Cost-Effectiveness Model Greatly Exaggerates Costs of PCSK9 Inhibitors

To The Editor:

The August 16 article “Analysis: PCSK9 inhibitors not cost-effective, could greatly increase US health care costs,” highlights a study published in JAMA that provides an inaccurate portrayal of the value of PCSK9 inhibitors and may be used to support payer policies that make it more difficult for patients to access them.

Cardiovascular disease (CVD) is the number one killer in the U.S, responsible for $600 billion in costs each year. Driving down the cost of CVD complications is imperative, and innovative therapeutics are our best hope. 

The FDA is clear that PCSK9 inhibitors should be used in patients with clinical atherosclerotic cardiovascular disease, who are receiving maximally tolerated statin treatment and require additional lowering of LDL, as well as patients with heterozygous or homozygous familial hypercholesterolemia.  Yet, most of the concern from payers assume that all patients with elevated LDL will be treated. 

The authors assert that these medications would add $120 billion in annual costs.  In fact, these estimates are around 400 times higher than the 2016 analyst consensus estimates for PCSK9 inhibitors. Compared to other economic analyses, this paper appears to focus on a population at a 3-4 times lower risk of CV events and uses list prices rather than the prices actually negotiated with payers after rebates and discounts.  In addition, the model overestimates the number of patients who will receive PCSK9 inhibitors, intended for use in high-risk patients who have exhausted all other options.  All of this inflates the cost estimates.

Using unrealistic assumptions rather than probable scenarios, can actually prevent patients from getting the medicines that you, their doctors, prescribe — which may result in harm to those most in need. 

Despite the authors’ claims, the value-based price range for these drugs is far from settled; a previous publication concluded that PCSK9 inhibitors are cost-effective at prices in the market today. 

Nearly a year after the FDA approved these medicines, approximately two-thirds of patients prescribed a PCSK9 inhibitor continue to struggle to get these medicines from their insurer. Unfortunately, “utilization management criteria” and paperwork have all but closed the door for many patients who fit the FDA prescribing information. It’s time to stop blocking appropriate patients from getting access to the medicines their doctors believe they need, and studies like this do them no favors.

Health experts must not focus on scare tactics, but rather on how we can collaborate and agree on how to assess value in the complex market-based U.S. system, with the shared goal that patients get access to the medicines they need. 


Joshua J. Ofman, M.D., MSHS
Senior Vice President, Amgen