The FDA’s approval of the first proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor brought about excitement for some with regards to the effectivenss of the new class of drugs in lowering cholesterol. It also raised concerns about the high costs associated with the medications.

On July 24, the FDA approved alirocumab (Praluent, Regeneron Pharmaceuticals and Sanofi Aventis) as an adjunct to diet and maximally tolerated statin therapy for patients with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease who have not been able to lower their low-density lipoprotein (LDL) cholesterol. By Aug. 27, the agency is also expected to approve evolocumab (Repatha, Amgen), another PCSK9 inhibitor.

Although alirocumab has been proven to lower LDL cholesterol by more than 50 percent compared with other current therapies, it also comes at a high price. The wholesale acquisition cost of alirocumab is $40 per day for the 75 mg and 150 mg doses, or nearly $15,000 per year. The costs to Medicare and insurers will vary, but they will be more expensive than the current standard of care of statins alone or statins with ezetimibe, niacin or fibrates, according to Christopher P. Cannon, MD, a steering committee member of the Phase 3 program that led to alirocumab’s approval.

Cannon said the drug would most commonly be used in patients with HeFH. He added that it could be an option for others who cannot tolerate statins or cannot lower their cholesterol by using statins alone.

Patients receive one injection of alirocumab every two weeks. In clinical trials, more than 85 percent of patients continued taking alirocumab after a year, which Cannon said was a higher rate than with most oral medications.

“The big issue is its cost,” Cannon, a cardiologist at Brigham and Women’s hospital in Boston, told Cardiovascular Business. “One has to balance the cost of the agent versus other available therapies. One would use the lower cost therapies first, but there are patients where this will be needed. If it’s needed, then we would add this therapy… We have to sort through who are the patients who will get benefit that’s reasonable for the cost.”

The Institute for Clinical and Economic Review (ICER), a nonprofit healthcare research organization, is currently reviewing the clinical effectiveness and long-term economic value of alirocumab and evolocumab. ICER plans on releasing a report on the PCSK9 inhibitors for public comment on Sept. 8.

On Oct. 27 in Boston, the independent New England Comparative Effectiveness Public Advisory Council will debate the evidence from the PCSK9 report, provide input from clinical experts, drug manufacturers and patient groups, vote on the strength of the evidence for various patient populations and recommend how the evidence could be applied in practice or coverage policy.

“There are still lots of questions about larger patient groups,” Steven D. Pearson, MD, the founder and president of ICER, told Cardiovascular Business. “Most of the time, people are talking about adding this drug to statins, but there are a large number of patients who aren’t on statins for one reason or another. How those patients will be managed is going to be an important question.”

ICER evaluates medications and other therapies that are potential blockbusters and are associated with high costs. Last year, it issued a report on hepatitis C drugs. ICER is also currently examining CardioMEMS heart failure system (St. Jude Medical) and sacubitril/valsartan (Entresto, Novartis), an oral medication that the FDA approved on July 8 to treat patients with heart failure and reduced ejection fraction.

“The reason that [PCSK9 inhibitors] ended up on our list of topics was because people viewed them as significant additions to the treatment options for patients with high cholesterol,” Pearson said. “There are a lot of patients with high cholesterol. I think [PCSK9 inhibitors] are going to be very important clinically. I think they will have an important impact on healthcare budgets. Our job is to help sort out whether they provide a good value both for patients and the healthcare system.”