Costs and Patient Access to PCSK9 Inhibitors

Study Questions:

What is the patient access to PCSK9 inhibitors (PCSK9i) among those who are prescribed therapy?


Using pharmacy transaction data from Symphony Health Solutions, the authors evaluated 45,029 patients who were newly prescribed PCSK9i in the United States between August 1, 2015, and July 31, 2016. Main outcomes included the proportion of PCSK9i prescriptions approved and abandoned (approved but unfilled); multivariable analyses examined factors associated with approval/abandonment including payer, prescriber specialty, pharmacy benefit manager, out-of-pocket cost (copay), clinical diagnoses, lipid-lowering medication use, and low-density lipoprotein cholesterol (LDL-C) levels.


Of patients given an incident PCSK9i prescription, 51.2% were women, 56.6% were ≥65 years, and 52.5% had governmental insurance. Of the patients given a prescription, 20.8% received approval on the first day, and 47.2% ever received approval. Of those approved, 65.3% filled the prescription, resulting in 30.9% of those prescribed PCSK9i ever receiving therapy. After adjustment, patients who were older, male, and had atherosclerotic cardiovascular disease (ASCVD) were more likely to be approved, but approval rates did not vary by patient LDL-C level or statin use. Other factors associated with drug approval included having government versus commercial insurance (odds ratio [OR], 3.3) and those filled at a specialty versus retail pharmacy (OR, 1.96). Approval rates varied nearly threefold among the top 10 largest pharmacy benefit managers. Prescription abandonment by patients was most associated with copay costs (C-statistic, 0.86); with abandonment rates ranging from 7.5% for those with $0 copay to >75% for copays >$350.


In the first year of availability, only one-half of patients prescribed a PCSK9i received approval, and one-third of approved prescriptions were never filled owing to copay.


Criteria for insurer approval for patients prescribed a PCSK9i in our lipid clinic in Michigan were much more stringent during the period of this study than that of Symphony Health Solutions, who track outcomes of prescriptions as a service. Most insurers for our patients require familial hypercholesterolemia be documented by LDL-C >190 mg/dl and genetic testing or high probability by tendon xanthomas, family history and premature coronary heart disease, and for clinical ASCVD by an LDL-C >100 mg/dl with medical record (and some required pharmacy records) documentation of a trial of the highest tolerated dose of two high-intensity statins and at least one of the moderate-intensity statins unless documented rhabdomyolysis. During this past year, response to written appeals has improved considerably. Approval was about 20% and now 50%, but utilization remains low because of copays.

Clinical Topics: Diabetes and Cardiometabolic Disease, Dyslipidemia, Prevention, Atherosclerotic Disease (CAD/PAD), Lipid Metabolism, Nonstatins, Novel Agents, Primary Hyperlipidemia, Statins

Keywords: Atherosclerosis, Cholesterol, LDL, Coronary Artery Disease, Dyslipidemias, Genetic Testing, Health Expenditures, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Hyperlipoproteinemia Type II, Insurance Carriers, Medical Records, Pharmacies, Pharmacy Service, Hospital, Primary Prevention, Rhabdomyolysis

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