Cost-Effectiveness of PCSK9 Inhibitor Therapy in HeFH or ASCVD

Study Questions:

What is the cost-effectiveness of PCSK9 inhibitors and potential effect on US health care spending on the Food and Drug Administration–approved indications including heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease (ASCVD)?


The Cardiovascular Disease Policy Model included the entire population of US adults aged 35-94 years in 2015, and followed them over their lifetime (until death or survival to 95 years of age). The model incorporated 2015 annual PCSK9 inhibitor costs of $14,350 (based on mean wholesale acquisition costs of evolocumab and alirocumab) and adopted a health-system perspective.


Adding PCSK9 inhibitors to statins in HeFH was estimated to prevent 316,300 major adverse cardiac events (MACE) at a cost of $503,000 per quality-adjusted life-year (QALY) gained compared with adding ezetimibe to statins. In ASCVD, adding PCSK9 inhibitors to statins was estimated to prevent 4.3 million MACE compared with adding ezetimibe at $414,000 per QALY. Reducing drug costs to ≤$4,536 per year per patient would be needed for PCSK9 inhibitors to be cost-effective at <$100,000 per QALY. At 2015 prices, PCSK9 inhibitor use in all eligible patients was estimated to reduce CV care costs by $29 billion over 5 years, but drug costs increased by an estimated $592 billion (a 38% increase over 2015 prescription drug expenditures).


Assuming 2015 prices, PCSK9 inhibitor use in patients with HeFH or ASCVD did not meet generally acceptable incremental cost-effectiveness thresholds and was estimated to increase US health care costs substantially. Reducing annual drug prices from >$14,000 to $4,536 would be necessary to meet a $100,000 per QALY threshold.


Each of the available PCSK9 inhibitors reduces the low-density lipoprotein cholesterol (LDL-C) by about 60% regardless of statin therapy. Accurate cost/benefit analysis of therapy superimposed on maximal tolerated dosing of statins or alone in statin-intolerant patients awaits the results of the 5-year outcome data. And it may well vary by baseline LDL-C. Importantly, this analysis did not consider the evidence that CV event rates in HeFH patients who have lifelong exposure to very high LDL-C levels are three- to four-fold that of adults patients with equivalent LDL-C >190 mg/dl. The eventual cost/benefit will depend on additional value over statins in HeFH and high- and intermediate-risk individuals.

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