Cost Effectiveness of Sequencing Vedolizumab as First-Line Biologic in Ulcerative Colitis and Crohn's Disease in Canada: An Analysis Using Real-World Evidence from the EVOLVE Study.

INTRODUCTION: Vedolizumab is a gut-selective anti-lymphocyte trafficking biologic indicated for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) and Crohn's disease (CD) in Canada. OBJECTIVE: The objective of this study was to evaluate the cost effectiveness of treatment sequencing for UC and CD from a public healthcare payer perspective, leveraging new real-world evidence from the literature and the EVOLVE study, a retrospective chart review. METHODS: Using separate decision tree/Markov models to assess cost effectiveness for UC and CD, two sequencing approaches were estimated for adult patients (≥ 18 years) diagnosed with UC or CD who were biologic-naïve: vedolizumab as first-line biologic followed by anti-tumor necrosis factor (TNF)-α versus first-line anti-TNFα followed by vedolizumab. Treatment effectiveness (response and remission), surgery rates, dose escalation and regain of response and safety inputs were estimated from EVOLVE, a retrospective chart review of real-world data, and evidence synthesis from the literature, whereas costs and utilities were estimated from health technology assessment reports, clinical trials, and the literature. Biosimilar costs were used for anti-TNFα. Both models simulated a 5-year time horizon and discounted costs and outcomes at 1.5%. Probabilistic base-case analyses (n = 10,000) reported total costs (2023 Canadian dollars) and quality-adjusted life-years (QALYs). Several scenario analyses were conducted to explore robustness of results. RESULTS: In UC, vedolizumab as a first-line biologic followed by anti-TNFα resulted in an incremental gain of 0.09 QALYs (2.46 vs. 2.55) and saved $7179 ($134,028 vs. $126,848), making this a dominant strategy compared with first-line anti-TNFα followed by vedolizumab. In CD, use of vedolizumab as a first-line biologic resulted in an incremental gain of 0.04 QALYs (3.35 vs. 3.39) at an incremental cost of $50,631 ($89,850 vs. $140,381) versus first-line anti-TNFα followed by vedolizumab (incremental cost-effectiveness ratio of $1,265,775 per QALY). CONCLUSIONS: Based on this analysis, sequencing vedolizumab as a first-line biologic prior to anti-TNFα in UC and CD provided additional clinical benefit to patients. In UC, vedolizumab as a first-line biologic also saved healthcare system costs compared with anti-TNFα, whereas in CD, vedolizumab provided incremental benefit at an incremental cost, which was not considered cost effective at a threshold of $50,000/QALY.

Correction: Valuation of the EQ-5D-5L in Taiwan.

[This corrects the article DOI: 10.1371/journal.pone.0209344.].

Health state utilities for beta-thalassemia: a time trade-off study.

BACKGROUND: Beta-thalassemia (BT) is an inherited blood disorder characterized by reduced levels of functional hemoglobin resulting in phenotypes ranging from clinically asymptomatic to severely anemic. Patients with BT may require lifelong regular blood transfusions supported by appropriate iron chelation therapy (ICT). This study aimed to determine how the UK general population values BT health states associated with differing transfusion burden and ICT. METHODS: Composite time trade-off (cTTO) methodology was employed to elicit health state utilities in BT. Relevant BT literature related to symptom and quality-of-life impact, including physical, functional, and emotional well-being, and safety profiles of BT treatments were considered when drafting health state descriptions. Eleven health state descriptions were developed and validated by hematologists and patient advocates for clinical accuracy and completeness. 200 individuals from the UK general population participated in the cTTO interviews. RESULTS: The mean age of participants was 41.50 years (SD 16.01, range 18-81); 88 (46.8%) were female. Utility values ranged from 0.78 (SD 0.34) for non-transfusion dependent BT with oral ICT to 0.37 (SD 0.50) for high transfusion burden with subcutaneous ICT in transfusion-dependent BT. CONCLUSIONS: This study provides health utilities for a range of BT health states from the UK general population perspective. Importantly, lower transfusion burden and lower burden of anemia were associated with higher utilities. To a lesser extent, differential modes of ICT were found to impact utility valuations in patients with BT. The utilities obtained in this study can be employed as inputs in cost-effectiveness analyses of BT therapies.

