Payer Approval Rates for PCSK9 Inhibitor Therapy

Study Questions:

Proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) are available for familial hypercholesterolemia (FH) and atherosclerotic cardiovascular disease (ASCVD) requiring additional lipid lowering beyond dietary measures and statin use. What are payer approval and rejection rates for PCSK9i prescriptions and the potential factors influencing these rates?


A retrospective, descriptive cohort study was performed using nationwide pharmacy claims linked to electronic medical records from a nationwide data warehouse. The data set includes >220 million patients from all 50 states and all payer types with 5,140 distinct health plans. PCSK9i prescriptions were submitted for 51,466 patients. The main outcome was approval or rejection of PCSK9i prescription claims. Factors associated with approval and rejection of these medications in the United States were assessed.


Of those prescribed a PCSK9i, 47.0% were approved for coverage by the payer. Variables that were associated with PCSK9i approval included age >65 years (p < 0.01), history of ASCVD (p < 0.01), prescription by a cardiologist or nonprimary care provider (p < 0.01), statin intolerance (p = 0.03), longer statin duration (p = 0.01), and noncommercial payers (p < 0.01). Higher low-density lipoprotein (LDL) cholesterol levels were not associated with higher approval rates. Commercial third-party payers had the lowest approval rates (24.4%) and Medicare had the highest (60.9%).


Rates of approval for PCSK9i therapy are low, even for patients who appear to meet labeled indications. Although a combination of clinical characteristics increases the likelihood of approval, payer type is the most significant factor.


This is a very important study of the US experience, and our lipid clinic experience is similar. The results reflect the indications for PCSK9 antibody treatment, which include ASCVD with LDL cholesterol >100 mg/dl despite the highest tolerated dose of statins and the relative rarity of FH (about 1/250 worldwide). Variables not included in this study that are relevant include the commercial payers requiring documentation of statin intolerance that is nigh impossible to obtain from patients and their referring physicians, and the requirement for genetic testing or ‘definite familial hypercholesterolemia’ by the Simon-Broome or Dutch Lipid Network criteria, each of which excludes many of the FH population, based on recent studies. Appeals are a costly process, but have been successful in about 25% of cases.

Clinical Topics: Diabetes and Cardiometabolic Disease, Dyslipidemia, Prevention, Lipid Metabolism, Nonstatins, Novel Agents, Primary Hyperlipidemia, Statins

Keywords: Atherosclerosis, Cholesterol, LDL, Dyslipidemias, Electronic Health Records, Genetic Testing, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Hyperlipoproteinemia Type II, Insurance, Health, Reimbursement, Lipids, Medicare, Pharmaceutical Services, Pharmacies, Primary Prevention, Proprotein Convertases, Subtilisin

< Back to Listings