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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.

Background An important question in the valuation of children’s health is whether the preferences of younger individuals should be captured within value sets for measures that are aimed at them. This depends on whether younger individuals can complete valuation exercises and whether their preferences differ from those of adults. This study compared the preferences of adults and adolescents for EQ-5D-Y-3L health states using latent scale values elicited from a discrete choice experiment (DCE). Methods An online DCE survey, comprising 15 pairwise choices, was provided to samples of UK adults and adolescents (aged 11–17 y). Adults considered the health of a 10-year-old child, whereas adolescents considered their own health. Mixed logit models were estimated, and comparisons were made using relative attribute importance (RAI) scores and a pooled model. Results In total, 1000 adults and 1005 adolescents completed the survey. For both samples, level 3 in pain/discomfort was most important, and level 2 in self-care the least important, based on the relative magnitudes of coefficients. The RAI scores (normalized on self-care) indicated that adolescents gave less weight relative to adults to usual activities (1.18 v. 1.51; P < 0.05), pain/discomfort (1.77 v. 3.12; P < 0.01), and anxiety/depression (1.64 vs. 2.65; P < 0.01). The pooled model indicated evidence of differences between the two samples in both levels in pain/discomfort and anxiety/depression. Limitations The perspective of the DCE task differed between the 2 samples, and no data were collected to anchor the DCE data to generate value sets. Conclusions Adolescents could complete the DCE, and their preferences differed from those of adults taking a child perspective. It is important to consider whether their preferences should be incorporated into value sets.

The introduction of the EQ-5D-5L offered an opportunity to develop a standardised valuation protocol, the EQ-VT protocol, with improved methods for health state valuation that enables comparison of the resulting value sets between countries. This chapter summarises the process of developing and strengthening the methods for valuing EQ-5D-5L in the EQ-VT protocol which underpins the valuation studies reported in this book. This includes an overview of the methodological research programme that informed the initial EQ-VT protocol and a description of the key elements of the protocol and the included valuation techniques, i.e. composite time trade-off and discrete choice experiments. This chapter also discusses the first wave of EQ-5D-5L valuation studies which used the protocol and the resulting conclusions; the subsequent modification and strengthening of the EQ-VT protocol including a quality control procedure; and experience with use of the improved EQ-VT protocol in the subsequent waves of EQ-5D-5L valuation studies. The chapter concludes with an overview of the lessons learned during this journey of evidence-based refinement of the EQ-VT protocol from version 1.0 to the current version 2.1.

Most of rare disease (RD) patients are assisted in their homes by their family as informal caregivers, causing a substantial burden among family members devoted to care. The role of informal caregivers has been associated with increased levels of stress, poor physical/mental health and impaired HRQOL. The present study assessed the impact on HRQOL and perceived burden of long-term informal caregiving, as well as the inter-relationships of individuals affected by different RD in six European countries, taking advantage of the data provided by the BURQOL-RD project (France, Germany, Italy, Spain, Sweden and UK). Correlation analysis was used to explore the relation between caregiver HRQOL and caregiver burden (Zarit Burden Interview). Multinomial logistic regression models were used to explore the role of explanatory variables on each domain of caregivers HRQOL measured by EQ-5D. Caregivers’ HRQOL is inversely correlated with burden of caring. Mobility dimension of EQ-5D was significantly associated with patients age, time devoted to care by secondary caregivers, patient gender and patient utility index. Patients’ age, burden scores and patient utility index significantly predict the capacity of caregivers to perform activities of daily living. Employed caregivers are less likely of reporting ‘slight problems’ in pain/discomfort dimensions than unemployed caregivers. The EQ-5D instrument is sensitive to measure differences in HRQOL between caregivers with different levels of burden of care.

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.

