Ontario expands coverage for compassionate drug access
TheStar.com – healthzone.ca/health/features – Star gets action
January 21, 2011. Tanya Talaga, Queen’s Park Bureau
A dying man’s fight to gain access to a prohibitively expensive, life-saving medication has moved the government to improve drug access for all those in Ontario suffering from rare disorders.
The province will now consider requests to cover drugs in cases where a patient with a rare disease has been urgently hospitalized due to an immediate life-, limb- or organ-threatening condition.
Diane McArthur, the executive officer of the Ontario Public Drug Programs, will consider requests to cover drugs in these cases.
The story of Lucas Maciesza, a Mount Forest man who nearly died before gaining access to the $500,000-a-year drug treatment, was the subject of a series of Star stories last year.
The series focused on why some patients living in one part of the province can access life-saving medication, while others in different parts of the province cannot.
The government paid for the cost of Soliris for a North Bay woman on compassionate grounds, but did not pay for Maciesza’s.
The provincial government will now pick up the cost of Maciesza’s treatment.
The government receives about 250 applications each year for compassionate access to prohibitively expensive drugs for rare disorders.
The policy change is meant to equalize every patient’s chance, regardless of where they live, to access the medication they need.
“This is cool, oh my goodness, I’m grinning ear to ear,” said Maciesza.
The 26-year-old has paroxysmal nocturnal hemoglobinuria (PNH), an extremely rare blood disorder. Before he started Soliris, his kidneys were failing, he was bleeding internally and he was at risk of stroke.
His family could not afford Soliris and has been in a battle for months with the ministry of health to secure coverage.
However, London Health Sciences Centre stepped in and began to pay for the drug, deciding weekly if it could afford it or not, out of its budget.
Since receiving the intravenous drug, Maciesza has had a remarkable turnaround, but he and his family have lived in fear that each week would be his last on the drug due to cost.
Just this past Wednesday, Lucas was driving to the hospital for his infusion, but then had to go home when officials called to say “they weren’t going to cover it,” said his father, Rick Maciesza.
“If this disease doesn’t take Lucas, I think the stress of it — the disease and the funding problems — will take all of us,” Rick Maciesza said.
Then, on Thursday, Lucas’s physician, Dr. Ian Chin-Yee, called to say he had secured hospital funding for another week.
The policy change means there will be interim coverage of the drug for a period of six months once the patient leaves hospital and the province is in negotiations to cover the medication with the manufacturer.
McArthur is in negotiations with the U.S.-based manufacturer, Alexion, in a bid to secure a better price.
The drug company has previously said the drug costs so much because developing medication for such a rare disease comes at a great cost to pharmaceutical firms.
“In the last four or five weeks, I have been better and better each day,” Maciesza said. “I have drive, motivation now. I feel great, healthy. It’s going to be a good year.”
Chin-Yee, chief of hematology and professor of medicine at the University of Western Ontario, has spearheaded a movement to get Soliris covered by the province.
“In Lucas’s case, they are basically providing it for him,” he said. “The bigger problem is, do we have to come in with life-and-limb threatening (conditions) before patients get it?”
Chin-Yee fears without universal coverage, the ministry of health may suddenly believe a patient is no longer in a life-and-limb threatening state and then they could pull funding.
“It is a step forward,” he said, and cautioned the government is not saying the drug is available for all PNH patients.
But this is a “poor response” to the problem, said Progressive Conservative MPP Christine Elliott (Whitby-Oshawa). People must be urgently hospitalized and the requested drug needs to be tied to it.
“Many people with rare disorders need drugs, but they aren’t hospitalized,” she said. “This is a really, really narrow opportunity that might help one or two people, but not a real benefit for the many.”
The Liberals are just trying to get rid of the problem, she said. “It is an off-the-cuff, let’s-get-this-problem- off-our-back solution and it won’t offer the help it says it will,” she said.
Barry Katsof, founder of the Canadian Association of PNH patients, agreed it isn’t time to celebrate yet.
“You basically have to be on death’s door in the hospital before you’ll get this,” Katsof said from Montreal. He has been on Soliris for 2 1/2 years. Quebec covers the drug.
The problem here is PNH patients may suffer kidney or liver failure before Ontario decides whether or not it will cover the drug.
“They are pushing you to death’s door. They are playing Russian roulette here,” he said. “There are PNH patients who need the drug now, but they aren’t in the hospital.”
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