Kevin Maki, PhD: Balancing Cost and Benefit With PCSK9 Inhibitors

Kevin Maki, PhD, the chief scientist at the Midwest Biomedical Research Center for Metabolic and Cardiovascular Health, sat down with MD Magazine to discuss the pros and cons of PCSK9 inhibitors.

Despite their overall success and great benefit in treating patients, their high cost has created a debate among experts and patients about whether or not the benefits are worth the costs of the medication. Maki, an expert in the lipid space, explained that in some patients, there must be a line of decisiveness where the benefit they would receive justifies the price of the therapy.

Maki also addressed the issue of high-priced drugs that the health care system in the United States is currently faced with, debating the complicated issue. The news of CVS's providing of real-time cost prescription data might help, according to Maki.

Kevin Maki, PhD, Chief Scientist at the Midwest Biomedical Research Center for Metabolic and Cardiovascular Health:

Well, I think that things may change [with proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors]. We're expecting new outcomes data from the ODYSSEY outcomes trial that's just ending, and so the results are going to be available next year. We have 1 trial now, FOURIER, which showed a benefit, the trial was very short, though. The follow-up period averaged only 2.2 years, and so the full benefit probably was not realized in the patients who participated in that trial. With ODYSSEY outcomes, the follow-up time will be longer. I think we'll see more evidence of benefit, and then the conversation becomes one where the question is really who are the appropriate patients in whom the health care system can afford to provide this very effective treatment?

I'm saying that now with the assumption that ODYSSEY outcomes will produce the expected results we would be very surprised if that were not the case because we have so much evidence now that lowering atherogenic cholesterol is effective for reducing event risk. It's not just the amount of lowering, it is how long it stays low. So because of that, we expect favorable results with ODYSSEY outcomes. If that's the case, then I think there's gonna be a much stronger argument for treating more people with PCSK9 inhibitors.

Then, it becomes a trade-off between benefit versus cost. I think they're both important because, in the end, you know, economics is the study of limited resources with alternative applications. If we're spending money on PCSK9 inhibitors, you know, there may not be money available to spend on other things. So, in patients who have high enough risk to justify the treatment, I think it's going to make very good sense.

Ultimately payers, and as a society, we have to figure out what the thresholds are that we're going to consider when deciding what makes sense economically and what doesn't make sense. These are very complicated issues and we do not have great systems right now for dealing with those.

I think the CVS approach is very helpful because I know in many cases that people are prescribed a medication that's very expensive, [and] their physician really has no idea what the cost is and the patient doesn't know that there is an alternative that might be less expensive. As one example, recently, I heard of a patient who was prescribed a medication that was something along the lines of $600 per month for the medication. It was a combination of 2 things, but if each of the things was taken individually, the price would have been about one-tenth of that.

These types of things do occur. Physicians don't know what the costs are when they're writing the prescription often, and patients don't know what the costs are when their physician is recommending something. So there really isn't that opportunity to have a discussion about "is there an alternative that might be less expensive?" Like many places in our health care system, we don't have good systems to manage expenses because we are ultimately going to have to manage expense versus benefit, and the current system doesn't lend itself very well to doing that.