have been mostly investigated among White, middle-class samples. Framed by situated expectancy-value theory, the current study investigated: (1) the associations between parents' science support in 9th grade and Latine adolescents' science intrinsic value, utility value, and STEM career expectations in 11th grade, and (2) whether these indicators and the relations among them differed by adolescents' gender and parents' education. METHODS: Study participants included Latine adolescents (n = 3060; Mage = 14.4 years old; 49% female) in the United States from the High School Longitudinal Study of 2009. RESULTS: Analyses revealed a significant, positive association between parents' science support and Latine adolescents' science utility value. Additionally, there was a significant, positive association between parents' science support and Latinas' science intrinsic value, but not for Latinos' science intrinsic value. Latine adolescents' science utility value, but not their science intrinsic value, predicted their concurrent STEM career expectations. Though there were no significant mean level differences in adolescents' science utility value or parents' science support based on adolescents' gender, the measure of adolescents' science intrinsic value varied across girls and boys. Finally, adolescents whose parents had a college degree received greater science support from parents compared to adolescents whose parents had less education than a college degree. CONCLUSION: Findings suggest parents' science support and adolescents' intrinsic and utility values have potential associations with Latine adolescents' STEM career expectations near the end of high school.

The Impact of Hearing Loss and Its Treatment on Health-Related Quality of Life Utility: a Systematic Review with Meta-analysis.

BACKGROUND: Hearing loss significantly impacts health-related quality of life (QoL), yet the effects of current treatments on QoL utility remain uncertain. Our objective was to describe the impact of untreated and treated hearing loss on QoL utility to inform hearing healthcare policy. METHODS: We searched databases for articles published through 02/01/2021. Two independent reviewers screened for articles that reported elicitation of general QoL utility values for untreated and treated hearing loss health states. We extracted data and quality indicators from 62 studies that met the inclusion criteria. RESULTS: Included studies predominately used observational pre/post designs (61%), evaluated unilateral cochlear implantation (65%), administered the Health Utilities Index 3 (HUI3; 71%), and were conducted in Europe and North America (84%). In general, treatment of hearing loss improved post-treatment QoL utility when measured by most methods except the Euro-QoL 5 dimension (EQ-5D). In meta-analysis, hearing aids for adult mild-to-moderate hearing loss compared to no treatment significantly improved HUI3-estimated QoL utility (3 studies; mean change=0.11; 95% confidence interval (CI): 0.07 to 0.14) but did not impact EQ-5D-estimated QoL (3 studies; mean change=0.0; 95% CI: -0.03 to 0.04). Cochlear implants improved adult QoL utility 1-year post-implantation when measured by the HUI3 (7 studies; mean change=0.17; 95% CI: 0.11 to 0.23); however, pediatric VAS-estimated QoL utility was non-significant (4 studies; mean change=0.12; 95% CI: -0.02 to 0.25). The quality of included studies was limited by failure to report missingness of data and low survey response rates. Our study was limited by heterogeneous study populations and designs. FINDINGS: Treatment of hearing loss significantly improves QoL utility, and the HUI3 and VAS were most sensitive to improvements in hearing. Improved access to hearing healthcare should be prioritized. SYSTEMATIC REVIEW REGISTRATION: PROSPERO: CRD42021253314.

How Do Patients With Type 2 Diabetes Mellitus Value the Importance of Outcomes? An Overview of Reviews.

