OBJECTIVES

To develop a new cost-effectiveness model, using real-world data, to predict long-term health outcomes and costs of patients with familial hypercholesterolaemia (FH), with and without treatment, and considering the increased effect of low-density lipoprotein cholesterol (LDL-C) over time on cardiovascular risk (known as cholesterol burden).

METHODS

We used routinely collected healthcare and mortality data from FH patients in the UK to estimate parametric survival models for cardiovascular events given age, sex, LDL-C, prior cardiovascular history, and to estimate the effect of treatment on LDL-C. We developed a new cohort Markov model over a lifetime horizon to predict health outcomes (e.g., quality-adjusted life years, QALYs) and UK National Health Service costs. Since the routine data was from treated patients, we estimated the counterfactual risk had they not been treated with and without accounting for cholesterol burden.

RESULTS

Accounting for cholesterol burden, treatment increases event-free survival by up to 30 years with greater benefits in younger patients, men, those with higher LDL-C and prior cardiovascular history. Treatment results in savings in those with LDL-C < 4.5 mmol/L and, in most patients, is cost-effective (incremental net health benefit at £15,000/QALY up to 2.4 QALYs). If cholesterol burden is not considered, the model predicts smaller benefits from treatment (e.g., 11 years event-free survival gain) and, in some patients, it is not cost-effective (e.g., patients < 18 years with LDL-C < 4 mmol/L).

CONCLUSIONS

Treatment results in large health benefits and is highly cost-effective, but magnitude of benefits depends on whether cholesterol burden is considered, particularly in younger patients. Further research is warranted on effect size of cholesterol burden and how best to consider it in cost-effectiveness modelling.