Testing "Pits" Time Trade-Off: Can Data Quality be Improved by Removing Death From Valuation of Health States?

OBJECTIVES: Composite time trade-off (cTTO) often exhibits issues such as a value clustering at -1, potentially due to suboptimal valuation task design. We developed a novel time trade-off which uses the "pits" state as an anchor state and enables a unified time trade-off procedure for all health states (referred to as "pTTO"). This study aimed to test the pTTO for valuation of EQ-5D-5L health states. METHODS: A total of 120 members of the general public were invited to value 3 pairs of dominant/dominated EQ-5D-5L states using both cTTO and pTTO and the "pits" state using cTTO. We compared the pTTO with cTTO in terms of feasibility (interviewer-rated task-completing difficulty), acceptability (participant-reported task experience), value distribution, logical consistency, and discriminatory ability (using the standardized response mean, an effect size measure, derived from 3 pairwise comparisons of states with dominant/dominated relationship). RESULTS: The pTTO demonstrated similar feasibility to cTTO but slightly lower acceptability. The pTTO values exhibited a smoother and more continuous distribution compared with cTTO values. Relative to the cTTO, the pTTO showed higher individual-level logical consistency rate for the pairs comprising severe states (stringent criterion: 53.04% vs 17.39%; less stringent criterion: 82.61% vs 78.26%). The standardized response mean value based on pTTO values were higher than those based on cTTO values for the pair comprising severe states (0.388 vs -0.140). CONCLUSIONS: The pTTO appeared to be feasible and acceptable to value EQ-5D-5L states. The pTTO values were less clustered, more logically consistent, and discriminative compared with the cTTO values for severe health states.

Preference-based utility weights for the Individualized Neuromuscular Quality of Life Questionnaire (INQoL), with a focus on non-dystrophic myotonia (NDM).

INTRODUCTION: The Individualized Neuromuscular Quality of Life Questionnaire (INQoL) is used to measure quality of life in neuromuscular disorders such as non-dystrophic myotonia (NDM). Here we report methods to estimate utilities, with a focus on NDM, from this questionnaire based on two preference elicitation exercises. METHODS: Eight items from the INQoL were selected with input from three neuromuscular disorder clinical experts with expertise in treating NDM. A discrete choice experiment (DCE) survey of UK general public respondents (n = 508) described outcomes defined by the INQoL items. The same 8 items were also valued using time trade-off (TTO) face-to-face interviews (n = 200). A hybrid regression modelling approach combined both datasets to inform the utility weights. RESULTS: Hybrid modelling of DCE and TTO data in conjunction improved out-of-sample predictive accuracy. The selected INQoL utility model indicates substantial disutility associated with all eight dimensions of health, with the greatest losses associated with subjective items such as pain and depression. DISCUSSION: The hybrid modelling approach allows us to combine data from the two methodologies and maximize the information from each to inform the utility weights for the INQoL. The TTO is the more conventional valuation method, but combined with the larger DCE study produced better descriptive coverage. This is a relatively novel method for estimating weights which we think is particularly well suited to economic evaluations of orphan drugs.

Digital thErapy For Improved tiNnitus carE Study (DEFINE): Protocol for a randomised controlled trial.

Tinnitus is a common health condition, affecting approximately 15% of the UK population. The tinnitus treatment with the strongest evidence base is Cognitive Behavioural Therapy (CBT), with standard tinnitus therapy typically augmented with education, relaxation and other techniques. Availability of CBT and conventional tinnitus therapy more broadly is limited for tinnitus sufferers. The DEFINE trial aims to assess whether smartphone-delivered tinnitus therapy, the Oto app, is as effective as current standard care, one-to-one therapist-delivered tinnitus treatment for the treatment of tinnitus in adults. The trial is registered in the ISRCTN Registry: ISRCTN99577932. DEFINE is an open-label, non-inferiority, prospective, parallel design, randomised-controlled trial. Recruitment, interventions and assessments will be remote, enabling UK-wide participant involvement. 198 participants aged 18 years or more will be recruited via social media advertisement or via primary care physicians. A screening process will identify those with tinnitus that impacts health-related quality of life, and following consent smartphone-based audiometry will be performed. Randomisation 1:1 to the Oto app or one-to-one therapist-led tinnitus therapy will be performed centrally by computer, matching groups for age, sex and hearing level. Following participant allocation, the Oto app will be provided for immediate use, or a one-to-one remote therapy appointment booked to occur within approximately 1 week, with up to 6 sessions delivered. Participant outcomes will be collected at 4,12, 26 and 52 weeks via questionnaire and phone call. The primary outcome is the change in Tinnitus Functional Index (TFI) total score measured at 26 weeks following allocation. Adverse events will be recorded. A health economic evaluation in the form of a cost-utility analysis will be performed using data from participant submitted EuroQol 5D-5L and Health Utilities Index Mark 3 scores and resource use data. Trial results will be made publicly available, including a plain English summary.

