How does cholesterol burden change the case for investing in Familial Hypercholesterolaemia? A cost-effectiveness analysis.

This study aimed to ascertain how the long-term benefits and costs of diagnosis and treatment FH vary by prognostic factors and ‘cholesterol burden’, which is the effect of long-term exposure to LDL-C on cardiovascular disease (CVD) risk. If cholesterol burden is considered, diagnosis resulted in positive net health gain (i.e., it is cost-effective) in all individuals with pre-treatment LDL-C ≥ 4 mmol/L, and in those with pre-treatment LDL-C ≥ 2 mmol/L aged ≥50 years or who have CVD history. If cholesterol burden is not considered, diagnosis resulted in lower net health gain, but still positive in children aged 10 years with pre-treatment LDL-C ≥ 6 mmol/L and adults aged 30 years with pre-treatment LDL-C ≥ 4 mmol/L.  Economic evaluations of FH interventions should consider the sensitivity of the study conclusions to cholesterol burden, particularly where interventions target younger patients.