OBJECTIVES: Recently published clinical guidelines recommend the use of ezetimibe and Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) inhibitors for patients with persistent high LDL-C despite treatment with maximal tolerated statin dose. The objective of this study was to systematically review the cost-effectiveness of ezetimibe and PCSK9 inhibitors for these patients.

METHODS: We searched PubMed, Embase, and Cochrane Database of Systematic Reviews to identify studies published from May 2007 to April 2017. Full-text, original articles evaluating the economic value of ezetimibe and PCSK9 inhibitors were selected. From the selected articles, two reviewers independently extracted data on the study population, comparators, economic model, perspective, time horizon, discount rate, type of costs, clinical data source, economic and clinical outcomes, sensitivity analyses, funding source, and country.

RESULTS: A total of 13 articles meeting all inclusion criteria were reviewed. Cost-effectiveness analyses were conducted in six different countries including the US (n=5) and UK (n=4). All studies selected a lifetime horizon except one study with a 5-year time horizon. Only two studies adopted a societal perspective. Of the 13 studies, seven studies included ezetimibe in their economic evaluations. Ezetimibe monotherapy was considered to be cost-effective compared with no treatment. The ezetimibe/simvastatin combination therapy was also cost-effective compared with statin therapy. However, ezetimibe combined with other statins was not always considered to be cost-effective compared with statin therapy. Five studies included PCSK9 inhibitors, and one study included both ezetimibe and PCSK9 inhibitor in their cost-effectiveness analyses. The PCSK9 inhibitor monotherapy was not cost-effective compared with statins or ezetimibe. The cost-effectiveness of the PCSK9 inhibitor/statin combination therapy was not consistent across studies.

CONCLUSIONS: Given the current cost of ezetimibe and PCSK9 inhibitors, their cost-effectiveness results depended largely on the types of statins being used together and comparator treatments. Cost reductions of ezetimibe and PCSK9 inhibitors may potentially impact their cost-effectiveness in future.