management of patients eligible for proprotein convertase subtilisin/kexin type 9 (pcsk9) inhibitors: Economic impact and reform proposals

Background: PCSK9 inhibitors are indicated for primary hypercholesterolemia (heterozygous familial and non-familial) and for homozygous familial hypercholesterolemia (only evolocumab). They are subject to a drug registry for patient eligibility and follow-up. Patient access to PCSK9 inhibitors is still very limited and varies across regions. Aim: This paper aimed to investigate the cost of the PCSK9-treated patient pathway and main barriers to patient access to medicines and to suggest some changes to this pathway. Methods: The research was conducted through a literature review and an expert panel, which was surveyed through a Delphi approach. The panel included seven clinicians and five pharmacists. Results: If patients are exempt from co-payment, the Italian National Health Service spends €169.70 and €276.93 per year to manage the PCSK9-treated patient pathway, depending on registry coverage (six or three treatment months, respectively). Non-exempt patients pay €176,43 and €271.53 (for six and three months of coverage, respectively) per year. The referral process and the length of the pre-PCSK9 treatment period were considered the main barriers to access. The panelists suggested that the pre-PCSK9 treatment period be shortened to three months; they recommended six months of registry coverage, thereby decreasing the costs compared to three months' coverage; and they advised that outpatient specialists also be authorized to manage the follow-up. These actions may reduce pathway costs and enhance awareness of the disease. Discussion: The study shows that the pathway of patients eligible for PCSK9 inhibitors can be improved. The surveyed experts integrated pathway-specific recommendations with broader advice to increase awareness on hypercholesterolemia in both citizens and general practitioners.