Aderenza, persistenza e analisi di costo-efficacia in pazienti in trattamento con inibitori di PCSK9

Introduction. Dyslipidemia is a metabolic disorder characterized by alterations in serum lipid levels. PCSK9 has emerged as a therapeutic target for hypercholesterolemia, with three main agents: alirocumab, evolocumab, and inclisiran currently available. While the monoclonal antibodies alirocumab and evolocumab inhibit circulating PCSK9, inclisiran is a siRNA that reduces PCSK9 synthesis at the hepatic level. These therapies are mainly indicated for secondary prevention of ASCVD and for severe primary hypercholesterolemia. This study aimed to assess adherence, persistence, and cost-effectiveness of PCSK9 inhibitors in a real-world clinical setting within a local health authority (ASL). Materials and methods. A retrospective analysis was conducted on administrative pharmacy database. Patients naïve to PCSK9 inhibitors between 12 April 2023 and 28 February 2025 were included. Adherence was measured via the Medication Possession Ratio (MPR), while persistence to treatment was defined as the time from treatment initiation to its discontinuation. A 5-year cost-effectiveness analysis from the NHS perspective was conducted using ex-factory prices. Incremental Cost-Effectiveness Ratios (ICERs) were calculated based on 5-year treatment persistence. Results. A total of 103 patients were analyzed: 34 received alirocumab, 27 evolocumab, and 42 inclisiran. Median age was 68 years, with 61.17% male. Mean MPR was 0.9341 (alirocumab), 0.8614 (evolocumab), and 0.9457 (inclisiran). One-year persistence was 47.05% (alirocumab), 70.37% (evolocumab), and 83.33% (inclisiran). Alirocumab had the lowest 5-year cost (€25,529.70), followed by evolocumab (+0.16%) and inclisiran (+4.80%). Evolocumab demonstrated the most favorable ICER (€1.90 per percentage point gained), while inclisiran showed the highest persistence but at a higher incremental cost (€37.43/p.p.). Conclusions. Inclisiran showed the highest adherence and persistence, potentially due to its biannual dosing. Evolocumab exhibited the best cost-effectiveness profile. This study highlights the importance of integrating real-world evidence and pharmacoeconomic assessments in therapeutic decisions. The clinical pharmacist plays a critical role in leveraging such data to support sustainable and value-based healthcare models.