Amgen has announced new data from two studies that showed that for appropriate patients (on-label) in the US, the majority of prescription claims for PCSK9 inhibitors, such as Repatha (evolocumab), were initially rejected. Additionally, one of the studies showed no major differences in patient characteristics across those approved and denied, suggesting a utilization management process that is not driven by any observable clinical criteria. The studies were presented at the American College of Cardiology 66th Annual Scientific Session.

"While it is important to ensure that PCSK9 inhibitors are used in appropriate cases, our data suggest that the current approval process is lengthy and highly variable by payer. High initial rejection and slow approval rates may be preventing patients who could truly benefit from getting these drugs," said Ann Marie Navar, M.D., Ph.D., assistant professor of medicine at the Duke Clinical Research Institute and lead study investigator. "This study highlights the need to better investigate the impact of policies around drug access on the utilization of novel therapies, including pricing, payments and reimbursement, and the approval process."

In a retrospective study presented as Featured Clinical Research, researchers evaluating 45,029 new PCSK9 inhibitor prescription claims found an average of 79.2 percent were initially rejected across commercial and Medicare plans, and of those, 52.8 percent were ultimately rejected. Additionally, 34.7 percent of prescriptions were abandoned (unfilled) by the patient. Rejection rates varied by prescribing provider and payer (p<0.0001). Of prescription claims submitted to commercial payers, 71.2 percent were ultimately rejected. Among prescription claims submitted to government payers, 40.0 percent were ultimately rejected. This analysis did not specifically look at the patient characteristics of those denied and approved. (Abstract 415-08)

"Individuals with familial hypercholesterolemia are by definition at high risk for heart attacks in the prime of their lives. PCSK9 inhibitors were developed and approved with FH patients in mind and yet, too often, they are being denied appropriate therapy for their genetic condition," said Katherine Wilemon, founder and chief executive officer of The FH Foundation. "All stakeholders in the health care arena need to live up to their responsibility to get the right treatments to the right patients."

Results of a second retrospective study of 44,234 new PCSK9 inhibitor prescription claims showed 83 percent of PCSK9 inhibitor prescription claims were initially rejected, and of those, 57 percent were ultimately rejected. Final rejection rates were higher in commercially insured patients (69.5 percent) compared to Medicare patients (42.3 percent). When comparing characteristics of approved and denied patients, there were no major differences in baseline statin use, statin intensity, ezetimibe use or history of co-medication use, including antiplatelet therapy, a clinical characteristic highly suggestive of atherosclerotic cardiovascular disease (ASCVD). (Abstract 1258-435)

"The similarities in clinical profiles between accepted and rejected patients suggest concerning inconsistencies in the approval-rejection process," said Seth Baum, M.D., president of the American Society for Preventive Cardiology and lead study investigator. "These results deepen concerns that without meaningful improvement in the burdensome processes and complex access issues, many of our high-risk ASCVD and FH patients with uncontrolled LDL cholesterol levels will continue to be denied or delayed access to a PCSK9 inhibitor, an important and approved treatment option."

In the US, there are approximately 11 million people with ASCVD and/or familial hypercholesterolemia (FH) who have uncontrolled levels of low-density lipoprotein cholesterol (LDL-C) over 70 mg/dL, despite treatment with statins or other cholesterol-lowering therapies.1,2

Estimates based on these access restrictions and real world data suggest at least 100,000 heart attacks and strokes could have been avoided last year in the U.S. alone if all of the appropriate high-risk patients were actually treated with Repatha.3,4

Amgen is committed to providing personalized support services for patients and providers in the U.S. through its RepathaReady™ program. RepathaReady is a comprehensive suite of services to help patients and providers, including a Repatha co-pay card for eligible commercial patients, insurance coverage support and injection training.

Amgen also provides patient assistance for its medicines marketed in the U.S. in a variety of ways, including free medicines through The Amgen Safety Net Foundation for qualifying individuals with no or limited drug coverage.

Payer policies that restrict the ability of appropriate patients to access medicines are not limited to PCSK9 inhibitors. Other organizations have recently highlighted concerns with payer utilization management practices, including the American College of Cardiology and the American Medical Association along with a coalition of 16 other organizations.