Quality of life and societal costs in hypertrophic cardiomyopathy:protocol of the AFFECT-HCM study

Background: Ever since the first description of hypertrophic cardiomyopathy (HCM), the most common genetic cardiac disease, tremendous progress has been made in the evaluation and management of HCM patients, but little attention has been focused on the impact of HCM on societal costs and quality of life (QoL). Aims: This paper describes the study protocol for the AFFECT-HCM study into burden of disease (BoD), which aims to estimate health-related QoL and societal costs in HCM patients and genotype-positive phenotype-negative (G+/P−) relatives during a one-year follow-up study, and relate this to the phenotypical HCM expression. Methods: A total of 400 Dutch HCM patients and 100 G+/P− subjects will be followed for one year in a prospective, multi-centre, prevalence-based BoD study. Societal costs will be measured via a bottom-up approach using the cost questionnaires iMCQ and iPCQ. For QoL, the generic EQ-5D-5L and disease-specific Kansas City Cardiomyopathy Questionnaire will be used. QoL and societal costs will be compared with phenotype-specific HCM characteristics and other determinants to identify factors that influence BoD. Accelerometry will test the correlation between BoD and physical activity. Conclusion: The AFFECT-HCM study will evaluate the BoD in HCM patients and G+/P− subjects to improve the understanding of the societal and economic impact of HCM.</p

A comparison of the responsiveness of EQ-5D-5L and the QOLIE-31P and mapping of QOLIE-31P to EQ-5D-5L in epilepsy

Objective: To investigate the responsiveness of and correlation between the EQ-5D-5L and the QOLIE-31P in patients with epilepsy, and develop a mapping function to predict EQ-5D-5L values based on the QOLIE-31P for use in economic evaluations. Methods: The dataset was derived from two clinical trials, the ZMILE study in the Netherlands and the SMILE study in the UK. In both studies, patients’ quality of life using the EQ-5D-5L and QOLIE-31P was measured at baseline and 12 months follow-up. Spearman’s correlations, effect sizes (EF) and standardized response means (SRM) were calculated for both the EQ-5D-5L and QOLIE-31P domains and sub scores. Mapping functions were derived using ordinary least square (OLS) and censored least absolute deviations models. Results: A total of 509 patients were included in this study. Low to moderately strong significant correlations were found between both instruments. The EQ-5D-5L showed high ceiling effects and small EFs and SRMs, whereas the QOLIE-31P did not show ceiling effects and also showed small to moderate EFs and SRMs. Results of the different mapping functions indicate that the highest adjusted R2 we were able to regress was 0.265 using an OLS model with squared terms, leading to a mean absolute error of 0.103. Conclusions: Results presented in this study emphasize the shortcomings of the EQ-5D-5L in epilepsy and the importance of the development of condition-specific preference-based instruments which can be used within the QALY framework. In addition, the usefulness of the constructed mapping function in economic evaluations is questionable

Exploring Outcomes to Consider in Economic Evaluations of Health Promotion Programs: What Broader Non-Health Outcomes Matter Most?

Background Attention is increasing on the consideration of broader non-health outcomes in economic evaluations. It is unknown which non-health outcomes are valued as most relevant in the context of health promotion. The present study fills this gap by investigating the relative importance of non-health outcomes in a health promotion context. Method We investigated the relative importance of ten non-health outcomes of health promotion programs not commonly captured in QALYs. Preferences were elicited from a sample of the Dutch general public (N = 549) by means of a ranking task. These preferences were analyzed using Borda scores and rank-ordered logit models. Results The relative order of preference (from most to least important) was: self-confidence, insights into own (un)healthy behavior, perceived life control, knowledge about a certain health problem, social support, relaxation, better educational achievements, increased labor participation and work productivity, social participation, and a reduction in criminal behavior. The weight given to a particular non-health outcome was affected by the demographic variables age, gender, income, and education. Furthermore, in an open question, respondents mentioned a number of other relevant non-health outcomes, which we classified into outcomes relevant for the individual, the direct social environment, and for society as a whole. Conclusion The study provides valuable insights in the non-health outcomes that are considered as most important by the Dutch general population. Ideally, researchers should include the most important non-health outcomes in economic evaluations of health promotio

Exploring the identification, validation, and categorization of costs and benefits of education in mental health: the PECUNIA project

BackgroundMental health problems can lead to costs and benefits in other sectors (e.g. in the education sector) in addition to the healthcare sector. These related costs and benefits are known as intersectoral costs and benefits (ICBs). Although some ICBs within the education sector have been identified previously, little is known about their extensiveness and transferability, which is crucial for their inclusion in health economics research.ObjectivesThe aim of this study was to identify ICBs in the education sector, to validate the list of ICBs in a broader European context, and to categorize the ICBs using mental health as a case study.MethodsPreviously identified ICBs in the education sector were used as a basis for this study. Additional ICBs were extracted from peer-reviewed literature in PubMed and grey literature from six European countries. A comprehensive list of unique items was developed based on the identified ICBs. The list was validated by surveying an international group of educational experts. The survey results were used to finalize the list, which was categorized according to the care atom.ResultsAdditional ICBs in the education sector were retrieved from ninety-six sources. Fourteen experts from six European countries assessed the list for completeness, clarity, and relevance. The final list contained twenty-four ICBs categorized into input, throughput, and output.ConclusionBy providing a comprehensive list of ICBs in the education sector, this study laid further foundations for the inclusion of important societal costs in health economics research in the broader European context

Comparison of statistical analysis methods for object case best–worst scaling

KLC received financial support for his research stay at the Department of Health Economics, Center for Public Health, Medical University of Vienna through the ‘Erasmus+ staff mobility grant’

The identification of economically relevant health and social care services for mental disorders in the PECUNIA project

Background: Health economic research is still facing significant problems regarding the standardization and international comparability of health care services. As a result, comparative effectiveness studies and cost-effectiveness analyses are often not comparable. This study is part of the PECUNIA project, which aimed to improve the comparability of economic evaluations by developing instruments for the internationally standardized measurement and valuation of health care services for mental disorders. The aim of this study was to identify internationally relevant services in the health and social care sectors relevant for health economic studies for mental disorders. Methods: A systematic literature review on cost-of-illness studies and economic evaluations was conducted to identify relevant services, complemented by an additional grey literature search and a search of resource use measurement (RUM) questionnaires. A preliminary long-list of identified services was explored and reduced to a short-list by multiple consolidation rounds within the international research team and an external international expert survey in six European countries. Results: After duplicate removal, the systematic search yielded 15,218 hits. From these 295 potential services could be identified. The grey literature search led to 368 and the RUM search to 36 additional potential services. The consolidation process resulted in a preliminary list of 186 health and social care services which underwent an external expert survey. A final consolidation step led to a basic list of 56 services grouped into residential care, daycare, outpatient care, information for care, accessibility to care, and self-help and voluntary care. Conclusions: The initial literature searches led to an extensive number of potential service items for health and social care. Many of these items turned out to be procedures, interventions or providing professionals rather than services and were removed from further analysis. The resulting list was used as a basis for typological coding, the development of RUM questionnaires and corresponding unit costs for international mental health economic studies in the PECUNIA project.</p