Cost-Effectiveness Assessments of PCSK9 Inhibitors

Study Questions:

Are proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors cost-effective when added to statin therapy?


Results from the FOURIER (Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk) trial were used to determine the cost-effectiveness of a PCSK9 inhibitor and statin therapy as compared to statin therapy alone. A Markov model was created to examine the cost-effectiveness of the two lipid-lowering strategies (statin plus PCSK9 inhibitor vs. statin alone) over a time horizon of a lifetime. The model used a hypothetical cohort of 1,000 patients that resembled the FOURIER population as a baseline group. Observed rates of events, outcomes, cost of care, and health insurance were derived from existing literature. The primary outcomes of interest included incremental cost-effectiveness ratio from a health system perspective, and the return on investment from a private payer perspective. For both outcomes, the annual PCSK9 inhibitor drug price was assumed to be $14,300, with a lapse in US patent protection that would reduce the price by 43% in year 12. Costs were reported in 2016 US dollars.


Using a model which included 1,000 hypothetical patients with attributes similar to those of the FOURIER trial cohort, at the current drug price, the incremental cost-effectiveness ratio of statin plus PCSK9 inhibitor therapy was $337,729 per quality-adjusted life-year. The probabilistic sensitivity analysis found that a statin plus PCSK9 inhibitor strategy had a low probability (<1%) of being cost-effective at the commonly accepted societal threshold of $100,000 per quality-adjusted life-year. A PCSK9 inhibitor produced a negative return on investment of 86% for private payers. In the threshold analysis, the price of PCSK9 inhibitors would need to drop 62%, to $5,459 per year, to reach $100,000 per quality-adjusted life-year.


The authors concluded that at current prices, the addition of a PCSK9 inhibitor to statin therapy is estimated to provide an additional quality-adjusted life-year for $337,729. Significant discounts are necessary to meet conventional cost-effectiveness standards.


These models provide important information for all of those interested in providing high-quality care with an acceptable cost. For PCSK9 inhibitor therapy to be widely used, prices will likely need to be reduced.

Clinical Topics: Diabetes and Cardiometabolic Disease, Dyslipidemia, Prevention, Lipid Metabolism, Nonstatins, Novel Agents, Statins

Keywords: Cost-Benefit Analysis, Dyslipidemias, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Insurance, Health, Lipids, Outcome Assessment (Health Care), Primary Prevention, Proprotein Convertases, Quality-Adjusted Life Years, Risk, Subtilisins

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