The Food and Drug Administration this week is expected to approve the first in a new class of cholesterol-lowering medications that could represent the most significant advance in cardiology since statin drugs hit the market decades ago.
But, in part because the new treatments have yet to demonstrate that they reduce the incidence of strokes and heart attacks, their approval appears set to trigger the latest fight over the high price of many drugs in the United States.
Experts have estimated that the new medications could cost $7,000 to $12,000 a year. That’s far less than expensive specialty drugs for cystic fibrosis, certain cancers and hepatitis C, but it is far more expensive than many existing statin drugs, which now come in cheap generic forms. The new medications are known as PCSK9 inhibitors because they block a substance that hinders the liver’s ability to remove bad, or LDL, cholesterol from the blood.
Should the FDA approve the new medications, it probably would do so for only some patients initially — for instance, those who can’t get their cholesterol levels low enough with statins or those with an inherited disorder that can severely elevate LDL levels. But some health-care providers and insurers already are fretting about the collective cost of the drugs should they eventually be used to treat millions of Americans with elevated cholesterol levels.
“This sets up an epic battle between insurers and physicians and patients,” said Steven Nissen, chairman of cardiovascular medicine at the Cleveland Clinic, who is involved in studies testing two of the new treatments under development. “It’s an interesting dilemma.”
Nissen said he thinks the FDA would be justified in approving the drugs, even for limited populations, because they have shown a remarkable ability to reduce bad cholesterol levels in clinical trials and are likely to help reduce cardiovascular events over the long term. That said, he acknowledged that such a scenario could create conflict between doctors who might want to prescribe the drugs for a broader swath of patients and payers who might not be willing to approve such “off label” uses without more data.
“It does set up a problem,” Nissen said. “How that gets adjudicated will be very important for individual patients but also for society in general.”
Last month, an FDA advisory committee voted in favor of approving two injectable PCSK9 inhibitors for use in certain patient populations. The agency does not have to follow the advice of its advisory committees, but it often does.
The first of those drugs, produced by Sanofi and Regeneron, could be approved by the FDA this week. The second, developed by Amgen, won approval Tuesday in Europe and could receive approval for use in the United States this summer. Drug giant Pfizer also has a PCSK9 inhibitor under development.
The research firm Global Data has projected that the entire class of PCSK9 drugs could generate sales of $17.8 billion by 2023.
Advocates say the new drugs fill a need for some of the estimated 73 million Americans who suffer from high LDL cholesterol. Even with such well-known statin drugs as Lipitor, some patients still can’t get their cholesterol to desirable levels. Some do not tolerate the drugs well. Others suffer from hypercholesterolemia, an inherited disorder that can severely elevate LDL levels.
In clinical trials, PCSK9 inhibitors have shown the ability to reduce bad cholesterol levels by about 60 percent, even for some people already taking statins. But there are no definitive answers yet about whether that will translate into fewer heart attacks, strokes and other heart problems.
“It presents a challenge for us,” said Troyen Brennan, chief medical officer for CVS Caremark, one of the country’s largest managers of prescription drug benefits. “Although there’s good information that these medications lower cholesterol, we don’t have good data on patient outcomes. . . . We basically think the statin medications are going to be totally appropriate for the overwhelming majority of patients.”
Brennan said if the FDA approves the new drugs, CVS probably will require proof that patients prescribed PCSK9 inhibitors cannot tolerate statins or already have tried them without success.
“Employers and insurers pay us to make wise decisions about what to cover,” Brennan said. “We’re trying to be responsible members of the health-care community. We don’t have limitless resources.”
Express Scripts, another prominent pharmacy benefit manager, also plans to closely monitor the use of PCSK9 inhibitors. Steve Miller, the company’s chief medical officer, said that although select patients might take the drugs initially, the potential costs are enormous if millions of Americans eventually end up on the medications.
“It has the potential to be the biggest category in the history of pharmaceuticals,” Miller said.
He said Express Scripts has had far more “collaborative discussions” with the companies developing PCSK9 drugs than it did with the firms that developed drugs to treat hepatitis C. Express Scripts vehemently opposed the $84,000 price tag of Gilead Sciences’ Sovaldi treatment and decided last year to exclusively offer a competing drug in exchange for discounts on the price from the manufacturer.
Miller said the company could take a similar approach with makers of the new PCSK9 drugs. “We have made it very clear to them that we’re willing to go exclusive with one drug or the other, if they don’t competitively price these drugs,” he said.
The companies behind the new drugs have not yet disclosed what they will cost. But they argue that if the drugs work as intended, they could save money over the long term.
In a statement, Amgen noted that despite the huge annual spending on cardiovascular disease in the United States, many patients can’t keep their LDL levels in check. Providing new drugs to help accomplish that goal ultimately could be “part of the solution to rising healthcare costs,” the company said.