Regeneron Pharmaceuticals and partner Sanofi report new data this morning from a large, highly-anticipated trial suggesting that their drug alirocumab (Praluent) may reduce the risk of death for people with high cholesterol who have recently suffered heart attacks or strokes and are at risk of having another one or dying.
And in an unusual move, Regeneron—based on input from the nonprofit drug-pricing watchdog, the Institute for Clinical and Economic Review (ICER)—vows to cut the cost of the drug if it can get certain concessions from insurers. Regeneron and Amgen, which makes a competing drug, have struggled mightily to get payers to cover these treatments because of their high cost.
ICER often comes up with recommended prices that are far below those set by drug makers. Rarely do companies respond by actually lowering prices the way Regeneron promises to do. The price cut is also notable because the latest clinical data from Regeneron, released today at the American College of Cardiology’s yearly meeting, shows that alirocumab not only lowers the risk of heart attacks like Amgen’s does, but might also lower the chance of death as well—something the rival drug hasn’t been shown to do. Doctors say this finding, though not statistically clear-cut (more on that below), could still drive greater adoption of the expensive cholesterol drug.
The news comes as the high price of prescription drugs remains a big political flashpoint; both new U.S. Health and Human Services Secretary Alex Azar and FDA Commissioner Scott Gottlieb amped up the pressure on drug companies, hospitals, and insurers this week to change payment schemes and increase drug price transparency to help drive down healthcare costs.
Regeneron’s (NASDAQ: [[ticker:REGN]]) alirocumab and Amgen’s (NASDAQ: [[ticker:AMGN]]) evolocumab (Repatha), both approved in 2015, belong to a new class of cholesterol lowering medicines called PCSK9 inhibitors, which has been a key battleground between payers and drugmakers. Despite their striking ability to lower gobs of LDL-C or “bad” cholesterol, their sales have been meager compared to Wall Street analysts’ initial bullish multi-billion dollar projections; alirocumab tallied $194 million last year worldwide, evolocumab $319 million. Insurers often balk at their high list prices, some $14,000 apiece per patient, per year.
Problems with patient access have been documented in numerous studies. An Amgen-backed study released yesterday, for instance, found 65 percent of commercially insured and Medicare patients trying to get a PCSK9 blocker were denied access by their health plans in 2016. Cardiologists interviewed by Xconomy say the drugs are almost always rejected upon first request. Months can go by before a patient gets them.
“I don’t have a single patient on a PCSK9 inhibitor,” says Roxana Mehran, director of interventional cardiovascular research and clinical trials at the Icahn School of Medicine at Mount Sinai. “It’s so difficult, I give up.”
Regeneron launched its multi-year, 18,924-patient study, “Odyssey Outcomes,” in 2012 to try to show that alirocumab, when used along with standard treatment with other heart drugs known as statins, can improve patient outcomes, not just cut cholesterol. The study is the second of its kind—following a similar, 27,564-patient trial from Amgen last year. Both studies have been seen as critical to proving PCSK9 blockers are worth their high price tags.
Despite the widespread use of statins, heart disease remains the leading cause of death, according to the Centers for Disease Control and Prevention. Regeneron and Amgen have been hoping to make PCSK9 blockers available to more and more of the millions of patients who can’t reduce their dangerous cholesterol levels with statins, or can’t tolerate them because of side effects. (PCSK9 inhibitors block a protein that interferes with the body’s clearance of bad cholesterol from the blood and are administered, via injection, once or twice a month.)
Before releasing its Odyssey data, Regeneron shared them with ICER so that it could compile an independent review. ICER today says alirocumab is worth between $4,500 and $8,000 per year for the specific group of “high-risk” patients who benefited the most from treatment in Regeneron’s trial—and $2,300 to $3,400 per year for everyone else. The high-risk group is the estimated 1.3 million people in the U.S. and Europe who have had heart attacks or strokes and still can’t get their LDL-C levels below 100 mg/dL of blood despite treatment with statins. Regeneron will now focus its commercial efforts on those patients, and plans to reach out to payers with a deal: provide “straightforward access for high-risk patients,” and it will lower the price of alirocumab, according to a press release.
Regeneron spokesperson Hala Mirza says the size of the price cuts will depend on
