Flawed JAMA Study Misrepresents Costs And Benefits Of New Cholesterol Drugs

To The Editor:

The August 17 article “PCSK9 Inhibitor Prices Should Fall Two-Thirds for Cost-effectiveness: Analysis,” offers a biased view of 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 one death every 40 seconds and $600 billion in costs each year. Driving down the cost of CVD complications is imperative, and innovative therapeutics are our best hope.  PCSK9 inhibitors are intended for patients who have exhausted all other options, including statins.   

This study uses a model with assumptions that over-estimate the size and underestimate the risk of the treated population resulting in exaggerated costs and low estimated value.  In addition, using list price rather than the price payers actually pay after rebates and discounts inflates the cost estimates.  Proposing prices based on arbitrary cost-effectiveness thresholds is what some price controlled healthcare systems outside of the U.S. use to ration care.

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. 

Additionally, its authors’ ties to the group ICER further clouds the study’s conclusions. ICER has been repeatedly criticized for using questionable methodologies that produce drug pricing recommendations to support the insurers.

A balanced discussion about value is critical to inform complex decisions, but analyses should identify ranges and uncertainties, not assign arbitrary prices and ring “alarm bells” with exaggerated cost estimates.  Health experts should focus on collaborating to both assess and unlock the value of innovation, while ensuring patients get access to the medicines they need.


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