Leonard S. Schleifer, president and chief executive officer of Regeneron Pharmaceuticals Inc., listens during the Bloomberg "Focus on Pharma" event in New York, U.S., on Wednesday, June 29, 2016. Schleifer discussed the need for the pharmaceutical industry to do a better job explaining the value proposition of products they bring out and the need to price drugs at a more reasonable rate. Photographer: Eric Thayer/Bloomberg Leonard Schleifer (Photo: Eric Thayer/BB)

Regeneron Pharmaceuticals Inc. and Sanofi are taking the unprecedented step of cutting the price of their $14,000-a-year heart drug Praluent and narrowing its use to high-risk patients shown to benefit most in an effort to get insurers to ease restrictions on its use.

A highly anticipated study of the injected drug found it reduces complications including heart attacks and strokes by 15 percent, less than the 20% benefit analysts say would be needed to force insurers to loosen their stranglehold.

The companies honed in on a smaller group of patients who responded even better, and said they would cut the cost for that group in exchange for allowing them better access.

(Related: `Rigged’ System Blocks Lower-Cost Drugs, Head of FDA Says)

The new price will be in line with a recommendation from the Institute for Clinical & Economic Review, which found Praluent would offer good value at a cost of $4,500 to $8,000 a year in high-risk patients. The study, presented Saturday at the American College of Cardiology meeting in Orlando, Florida, found those patients got a 24% benefit, including a 29% reduction in death.

Opening Move

“Today, we have made the opening move and we challenge payers to respond by making Praluent readily accessible to high-risk patients,” said Regeneron Chief Executive Officer Leonard Schleifer, in a conference call with analysts.

The 19,000 patient study yielded similar disappointing results to one generated a year ago by Amgen Inc.’s rival treatment, Repatha. Both drugs are in a class of medicines known as PCSK9 inhibitors that were once hoped to slash heart complications as dramatically as they help lower cholesterol. But last year’s findings on Repatha sank Amgen’s stock and failed to spark sales, even after the company offered a money-back guarantee.

The new study included people vulnerable to heart problems after they’d survived a heart attack or severe chest pain caused by reduced blood flow to the organ within the previous year. Praluent slashed bad cholesterol by more than half after four years. Heart attacks, strokes, hospitalizations and deaths from heart disease occurred in 9.5% of Praluent patients, compared with 11.1% of placebo patients.

The companies and the researchers who oversaw the trial focused on one subset of the patients, accounting for about 30% of the group, whose bad “LDL” cholesterol levels remained stubbornly high despite aggressive treatment to lower it. Those patients responded best. Their results:

Complications in the highest-risk group Praluent Patients Placebo Patients Heart attack, stroke, hospitalization, death 11.5% 14.9% Death from any cause 4.1% 5.7%

“The majority of our benefit was accounted for by these people who had the highest LDL and the highest risk,” said George Yancopoulos, chief scientific officer at Tarrytown, New York-based Regeneron. “We have to get the whole world to realize this is cost-effective for society and we have to get this drug to the patients who will benefit the most.”

The findings may sway insurance companies and pharmacy benefit managers, which had urged the drugmakers to identify patients most likely to benefit. Some clinical trial purists, however, may say the mortality results aren’t definitive because the companies decided to analyze those findings after the study was complete, rather than planning it in advance.

Reduce Death

The results show for the first time that a PCSK9 inhibitor can reduce death, which is what everyone in the field was hoping to see, said Deepak Bhatt, executive director of interventional cardiovascular programs at Brigham & Women’s Hospital in Boston and one of the trial’s researchers. While there’s a benefit for all patients, the investigators honed in on the highest-risk group because of the chance to make a difference for patients, he said.

“If it was pennies a day, I would say use it in everybody,” Bhatt said. “But here it’s really an issue of cost. In terms of trying to be judicious with health care resources, it seems like this is the population to target.”

The findings, while “a true win,” aren’t going to change prescribing practices and boost access overnight, said Mary Norine Walsh, president of the American College of Cardiology. Use of the medicine depends heavily on insurance coverage, she said.

‘Budget Impact’

ICER said it plans to evaluate the impact of the drug in a later report, but that given the number of potential patients, which it estimated at 300,000 to 400,000 a year, “the budget impact may be substantial.”

Yancopoulos said the changes will result in a trifecta for patients: data that proves Praluent’s benefit, a lower price that makes it undeniably cost effective, and a requirement that payers break the gridlock and allow its use.

In the high-risk group, Praluent would keep alive one of every three patients who otherwise would have died, said Elias Zerhouni, president of research and development at Paris-based Sanofi.

“We save people’s lives from day one,” he said.

—With assistance from Kenneth Pringle and Ros Krasny.

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