OBJECTIVES: We aimed to assess how patients value the importance of type 2 diabetes mellitus (T2DM) related outcomes. METHODS: Overview of systematic reviews (SRs) reporting patients' utilities or disutilities for T2DM outcomes. We searched 3 databases from inception until June 2021. Study selection and data extraction were conducted in pairs. We evaluated the quality of SRs with the Joanna Briggs Institute Checklist, and the overlap with the corrected covered area. We estimated descriptive statistics, and, when possible, conducted metanalysis. RESULTS: We identified 11 SRs, including 119 studies and 70 outcomes. Most reviews were high-quality SRs. The outcomes with the lowest utilities were hypoglycemia with very severe symptoms (acute complications), stroke (macrovascular complications), diabetic peripheral neuropathy with severe pain (microvascular complications), extreme obesity (comorbidities), and insulin only or combined (management of diabetes). Good/excellent glucose control and noninsulin injectable showed higher values than T2DM without complications. The outcomes with the highest disutilities were amputation, depression, major hypoglycemia, stroke, and management using only insulin. CONCLUSIONS: We provide standardized, reliable utility values (or associated disutilities) for T2DM, acute, microvascular and macrovascular complications, related comorbidities and treatments that may support judgments when making clinical recommendations, designing decision support tools, and developing interventions and economic analysis.

Modifying the Composite Time Trade-Off Method to Improve Its Discriminatory Power.

OBJECTIVES: In cost-effectiveness analysis of health technologies, health state utilities are needed. They are often elicited with a composite time trade-off (cTTO) method, particularly for the widely used EQ-5D-5L. Unfortunately, cTTO discriminatory power is hindered by (1) respondents' nontrading (NT) of time for quality, (2) censoring of utilities at -1, and (3) poor correlation of negative utilities with state severity. We investigated whether modifying cTTO can mitigate these effects. METHODS: We interviewed online 478 students (February to April, 2021) who each valued the same 10 EQ-5D-5L health states in 1 of 3 arms. Arm A used a standard cTTO, expanded with 2 questions to explore reasons for NT and censoring. Arms B and C used a time trade-off with modified alternatives offered to overcome loss aversion, to unify the tasks for positive and negative utilities, and to enable eliciting utilities < -1. RESULTS: In arms B and C, we observed less NT than in A (respectively, 4% and 4% vs 10%), more strictly negative utilities (38% and 40% vs 25%), and more utilities ≤ -1 (18% and 30% vs 10%). The average utility of state 55555 dropped to -2.15 and -2.52 from -0.53. Enabling finer trades in arm A reduced NT by 70%. Arms B and C yielded an intuitive association between negative utilities and state severity. These arms were considered more difficult and resulted in more inconsistencies. CONCLUSIONS: The discriminatory power of cTTO can be improved, but it may require increasing the difficulty of the task. The standard cTTO may overestimate the utilities, especially of severe states.

Determinants of health-related quality of life in people with Human Immunodeficiency Virus, failing first-line treatment in Africa.

BACKGROUND: Antiretroviral treatment improves health related quality of life (HRQoL) of people with human immunodeficiency virus (PWH). However, one third initiating first-line treatment experience virological failure and the determinants of HRQoL in this key population are unknown. Our study aims to identify determinants of among PWH failing antiretroviral treatment in sub-Saharan Africa. METHODS: We analysed data from a cohort of PWH having virological failure (> 1,000 copies/mL) on first-line ART in South Africa and Uganda. We measured HRQoL using the EuroQOL EQ-5D-3L and used a two-part regression model to obtain by-country analyses for South Africa and Uganda. The first part identifies risk factors that were associated with the likelihood of participants reporting perfect health (utility = 1) versus non-perfect health (utility < 1). The second part identifies risk factors that were associated with the EQ-5 L-3L utility scores for participants reporting non-perfect health. We performed sensitivity analyses to compare the results between the two-part model using tobit models and ordinary least squares regression. RESULTS: In both countries, males were more likely to report perfect health and participants with at least one comorbidity were less likely to report perfect health. In South Africa, participants with side effects and in Uganda those with opportunistic infections were also less likely to report perfect health. In Uganda, participants with 100% ART adherence were more likely to report perfect health. In South Africa, high HIV viral load, experiencing ART side effects, and the presence of opportunistic infections were each associated with lower HRQoL, whereas participants with 100% ART adherence reported higher HRQoL. In Uganda participants with lower CD4 count had lower HRQoL. CONCLUSION: Markers of advanced disease (opportunistic infection, high viral load, low CD4), side effects, comorbidities and lack of ART adherence negatively impacted HRQoL for PWH experiencing virological failure. TRIAL REGISTRATION: ClinicalTrials.gov: NCT02787499.

