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Powerful new class of cholesterol drugs offers hope for some patients

More than 30 million Americans take statins to lower their cholesterol, according to estimates. But these popular drugs don't work for everyone. Now the FDA may be poised to approve a powerful new class of drugs that can attack cholesterol levels in a different way. Hari Sreenivasan talks to Dr. Steven Nissen of the Cleveland Clinic and Dr. Harlan Krumholz of the Yale School of Medicine.

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    It's estimated that more than 30 million Americans are taking medications like Lipitor and Zocor to lower their overall cholesterol levels.

    But, as popular as these drugs are, they don't work for everyone. Many cannot tolerate them easily, and, for some people, they simply don't lower their cholesterol significantly.

    Now the FDA appears poised to approve a powerful new class of drugs that could be used with the statins to attack cholesterol levels in a more targeted way. It amounts to a new method for cutting down LDL, often referred to as bad cholesterol.

    An FDA advisory committee has backed the approval of two new drugs in this class for some groups of people.

    Hari Sreenivasan picks up the story from there.


    The FDA will decide later this summer whether to follow the panel's recommendations. But it usually does so. And the drugs, which are injected once or twice a month, have been shown to be effective at lowering bad cholesterol in early trials.

    Still, larger trials need to be completed to see if they prevent heart attacks and strokes. And then there's the question of how many people may ultimately take them. More than 70 million Americans have high LDL. Initially, they may cost around $10,000 a year per patient.

    We talk to two cardiologists about these questions.

    Dr. Stephen Nissen is the chair of cardiology at the Cleveland Clinic. For the record, he is leading a study of one of the drugs and is on the steering committee for a Pfizer clinical trial. He takes no money from the companies. And Dr. Harlan Krumholz is professor of cardiovascular medicine at the Yale School of Medicine.

    So, Dr. Nissen, I want to ask, why is this class of drugs so interesting to you? What does this do that the medications that are on the shelf today don't do?

    DR. STEPHEN NISSEN, Chairman, Department of Cardiology, Cleveland Clinic: Well, LDL cholesterol is the primary driver of coronary heart disease, the most important risk factor that we can modify.

    And we have great drugs called statin drugs, drugs like Zocor and Lipitor and so on, that are very effective at lowering LDL cholesterol and preventing heart attack, stroke and death.

    But not everybody gets an adequate response to those drugs. Some people are genetically programmed to have very high levels of bad cholesterol, LDL, and they just don't get well enough with those drugs. And there are other people that are intolerant of the drugs.

    So we have needed another class of drugs. These drugs are really powerful. They lower LDL anywhere from 50 to 70 percent. And that's additive on top of the effects of the statin drugs. So they're very exciting, very powerful.


    So, without getting too far into the weeds, in lay terms, how do they work? How are they different?


    They increase the activity of the receptors in the liver that are responsible for removing LDL cholesterol.

    When you interfere with a protein that's involved in degrading those receptors, the receptors become more active, and they're removing a lot of cholesterol from the bloodstream. And, of course, it's cholesterol in the bloodstream that ends up as plaques in the coronary arteries that lead to heart attacks and all the consequences.


    Dr. Krumholz, I mentioned that the FDA has started to approve this. But it almost seems like this drug is on a fast track. You have some concerns about that?

    DR. HARLAN KRUMHOLZ, Professor, Section of Cardiovascular Medicine, Yale School of Medicine: Well, let me say, I think these are remarkable drugs and they're a remarkable story.

    There were some observations of families that led to molecular biology studies, which have very quickly led to these drugs. And I think there is a lot of excitement about them. But we have to remember that the verdict is not in yet.

    So it is true that bad cholesterol, this LDL cholesterol, contributes to heart disease. And it would seem in theory that, if we could lower it to very low levels, that we will benefit patients. But these drugs and many other drugs, they have a lot of different effects. And what we really need to understand is, at the end of the day, if people take these drugs, can they expect that they will live longer or better?

    And just knowing what it does to the cholesterol is a hint. It's an assurance that that's likely, that it's possible. And there is other information about these drugs that make us hopeful that that's true, but there are a lot of studies in the field now that are going to be counting events, determining whether people really benefit from these drugs.

    And until those studies are completed, we won't know for sure. So, we want to temper our enthusiasm a little bit. These drugs may be reserved for the people who need them most. We need to be careful about not overreaching with the information we have so far.


    So, Dr. Nissen, by the time these trials that Dr. Krumholz was talking about finish up, how long until a drug like this from this class gets to market?


    Well, the FDA is going to make a decision this summer, and most of us anticipate that they will follow the advice of the advisory panel and approve the drugs perhaps not for a broad population, but for a limited population.

    The FDA is acting carefully. They know that the clinical trials that are going to measure the outcomes we care about, like heart attack and stroke and death, are ongoing. At least one of these trials with 27,000 patients is actually completely enrolled and should report in 2017.

    So, what they're doing is providing early access to the drugs for those people that have the most need, while waiting to give a broad label for the drugs until we have the kind of outcome studies that Dr. Krumholz wants and I want to. We really do need the outcome trials, but it's about balancing what's in the public interest.

    And I agree with the FDA. I think it's in the public interest to get the drugs to market now for the people that are most in need.


    Dr. Krumholz, how much are some of the drugs going to cost? Is it the standard curve, where the first ones cost a lot and, once you make a billion of them, they get cheaper?


    I think it's hard for me to determine on what basis we're pricing drugs now. If you look at many of the cancer drugs, if you look at the hep C drugs, they're really coming out at extraordinarily high cost.

    And it's hard to know exactly where this will be priced. I think it's impossible to know about the value that the drugs are bringing until we have these other studies completed and we know what effect the drugs have on people's lives.

    So I'm in agreement with Dr. Nissen. I think it's important to give early access, if possible, to people who have little choice or for whom cholesterol represents their biggest risk and for whom they're not really responding to or can't tolerate other options. But we need to be careful.

    And what's important here is, we don't know the value at all until we know what benefit it's providing. Once we know that, we can put in perspective what the costs are. But these could break the bank, because there are so many people at risk for heart disease. We're going to need to be thoughtful about the way in which these drugs are used and we're going to need to be careful in thinking about, exactly what is the size benefit for what we're paying for?


    All right, Harlan Krumholz and Stephen Nissen, Doctors, thank you so much for joining us.


    Thank you.


    Thank you, Hari.

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