An EQ-5D-Y-3L Value Set for Belgium.

BACKGROUND: In Belgium, a value set for children and adolescents for a generic health-related quality-of-life measure is not available. To inform drug pricing and make resource allocation decisions for children and adolescents, national tax-payers' preferences for youth health states should be known. OBJECTIVE: We aimed to obtain a value set for EQ-5D-Y-3L in Belgium, following the international youth valuation protocol for data collection. METHODS: Composite Time Trade-Off interviews were conducted in a sample of 200 adults, either face to face or via video conferencing. Another sample of 1000 adults completed an online discrete choice experiment survey. All adults were asked to take the perspective of a 10-year-old child for both methods. Both samples were representative for Belgium in terms of age, sex and region. A latent class analysis was selected to obtain the relative importance of the five dimensions and their levels based on the discrete choice experiment data, which were anchored with the composite Time Trade-Off censored value for the worst health state (33333). RESULTS: Preferences from Belgian adults revealed a mean censored value for 33333 for children and adolescents of - 0.475. All the estimated coefficients of the model with 4 latent classes were statistically significant and showed higher disutility as severity levels increase. The most important health dimension was pain/discomfort, followed by feeling sad/worried/unhappy. CONCLUSIONS: This study presents the Belgian EQ-5D-Y-3L value set, which will be included in the Belgian pharmacoeconomic guidelines. The value set enables the calculation of quality-adjusted life-years in children and adolescents, allowing a cost-effectiveness evaluation of health technologies and their youth-specific price setting.

Accounting for Unobservable Preference Heterogeneity and Evaluating Alternative Anchoring Approaches to Estimate Country-Specific EQ-5D-Y Value Sets: A Case Study Using Spanish Preference Data.

OBJECTIVES: The EuroQol Group published the EQ-5D-Y valuation protocol that recommends 2 valuation techniques to elicit preferences: composite time trade-off (C-TTO) and discrete choice experiments (DCEs). The protocol left the decision of what modeling approach to use open for researchers. Our aims were to explore modeling strategies allowing generation of EQ-5D-Y value sets and to produce an EQ-5D-Y Spanish value set. METHODS: We used EQ-5D-Y DCE and C-TTO data collected in Spain following the protocol and adopted a staged approach for our modeling exercise. First, we selected the best performing DCE latent class model and evaluated models from 2 to 10 classes. We selected the preferred model based on best goodness of fit in terms of the Bayesian information criterion. We considered 2 anchoring approaches to estimate utility values: (1) pits state anchoring and (2) hybrid models (using all available C-TTO responses). All analysis were weighted to be representative of the Spanish population. RESULTS: We collected 1005 DCE and 200 C-TTO interviews. We selected a DCE model including 4 classes. Hybrid models using all available C-TTO observations produced a narrower range of values than the pits state anchoring approach. CONCLUSIONS: In this article, we have presented an EQ-5D-Y value set that can be used for cost-utility analysis in Spain. The international EQ-5D-Y valuation protocol should be updated to include a different set of health states for the C-TTO experiment if researchers wish to use alternative anchoring approaches to the "pits state."

Does Changing the Age of a Child to be Considered in 3-Level Version of EQ-5D-Y Discrete Choice Experiment-Based Valuation Studies Affect Health Preferences?

