Eculizumab, dubbed “the world’s most expensive drug”, and described as “a step change in the management of atypical haemolytic uraemic syndrome (aHUS)”, is being made available on the NHS in England.
But the National Institute for Health and Care Excellence (NICE) has attached conditions to the use of eculizumab (Soliris), which costs £340,200 per patient per year, according to guidance produced as part of NICE’s highly specialised technologies programme.
NICE estimates that costs to the NHS in the first year of use could be £57.8m and would increase as more patients are diagnosed.
NICE has linked the approval to several conditions, including that the drug should be made available through an expert centre and that the number of people diagnosed with aHUS and treated with eculizumab are monitored. It also wants a national clinical protocol for starting and stopping treatment, as well as research into stopping treatment and possible dose adjustments.
“The long-term budget impact of eculizumab for treating aHUS is uncertain but will be considerable,” the guidance states. “NHS England and Alexion Pharma should consider what opportunities might exist to reduce the cost to the NHS.”
NICE chief executive Andrew Dillon says the committee accepted that the drug is a “step change in the management of aHUS” and was a “significant innovation for a disease with a high unmet clinical need”.
“The drug is, however, very expensive,” he adds. “The budget impact of eculizumab would be lower if the potential for adjusting the dose of the drug and stopping treatment was explored.”