Background: Decision makers have huge problems when attempting to attribute social value to the improvements achieved by new drugs, especially when considering the use of orphan drugs for rare diseases. We present the results of a pilot study aimed to investigate patient preferences regarding public funding for drugs used to treat rare diseases.Methods: An online questionnaire was used as a discrete choice experiment (DCE) survey to explore the preferences of patients with cystic fibrosis and haemophilia in Italy. The questionnaire focused on relevant issues that were defined in a review of the literature. A conditional logistic model showed preferences for specific attributes.Results: A total of 54 questionnaires (20% response rate) were completed. The issues that received the greatest attention were improvement in health, treatment cost and value for money. However, disease severity and the availability of other treatments were important social values that could not be ignored.Conclusions: The findings presented here provide evidence as to what patients with cystic fibrosis or haemophilia think are the most important considerations on which to base decisions in health technology scenarios, and regarding the priorities for funding.

Newborn screening programs are a fundamental tool for secondary prevention or pre-symptomatic detection of certain conditions. The implementation of a newborn screening program requires an evaluation of effectiveness, safety, cost-effectiveness, feasibility and budget impact. Economic evaluation aims to contribute to the sustainability and solvency of health systems, especially when it comes to informing about financing health interventions with public funds. This funding must be justified on the basis of robust evidence of effectiveness, safety, cost-effectiveness, and acceptability. One of the most important limitations when evaluating the cost-effectiveness of a newborn screening program for hereditary disorders or congenital errors of metabolism is the scarcity of scientific evidence that limits the robustness of the economic analysis. Given the low availability of data, the use of expert opinion as a data source is unavoidable to complete the information. However, two main problems make it difficult to synthesize data obtained from various sources: biases and heterogeneity. Moreover, the measurement of quality-adjusted life years (QALYs) in pediatric populations poses serious methodological challenges. In Spain, although there is some heterogeneity in the supply of newborn screening programs between regions, guidelines are being established based on the best available scientific evidence to achieve the homogenization of newborn screening policies and programs at national level.Los programas de cribado neonatal son una herramienta fundamental para la prevención secundaria o detección presintomática de determinadas afecciones. La implantación de un programa de cribado neonatal requiere necesariamente de una evaluación de su efectividad, seguridad, coste-efectividad, factibilidad e impacto presupuestario. La evaluación económica pretende contribuir a la sostenibilidad y solvencia de los sistemas sanitarios, especialmente a la hora de informar sobre la posible financiación, con fondos públicos, de intervenciones sanitarias como el cribado poblacional. Esta financiación debe justificarse en base a pruebas robustas de efectividad, seguridad, coste-efectividad y aceptabilidad. Una de las limitaciones más importantes a la hora de evaluar el coste-efectividad de un programa de cribado neonatal de trastornos hereditarios o de errores congénitos del metabolismo es la escasez de evidencia científica que limita la solidez y robustez del análisis de evaluación económica. Dada la baja disponibilidad de datos, el uso de la opinión de expertos como fuente de datos es inevitable para completar la información necesaria. Sin embargo, dos problemas principales dificultan la síntesis de datos obtenidos de varias fuentes: sesgos y heterogeneidad. Por otro lado, la medición de los años de vida ajustados por calidad (AVAC) en poblaciones pediátricas plantea serios desafíos metodológicos en un análisis de evaluación económica. En España, aunque existe cierta heterogeneidad en la oferta de programas de cribado neonatal entre CC.AA., se están estableciendo directrices basadas en la mejor evidencia científica disponible para conseguir la homogeneización de políticas y programas de cribado neonatal a nivel nacional.

Objectives Discrete choice experiments (DCEs) can be used to obtain latent scale values for the EQ-5D-Y, but these require anchoring at 0 = dead to meet the conventions of quality-adjusted life year (QALY) estimation. The primary aim of this study is to compare four preference elicitation methods for obtaining anchors for latent scale EQ-5D-Y values. Methods Four methods were tested: visual analogue scale (VAS), DCE (with a duration attribute), lag-time time trade-off (TTO) and the location-of-dead (LOD) approach. In computer-assisted personal interviews, UK general public respondents valued EQ-5D-3L health states from an adult perspective and EQ-5D-Y health states from a 10-year-old child perspective. Respondents completed valuation tasks using all four methods, under both perspectives. Results 349 interviews were conducted. Overall, respondents gave lower values under the adult perspective compared to the child perspective, with some variation across methods. The mean TTO value for the worst health state (33333) was about equal to dead in the child perspective and worse than dead in the adult perspective. The mean VAS rescaled value for 33333 was also higher in the child perspective. The DCE produced positive child perspective values and negative adult perspective values, though the models were not consistent. The LOD median rescaled value for 33333 was negative under both perspectives and higher in the child perspective. Discussion There was broad agreement across methods. Potential criteria for selecting a preferred anchoring method are presented. We conclude by discussing the decision-making circumstances under which utilities and QALY estimates for children and adults need to be commensurate to achieve allocative efficiency.

