A snapshot of access to PCSK9 inhibitors during the first year after their approval by the U.S. Food and Drug Administration has shown that access is limited, with less than a third ever receiving the drug after it being prescribed, and that rejection rates vary by payer and pharmacy benefit manager, according to research presented at ACC.17.

Ann Marie Navar, MD, PhD, et al., examined the fate of the first prescription for PCSK9 in 45,029 patients (median age 66 years, 51 percent women) between Aug. 1, 2015 and July 31, 2016, using a database encompassing 90 percent of retail, 60 percent of mail order and 70 percent of specialty pharmacies. The prescriber was a cardiology practice for 48.2 percent and for 36.9 percent it was a general practice. The composition of payers was 39.7 percent commercial and 51.9 percent government. Only 9.8 percent of patients participated in a coupon program.

The prescription was initially rejected for 79.2 percent of patients. Ultimately, the PCSK9 prescription was approved in 47.2 percent of patients, and dispensed for 30.9 percent. Notably, 34.7 percent of approved prescriptions were never picked up.

The median time to approval was three days and the median time to dispensing was 9.9 days. The PCSK9 inhibitor was dispensed more than a month after the prescription for 25 percent of patients.

The rates of rejection ranged from 33.1 to 74.7 percent across pharmacy benefit managers, from 37.9 to 83.5 percent across government–paid insurance programs, and from 33.2 to 77.6 percent across commercial payers. This variability could suggest that factors other than clinical contribute to the rejections, said Navar. She also noted that the prior authorization processes need to balance cost containment efforts and provider burden, and that the prolonged time to dispensing of the drug could have an impact on utilization and provider willingness to prescribe the drug.

On multivariable logistic regression analysis, some of the factors associated with receiving PCSK9 therapy were use of a mail order pharmacy (odds ratio [OR], 4.43), prescription by a cardiologist (OR, 1.60) and commercial plus government health insurance (OR, 4.35). Patients in a coupon program were nearly 17–fold more likely to receive therapy.

Among the limitations of the study are the lack of indication for the PCSK9 inhibitor and clinical data, and it is unknown whether some rejections may have been clinically appropriate. Not all pharmacies were included in the analysis, and ultimate payer attribution is difficult to make because of complex interactions.

"Further details are needed to clarify the cost of the patient copay among those who both received and those who had approved prescriptions," said Kim A. Eagle, MD, MACC, editor–in–chief of ACC.org. "It is clear that cost will continue to limit access to this important class of medications."