Format

Send to

Choose Destination
JAMA. 2016 Aug 16;316(7):743-53. doi: 10.1001/jama.2016.11004.

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

Author information

1
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 Francisco4Center.
2
Division of General Internal Medicine, Columbia University Medical Center, New York, New York7College of Physicians and Surgeons, Columbia University, New York, New York.
3
Department of Medicine, Center for Vulnerable Populations, University of California, San Francisco2Department of Medicine, University of California, San Francisco8Division of General Internal Medicine, Zuckerberg San Francisco General Hospital, San Franci.
4
Department of Medicine, Center for Vulnerable Populations, University of California, San Francisco2Department of Medicine, University of California, San Francisco.
5
Institute for Clinical and Economic Review, Boston, Massachusetts.
6
Department of Medicine, University of California, San Francisco.
7
Department of Medicine, Center for Vulnerable Populations, University of California, San Francisco.
8
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.

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.

PMID:
27533159
DOI:
10.1001/jama.2016.11004
[Indexed for MEDLINE]

Supplemental Content

Full text links

Icon for Silverchair Information Systems
Loading ...
Support Center