Dutch Nationwide Implementation Program for familial hypercholesterolemia case finding in children: a cost-effectiveness and return on investment analysis

Abstract

Abstract Background The Netherlands is one of the few countries in the world that actively screen for familial hypercholesterolemia (FH). Objectives We investigated the return on investment (ROI) and cost-effectiveness of the Dutch nationwide implementation program for FH case-finding and preventive treatment in children compared to later detection and treatment from both a societal and healthcare perspective in the Netherlands. Methods Cascade case finding and early preventive treatment were modelled to simulate the progression of disease and costs of ten-year-olds suspected of having heterozygous FH (HeFH) over a lifetime. The model consisted of three health states: alive without coronary heart disease (CHD), alive with CHD, and dead. The decision was to compare the nationwide implementation program for FH cascade case-finding in children and immediate treatment with statins compared to “usual care” later detection and treatment. The prevalence of HeFH in this target population was 51.2% as per the Dutch Health Program for FH case-finding, and the sensitivity and specificity of testing was 100%. Contemporary Mendelian Randomisation Analysis data was used to quantify the risk of a first coronary heart disease event as a function of age and total lifetime exposure to low-density-lipoprotein cholesterol. Costs and outcome were sourced from the Dutch data and other published sources. ROI for the Dutch nationwide implementation program for FH case-finding and cost-effectiveness analyses using incremental cost-effectiveness ratios (ICERs) (cost per quality-adjusted life years (QALYs) gained), compared with current usual care were the main outcomes of interest. All future benefits and costs were discounted annually by 1.5% and 4% respectively. Results Over the lifetime horizon, the ROI for the Dutch nationwide implementation program for FH case-finding was €16.52 (i.e. every euro spent on the program resulted in a return of €16.52). From the societal perspective, the Dutch nationwide implementation program for FH case-finding and early treatment with statins was the dominant strategy (health improvement and cost saving) compared to usual care. From a healthcare perspective, the Dutch nationwide implementation program for FH case-finding and early treatment with statins would gain 2.64 QALYs per person, at an additional cost of €7,178 (both discounted). These equated to an ICER of €2,668 per QALY gained. Conclusions From the societal perspective, the the Dutch nationwide implementation program for FH case-finding represented excellent value for investment for Netherlands, being not only health saving but also cost saving form a societal perspective and cost effective from a healthcare perspective. Funding Acknowledgement Type of funding sources: None.