NICE has today issued final draft guidance recommending eculizumab (Soliris, Alexion) for funding for treating atypical Haemolytic Uraemic Syndrome (aHUS). The draft recommendation depends on the following arrangements being in place:

• coordination of eculizumab use through an expert centre

• monitoring systems to record the number of people with a diagnosis of aHUS and the number who have eculizumab, and the dose and duration of treatment

• a national protocol for starting and stopping eculizumab for clinical reasons

• a research programme with robust methods to evaluate when stopping treatment or dose adjustment might occur. 

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.

Eculizumab is being evaluated as part of a new programme at NICE that looks at highly specialised technologies which may benefit people with very rare diseases at a high price.

Commenting on the draft guidance, NICE Chief Executive Sir Andrew Dillon said: “The Committee accepted that eculizumab is a step change in the management of aHUS and can be considered a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.

“Eculizumab offers people with aHUS the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage.

For more details, go to: https://www.nice.org.uk/news/press-and-media/nice-draft-guidance-recommends-eculizumab-soliris-for-treating-very-rare-life-threatening-blood-disorder