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. EuroQol Research Foundation info:eu-repo/semantics/publishedVersion

Background The EQ-5D is one of the most used generic health-related quality-of-life (HRQOL) instruments worldwide. To make the EQ-5D suitable for use in economic evaluations, a societal-based value set is needed. Indonesia does not have such a value set. Objective The aim of this study was to derive an EQ-5D-5L value set from the Indonesian general population. Methods A representative sample aged 17 years and over was recruited from the Indonesian general population. A multi-stage stratified quota method with respect to residence, gender, age, level of education, religion and ethnicity was utilized. Two elicitation techniques, the composite time trade-off (C-TTO) and discrete choice experiments (DCE) were applied. Interviews were undertaken by trained interviewers using computer-assisted face-to-face interviews with the EuroQol Valuation Technology (EQ-VT) platform. To estimate the value set, a hybrid regression model combining C-TTO and DCE data was used. Results A total of 1054 respondents who completed the interview formed the sample for the analysis. Their characteristics were similar to those of the Indonesian population. Most self-reported health problems were observed in the pain/discomfort dimension (39.66%) and least in the self-care dimension (1.89%). In the value set, the maximum value was 1.000 for full health (health state ‘11111’) followed by the health state ‘11112’ with value 0.921. The minimum value was −0.865 for the worst state (‘55555’). Preference values were most affected by mobility and least by pain/discomfort. Conclusions We now have a representative EQ-5D-5L value set for Indonesia. We expect our results will promote and facilitate health economic evaluations and HRQOL research in Indonesia. Electronic supplementary material The online version of this article (doi:10.1007/s40273-017-0538-9) contains supplementary material, which is available to authorized users.

