OBJECTIVES: PCSK9 inhibitors (PCSK9i) are effective lipid lowering treatments (LLT) but their high cost has created affordability issues for healthcare systems. In Ireland, a Managed Access Protocol (MAP) restricts access to PCSK9i’s high risk subgroups in which they are considered most cost-effective. This study aimed to determine patients for whom additional LLT is recommended by ESC guidelines, but are ineligible for PCSK9i reimbursement due to MAP criteria.

METHODS: Patient data from St. James’s Hospital Lipid Clinic during October-December 2021 was analysed. Factors included in the analysis were low density lipoprotein (LDL) levels, history of cardiovascular disease (CVD), heterozygous familial hypercholesterolaemia (HeFH), ESC CVD risk classification, and LLT. Patients not reaching their ESC LDL targets despite high intensity LLT (atorvastatin ≥40mg & ezetimibe, rosuvastatin ≥20mg & ezetimibe), or due to statin intolerance were identified. Then this cohort was compared to MAP clinical criteria (genetically proven HeFH diagnosis or previous myocardial infarction (MI) or coronary artery bypass graft (CABG)) as well as its LDL cut-off ≥4mmol/L.

RESULTS: 129 patients being treated primarily for hypercholesterolemia formed the cohort for analysis. Of these, 50% were on high intensity LLT (n=40) or intolerant of statins (n=24). 69% of the 64 patients (n=44) had LDL levels above their ESC guideline target. These patients were the group who may benefit from treatment with a PCSK9i. Of the 44 patients, 55% (n=24) met MAP clinical criteria (HeFH=20 patients, MI/CABG=4). Of these 24 patients, only 38% (n=9) also met MAP LDL criteria. In summary, of those patients on high intensity LLT or intolerant of statins with LDL above the ESC target, 20% met MAP criteria.

CONCLUSIONS: A MAP has been created due to excessive drug pricing. Most patients not attaining their LDL goals on high intensity LLT, or intolerant of LLT, are ineligible for PCSK9i’s due to MAP reimbursement criteria.