Intended for healthcare professionals


Company withholds lifesaving drug in row with Australian government over its use

BMJ 2014; 349 doi: (Published 26 September 2014) Cite this as: BMJ 2014;349:g5869
  1. Amy Coopes
  1. 1Sydney

A lifesaving drug treatment for the ultra-rare but fatal blood disorder atypical haemolytic uraemic syndrome (aHUS) is at the centre of an unprecedented funding stand-off in Australia that has left patients in limbo and is being closely watched abroad, experts have said.

The global drug firm Alexion last week walked away from the Australian government’s offer of A$63m (£33.9m; €43.4m; US$55.1m) in funding over five years at full price (A$500 000 a patient each year) to subsidise its humanised monoclonal antibody, eculizumab (Soliris), under the country’s Pharmaceutical Benefits Scheme. Alexion objected to the recommended terms of use, under which patients would be taken off the drug if the markers of their condition returned to normal; the company wanted patients to be put on the drug for life.

The condition of aHUS is caused by chronic, uncontrolled activation of the alternative complement pathway and results …

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