Introduction. Health utilities are widely used in health care. The distributions of utilities differ between countries; some countries more often report worse than dead health states, while mild states are valued more or less the same. We hypothesize that cultural values explain these country-related utility differences. Research Question. What is the effect of sociodemographic background, methodological factors, and cultural values on differences in health utilities? Methods and Analyses. Time tradeoff data from 28 EQ-5D valuation studies were analyzed, together with their sociodemographic variables. The dependent variable was (Formula presented.), the utility difference between mild and severe states. Country-specific cultural variables were taken from the World Values Survey. Multilevel models were used to analyze the effect of sociodemographic background, methodology (3L v. 5L), and cultural values on (Formula presented.). Intraclass correlation (ICC) for country variation was used to assess the impact of the predicting variables on the variation between countries. Results. Substantial variation in (Formula presented.) was found between countries. Adding cultural values did not reduce ICCs for country variation. Sociodemographic background variables were only weakly associated with (Formula presented.) and did not affect the ICC. (Formula presented.) was 0.118 smaller for EQ-5D-5L studies. Discussion. (Formula presented.) varies between countries. These differences were not explained by national cultural values. In conclusion, despite correction for various variables, utility differences between countries remain substantial and unexplained. This justifies the use of country-specific value sets for instruments such as the EQ-5D. Fil: Roudijk, Bram. Radboud University Medical Center; Países Bajos Fil: Donders, A. Rogier T.. Radboud University Medical Center; Países Bajos Fil: Stalmeier, Peep F. M.. Radboud University Medical Center; Países Bajos Fil: Luo, Nan. Radboud University Medical Center; Países Bajos Fil: Viney, Rosalie. Radboud University Medical Center; Países Bajos Fil: Andrade, Monica Viegas. Radboud University Medical Center; Países Bajos Fil: Gudex, Claire. Radboud University Medical Center; Países Bajos Fil: de Pouvourville, Gerard. Radboud University Medical Center; Países Bajos Fil: Greiner, Wolfgang. Radboud University Medical Center; Países Bajos Fil: Scalone, Luciana. Radboud University Medical Center; Países Bajos Fil: Tsuchiya, Aki. Radboud University Medical Center; Países Bajos Fil: Golicki, Dominik. Radboud University Medical Center; Países Bajos Fil: Ferreira, Pedro. Radboud University Medical Center; Países Bajos Fil: Prevolnik-Rupel, Valentina. Radboud University Medical Center; Países Bajos Fil: Badia, Xavier. Radboud University Medical Center; Países Bajos Fil: Hsieh, Ching-Lin. Radboud University Medical Center; Países Bajos Fil: Jelsma, Jennifer. Radboud University Medical Center; Países Bajos Fil: Santos, Marisa. Radboud University Medical Center; Países Bajos Fil: Xie, Feng. Radboud University Medical Center; Países Bajos Fil: Purba, Fredrick. Radboud University Medical Center; Países Bajos Fil: Ikeda, Shunya. Radboud University Medical Center; Países Bajos Fil: Shiroiwa, Takeru. Radboud University Medical Center; Países Bajos Fil: Stolk, Elly. Radboud University Medical Center; Países Bajos Fil: Jo, Min-Woo. Radboud University Medical Center; Países Bajos Fil: Ramos Goñi, Juan Manuel. Radboud University Medical Center; Países Bajos Fil: Augustovski, Federico Ariel. Consejo Nacional de Investigaciones Científicas y Técnicas. Oficina de Coordinación Administrativa Parque Centenario. Centro de Investigaciones en Epidemiología y Salud Pública. Instituto de Efectividad Clínica y Sanitaria. Centro de Investigaciones en Epidemiología y Salud Pública; Argentina Fil: Rey Ares, Lucila. Radboud University Medical Center; Países Bajos Fil: Devlin, Nancy. Radboud University Medical Center; Países Bajos Fil: Shah, Koonal. Radboud University Medical Center; Países Bajos Fil: Pattanaphesaj, Juntana. Radboud University Medical Center; Países Bajos Fil: Tongsiri, Sirinart. Radboud University Medical Center; Países Bajos

A standardized 5-level EuroQol 5-dimensional questionnaire (EQ-5D-5L) valuation protocol was first used in national studies in the period 2012 to 2013. A set of problems encountered in this initial wave of valuation studies led to the subsequent refinement of the valuation protocol. To clarify lessons learned and how the protocol was updated when moving from version 1.0 to the current version 2.1 and 2.0, this article will (1) present the challenges faced in EQ-5D-5L valuation since 2012 and how these were resolved and (2) describe in depth a set of new challenges that have become central in currently ongoing research on how EQ-5D-5L health states should be valued and modeled. Copyright (c) 2019, ISPOR-The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

In this article, we describe the eq5dds command, which presents the results of a descriptive system using individuals’ responses for mobility, self care, usual activities, pain or discomfort, anxiety or depression, and visual analog scale from the EQ-5D health-related quality-of-life instrument (in its 3L and 5L versions). The command presents each of the tables and the figures recommended in the official user guide of the instrument (developed by the EuroQol Group).

The time trade-off (TTO) valuation technique is widely used to determine utility values of health outcomes to inform quality-adjusted life-year (QALY) calculations for use in economic evaluation. Protocols for implementing TTO vary in aspects such as the trade-off framework, iteration procedure and its administration model and method, training of respondents and interviewers, and quality control of data collection. The most widely studied and utilized TTO valuation protocols are the Measurement and Valuation of Health (MVH) protocol, the Paris protocol and the EuroQol Valuation Technology (EQ-VT) protocol, all developed by members of the EuroQol Group. The MVH protocol and its successor, the Paris protocol, were developed for valuation of EQ-5D-3L health states. Both protocols were designed for a trained interviewer to elicit preferences from a respondent using the conventional TTO framework with a fixed time horizon of 10 years and an iteration procedure combining bisection and titration. Developed for valuation of EQ-5D-5L health states, the EQ-VT protocol adopted a composite TTO framework and made use of computer technology to facilitate data collection. Training and monitoring of interviewers and respondents is a pivotal component of the EQ-VT protocol. Research is underway aiming to further improve the EuroQol protocols, which form an important basis for the current practice of health technology assessment in many countries.

