PCSK9 Inhibitors: Aligning Prices With Outcomes

It was only a few short years ago that the world of health care was abuzz with launch of the then new class of hyperlipidemia treating medications that targets proprotein convertase subtilisin/kexin type 9 (PCSK9s). While there was a lot of excitement within the treatment community for another treatment option with the ability to reduce LDL, it seemed that the managed care community was more on edge. The movement of the treatment of a condition treated with traditional drugs into the specialty space had the potential to be budget busting. While I think that some of the worry within managed care was overblown likely due to the proximity of the PCSK9 launch after the first of the curative hepatitis C drugs, it was definitely good for us within managed care to be cautious of the unknowns surrounding these drugs.

Now, a few years later it does not seem as though the PCSK9 inhibitors have left the budget impacts some feared they might. I think much of this has to do with the initial reaction within managed care and the tight controls we used to ensure proper use. While watching this unfold, the question in many of our minds has been if the use of a PCSK9 inhibitor leads to any meaningful outcome or if their use just leads to the chasing of lower lab levels.

In the recent months, we have begun to get the answers about outcomes we have been waiting for. Both Amgen and Sanofi/Regeneron have released data from outcomes-based trials of their respective PCSK9 agents. While there is not space here to give a complete review of the released results in this article, I do want to look at a short review of the primary endpoints. For Repatha (evolocumab; Amgen), they studied within the Fourier trials a primary composite outcome of incidence of cardiovascular death, MI, stroke, hospitalization for unstable angina, or coronary revascularization. The study found that this outcome occurred in 12.6% of subjects in the Repatha group versus 14.6% of the placebo group. The Odyssey trial used to study the outcomes for Praluent (alirocumab; Sanofi/Regeneron) looked for a similar composite outcome of the time to first occurrence of coronary heart disease death, nonfatal myocardial infarction, unstable angina requiring hospitalization or ischemic stroke. It was found to be lower in the Praluent group vs the placebo group (9.5 vs. 11.1 percent). The primary results for both of these studies were found to be statistically significant.

It appears that this data, along with the ICER report on the cost-effective thresholds for these drugs, have helped the manufacturers to rethink their pricing strategies for these drugs. The outcomes data is likely not as robust as they were hoping for and their willingness to concede on pricing appears to reflect this. It was recently announced that Sanofi/Regeneron have reached an agreement with Express Scripts to rebate Praluent to meet ICER cost-effective pricing levels in exchange for simplified prior authorization criteria. Other negotiations have been rumored and will likely be announced within the near future.

I think this is the right step to take within this therapeutic area. The outcomes data gives us confidence that there are patients that will benefit from using PCSK9s. By lowering their costs, we should see a win-win-win situation for patients, payers, and manufacturers by providing easier access within cost-effective thresholds. As we see other specialty drugs developed for traditional conditions, I hope to see this kind of data and price negotiation happen to allow for access to drugs without breaking budgets.

—Russ J Spjut, PharmD​

Russ J Spjut, PharmD, is owner of Formulary Intel Consulting. He is a residency trained pharmacist in managed care with experience in both commercial and Medicare Part D PBM operations. He has been involved in formulary management, P&T committee presentations, clinical program development, formulary strategy, clinical analysis, client management, and review of coverage determination requests for a major health care management company.

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