Why the New Statin Guidelines will Endure

This week the discussions about the new cholesterol guidelines from the American College of Cardiology and the American Heart Association took a turn for the worse. I wonder if the rancorous public debate over the risk calculator will divert attention from some truly remarkable features.

The principles articulated by these guidelines are worthy of notice – and will endure far past this debate about how to calculate risk.

So what are the principles?

1) Do not focus on the cholesterol levels as a drug therapy goal. The goal of drug treatment in prevention, after lifestyle interventions have been exhausted, is to lower risk. That sounds simple but for a long time we thought that the goal was to reduce your bad cholesterol and raise your good cholesterol. We assumed that this change was equivalent to lowering your risk.

The medical profession, industry, and government embraced the idea that lowering the bad cholesterol was the same as lowering your risk of heart attack and stroke. Physicians were being assessed by their success in lowering their patients’ cholesterol levels – and sometimes their pay was tied to it. Board questions tested our knowledge of the levels that our patients should reach. Advertisements and studies, many sponsored by industry, promoted how different drug combinations could improve our ability to help our patients reach target. Patients were urged to know their numbers – and public service announcements and direct to consumer advertising pushed the message.

But, meanwhile, studies were repeatedly showing that lowering the bad cholesterol and raising the good cholesterol did not always reduce risk – and sometimes increased it. These things were not equivalent. And even though we are confident in the role of cholesterol in causing heart disease and stroke, there was growing concern that drugs that change the levels can have other effects that might offset any benefit.

So the new guideline moved away from the target levels that have been a dominant influence on practice for decades.

2) Use proven medications. A corollary to the recognition that drug treatment that improves the cholesterol levels may not lower risk is that the only way to know is to do a study of the effect of the drug on risk. So the guidelines endorsed strongly that we should be using drugs that are shown to reduce risk. This is a major statement, essentially saying that knowing what a drug does to your lab tests is not sufficient to know if it is helping you.

We are fortunate that the statin drugs reduce risk, remarkably well. And so these are the drugs that are endorsed.

The guidelines are clear that drugs and strategies that improve cholesterol levels but are not known to reduce risk should be relegated to the back shelf. Those drugs include ezetimibe (Zetia) and its combinations with other drugs like ezetimibe and simvastatin (Vytorin) or ezetimibe and atorvastatin (Liptuzet), all Merck products.

3) Make decisions about drug treatment based on whether it is worth it to you. That is, know the likely size of the benefit to you. What are you going to gain? That gets us to the controversy. To know the benefit you need to know your risk. If your risk is already low, then drug treatment is unlikely to do much for you.

The controversy about the risk calculator surrounds its accuracy. Such discussions are useful – and there should be room to debate the best approach to measure risk.

But the even more important point is that the guidelines set a threshold for drug treatment of 7.5% over 10 years. Now this is a problem if people take this as a requirement rather than a recommendation.

As a recommendation, whether the calculator is exactly right becomes less important. Patients may not think much differently about a risk that is 1 in 13 or 1 in 8 or 1 in 20. That is not to say that we should not care about having an accurate risk calculator, but just that for patients the difference may not matter that much.

It is critical that the guideline recommendation be understood as exactly that, rather than as a requirement. We need to communicate that the decision about treatment is a personal one. What I consider high risk and worth treatment may be different from what you would. We should not be imposing any threshold for treatment on patients; we should be fostering informed decisions.

Final point. The guidelines are a remarkable step forward. Amidst the debate about the calculator’s accuracy, it is important to remember that a small group of individuals, steeped in years of practice focusing on cholesterol levels, were able articulate a clear set of principles that will change the direction of practice. These individuals are not rebels; they are stalwarts of the prevention community. They are people who likely have given hundreds of talks about target levels. They are people who were trusted to write of the most important guidelines in our profession because of their credibility and expertise.

Even though several of the most recent guidelines from the American College of Cardiology and the American Heart Association had recommended moving away from cholesterol targets, everyone was waiting for what this group would say and there was pressure not to depart from the last version. The courage of the Guidelines group to depart so definitively from a tenet of prevention is truly amazing. And it is because they were willing to assess the medical literature dispassionately and come to a decision based on what we know.

What they changed will endure: focus on what matters (your risk), decide if the benefit to you is worth the risk and cost of the drug (know your risk and the likelihood it will be meaningfully reduced with treatment), and preferentially use medications that reduce your risk (rather than make your lab tests look good with an unknown effect on your risk).

I hope that what they achieved will be told and remembered.