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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 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 © 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Background: Health states were quantified based on discrete choice (DC) modeling and visual analogue scale (VAS) values using the five-level version of the EQ-5D (EQ-5D-5L). The aim of this study was to determine the extent of the relationship between DC derived values (indirect method) and VAS values (direct method)., Methods: Data were collected in Canada, the United Kingdom, the Netherlands, and the United States. Respondents were asked to perform paired comparisons between two EQ-5D-5L health states for DC. In total, 400 different EQ-5D-5L states were included. After each DC task, respondents were prompted to score the two states one after another on a VAS. Intraclass correlation coefficients were calculated between DC and VAS values and illuminating graphs were designed., Results: Approximately 400 respondents participated from each country. High similarity [individual intraclass correlation coefficients (ICC) >0.85] of DC and moderate correspondence of VAS values were observed for the four countries. Cross-country comparison of DC values shows a nonlinear relationship to the VAS values., Conclusion: EQ-5D-5L derived DC and VAS values show a close but nonlinear relationship. Given the obvious biases associated with the VAS, DC methods based on ordinal responses may be a better alternative.

Background: Resource allocation decision making in the Caribbean can be greatly enhanced by the introduction of cost per quality-adjusted life-year (QALY) analysis on the basis of local preferences. In the valuation literature there have been recommendations for the elicitation methods of the EuroQol five-dimensional questionnaire (EQ-5D) that combine discrete-choice experiment (DCE) for bulk valuation with a time trade-off component for rescaling., Objectives: To create a three-level EQ-5D value set for Trinidad and Tobago using an elicitation method that takes into account the local constraints, and that can be easily deployed in other Caribbean islands., Methods: A D-efficient DCE was completed by a representative sample of 307 adults. A time trade-off procedure was used to obtain values for rescaling the DCE model on a scale anchored at 0 (dead) and 1 (full health)., Results: A mixed logit analysis of the DCE data produced an internally valid model that is similar to the results obtained in earlier pilot studies., Conclusions: This EQ-5D value set allows cost per QALY analyses to be carried out on the basis of preferences from Trinidad and Tobago, and the approach to the DCE design can be taken for similar value sets to be created in the small, resource-constrained health systems of the Caribbean. Some guidelines for the initial application and introduction of cost per QALY analysis into the Trinidad and Tobago health system are also presented., (Copyright © 2016 International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc. All rights reserved.)

Objective: Cost per quality-adjusted life year data are required for reimbursement decisions in many Central and Eastern European (CEE) countries. EQ-5D is by far the most commonly used instrument to generate utility values in CEE. This study aims to systematically review the literature on EQ-5D from eight CEE countries., Methods: An electronic database search was performed up to 1 July 2015 to identify original EQ-5D studies from the countries of interest. We analysed the use of EQ-5D with respect to clinical areas, methodological rigor, population norms and value sets., Results: We identified 143 studies providing 152 country-specific results with a total sample size of 81,619: Austria (n = 11), Bulgaria (n = 6), Czech Republic (n = 18), Hungary (n = 47), Poland (n = 51), Romania (n = 2), Slovakia (n = 3) and Slovenia (n = 14). Cardiovascular (21 %), neurologic (17 %), musculoskeletal (15 %) and endocrine, nutritional and metabolic diseases (13 %) were the most frequently studied clinical areas. Overall, 112 (78 %) of the studies reported EQ VAS results and 86 (60 %) EQ-5D index scores, of which 27 (31 %) did not specify the applied tariff. Hungary, Poland and Slovenia have population norms. Poland and Slovenia also have a national value set., Conclusions: Increasing use of EQ-5D is observed throughout CEE. The spread of health technology assessment activities in countries seems to be reflected in the number of EQ-5D studies. However, improvement in informed use and methodological quality of reporting is needed. In jurisdictions where no national value set is available, in order to ensure comparability we recommend to apply the most frequently used UK tariff. Regional collaboration between CEE countries should be strengthened.

Discrete-choice experiments (DCEs) have become a commonly used instrument in health economics and patient-preference analysis, addressing a wide range of policy questions. An important question when setting up a DCE is the size of the sample needed to answer the research question of interest. Although theory exists as to the calculation of sample size requirements for stated choice data, it does not address the issue of minimum sample size requirements in terms of the statistical power of hypothesis tests on the estimated coefficients. The purpose of this paper is threefold: (1) to provide insight into whether and how researchers have dealt with sample size calculations for healthcare-related DCE studies; (2) to introduce and explain the required sample size for parameter estimates in DCEs; and (3) to provide a step-by-step guide for the calculation of the minimum sample size requirements for DCEs in health care.

