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High tierRandomized Controlled TrialCitation verified

Evolocumab and Clinical Outcomes in Patients with Cardiovascular Disease

Marc S Sabatine, Robert P Giugliano, Anthony C Keech, Terje R Pedersen, FOURIER Steering Committee and Investigators - New England Journal of Medicine, 2017

Evolocumab, a PCSK9 inhibitor, lowered LDL by 59% (to a median of 30 mg/dL) on top of statins and reduced the primary cardiovascular composite (HR 0.85). It is strong evidence that even very low LDL is safe and beneficial - but it is also a key skeptic exhibit: the absolute benefit was modest, and the short median follow-up (2.2 years) was too brief to test whether halving LDL extends life.

Key findings

Effect measures

  • Hazard Ratio: 0.85 (primary composite)95% CI 0.79-0.92
  • Absolute Risk Reduction: 1.5 percentage points (9.8% vs 11.3%)

Why this evidence tier (High)

Risk of bias:
Very large double-blind randomized placebo-controlled trial.
Precision:
Large event count; precise estimate.
Directness:
Hard clinical composite; tests a third LDL-lowering mechanism (PCSK9 inhibition).
Consistency:
Event reduction concordant with statin/ezetimibe evidence; the short follow-up leaves the mortality question open.
Funding / COI:
Manufacturer-funded (Amgen); several authors were Amgen employees.

High certainty that PCSK9-driven LDL lowering reduces events safely at very low LDL; the short follow-up means the trial could not test a mortality effect, a genuine limitation skeptics rightly emphasise.

Population:
27,564 patients with established atherosclerotic cardiovascular disease and LDL >=70 mg/dL already on statins, randomized to the PCSK9 inhibitor evolocumab vs placebo; median follow-up 2.2 years.
Conflicts of interest:
Industry-funded by Amgen, which manufactures evolocumab. Several co-authors were Amgen employees.
Funding:
Amgen (manufacturer of evolocumab); NCT01764633.

Limitations

  • Short median follow-up (2.2 years), too brief to test a mortality effect; absolute benefit modest.
  • Manufacturer-funded.

How this study is used