Family Heart Foundation’s FOCUS analysis shows that regardless of medical need, prescriptions for PCSK9 inhibitors are being rejected at high rates

Pasadena, Calif. – The Familial Hypercholesterolemia Foundation today published a study in Circulation revealing high rejection rates for PCSK9 inhibitors in the two at-risk patient populations approved for this novel cholesterol-lowering class of therapy.

the Family Heart Foundation’s FOCUS (FH Optimal Care in the United States) study found that 63 percent of patients with presumed FH and 58 percent of patients with established atherosclerotic cardiovascular disease (ASCVD) had rejected claims for PCSK9 inhibitor therapy despite sub-optimal low-density lipoprotein cholesterol (LDL-C) levels on statins. Conversely, ezetimibe, a cholesterol-lowering medication also shown to reduce cardiovascular event rates, was rejected 9 percent and 8 percent of the time, respectively, in the two groups.

“Over 1 million people in the United States living with FH have not been accurately diagnosed, although many will show significant signs of coronary heart disease by the time they are 30 or 40 years old. This study shows that individuals with FH are now experiencing an additional barrier to life-saving care: lack of access to needed therapies,” said Katherine Wilemon, Founder and CEO of the Family Heart Foundation.

In the FOCUS study published today in Circulation, the Family Heart Foundation examined diagnostic information, pharmacy claims adjudication, and laboratory data on over 1.2 million Americans prescribed either a PCSK9 inhibitor or ezetimibe from their national database that combines claims and lab result data.

The two populations studied were patients with presumed FH based on Make Early Diagnosis, Prevent Early Death (MEDPED) criteria with LDL-C levels greater than 190 mg/dL despite being on moderate or high-intensity statins and patients with ASCVD whose LDL-C was greater than 100 mg/dL despite being on moderate or high-intensity statins.

“This study shows that high-risk patients are being denied effective, FDA-approved treatment to address the unmet need for LDL-C lowering. In light of the FOURIER results for PCSK9 inhibitors showing a significant decrease in the risk for a heart attack, we know that denying this treatment is leaving some patients at undue risk,” said Dr. Daniel Rader, Seymour Gray Professor of Molecular Medicine at the University of Pennsylvania and Chief Scientific Advisor to the Family Heart Foundation.

FH is a life-threatening genetic disorder that causes high LDL-cholesterol from birth and significantly increases the risk for early heart disease and heart attacks, independent of diet and lifestyle. An estimated 30 million or 1 in 250 people across all races and ethnicities are impacted by FH. Often undistinguished in clinical care from other causes of high cholesterol, FH remains under-diagnosed and undertreated.

About the Family Heart Foundation
The mission of the Family Heart Foundation, a patient-centered research and advocacy nonprofit organization, is to save lives by contributing to scientific research that leads to greater understanding and improved diagnosis of familial hypercholesterolemia worldwide.

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2 Responses to “Family Heart Foundation’s FOCUS analysis shows that regardless of medical need, prescriptions for PCSK9 inhibitors are being rejected at high rates”

  1. Colleen McCready

    HUGE thank you for everyone’s work on this very important and extremely needed paper-my hope is that this illuminates the issues our FH population has with drug access and that it creates a dialogue with the insurance companies, hopefully making access much easier in this underserved patient group.

  2. Annette Sophocles

    So glad to know that I am not the only one. I have applied four times and been rejected four times for PCSK9 medication. I am FH and have had two heart surgeries. We are five generations of FH patients and are very concerned about educating both physicians and the insurance companies of the benefits of early diagnosis and treatment of this curable disease. Thank you for your efforts !


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