Economic evaluations likely undervalue the benefits of interventions in populations receiving both health and social services, such as frail elderly, by measuring only health-related quality of life. For this reason, alternative preference-based instruments have been developed for economic evaluations in the elderly, such as the ICECAP-O. The aim of this paper is twofold: (1) to evaluate the cost-effectiveness using a short run time frame for an integrated care model for frail elderly, and (2) to investigate whether using a broader measure of (capability) wellbeing in an economic evaluation leads to a different outcome in terms of cost-effectiveness. We performed univariate and multivariate analyses on costs and outcomes separately. We also performed incremental net monetary benefit regressions using quality adjusted life years (QALYs) based on the ICECAP-O and EQ-5D. In terms of QALYs as measured with the EQ-5D and the ICECAP-O, there were small and insignificant differences between the instruments, due to negligible effect size. Therefore, widespread implementation of the Walcheren integrated care model would be premature based on these results. All results suggest that, using the ICECAP-O, the intervention has a higher probability of cost-effectiveness than with the EQ-5D at the same level of WTP. In case an intervention's health and wellbeing effects are not significant, as in this study, using the ICECAP-O will not lead to a false claim of cost-effectiveness of the intervention. On the other hand, if differences in capability QALYs are meaningful and significant, the ICECAP-O may have the potential to measure broader outcomes and be more sensitive to differences between intervention and comparators.

Considerations beyond cost-effectiveness are important in reimbursement decision making. We assessed the importance of disease severity in drug reimbursement decision making in Belgium, France, The Netherlands and Sweden. We investigated scientific literature and policy documents and conducted three interviews in each country (four in The Netherlands) with persons involved in drug reimbursement. Disease severity is an important consideration, especially where the level is high. The Netherlands operationalizes disease severity using the proportional shortfall approach. Sweden uses categories to give an indication of the level of severity. In The Netherlands and Sweden, severity only implicitly plays a role in the decision whether to reimburse a drug, whereas in Belgium and France it also explicitly plays a role in determining the willingness to use public resources. Interviewees acknowledged that as well as a qualitative description of the disease, quantitative information may also be useful as input for decision making. None of them, however, considered this to be of decisive importance. Although disease severity is important in drug reimbursement decision making in all four countries, all seem to struggle in explicitly specifying its actual role. Belgium and France are the most explicit by using levels of severity in setting reimbursement levels; all four countries could, however, improve the transparency of its actual importance relative to the other criteria in the decision-making process., (Copyright © 2014 Elsevier Ireland Ltd. All rights reserved.)

Background: The aim of our study is to compare the validity of a generic preference-based Quality of Life (QoL) instrument for adults to that of a generic child-specific QoL instrument in children and adolescents with attention deficit hyperactivity disorder (ADHD)., Methods: EQ-5D and KIDSCREEN-10 data were collected using a questionnaire survey performed among parents with a child or adolescent diagnosed with ADHD. The measurements were compared to assess (dis)similarities of the instruments' constructs and responsiveness to different health states. Principal component analysis (PCA) with varimax rotation was used to identify factors underlying the constructs of both instruments. Instruments' index scores of respondents with different treatment and comorbidity profiles were compared using Student's t tests. Cohen's effect sizes were calculated for an indirect comparison of the instruments' responsiveness and discriminating ability. Separate analyses were performed in children aged 8-12 and 13-18 years., Results: A strong relation was found between the EQ-5D and KIDSCREEN-10 index scores. However correlations between EQ-5D and KIDSCREEN-10 items were moderate or low. The PCA identified five separate factors of quality of life. A physical and a mental factor included a combination of three EQ-5D dimensions and six KIDSCREEN-10 items; the remaining EQ-5D and KIDSCREEN-10 items constituted complementary factors without any overlap between the separate instruments. Scores of both instruments differed significantly according to respondents' response to treatment and comorbidity profile. Cohen's effect sizes indicated comparable results of the instruments' responsiveness and discriminative ability., Conclusions: The results highlight that the instruments measure different constructs of QoL in children with ADHD. Despite this, the analyses showed comparable responsiveness and discriminative ability of the instruments. These results suggest that for economic evaluations, the EQ-5D is an appropriate and valid instrument for measuring QoL in children.

