This week a new class of cholesterol-lowering drugs, PCSK9 inhibitors, were announced with tremendous fanfare. These drugs cleared the first approval hurdle through the FDA and are expected to be commercially available in the fall.
So what are these compounds, why is everyone so excited about them, and, most importantly, what does this news mean for you?
LDL receptors in the liver remove LDL (bad) cholesterol from the blood so the more LDL receptors you have, the lower your LDL cholesterol level. PCSK9 is a naturally-occurring protein that reduces the number of liver LDL receptors, thus increasing your LDL cholesterol level.
Researchers became interested in the PCSK9 protein when they found that people who had genetically low levels of PCSK9 had naturally very low LDL levels.
These new drugs, PCSK9 inhibitors, are designed to destroy the PCSK9 protein resulting in more liver LDL receptors and lower LDL levels. And the effect is not small – these treatments can lower cholesterol levels significantly more than those achieved with the highest doses of the most powerful statins. They are self-injected about every two weeks, and appear to be well tolerated in the trials conducted so far, although some users reported memory issues. Long term trials have not been completed so we don’t know if treatment with these drugs will translate into improved patient outcomes or will result in other side effects.
Many cardiologists are giddy about the sheer magnitude of the cholesterol lowering effect. It has the drug manufacturers, Sanofi and Amgen, giddy as well! These treatments are expected to cost approximately $10,000 per patient per year and as many as 10 million Americans may be candidates for the drugs.
Just in case you don’t have a calculator handy, that translates to $100 billion per year. Every year.
So what does this mean for you? You will soon have a new option for lowering cholesterol – assuming your insurance company agrees to pay for it. Given the astronomical cost, it’s likely insurance coverage will be limited and very selective, at least initially, with only those patients who have familial hypercholesterolemia (genetically driven very high LDLs) being approved.
Even if coverage is wider, you should:
• Be on the lookout for the long term trial data to be sure the drugs benefits go beyond simply making the LDL number look good.
• Be skeptical about the reported rates of side effects. Statin trials reported less than 2% of people affected by muscle achiness. In practice, that proportion is closer to 20%.
Here’s the bottom line: PCSK9 inhibitors treat ONE risk factor for heart disease at an enormous cost with unclear long term impacts. For context, statins have been lowering cholesterol for nearly 30 years and heart disease is still the number one killer of Americans.
The tremendous cost — up to $100 billion per year — will be passed through insurers to each and every one of us.
Finally, imagine if we used the money in a different way. High cholesterol, high blood pressure, high blood sugar, excess weight are all risk factors for heart disease and are all food related. So instead of treating the SYMPTOMS of a poor diet, how impactful could we be if we treated the CAUSE: diet itself? $100 billion per year pays for a LOT of food. Imagine the possibilities if doctors prescribed the right foods instead of expensive drugs. The costs would be dramatically lower and the outcomes dramatically better.