OBJECTIVES: There has been some debate about the choice of perspective and the age of the child considered when completing preference elicitation tasks in the 3-level version of EQ-5D-Y (EQ-5D-Y-3L) valuation protocol. This study aimed to clarify the impact on latent scale EQ-5D-Y-3L values of varying the age of the child experiencing the health state considered by respondents completing the discrete choice experiment (DCE) tasks of the protocol. METHODS: We conducted an online DCE with a representative sample of 1000 adults in the United Kingdom and 1000 adults in the United States. Respondents selected the health state they prefer from a series of DCE paired EQ-5D-Y-3L health state comparisons using their own perspective and that of a hypothetical child from the following age groups: "5-7 years old," "8-10 years old," "11-13 years old," and "14-15 years old." Data analysis was conducted using separate multinomial logit models for each perspective and country. We also estimated combined models including data from each possible pair of perspectives and used interactions between EQ-5D-Y-3L levels and perspective to determine whether any differences were statistically significant. RESULTS: No statistically significant differences in coefficients between perspectives were found in the United States. In the United Kingdom, there were differences between the own perspective and the 5 to 7 years old perspective (looking after myself level 3) and between the 5 to 7 years old perspective and the 8 to 10 years old perspective (usual activities level 3). CONCLUSIONS: Our results suggest that there is minimal impact on latent scale values when using different ages of the hypothetical child in the current EQ-5D-Y-3L valuation protocol.

Exploring the Comparability of Face-to-Face Versus Video Conference-Based Composite Time Trade-Off Interviews: Insights from EQ-5D-Y-3L Valuation Studies in Belgium and Spain.

BACKGROUND: Face-to-face interviews are recommended for the collection of composite time trade-off (cTTO) data. The coronavirus disease 2019 (COVID-19) social distancing measures made researchers consider videoconferencing interviews as an alternative mode of administration, but little evidence about the implementation of videoconferencing in valuation studies is available. This study provides insights into the effect of videoconferencing on the quality of data, evaluating interviewers' and respondents' engagement level in videoconferences compared with face-to-face interviews. METHODS: We used cTTO data collected in Belgium and Spain following the EQ-5D-Y-3L valuation protocol. Due to the COVID-19 pandemic, both projects interrupted the face-to-face data collection before reaching the target sample. The remaining interviews were conducted by videoconference. We compared both modes of administration in terms of interviewers' engagement (task duration and number of moves in each example) and respondents' engagement (task duration and proportions of specific response values, in half-year units). To minimise interviewers' learning effects, we split our sample into three groups: (1) first 20 interviews conducted face-to-face; (2) subsequent interviews conducted face-to-face; and (3) videoconferencing interviews. RESULTS: The comparison between videoconferencing and subsequent face-to-face interviews showed the interviewer's engagement was not affected by the mode of administration as almost no significant results were found either in the task duration or the numbers of moves shown in the examples. Similarly, none of the respondents' task duration or proportion of specific responses or half-year units were affected by the mode of administration in either of the two countries. CONCLUSIONS: No evidence suggested that the quality of cTTO data is reduced when using videoconferencing compared with face-to-face interviews.

Socio-demographic indicators of self-reported health based on EQ-5D-3L: A cross-country analysis of population surveys from 18 countries.

Background: Generic health-related quality of life instruments, such as the EQ-5D, are increasingly used by countries to monitor population health via general population health surveys. Our aim was to demonstrate analytic options to measure socio-demographic differences in self-reported health using the EuroQol Group's archive of EQ-5D-3L population surveys that accumulated over the past two decades. Methods: Analyses captured self-reported EQ-5D-3L data on over 100,000 individuals from 18 countries with nationally representative population surveys. Socio-demographic indicators employed were age, sex, educational level and income. Logistic regression odds ratios and the health concentration index methodology were used in the socio-demographic analysis of EQ-5D-3L data. Results: Statistically significant socio-demographic differences existed in all countries (p < 0.01) with the EQ VAS based health concentration index varying from 0.090 to 0.157 across countries. Age had generally the largest contributing share, while educational level also had a consistent role in explaining lower levels of self-reported health. Further analysis in a subset of 7 countries with income data showed that, beyond educational level, income itself had an additional significant impact on self-reported health. Among the 5 dimensions of the EQ-5D-3L descriptive system, problems with usual activities and pain/discomfort had the largest contribution to the concentration of overall self-assessed health measured on the EQ VAS in most countries. Conclusion: The EQ-5D-3L was shown to be a powerful multi-dimensional instrument in the analyses of socio-demographic differences in self-reported health using various analytic methods. It offered a unique insight of inequalities by health dimensions.

