Background and importance Hypercholesterolaemia produces a higher risk of atherosclerosis and cardiovascular events. The proprotein convertase subtilisin kexin type 9 inhibitors (PCSK9i), evolocumab and alirocumab, were approved by the European Medicines Agency in 2015, and they are available to manage patients who have not achieved the target cholesterol levels or who are intolerant to the standard treatment with statins or ezetimibe. Nevertheless, due to their high budgetary impact, it is crucial to find measures to optimise their use.

Aim and objectives The aim of the study was to analyse the effectiveness and costs of the optimised PCSK9i regimen compared to the standard dosage regimen.

Material and methods A retrospective cohort study was conducted in patients who began using PCSK9i between September 2017 and September 2021. In patients with a reduction in low-density lipoprotein cholesterol (LDLc) greater than 50% or who have reached their target value, alirocumab 150 mg/4 weeks or evolocumab 140 mg/21 days were proposed for optimisation of the dosage. Demographic, clinical and pharmacotherapeutic data were collected. Treatment efficacy was calculated as percent reduction in LDLc from baseline at treatment initiation to the end of the study period. The collected data were analysed using a Student’s test through the SPSS programme.

Results Twenty-two patients were included, 9 males, with a median age of 62 (range 42–82) years, median treatment time was 22.52 (1.27–49.30) months and initial LDLc values of 161 (101–237) mg/dL. Fifteen patients (68%) were treated with alirocumab. Two patients discontinued treatment. Two patients were excluded because the treatment was not effective. Nine patients (45%) were proposed to optimise doses. In these patients the mean LDLc value was 75.66±41.21 mg/dL and a reduction of 48.33%±26.87, while in patients on standard doses it was 90.72±59.70 mg/dL and 51.52%±26.72, respectively. The difference was not significant (p>0.05). The optimised doses involve a saving of €2040.97/patient/year in alirocumab and €1417.65/patient/year in evolocumab.

Conclusion and relevance The optimised use of PCKS9i is an effective measure and would mean a reduction in the direct costs in the treatment of hypercholesterolaemia.

It is necessary to search for strategies that help to reduce the budgetary impact to optimise health resources without damaging treatment effectiveness.

Conflict of interest No conflict of interest