Company withholds lifesaving drug in row with Australian government over its useBMJ 2014; 349 doi: https://doi.org/10.1136/bmj.g5869 (Published 26 September 2014) Cite this as: BMJ 2014;349:g5869
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 condition of aHUS is caused by chronic, uncontrolled activation of the alternative complement pathway and results in thrombotic microangiopathy—which, if untreated, leads to organ failure, predominantly of the kidneys. Its aetiology is diffuse, with mutations in multiple factors implicated, and eculizumab is currently the only available treatment.1
Eculizumab was approved for treatment of aHUS by the US Food and Drug Administration and the European Medicines Agency in 2011 and by the Australian Therapeutic Goods Administration in 2012, but arguments over the prescribed length of treatment have stymied a local funding deal, said Sue Hill, chair of the independent Pharmaceutical Benefits Advisory Committee (PBAC). “In 22 years in the pharmaceutical sector I’ve never seen anything like this,” said Hill.
On 18 September the health minister, Peter Dutton, announced cabinet approval for the Pharmaceutical Benefits Scheme’s listing of eculizumab under the restricted terms recommended by PBAC. The committee has recommended case reviews of patients at six and 12 months, with a view to closely supervised and managed withdrawal if patient markers have returned to normal and are stable.2
In what Hill described as an “unprecedented” move, Alexion has refused to agree the funding offer for the drug and is seeking further talks with the government, arguing that the treatment window—which it recommends should be lifelong—ought to be at clinicians’ discretion.
David Kwasha, Australasia managing director at Alexion, said, “We believe that this approach raises significant safety concerns for patients, who would be put at constant risk for life threatening complications since aHUS is a genetic disease and sudden and catastrophic symptoms can occur in affected patients without warning at any time.” The company has committed to ongoing provision of eculizumab on compassionate grounds to 11 aHUS patients in Australia, but until a funding agreement is finalised it will not guarantee the supply to new patients.
The president of the aHUS advocacy group, Kerri Grey—whose son Ashley, 6, is among the 11 patients—described the delays as “unconscionable.” She said, “Both parties talk about putting patient lives at risk, and that’s why they are fighting.” Every day that patients were forced to await access to Soliris, their lives were at risk, she added.
Hill said that PBAC was unable to agree to a recommendation of lifelong treatment because the underlying mutations of the disease were diverse and little had been established about the natural history of the condition or its response to eculizumab.3 The drug’s effectiveness in treating aHUS had been measured only through single arm open label and observational studies, with no randomised clinical trial to date,4 she said.
“My assessment is that, given that this is the only product that Alexion has, they’re trying to ensure maximum return by ensuring that people stay on it for as long as possible,” said Hill.
She added that a broader principle was at stake for Alexion, as funding and supply negotiations were also under way in Canada and the United Kingdom, where the National Institute for Health and Care Excellence had flagged similar limitations to those contemplated by Australia.5 “Although Australia is probably a relatively small market in the scheme of things, I think [that] from the point of view of potentially influencing other countries they want to make sure everyone toes the party line, which is long term supply,” she said.
Dutton has held firm, saying that the government “has accepted and will adhere to” PBAC’s recommendations on eculizumab and “deals with the vast majority of companies in a way that provides positive outcomes.”
Cite this as: BMJ 2014;349:g5869