Introduction and objectives: A cost-effectiveness model was developed to evaluate the efficiency of different preventive strategies in familial hypercholesterolemia (FH) in comparison with routine clinical practice (CP): atorvastatin monotherapy, 40 mg (A40) or 80 mg (A80, and atorvastatin combined with ezetimibe, 10 mg (A40+E10 or A80+E10).
Methods: A longitudinal population model with a time horizon for life-expectancy was developed within the context of the Spanish public healthcare system. Life tables for the Spanish population (2002) were modified using the standardized mortality rate for individuals with FH. Effectiveness was expressed in life-years gained (LYG), after taking into account reductions for risk (ie, Framingham risk score) and cardiovascular mortality. The costs (in 2005 terms) of the intervention (CI) and care (CC) were discounted at 6%, while effects were discounted at 3%.
Results: Routine CP, based on the Spanish FH registry: 1.97 LYG per patient vs. no treatment; CI euro5321, CC euro23,389. A40: 2.59 LYG; reduction in CC compared with CP 4.5%; total costs (TC) euro30 569. A80: 2.75 LYG; reduction in CC 6.4%; TC euro30 133. A40+E10: 3.38 LYG; reduction in CC 14.3%; TC euro36 104. A80+E10: 3.62 LYG; reduction in CC 17.6%; TC euro35 317. From most to least efficient strategy, the incremental cost-effectiveness per LYG compared with CP was: a) A80: euro1821; b) A40: euro3012; c) A80+E10: euro4021, and d) A40+E10: euro5250.
Conclusions: Preventive treatment of FH with atorvastatin was cost-effective. The greatest cost-effectiveness was obtained with atorvastatin monotherapy, 80 mg. The addition of ezetimibe could produce further benefits at an acceptable incremental cost.