Think of the Children: A Discussion of the Rationale for and Implications of the Perspective Used for EQ-5D-Y Health State Valuation

_Objectives:_ The recently published EQ-5D-Y valuation protocol prescribes the general public values EQ-5D-Y health states for a 10-year-old child. This child perspective differ

EQ-5D-Y-5L: developing a revised EQ-5D-Y with increased response categories.

Kreimeier S, Astrom M, Burstrom K, et al. EQ-5D-Y-5L: developing a revised EQ-5D-Y with increased response categories. Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation. 2019;28(7):1951-1961.PURPOSE: EQ-5D-Y is a generic measure of health status for children and adolescents aged 8-15 years. Originally, it has three levels of severity in each dimension (3L). This study aimed to develop a descriptive system of EQ-5D-Y with an increased number of severity levels and to test comprehensibility and feasibility. METHODS: The study was conducted in Germany, Spain, Sweden and the UK. In Phase 1, a review of existing instruments and focus group interviews were carried out to create a pool of possible labels for a modified severity classification. Participants aged 8-15 rated the severity of the identified labels in individual sorting and response scaling interviews. In Phase 2, preliminary 4L and 5L versions were constructed for further testing in cognitive interviews with healthy participants aged 8-15 years and children receiving treatment for a health condition. RESULTS: In Phase 1, a total of 233 labels was generated, ranging from 37 (UK) to 79 labels (Germany). Out of these, 7 to 16 possible labels for each dimension in the different languages were rated in 255 sorting and response scaling interviews. Labels covered an appropriate range of severity on the health continuum in all countries. In Phase 2, the 5L version was generally preferred (by 68-88% of the participants per country) over the 4L version. CONCLUSIONS: This multinational study has provided a version of the EQ-5D-Y with 5 severity levels in each dimension. This extended version (EQ-5D-Y-5L) requires testing its psychometric properties and its performance compared to that of the original EQ-5D-Y-3L

An exploration of methods for obtaining 0 = dead anchors for latent scale EQ-5D-Y values

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

An integrated molecular risk score early in life for subsequent childhood asthma risk.

BACKGROUND Numerous children present with early wheeze symptoms, yet solely a subgroup develops childhood asthma. Early identification of children at risk is key for clinical monitoring, timely patient-tailored treatment, and preventing chronic, severe sequelae. For early prediction of childhood asthma, we aimed to define an integrated risk score combining established risk factors with genome-wide molecular markers at birth, complemented by subsequent clinical symptoms/diagnoses (wheezing, atopic dermatitis, food allergy). METHODS Three longitudinal birth cohorts (PAULINA/PAULCHEN, n = 190 + 93 = 283, PASTURE, n = 1133) were used to predict childhood asthma (age 5-11) including epidemiological characteristics and molecular markers: genotype, DNA methylation and mRNA expression (RNASeq/NanoString). Apparent (ap) and optimism-corrected (oc) performance (AUC/R2) was assessed leveraging evidence from independent studies (Naïve-Bayes approach) combined with high-dimensional logistic regression models (LASSO). RESULTS Asthma prediction with epidemiological characteristics at birth (maternal asthma, sex, farm environment) yielded an ocAUC = 0.65. Inclusion of molecular markers as predictors resulted in an improvement in apparent prediction performance, however, for optimism-corrected performance only a moderate increase was observed (upto ocAUC = 0.68). The greatest discriminate power was reached by adding the first symptoms/diagnosis (up to ocAUC = 0.76; increase of 0.08, p = .002). Longitudinal analysis of selected mRNA expression in PASTURE (cord blood, 1, 4.5, 6 years) showed that expression at age six had the strongest association with asthma and correlation of genes getting larger over time (r = .59, p < .001, 4.5-6 years). CONCLUSION Applying epidemiological predictors alone showed moderate predictive abilities. Molecular markers from birth modestly improved prediction. Allergic symptoms/diagnoses enhanced the power of prediction, which is important for clinical practice and for the design of future studies with molecular markers

The EQ Health and Wellbeing: Overview of the Development of a Measure of Health and Wellbeing and Key Results

Objectives: Existing measures for estimating quality-adjusted life-years are mostly limited to health-related quality of life. This article presents an overview of the development the EQ Health and Wellbeing (EQ-HWB), which is a measure that encompasses health and wellbeing. Methods: Stages: (1) Establishing domains through reviews of the qualitative literature informed by a conceptual framework. (2) Generation and selection of items to cover the domains. (3) Face validation of these items through qualitative interviews with 168 patients, social care users, general population, and carers across 6 countries (Argentina, Australia, China, Germany, United Kingdom, United States). (4) Extensive psychometric testing of candidate items (using classical, factor analysis, and item response theory methods) on > 4000 respondents in the 6 countries. Stakeholders were consulted throughout. Results: A total of 32 subdomains grouped into 7 high-level domains were identified from the qualitative literature and 97 items generated to cover them. Face validation eliminated 36 items, modified 14, and added 3. Psychometric testing of 64 items found little difference in missing data or problems with response distribution, the conceptual model was confirmed except in China, and most items performed well in the item response theory in all countries. Evidence was presented to stakeholders in 2 rounds of consultation to inform the final selection of items for the EQ-HWB (25-item) and the short version of EQ-HWB (9-items). Conclusions: EQ-HWB measures have been developed internationally for evaluating interventions in health, public health, and social care including the impact on patients, social care users, and carers

Proceedings of Patient Reported Outcome Measure’s (PROMs) Conference Oxford 2017: Advances in Patient Reported Outcomes Research

