The National Institute for Health and Care Excellence (NICE) has recommended eculizumab (Soliris) for treating a rare life-threatening blood disorder called atypical Haemolytic Uraemic Syndrome (aHUS).
aHUS is a life-threatening disease affecting around 200 people in England, with 20-30 new patients diagnosed with the condition each year. It causes inflammation of blood vessels and the formation of blood clots throughout the body. People with aHUS are at constant risk of sudden and progressive damage to, and failure of vital organs, particularly the kidneys.
NICE Chief Executive Sir Andrew Dillon said: “aHUS is a very distressing condition that imposes a significant burden on those with the condition and their carers and families. We are therefore pleased to be able to recommend eculizumab for funding.”
“The Committee accepted that eculizumab is a step change in the management of aHUS and can be considered a significant innovation for a disease with a high unmet clinical need. It offers people with aHUS the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.”
Nevertheless, Mr Dillon said the drug is very expensive: “The Committee felt that the budget impact of eculizumab would be lower if the potential for adjusting the dose of the drug and stopping treatment was explored.
“This is reflected in the guidance which recommends eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for clinical reasons. In the meantime NHS England and the company should consider what opportunities might exist to reduce the cost of eculizumab to the NHS.”