To What Extent Do Patient Preferences Differ From General Population Preferences?

OBJECTIVES: In some countries including Germany, value sets based on general population preferences are not acceptable for decision-makers in healthcare because the impact of the reference group-general population versus patients-on utility decrements is questioned. The objective of this study was to explore potential differences in patient versus general population health preferences and a way of combining both preferences in economic evaluation. METHODS: EQ-5D-5L general population preferences were available from national value sets in Germany and Spain. Patient preferences were obtained by conducting discrete choice experiments with patients with rheumatism and patients with diabetes mellitus in Germany and Spain using an online panel. The econometric approach was based on the conditional logit framework. Latent values were anchored using the national value sets. RESULTS: A total of 1700 patients (Germany, n = 937; Spain, n = 763) were included in the analysis. In both countries, patients gave more importance to mobility, self-care, or usual activities and less importance to pain/discomfort and anxiety/depression than the general population. The size of these differences was larger in Germany than in Spain. In Germany, preferences reported by both patient groups were more similar than in Spain. CONCLUSION: Patient preferences differ from preferences derived from the general population. In contrast to the general population, patients gave more importance to the functional dimensions than to symptoms in both countries. The extent of the differences depends on the disease and the country. For countries preferring patient preferences, a possible way of incorporating the patient perspective in health state valuation was suggested and needs to be further explored.

Comparison of Adult and Adolescent Preferences Toward EQ-5D-Y-3L Health States.

OBJECTIVES: The international EQ-5D-Y-3L valuation protocol suggests obtaining preferences for EQ-5D-Y-3L health states from a sample of the general adult population. There is discussion around involving children and adolescents in the processes of preference elicitation and decision making. The objective of this study was to compare the preferences for EQ-5D-Y-3L obtained from adolescents for themselves to those of adults considering a hypothetical 10-year-old child in Germany, Slovenia, and Spain. METHODS: A sample of a minimum of 700 adolescents and 1000 adults in each country was recruited through online panels. An online discrete choice experiment was used to obtain health-state preferences. For the purposes of comparison, all coefficients were rescaled to a 1 (best) to 0 (worst) scale. The differences between preferences in both samples were analyzed via the relative attribute importance of health dimensions from the mixed logit models. RESULTS: Statistically significant differences between the preferences for EQ-5D-Y-3L states given by the 2 samples were observed in all 3 countries. The overall relative attribute importance was similar between adolescents and adults; adolescents usually gave more importance to mobility and self-care, and less to anxiety/depression. The rank-order of the dimension levels between adults and adolescents differs in all 3 countries. CONCLUSIONS: Preferences toward EQ-5D-Y-3L states differ if estimated by adults taking the perspective of a child or by the adolescents themselves. Although it seems possible to obtain adolescents' preferences for inclusion in EQ-5D-Y-3L value sets, the desirability and acceptance of their preferences by researchers and decision makers need to be explored further.

Cost-effectiveness of adding Sativex® spray to spasticity care in Belgium: using bootstrapping instead of Monte Carlo simulation for probabilistic sensitivity analyses.

BACKGROUND: Uncertainty in model-based cost-utility analyses is commonly assessed in a probabilistic sensitivity analysis. Model parameters are implemented as distributions and values are sampled from these distributions in a Monte Carlo simulation. Bootstrapping is an alternative method that requires fewer assumptions and incorporates correlations between model parameters. METHODS: A Markov model-based cost-utility analysis comparing oromucosal spray containing delta-9-tetrahidrocannabinol + cannabidiol (Sativex®, nabiximols) plus standard care versus standard spasticity care alone in the management of multiple sclerosis spasticity was performed over a 5-year time horizon from the Belgian healthcare payer perspective. The probabilistic sensitivity analysis was implemented using a bootstrap approach to ensure that the correlations present in the source clinical trial data were incorporated in the uncertainty estimates. RESULTS: Adding Sativex® spray to standard care was found to dominate standard spasticity care alone, with cost savings of €6,068 and a quality-adjusted life year gain of 0.145 per patient over the 5-year analysis. The probability of dominance increased from 29% in the first year to 94% in the fifth year, with the probability of QALY gains in excess of 99% for all years considered. CONCLUSIONS: Adding Sativex® spray to spasticity care was found to dominate standard spasticity care alone in the Belgian healthcare setting. This study showed the use of bootstrapping techniques in a Markov model probabilistic sensitivity analysis instead of Monte Carlo simulations. Bootstrapping avoided the need to make distributional assumptions and allowed the incorporation of correlating structures present in the original clinical trial data in the uncertainty assessment.