Matching-Adjusted Indirect Comparison of the Long-Term Efficacy Maintenance and Adverse Event Rates of Lebrikizumab versus Dupilumab in Moderate-to-Severe Atopic Dermatitis.

INTRODUCTION: Lebrikizumab and dupilumab are monoclonal antibodies approved for treating moderate-to-severe atopic dermatitis (AD). Both have demonstrated efficacy and safety over the 16-week SOLOs and ADvocate trials. However, AD is a chronic and relapsing inflammatory disease, and the long-term maintenance of efficacy is critical for achieving disease control from the perspective of patients, physicians, and regulatory agencies. This study aims to compare the long-term efficacy and safety of lebrikizumab every 4 weeks (Q4W) and dupilumab every week or every 2 weeks (QW/Q2W) among adult patients who have achieved treatment efficacy following the induction period of 16 weeks. METHODS: Lebrikizumab's efficacy was assessed using individual patient data (IPD) from the ADvocate 1 and 2 monotherapy trials. Dupilumab's efficacy was evaluated using aggregate data from the adult-exclusive SOLO-CONTINUE trial. Due to the absence of a common comparator trial arm, we employed an unanchored matching-adjusted indirect comparison (MAIC), a robust methodology widely accepted by health technology assessment (HTA) agencies. This re-weights ADvocate IPD to align with SOLO-CONTINUE's prognostic factors and effect modifiers. We compared lebrikizumab's adjusted outcomes with dupilumab outcomes at week 52, focusing on 75% improvement in the Eczema Area and Severity Index from baseline (EASI-75), Investigator's Global Assessment (IGA) score of 0 or 1, and overall adverse event (AE) rates. Sensitivity analyses were conducted to test various combinations of matching variables. RESULTS: Adults on lebrikizumab Q4W were more likely to maintain IGA 0/1 through the 36-week maintenance period (weeks 16-52) compared with those on dupilumab QW/Q2W [risk ratio (RR) 1.334; 95% confidence interval (CI) 1.02-1.74; p = 0.035]. Both treatments demonstrated comparable efficacy in terms of EASI-75 maintenance (RR 0.937; 95% CI 0.78-1.13; p = 0.490) and similar AE rates (RR 1.052; 95% CI 0.90-1.23; p = 0.526). Sensitivity analyses substantiated these findings. CONCLUSIONS: Our findings suggest that lebrikizumab Q4W may provide equal or superior long-term maintenance of efficacy measured with EASI-75 and IGA 0/1 compared with dupilumab QW/Q2W, with the advantage of requiring less frequent doses.

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

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

Exploring non-iterative time trade-off methods for valuation of EQ-5D-5L health states.

INTRODUCTION: The composite time trade-off (cTTO) method is used as the primary method for valuing EQ-5D-5L health states, but it requires intensive interviewer training and stringent quality control, which increases the burden of conducting cTTO studies. In this study, two non-iterative variants of the TTO method, non-stopping TTO (nTTO) and open-ended TTO (oTTO), were tested head-to-head with the cTTO method aiming to reduce the administration burden. METHODS: 31 EQ-5D-5L health states from an orthogonal array was selected and valued by a general public sample in China. Data were collected by 7 interviewers with all interviewers performed an equal number of interviews using all three TTO methods. We compared the value distribution, logical consistency, administration burden, and modeling performance of these three TTO methods. RESULTS: In total, 422 participants participated in the valuation interviews, with 139 using the nTTO method, 140 using the oTTO method, and 143 using the cTTO method. Both oTTO and nTTO methods saved around 10 min for conducting an interview. The mean values of three methods were similar with each method showed different characteristics in their value distributions. cTTO outperformed the other two methods in terms of modeling performance. DISCUSSION: Both non-iterative TTO methods showed potential for valuing EQ-5D health states, although their data distributions and modeling performance were inferior to the cTTO method. The results of this study showed the potential of these two alternative non-iterative TTO methods.

