NICE final draft guidance recommends Soliris, but saying it is 'very expensive'

UK health costs watchdog the National Institute for Health and Care Excellence (NICE) has today issued final draft guidance recommending US drugmaker Alexion Pharmaceuticals’ (Nasdaq: ALXN) Soliris (eculizumab) for funding for treating atypical hemolytic uremic syndrome (aHUS) under the National Health Service in England as the first and only treatment for patients with aHUS.

The draft recommendation depends on the following arrangements being in place: • coordination of eculizumab use through an expert center • monitoring systems to record the number of people with a diagnosis of aHUS and the number who have eculizumab, and the dose and duration of treatment • a national protocol for starting and stopping eculizumab for clinical reasons • a research program with robust methods to evaluate when stopping treatment or dose adjustment might occur.

Eculizumab is being evaluated as part of a new programme at the NICE that looks at highly specialised technologies which may benefit people with very rare diseases at a high price. Eculizumab costs £3,150 ($4,939) per 30ml vial in the UK, according to the NICE.