Sickle cell: NICE approves voxelotor after initially rejecting it over uncertain evidence

Up to 4000 patients could be offered voxelotor (Oxbryta), a treatment for sickle cell disease which may reduce the need for blood transfusions, after the drug was recommended by the UK National Institute for Health and Care Excellence (NICE) in final draft guidance.1Voxelotor, a once daily pill, will now be available on the NHS in England for people aged 12 and older and for whom hydroxycarbamide alone is insufficient or where the patient cannot take hydroxycarbamide. NICE has said clinical trial data suggest the drug may improve anaemia compared with standard care, reduce the need for repeated blood transfusions, and improve self-reported quality of life.The approval comes after NICE initially rejected the drug in February, noting that “how well voxelotor works is uncertain” because the key clinical trial was short and the trial population did not represent the NHS patient population.2 Despite this, NICE had said that the “greater uncertainty…
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