Familial Hypercholesterolemia (FH)
The FH Foundation welcomes Amgen's  decision  to substantially lower the list price of its novel lipid-lowering therapy, the PCSK9 inhibitor Repatha, by 60%. PCSK9 inhibitors were approved by the Food and Drug Administration (FDA) in 2015 for the treatment of individuals with familial hypercholesterolemia (FH), with or without atherosclerotic cardiovascular disease on maximally tolerated statins, in need of additional LDL-cholesterol (LDL-C) lowering.

Published FH Foundation analysis has shown both high insurance plan rejection rates and a high abandonment rate of approved PCSK9 inhibitor prescriptions. Real world data shows that high copays impact an individual's ability and/or willingness to take the medications their physicians prescribe. The lower list price of Repatha is expected to significantly reduce out-of-pocket costs for Medicare patients in particular. 

We hope Amgen’s lower list price, in addition to Sanofi/Regeneron's program to offer substantial rebates for its PCSK9 inhibitor, Praluent, will make these important LDL-C lowering treatments more affordable and available to individuals who need them, including those with FH.

Our work does not end here.  

FH is the leading genetic cause of heart disease in the world. The biggest barrier to care continues to be the lack of understanding and recognition of FH. Nine out of 10 people born with FH are unaware of their risk and the urgent need for lifesaving interventions. For many individuals with FH, appropriate treatment is first-line statin therapy, while others need combination therapy, including a PCSK9 inhibitor, to reach the lower LDL-C levels needed to reduce cardiac event risk.

The FH Foundation is concerned that m any health plans and pharmacy benefit managers continue to "define" FH using diagnostic criteria that are out-of-line with the realities of clinical practice in the U.S.

To help ensure that prior authorization criteria for FH do not inadvertently exclude appropriate, high-risk FH patients, the FH Foundation developed Guidance for Prior Authorization Criteria for PCSK9 inhibitors for Familial Hypercholesterolemia Treatment in Adults , endorsed by the American Society of Preve ntive Cardiology. 

We will continue to engage with health plans and PBMs, as well as others in the healthcare system willing to work with us to improve the understanding, identification, and appropriate treatment of FH to prevent heart disease and save lives. Today, during the  Cardiometabolic Health Congress , we will join the Association of Black Cardiologists to discuss overcoming treatment barriers for high-risk and minority patients.