A systematic review of health state utility values and psychometric performance of generic preference-based instruments for children and adolescents with mental health problems.

AIMS: This paper aims to systematically identify reported health state utility values (HSUVs) in children and adolescents with mental health problems (MHPs) aged less than 25 years; to summarise the techniques used to elicit HSUVs; and to examine the psychometric performance of the identified multi-attribute utility instruments (MAUIs) used in this space. METHODS: A systematic review was conducted following PRISMA guidelines. Peer-reviewed studies published in English, reporting HSUVs for children and adolescents with MHPs using direct or indirect valuation methods were searched in six databases. RESULTS: We found 38 studies reporting HSUVs for 12 types of MHPs across 12 countries between 2005 and October 2021. Attention deficit hyperactivity disorder (ADHD) and depression are the most explored MHPs. Disruptive Behaviour Disorder was associated with the lowest reported HSUVs of 0.06 while cannabis use disorder was associated with the highest HSUVs of 0.88. Indirect valuation method through the use of MAUIs (95% of included studies) was the most frequently used approach, while direct valuation methods (Standard Gamble, Time Trade-Off) were only used to derive HSUVs in ADHD. This review found limited evidence of the psychometric performance of MAUIs used in children and adolescents with MHPs. CONCLUSION: This review provides an overview of HSUVs of various MHPs, the current practice to generate HSUVs, and the psychometric performance of MAUIs used in children and adolescents with MHPs. It highlights the need for more rigorous and extensive psychometric assessments to produce evidence on the suitability of MAUIs used in this area.

Quantifying health-related quality of life in Malaysian type 2 diabetes: focusing on complication types and severity.

PURPOSE: There is a knowledge gap of health utility values for Type 2 Diabetes Mellitus (T2DM) complications in Malaysia. This study aimed to estimate EQ-5D-5L utility values and evaluate health-related quality of life (HRQoL) for Malaysian T2DM associated with complications and clinical characteristics. METHODS: A cross-sectional study was conducted on T2DM patients at a tertiary hospital outpatient using the Malay and English version of the EQ-5D-5L questionnaire. Health utility values were derived using the Malaysian EQ-5D-5L value set. Ordinary least squares (OLS) multivariable regression model was used to estimate the health utility decrements associated with T2DM-related complications and clinical characteristics. RESULTS: A total of 513 T2DM patients were recruited. Overall, pain was the most affected of all five EQ-5D-5L dimensions. Patients with foot ulcer, amputation, severe heart failure and frequent hypoglycemia reported more problems collectively in all EQ-5D-5L dimensions. Older age, lower education level, longer duration of T2DM, urine protein creatine index (UPCI) > 0.02 g/mmol, and injection therapy were significantly associated with lower EQ-5D-5L utility values (p < 0.004, Bonferroni adjusted). The lowest unadjusted utility values were reported for severe heart failure 0.65 (interquartile range, IQR 0.50), frequent hypoglycemia 0.74 (0.22) and being amputated 0.78 (0.47). In the multivariable regression model after controlling for sociodemographic and clinical characteristics, the largest utility value decrement was observed for amputation (- 0.158, SE 0.087, p = 0.05), frequent hypoglycemia (- 0.101, SE 0.030, p = 0.001), myocardial infarction (-0.050, SE 0.022, p = 0.022) and obesity (-0.034, SE 0.016, p = 0.029). CONCLUSION: Larger utility value decrements were found for severe stages of complications. These findings suggest the value of defining severity of complications in utility elicitation studies. The utility decrement quantified for different T2DM complication severity will be useful for economic evaluations within diabetic-related fields.

Assessment of health-related quality of life in Australian patients with idiopathic pulmonary fibrosis: a comparison of the EQ-5D-5L and the AQoL-8D.