Valuation of EQ-5D-5L health states from cancer patients' perspective: a feasibility study.

OBJECTIVES: To assess the feasibility of estimating an EQ-5D-5L value set using a small study design in cancer patients and to compare the EQ-5D-5L values based on the preferences of cancer patients with those of the general public. METHODS: Patients with clinically diagnosed cancers were recruited from two hospitals in Shanghai, China. In face-to-face interviews using the EQ-PVT survey, health states were valued by cancer patients using both cTTO and DCE methods. cTTO data was modelled alone or jointly with DCE data. Forty-eight models using different model specifications (cross-attribute level effect [CALE] and additive models), random/fixed effects model assumptions, data heteroscedasticity and censoring were estimated. The best performed model was identified in terms of monotonicity of estimated model coefficients and out-of-sample prediction accuracy. RESULTS: Data collected from 221 cancer patients who participated in the study were included. The hybrid CALE model using both TTO and DCE data performed best in terms of prediction accuracy (Lin's concordance coefficient = 0.989; root mean squared error = 0.058) and suggested that pain/discomfort and anxiety/depression were the most undesirable health problems. Compared to values based on general Chinese public's health preferences, the values based on cancer patients' preferences were much higher and lower for health states characterized by extreme mobility problems and severe/extreme pain or discomfort, respectively. CONCLUSION: This study demonstrated the feasibility of using a small design to develop EQ-5D-5L value sets based on cancer patients' health preferences. Since there were signs of differences between preferences of patients and general population, it may be valuable to develop patient-specific value sets and use them in clinical decision making and economic evaluations.

Reduced quality of life, more technical challenges, and less study motivation among paramedic students after one year of the COVID-19 pandemic - a survey study.

INTRODUCTION: Despite the lack of knowledge about the SARS-CoV2 virus, the lack of personal protection gear among frontline healthcare workers, and lack of vaccines in the beginning of the pandemic, paramedic students in Norway contributed to the National response against the COVID-19 pandemic by working in test-stations, ambulance services, ambulance decontamination stations etc. Despite fear of contracting the COVID-19 reported by healthcare workers worldwide, paramedic students in Norway reported higher-than-average quality of life after four months of the COVID-19 pandemic (first pandemic wave). In this study we aimed to investigate how students reported their quality of life, study motivation and job satisfaction after one year of living with the COVID-19 pandemic. METHOD: At two data collection point, all paramedic students enrolled at Oslo Metropolitan University were invited to participate in a digital, online, self-administered survey. The first data collection was in June 2020 (the first pandemic wave), while the second data collection was in March 2021 (the third pandemic wave). Results from both samples were analyzed independently with descriptive statistics. Differences between the groups were analyzed using an independent T-test and Mann-Whitney-U test to discover changes over time. Multiple linear regression analysis was used to estimate the difference attributable to timing (first vs. the third wave), seniority, and student gender. RESULTS: The samples consist of slightly more female students than male students. The mean age in both samples is 24.6 years. Despite the higher-than-average level of quality of life in the first pandemic wave, results show that there was a significant reduction in students' health-related quality of life (p < 0.001, B -0.059, SE 0.016), study motivation (p = 0.002, 95% CI:0.09,0.41), and job satisfaction (p = 0.005, 95% CI:1.62,9.00) after the third pandemic wave in Norway. Surprisingly, students experienced more technical challenges in the third wave, e.g., poor internet connection, sound pollution, and poor picture quality, despite more experience among students and teachers. CONCLUSION: Our results show that paramedic students had significant worsening experiences in the late pandemic wave compared to the first pandemic wave. Universities and governments should learn from the COVID-19 pandemic to develop better preparedness plans for future pandemics and knowledge about students' well-being should be considered in future preparedness plans for higher education and the government plans for the education of front-line healthcare workers during a pandemic to facilitate the continuation of necessary education.

Urban/rural differences in preferences for EQ-5D-5L health states: a study of a multi-ethnic region in China.

