IMPORTANCE
Although PCSK9 inhibitors (PCSK9i) were approved in 2015, their high cost has led to strict prior authorization practices and high copays, and use of PSCK9i in clinical practice has been low.
OBJECTIVE
To evaluate patient access to PCSK9i among those prescribed therapy.
DESIGN, SETTING, AND PARTICIPANTS
Using pharmacy transaction data, we evaluated 45,029 patients newly prescribed PCSK9i in the United States between 08/01/2015 and 07/31/2016.
MAIN OUTCOMES AND MEASURES
Main outcomes were: the proportion of PCSK9i prescriptions approved and abandoned (approved but unfilled); multivariable analyses examined factors associated with approval/abandomnent including payor, prescriber specialty, pharmacy benefit manager (PBM), out-of-pocket cost (copay), clinical diagnoses, lipid lowering medication use, and low density lipoprotein cholesterol (LDL-C) levels.
RESULTS
Of patients given an incident PCSK9i 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 ASCVD were more likely to be approved, but approval rates did not vary by patient low density lipoprotein (LDL-C) level nor statin use. Other factors associated with drug approval included: having government versus commercial insurance (odds ratio [OR] 3.3, 95% confidence interval [CI] 2.8–3.8), and those filled at a specialty versus retail pharmacy (OR 1.96, 95% CI 1.66–2.33). Approval rates varied nearly 3-fold among the top 10 largest PBMs. 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 more than 75% for copays greater than $350. Limitations include that the analysis was conducted in the first year of availability before outcomes trials results.
CONCLUSIONS AND RELEVANCE
In the first year of availability, only half of patients prescribed a PCSK9 received approval, and one third of approved prescriptions were never filled due to copay.