PCSK9 Use Remains Limited Among Familial Hypercholesterolemia Patients

Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors continue to be used limitedly among eligible patients with familial hypercholesterolemia (FH), despite proven benefits.

In new trial data planned for presentation at the ACC.20 Together with Word Congress of Cardiology (ACC/WCC) Scientific Sessions this year, investigators found the drug class—despite having been a marketed adjunct therapy to maximally tolerated statin treatment in patients with FH since 2015—is of notably lesser use than statins.

The retrospective analysis, led by Candace Jackson of the Mayo Clinic, assessed administrative claims data from the OptumLabs Data Warehouse from October 2016 to January 2019. The team sought to assess treatment patterns in the FH patient population—a group burdened with an inherited disorder putting individuals at notable risk of premature cardiovascular disease.

Investigators included individuals diagnosed with FH based on ICD codes, with differences in treatment between various patient groups and demographics evaluated with chi-square tests for categorical variables.

The patient population included 65,210 individuals with an FH diagnosis. Mean patient age was 63.3 years old, with a majority (52%) being women, and one-eighth (12.7%) were African-American.

Compared to the 60.7% of patients with a statin medication fill and no PCSK9 inhibitor prescription, just 1.6% (n = 1048) overall had any medication fill for a PCSK9 inhibitor—with or without other medications.

More than 22,000 patients (35.1%) with FH were untreated during the observed period.

Though women were more likely to be treated with a PCSK9 inhibitor than men (1.8% vs 1.5%, P <.01), they were also more likely to be untreated overall (39% vs 32.8%, P <.01).

Three observed minority groups—African-American (1%), Asian (1.1%), and Hispanic (1.1%)&mdash;were less likely to be treated with a PCSK9 inhibitor than white patients (1.9%).

The availability and use of PCSK9 inhibitors since making the cardiovascular market a half-decade ago has been marked by concerns regarding their cost and patient coverage for care. In an interview with HCPLive® while discussing ACC 2020, Roxana Mehran, MD, noted the impact of promising, new PCSK9 inhibitor clinical data is reduced by these facts.

“We all need to think about how we incorporate the use of these novel agents into a better, more effective, more swift way of reducing LDL and pushing the envelope for our patients—not just those who are at high risk, but also in primary prevention,” Mehran noted. “Of course, it's a cost issue, but the more of these agents that become available, I think there will be a lot of competitive pricing.”

While Jackson and colleagues’ findings evidence their limited use among patients with FH, they also indicate particular issues of usage among differing patient groups overall.

“The utilization of PCSK9 inhibitors in FH patients remains low compared with statin utilization, and significant differences exist based on demographic factor,” investigators concluded.

The study was planned for presentation at ACC 2020.