A33-Effects of Out-of-Pocket (OOP) Payments and Financial Distress on Quality of Life (QoL) of People with Parkinson’s (PwP) and their Carer

EuReCa ONE—27 Nations, ONE Europe, ONE Registry A prospective one month analysis of out-of-hospital cardiac arrest outcomes in 27 countries in Europe

AbstractIntroductionThe aim of the EuReCa ONE study was to determine the incidence, process, and outcome for out of hospital cardiac arrest (OHCA) throughout Europe.MethodsThis was an international, prospective, multi-centre one-month study. Patients who suffered an OHCA during October 2014 who were attended and/or treated by an Emergency Medical Service (EMS) were eligible for inclusion in the study. Data were extracted from national, regional or local registries.ResultsData on 10,682 confirmed OHCAs from 248 regions in 27 countries, covering an estimated population of 174 million. In 7146 (66%) cases, CPR was started by a bystander or by the EMS. The incidence of CPR attempts ranged from 19.0 to 104.0 per 100,000 population per year. 1735 had ROSC on arrival at hospital (25.2%), Overall, 662/6414 (10.3%) in all cases with CPR attempted survived for at least 30 days or to hospital discharge.ConclusionThe results of EuReCa ONE highlight that OHCA is still a major public health problem accounting for a substantial number of deaths in Europe.EuReCa ONE very clearly demonstrates marked differences in the processes for data collection and reported outcomes following OHCA all over Europe. Using these data and analyses, different countries, regions, systems, and concepts can benchmark themselves and may learn from each other to further improve survival following one of our major health care events

Konzeptionelle und methodische Weiterentwicklung der Lebensqualitätsmessung bei Kindern und Jugendlichen am Beispiel des EQ-5D-Y

Kreimeier S. Konzeptionelle und methodische Weiterentwicklung der Lebensqualitätsmessung bei Kindern und Jugendlichen am Beispiel des EQ-5D-Y. Bielefeld: Universität Bielefeld; 2020

Development of the German version of EQ-5D-Y-5L to measure health-related quality of life in children and adolescents: Identification of response labels and pilot testing

Kreimeier S, Greiner W. Entwicklung der deutschen Version des EQ-5D-Y-5L zur Messung der gesundheitsbezogenen Lebensqualität bei Kindern und Jugendlichen: Identifikation von Antwortformulierungen und Pilotierung. Zeitschrift fur Evidenz, Fortbildung und Qualitat im Gesundheitswesen. 2019;144-145:24-34.OBJECTIVE: EQ-5D-Y-3L is a generic, youth-specific instrument to measure health-related quality of life (HRQoL). It contains five dimensions with three severity levels each. The aim of this study was to identify response levels to extend the German EQ-5D-Y version to four or five severity levels (4L or 5L) and to conduct a pilot test of both versions.; METHODS: In phase 1, we reviewed existing youth-specific HRQoL instruments and conducted focus group interviews with healthy children and adolescents to identify potential new labels. In individual sorting and response scaling interviews, participants aged 8 to 15 years rated the severity of the identified labels. Based on the results, a 4L and a 5L version were developed. In phase 2, the feasibility of those versions was tested in cognitive interviews with healthy children and adolescents as well as those with a health condition.; RESULTS: In phase 1, 12 to 16 labels were identified for each dimension. These were rated by 64 children and adolescents in the sorting and response scaling interviews. The included labels covered different severity levels of health impairments. In phase 2, 88% of the 33 children and adolescents preferred the 5L version as it was easier for them to report on their own health in more detail.; CONCLUSION: Involving the target group of children and adolescents, a German EQ-5D-Y version with five severity levels was developed (EQ-5D-Y-5L) that can be used in children and adolescents aged 8 to 15 years. However, the psychometric properties of the instrument need further investigation. In addition, value sets need to be to developed before the questionnaire is suitable for all fields of application. Copyright © 2019. Published by Elsevier GmbH

EQ-5D-Y as a Health-Related Quality of Life Instrument for Children and Adolescents: The Instrument's Characteristics, Development, Current Use, and Challenges of Developing Its Value Set

Kreimeier S, Greiner W. EQ-5D-Y as a Health-Related Quality of Life Instrument for Children and Adolescents: The Instrument's Characteristics, Development, Current Use, and Challenges of Developing Its Value Set. VALUE IN HEALTH. 2019;22(1):31-37.Objectives: Interest in the measurement of health-related quality of life (HRQoL) in children and adolescents has been increasing, and appropriate instruments are required for this target group. This article focuses on the EQ-5D-Y instrument, presenting an overview of its characteristics, development, and current use, and includes a discussion of methodological and conceptual issues related to the valuation of child health and the development of an EQ-5D-Y value set. Methods: This article brings together the experiences of the research team that developed and validated the EQ-5D-Y, supplemented by information derived from EQ-5D-Y study registrations on the EuroQol Group's website. Results: EQ-5D-Y is a child-specific and age-appropriate measure of HRQoL. Study registration data show that the instrument's use has steadily increased since its first publication. It has been used in various types of studies and in different disease areas. Currently there is no value set for EQ-5D-Y, and so its use in cost-utility analysis (CUA) is limited. There are methodological and conceptual issues that affect the design of valuation studies for child health. Issues that are discussed include the need for separate value sets for children and adolescents, the choice of appropriate reference samples and valuation techniques, and the framing of the tasks. Conclusions: Research on EQ-5D-Y and its use has increased in the last years. Further research is required to clarify methodological issues regarding health state valuation in children and adolescents. This will support the development of a value set for EQ-5D-Y and the use of EQ-5D-Y in CUA. Copyright (c) 2019, ISPOR-The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. This is anopenaccess article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)