Alexion Pharma’s ultra-rare drug Soliris has not been backed by British ministers, even though it received a positive endorsement by a medical advisory board.

The firm’s drug, which is licensed to treat the exceptionally rare atypical haemolytic uraemic syndrome (aHUS), costs $400,000 (£253,761) per patient, per year in the US, and has similar costs in Europe - it is currently the most expensive single treatment in the world.

Because of the rarity of the disease this drug treats – and the high costs involved – a specialised agency called the Advisory Group for National Specialised Services has considered the treatment, rather than NICE.

They issued a positive recommendation for the drug, but its advice has not been backed by ministers for the British Parliament, who have taken the unusual decision of overruling AGNSS.

The Advisory Group said in statement: “We concluded that [Soliris] would help save lives and improve the quality of life for children (among whom the condition is particularly prevalent) and adults with atypical haemolytic uraemic syndrome.

“[But] ministers have decided not to recommend national commissioning of [Soliris] for the treatment of atypical haemolytic uraemic syndrome at this stage. The drug will be referred to NICE for further review.”

From April, NICE will be able to assess these types of high cost, low volume medicines regularly instead of the AGNSS, under reforms from the government. 

But Alexion is not happy with this change or the ministers' decision, and said it was “gravely disappointed” by the announcement, and the “underlying departure by the UK Government from an agreed formal process”. 

aHUS is an ultra-rare, life-threatening, chronic, genetic disease that progressively damages vital organs, often leading to sudden and significant damage to the kidney, brain, heart, and other vital organs, and premature death.

More than half of patients have kidney failure, require dialysis or die within one year of diagnosis. In November 2011, the European Commission approved Soliris as the first and only treatment for children and adults with aHUS. 

Alexion said “it is committed to working with the health ministers to make Soliris available to children and adults as quickly as possible”, and adds that it is calling upon the ministers to accept the recommendation from the AGNSS.

More work needed on rarer drugs

Meanwhile, patients with rare life threatening conditions in the UK are being urged to join forces and call for more access to clinical trials and developing medicines, following a Parliamentary debate on the topic this week.

Health minister Anna Soubry responded to an adjournment debate in Westminster Hall led by Cotswold MP Geoffrey Clifton-Brown, who has taken the issue on following the launch of a campaign by one of his constituents, who is suffering from motor neurone disease

Les Halpin, aged 55, from Gloucestershire, said he was “shocked” when he found that the only drug approved for the illness was the same as was prescribed to his friend who had the condition 20 years ago.

A statistician by training, Les established the campaign ‘Empower: Access to Medicine’ last year to put the patient front and centre in calling for easier and quicker access to treatments for rare terminal illnesses and to get involved with the development of new drugs.

The Westminster debate discussed how individuals can be given greater freedom to undergo trials, which would reduce the current average time of ten years it takes to bring any new drug to market.

Anna Soubry said that the debate “was Parliament at its very best,” adding that the issue had “cross-party support” and the government’s priority was “to have NHS patients receive the most appropriate treatment for their condition”.

In the debate, Geoffrey Clifton-Brown specifically asked ministers to consider the following solutions:

•    Abolish strict liability in the area of drug development and replace it with liability based on negligence

•    Revise the definition of negligence so that, in deciding whether it was negligent or not to seek to develop a new drug, account is taken of the consequences of doing nothing as well as the consequences of trying to do something

•    Change the law on waivers so that any patient who is prepared to try a new medicine, together with the risk that it may have unknown side effects, is at liberty to do so

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