The emergence of two medications that lower cholesterol through a novel mechanism is stirring the medical community with excitement even as medical policy experts highlight the drugs' stratospheric costs.
Long Island doctors are citing the unique nature of the medications and have reported varying degrees of success when patients attempt to fill prescriptions. The medications, which are injected, need to be taken only once or twice a month.
Praluent and Repatha fall into the class of medications known as "biologics," and use an engineered molecule to home in on a protein in the liver called PCSK9 to inhibit its activity. The inhibition lowers LDL -- low-density lipoprotein -- the so-called bad form of cholesterol.
The drugs mimic biological processes observed in people who have naturally low LDL. Praluent was approved in July; Repatha last week.
But prices on the new medications dramatically overshadow those of statin drugs, the old medicine cabinet standbys, which cost only pennies per dose. Each of the new drugs runs more than $14,000 a year.
10 million qualifyOverall, 73 million people nationwide are on cholesterol-lowering medication and at least 10 million qualify for the new drugs, medical policy experts say.
Dr. Thomas Pappas, director of the cardiac catheterization laboratory at St. Francis Hospital in Flower Hill, has prescribed Praluent to one patient so far, but the patient's insurer has yet to cover the medication's cost.
"The roadblock is ongoing and we are trying to get it authorized," Pappas said. "I think this is happening because the drug is so new and different from the others we use on a routine basis."
He added, "I think it's encouraging that there is a second drug out there because competition usually helps drive prices down."
Competition, however, may not force a price decline, medical policy experts say. For example, two leading and competing forms of engineered insulin -- Levemir and Lantus -- have been rising in price in tandem over the past five years, cost analyses show.
Each of those drugs has risen 189 percent since 2010 from $86 per month to $249 every 30 days, according to the Alliance of Community Health Plans, a national organization representing health plans and medical providers. Identical hikes in medication costs is called shadow pricing, experts say.
Despite high price tags on the new cholesterol medications, some doctors have had no problem prescribing them.
Dr. Kevin Marzo, chief of cardiology at Winthrop-University Hospital in Mineola, said some of his patients have entered a new frontier of cholesterol lowering. And his patients' insurers didn't balk at the cost.
He said the prescriptions were written for patients with familial hypercholesterolemia, a condition in which LDL is astoundingly high and uncontrolled by statins. These were the patients for whom the new drugs were developed, he said.
Insurance coverage varies"I have several patients who have familial hypercholesterolemia and I prescribed the medication [Praluent] because I had trouble getting their LDLs under 200 with statins." Less than 100 milligrams of LDL cholesterol per deciliter of blood is considered optimal; greater than 189 is considered very high, according to the National Heart, Lung, and Blood Institute.
"For my patients, it was effortless," Marzo said of obtaining coverage. "But again, these are patients who have high cholesterol that runs in their families."
At Franklin Hospital in Valley Stream, Dr. David Friedman said he has yet to prescribe either medication.
"The approval of these drugs came with some pretty strict guidelines from the FDA. The challenge will be whether these are the right drugs for patients who don't fit the guidelines."
Friedman, chief of the heart failure service at Franklin, a division of the North Shore-Long Island Jewish Health System, said some doctors may prescribe the medications "off-label." That refers to prescribing the drugs to patients who don't meet the guidelines under which the medications were approved.
Clare Krusing, spokeswoman for the health insurance industry's trade organization, America's Health Insurance Plans, said it's up to individual insurers to decide whether they will cover the medications. She said insurers consider a variety of factors when determining coverage.
Key factors are whether a medication is being prescribed according to treatment guidelines or if it is being prescribed off-label, Krusing said.
Medical policy experts, however, worry about the continuing upward climb of prescription medications, especially those that are biologically engineered.
The Pew Foundation has written a position paper on the new cholesterol medications and noted that they belong to an overall class of drugs -- biologics -- that are prescribed to fewer than 2 percent of all patients. But the drugs as a group are projected to account for more than half of all medication expenditures by 2020, according to the report.
Praluent is a product of Regeneron Pharmaceuticals in Westchester County and French drugmaker Sanofi. Repatha is made by Amgen in California.
Pappas said the drugs reduce cholesterol through a revolutionary new mechanism, but clinical trials have yet to prove whether they have broader application, despite scores of patients who say they cannot tolerate statins.
Statins have been associated with muscle pain and weakness in some patients. Others have complained of blurred vision and memory loss. An estimated 73 million people in the United States are on cholesterol-lowering medications, according to the Pew Foundation.