PURPOSE: Idiopathic pulmonary fibrosis (IPF) is a progressive and debilitating chronic lung disease with a high symptom burden, which has a substantial impact on health-related quality of life (HRQoL). Our study aimed to assess the suitability of the EuroQol five-dimension (EQ-5D-5L) and the Assessment of Quality of Life- eight-dimension (AQoL-8D) questionnaires in measuring HRQoL as health state utility values (HSUVs) in an Australian IPF cohort. METHODS: Data for estimation of health state utility values (HSUVs) were collected from participants of the Australian IPF Registry (AIPFR) using self-administered surveys which included the EQ-5D-5L and the AQoL-8D. Data on lung function and disease specific HRQoL instruments were collected from the AIPFR. Performance of the two instruments was evaluated based on questionnaire practicality, agreement between the two instruments and test performance (internal and construct validity). RESULTS: Overall completion rates for the EQ-5D-5L and AQoL-8D were 96% and 85%, respectively. Mean (median) HSUVs were 0.65 (0.70) and 0.69 (0.72) for the EQ-5D-5L and AQoL-8D, respectively. There was reasonable agreement between the two instruments based on the Bland-Altman plot mean difference (-0.04) and intraclass correlation coefficient (0.84), however there were some fundamental differences. A larger range of values was observed with the EQ-5D-5L (-0.57-1.00 vs 0.16-1.00). The EQ-5D-5L had a greater divergent sensitivity and efficacy in relation to assessing HSUVs between clinical groupings. The AQoL-8D ,however, had a higher sensitivity to measure psychosocial aspects of HRQoL in IPF. CONCLUSION: The EQ-5D-5L demonstrated superior performance when compared to AQoL-8D in persons with IPF. This may be attributable to the high symptom burden which is physically debilitating to which the EQ-5D-5L may be more sensitive.

A cross-sectional study of symptoms and health-related quality of life in menopausal-aged women in China.

OBJECTIVE: To measure symptoms and health-related quality of life in a cross-sectional cohort of menopausal-aged women in China. METHOD: A cross-sectional survey was conducted in a general population cohort of 2,000 Chinese females over the age of 45 years. Patients completed the Chinese version of the EuroQol-5D five level (EQ5D5L) health-related quality of life instrument via Personal Digital Assistant. Raw scores were converted to utility tariffs using value sets for China. Statistical analysis included Pearson's chi-square test, z test for multiple comparisons with adjustment by the Bonferroni method, independent-sample t-test, ANOVA, and adjustment by the Tukey method for multiple comparison. Results were considered statistically significant when p < 0.05 and the study was reported according to the STROBE recommendations. RESULTS: In a cohort of 2000 women, 732 (37%) were premenopausal, 798 (40%) were perimenopausal and 470 (23%) were postmenopausal. Perimenopausal women reported significantly more symptoms (91%) compared to premenopausal (77%) and postmenopausal (81%) women. Health-related quality of life was significantly lower in symptomatic perimenopausal women compared to premenopausal (0.919, p < 0.05) and postmenopausal (0.877, p < 0.05) women. Within each group there was a statistically significant difference between the health-related quality of life of women with symptoms compared to without symptoms. CONCLUSION: The perimenopausal phase of menopause is associated with significantly more symptoms and significantly lower HRQoL compared to premenopausal and postmenopausal phases.

Why is this study needed?• It is important to understand the impact of menopausal symptoms on women's quality of life so that decisions can be made about what healthcare services to provide and what treatments to fund.• Currently there are only a few older studies investigating menopausal symptoms in the perimenopausal phase, which is characterised by hormone fluctuations and symptoms likely to impact quality of life, and lasts up to four years. What is the key problem this manuscript addresses?• This research measures symptoms and the impact on quality of life of menopausal women in China. What are your main results and what do they mean?• Women in the perimenopausal phase are disproportionately affected by symptoms compared to premenopausal and postmenopausal women.• Perimenopausal women have significantly lower HRQoL compared to premenopausal and postmenopausal women.• This information is critical to informing pharmacoeconomic decisions about healthcare provision and publicly funded treatments in China.

