PCSK9-Inhibitor Coverage Often Denied for Patients With Familial Hypercholesterolemia

Fran Lowry

May 03, 2017

STANFORD, CA — Patients at high risk for atherosclerotic cardiovascular disease (ASCVD), including those who have familial hypercholesterolemia (FH) and who have had an inadequate response to statins, are being denied access to PCSK9 inhibitors, even though the nonstatin lipid-lowering therapies have been approved for use in such patients since 2015, according to a new study[1].

"Patients who have a very high risk for cardiovascular disease, either because they have a genetic predisposition to it or because they already have had a heart attack, are still having a hard time getting [proprotein convertase subtilisin kexin type 9] PCSK9 inhibitors, even when they are on standard therapies and still have a very high cholesterol," first author Dr Joshua Knowles (Stanford University, Stanford, CA) told heartwire from Medscape.

The study is published in the April 26, 2017 issue of Circulation.

"Our major goal in doing this study is to bring attention to the fact that we all need to come together and figure out better ways of identifying very high-risk patients, making sure they are on the right therapies already, and then when that is not enough, to expedite the approval process for those other new therapies. The PCSK9 inhibitors are already approved and are beneficial for these patients. We want to get the right drugs to the right people at the right time," Knowles said.

The aim of the current study, dubbed the FH Optimal Care of the US (FOCUS) study and designed by the FH Foundation, was to assess current treatment patterns of FH patients as well as rejection rates of PCSK9 inhibitors in high-risk FH or ASCVD patients.

Knowles and his team used the FOCUS data set that had both diagnostic information and pharmacy-claims adjudication data for over 140 million individuals.

They were able to link the pharmacy-claims adjudications with clinical characteristics of the patients based on diagnostic codes and laboratory results.

Of these, 1.12 million individuals had a claim for either a PCSK9 inhibitor or ezetimibe (Zetia, Merck).

The researchers were able to identify 5795 individuals with laboratory characteristics consistent with FH and 415,133 individuals with atherosclerotic cardiovascular disease.

Among the FH patients, 237 had an LDL-C value >190 mg/dL despite being on statins to lower their cholesterol. However, the majority (63.3%) of prescriptions for PCSK9 inhibitors in this group of patients were rejected. By comparison, only 9% of prescriptions for ezetimibe were rejected.

Among the ASCVD patients, 1622 had an LDL-C value >100 mg/dL despite being on statins to lower their cholesterol. Among these patients, 57.5% of PCSK9-inhibitor prescriptions were rejected. By comparison, only 8.2% of prescriptions for ezetimibe were rejected.

"PSCK9 inhibitors are new injectable pharmaceutical agents that are very expensive, and ezetimibe is an oral medication that has long been second-line therapy and is set to become generic," Knowles said. "It's not surprising that the PSCK9 inhibitors were rejected, but the results are still eye-opening."

Similar results were presented by Dr Ann Marie Navar (Duke Clinical Research Institute, Durham, NC) in March at the American College of Cardiology 2017 Scientific Sessions and reported then by heartwire .

Commenting on the current study in email correspondence, Navar said, "In our study, fewer than one in three adults was prescribed a PCSK9 inhibitor in the first year of availability received therapy. This study shows that rejection rates are high even among patients who appear to meet clinical criteria for therapy. This raises concern that the current utilization management process is filtering out appropriate-therapy candidates."

A Critically Important Study

Navar added that the FOCUS study is critically important "as we seek to continue to move the needle in reducing the burden of cardiovascular disease. We now know that PCSK9 therapy can lower the risk of cardiovascular disease events in high-risk adults. However, the effectiveness of these therapies looks to be severely limited by barriers to patient access."

She added: "As a practicing cardiologist myself, I suspect most prescribers will not be surprised by these results. Providers already spend an enormous amount of time and resources navigating the prior authorization process, which is often complicated, prolonged, and frustrating."

Navar predicted that the stress on the system will only increase as demand for PCSK9 therapy increases.

"We have to find a better system to manage costs while maximizing benefit of these novel therapies to our patients," she said.

The study was supported by the FH Foundation. Knowles reported research grants to his institution from Amgen and the AHA. Disclosures for the coauthors are listed in the paper. Navar reported research support from the National Institutes of Health, Amgen, and Sanofi & Regeneron Pharmaceuticals and consulting for Amgen and Sanofi.

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