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Research Letter
September 2017

Revisiting Outcomes–Based Pricing Propositions for the PCSK9 Inhibitor Evolocumab

Author Affiliations
  • 1Department of Pharmacy and Therapeutics, School of Pharmacy, University of Pittsburgh, Pittsburgh, Pennsylvania
JAMA Intern Med. 2017;177(9):1388-1390. doi:10.1001/jamainternmed.2017.3143

The approval of the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor evolocumab was based on the 61% reduction in the surrogate outcome of low-density lipoprotein cholesterol shown in clinical trials,1 which, based on the low-density lipoprotein hypothesis, was expected to translate into a 36% to 39% reduction in the risk of cardiovascular events.2 Under these conditions, Kazi et al2 and Arrieta et al3 predicted PCSK9 inhibitors would be cost-effective at an annual price of $4250 to $4500, and the Institute for Clinical and Economic Review recommended an annual price of $2177,4 estimates considerably lower than the price Amgen set for evolocumab($14 000). The results of the Further Cardiovascular Outcomes Research with PCSK9 Inhibition in Subjects with Elevated Risk (FOURIER) trial,5 powered to evaluate the efficacy of evolocumab in preventing cardiovascular events, were recently published. In the FOURIER trial,5 evolocumab was associated with a 27% and 21% reduction in the risk of myocardial infarction (MI) and stroke, respectively. There was no reduction in cardiovascular death or all-cause mortality. Following the publication of these results, which failed to meet the expectations generated by previous predictions,2 Amgen announced its willingness to engage in contracts where the cost of evolocumab would be refunded for patients who have an MI or a stroke while using the drug. In this article, I simulate the annual price of evolocumab under the outcome-based contracts proposed by Amgen, and put my estimates in the context of the available evidence on the cost-effectiveness of PCSK9 inhibitors.