A pricey new drug designed to slash harmful cholesterol levels in heart patients has been shown to reduce the risk of a heart attack or stroke by 20 percent. It’s an important breakthrough, but a big question remains: Is the benefit big enough to warrant the drug’s $14,000 price tag?
The results of a two-year study of the drug, called Repatha (evolocumab), were announced at a meeting of the American College of Cardiology and published in the New England Journal of Medicine. The study, which involved more than 27,000 men and women, cost about $1 billion and was paid for by Amgen, the maker of the drug, the New York Times reported.
The drug was designed for people already taking the maximum dose of cholesterol-lowering statins but who had been unable to lower their cholesterol level sufficiently to ward off heart disease, in some cases because of an inherited genetic condition. Study participants were ages 40 to 85 and already taking statins; most already had suffered a heart attack or stroke.
The results of the trial were highly anticipated. Repatha is part of class of cholesterol drugs targeting the PCSK9 protein to control cholesterol and heart-disease risk. Inhibiting the protein can cause the level of LDL, or “bad” cholesterol, to tumble dramatically to unheard-of levels — from 92 milligrams per deciliter to 30 milligrams.
In the study, 5.9 percent of patients who took both Repatha and a statin suffered a heart attack or stroke or died, compared with 7.4 percent of patients who took a statin and a placebo — a 20 percent reduction.
But while an accompanying editorial in the journal called it a “landmark study,” others expected a greater reduction in risk for its cost.
“It’s a small reduction for a super-expensive drug,” cardiologist John Mandrola with Baptist Health in Louisville, Ky., told USA Today.
Yale University cardiologist Harlan Krumholz, writing about the study for NPR, said the trial represents “good, tangible evidence that the drug can reduce risk,” but added that the size of the benefit is about the same as is achieved with statins. “No new records on risk reduction were set.”
However, for people at high-risk for heart attack or stroke who are unable to lower their cholesterol with traditional statins alone or who cannot tolerate statins, the drug offers physicians a new tool to help them.
In a prepared statement about the study’s results, drugmaker Amgen called it a “game-changer for high-risk patients” for whom nothing else has worked. The company estimates there are about 11 million Americans who fall into this category. Amgen also noted that despite worries about the side effects of dropping LDL levels so low, there were no apparent safety concerns during the course of the study.
To help insurers who might be balking at covering the drug’s $14,000 price, Amgen also added this sweetener: It will refund the cost to the insurer if the patient has a heart attack or stroke while taking the drug.