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Study Finds Prior Authorization Mandates for PCSK9 Drugs Raise Questions of Access


Researchers were especially concerned about barriers for patients with familial hypercholesterolemia, whose needs would seem clear cut but who nonetheless faced costly hurdles, such as genetic testing.

A recent study finds that prior authorization requirements imposed by payers to prevent overuse of PCSK9 inhibitors are so burdensome they raise questions whether patients with clear need for the cholesterol drugs are being denied access.

The study, by researchers at the Perelman School of Medicine and published January 11, 2018, in the journal Circulation: Cardiovascular Quality and Outcomes, puts numbers behind the outcry heard over the past 2 years from cardiologists, endocrinologists, and other physicians who try to prescribe proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, a powerful class of injectable drugs approved for patients with atherosclerotic cardiovascular disease (ASCVD) and a rare genetic condition, familial hypercholesterolemia (FH).

PCSK9 inhibitors work to reduce low-density lipoprotein (LDL), or “bad,” cholesterol by blocking a protein that prevents the liver from carrying cholesterol out of the body. In clinical trials, 2 approved drugs in the class, alirocumab and evolocumab, have been shown to reduce LDL cholesterol by as much as 60% when patients take it with metformin. Both were approved in 2015.

However, price tags topping $14,000 per year, and the prospect of up to 11 million patients being eligible for the drugs, caused health plans and pharmacy benefit managers to put strict protocols in place for patients to gain access. According to the findings of the new study, those prior authorization requirements may be too strict.

The researchers examined a proprietary database with information on prior authorization policies from 3872 plans affecting 95% of Americans with prescription drug coverage, or 275 million people. All types of plans were represented: commercial, health insurance exchange, Medicare, and Medicaid. The researchers found:

  • Between 82% and 97% of Americans are enrolled in plans that require prior authorization for PCSK9 inhibitors.
  • Many plans require medical records, such as lab results, to be submitted before covering PCSK9 inhibitors, but do not require this when doctors prescribe ezetimibe or liraglutide.
  • Some documentation may be beyond a physicians’ ability to obtain. Examples include adherence measures contained in pharmacy data, or what LDL cholesterol levels were before treatment began, for patients who started statins and then changed doctors.
  • Many plans will only fill PCKS9 inhibitor prescriptions written by a specialist, such as a cardiologist or a lipid specialist, requiring a referral from a primary care physician and an extra copayment from the patient.
  • Some payers require genetic testing results to cover the drugs for FH patients, but will not pay for the genetic tests.
  • While clinical guidelines call for use of PCKS9 inhibitors if patients cannot reach LDL cholesterol goals with high-intensity statins, many were forced to try several other lipid-lowering agents first before the higher-cost drugs were approved.

The study echoes results presented at scientific meetings, as well as reports from clinicians and pharmaceutical leaders over the past year to The American Journal of Managed Care® (AJMC®). A pair of abstracts were presented in 2017 at the meeting of the American College of Cardiology, and in December, results presented at the American Heart Association showed one-third of patients who started a PCSK9 inhibitor stopped taking it for lack of insurance coverage or high out-of-pocket costs.

Jay Edelberg, MD, PhD, vice president and head of Cardiovascular Development and Cardiovascular Affairs at Sanofi, the maker of alirocumab (Praluent), told AJMC® at the time that he had never seen anything like the prior authorization challenges with PCSK9 inhibitors, especially for patients with FH. An author of this new study shared that observation in a statement.

“Of particular concern was that patients with FH, where the need for PCSK9 inhibitors is more straightforward, faced the same cumbersome requirements as did patients with ASCVD,” said Michael Parmacek, MD, chair of medicine at the Perelman School, at the University of Pennsylvania.

Researchers found that administrative burdens for PCSK9 inhibitors were substantial, requiring between 3 and 11 more items on the forms than comparator drugs, ezetimibe and liraglutide. This increased the likelihood of errors, omissions, and delays.


Doshi JA, Puckett JT, Parmacek MS, Rader DJ. Prior authorization requirements for proprotein convertase subtilisin/kexin type 9 inhibitors across US private and public payers [published online January 11, 2018]. Circulation Cardiovasc Qual Outcome 11:e003939.

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