Development of an EQ-5D Value Set for India Using an Extended Design (DEVINE) Study: The Indian 5-Level Version EQ-5D Value Set.

OBJECTIVES: This study aimed to develop the Indian 5-level version EQ-5D (EQ-5D-5L) value set, which is a key input in health technology assessment for resource allocation in healthcare. METHODS: A cross-sectional survey using the EuroQol Group's Valuation Technology was undertaken in a representative sample of 3548 adult respondents, selected from 5 different states of India using a multistage stratified random sampling technique. The participants were interviewed using a computer-assisted personal interviewing technique. This study adopted a novel extended EuroQol Group's Valuation Technology design that included 18 blocks of 10 composite time trade-off (c-TTO) tasks, comprising 150 unique health states, and 36 blocks of 7 discrete choice experiment (DCE) tasks, comprising 252 DCE pairs. Different models were explored for their predictive performance. Hybrid modeling approach using both c-TTO and DCE data was used to estimate the value set. RESULTS: A total of 2409 interviews were included in the analysis. The hybrid heteroscedastic model with censoring at -1 combining c-TTO and DCE data yielded the most consistent results and was used for the generation of the value set. The predicted values for all 3125 health states ranged from -0.923 to 1. The preference values were most affected by the pain/discomfort dimension. CONCLUSIONS: This is the largest EQ-5D-5L valuation study conducted so far in the world. The Indian EQ-5D-5L value set will promote the effective conduct of health technology assessment studies in India, thereby generating credible evidence for efficient resource use in healthcare.

Quality appraisal for systematic literature reviews of health state utility values: a descriptive analysis.

BACKGROUND: Health state utility values (HSUVs) are an essential input parameter to cost-utility analysis (CUA). Systematic literature reviews (SLRs) provide summarized information for selecting utility values from an increasing number of primary studies eliciting HSUVs. Quality appraisal (QA) of such SLRs is an important process towards the credibility of HSUVs estimates; yet, authors often overlook this crucial process. A scientifically developed and widely accepted QA tool for this purpose is lacking and warranted. OBJECTIVES: To comprehensively describe the nature of QA in published SRLs of studies eliciting HSUVs and generate a list of commonly used items. METHODS: A comprehensive literature search was conducted in PubMed and Embase from 01.01.2015 to 15.05.2021. SLRs of empirical studies eliciting HSUVs that were published in English were included. We extracted descriptive data, which included QA tools checklists or good practice recommendations used or cited, items used, and the methods of incorporating QA results into study findings. Descriptive statistics (frequencies of use and occurrences of items, acceptance and counterfactual acceptance rates) were computed and a comprehensive list of QA items was generated. RESULTS: A total of 73 SLRs were included, comprising 93 items and 35 QA tools and good recommendation practices. The prevalence of QA was 55% (40/73). Recommendations by NICE and ISPOR guidelines appeared in 42% (16/40) of the SLRs that appraised quality. The most commonly used QA items in SLRs were response rates (27/40), statistical analysis (22/40), sample size (21/40) and loss of follow up (21/40). Yet, the most commonly featured items in QA tools and GPRs were statistical analysis (23/35), confounding or baseline equivalency (20/35), and blinding (14/35). Only 5% of the SLRS used QA to inform the data analysis, with acceptance rates of 100% (in two studies) 67%, 53% and 33%. The mean counterfactual acceptance rate was 55% (median 53% and IQR 56%). CONCLUSIONS: There is a considerably low prevalence of QA in the SLRs of HSUVs. Also, there is a wide variation in the QA dimensions and items included in both SLRs and extracted tools. This underscores the need for a scientifically developed QA tool for multi-variable primary studies of HSUVs.