Purpose: The purpose of this study is to compare the health-related quality of life (HrQoL) of preschool children who stutter (CWS) and a reference population of children who do not stutter, and to evaluate the association between stuttering severity and HrQoL., Methods: Baseline data were used from 197 children participating in a multicenter Randomized Clinical Trial in the Netherlands. Information on stuttering severity and time since onset (TSO) of stuttering was obtained from the baseline evaluation by speech- and language therapists. Stuttering severity was measured using the SSI-3. HrQoL was assessed using proxy versions of two Child Health Questionnaires (ITQOL-97 and CHQ-PF28), the Health Utility Index 3 (HUI3) and the EuroQoL EQ-VAS (EQ-VAS)., Results: While the outcomes on the EQ-VAS and the HUI3 showed that the HrQoL of CWS is slightly poorer than that of the Dutch reference population, results on the different dimensions of the CHQ-instruments did not reveal any difference in scores between stuttering children and reference groups. Within the group of CWS, two ITQOL-97 and four CHQ-PF28 scales showed statistically different scores for children in different SSI stuttering severity or TSO categories. However, the effect sizes showed that these differences were so small that they could be considered negligible., Conclusion: The results of this study do not reveal a diminished HrQoL for preschool CWS. Future research should include a larger cohort of children with severe stuttering, study the longitudinal course of HrQoL and incorporate additional parameters such as the characteristics of the child and his environment., Educational Objectives: The reader will be able to: (a) summarize the current evidence base on HrQoL in people who stutter; (b) describe the HrQoL of preschool CWS on different HrQoL measures; (c) describe the relationship between stuttering severity and HrQoL in preschool CWS., (Copyright © 2014 Elsevier Inc. All rights reserved.)

Aims: To investigate the feasibility of discrete choice experiments for valuing EQ-5D-5L states using computer-based data collection, the consistency of the estimated regression coefficients produced after modeling the preference data, and to examine the similarity of the values derived across countries., Methods: Data were collected in Canada, England, The Netherlands, and the United States (US). Interactive software was developed to standardize the format of the choice tasks across countries, except for face-to-face interviewing in England. The choice task required respondents to choose between 2 suboptimal health states. A Bayesian design was used to generate 200 pairs of states that were randomly grouped into 20 blocks. Each respondent completed 1 block of 10 pairs. A main-effects probit model was used to estimate regression coefficients and to derive values., Results: Approximately 400 respondents participated from each country. The mean time to perform 1 choice task was between 29.2 (US) and 45.2 (England) seconds. All regression coefficients were statistically significant, except level 2 for Usual Activities in The Netherlands (P=0.51). Predictions for the complete set of 3125 EQ-5D-5L health states were similar for the 4 countries. Intraclass correlation coefficients between the countries were high: from 0.80 (England vs. US) through 0.98 (Canada vs., Us) Conclusions: Derivation of value sets from the general population using computer-based choice tasks for the EQ-5D-5L is feasible. Parameter estimates were generally consistent and logical, and health-state values were similar across the 4 countries.

Background: Productivity costs are often ignored in economic evaluations. In order to facilitate productivity cost inclusion, it has been suggested to estimate productivity costs indirectly using quality of life data., Objective: This study aimed to derive and validate an algorithm for predicting productivity losses on the basis of quality-of-life data using the EQ-5D-3L., Methods: A large representative sample of the Dutch general public (n = 1,100) was asked in a web-based questionnaire to state their expected level of productivity in terms of absenteeism and presenteeism for multiple EQ-5D health states. Based on these data, two generalized estimating equations (GEE) models were constructed: (1) a model predicting levels of absenteeism and (2) a model predicting presenteeism. The models were validated by comparing model predictions with conventionally measured productivity within a group of low back pain patients., Results: Predicted absenteeism levels based on EQ-5D health state closely resembled conventionally measured absenteeism levels. Productivity losses related to presenteeism seemed somewhat overestimated by our prediction model. Measured and predicted productivity were moderately but highly significantly correlated., Conclusions: Overall, it appears possible to make reasonable productivity predictions based on EQ-5D data. Further exploration and validation of prediction algorithms remains necessary, however, especially for presenteeism.

Background: The effect of lead time in time trade-off (TTO) valuation is not well understood. The purpose of this study was to investigate the effects on health-state valuation of the length of lead time and the way the lead-time TTO task is displayed visually., Methods: Using two general population samples, we compared three lead-time TTO variants: 10 years of lead time in full health preceding 5 years of unhealthy time (standard); 5 years of lead time preceding 5 years of unhealthy time (experimental); and 10 years of lead time and 5 years of unhealthy time, presented with a visual aid to highlight the point where the lead time ends (experimental). Participants were randomized to receive one of the lead-time variants, as administered by a computer software program., Results: Health-state values generated by TTO valuation tasks using a longer lead time were slightly lower than those generated by tasks using a shorter lead time. When lead time and unhealthy time were presented with visual aids highlighting the difference between the lead time and unhealthy time, respondents spent more time considering health states with a value close to 0., Conclusions: Different lead-time time trade-off variants should be carefully studied in order to achieve the best measurement of health-state values using this new method.

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'.

