OBJECTIVES: An elevated level of low-density lipoprotein cholesterol (LDL-C) constitutes an important modifiable risk factor for cardiovascular disease (CVD). Individuals with heterozygous familial hypercholesterolaemia (HeFH) are particularly vulnerable to CVD events and often do not achieve adequate reduction of LDL-C with standard of care (SoC). The objective of this analysis was to demonstrate the clinical and economic value of LDL-C lowering with evolocumab in HeFH patients from the Bulgarian public health care perspective. METHODS: A disease-specific measure of health benefit was devised: Effectively treated patient-years (ETPYs) combine length of life with the likelihood of attaining best-practice recommendations on LDL-C lowering aimed at reducing CVD events. ‘Effective treatment’ was defined as a reduction in LDL-C levels of ≥50%. Considering a life-long treatment duration, a Markov model was adapted to compare the addition of evolocumab to SoC (high-intensity statins) versus SoC alone. Demographics, baseline characteristics and efficacy were taken from pertinent trial data. The model uses the relationship between LDL-C lowering and reduced CVD event rates observed in the Cholesterol Treatment Trialists' Collaboration (CTTC) meta-analyses. RESULTS: The total incremental costs of evolocumab added to SoC versus SoC alone are BGN120,111 while adding 9.35 ETPYs over lifetime. These results imply an incremental cost per ETPY of BGN12,846 (US$7,258; €6,559). The use of evolocumab is associated with a relative reduction in the CVD event rate by 46% (22% per 1 mmol/L), consistent with CTTC. CONCLUSIONS: The addition of evolocumab to SoC may be considered cost-effective in light of an additional expense per patient-year gained in which individuals with HeFH receive effective treatment under the terms of international prevention guidelines. ETPYs are an intuitive and clinically meaningful measure of patient benefit that, in relation to costs, can support healthcare decision-making not subject to the cost-per-QALY paradigm, and that recognises and considers process-oriented factors of value, specifically quality of care.