At present, health technology assessment (HTA) guidelines of many countries including Thailand have recommended EQ-5D as the preferred method for assessing utility. This study aims to generate an EQ-5D-5L value set based on societal preferences of Thai population.A 1,207 representative sample was recruited using a stratified multi-stage quota sampling technique. Face-to-face, computer-assisted interviews using the EuroQol Valuation Technology (EQ-VT) software were employed. To elicit preference score, each respondent was asked to value health states using composite time trade-off (cTTO), and discrete choice experiment (DCE). All data were integrated and analyzed using a hybrid regression model to estimate the value set.Characteristics of 1,207 participants were generally similar to those of Thai general population. The coefficients generated from a hybrid model were logically consistent. The second best value is 0.9436 for health state 11121 and the worst state (55555) value is -0.4212. Mobility shows the greatest impact to utility decrement.Our study developed a Thai value set for EQ-5D using hybrid model. The findings from this study are of important to facilitate health technology assessment studies to inform policy decision-making as well as to promote the use of EQ-5D-5L in various health research in Thailand.

ObjectivesTo 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.MethodsAn 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).ResultsOf 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.ConclusionsAn 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.

Background An international valuation protocol exists for obtaining societal values for each of the 3125 health states of the five-level EuroQol-five dimensions (EQ-5D-5L) questionnaire. A feedback module (FM) that can be related to theoretical models used in behavioral economics was recently included in this protocol. Objectives Our objective was to assess the impact of using an FM to estimate an EQ-5D-5L value set in Hong Kong. Methods EQ-5D-5L health states were elicited using a composite time trade-off (C-TTO) and a discrete-choice (DC) experiment. Use of the FM according to participant characteristics and the impact of the FM on the number of inconsistent C-TTO responses were assessed. We employed a main-effects hybrid model that combined data from both elicitation techniques. Results In total, 1014 individuals completed the survey. The sample was representative of the general Chinese Hong Kong population in terms of sex, educational attainment, marital status, and most age groups but not for employment status. The use of the FM reduced the number of C-TTO inconsistencies. Participant characteristics differed significantly between those who used and did not use the FM. The model without a constant resulted in logical consistent coefficients and was therefore selected as the model to produce the value set. The predicted EQ-5D-5L values ranged from −0.864 to 1. Conclusions The use of an FM to allow participants to exclude C-TTO responses reduced the number of inconsistent responses and improved the quality of the data when estimating an EQ-5D-5L value set in Hong Kong. Electronic supplementary material The online version of this article (doi:10.1007/s40271-017-0278-0) contains supplementary material, which is available to authorized users.

To derive a value set from Uruguayan general population using the EQ-5D-5L questionnaire and report population norms. General population individuals were randomly assigned to value 10 health states using composite time trade off and 7 pairs of health states through discrete choice experiments. A stratified sampling with quotas by location, gender, age and socio-economic status was used to respect the Uruguayan population structure. Trained interviewers conducted face-to-face interviews. The EuroQol valuation technology was used to administer the protocol as well as to collect the data. OLS and maximum likelihood robust regression models with or without interactions were tested. We included 794 respondents between 20 and 83 years. Their characteristics were broadly similar to the Uruguayan population. The main effects robust model was chosen to derive social values. Values ranged from −0.264 to 1. States with a misery index = 6 had a mean predicted value of 0.965. When comparing the Uruguayan population with the Argentinian EQ-5D-5L crosswalk value set, the prediction for states which differed from full health only in having one of the dimensions at level 2 were about 0.05 higher in Uruguay. The mean index value, using the selected Uruguayan EQ-5D-5L value set, for the general population in Uruguay was 0.895. In general, older people had worse values and males had slightly better values than females. We derived the EQ-5D-5L Uruguayan value set, the first in Latin America. These results will help inform decision-making using economic evaluations for resource allocation decisions.

