FDA approves evolocumab for prevention of MI, stroke, coronary revascularization

Seth J. Baum, MD, FACC, FACPM, FAHA, FNLA, FASPC

First, I’m thrilled. It was completely appropriate and completely expected. I was especially happy that they mentioned that the drug was approved for monotherapy as well, so that was interesting.
The implications on clinical practice will hinge on the payer’s approach or payer’s response to the change in the indication for the drug. The drug has always been indicated for a certain type of individual, one who has a need for further LDL reduction on a maximally tolerated statin dose; however, they’ve been denying the drug up to rates of 80%. There have been a number of papers that have clearly shown that many of these denials are inappropriate, so that’s established.
Now we have an additional indication though, which is to reduce the risk of heart attack, stroke and revascularization. The question will be, will there be liability associated with continued denials of the drug? In other words, now that there’s an indication for heart attack, stroke and revascularization, what happens if they deny an individual, and that individual then has a heart attack, stroke or revascularization? Are there legal implications? That’s my question. That will drive whether or not the payers will suddenly start approving.
I have no understanding of how cost is derived and determined, so I’m not sure that this will affect the cost. I don’t see why this would affect cost.
Access is all about the payer. Access has been terribly limited. Now there are indications that include cardiovascular endpoints, heart attack and stroke in particular, and it should make it much harder for the payers to deny access, but that remains to be seen.
I can give you an example, and the example is an individual who has given me the right to use her name. It is Donnette Smith, and the reason why she’s particularly relevant is that she’s the president of Mended Hearts. She has familial hypercholesterolemia. She told me that her LDL has been up to 300 mg/dL. She’s had five cardiovascular procedures, and she has been denied PCSK9 inhibitors repeatedly by Blue Cross and Blue Shield of Alabama. I’ve been trying very hard through tweeting and other internet modalities to raise awareness about her because here’s a woman who has devoted her life to helping others with cardiovascular disease, and she’s being denied an appropriate therapy. Let’s see what happens with her after the indication changes. She can be a great test case.
Many people have made a lot about the fact that mortality was not reduced in FOURIER, and you see that there’s no indication for reduction of mortality. It’s just for heart attack, stroke and revascularization. Nobody expected a mortality rate reduction in a 2.2-year trial in modern-day medicine. You just won’t see that. It’s not going to happen with lipid-lowering therapy. It hasn’t happened. It will not happen. The absence of mortality indication should never be used against prescribing the drug. I would caution everybody about that.

Robert H. Eckel, MD

This is a timely and important approval with substantial implications on clinical practice, although it is conditional based on the FDA indications for the use of evolocumab.
This will hopefully affect the cost of this medication, but we’ll need to wait for data from the ODYSSEY OUTCOMES trial with similar benefit before the competition is there for cost reduction.
There are many patients who need this class of drugs and the indications are clear. Now it is up to pricing.