PCSK9 inhibitors - the latest and greatest cholesteorl lowering medications have been in the news everywhere. 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. The more LDL receptors you have, the lower your LDL cholesterol level. PCSK9 is a protein that reduces the number of liver LDL receptors. Doctors became interested in this protein when they found that certain individuals with genetically very low levels of PCSK9 had naturally low LDL levels.
PCSK9 inhibitors are essentially antibodies which destroy the PCSK9 protein. Lower PCSK9 levels mean more liver LDL receptors, leading to lower LDL levels. And the effect is not small – PCSK9 inhibitor treatment can lead to cholesterol levels lower than those achieved with the highest doses of the most powerful statins. PCSK9 inhibitors are injected under the skin once to twice per month. They appear well tolerated in the trials conducted so far, although there have been some patients who experienced 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.
The sheer magnitude of the cholesterol lowering effect has many cardiologists giddy. It has the companies that make these compounds (Sanofi and Amgen) giddy as well! Their drugs (Praluent® and Repatha®, respectively) have cleared the first approval hurdle through the FDA and are on schedule to be commercially available in the fall. Treatment with Praluent® and Repatha® is also expected to cost approximately $10,000 per patient per year and it is estimated that 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.
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 that coverage will be limited and very selective, at least initially, with only those patients who have genetically driven very high LDLs (familial hypercholesterolemia) being eligible.
Even if coverage is wider: 1. 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; and 2. Be a bit skeptical about the reported rates of side effects. The statin trials had less than 1-2% of people affected by muscle achiness. In practice, that proportion is closer to 10 or even 20%. 3. Understand that wider coverage will come with a very high price. Covering the costs for everyone who may be eligible would cause health insurance premiums to go up by $1200 per family per year.
Bottom line: PCSK9 treat ONE risk factor for heart disease - at an enormous cost - with unclear long term impacts. After all, statins have been lowering cholesterol for nearly 30 years and heart disease is still the number one killer of Americans.
Finally, imagine if we used the money that we will be spending on PCSK9s 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 – the 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.