Cost-effectiveness of PCSK9 Inhibitor Therapy in Patients With Heterozygous Familial Hypercholesterolemia or Atherosclerotic Cardiovascular Disease.

Pubmed ID: 27533159

Journal: JAMA

Publication Date: Aug. 16, 2016

Affiliation: Department of Medicine, Center for Vulnerable Populations, University of California, San Francisco2Department of Medicine, University of California, San Francisco3Department of Epidemiology and Biostatistics, University of California, San Francisco8Divisi.

MeSH Terms: Humans, Male, Adult, Female, Aged, Aged, 80 and over, Cardiovascular Diseases, United States, Middle Aged, Anticholesteremic Agents, Atherosclerosis, Cholesterol, LDL, Cost-Benefit Analysis, Quality-Adjusted Life Years, Hyperlipoproteinemia Type II, Drug Costs, Antibodies, Monoclonal, Ezetimibe, Proprotein Convertase 9, Proprotein Convertases, Serine Endopeptidases, Uncertainty, Antibodies, Monoclonal, Humanized

Authors: Bibbins-Domingo K, Coxson PG, Guzman D, Moran AE, Penko J, Kazi DS, Ollendorf DA, Pearson SD, Tice JA

Cite As: Kazi DS, Moran AE, Coxson PG, Penko J, Ollendorf DA, Pearson SD, Tice JA, Guzman D, Bibbins-Domingo K. Cost-effectiveness of PCSK9 Inhibitor Therapy in Patients With Heterozygous Familial Hypercholesterolemia or Atherosclerotic Cardiovascular Disease. JAMA 2016 Aug 16;316(7):743-53.

Studies:

Abstract

IMPORTANCE: Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors were recently approved for lowering low-density lipoprotein cholesterol in heterozygous familial hypercholesterolemia (FH) or atherosclerotic cardiovascular disease (ASCVD) and have potential for broad ASCVD prevention. Their long-term cost-effectiveness and effect on total health care spending are uncertain. OBJECTIVE: To estimate the cost-effectiveness of PCSK9 inhibitors and their potential effect on US health care spending. DESIGN, SETTING, AND PARTICIPANTS: The Cardiovascular Disease Policy Model, a simulation model of US adults aged 35 to 94 years, was used to evaluate cost-effectiveness of PCSK9 inhibitors or ezetimibe in heterozygous FH or ASCVD. The model incorporated 2015 annual PCSK9 inhibitor costs of $14,350 (based on mean wholesale acquisition costs of evolocumab and alirocumab); adopted a health-system perspective, lifetime horizon; and included probabilistic sensitivity analyses to explore uncertainty. EXPOSURES: Statin therapy compared with addition of ezetimibe or PCSK9 inhibitors. MAIN OUTCOMES AND MEASURES: Lifetime major adverse cardiovascular events (MACE: cardiovascular death, nonfatal myocardial infarction, or stroke), incremental cost per quality-adjusted life-year (QALY), and total effect on US health care spending over 5 years. RESULTS: Adding PCSK9 inhibitors to statins in heterozygous FH was estimated to prevent 316,300 MACE at a cost of $503,000 per QALY gained compared with adding ezetimibe to statins (80% uncertainty interval [UI], $493,000-$1,737,000). In ASCVD, adding PCSK9 inhibitors to statins was estimated to prevent 4.3 million MACE compared with adding ezetimibe at $414,000 per QALY (80% UI, $277,000-$1,539,000). Reducing annual drug costs to $4536 per patient or less would be needed for PCSK9 inhibitors to be cost-effective at less than $100,000 per QALY. At 2015 prices, PCSK9 inhibitor use in all eligible patients was estimated to reduce cardiovascular care costs by $29 billion over 5 years, but drug costs increased by an estimated $592 billion (a 38% increase over 2015 prescription drug expenditures). In contrast, initiating statins in these high-risk populations in all statin-tolerant individuals who are not currently using statins was estimated to save $12 billion. CONCLUSIONS AND RELEVANCE: Assuming 2015 prices, PCSK9 inhibitor use in patients with heterozygous FH or ASCVD did not meet generally acceptable incremental cost-effectiveness thresholds and was estimated to increase US health care costs substantially. Reducing annual drug prices from more than $14,000 to $4536 would be necessary to meet a $100,000 per QALY threshold.