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The Globe and Mail

Brief relief promised for six Canadians with rare soft-bone disease

Theresa Luckevich, 62, a participant in a clinical trial for the drug Strensiq is now waiting for a free supply of the expensive medicine which helps control her hypophosphatasia condition.

Michelle Siu/The Globe and Mail

The maker of one of the world's most expensive drugs has agreed to provide at least three months' worth of free medicine to six Canadians who took part in a clinical trial, a decision that comes as the company and provincial governments prepare to resume pricing negotiations that could lead to wider access to Strensiq, a treatment for a rare soft-bone disease.

Among the patients who will receive more Strensiq are Theresa and Rosanne Luckevich, a pair of Ontario sisters who lost access to the injectable drug a year ago after the Winnipeg-based trial in which they had participated for seven years wrapped up.

"I'm infinitely grateful. It's a life changer," said Rosanne, a 61-year-old sculptor who lives in Toronto. "It's the difference between being an invalid and just living your life like everybody else."

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The Luckevich sisters, whose story appeared in The Globe and Mail last month, were both diagnosed as infants with hypophosphatasia (HPP,) a rare genetic disorder in which bones fail to mineralize, leaving sufferers prone to fractures and chronic pain. In its most severe incarnation, HPP can be fatal for babies.

Strensiq is the most expensive product that Canada's Common Drug Review – which advises government drug plans on whether to cover new medications – has ever evaluated, and the drug's price is laying bare the challenge that public and private insurers face as more astonishingly expensive treatments for rare diseases hit the market.

HPP, which is believed to affect about 100 people in this country, had no treatment until a Montreal biochemist invented a successful enzyme-replacement therapy for the disease a little more than a decade ago.

Connecticut-based Alexion Pharmaceuticals Inc. bought the drug in 2011, secured regulatory approval for it, then set a sticker price so high that Canada's public drug plans balked at paying for it – more than $1-million a year in some cases, depending on a patient's weight.

Neither Rosanne nor Theresa, a 62-year-old former nurse who lives northwest of Toronto in Brampton, could afford to pay out-of-pocket for Strensiq after their last shipments of trial medication ran out last May.

Both have seen their heath decline since then, with fresh leg fractures renewing the chronic pain they endured before starting on Strensiq.

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Rosanne feels worse today than she did before starting the trial. "Everything in the [art] studio, all the work I do, I feel like it takes me four times longer now than when I was on the medication," she said. "It's real acute pain. It's not just a mild ache."

Rosanne is one of eight siblings, four of whom have HPP. One of her brothers, Michael Luckevich, who lives in Seattle, stopped taking Strensiq in protest about a month ago after his U.S. insurer told him the half-dose on which he had started came with a sticker price of U.S. $1,048,320 a year.

Mr. Luckevich, 58, said Tuesday that his pain has grown worse since he stopped treatment, but that he has no intention of ordering another shipment until Alexion agrees to lower the price for patients like his sisters.

Back in Canada, the Luckevich sisters could not turn for financial help to the government program that helps patients with catastrophic annual prescription-drug bills because Ontario, like every other province and territory, is refusing to cover Strensiq unless Alexion can seal a deal with the pan-Canadian Pharmaceutical Alliance (pCPA,) which negotiates confidential group discounts on behalf of public drug plans.

The pCPA walked away from negotiations at the beginning of this year, but a spokeswoman for Alexion said the company has been told verbally that the alliance is now prepared to come back to the table.

"The pCPA indicated [last week] that we should be hearing from them within the week of their intention to resume the negotiations," said Emily Vlasak, Alexion's associate director of corporate communications. "We look forward to that opportunity." (A spokesman for Alberta Health, which is co-leading the Strensiq talks on behalf of the pCPA, would say only that, "any further communications on next steps will flow directly from pCPA to the company.")

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In the meantime, Ms. Vlasak said that the trial participants should have had access to Strensiq through the company's post-trial program all along.

Cheryl Rockman-Greenberg, the physician who ran the Winnipeg trial, said company officials told her last year that participants would be limited to a two-month supply of the drug, hardly sufficient for patients who need it for life.

As The Globe was reporting on the Luckevich family's story, Dr. Rockman-Greenberg reapplied on their behalf for post-trial access.

Ms. Vlasak said that if, at the end of three months, a re-evaluation shows the six former trial participants are deriving a "clinical benefit" from the drug, they'll receive another three-months' supply.

She cautioned, however, that Alexion can't provide "blanket approval for endless free therapy."

Alexion's hope is that it can reach a deal with the pCPA that would lead to provincial governments picking up the cost of Strensiq for most HPP patients.

For her part, Theresa Luckevich said she will remain skeptical of Alexion's promise until she receives the drug. A date for the first shipment has not yet been set.

"I'm glad it's going to happen, but I'm like Doubting Thomas," she said. "I'll believe it when it happens."

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