Cost-effectiveness of evolocumab in patients with familial hypercholesterolemia in Belgium

Background and Aims: Evolocumab, a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, is indicated for the reduction of cardiovascular (CV) risk by lowering low-density lipoprotein cholesterol (LDL-C). We assessed the cost-effectiveness of evolocumab added to optimized standard of care (SoC), i.e. maximally tolerated statin with ezetimibe, in patients with Familial Hypercholesterolemia (FH) in Belgium. Methods: A previously published Markov model was adapted to the Belgian context. Patient characteristics, aligned with reimbursement criteria, were taken from a cohort of Belgian patients with FH. Baseline CV event (myocardial infarction, ischemic stroke, CV death) rates, utilities and costs were taken from published sources. The model used an evolocumab LDL-C reduction of 59%, as observed in the FOURIER trial, and the relationship between LDL-C lowering and CV event reduction from the Cholesterol Treatment Trialists’ Collaboration (CTTC) 2010 meta-analysis. An annual cost of evolocumab of €5,440 was utilized, based on its list price (before any potential discounts). Costs and health outcomes were evaluated over a lifetime horizon from payer perspective. Results: In primary prevention (PP) patients with FH and LDL-C ≥130 mg/dL, the incremental cost-effectiveness ratio (ICER) was €45,484. In secondary prevention (SP) patients with FH and LDL-C ≥100 mg/dL, the ICER was €36,627. In a combined PP (24%) and SP (76%) population, weighted according to evolocumab use observed in Belgium, the ICER was €38,770 (see table). [Formula presented] Conclusions: In Belgian patients with FH eligible for reimbursement, the addition of evolocumab to optimized SoC results in an ICER below generally accepted willingness-to-pay thresholds by Belgian authorities and may be considered cost-effective.