The National Institute for Health and Care Excellence has recommended eculizumab (Soliris) for treating a rare life-threatening blood disorder called atypical Haemolytic Uraemic Syndrome (aHUS).
The guidance is the first to be produced as part of NICE’s highly specialised technologies programme to evaluate treatments for very rare conditions.
aHUS is a life-threatening disease affecting around 200 people in England, with 20-30 new diagnoses each year. It causes inflammation of blood vessels and the formation of blood clots throughout the body, putting patients at constant risk of damage to vital organs, particularly the kidneys.
Eculizumab, manufactured by Alexion, offers people with aHUS the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.
It is given intravenously in adults as initial treatment at a dose of 900mg for four weeks, then as maintenance treatment at a dose of 1200mg on week five and then every 12-16 days.
It costs £3,150 per 30 ml vial (excluding VAT). NICE’s estimates that the drug will cost the NHS up to £58m in the first year, rising to £82m after five years.
It is currently funded by NHS England through interim specialised commissioning arrangements.
NICE chief executive Sir Andrew Dillon said: “aHUS is a very distressing condition that imposes a significant burden both on those with the condition and their carers and families. We are therefore pleased to be able to recommend eculizumab for funding” said.
He said the drug represented a “step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need”. “The drug is, however, very expensive,” he added.
He said NICE felt the budget impact of eculizumab for the NHS would be lower if the potential for dose adjustment or stopping treatment altogether where possible “was explored” in future by the manufacturer.
At present the drug’s product notes suggest it should continue for the patient’s “lifetime”.