The National Institute for Health and Care Excellence has taken a u-turn on its decision to bar NHS patients from access to Amgen’s cholesterol buster Repatha, the world’s first approved PCSK9 inhibitor.
European regulators approved the drug last year for patients with uncontrolled cholesterol, after clinical data showed that it cut low-density lipoprotein-cholesterol (LDL-C) by some 55%-75% versus a placebo, and by around 35% to 45% compared with ezetimibe (Merck & Co’s Zetia).
But the cost regulator sparked outrage when it issued draft guidelines rejecting Repatha (evolocumab) for NHS patients with high cholesterol and mixed dyslipidaemia, because of a lack of clinical trials measuring its direct effect on cardiovascular events and uncertainty over whether the drug would actually reduce angina, heart attacks and strokes.
However, in a revised appraisal consultation document, NICE is now backing Repatha, alone or in combination with other lipid-lowering therapies, as an option in various scenarios, but only at a dosage of 140mg every two weeks and as long as the company continues to offer discount agreed in the patient access scheme.
The Institute is supporting use of Repatha in patients with primary non-familial hypercholesterolaemia or mixed dyslipidaemia with progressive cardiovascular disease (CVD), who also have persistently high LDL-C concentrations despite maximum lipid-lowering therapy and can’t take statin therapy.
Patients with primary heterozygous-familial hypercholesterolaemia or mixed dyslipidaemia with progressive, symptomatic CVD and persistently high LDL-C concentrations despite maximum lipid-lowering therapy also stand to gain access to the drug, as do those with severe primary heterozygous-familial hypercholesterolaemia or mixed dyslipidaemia without CVD, if they continue to have high levels of cholesterol despite maximum lipid-lowering therapy and can’t take statins.
Amgen has welcomed NICE’s decision. “NICE has reviewed the technical responses to its initial appraisal, listened to patients and healthcare professionals and proposed a recommendation for the use of Repatha by the NHS in England and Wales,” said Tony Patrikios, Executive Medical Director, Amgen UK & Ireland.
“However, beyond these draft recommendations, another priority will be patients with existing cardiovascular disease who fail to reduce their LDL-C to a satisfactory level despite the use of statins and who remain at high risk. For them, access to new treatment options is critically important to help avoid a heart attack or stroke,” he stressed.
More than seven million people in the UK take statins daily to help them lower LDL cholesterol in the blood, but a significant number report side effects such as joint pain, fatigue, and nausea and, according to Amgen, in up to 60% of cases statins alone are not enough to control high cholesterol.
Repatha’s annual cost per patient comes in at around £4,448.60 for 140mg every two weeks, and £6123.60 for 420 mg monthly.