{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,8]],"date-time":"2026-10-08T19:06:21Z","timestamp":1791486381420,"version":"4.3.4"},"reference-count":39,"publisher":"American Medical Association (AMA)","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Pediatr"],"published-print":{"date-parts":[[2023,6,1]]},"abstract":"Importance<\/jats:title>The Netherlands is one of the few countries that has a long-term history of active screening for familial hypercholesterolemia (FH), enabling health-economic analyses.<\/jats:p><\/jats:sec>Objective<\/jats:title>To investigate cost-effectiveness and the return on investment (ROI) of a nationwide cascade case-finding and preventive treatment program starting with identification of FH in children and treatment, from both a societal and health care perspective.<\/jats:p><\/jats:sec>Design, Setting, and Participants<\/jats:title>Cascade case-finding and early preventive treatment were modeled to simulate the progression of disease and costs of 10-year-olds suspected of having heterozygous FH over a lifetime. The model consisted of 3 health states: alive without coronary heart disease (CHD), alive with CHD, and deceased. Mendelian randomization analysis was used to quantify the risk of a first CHD event as a function of age and total lifetime exposure to low-density lipoprotein cholesterol. Cost-effectiveness was defined as \u20ac20\u202f000 ($21\u202f800) per QALYs (quality-adjusted life-years) gained, using incremental cost-effectiveness ratios (ICERs). All future benefits and costs were discounted annually by 1.5% and 4%, respectively.<\/jats:p><\/jats:sec>Interventions<\/jats:title>The study compared 2 strategies: (1) cascade screening and initiation of treatment with statins in children (mean age, 10 years) and (2) no screening, later detection, and treatment.<\/jats:p><\/jats:sec>Main Outcomes and Measures<\/jats:title>Outcome of interest included cost, detection, and successful treatment of FH in terms of life-years gained and QALYs. The clinical and cost outputs for each model in the 2 scenarios (early detection and treatment and later detection and treatment) were totaled to determine the overall cost-effectiveness and ROI attributed to implementation of the Dutch FH program.<\/jats:p><\/jats:sec>Results<\/jats:title>In this model constructed to simulate the progression of FH in 1000 hypothetical 10-year-olds, from a health care perspective, the program would gain 2.53 QALYs per person, at an additional cost of \u20ac23\u202f365 ($25\u202f468) (both discounted). These equated to an ICER of \u20ac9220 ($10\u202f050) per QALY gained. From the societal perspective, the detection and treatment program were cost saving over a lifetime compared with no cascade screening for FH. The ROI for the detection and treatment program for FH in children was \u20ac8.37 ($9.12).<\/jats:p><\/jats:sec>Conclusions and Relevance<\/jats:title>The findings of this study suggest that the early detection and treatment program for FH in children may offer a good value for investment, being both health and cost saving. The findings and interpretations are conditional on assumptions inherent in the health economic model.<\/jats:p><\/jats:sec>","DOI":"10.1001\/jamapediatrics.2023.0763","type":"journal-article","created":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T15:01:15Z","timestamp":1682953275000},"page":"625","source":"Crossref","is-referenced-by-count":45,"title":["Cost-effectiveness and Return on Investment of a Nationwide Case-Finding Program for Familial Hypercholesterolemia in Children in the Netherlands"],"prefix":"10.1001","volume":"177","author":[{"given":"Zanfina","family":"Ademi","sequence":"first","affiliation":[{"name":"Centre for Medicine Use and Safety, Faculty of Pharmacy and Pharmaceutical Sciences, Monash University, Melbourne, Victoria, Australia"},{"name":"School of Public Health and Preventive Medicine, Monash University, Melbourne, Victoria, 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