From the Journals

Insurance approvals are hard to get for new PCSK9 inhibitors



Insurance approval rates for proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors are strikingly low, according to a study published in Circulation.

Two approved PCSK9i drugs, alirocumab and evolocumab, were approved for coverage just 47% of the time. Because of their high price tag – about $14,000 per year – researchers had expected low rates of insurance coverage for the novel drugs, which encourage degradation of low-density lipoprotein cholesterol by blocking preserving LDL-C receptors in hepatocytes. However, they were surprised at the size of the problem.

“I think we knew that physicians were having challenges, but the number [of coverage approvals] was maybe lower than we expected,” Robert Yeh, MD, the study’s lead investigator, said in an interview. “Physicians and patients need to be aware that in perhaps the majority of cases, when they decide these drugs are going to be ones they want to prescribe, there may be a long procedure ahead of them, and they may ultimately not succeed in getting those medications.”

PCSK9 inhibitors are specifically approved for patients with familial hypercholesterolemia, or those with atherosclerotic cardiovascular disease who are unable to achieve satisfactory lipid levels through dietary measures and statin use.

Yet insurance coverage rates were low, even when patients met labeled indications. When the researchers examined the factors associated with insurance coverage, the most important factor was the type of insurance: Commercial third-party insurers approved the drugs about 24% of the time, while Medicare approved them nearly 61% of the time (P less than .01).

Older age, prescriptions by a cardiologist or other specialist, and a diagnosis of clinical atherosclerotic cardiovascular disease (ASCVD) were also associated with higher rates of coverage by insurers (Circulation. 2017 Oct 30. doi: 10.1161/CIRCULATIONAHA.117.028430).

The researchers analyzed data from 9,357 patients with a prescription for a PCSK9 inhibitor. About 60% of the patients had a diagnosis of clinical ASCVD. In all, 4,397 patients (47%) had their prescriptions for PCSK9 inhibitor therapy covered, and 53% of coverage requests were rejected. Nearly 65% of patients who received approval went on to fill their prescription.

There was no association between LDL-C level and the likelihood of approval. In the 32 cases in which the LDL-C levels were 330 mg/dL or greater, 59% of patients were not approved for coverage.

Noncommercial payers were more likely to approve the medication (odds ratio, 12.32; 95% confidence interval, 7.09-21.39), as was Medicare (OR, 5.37; 95% CI, 4.23-6.80).

A recent cost-effectiveness analysis of evolocumab added to standard therapy showed that an annual cost of $9,669 would achieve an incremental cost-effectiveness ratio of $150,000 per quality-adjusted life year gained among patients with LDL levels 70 mg/dL or greater. (JAMA Cardiol. 2017;2[10]:1069-78). Evolocumab is currently listed at $14,523, putting it above that value.

“Now that clinical trial outcome data demonstrating reductions in major cardiovascular events and formal cost-effectiveness studies have identified value-based prices for these medications, it would be hoped that progress can be made such that a greater proportion of eligible patients can be treated,” Gregg C. Fonarow, MD, professor of cardiovascular medicine and science director at the Ahmanson-UCLA Cardiomyopathy Center, Los Angeles, and the lead author of the cost-effectiveness study, said in an interview.

Both sets of findings cut to the heart of the debate over pricing of new medications. PCSK9 inhibitors have a clear benefit in lowering LDL and reducing risk of heart attack, but the drugs’ price tags may limit their availability.

“We need to not be afraid to discuss these issues in plain terms that are not emotional, so that we can get a better sense of what is the amount to spend on a therapy, even when it is effective but potentially pricey,” said Dr. Yeh, director of the Richard and Susan Smith Center for Outcomes Research in Cardiology at the Beth Israel Deaconess Medical Center, Boston. “This is a really important business, philosophical, medical, and ethical debate.”

The study received no external funding. Dr. Yeh reported having no financial disclosures. Dr. Fonarow has consulted for Amgen.

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