OBJECTIVES: This study aimed to compare health preferences for EQ-5D-5L health states between urban and rural populations in China. METHODS: This study used pooled secondary data from two EQ-5D-5L valuation studies. Participants were recruited from Guizhou province and Chongqing municipality, China using quota sampling. Each participant was interviewed face-to-face to value a set of 15 or 16 out of 30 EQ-5D-5L health states using time trade-off (TTO) methods including composite TTO and other two variants. Regression analysis was used to compare health state preferences between urban and rural participants. RESULTS: A total of 597 participants (urban: 55.44%; rural: 44.56%) completed the valuation interviews. Both univariate and multivariable linear regression analyses showed that rural participants tended to value health states lower than urban participants regardless of severity of health states. The unadjusted and adjusted overall mean differences between the two groups were - 0.041 (95% confidence interval [CI] - 0.077, - 0.004, p-value = 0.031) and - 0.040 (95% CI - 0.078, - 0.002, p-value = 0.038), respectively. Predictions for the 3125 health states based on rural participants' health preferences were lower than those based on urban participants' health preferences. CONCLUSION: There were small, yet statistically significant, differences in EQ-5D-5L health states preferences between urban and rural populations in China. Future study aiming at establishing a national value set should pay more attention to the sample representativeness.

Cross-Attribute Level Effects Models for Modeling Modified 5-Level Version of EQ-5D Health State Values: Is Less Still More?

OBJECTIVES: Cross-attribute level effects (CALE) model has demonstrated better predictive accuracy for out-of-sample health states than the conventional additive main-effects model in cross-validation analysis of the 5-level version of EQ-5D (EQ-5D-5L) composite time trade-off (cTTO) datasets. In this study, we aimed to further test the performance of CALE model using a different design and modified EQ-5D-5L states. METHODS: A total of 29 EQ-5D-5L self-care bolt-off states, 30 EQ-5D-5L states, and 31 EQ-5D-5L vision bolt-on states were selected from the same orthogonal array. A total of 600 university students were interviewed face-to-face to value a subset of these health states using the cTTO method. For each type of health state, we fitted both the conventional main-effects model and the CALE model. Predictive accuracy was assessed in a series of cross-validation analysis using the leave-one-state-out method. RESULTS: Overall, the CALE model outperformed the conventional model for each of the 3 types of health states in predicting the cTTO values of out-of-sample health states. The prediction accuracy of using the CALE model improved with the number of dimensions in health states, for example, the MAE decreased about 24%, 67%, and 77% for the EQ-5D-5L self-care bolt-off, EQ-5D-5L, and EQ-5D-5L vision bolt-on states, respectively, when using CALE models. CONCLUSION: Our study supported the strengths of the CALE model for modelling the utility values of both original and modified EQ-5D-5L health states. Investigators with limited resources may consider using the CALE model to lower the costs for their valuation studies for EQ-5D-5L or similar health state descriptive systems.

Censoring in the time trade-off valuation of worse-than-dead EQ-5D-5L health states: can a time-based willingness-to-accept question be the solution?

PURPOSE: The left censoring of values at -1 by design of the composite time trade-off (cTTO) tasks leads to excessive amount of -1 values in some EQ-5D-5L valuation studies. This study aimed to investigate whether a time-based willingness-to-accept (tWTA) question can be used to elicit values lower than -1 and improve the estimation of EQ-5D-5L values. METHODS: At the end of each cTTO task in the Taiwanese EQ-5D-5L valuation study, if the value of the health state was indicated to be lower than -1, a tWTA question eliciting the indifference point between a hypothetical life (i.e. x number of years in full health followed by 10 years in the health state) and immediate death was used to estimate its uncensored value. We compared the statistical characteristics of the censored and uncensored data. RESULTS: Four hundred and twenty-nine of 1,000 respondents were offered the tWTA question in a total of 1,071 cTTO tasks. In 79.55% of those tasks, indifference was not reached. Spearman's correlation with level summary score was -0.41 and -0.40 for negative uncensored and censored data, respectively. The logical inconsistency rates of the uncensored and censored data were 0.88% vs. 0.29%, respectively. Modelling of the uncensored data resulted in coefficients with greater uncertainty and much lower predictions. CONCLUSIONS: The elicitation of values lower than -1 using a tWTA question that grants more time for trading seems not a promising solution to the value censoring of the cTTO tasks. Other strategies for valuation of very poor health states should be explored.