International Valuation Protocol for the EQ-5D-Y-3L.

The EQ-5D-Y-3L is a generic, health-related, quality-of-life instrument for use in younger populations. Some methodological studies have explored the valuation of children's EQ-5D-Y-3L health states. There are currently no published value sets available for the EQ-5D-Y-3L that are appropriate for use in a cost-utility analysis. The aim of this article was to describe the development of the valuation protocol for the EQ-5D-Y-3L instrument. There were several research questions that needed to be answered to develop a valuation protocol for EQ-5D-Y-3L health states. Most important of these were: (1) Do we need to obtain separate values for the EQ-5D-Y-3L, or can we use the ones from the EQ-5D-3L? (2) Whose values should we elicit: children or adults? (3) Which valuation methods should be used to obtain values for child's health states that are anchored in Full health = 1 and Dead = 0? The EuroQol Research Foundation has pursued a research programme to provide insight into these questions. In this article, we summarized the results of the research programme concluding with the description of the features of the EQ-5D-Y-3L valuation protocol. The tasks included in the protocol for valuing EQ-5D-Y-3L health states are discrete choice experiments for obtaining the relative importance of dimensions/levels and composite time-trade-off for anchoring the discrete choice experiment values on 1 = Full Health and 0 = Dead. This protocol is now available for use by research teams to generate EQ-5D-Y-3L value sets for their countries allowing the implementation of a cost-utility analysis for younger populations.

A Qualitative Research for Defining Meaningful Attributes for the Treatment of Inflammatory Bowel Disease from the Patient Perspective.

INTRODUCTION: Crohn's disease (CD) and ulcerative colitis (UC) are chronic, inflammatory bowel diseases (IBD). Each class and type of medication available for the treatment of IBD has distinct characteristics and long-term effects that a patient may consider. We present the results of qualitative research that aimed to develop a descriptive framework that outlines the most relevant disease and/or treatment attributes for IBD treatment decisions and focuses on the patient perspective. METHODS: This research employed a three-step approach: a literature review to identify a broad list of attributes, a focus group meeting including patients and clinicians to assess the relevance of the attributes, and two rounds of voting to name and define each attribute. The literature review was used to develop the initial list of attributes. Although the same attributes were defined for both UC and CD, the relative importance of each attribute to UC or CD was considered. The list of attributes was discussed and evaluated in the focus group meeting, which included eight patient representatives and nine gastroenterologists. Using feedback elicited from the focus group meeting, the research team developed a draft of the descriptive framework that grouped the attributes into domain subsets. All members of the focus group participated in two subsequent rounds of structured, online voting, which was used to refine the wording to name and define each attribute. Additionally, participants ranked all the attributes included in the descriptive framework to suggest which attributes were less relevant and could be omitted. RESULTS: Among 574 publications retrieved from the databases and registries, we identified 32 eligible publications, and an initial list of attributes was developed. This list was refined during the focus group meeting, resulting in a draft descriptive framework of attributes within subsets of domains. The final descriptive framework was developed based on structured rounds of online voting to further refine attribute names and definitions. In the final descriptive framework, a total of ten attributes were identified: abdominal pain, other disease-related pain, bowel urgency, fatigue, risk of cancer and serious infections within the next 10 years, risk of mild to moderate complications, aesthetic complications related to treatment, emotional status, sexual life, and social life and relationships. These attributes were distributed across three domains: efficacy, complications and risk, and health-related quality of life. CONCLUSIONS: Through the identification of the ten most relevant attributes that influence patient decision making for IBD treatments, we developed a descriptive framework that should be considered by physicians when discussing IBD treatment options with their patients. The results of our qualitative research may also be helpful for the development of future IBD clinical studies and quantitative research.

