FDA approves new cholesterol drug - at $14,600 a year

Up to 10 million Americans will soon have a new option for lowering their cholesterol – at a price of $14,600 a year.

The Food and Drug Administration surprised much of the medical community Friday by broadly approving a new cholesterol drug for a vast potential patient population. The agency approved Praluent for people with an inherited condition that causes very high levels of LDL, or bad cholesterol, as well as for the millions of Americans who have had heart attacks, strokes or other types of heart disease and whose LDL is higher than it should be.

While studies show that Praluent can dramatically lower LDL, or bad cholesterol, there is no definitive evidence yet that it actually prevents heart attacks or strokes, and there is limited data on its side effects.

Praluent will cost far more than statins, the dominant cholesterol treatment today, which are available generically for about $250 a year. Statins have been shown to both lower LDL and reduce the risk of heart attacks and strokes.

Praluent, made by Sanofi and Regeneron Pharmaceuticals, belongs to a new class of injectible cholesterol drugs called PCSK9 inhibitors, the first major new type of cholesterol-lowering medication since statins were released in 1987. The FDA is scheduled to decide whether to approve a second drug in the same family, Amgen's Repatha, in the next month. Pfizer is developing a similar drug.

The drugs are predicted to be blockbusters many times over, adding billions of dollars to prescription drug costs, said Steve Miller, senior vice president and chief medical officer at Express Scripts, a leading pharmacy benefit manager. The USA already spends $300 billion on medications. Those costs can hit taxpayers, not just consumers, as Medicare, Medicaid and other public health programs struggle to keep pay for new medications.

Dan Durham, interim CEO of America's Health Insurance Plans, an industry group, called Praluent's price "exorbitant."

A growing number of people have been concerned by the high price of prescription drugs, which increased 13% last year. New cancer therapies are commonly priced at more than $100,000 a year. Unlike cancer drugs, which are taken for a short course of treatment, Praluent will be taken for the rest of a patient's life, adding to the cost.

Many cardiologists have been excited about PCSK9 inhibitors because of their ability, when combined with statins, to lower LDL to levels never before thought possible.

High levels of LDL increase the risk of heart attacks and strokes. About a third of American adults have high LDL cholesterol, according to the Centers for Disease Control and Prevention.

By themselves, statins can reduce LDL by up to 50%, cutting levels to about 70 milligrams per deciliter of blood, said cardiologist Cam Patterson, chief operating officer at New York-Presbyterian Hospital/Weill Cornell Medical Center. Adding Repatha or Praluent to a statin can cut cholesterol by a total of 70% or so, dropping LDL levels below 50 milligrams per deciliter, Patterson said.

But Patterson said he's concerned both about the lack of evidence that Praluent or similar drugs actually save lives, as well as a lack of information about the long-term side effects. While smaller studies can give doctors a glimpse of side effects, the true risks of less common but serious side effects often becomes clear only after drugs are approved, when large numbers of people are already taking them.

Definitive studies about Praluent aren't due to be completed until 2017.

"I am shocked that the FDA had opened the door this wide for a new medication," Patterson said. "The FDA has put themselves in a situation where they may have to turn around and reverse their decision."

If patients are harmed and decide to sue, Patterson said, "the FDA is not going to be on the hook. It's the doctor and hospitals and front-line caregivers who are going to be on the hook."

Many people once assumed that drugs that lower cholesterol would prevent heart attacks. Several recent clinical trials have proven this assumption wrong.

In 2006, for example, Pfizer halted development of an experimental cholesterol-lowering drug called torcetrapib after studies showed that patients who combined it with statins had higher death rates than those taking statins alone.

"As a cardiologist, I hope they (the new cholesterol drugs) put me out of business," said Harlan Krumholz, a professor at Yale Medical School. "But for some people, their enthusiasm about the potential of the drugs has blinded them to the amount of evidence that's available."

Praluent's price tag -- which is even higher than many analysts had predicted -- could have a huge impact not just for patients trying to afford their medications, but for the whole health-care system, Miller said. Insurance companies could decide to raise premiums for everyone in order to pay for them.

"This will grow to be the largest drug class in history," Miller said.

Praluent and similar drugs are costly because, like some of the newer, high-priced cancer therapies, they are man-made antibodies. Praluent targets a specific protein, called PCSK9, which reduces the number of receptors on the liver that remove LDL cholesterol from the blood. By blocking PCSK9's ability to work, more receptors are available to get rid of LDL cholesterol from the blood. That lowers LDL.

Covering the cost of the new drugs could lead insurance companies to raise premiums for everyone, Miller said.

Insurance plans are likely to limit coverage, Miller said. Instead of charging a standard copay, insurance plans could put Repatha and Praluent into the "specialty pharmacy" category, used for the most expensive drugs, such as those used to treat rheumatoid arthritis.

If PCSK9 inhibitors prevent heart attacks and strokes, they could eventually reduce some health costs, by keeping patients out of the hospital or nursing home.

So far, research published in April found that serious side effects were rare.

About 1.2% of those taking Praluent experienced neurocognitive problems, such as memory impairment or confusion, compared to 0.5% of those taking statins. About 5.4% of those taking Praluent had muscle pain, compared to 2.9% of those given statins. About 2.9% of those taking Praluent had eye problems, compared to 1.9% of those taking statins, according to a study in The New England Journal of Medicine.