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30 Sep 2014

Almost 100 patients suffering a rare and potential deadly blood disease will start receiving treatment after a major drug company dropped its objections to the terms of its subsidised supply through the Pharmaceutical Benefits Scheme.

About 70 Australians afflicted with atypical Haemolytic Uraemic Syndrome (aHUS) will get subsidised access to the life-saving but very expensive medicine Soliris after manufacturer Alexion Pharmaceuticals agreed to conditions set by the  powerful Pharmaceutical Benefits Advisory Committee (PBAC) for its supply through the PBS.

In a major backdown, Alexion said it would supply the drug even though it did not agree with the PBAC criteria.

Soliris costs $500,000 to treat a single patient for year, and Federal Cabinet has set aside $63 million over the next four years to subsidise access to to the drug, which has been formulated to treat aHUS – which can cause blood clots to form in small blood vessels throughout the body, potentially leading to stroke, heart attack, kidney failure and death.

About 35 people, including children, are diagnosed with the disease every year, and Soliris has been hailed as a breakthrough treatment that not only can control symptoms and the severity of attacks, but can restore critical organ function and lead to remission in some patients.

But until now the treatment’s prohibitively high cost  has put it out of the reach of most sufferers.

In approving the listing of Soliris on the PBS, Cabinet followed the advice of the PBAC that although Soliris demonstrated significant clinical benefits in the short-term, there was little evidence to support its sustained use in patients who had experienced remission.

Following a meeting in late August, the PBAC recommended that Soliris be supplied through the PBS, but that therapy be discontinued for patients in remission at 12 months – subject to ongoing monitoring and an immediate resumption of treatment at any sign of a relapse.

“In reaching this conclusion the PBAC noted, among other matters, that the vast majority of the benefit observed in patients receiving occurs in the first six months of treatment,” the Committee said in a statement.

Health Minister Peter Dutton said the criteria set by the PBAC for commencing treatment with Soliris, its termination and possible recommencement, reflected “principles of good medical practise”.

But the drug firm Alexion, which makes Soliris, had voiced strong objections to supplying the drug on the terms set by the Government.

Managing Director of Alexion’s Australian subsidiary, David Kwasha, told PharmaDispatch in early September the company could not accept what it considered to be “inhumane treatment conditions”.

“It’s dangerous, clinically inappropriate, and goes against dose administration guidelines,” Mr Kwasha said. “We all agree that this needs to be resolved quickly, we have this one issue, but we just can’t agree to the experiment being proposed by the PBAC.”

He said the nature of the condition meant that, even though the PBAC criteria allowed for the reintroduction of therapy where there is a relapse, “for some patients it could be too late”.

The stand-off threatened to delay patient access to the life-saving drug, and PBAC Chair Associate Professor Suzanne Hill accused Alexion of causing unnecessary alarm by grossly misrepresenting the PBAC’s recommendation.

“Let me be clear about what the PBAC recommended,” she told Medical Observer. “We have not recommended that all patients must stop receiving treatment after 12 months.”

Mr Kwasha told The Saturday Telegraph that "we still disagree that patients should come off it after 12 months".

But the company has caved into mouting pressure after Health Minister Peter Dutton sought to put the onus for any delay in the supply of Soliris on to Alexion.

“The Government has acted upon the PBAC’s expert advice and, as a result, aHUS patients should be aware that the Government is ready to make this treatment available to them and is awaiting the response of the drug sponsor Alexion to the independent PBAC’s recommendation for the listing of Soliris,” the Minister said.

Adrian Rollins

Published: 30 Sep 2014