Normative Profile of Health-Related Quality of Life for Hong Kong General Population Using Preference-Based Instrument EQ-5D-5L.

OBJECTIVES: To establish a normative profile of health-related quality of life (HRQoL) for Hong Kong (HK) Chinese residents aged 18 years and above and to examine the relationship between socioeconomic characteristics and health conditions and the preference-based health index. METHODS: We recruited 1014 representative Cantonese-speaking residents across 18 geographical districts. The normative profiles of HRQoL were derived using established HK value sets. Mean values were computed by sex, age group, and educational attainment to obtain the EQ-5D HK normative profile for the general HK population. To explore the relationships among potential covariates (socioeconomic characteristics and health conditions) and the HK health index, a multivariable homoscedastic Tobit regression model was employed for the analysis. RESULTS: The mean index value was 0.919 using the EQ-5D-5L HK value set. Younger ages reported greater problems with anxiety or depression than did older ages, whereas older ages reported greater problems with pain or discomfort than did younger ages. Persons with higher educational attainment and those who reported higher life satisfaction reported significantly higher health index scores (P < .05). On the contrary, receiving government allowance and having experienced a serious illness were significantly associated (P < .05) with a lower health index. CONCLUSIONS: The norm values fully represent the societal preferences of the HK population, and knowledge of societal preferences can enable policy makers to allocate resources and prioritize service planning. The study was conducted with the EuroQol International EQ-5D-5L Valuation Protocol and therefore enabled us to compare the EQ-5D-5L values with other countries to facilitate understanding of societal preferences in different jurisdictions.

A hybrid modelling approach for eliciting health state preferences: the Portuguese EQ-5D-5L value set.

BACKGROUND: The EQ-5D is a generic preference-based quality of life measure considered useful for supporting clinical and policy decisions by providing utility values that can easily be converted into quality-adjusted life years to be integrated in cost-utility economic evaluations. Although the three-level classification system of the EuroQol questionnaire (EQ-5D-3L) is still the most popular preference-based instrument used worldwide, several studies reported a ceiling effect on this version, especially in healthy and/or young individuals. In 2009, the EuroQol Group introduced a five-level EQ-5D, which expands the descriptive system from three to five levels within the same five dimensions. For this version to be used in health economic evaluation, societal values need to be assigned to the 3125 health states generated by this instrument. OBJECTIVES: The aims of this study were to elicit the EQ-5D-5L health state preferences from the general Portuguese population and to derive the Portuguese value set for the EQ-5D-5L. METHODS: A representative sample of the Portuguese general population aged above 18 years was stratified by age and gender (n = 1451). Between October 2015 and July 2016, 28 interviewers carried out a series of 1-h-long computer-assisted personal interviews following the EuroQol Valuation Technology protocol. Each interview included the valuation of ten health states using the composite time trade-off (cTTO) and seven pairs of discrete choice experiments (DCEs). A standardized tool for quality control was used to assess the quality of the data as well as direct supervision and cross-examination of 10% of the global sample size. Data from both cTTO and DCE valuation tasks were modelled using a censored heteroskedastic hybrid model. RESULTS: Interviewers complied with the quality control protocol in providing high-quality valuation data. The hybrid econometric model had consistent and significant parameters. The derived societal values for the Portuguese population ranged from - 0.603 to 1. CONCLUSION: This study provided the Portuguese value set for the EQ-5D-5L on the basis of a hybrid econometric model using cTTO and DCE data. These results represent the preferences of the Portuguese population and are recommended to inform health decision-making in Portugal.