Censoring Time Trade-off Values at 0 Versus at -1: How Does the Assumption for Worse-Than-Dead Time Trade-off Values Affect the Modeling of EQ-5D-5L Valuation Data?

OBJECTIVES: A recent study found that negative utility values elicited using composite time trade-off (TTO) were barely associated with the severity of EQ-5D-5L health states, suggesting poor discriminative ability. Assuming negative values provide limited information, this study aimed to explore the usefulness of censoring negative TTO values at 0 in modeling EQ-5D-5L valuation data. METHODS: We analyzed EQ-5D-5L valuation data from China, The Netherlands, Canada, Singapore, and Thailand. For each data set, we estimated value sets using 2 Tobit models, one left-censored at -1 (current practice) and one left-censored at 0 (our proposed method), and compared the model performances. We hypothesized that censoring at 0 and censoring at -1 would produce similar values, though on slightly different scales. RESULTS: When censoring at 0, logical inconsistencies and statistical significance were improved but the value range was compressed. In the cross-attribute level effects model, the 3-level parameters were similar between the models censored at 0 and -1, but the rank order of some dimension parameters was altered. Health state values predicted by the 2 censoring models approximated a perfect agreement after rescaling. CONCLUSIONS: Censoring TTO values at 0 improved model estimation and fit but produced higher utility values than models censoring at -1. Investigators of future EQ-5D value set studies using the composite TTO method are advised to examine the validity of negative TTO values before choosing modeling strategies.

Completing the time trade-off with respondents who are older, in poorer health or with an immigrant background in an EQ-5D-5L valuation study.

OBJECTIVES: To determine the effects of age, immigrant background, and poor self-reported health in a general population sample on the probability of non-completion or slow completion of the time trade-off (TTO). METHODS: We used data from an interrupted Norwegian EQ-5D-5L valuation study conducted between 2019 and 2020. All participants responded to background items, irrespective of completion. We used mixed effect logistic regression analysis to assess the effect of old age, poor health, and immigrant background on the probability of non-completion of the TTO, and, for those who completed the TTO, of slow completion times. RESULTS: First experiences from a Norwegian valuation study were that 29 (5.5%) respondents failed to complete the TTO tasks. For those reporting age over 65 years, poor health, or an immigrant background, 12% failed to complete the TTO. Adjusted odds ratios for predictors of non-completion were statistically significant (age > 65 years, 8.3; EQ-VAS ≤ 50, 3.49; immigrant background, 4.56). Being over 65 years or with an immigrant background also predicted slow completion of both the introduction and TTO tasks. CONCLUSIONS: High age, poor health, and immigrant status increased the risk of not being able to complete the TTO tasks, and of slow completion. Higher non-completion rates and increased completion times suggest that elements of the TTO may be demanding for some respondent groups, with possible implications for representativeness.

EQ-5D-5L measurement properties are superior to EQ-5D-3L across the continuum of health using US value sets.

OBJECTIVE: The objective of this study was to compare the measurement properties of the US EQ-5D-3L, EQ-5D-5L, and -5L to -3L crosswalk value sets (3L; 5L; 5L > 3L) across the spectrum of health. METHODS: The three scoring approaches were compared in terms of range of scale, percent of worse-than-dead health states, and mean single-level transitions. Discriminative ability was compared by leveraging two cross-sectional datasets. A novel method was used to visualize and compare the responsiveness of 3L and 5L scoring approaches across EQ VAS values. RESULTS: The US 5L value set had the broadest range of scale at 1.573 (vs. 1.109 for 3L and crosswalk). The crosswalk had the smallest mean single-level transition of 0.061 (vs. 0.078 for 5L and 0.111 for 3L). The 5L value set tended to be more discriminative/greater statistical efficiency than the crosswalk (F-statistic ratio: 1.111, 95% CI 0.989-1.240) and 3L (F-statistic ratio: 1.102 95% CI 0.861-1.383) across levels of general health. The 5L was the most responsive value set between EQ VAS values of 25 and 75. CONCLUSION: These results imply greater sensitivity of the 5L to health changes and potentially lower incremental cost-utility ratios compared to the 3L.

Normal B-cell ranges in infants: A systematic review and meta-analysis.

