The PCSK9 Inhibitor Conundrum

September 23, 2016

A guest blog by Seth Baum, M.D.

Medicine is at a crossroads; cardiology in particular. I saw this fact magnified at the American Society for Preventive Cardiology’s recent town hall on access barriers to PCSK9 inhibitors. The event was entitled, “Unraveling a Therapeutic Conundrum,” and the conundrum is this: Will we embrace the potential that these innovative drugs hold for patients, or will we use them as a scapegoat for soaring health care costs?

First, let’s all at least agree that, without pharmaceutical innovations, we would never have made such a large dent in heart attack and stroke incidents. Statins and novel mediations for heart failure have contributed to our remarkable success in these areas. So before we denigrate the very innovations that make successes like these possible, let’s examine the impact of pharmaceutical innovations, specifically PCSK9 inhibitors.

These treatments unequivocally and predictably lower LDL-C (the “bad” cholesterol) by around 60%. And this reduction is on top of statin therapy! By way of example, a person on a statin with an LDL-C of 200 would typically achieve an LDL-C of 80 after adding one of these medicines.

Also, there is a confirmed relationship between the drop in LDL and one’s risk for heart attack, stroke or death. Though not yet proven (studies are ongoing), such a reduction in cholesterol would dramatically reduce risk. A recent Journal of the American Medical Association article by Kazi et al. concluded that 4.6 million heart attacks, strokes and deaths could be averted through the use of PCSK9 inhibitors.

Yet the researchers weighed in against the medications because they are not “cost effective.”

Their conclusion, however, uses flawed metrics and makes inaccurate assumptions.  For instance, it utilizes a faulty assessment of value, QALYs or Quality-Adjusted Life Years. Sparing you the details, let me simply state that the Affordable Care Act prohibited such a metric; and the Europeans are abandoning QALYs as well.

Further, Kazi assumed all patients would be on these drugs for life, eliminating the possibility of future innovations. This, of course, is a false premise. Innovative medicines or procedures often negate the need for older approaches.

Perhaps most egregious, however, is the fact that Kazi and team used an inaccurate drug price for their calculations. The list price cited by the authors fails to consider the impact of pharmacy benefit managers, middlemen who negotiate drug prices for insurance providers. When PBMs negotiate prices for medications like PCSK9 inhibitors, they typically purchase well below the list price, and even get rebates down the road. Thus the cost of the PCSK9 inhibitors is not the list price cited by Kazi and many others; it is much lower.

Finally, the PCSK9 inhibitor conundrum demands that we consider a cost factor that analysts and researchers often overlook: What is the cost, and the value, of a life? How do we measure this intangible and all-important metric?

I can’t answer this question. I can tell you, though, that withholding medications from those who might benefit would exact a price I’m unwilling to pay: my personal ethos and professional integrity.

Seth Baum, MD, is a preventive cardiologist, cholesterol expert and member of the Alliance for Patient Access.

Categorized in: Blog

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