What evidence is there to support the use of PCSK9 inhibitors in primary prevention?
The evidence of benefit for the use of PCSK9 inhibitors in primary prevention is lacking. This is reinforced by a 2018 cost-effectiveness analysis and a 2019 cost-effectiveness analysis by Kazi et al., which found that PCSK9 inhibitors did not meet a willingness to pay (WTP) threshold of $100,000 per additional quality-adjusted life year (QALY). Despite the dearth of evidence for primary prevention with PCSK9 inhibitors, there is heavy industry marketing and enthusiasm surrounding new therapies that may influence some patients and providers to consider its use. The Work Group determined a Strong against recommendation was warranted based on the lack of evidence for reducing CV risk, the unknown long-term adverse event/safety profile in a primary prevention population, and the treatment's prohibitive cost. More research on the safety and effectiveness of PCSK9 inhibitors, specifically in the primary prevention populations, is needed.