SOURCES: Thomas Whayne, M.D., Ph.D., director, Lipid Management Clinic, University of Kentucky, Gill Heart Institute, Lexington; Jack Hoadley, Ph.D., research professor, Georgetown University Health Policy Institute, Washington, D.C.; Brent Reed, PharmD., assistant professor, pharmacy practice and science, University of Maryland School of Pharmacy, Baltimore
THURSDAY, June 18, 2015 (HealthDay News) -- A new class of powerful cholesterol drugs is poised to hit the market, and doctors are both hopeful about their potential, and worried that insurers won't pay for them.
The drugs, known as PCSK9 inhibitors, can drastically cut LDL cholesterol -- the "bad" kind linked to increased risks of heart attack and stroke. And they are expected to open up a new option for people who cannot take statins, the drugs that have been the standard for cholesterol-lowering since the 1980s.
Last week, an advisory panel to the U.S. Food and Drug Administration recommended the agency approve two PCSK9 inhibitors: alirocumab (Praluent) and evolocumab (Repatha).
The FDA, which usually follows the recommendations of its advisory panels, is expected to OK both drugs.
Some cardiologists have heralded PCSK9 inhibitors as a breakthrough -- particularly for patients who can't take statins because of side effects, such as intolerable muscle pain, and for those whose LDL does not decline enough with statins or other current drugs.
"I think this is fantastic news for those patients," said Dr. Thomas Whayne, director of the Lipid Management Clinic at the University of Kentucky's Gill Heart Institute.
The downside, as Whayne sees it, is that insurers may not be willing to pay in all cases. "I think we'll have some tremendous battles with pharmacy benefit managers," he said.
Why? Because PCSK9 inhibitors are complex, injectable drugs called monoclonal antibodies, which are expensive to produce. And they are expected to be priced accordingly -- running up to $12,000 a year, according to a recent estimate from CVS Health, one of the nation's largest pharmacy benefit managers.
By comparison, many statins are available as generics, and can cost as little as a few dollars a month, according to Consumer Reports.
CVS Health warned that PCSK9 inhibitors could put a "great cost" on the health-care system. As many as 15 million Americans could be candidates for the drugs, the company said -- and those people would be taking the medications for years, if not decades.
It's likely that some patients will have difficulty getting insurance coverage, agreed Jack Hoadley, a research professor at Georgetown University's Health Policy Institute, in Washington, D.C.
"In these situations, the insurance companies can end up looking like the bad guy if they put up obstacles," Hoadley said.
On the other hand, he added, there are legitimate reasons for payers to balk: The new drugs do slash LDL levels -- by as much as 60 percent -- but they haven't been studied long enough to know whether they actually prevent heart attacks and strokes.
"What we don't have yet is evidence that these drugs save lives," Hoadley said.
Plus, it's not entirely clear which people with high cholesterol would fare better with a PCSK9 inhibitor rather than a statin or other standard medication.
"There will be some ambiguity about which patients are the right candidates," Hoadley said.
And that will be a "real challenge" when the medications hit the market, said Brent Reed, an assistant professor of pharmacy practice and science at the University of Maryland, in Baltimore.
"I think the first patients to receive this drug will be those with familial hypercholesterolemia," Reed said, referring to a genetic condition that causes very high LDL levels that often resist statin treatment.
Beyond that group, though, things get murkier.
People who are "statin-intolerant," because of side effects such as muscle pain, seem like obvious candidates. But, Reed said, statin intolerance is not simply defined: Studies show that people who have, or perceive, statin side effects often do much better if they try again -- with a different statin or a different drug dose.
Reed said he wouldn't be surprised if insurers required proof of true statin intolerance before they would approve a PCSK9 inhibitor.
Whayne pointed to another group that could benefit from the new drugs: People at high risk of heart attack or stroke -- because of multiple risk factors, like diabetes and high blood pressure -- whose LDL levels do not respond adequately to statins.
But again, that's a gray area. If a doctor wants to further reduce a patient's LDL, an insurer can question the necessity. The latest guidelines from the American College of Cardiology and the American Heart Association say it's the statin treatment that is important, but downplay the need for getting LDL to a "target" number.
Whayne said he can foresee doctors "battling" insurers in such cases.
Still, Whayne also stressed that doctors will have to be selective about PCSK9 drugs. "This should not be a casual prescription," he said.
For his part, Reed said the unknowns about PCSK9 inhibitors -- not only their long-term effectiveness, but their safety -- will make him cautious. "Until I see compelling evidence that they improve patients' outcomes, I can't really see choosing them over a statin in most cases," he said.
Hoadley noted: "The FDA will probably approve them, but the FDA process does not look at whether these drugs are any better than the alternatives." That, he said, will require studies that compare PCSK9 inhibitors with statins.
The U.S. National Heart, Lung, and Blood Institute has advice on cutting LDL cholesterol.