28 January 2015

UK DAILY MAIL: NHS to pay for SOLIRIS (USA Alexion Pharm.) for atypical Haemolytic Uraemic Syndrome @ GBP 340,000/year.

A drug costing up to £9million per patient is to be made available on the NHS - making it the health service’s most expensive medicine.
Eculizumab - also known as Soliris - will be offered to about 200 people suffering from a rare kidney condition, the treatments watchdog confirmed last night.
The National Institute for Health Care and Excellence (NICE) said the drug – which will cost the NHS up to £82million a year – would allow patients to live independently for decades.


Sir Andrew Dillon, left, of the National Institute for Health Care and Excellence (NICE) said he was pleased to have authorised the new wonder drug Soliris, also known as Eculizumab to treat a rare kidney condition
A NICE spokeswoman confirmed last night that the drug would be the most expensive funded by the NHS.
The drug will treat atypical Haemolytic Uraemic Syndrome (aHUS) - a life-threatening disease affecting around hundreds of people in England.
It causes inflammation of blood vessels and the formation of blood clots throughout the body.
There are only around 200 sufferers, with 20 to 30 new patients with the condition diagnosed each year.
Patients are constantly at risk of sudden damage and failure of their vital organs, particularly the kidneys.
NICE said the drug – which will be funded on an interim basis - was a ‘significant innovation’ and offered ‘gains of a magnitude that is rarely seen for any new drug treatment’.
Its chief executive Sir Andrew Dillon conceded the drug would prove to ‘very expensive’ and recommended NHS England to find ways of reducing the cost.
The decision to provide Eculizumab, developed and marketed by US firm Alexion Pharmaceuticals, was only approved on the basis of ultra-rare conditions, rather than being assessed on its standard value-for-money formula.
The health watchdog said that it was unfair to apply the usual rules because it was such a rare condition – meaning drug companies had to recoup their research costs from only a handful of patients.
With the drug offering an extra 25 years of good-quality life, NICE deemed it worth he annual £340,000 cost per patient to the NHS.
But the decision will come as a blow to cancer patients after a quarter of treatments that can offer a few extra months of life were stopped on cost grounds this month.
Sir Andrew Dillon said: ‘aHUS is a very distressing condition that imposes a significant burden both on those with the condition and their carers and families. We are therefore pleased to be able to recommend Eculizumab for funding.
‘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.’
He added: ‘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.
‘This is reflected in the guidance which recommends eculizumab should be funded only if important conditions are met, including the development of rules for starting and stopping treatment for clinical reasons.
In the meantime NHS England and the company should consider what opportunities might exist to reduce the cost of eculizumab to the NHS.’

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