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Alexion ‘concerned’ over NICE draft

Published on 04/03/14 at 08:04am
Soliris image

Alexion Pharmaceuticals UK has expressed ‘concern’ that NICE has not yet allowed its rare disease treatment Soliris to be formally approved for NHS use.

Soliris (eculizumab) is the first 'ultra-orphan' drug to be reviewed by NICE under its new Highly Specialised Technologies (HST) process, which is expected to look at just three drugs per year.

As part of a complex picture, the watchdog has asked the company for more information on the manufacturing, research and development costs of the drug, which is a treatment for blood disorder atypical Haemolytic Uraemic Syndrome (aHUS).

“Before we can make a confident recommendation for routine use, which we estimate would cost the NHS about £58 million in the first year, rising to over £80 million in five years, we need more information,” NICE chief executive Sir Andrew Dillon explained.

Around 200 people in England suffer from the chronic condition, which causes severe inflammation and clots in blood vessels throughout the body - and which can lead to kidney impairment, heart failure and brain injury.

In around 70% of patients, aHUS is associated with a genetic abnormality of immune system proteins called complement.

NICE’s draft guidance, which comes shortly after Rare Disease Day, also asks NHS England what considerations with regard to its commissioning budget should be taken into account in formulating a recommendation.

There is no question that NICE thinks the drug works as an effective treatment for aHUS - and it has made it available on an interim basis, pending the outcome of this appraisal.

Soliris acts to block pro-thrombotic and pro-inflammatory processes which in aHUS can lead to cellular damage in small blood vessels, renal failure and damage to other organs.

“The committee has so far concluded that eculizumab produced substantial QALY gains of a magnitude rarely seen for a new drug treatment,” Alexion said in a statement.

The company’s main issue is that, nearly three years after the government commenced its evaluation, and a year after Soliris was referred to NICE, there is still no formal recommendation.

“We are concerned that there hasn’t been a decision,” Jon Beauchamp, vice president, medical affairs EMEA at Alexion, told Pharmafile.

AGNSS

The now-defunct Advisory Group for National Specialised Services (AGNSS) - whose role in cost-assessing low volume, high cost ultra-orphan drugs NICE took over - has already recommended Soliris, as has NHS England.

However, Beauchamp concedes that the company is happy with NICE’s clinical appraisal of the product and reaffirmed the company’s desire to work with the watchdog.

But cost remains the key stumbling block. Sir Andrew said: “Alexion insisted that its information about the overall cost of eculizumab be kept confidential and so NICE is unable to share these details of the Alexion submission with stakeholders. We’re disappointed about this decision, for which we have not had an adequate explanation.”

A spokesman added: “In our technology appraisals programme costs are always provided, unless the drug is being provided under a patient access scheme.” NICE will look at any new data at the next meeting of the Evaluation Committee in April.

The prognosis for people with aHUS is poor, with early mortality rates ranging from 10% to 15% in the initial, acute phase of the disease: up to 70% of sufferers develop end stage kidney failure requiring dialysis and transplantation.

Alastair Kent, director of Genetic Alliance UK, said: “We are […] pleased to see consultation with NHS England on the wider specialised commissioning landscape and the considerations that may be necessary resulting from NICE guidance.”

He believes this dialogue “should be ongoing throughout all HST evaluations to ensure a more consistent approach to management of the NHS England specialised commissioning budget”.

Soliris is also approved in the US, European Union and Japan to treat another ultra-rare disease, paroxysmal nocturnal hemoglobinuria (PNH), characterised by the destruction of red blood cells.

Adam Hill

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