Objective To estimate the effect of sequence on response precision and response behavior in health valuation studies. Methods Time trade-off (TTO) and paired comparison responses from six health valuation studies—four US, one Spanish, and one Dutch—were examined (22,225 respondents) to test whether task sequence influences response precision (e.g., rounding), response changes, and median response times. Each study used a computer-based instrument that randomized task sequence among a national sample of adults, age 18 years or older, from the general population. Results For both TTO and paired comparisons, median response times decreased with sequence (i.e., learning), but tended to flatten after the first three tasks. Although the paired comparison evidence demonstrated that sequence had no effect on response precision, the frequency of rounded TTO responses (to either 1-year or 5-year units) increased with sequence. Conclusions Based on these results, randomizing or reducing the number of paired comparison tasks does not appear to influence response precision; however, generalizability, practicality, and precautionary considerations remain. Overall, participants learned to respond efficiently within the first three tasks and did not resort to satisficing, but may have rounded their TTO responses.

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.

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. Method: 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. Conclusion: 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..

Ranolazine is an antianginal agent that was approved in the EU in 2008 as an add-on therapy for symptomatic chronic angina pectoris treatment in patients who are inadequately controlled by, or are intolerant to, first-line antianginal therapies. These patients’ quality of life is significantly affected by more frequent angina events, which increase the risk of revascularization. To assess the cost-utility of ranolazine versus placebo as an add-on therapy for the symptomatic treatment of patients with chronic angina pectoris in Spain. A decision tree model with 1-year time horizon was designed. Transition probabilities and utility values for different angina frequencies were obtained from the literature. Costs were obtained from Spanish official DRGs for patients with chronic angina pectoris. We calculated the incremental cost-utility ratio of using ranolazine compared with a placebo. Sensitivity analyses, by means of Monte Carlo simulations, were performed. Acceptability curves and expected value of perfect information were calculated. The incremental cost-utility ratio was €8,455 per quality-adjusted life-year (QALY) per patient in Spain. Sensitivity analyses showed that if the decision makers’ willingness to pay is €15,000 per QALY, the treatment with ranolazine will be cost effective at a 95 % level of confidence. The incremental cost-utility ratio is particularly sensitive to changes in utility values of those non-hospitalized patients with mild or moderate angina frequency. Ranolazine is a highly efficient add-on therapy for the symptomatic treatment of chronic angina pectoris in patients who are inadequately controlled by, or intolerant to, first-line antianginal therapies in Spain.

Background The conventional method for modeling of the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) health state values in national valuation studies is an additive 20-parameter main-effects regression model. Statistical models with many parameters are at increased risk of overfitting—fitting to noise and measurement error, rather than the underlying relationship. Objectives To compare the 20-parameter main-effects model to simplified, nonlinear, multiplicative regression models in terms of how accurately they predict mean values of out-of-sample health states. Methods We used data from the Spanish, Singaporean, and Chinese EQ-5D-5L valuation studies. Four models were compared: an 8-parameter model with single parameter per dimension, multiplied by cross-dimensional parameters for levels 2, 3, and 4; 9- and 11-parameter extensions with handling of differences in the wording of level 5; and the "standard" additive 20-parameter model. Fixed- and random-intercept variants of all models were tested using two cross-validation methods: leave-one-out at the level of valued health states, and of health state blocks used in EQ-5D-5L valuation studies. Mean absolute error, Lin concordance correlation coefficient, and Pearson R between observed health state means and out-of-sample predictions were compared. Results Predictive accuracy was generally best using random intercepts. The 8-, 9-, and 11-parameter models outperformed the 20-parameter model in predicting out-of-sample health states. Conclusions Simplified nonlinear regression models look promising and should be investigated further using other EQ-5D-5L data sets. To reduce the risk of overfitting, cross-validation is recommended to inform model selection in future EQ-5D valuation studies.

