In the first draft guidance produced by its Highly Specialised Technologies programme, the National Institute for Health and Care Excellence (NICE) has asked Alexion Pharma UK why its rare blood disorder drug treatment Soliris (eculizumab) costs so much.
Soliris is used in the treatment of atypical Haemolytic Uraemic Syndrome (aHUS), a chronic condition that causes severe inflammation of blood vessels and the formation of blood clots in small blood vessels throughout the body. The prognosis for people with aHUS - estimated at over 200 in England - is poor, with early mortality rates of 10%-15% in the initial, acute phase of the disease, and the majority - up to 70% - developing end-stage kidney failure requiring dialysis and transplantation.
Soliris is an effective treatment for aHUS, and the NHS has already recognised this by making it available on an interim basis pending the outcome of NICE’s appraisal, says the Institute’s chief executive, Sir Andrew Dillon.
“However, 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. 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,” he adds.
Sir Andrew also notes that Alexion has insisted that its information about the overall cost of the drug be kept confidential, so NICE is unable to share these details of the firm’s submission with stakeholders.
“We’re disappointed about this decision, for which we have not had an adequate explanation,” he says.
The Institute is also asking NHS England for advice on what considerations relating to the management of its specialised commissioning budget it should take into account in formulating a recommendation.
Commenting, Alexion says that NICE’s advisory committee has concluded so far that Soliris - the first ultra-orphan drug to be reviewed under the Institute’s new HST process - produced substantial Quality-Adjusted Life Year (QALY) gains “of a magnitude rarely seen for a new drug treatment - let alone for an ultra-rare disorder.”
However, the firm expresses its concern that, nearly three years after the government commenced its evaluation of Soliris, and more than a year after the drug was referred to NICE, the advisory committee is still unable to provide a formal recommendation whether it should be nationally commissioned.
Alexion says that NICE has asked it to provide additional information related to the budget impact of Soliris, but has not requested any further information regarding its clinical effectiveness.
• Based on a treatment cost of £340,200 per adult patient in the first year, and assuming a patient cohort of 170, NICE estimates that Soliris would cost the NHS £57.8 million in the first year, rising to £82 million in year five.
NICE’s preliminary recommendations are available for public consultation until midday on March 25. The information sought from Alexion and NHS England will be considered at the next meeting of NICE’s evaluation committee, in April.