The National Institute for Health and Care Excellence says it plans to recommend the use of a drug to treat a very rare blood disorder which is expected to cost the NHS up to £58 million in its first year, rising to £82 million after five years.

In new draft guidance, NICE says it proposes to recommend the use of Alexion’s Soliris (eculizumab) in the treatment of atypical Haemolytic Uraemic Syndrome (aHUS), an extremely rare but life-threatening disease which causes inflammation of blood vessels and the formation of blood clots throughout the body.

The approximately 200 people in England who are affected by aHUS are at constant risk of sudden and progressive damage to and failure of their vital organs, especially the kidneys.

Soliris is being evaluated as part of a new programme at NICE which looks at highly-specialised technologies which may benefit people with very rare diseases at a high price. The drug is currently funded by NHS England through interim specialised commissioning arrangements.

Soliris radically improves the quality of life for people with aHUS, and it is a significant breakthrough in the management of the condition, said NICE chief executive Sir Andrew Dillon.

The drug is however, very expensive, and the draft guidance recommends that it be funded only if important conditions are met.  These include coordinating its use through an expert centre and putting place systems for monitoring how many people are diagnosed with aHUS and how many receive Soliris, at what dose and for how long. 

The programme also needs to develop protocols for starting and stopping treatment with Soliris for clinical reasons and to introduce a research programme to collect data to evaluate when stopping treatment or adjusting the dose of the drug might occur, says the draft guidance.

Also, given that the budget impact of Soliris will be considerable, it recommends that NHS England and Alexion should consider what opportunities might exist to reduce the drug’s overall cost to the NHS, NICE adds.

Commenting on the draft guidance, Alexion said that it supports the drug’s use consistent with the label approved by the European Medicines Agency (EMA), which specifically directs that treatment is recommended to continue for the patient’s lifetime, unless discontinuation is clinically indicated.

“We believe that it is important that NICE work within its remit and that decisions regarding continuation of eculizumab should be made by the treating physician based on best clinical judgement,” said the firm’s CEO, Leonard Bell.

The firm will be providing specific comments to NICE to address their conditions, Dr Bell added.

A consultation is now underway until September 25.