Inequalities in health-related quality of life according to age, gender, educational level, social class, body mass index and chronic diseases using the Spanish value set for Euroquol 5D-5L questionnaire.

BACKGROUND: Reducing health inequalities on the basis of social factors has been a key driver in the development of Public Health policies. Health-related quality of life is a global indicator useful to assess health inequalities within a society. The objective of this study was to identify inequalities on health by analysing the interactive effects of gender, age, educational level, social class, body mass index and chronic diseases on health-related quality of life in a Spanish population sample. METHODS: We used data from the Spanish National Health Survey 2011-2012. Health-related quality of life was measured by the EQ-5D-5L instrument applying the Spanish value set. Probability of being in perfect health was ascertained by logistic regression models including gender, age, educational level, body mass index and social class and the corresponding terms of interaction. A two-part model combining logistic regression analysis and generalized linear models was applied to calculate the adjusted utility loss associated with chronic conditions (disutility values). RESULTS: The sample used for analysis contained 18,450 individuals. The mean age was 50 years, 51.3% were women, 55% were overweight or obese and 46.7% had low social status. The mean utility was 0.94 in men and 0.89 in women. Elderly women, obese people, those of low social class and those with chronic conditions had significant lower utility values. Within the regression analysis, interaction assessment revealed that the detrimental effect of obesity disappeared in higher social classes. Utility values for all chronic conditions considered were lower in women than in men and were on a gradient within social class, the lowest for individuals declaring stroke. The greatest decrease on health-related quality of life was determined by declaration of stroke (17.6%) or mental diseases (18.6%). CONCLUSIONS: The interactive effects of gender, age, educational level, social class, body mass index and chronic diseases on health-related quality of life in the Spanish population revealed important inequalities in health. Social class acted as a modulator of the stigma associated with obesity. Chronic conditions producing loss of autonomy had the greatest impact on reduction of health-related quality of life. This is the first study using the Spanish EQ-5D-5L value set to estimate utilities.

Valuation of the EQ-5D-5L in Taiwan.

OBJECTIVES: To date, a value set for the EQ-5D-5L based on the health state preferences of the general Taiwanese population has not been available. This study aimed to develop a Taiwanese value set for EQ-5D-5L to facilitate health technology assessment for medical products and services. METHODS: An international standardized protocol for EQ-5D-5L valuation studies developed by the EuroQol group was adopted. Adult members of the general public were recruited from six geographic regions in Taiwan. In computer-based face-to-face interviews, each participant completed 10 composite time trade-off (C-TTO) tasks and 7 discrete choice experiment (DCE) tasks. The C-TTO and DCE data were modeled alone or in combination (using hybrid models) with additive models containing 20 dummy variables as main effects. The model performance was assessed both quantitatively and qualitatively (mainly logical consistency and prediction patterns). RESULTS: Of 1,073 recruited participants, 1,000 completed the study. Approximately 13% of observed utility values were -1 in the C-TTO tasks. The hybrid model, using all available data that assumed C-TTO response values left-censored at -1 and with main effects coefficients with logical consistency (monotonicity), was considered as the most appropriate model. The predicted utility ranged from -1.0259 to 1. CONCLUSIONS: An EQ-5D-5L value set was developed for Taiwan using an established study protocol and a representative sample of the general population. This may facilitate health economic evaluations and decision making on resource allocation under Taiwan's national health insurance program in the future.

Valuation of EuroQol Five-Dimensional Questionnaire, Youth Version (EQ-5D-Y) and EuroQol Five-Dimensional Questionnaire, Three-Level Version (EQ-5D-3L) Health States: The Impact of Wording and Perspective.