BACKGROUND: During the first year of life, B-cell level is a valuable indicator of whether external factors, such as exposure to B-cell-depleting therapies, have an adverse impact on immune system development. However, there are no standard reference ranges of B-cell levels in healthy infants by age. OBJECTIVE: Our aim was to estimate the normal range of B-cell levels in infants, by age, during the first year of life by pooling data from published studies. METHODS: Studies reporting B-cell levels measured by using flow cytometry and CD19 markers in healthy infants were identified via a systematic literature review. Quality and feasibility assessments determined suitability for inclusion in meta-analyses by age group and/or continuous age. Means and normal ranges (2.5th-97.5th percentile) were estimated for absolute and percentage B-cell levels. Sensitivity analyses assessed the impact of various assumptions. RESULTS: Of the 37 relevant studies identified, 28 were included in at least 1 meta-analysis. The means and normal ranges of B-cell levels were found to be 707 cells/µL in cord blood (range 123-2324 cells/µL), 508 cells/µL in infants aged 0 to 1 month (range 132-1369 cells/µL), 1493 cells/µL in infants aged 1 to 6 months (range 416-3877 cells/µL), and 1474 cells/µL in infants older than 6 months (range 416-3805 cells/µL). The continuous age model showed that B-cell levels peaked at week 26. Trends were similar for the percentage B-cell estimates and in sensitivity analyses. CONCLUSION: These meta-analyses provide the first normal reference ranges for B-cell levels in infants, by week of age, during the first year of life.

Estimating the EQ-5D-5L value set for the Philippines.

BACKGROUND: The Philippines has recommended the use of Quality-Adjusted Life Years (QALYs) in government health technology assessments (HTA). We aimed to develop a value set for the EQ-5D-5L based on health preferences of the healthy general adult population in the Philippines. METHODS: Healthy, literate adults were recruited from the Philippine general population with quota targets based on age, sex, administrative region, type of residence, education, income, and ethnolinguistic groups. Each participant's preference was elicited by completing Composite Time Trade-Off (C-TTO) and Discrete Choice Experiment (DCE) tasks. Tasks were computer-assisted using the EuroQol Valuation Technology 2.0. To estimate the value set, we explored 20- and 8-parameter models that either use c-TTO-only data or both c-TTO and DCE (also called hybrid models). Final model choice was guided by principles of monotonicity, out-of-sample likelihood, model fit, and parsimony. RESULTS: We recruited 1000 respondents with demographic characteristics that approximate the general population such as 49.6% Female, 82% Roman Catholic, 40% in urban areas, and 55% finished high school. None of the 20-parameter models demonstrated monotonicity (logical worsening of coefficients with increasing severity). From the 8-parameter models, the homoscedastic TTO-only model exhibited the best fit. From this model, mobility and pain/ discomfort had the highest effect on utilities. CONCLUSION: The selected model for representing the Philippine general population preferences for EQ-5D-5L health states was an 8-parameter homoscedastic TTO-only model. This value set is recommended for use in QALY calculations in support of HTA-informed coverage decisions in the Philippines.

Investigating 5-Level EQ-5D (EQ-5D-5L) Values Based on Preferences of Patients With Heart Disease.

OBJECTIVES: Several studies have shown that patients with heart disease value hypothetical health states differently from the general population. We aimed to investigate the health preferences of patients with heart disease and develop a value set for the 5-level EQ-5D (EQ-5D-5L) based on these patient preferences. METHODS: Patients with confirmed heart disease were recruited from 2 hospitals in Singapore. A total of 86 EQ-5D-5L health states (10 per patient) were valued using a composite time trade-off method according to the international valuation protocol for EQ-5D-5L; 20-parameter linear models and 8-parameter cross-attribute level effects models with and without an N45 term (indicating whether any health state dimension at level 4 or 5 existed) were estimated. Each model included patient-specific random intercepts. Model performance was evaluated for out-of-sample and in-sample predictive accuracy in terms of root mean square error. The discriminative ability of the utility values was assessed using heart disease-related functional classes. RESULTS: A total of 576 patients were included in the analysis. The preferred model, with the lowest out-of-sample root mean square error, was a 20-parameter linear model including N45. Predicted utility values ranged from -0.727 for the worst state to 1 for full health; the value for the second-best state was 0.981. Utility values demonstrated good discriminative ability in differentiating among patients of varied functional classes. CONCLUSIONS: An EQ-5D-5L value set representing the preferences of patients with heart disease was developed. The value set could be used for patient-centric economic evaluation and health-related quality of life assessment for patients with heart disease.