Eculizumab for treating atypical haemolytic uraemic syndrome

Eculizumab, within its marketing authorisation, is recommended for funding for treating atypical haemolytic uraemic syndrome, only if all the following arrangements are in place:

• coordination of eculizumab use through an expert centre

• monitoring systems to record the number of people with a diagnosis of atypical haemolytic uraemic syndrome 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 programme with robust methods to evaluate when stopping treatment or dose adjustment might occur.

The long‑term budget impact of eculizumab for treating atypical haemolytic uraemic syndrome is uncertain but will be considerable. NHS England and the company (Alexion Pharma UK) should consider what opportunities might exist to reduce the cost of eculizumab to the NHS.