BACKGROUND: Valuations of health states were affected by the wording of the two instruments (EQ-5D-3L and EQ-5D-Y) and by the perspective taken (child or adult). OBJECTIVES: There is a growing demand for value sets for the EQ-5D-Y (EQ-5D instrument for younger populations). Given the similarities between EQ-5D-Y and EQ-5D-3L, we investigated whether valuations of health states were affected by the differences in wording between the two instruments and by the perspective taken in the valuation exercise (child or adult). STUDY DESIGN: Respondents were randomly assigned to EQ-5D-3L or EQ-5D-Y (instrument) and further into two groups that either valued health states for an adult or for a 10-year-old child (perspective). The valuation tasks were composite time trade-off (C-TTO) and discrete choice experiments (DCE), including comparisons with death (DCE + death). Members of the adult general population in four countries (Germany, Netherlands, Spain, England) participated in computer-assisted personal interviews. METHODS: Two-way multivariate analysis of variance (MANOVA) and post hoc tests were used to compare C-TTO responses and chi-square tests were conducted to compare DCE + death valuations. RESULTS: A significant interaction effect between instrument and perspective for C-TTO responses was found. Significant differences by perspective (adult and child) occurred only for the EQ-5D-3L. Significant differences in values between instruments (EQ-5D-3L and EQ-5D-Y) occurred only for the adult perspective. Both significant results were confirmed by the DCE + death results. When comparing EQ-5D-3L for adult perspective and EQ-5D-Y for child perspective, values were also significantly different. CONCLUSIONS: The results identified an interaction effect between wording of the instrument and perspective on elicited values, suggesting that current EQ-5D-3L value sets should not be employed to assign values to EQ-5D-Y health states.

Handling Data Quality Issues to Estimate the Spanish EQ-5D-5L Value Set Using a Hybrid Interval Regression Approach.

BACKGROUND: The Spanish five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) valuation study was the first to use the EuroQol Valuation Technology protocol, including composite time trade-off (C-TTO) and discrete choice experiments (DCE). In this study, its investigators noticed that some interviewers did not fully explain the C-TTO task to respondents. Evidence from a follow-up study in 2014 confirmed that when interviewers followed the protocol, the distribution of C-TTO responses widened. OBJECTIVES: To handle the data quality issues in the C-TTO responses by estimating a hybrid interval regression model to produce a Spanish EQ-5D-5L value set. METHODS: Four different models were tested. Model 0 integrated C-TTO and DCE responses in a hybrid model and models 1 to 3 altered the interpretation of the C-TTO responses: model 1 allowed for censoring of the C-TTO responses, whereas model 2 incorporated interval responses and model 3 included the interviewer-specific protocol violations. For external validation, the predictions of the four models were compared with those of the follow-up study using the Lin's concordance correlation coefficient. RESULTS: This stepwise approach to modeling C-TTO and DCE responses improved the concordance between the valuation and follow-up studies (concordance correlation coefficient: 0.948 [model 0], 0.958 [model 1], 0.952 [model 2], and 0.989 [model 3]). We recommend the estimates from model 3, because its hybrid interval regression model addresses the data quality issues found in the valuation study. CONCLUSIONS: Protocol violations may occur in any valuation study; handling them in the analysis can improve external validity. The resulting EQ-5D-5L value set (model 3) can be applied to inform Spanish health technology assessments.

Choice Defines Value: A Predictive Modeling Competition in Health Preference Research.

OBJECTIVE: To identify which specifications and approaches to model selection better predict health preferences, the International Academy of Health Preference Research (IAHPR) hosted a predictive modeling competition including 18 teams from around the world. METHODS: In April 2016, an exploratory survey was fielded: 4074 US respondents completed 20 out of 1560 paired comparisons by choosing between two health descriptions (e.g., longer life span vs. better health). The exploratory data were distributed to all teams. By July, eight teams had submitted their predictions for 1600 additional pairs and described their analytical approach. After these predictions had been posted online, a confirmatory survey was fielded (4148 additional respondents). RESULTS: The victorious team, "Discreetly Charming Econometricians," led by Michal Jakubczyk, achieved the smallest χ2, 4391.54 (a predefined criterion). Its primary scientific findings were that different models performed better with different pairs, that the value of life span is not constant proportional, and that logit models have poor predictive validity in health valuation. CONCLUSIONS: The results demonstrated the diversity and potential of new analytical approaches in health preference research and highlighted the importance of predictive validity in health valuation.