The UK National Institute for Health and Care Excellence (NICE) has issued a request to Alexion Pharmaceuticals for more information regarding its expensive drug Soliris (eculizumab). Soliris is a proven effective treatment for rare disease atypical hemolytic-uremic syndrome (aHUS). The UK watchdog posted draft guidance asking clarification for the high cost of treatment from Alexion, along with a request for advice from NHS England regarding budget considerations.
Sir Andrew Dillon, Chief Executive at NICE, commented, “Eculizumab is an effective treatment for aHUS. The NHS has already recognized this by making it available on an interim basis, pending the outcome of NICE's appraisal. 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 5 years, we need more information.” According to the FDA, Soliris would cost patients more than $800,000 per year and impact NHS’ specialized commissioning budget about $96 million in the first year.
aHUS is a chronic condition which causes severe inflammation of blood vessels and formation of blood clots throughout the body. aHUS can affect the kidneys, brain, and the heart. Around 70% of patients with aHUS are suspected of having underlying genetic or acquired abnormality of proteins in the immune system. The disease is estimated to affect over 200 people in England.
Eculizumab blocks pro-thrombotic and pro-inflammatory processes to inhibit the disease process in the body. NICE specifically asked Alexion for manufacturing and R&D costs regarding eculizumab. “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. It has also asked NHS England for clarification on treatment costs for a highly specialized technology in the context of a highly specialized service.”
NICE said that Alexion insisted information about overall cost of eculizumab be kept confidential, making it difficult for the institute to share details with stakeholders. “We're disappointed about this decision, for which we have not had an adequate explanation.” The institute has yet to issue final guidance for Soliris to the NHS. NICE will consider any information submitted by Alexion in response to its request at the next meeting of the Evaluation Committee in April.