This article examines the effect that different specifications of the time trade-off (TTO) valuation task may have on values for EQ-5D-5L health states. The new variants of the TTO, namely lead-time TTO and lag-time TTO, along with the classic approach to TTO were compared using two durations for the health states (15 and 20 years). The study tested whether these methods yield comparable health-state values. TTO tasks were administered online. It was found that lag-time TTO produced lower values than lead-time TTO and that the difference was larger in the longer time frame. Classic TTO values most resembled those of the lag-time TTO in a 20-year time frame in terms of mean absolute difference. The relative importance of different domains of health was systematically affected by the duration of the health state. In the tasks with a 10-year health-state duration, anxiety/depression had the largest negative impact on health-state values; in the tasks with a 5-year duration, the pain/discomfort domain had the largest negative impact.

There is no scientific consensus on the optimal specification of the time trade-off (TTO) task. As a consequence, studies using TTO to value health states may share the core element of trading length of life for quality of life, but can differ considerably on many other elements. While this pluriformity in specifications advances the understanding of TTO from a methodological point of view, it also results in incomparable health state values. Health state values are applied in health technology assessments, and in that context comparability of information is desired. In this article, we discuss several alternative specifications of TTO presented in the literature. The defining elements of these specifications are identified as being either methodological, procedural or analytical in nature. Where possible, it is indicated how these elements affect health state values (i.e., upward or downward). Finally, a checklist for TTO studies is presented, which incorporates a list of choices to be made by researchers who wish to perform a TTO task. Such a checklist enables other researchers to align methodologies in order to enhance the comparability of health state values.

Introduction: This study was designed to test the feasibility and face validity of the composite time trade-off (composite TTO), a new approach to TTO allowing for a more consistent elicitation of negative health state values., Methods: The new instrument combines a conventional TTO to elicit values for states regarded better than dead and a lead-time TTO for states worse than dead., Results: A total of 121 participants completed the composite TTO for ten EQ-5D-5L health states. Mean values ranged from -0.104 for health state 53555 to 0.946 for 21111. The instructions were clear to 98 % of the respondents, and 95 % found the task easy to understand, indicating feasibility. Further, the average number of steps taken in the iteration procedure to achieve the point of indifference in the TTO and the average duration of each task were indicative of a deliberate cognitive process., Conclusion: Face validity was confirmed by the high mean values for the mild health states (>0.90) and low mean values for the severe states (<0.42). In conclusion, this study demonstrates the feasibility and face validity of the composite TTO in a face-to-face standardized computer-assisted interview setting.

Drug treatment and reimbursement is an area of ever growing complexity in health priority setting. This paper assesses the National Registry of Growth Hormone Treatment (LRG) responsible for making prioritisation decisions in the Dutch drug reimbursement system in the treatment of growth hormone, using the framework for fairness. We used qualitative research consisting of semi-structured interviews and focus group sessions combined with quantitative methods to audit the decisions of the forum. The rationing decisions of the forum demonstrate accountability for reasonableness by the conditions for transparency, relevance, and appeal. Most rationales for the decisions are public and transparent. The patients and paediatricians see decisions made by the LRG as clinical and therefore relevant decisions. They also refer to extensive appeal procedures. The case also raises important issues regarding the legitimacy of expert-based priority setting as the cyclic nature of guideline development conflicts with the need for maintaining strict rationing criteria. In 13% of the patients, the sick funds did cover treatment as the forum advised them to do, but according to guideline criteria it may be unlikely that these patients have growth hormone deficiency. According to the LRG, however, only 2% of the decisions are inconsistent with the guidelines, as some criteria on what to do in case of more uncertainty, shifted. For the forum, it seems rather unthinkable to go against the professional norms, in spite of formal national regulations. For the Health Care Insurance Board (CVZ), it was not considered possible to go against national regulations, especially as professional norms have shifted without informing policy makers and patient representatives.

The value of QALY gains for different patients may be recalculated using equity weights, but it is unclear which interpretation of equity should be used: severity of illness, fair innings or proportional shortfall. We set up an experiment to analyze which of these equity concepts best reflects people's distributional preferences. Sixty respondents assigned a priority rank to the treatment of 10 conditions using the paired comparison technique. We described these real-life conditions by their actual QALY profiles, i.e. in terms of age, disease free period, duration of disease, quality of life, and life years lost. Next we determined the priority rank order of the 10 conditions by the three equity concepts, using the weights that each equity concept attributes to the different units of the QALY profile describing the 10 conditions. To explore the social interpretation of equity, we compared the observed and theoretical rank orderings using Spearman correlations. All correlations were significant at a 0.05 level. Fair innings best predicted the observed rank order of the 10 conditions (r=0.95). Weaker correlations were found for proportional shortfall (r=0.82) and severity of illness (r=-0.65). This result calls attention to health policy, because actual health care decisions often reflect concerns of severity of illness. This raises the question if health care decision makers evaluate the claims of different patients for health care by appropriate criteria.