Background The values of the five-level EuroQol five-dimensional questionnaire (EQ-5D-5L) are elicited using composite time trade-off and discrete choice experiments. Unfortunately, data quality issues and interviewer effects were observed in the first few EQ-5D-5L valuation studies. To prevent these issues from occurring in later studies, the EuroQol Group established a cyclic quality control (QC) process. Objectives To describe this QC process and show its impact on data quality. Methods A newly developed QC tool provided information about protocol compliance, interviewer effects, and mean values by health state severity. In a cyclic process, this information is initially used to evaluate whether new interviewers meet minimal quality requirements and later to provide feedback about how their performance may be improved. To investigate the impact of this cyclic process, we compared the quality of the data in Dutch and Spanish valuation studies that did not have this QC process with that in the follow-up studies in the same countries that used the QC process. Data quality was measured using protocol violations, variability between interviewers, the proportion of inconsistent responders, and clustering of composite time trade-off values. Results In Spain, protocol violations were reduced from 87% in the valuation study to 5% in the follow-up study and in the Netherlands from 20% to 8%. In both countries, interviewers performed more homogeneously in the follow-up studies. The number of inconsistent respondents was reduced by 23.2% in Spain and 23.6% in the Netherlands. Values were less clustered in the follow-up studies. Conclusions The implementation of a strict QC process in EQ-5D-5L valuation studies increases interviewer protocol compliance and promotes data quality.

Discrete choice experiments (DCEs) are a promising alternative to more resource-intensive preference elicitation methods such as time trade-off (TTO), as pairwise comparisons are more amenable to online completion, which can save time and money. However, modeling DCE data produces latent utilities which are on an unknown scale. Therefore, latent utilities need to be transformed to a full health-dead scale before they can be used in quality-adjusted life year calculations. We aimed to explore transformation functions from DCE-derived latent utilities to TTO-derived health utilities. We used EQ-5D-5L valuation data from eight different countries that collected both DCE and TTO data by using a standardized protocol. Results found less variation in the function that transformed latent utilities to health utilities in the western countries than in the eastern countries. While a global transformation function is not recommended, results suggest that regional transformation functions could potentially be used to derive health utilities from DCE data. Copyright © 2016 John Wiley & Sons, Ltd.

Background Time trade-off (TTO)-based valuation studies for the three-level version of the EuroQol five-dimensional questionnaire (EQ-5D) typically started off with a ranking task (ordering the health states by preference). This was not included in the protocol for the five-level EQ-5D (EQ-5D-5L) valuation study. Objectives To test whether reintroducing a ranking task before the composite TTO (C-TTO) could help to reduce inconsistencies in C-TTO responses and improve the data quality. Methods Respondents were randomly assigned to three study arms. The control arm was the present EQ-5D-5L study protocol, without ranking. The second arm (ranking without sorting) preceded the present protocol by asking respondents to rank the target health states using physical cards. The states were then valued in random order using C-TTO. In the third arm (ranking and sorting), the ranked states remained visible through the C-TTO tasks and the order of valuation was determined by the ranking. The study used only 10 EQ-5D-5L health states. We compared the C-TTO–based inconsistent pairs of health states and ties. Results The final sample size was 196 in the control arm, 205 in the ranking without sorting arm, and 199 in the ranking and sorting arm. The percentages of ties by respondents were 15.1%, 12.5%, and 12.6% for the control arm, the ranking without sorting arm, and the ranking and sorting arm, respectively. The extra cost for adding the ranking task was about 15%. Conclusions The benefit does not justify the effort involved in the ranking task. For this reason, the addition of the ranking task to the present EQ-5D-5L valuation protocol is not an attractive option.

