OBJECTIVES : Describe published cost-utility analyses of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors.

METHODS : A systematic literature review was conducted according to the National Institute for Health and Care Excellence guidelines. Cost-utility analyses assessing addition of PCSK9 inhibitors to standard of care (SoC) of statins and/or ezetimibe in patients with cardiovascular (CV) disease were included. Studies in English, published between 01/01/2010 and 31/01/2020, were considered. Data on methods, inputs, assumptions, and outcomes were extracted.

RESULTS : We identified 16 published cost-utility analyses of PCSK9 inhibitors added to SoC in patients with CV disease. Nine analyses were conducted in the United States, four in Europe, and three in Asia-Pacific. All studies were based on Markov state transition models. Evolocumab added to SoC was the intervention in 12 evaluations, three considered any PCSK9 inhibitor added to SoC, and one alirocumab added to SoC. Baseline CV event rates reflecting real-world populations were used in 10 analyses. The remaining six used estimates reflecting randomized clinical trial populations. Ten studies did not report the actual baseline CV event rates figures used. Baseline levels of low-density lipoprotein cholesterol (LDL-C) ranged between 2.38 and 4.00 mmol/L. Relative LDL-C reduction from baseline ranged between 48% and 72%, translating into absolute LDL-C reductions between 1.37 and 2.59 mmol/L. In nine economic evaluations, the efficacy of PCSK9 inhibitors was modeled using LDL-C reduction as a surrogate endpoint for CV risk reduction, while seven directly used hazard ratios observed in clinical trials to model the reduction in the incidence of CV events.

CONCLUSIONS : Several cost-utility analyses of PCSK9 inhibitors used model inputs, such as event rates or hazard ratios, directly from clinical trials. These may differ from real-world evidence and therefore produce differing cost-effectiveness results. Publications of economic evaluations of PCSK9 inhibitors may benefit from more transparent reporting of parameters used in economic models.