Study Finds PCSK9s Are Not Cost-Effective, and Drugmakers Aren't Happy

A new class of cholesterol-fighting drugs would need to cost less than a third of its list price to be cost-effective, according to a study in JAMA.

The analysis, funded by the Institute for Clinical and Economic Review (ICER), was immediately panned by the makers of a pair of PCSK9 inhibitors, Amgen’s evolocumab (Repatha) and Sanofi-Regeneron’s alirocumab (Praluent). Both drugs were approved last summer and have list prices of about $14,000 a year.

Researchers found that if used on all eligible patients, the new class could cut hospitalizations and other costs by $29 billion a year, but the drugs would cost $568 billion. They found that the drugs would need to cost $4536 a year to meet accepted value standards.

Both drugs are approved to treat familial hypercholesterolemia, a rare genetic condition that elevates low-density lipoprotein (LDL) cholesterol, as well as atherosclerotic cardiovascular disease (ASCVD). Clinical trials have shown the drugs can cut LDL cholesterol levels by about 60%.

“This economic evaluation of PCSK9 inhibitors from the US health system perspective demonstrates that their use as indicated could substantially reduce (myocardial infarction), strokes, and cardiovascular deaths,” the authors write. “However, even if they prove highly effective,” in preventing cardiac events, “PCSK9 inhibitors are not cost-effective at 2015 prices.”

Sales for both drugs have been below estimates, with pharmacy benefit managers (PBMs) and health plans setting up protocols for who can have access to the drugs. Restricting the drugs to the most high-risk patients—which is exactly what some PBMs are doing—does not make PCSK9 inhibitors cost effective, the researchers found.

FDA approvals are more limited than those in Europe, while regulators await results from long-term safety trials. Those results are expected in late 2016 for alirocumab and early 2017 for evolocumab.

The study’s calculations are based on a simulation that used $100,000 as the value for a quality-adjusted life year. This is a standard level used in many cost-effectiveness studies, but Sanofi and Regeneron told Medical Marketing and Media that formal cost-effectiveness thresholds do not exist in the United States, and that $150,000 is a more accepted value.

Also, the drugmakers said, the study underestimates how many people will have CV events.

Amgen, meanwhile, pointed to a different study in Clinical Cardiology that did not find evolocumab’s price excessive; however, Amgen paid for the study.

John Rother, executive director for the Campaign for Sustainable Drug Rx Pricing, an advocacy group, said in a statement that the study affirms the group’s stance that PCKS9 inhibitors are overpriced.

“For more than a year, CSRxP has been calling attention to outrageously prices PCSK9 inhibitors like Repatha and Praluent,” he said. “The dangerous trend of high-priced drugs has to be reversed, and a common sense solution like pricing medications according to value and cost-effectiveness is one necessary step to fix the broken prescription drug market, but it must also be combined with increased transparency and competition.”

Reference

Kazi DS, Moran AE, Coxson PG, et al. Cost-effectiveness of PCSK9 inhibitor therapy in patients with heterozygous familial hypercholesterolemia or atherosclerotic disease. JAMA. 2016; 316(7):743-753.