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NICE backs first ‘ultra-orphan’ drug Soliris

Final guidance backs Alexion’s treatment for very rare blood clot condition
Solaris eculizumab Alexion

Alexion's Soliris has become the first drug to pass through a new UK assessment to determine the cost-effectiveness of medicines for very rare conditions.

The National Institute for Health and Care Excellence (NICE) declared Soliris (eculizumab) a cost-effective use of NHS resources to be available to treat the rare blood clot condition atypical haemolytic uraemic syndrome (aHUS) in England and Wales despite reservations about the drug's high price.

The guidance is the first to be issued as part of NICE's highly specialised technologies programme, designed to evaluate treatments for very rare conditions for which regular NICE assessment process are inappropriate.

aHUS qualifies as it is thought to affect just 200 people in England. As well as being rare the disease is also very serious, causing inflammation of the blood vessels and formation of blood clots throughout the body. This can sometimes lead to organ failure, with the kidneys most at risk.

Despite acknowledging the effectiveness of the drug NICE had previously balked at its high price tag, which would mean that total treatment would cost the NHS around £58m in the first year of recommendation, rising to £82m after five years.

These concerns have now been somewhat overcome, although cost still remains an issue and NICE has added some provisions that must be adhered to for the NHS to reimburse Soliris.

For instance treatment with the drug must be coordinated through an expert centre and monitoring systems must be put in place to record the number of people with a diagnosis of aHUS and the number who have eculizumab.

There should also be a national protocol for starting and stopping eculizumab for clinical reasons 
and a research programme to evaluate when stopping treatment or dose adjustment might occur.

NICE chief executive Sir Andrew Dillon commented: “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.

“The drug is, however, 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.”

Nevertheless the recommendation is something of a breakthrough for Alexion, whose business unit leader for aHUS Jon Beauchamp spoke to PMLiVE at the end of last year about NICE's new process for ultra-orphan drugs such as Soliris.

“It's certainly been a very protracted process, but it's important for patients - and for companies investing - to have certainty about how to get access,” he said.

“It's great that there is finally an NHS highly-specialised process and it has methods that are different from common medicines. But that process could still be a bit quicker - we were the first to go through it and certainly the process evolved as we went through it.”

Article by
Kirstie Pickering

29th January 2015

From: Sales



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