Potential barriers in lipid-lowering treatment with PCSK9 inhibitors from a healthsystem perspective. Comparative evidence from ten countries

Abstract

Background Gaps between clinical guidelines and attainment of LDL-C goals are evident across jurisdictions. Despite strong evidence of benefit from PCSK9-inhibitors (PCSK9i) treatment in eligible patients, significant underuse remains, suggesting considerable unmet need in clinical outcomes optimization. Purpose We investigated key performance endpoints across 10 countries to provide a comparative assessment of potential barriers in PCSK9i therapeutic integration from a healthcare system perspective. Methods We performed secondary analysis of peer-reviewed literature, health technology assessment reports, guidelines, clinical pathways since 2015 and constructed a comparative framework of pre-defined endpoints for 10 study countries (Japan, Italy, Spain, Australia, Canada, United Kingdom, Germany, France, Netherlands, USA). We identified 8 endpoints, which were clustered in 3 domains: (a) healthcare system characteristics, (b) demand-side policies, (c) medicine reimbursement policies, pertaining to cost-sharing. Approved PCSK9i indications were in scope. Results PCSK9i are reimbursed in all countries. Prescribing restrictions have been applied in all countries (Table 1). PCSK9i for primary prevention are reimbursed mainly in familial hypercholesterolemia, while additional criteria may apply depending on country and condition. PCSK9i are: (a) reserved mainly for very high- or high-risk cohorts (Japan, UK, Netherlands); (b) reimbursed for secondary prevention when additional ris…