Early detection and treatment of familial hypercholesterolemia in children cost effective and cost saving

The authors state that the interpretations and findings are conditional on assumptions basic in the health economic model.

Familial hypercholesterolemia occurs due to a genetic abnormality affecting the function, structure, or metabolism of the low-density lipoprotein (LDL) receptor, resulting in increased plasma LDL-C (LDL cholesterol). Several gene variants can underlie FH, including those involving the LDL receptor, apolipoprotein B, or PLSK9 (proprotein subtilisin/kexin type 9), which leads to a marked increase of plasma LDL-C from birth and, without intervention, throughout life.

The Netherlands has a long-term history of active screening for familial hypercholesterolemia, enabling health-economic analyses. Zanfina Ademi, Monash University, Melbourne, Victoria, Australia, and colleagues, therefore, investigated the cost-effectiveness and the ROI (return on investment) of a nationwide cascade case-finding and preventive treatment program starting with FH identification in children and treatment from both a health care and societal perspective.

For this purpose, early preventive treatment and cascade case-finding was modelled to stimulate the disease progression and costs of 10-year-olds doubted of having heterozygous familial hypercholesterolemia over a lifetime. The model comprised three health states: alive with coronary heart disease (CHD), alive without CHD, and deceased. The risk of a first CHD event was quantified as a function of age and total lifetime exposure to LDL cholesterol. Cost-effectiveness was defined as $21 800 per gain in QALYs (quality-adjusted life-years), using incremental cost-effectiveness ratios (ICERs). All future costs and benefits were discounted annually by 4% and 1.5%, respectively.

A comparison was drawn between two strategies; (1) cascade screening and treatment initiation with statin children (mean age ten years) and (2) no screening, later detection, and treatment.

The authors reported the following findings:

• In this model constructed to simulate the FH progression bin 1000 hypothetical 10-year-olds, from a healthcare perspective, the program would gain 2.53 QALYs per person at an additional cost of €23 365 ($25 468) (both discounted). These amount to an ICER of €9220 ($10 050) per gain in QALY.
• From the societal perspective, the treatment and detection treatment program were cost-saving over a lifetime versus no cascade screening for familial hypercholesterolemia.
• The ROI for the detection and treatment program for familial hypercholesterolemia in children was €8.37 ($9.12).

To conclude, early detection and treatment program for FH in children offers excellent value for investment, being both health and cost-saving and cost-effective from a healthcare perspective.

Reference:

Ademi Z, Norman R, Pang J, et al. Cost-effectiveness and Return on Investment of a Nationwide Case-Finding Program for Familial Hypercholesterolemia in Children in the Netherlands. JAMA Pediatr. Published online May 01, 2023. doi:10.1001/jamapediatrics.2023.0763