Seth Martin’s advanced lipids clinic at Johns Hopkins Medicine treats some of the sickest heart-disease patients around. But two doctors, two nurses and another staffer there are devoting much of their time to fighting with insurance companies over two advanced cholesterol drugs.
Nearly 90 percent of the patients Hopkins wants to put on expensive, powerful PCSK9 inhibitors are initially rejected, making heart disease perhaps the central stage in the battle over high U.S. drug prices. In the least, it’s the drug-pricing battle that may matter to the most Americans: Heart disease is the No. 1 cause of death in the country.
Two years after PCSK9 inhibitors from Amgen and Sanofi-Regeneron hit the market, patients are caught in the middle. The injectable drugs — which block a liver protein that prevents the organ from breaking down cholesterol — reduce bad cholesterol as much as 60 percent when combined with statins that have dominated cholesterol-reduction treatment for years. And they work for patients who get poor results from statins or can’t take them because of side effects, Martin said.
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Doctors largely agree that most people with cholesterol high enough to need treatment should stick with statins. Higher-risk patients should ask about newer treatments.
He added, “Most patients are rejected even when they clearly meet the criteria. There’s a process in place to make this as challenging as possible.”
That’s because the drugs also cost many times what statins do. Generic versions of familiar drugs, like Pfizer’s Lipitor, can be had for as little as $10 a month, compared to about $1,200 for Amgen’s Repatha or Sanofi’s Praluent. Statins are so cost-effective that some insurance carriers even waive co-payments to make sure patients take them.
That means the fight is over which patients should get which drugs, a debate patients join when making decisions for themselves. Two studies published in the last month will fuel even more arguments; they reached starkly different conclusions about whether Repatha and Praluent are worth the money. The latest results come against a backdrop of weak sales for the drugs. Amgen said insurers have approved just 44,000 U.S. patients for Repatha.
“It was developed for patients who are on the very best therapy but haven’t been able to drive their bad cholesterol lower,” said Joshua Ofman, senior vice president for global value, access and policy at Amgen. “Some of the controversy is because people don’t understand that this drug is intended to be used by patients who are already on a statin.”
Most people with high cholesterol do fine with statin treatment, Ofman said. Those drugs, whose sales peaked at nearly $40 billion but have been battered by generic competition since 2012, are effective in most patients and have few side effects in about 90 percent of cases. In the rest, the most common side effect is muscle pain, which can be severe enough to make patients seek alternatives.
“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.”
-Andrew Beamer, chair of cardiology at Berkeley Heights, N.J.-based Summit Medical Group
The patients who need more are tougher cases, many suffering from a genetic condition called familial hypercholesterolemia, or FH, which affects 1.3 million Americans. Patients who have the FH gene can develop heart disease as early as childhood, and many can’t get their cholesterol down to normal levels even with statins and a rigorous diet. Patients who have previously had a heart attack or stroke or are at risk of limb amputations because of poor circulation are also prime candidates for the more expensive drugs.
“This is absolutely an adjunctive therapy for statins, not a replacement,” Martin said. “We don’t just skip the statins and go straight to the [expensive] stuff.”
The problem has been convincing payers that the new drugs are worth the money. Insurers initially turned down as many as 80 percent of patients who were prescribed Amgen’s Repatha, Ofman said, though the numbers are improving slightly after a clinical study released in New England Journal of Medicine in March showed the drug did reduce hospitalizations for heart attacks and strokes but did not reduce mortality from heart disease. The most recent data show 71 percent of prescriptions are initially refused — with no clinical differences among the cases approved and rejected, Ofman said.
“Physicians have to fight very hard to get the drug even for the most high-risk patients,” he said. “But doctors say most patients who are clear candidates for Repatha or Praluent can get their prescriptions approved eventually, if they appeal the insurance denial with their doctor’s assistance.”