Drug firm ‘used patient as pawn’

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Health fears … Toula Lockley heart was unable to pump blood when she was denied crucial treatment this month. Picture: Supplied.

A DRUG company denied a critically ill woman access to a life-saving drug this month because it wanted to ramp up pressure on Health Minister Peter Dutton to subsidise its $500,000 per patient per year medicine.

Mr Dutton had to intervene to pay for the medicine Soliris to save the woman’s life.

The furious Health Minister told News Corp: “I won’t tolerate patients being used as pawns”.

“My job is to take care of patients and the few outlier companies that conduct themselves in that manner had better think again.”

Melbourne woman Toula Lockley, 42, suffers from a rare disease called aHUS that sees tiny blood vessels blocked, cutting off the blood supply to major organs.

Around 70 patients in Australia suffer from the disease and without Soliris 80 per cent of patients die or develop end stage organ failure within three years.

Toula’s heart was affected and was unable to pump blood.

Desperate ... Toula Lockley’s situation was “do or die no question”, her doctor says. Picture: Supplied
Desperate … Toula Lockley’s situation was “do or die no question”, her doctor says. Picture: Supplied Source:Supplied

Her doctor Annabel Tuckfield described her patients’ situation as “do or die no question”.

“She came into the emergency department blue, ready to arrest,” Dr Tuckfield told News Corp Australia.

The hospital provided Toula with an emergency dose of the medicine on September 6 and her doctor then began pleading with the drug company and the Health Minister to make more doses available to keep her alive.

Renal specialist Professor Jeff Szer said the manager of the Australian arm of the drug company Alexion, David Kwasha, spent 12 hours trying to make some doses of the drug available but was rebuffed by his head office in the US.

Mr Dutton had to step in on September 13 to pay for a second dose.

Disgrace ... the Health Minister had to intervene to pay for the medicine Soliris to save Toula Lockley’s life. Picture: Supplied.
Disgrace … the Health Minister had to intervene to pay for the medicine Soliris to save Toula Lockley’s life. Picture: Supplied.

Mr Kwasha told News Corp the company had provided the drug on compassionate ground to 11 Australian patients but his US head office had made it clear no further compassionate doses would not be made available until the government reached an agreement with the company to subsidise the drug.

The government’s expert advisory committee approved the drug for subsidy in March and the government last week announced it would provide $63 million to fund it.

However, the drug company is refusing to accept conditions placed on the funding that would see patients who recover taken off the drug after 12 months.

Clinicians have told News Corp Australia they were not happy with the original restrictions the government wanted to place on access to the drug fearing for patients who might not get access quickly again if they were taken off after 12 months.

Outrage ... Health Minister Peter Dutton had to step in on September 13 to pay for Toula’s second dose.
Outrage … Health Minister Peter Dutton had to step in on September 13 to pay for Toula’s second dose. Source:News Corp Australia

Late Friday the government’s expert Pharmaceutical Benefits Advisory Committee issued a statement clarifying the conditions it wants to place on funding for the drug.

“We have NOT recommended that all patients must stop receiving treatment after 12 months,” PBAC chairwoman Professor Sue Hill said.

“If a patient is responding well but does not have the disease fully under control, they will continue to receive subsidised treatment as long as is needed to maintain their health and as long as they continue to improve,” she said in a statement.

Patients who are in remission at 12 months will be taken off the drug and their condition will be continually monitored and if they have any sign of relapse, “even just an abnormal blood test” then the PBAC has recommended they be given the medicine again.

There is not enough evidence available at present on how long patients need to use the treatment and because it is so costly the PBAC wants the company to gather more evidence as the medicine is in use to refine the guidelines on its use.

Anger ... Health Minister Peter Dutton says he will not let patients be used as pawns.
Anger … Health Minister Peter Dutton says he will not let patients be used as pawns. Source:News Corp Australia

Mr Kwasha said Alexion was urgently seeking a meeting with the government this week after the PBAC’s clarification on funding conditions.

“The public statement looks more like what we are asking for,” he said.

“It seems like there could be a resolution,” he said.

Professor Szer who has been involved in the negotiations on the funding for the medicine has described the process as “a bit like children fighting in a schoolyard”.

He says clinicians “can live within the boundaries” of the PBAC’s latest conditions.

In its latest statement on the funding negotiations the company said last week it would immediately provide Soliris to patients with aHUS if the government “agrees to allow physicians to determine their patients medical needs as recommended by regulatory authorities in Australia and Europe”.

“In contrast, Government repeatedly imposes inhumane treatment conditions on aHUS patients.”

Doctors describe the effects of the drug as incredible with patients who’ve used it rapidly moving from a situation where they are on dialysis with swelling of the brain and at deaths door to being well enough to go back to work and play sport.

The company is negotiating subsidies with Canada and the UK at present and it is believed it does not want Australia’s funding conditions to set a precedent for those nations.

The case throws a spotlight on the tough tactics used by drug companies trying to force governments to pay exorbitant prices for new medicines.

Negotiations over another high cost $300,000 per patient per year medicine for cystic fibrosis called Kalydeco have reached a stalemate because the company won’t accept restrictions the government wants to place on funding.