Quality of life and societal costs in patients with dilated cardiomyopathy.

AIMS: Dilated cardiomyopathy (DCM) is a major cause of heart failure impairing patient wellbeing and imposing a substantial economic burden on society, but respective data is missing. This study aims to measure the quality of life (QoL) and societal costs of DCM patients. METHODS AND RESULTS: A cross-sectional evaluation of QoL and societal costs of DCM patients was performed through the 5-level EuroQol (EQ-5D-5 L) and the Medical Consumption Questionnaire (iMCQ) and Productivity Cost Questionnaire (iPCQ), respectively. QoL was translated into numerical values (i.e. utilities). Costs were measured from a Dutch societal perspective. Final costs were extrapolated to one year, reported in 2022 Euros, and compared between DCM severity according to NYHA classes. A total of 550 DCM patients from the Maastricht cardiomyopathy registry (mCMP-registry) were included. Mean age was 61 years, and 34% were women. Overall utility was slightly lower for DCM patients than the population mean (0.840 vs. 0.869, p = 0.225). Among EQ-5D dimensions, DCM patients scored lowest in 'usual activities'. Total societal DCM costs were €14 843 per patient per year. Cost drivers were productivity losses (€7 037) and medical costs (€4 621). Patients with more symptomatic DCM (i.e. NYHA class III or IV) had significantly higher average DCM costs per year compared to less symptomatic DCM (€31,099 vs. €11 446, p < 0.001) and significantly lower utilities (0.631 vs. 0.883, p < 0.001). CONCLUSION: DCM is associated with high societal costs and reduced QoL, in particular with high DCM severity.

Cost-Effectiveness of Emerging Treatments for Atopic Dermatitis: A Systematic Review.

BACKGROUND: Numerous therapies have recently emerged for treatment of patients with atopic dermatitis (AD), a common skin disease, and understanding their cost-effectiveness is of high importance for policy makers. This systematic literature review (SLR) aimed to provide an overview of full economic evaluations that assessed cost-effectiveness of emerging AD treatments. METHODS: The SLR was conducted in Medline, Embase, UK National Health Service Economic Evaluation Database and EconLit. Reports published by the National Institute for Health and Care Excellence, the Institute for Clinical and Economic Review and the Canadian Agency for Drugs and Technologies in Health were manually searched. Economic evaluations published from 2017 to September 2022 that compared emerging AD treatments with any comparator were included. Quality assessment was conducted by using the Consensus on Health Economic Criteria list. RESULTS: A total of 1333 references were screened after removing duplicates. Among those references, 15 that conducted a total of 24 comparisons were included. Most studies were from the USA, UK or Canada. Seven different emerging treatments were compared, mostly with usual care. In 15 comparisons (63%), the emerging treatment was cost-effective, and 11 out of 14 dupilumab comparisons (79%) reported that dupilumab was cost-effective. Upadacitinib was the only emerging therapy that was never classified as cost-effective. On average, 13 out of 19 quality criteria (68%) per reference were rated as fulfilled while manuscripts and health technology reports received generally higher quality assessment scores than published abstracts. DISCUSSION: This study revealed some discrepancies in the cost-effectiveness of emerging therapies for AD. A variety of designs and guidelines made comparison difficult. Therefore, we recommend that future economic evaluations use more similar modelling approaches to improve comparability of results. OTHERS: The protocol was published in PROSPERO (ID: CRD42022343993).

Economic evaluations of interventions focusing on child abuse and neglect in high-income countries: a systematic review.

Introduction: Child abuse and neglect are together considered to be an important public health problem with a high individual and societal burden. Different interventions have been developed to prevent, diagnose, or treat maltreatment. While their effectiveness has been synthesized in prior reviews, the analysis of their cost-effectiveness is less common. The aim of this study is to synthesize and analyse economic evaluations of interventions focusing on child abuse and neglect in high-income countries. Methods: A systematic literature review was performed using MEDLINE, EMBASE, EconLit, PsycInfo and NHS EED. This study follows the PRISMA guidelines and double scoring was performed. The review includes trial- and model-based economic evaluations of preventive, diagnostic, and treatment related interventions in children up to 18 years or their caregivers. Risk of bias was assessed using the CHEC-extended checklist. The results are presented in a cost-effectiveness plane. Results: Of 5,865 search results, the full texts of 81 were analyzed, resulting in the inclusion of 11 economic evaluations. Eight of the included studies focus on prevention of child abuse and neglect, one study on diagnosis, and two on treatment. The heterogeneity between studies did not allow for the quantitative pooling of results. Most interventions were cost-effective, with the exception of one preventive and one diagnostic intervention. Conclusion: This study was subject to some limitations, as no gray literature was included, and the selection of studies may have been arbitrary due to varying terminologies and methodologies in the field. However, the quality of studies was high, and several interventions showed promising results. Systematic review registration: https://www.crd.york.ac.uk/prospero/display_record.php?ID=CRD42021248485, identifier: CRD42021248485.

An introduction to health technology assessment and health economic evaluation: an online self-learning course.

Cardiovascular diseases impose an enormous burden on patients and society. New health technologies promise to lower this burden; however, novel treatments often come at a high cost. In the Netherlands, health technology assessment (HTA) is increasingly being used to inform policy bodies about the optimal distribution of scarce healthcare resources and to guide decision-making about financing and reimbursement. In particular, economic evaluations, as one pillar of HTA, are frequently used to compare the costs and effects of different interventions. This paper aims to define HTA and its relevance to healthcare policy as well as providing a comprehensive overview of the methodology of economic evaluations targeting health professionals and researchers with limited prior knowledge of this subject. Accordingly, different types of economic evaluations are introduced, together with their respective costs and outcomes. Further, the results of economic evaluations are explained, along with techniques for performing them and methods for coping with uncertainty. In addition to this paper-based learning format, each chapter is complemented by a video lecture with further information and practical examples, helping to better understand and analyse health economic studies.

A systematic literature review of economic evaluations and cost-of-illness studies of inherited cardiomyopathies.

Hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM) are commonly inherited heart conditions associated with a high risk of heart failure and sudden cardiac death. To understand the economic and societal disease burden, this study systematically identified and reviewed cost-of-illness (COI) studies and economic evaluations (EEs) of various interventions for HCM and DCM. A literature search was performed in MEDLINE, EMBASE, NHS EED, EconLit and Web of Science to identify COI studies and EEs published between 1 January 2010 and 28 April 2021. The selection of studies and their critical appraisal were performed jointly by two independent researchers. For the quality assessment, the 'Consensus on Health Economic Criteria' list was used. Two COI studies and 11 EEs were eligible for inclusion. Cost-effectiveness varied among interventions and depended on the targeted patient population. Both COI studies identified only hospitalisation costs in HCM. The mean study quality was high in EEs but low in COI studies. Most studies excluded costs for patients, caregivers and productivity losses. Overall, knowledge of the societal and economic burden of inherited cardiomyopathies is limited. Future research needs to include quality-adjusted life years and a broader range of costs to provide an information base for optimising care for affected patients.

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.