Supplemental Digital Content is available in the text., Background: The EQ-5D instrument is the most widely used preference-based health-related quality of life questionnaire in cost-effectiveness analysis of health care technologies. Recently, a version called EQ-5D-5L with 5 levels on each dimension was developed. This manuscript explores the performance of a hybrid approach for the modeling of EQ-5D-5L valuation data. Methods: Two elicitation techniques, the composite time trade-off, and discrete choice experiments, were applied to a sample of the Spanish population (n=1000) using a computer-based questionnaire. The sampling process consisted of 2 stages: stratified sampling of geographic area, followed by systematic sampling in each area. A hybrid regression model combining composite time trade-off and discrete choice data was used to estimate the potential value sets using main effects as starting point. The comparison between the models was performed using the criteria of logical consistency, goodness of fit, and parsimony. Results: Twenty-seven participants from the 1000 were removed following the exclusion criteria. The best-fitted model included 2 significant interaction terms but resulted in marginal improvements in model fit compared to the main effects model. We therefore selected the model results with main effects as a potential value set for this methodological study, based on the parsimony criteria. The results showed that the main effects hybrid model was consistent, with a range of utility values between 1 and −0.224. Conclusion: This paper shows the feasibility of using a hybrid approach to estimate a value set for EQ-5D-5L valuation data.

This chapter presents the data sources and methods of the book. General population surveys are accumulated from 24 countries (Table 2.1). Descriptive statistics are used to provide EQ-5D population norms by age and gender categories for EQ VAS, EQ-5D index values, and for the five dimensions. Correlations between country-specific EQ-5D data (EQ VAS and 5 dimensions) and country-specific economic and health system macro indicators are calculated in the cross-country analysis. Odds ratios and the health concentration index methodology are used in the socio-demographic analysis of EQ-5D data.

OBJECTIVE: To evaluate two different methods to obtain a dead (0)--full health (1) scale for EQ-5D-5L valuation studies when using discrete choice (DC) modeling. METHOD: The study was carried out among 400 respondents from Barcelona who were representative of the Spanish population in terms of age, sex, and level of education. The DC design included 50 pairs of health states in five blocks. Participants were forced to choose between two EQ-5D-5L states (A and B). Two extra questions concerned whether A and B were considered worse than dead. Each participant performed ten choice exercises. In addition, values were collected using lead-time trade-off (lead-time TTO), for which 100 states in ten blocks were selected. Each participant performed five lead-time TTO exercises. These consisted of DC models offering the health state 'dead' as one of the choices--for which all participants' responses were used (DCdead)--and a model that included only the responses of participants who chose at least one state as worse than dead (WTD) (DCWTD). The study also estimated DC models rescaled with lead-time TTO data and a lead-time TTO linear model. RESULTS: The DC(dead) and DCWTD models produced relatively similar results, although the coefficients in the DCdead model were slightly lower. The DC model rescaled with lead-time TTO data produced higher utility decrements. Lead-time TTO produced the highest utility decrements. CONCLUSIONS: The incorporation of the state 'dead' in the DC models produces results in concordance with DC models that do not include 'dead'.

Background Back, neck and shoulder pain are the most common causes of occupational disability. They reduce health-related quality of life and have a significant economic impact. Many different forms of physical treatment are routinely used. The objective of this study was to estimate the cost of physical treatments which, despite the absence of evidence supporting their effectiveness, were used between 2004 and 2007 for chronic and non-specific neck pain (NP), back pain (BP) and shoulder pain (SP), within the Spanish National Health Service in the Canary Islands (SNHSCI). Methods Chronic patients referred from the SNHSCI to private physical therapy centres for NP, BP or SP, between 2004 and 2007, were identified. The cost of providing physical therapies to these patients was estimated. Systematic reviews (SRs) and clinical practice guidelines (CPGs) for NP, BP and SP available in the same period were searched for and rated according to the Oxman and AGREE criteria, respectively. Those rated positively for ≥70% of the criteria, were used to categorise physical therapies as Effective; Ineffective; Inconclusive; and Insufficiently Assessed. The main outcome was the cost of physical therapies included in each of these categories. Results 8,308 chronic cases of NP, 4,693 of BP and 5,035 of SP, were included in this study. Among prescribed treatments, 39.88% were considered Effective (physical exercise and manual therapy with mobilization); 23.06% Ineffective; 13.38% Inconclusive, and 23.66% Insufficiently Assessed. The total cost of treatments was € 5,107,720. Effective therapies accounted for € 2,069,932. Conclusions Sixty percent of the resources allocated by the SNHSCI to fund physical treatment for NP, BP and SP in private practices are spent on forms of treatment proven to be ineffective, or for which there is no evidence of effectiveness.