Payers say they will pay for PCSK9 inhibitors, but only for patients who really need them. CVS Caremark — one of the two giant pharmacy-benefit managers along with Express Scripts, who help set the tone for other U.S. payers — didn't respond to requests for comment. It has warned that drugs like Repatha could cost the U.S. health-care system $200 billion.
"We've worked to make sure that the right patients get access to a drug they need," Express Scripts spokesman Brian Henry said in an email. "When the PCSK9 drugs first came to the market, we saw a lot of prescribing for patients who were not indicated for the drug. With additional education, that has lessened."
But even in more conventional doctors' offices than Hopkins' clinic, the new drugs are hard to get, according to the heads of cardiology at the two largest non-hospital-based medical practices in metro New York.
"It's very hard to get people on the drugs, period,'' said Andrew Beamer, chair of cardiology at Berkeley Heights, N.J.-based Summit Medical Group. "There is a risk in the prior authorization not only when you prescribe but also when you renew. They've done such a good job of making it expensive to ask that I don't even try to put people on. It's like self-censorship.''
Most patients will respond to statins, either alone or in tandem with Merck's Zetia, which goes for about $160 a month before discounts, Beamer said. But his practice has about seven patients on PCSK9 inhibitors. At 550-doctor CareMount Medical in Mount Kisco, N.Y., about three patients take the drugs, said director of cardiology Richard Keating.
"They're not the most common drugs we use," Keating said. "But they work very well, and the side-effect profile is quite benign.''
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The real dispute is over whether the PCSK9 inhibitors deliver enough bang to be worth the bucks. That was the subject of two competing studies that hit medical journals in August.
PCSK9 inhibitors reduce cholesterol but haven't been shown to reduce mortality, said Dhruv Kazi, associate professor at University of California San Francisco's medical school. Using estimates of how many deaths might be avoided, as well as an assumption that every extra year lived with a good quality of life is worth $100,000, he argues that the drugs are only worth about $4,200, based on findings published last month. Without assuming any improvement in mortality, he argues that the drugs are worth about $2,000 per year.
The lifetime bill for these drugs could be higher for many patients than even the one-time, $95,000 Hepatitis C treatments that have galvanized debate over drug prices. "Reducing the price of PCSK9 inhibitors remains the best approach to delivering the potential health benefits of PCSK9 inhibitors therapy at an acceptable cost," Kazi and colleagues wrote.
The other study, supported by Amgen, estimated the value of Repatha at $9,669 per year — less than its list price but consistent with prices paid after common discounts. To get there, researchers valued each quality-adjusted life year at $150,000 and assumed a higher rate of avoided heart attacks than in Kazi's study.
"That drives the majority of the difference," Ofman said.
What should patients make of this?
Most people with cholesterol high enough to need treatment should stick with statins, doctors largely agree. Higher-risk patients should ask about newer treatments. For now.
The wild card is whether cholesterol guidelines might move lower in the future, based on emerging research that indicates the inhibitors like Repatha can lower the buildup of cholesterol plaques in the coronary artery walls to levels not possible with statins alone, Beamer said. Price cuts for PCSK9 inhibitors and falling tolerance for cholesterol might be the keys to a big market in the future, Beamer argued.
"If the guidelines change, price cuts might expand the market," he said.
— By Tim Mullaney, special to CNBC