Updated NLA Guidance Identifies Patients in Which PCSK9i Use Provides Reasonable Value

The National Lipid Association (NLA) has released updated guidance for clinicians on identifying patients for which proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor therapy would provide reasonable value.

Currently, two PCSK9 therapies have been approved by the Food and Drug Administration. Alirocumab is indicated as an adjunct to diet, alone or in combination with other lipid-lowering therapies (eg, statins, ezetimibe) in adults with primary hyperlipidemia (including heterozygous familial hypercholesterolemia [HeFH]) to reduce LDL-C. Evolocumab is approved as an adjunct to diet, alone or in combination with other lipid-lowering therapies, in adults with primary hyperlipidemia (including HeFH to reduce LDL-C and in patients with homozygous familial hypercholesterolemia (HoFH) requiring additional lowering of LDL-C. Both therapies are also approved to reduce the risk of serious cardiovascular events in adults with established cardiovascular disease.

“Although cost-effectiveness informs only part of the decision making process, substantial reductions in acquisition costs for PCSK9 inhibitors in late 2018 and early 2019 have favorably affected economic considerations for PCSK9 mAb therapy for many patients,” the authors noted in the new statement paper “Enhancing the Value of PCSK9 Monoclonal Antibodies by Identifying Patients Most Likely to Benefit.”

Specifically, the authors identified 3 groups of patients on maximally tolerated statin therapy where reasonable cost-effectiveness (defined as <$100,000 per QALY) of PCSK9 therapy may be expected. These include:

• Patients at extremely high risk for atherosclerotic cardiovascular disease (ASCVD) (extensive burden of or active ASCVD, or ASCVD with extremely high risk cardiometabolic risk factors, including heterozygous familial hypercholesterolemia [HeFH]) with LDL-C ≥70mg/dL
• Patients at very high risk ASCVD (less extensive ASCVD and poorly controlled cardiometabolic risk factors) with LDL-C ≥100mg/dL
• Patients at high risk with less extensive ASCVD and well-controlled risk factors, or primary prevention HeFH or severe genetic hypercholesterolemia with LDL-C ≥130mg/dL

Recently, the manufacturers of both alirocumab and evolocumab announced significant price reductions for their products. “It is hoped that this assessment of the value following the recent price reduction for PCSK9 inhibitors will improve access for patient groups with the greatest likelihood of improved clinical outcomes and cost-effectiveness,” said Alan S. Brown, MD, FNLA, FACC, FAHA, President of the NLA.

The full NLA statement can be found here.