Two new drugs, Repatha and Praluent, were approved by the Food and Drug Administration several months ago amid much ballyhoo. Both are antibodies that specifically target PCSK9, a protein which reduces the number of receptors on the liver that remove LDL cholesterol from the blood. By blocking PCSK9’s ability to work, more receptors are available to clear LDL. This novel mechanism was proven safe and effective in clinical trials, lowering LDL cholesterol levels by 40% or more in patients already taking statin drugs. However, powerful treatment comes with a powerful cost-- over $14,000 per year for each patient.
This concern over pricing gained traction by a recent report by the Institute for Clinical and Economic Review (ICER), a nonprofit organization that gained credence by its previous warnings over the cost of new hepatitis C drug regimens. The analysis was a computer simulation of the adult American population calculating the price of purchasing the drug and the potential cost savings from reduced death, heart attack and strokes due to drug efficacy over the next 20 years. This cold-hearted mathematics puts a price tag on each additional “quality-adjusted life year”, which health economists use to determine cost-effectiveness. These calculations do not reflect scientific certitude and are obviously subject to criticism and alternative modeling. However, the ICER report is used by many large health plans to push back on the pharmaceutical industry and negotiate discounts. And since the ICER analysis priced the new drugs at $3,600-$4,800 a year, this could be a giant shove, not a nudge.
Although the methodology may be arcane, the issue is overt. Given the number of hypercholesterolemia patients requiring lifetime therapy and the known benefits of lowering LDL on morbidity and mortality, the indiscriminate utilization of these potent agents could break the healthcare bank. For the total American population, not a particular patient, is there a better alternative utilization of this money?
Now comes the doctors’ dilemma; what role should physicians play in this controversy? We are the ones actually prescribing the medication at the point of service. Do we abdicate responsibility under the rationalization that we are only doing our job and acting in the best interest of our patients? And these drugs lower LDL cholesterol better than any alternative. Or should we weigh in? In the big picture, there is a potential guns or butter situation. Also, the facts are that these drugs only lowered LDL, but whether they will prevent cardiovascular events is only surmised from our past experience with the lipid-lowering effect of statins. Statin efficacy is predicated on modulating the microvascular interface of endothelium and blood as well as decreasing LDL. The relative value of each is unknown and the PCSK9-inhibitors may differ from other agents, for better or worse. In my mind, a responsible stance is to prescribe these new drugs only in the population studied, i.e. patients with persistently elevated LDL despite optimal statin dosage or who cannot tolerate statins. We await the follow-up analysis in 2017 to see if the new drugs actually improve and lengthen lives as well as improve lipid profiles
By Norman Silverman, MD, with Ryan McKennon, DO and Ren Carlton