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Comment & Response
November 22/29, 2016

Cost-effectiveness of PCSK9 Inhibitor Therapy—Reply

Author Affiliations
  • 1Department of Medicine, University of California, San Francisco
  • 2College of Physicians and Surgeons, Columbia University, New York, New York
JAMA. 2016;316(20):2152. doi:10.1001/jama.2016.16292

In Reply Ms Mastey and Dr Johnstone are incorrect in their assessment of our results and the goals of our analysis. The Cardiovascular Disease Policy Model is calibrated to actual, observed CVD events in the general US population and predicts myocardial infarctions, strokes, and cardiovascular and noncardiovascular mortality within 1% of estimates from national vital statistics and the US National Hospital Discharge Survey (for the model base year of 2010; eTable 3 in the online supplement to the article). Our reference-case estimates of cardiovascular risk among patients with familial hypercholesterolemia are nearly identical to those observed in a pooled analysis of 6 US epidemiological cohorts.1 Doubling the predicted risk in sensitivity analyses improved the incremental cost-effectiveness ratio, but PCSK9 inhibitor therapy still did not meet conventionally accepted thresholds of cost-effectiveness. Our reference case did incorporate quality-of-life benefits from avoiding CVD events, and when we assumed even more severe disease-related disability in patients with CVD in sensitivity analyses—thus magnifying the potential benefits associated with PCSK9 inhibitor use—the results did not change materially (eTable 14 in the online supplement to the article).

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