NICE draft guidance recommends Alexion's Soliris for very rare life-threatening blood disorder

A drug to treat a very rare blood disorder affecting around 200 people in England has been recommended by the UK drugs watch dog the National Institute for Health and Care Excellence (NICE) in further draft guidance.

Produced by US drugmaker Alexion Pharmaceuticals’ (Nasdaq: ALXN), Solaris (eculizumab) treats atypical hemolytic uremic syndrome (aHUS), an extremely rare but life-threatening disease which 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 program at the NICE that looks at highly specialised technologies which may benefit people with very rare diseases at a high price.

Treatment cost of £340,200 per adult patient

The NICE estimates that this drug will cost the National Health Service up to £58million ($96.3 million) - based on a treatment cost of £340,200 per adult patient - in the first year, rising to £82million after five years. Solaris is currently funded by NHS England through interim specialised commissioning arrangements.

Commenting on the draft guidance, NICE chief executive Sir Andrew Dillon said: “Eculizumab radically improves the quality of life of the small number of people with aHUS. Until it became available, people with aHUS were at risk of kidney failure needing dialysis, other organ failure, and early death. Plasma therapy and dialysis were the main treatments, both of which had a limited impact on managing symptoms and reducing early deaths, but a substantial negative effect on a patient’s quality of life.”

Sir Andrew continued: “From the available evidence and from the testimony of clinicians and patients, families and carers, it is clear that eculizumab is a significant breakthrough in the management of aHUS. The drug offers people with the disease the possibility of avoiding end-stage renal failure, dialysis and kidney transplantation, as well as other organ damage. The drug is, however, very expensive. In making its decision the independent Evaluation Committee needed to take into account the total fixed budget for highly specialised services as a whole and how it is allocated.”

“The Committee felt that the budget impact of recommending eculizumab for aHUS in relation to the substantial benefits it offers would be lower if the potential for dose adjustment and stopping treatment was taken into account. Therefore the draft guidance recommends that eculizumab is funded only if important conditions are met. These include coordinating the use of eculizumab through an expert centre and putting in place systems for monitoring how many people are diagnosed with aHUS, how many receive the drug, at what dose and for how long. The program also needs to develop protocols for starting and stopping treatment with eculizumab for clinical reasons and introduce a research programme to collect data to evaluate when stopping treatment or adjusting the dose of the drug might occur,” he stated.

Given that the budget impact of eculizumab for treating aHUS will be considerable, the draft guidance also recommends that NHS England and the company should consider what opportunities might exist to reduce the overall cost of eculizumab to the NHS.

Alexion will address NICE conditions