Alexion will have to make a new case for the high cost of its ‘ultra-orphan’ drug Soliris before it can be recommended for use on the NHS in England and Wales to treat an extremely rare blood condition.
The National Institute for Health and Care Excellence (NICE), which assesses the cost-effectiveness of drugs for NHS use, issued draft guidance demanding more information from Alexion to explain the price tag of £340,200 per adult patient per year when used to treat atypical haemolytic uraemic syndrome (aHUS).
At current disease levels the total NHS spend on Soliris (eculizumab) would be £58m in the first year, rising to £80m over five years at predicted diagnosis rates.
“Our independent advisory committee has therefore asked for clarification from the company on aspects of the manufacturing, research and development costs of a medicinal product for the treatment of a very rare condition,” said NICE’s chief executive Sir Andrew Dillon.
Soliris is the first drug to be assessed by NICE through its new highly specialised technologies programme, which is designed to cover drugs for very rare conditions as decided in consultation with the Department of Health.
Alexion’s treatment fits into this category as aHUS – which involves the inflammation of blood vessel – is thought to only affect 170 people in the UK, with disease rates rising by 20 patients each year.
Despite these limited numbers, the individual impact of the disease is significant, leading to blood clots, heart failure and brain injury.
Mortality rates stand at 10 to15 per cent in the initial phase of the disease, while up to 70 per cent of patients need a kidney transplant or dialyses despite current standard therapy of plasma treatment.
NICE confirmed the benefits of Soliris for this group of patients and said that Alexion just needs to provide information about the budget impact rather than the drug’s clinical effectiveness.
As Soliris is the first drug to be assessed as a highly specialised technology uncertainties remain about pricing expectations, and NICE’s Sir Andrew confirmed the organisation has also asked national commissioning body NHS England for clarification on treatment costs for these medicines.
“Drugs for very rare conditions that affect just a few people in the country are inevitably more expensive than for more common diseases,” noted Sir Andrew.
In its response, Alexion said it was “pleased” that NICE has reaffirmed the effectiveness of Soliris, although the company was less positive about the time frame for a decision.
“Alexion is concerned that nearly three years after Government commenced its evaluation, and more than one year after eculizumab was referred to NICE, the Committee was still unable to provide a formal recommendation whether eculizumab should be nationally commissioned,” it said in a statement.
There will be further consultation until March 25, after which NICE will produce renewed guidance, and until a final decision is made Soliris will be available on NHS England for aHUS patients through interim commissioning arrangements.
