Study questions cost-effectiveness of PCSK9 inhibitors

Study data published Tuesday in JAMA indicate that the prices of PCSK9 inhibitors including Amgen's Repatha (evolocumab) and Sanofi and Regeneron Pharmaceuticals' Praluent (alirocumab) should be dramatically cut to make them cost-effective in the US. In the study, the researchers estimated that the treatments, which on average cost more than $14 000 per patient per year, could increase healthcare spending in the US by as much as $120 billion annually. Study author Kirsten Bibbins-Domingo remarked "this hopefully should prompt some discussion about what are reasonable or tolerable drug prices in the US." 

In the study, the investigators used a simulation model to assess the cost-effectiveness of PCSK9 inhibitors and ezetimibe when administered on top of statin therapy in the treatment of heterozygous familial hypercholesterolemia (FH) or atherosclerotic cardiovascular disease (ASCVD). The model adopted an annual cost of $14 350 for PCSK9 inhibitors based on the mean wholesale price of Repatha and Praluent. The main outcome measures of the study included major adverse cardiovascular events, incremental cost per quality-adjusted life-year (QALY) and total effect on US healthcare spending over a five-year period. 

Findings from the study suggested that the addition of PCSK9 inhibitors to statin therapy would prevent 316 300 major adverse cardiovascular events in patients with heterozygous FH at a cost of $503 000 per QALY versus adding ezetimibe to statins. Meanwhile, for ASCVD, the data indicated that PCSK9 inhibitors added to statins would prevent 4.3 million major adverse cardiovascular events at a cost of $414 000 per QALY compared with the addition of ezetimibe. 

Additionally, based on 2015 prices, the researchers estimated that while the use of PCSK9 inhibitors in all eligible patients would decrease cardiovascular care costs by $29 billion over five years, drug costs would rise by $592 billion over the same period. Conversely, they estimated that initiating statin therapy in all statin-tolerant people in this high-risk population would reduce healthcare spending by $12 billion. 

Commenting on the news, Amgen indicated that it "strongly believes in the clinical and economic value of Repatha," adding that the study presents "just one view" on the drug. The company added that other research identified a "value-based price" for Repatha as high as $15 000, exceeding both the current list price of the drug and the net price paid by purchasers in the US. 

In addition, Steven Nissen, chairman of cardiovascular medicine at the Cleveland Clinic, suggested it is too soon to attempt to determine the cost-effectiveness of PCSK9 inhibitors because their full benefits are not yet known. Amgen expects to report data from a study of Repatha's effects on cardiovascular outcomes in the first quarter of next year, while Regeneron estimated that interim outcome data for Praluent could be unveiled as soon as this year. 

Meanwhile, an analysis released last year by the Institute for Clinical and Economic Review (ICER) indicated that the prices of PCSK9 inhibitors should be slashed by as much as 67 percent to ensure their cost-effectiveness. 


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