Drugmakers condemn ICER-funded study on PCSK9s



A new class of cholesterol-lowering treatments, PCSK9 inhibitors, was deemed too expensive when compared to their value to patients, according to a new study partially funded by ICER.

The companies who market these drugs challenged the study's methodology and refuted its conclusions, with Sanofi and Regeneron saying that studies like this one do a “major disservice to patients.”

The study, published Tuesday in JAMA, said that PCSK9 inhibitors, like Amgen's Repatha and Sanofi's and Regeneron's Praluent, if used in all eligible patients, would reduce cardiovascular care costs like hospitalizations by $29 billion over five years, but that drug costs would simultaneously increase by an estimated $568 billion.

Using these drugs to either treat patients with heterozygous familial hypercholesterolemia as well as to reduce the risk of cardiovascular disease among the American public “would increase healthcare costs substantially,” they wrote.

See also: PBMs push forward toward outcomes-based pricing, despite challenges

At launch, Praluent had a wholesale acquisition cost of $14,600, which excludes discounts and rebates; Repatha cost slightly less, at $14,100. The study estimated that the price of the drugs would have to drop from roughly $14,000 to $4,563 to be cost-effective, at a $100,000 per quality-adjusted life year (QALY) threshold. QALY measures life expectancy combined with the quality of life-years remaining.

Amgen said in a statement that research published in Clinical Cardiology showed that Repatha has a “value-based” price of up to $15,000. Amgen financially supported that analysis.

The company also noted that the JAMA study is “another view” on the economic and clinical value of Repatha. “These types of assessments are focused on ringing alarm bells from a payer perspective, rather than focusing on a rigorous analysis that fully reflects the patient perspective of value,” Amgen said. This is not the first ICER-funded study that Amgen has come out against. In March, it criticized an upcoming ICER report that would compare the effectiveness of multiple myeloma drugs.

Sanofi and Regeneron also took issue with the JAMA study, specifically saying that the research understates the likelihood of cardiovascular events in the real world and shouldn't be based on total eligible patients.  

See also: Express Scripts to cover Repatha and Praluent

In addition, Sanofi and Regeneron said there were no “formal cost effectiveness thresholds in the U.S.," noting $150,000 per QALY is a “more generally accepted baseline threshold” rather than the $100,000 per QALY used in the JAMA study. They said that 75% of patients prescribed Praluent have been denied access by U.S. health plans.

Despite the broader debate about the prices of both drugs, sales of Repatha and Praluent have fallen short of analysts' initial blockbuster estimates. Repatha saw U.S. sales of $34 million in the first six months of 2016. Praluent brought in U.S. sales of $30 million during the same period.

Analysts told Stat that they believe sales will remain low until PCSK9 inhibitors can demonstrate that they lower heart risks. Regeneron has said it plans to report interim outcomes data by the end of 2016; Amgen expects to release results from its cardiovascular outcomes trial at the beginning of 2017.