Karol E. Watson, MD, PhD, FACC reviewing Kazi DS et al. JAMA 2016 Aug 16.
In a simulation model, using these cholesterol-lowering drugs in their target populations greatly increased net costs — but we don't yet have seminal data on patient outcomes.
Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors can greatly lower LDL-cholesterol levels, beyond the effects of statin therapy, in patients with heterozygous familial hypercholesterolemia (FH) or atherosclerotic cardiovascular disease (ASCVD). However, the annual U.S. drug cost is currently about $14,350 per patient. Researchers used a simulation model of all U.S. adults (age range, 35–94 years) to assess the cost-effectiveness of PCSK9-inhibitor therapy in patients with heterozygous FH or ASCVD who were taking a statin.
In patients with heterozygous FH, adding a PCSK9 inhibitor to statin therapy was estimated to prevent 316,300 lifetime major adverse cardiovascular events (MACE; cardiovascular death, nonfatal myocardial infarction, or stroke), compared with adding ezetimibe to statins, at an estimated cost of $503,000 per quality-adjusted life-year (QALY); the parallel estimated figures for patients with ASCVD were prevention of 4.3 million MACE events at a cost of $414,000 per QALY. At 2015 prices, PCSK9-inhibitor use in these high-risk populations would reduce cardiovascular-care costs by an estimated $29 billion within 5 years but would increase drug costs by an estimated $592 billion (a net increase of $565 billion). The analysis showed that annual per-patient drug costs for PCSK9 inhibitors would need to fall to $4,536 or less (vs. the current $14,350) to meet the incremental cost-effectiveness threshold of <$100,000 per QALY. In contrast, for these high-risk populations, initiating statins in all statin-tolerant patients who do not currently take them would save a net $12 billion.
CITATION(S):
Kazi DS et al. Cost-effectiveness of PCSK9 inhibitor therapy in patients with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease. JAMA 2016 Aug 16; 316:743.
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