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One of the most expensive medications in the world is about to be covered by public drug plans in Canada. The alliance that negotiates group drug discounts on behalf of Ottawa and the provinces has reached a pricing deal with the maker of Strensiq.michaelquirk

One of the most expensive medications in the world is about to be covered by public drug plans in Canada.

The alliance that negotiates group drug discounts on behalf of Ottawa and the provinces has reached a pricing deal with the maker of Strensiq, an injectable treatment for a rare and potentially fatal soft-bone disease that affects fewer than 100 Canadians.

Strensiq, which can cost more than $1-million per patient per year, is the most expensive prescription medication ever evaluated by Canada's Common Drug Review, the body that advises taxpayer-funded drug plans on whether they should pay for new drugs.

The Common Drug Review's expert committee recommended that Strensiq only be funded for patients who start taking it before they turn 18, saying the evidence was not robust enough to support covering the drug for patients who begin treatment as adults.

The pan-Canadian Pharmaceutical Alliance (pCPA) followed that advice in its negotiations with the drug company and, as a result, adults with hypophosphatasia, or HPP, have been left out of the deal.

"There were very few adults in the clinical trials. They just really didn't feel they had enough evidence to include adults in that recommendation," said Debbie Taillefer, the president of Soft Bones Canada, an advocacy group for patients with HPP.

She was diagnosed with HPP at 33 and won't receive public funding for Strensiq.

"We're still hopeful," Ms. Taillefer said. "The door isn't closed [for adults.] They just need more evidence."

HPP is a rare genetic disorder in which bones fail to mineralize, leaving sufferers prone to fractures and a lifetime of chronic pain. In its most severe incarnation, it can be fatal for babies.

Strensiq, the first treatment to be approved for HPP, was invented by a Montreal biochemist about a decade ago.

In 2011, Alexion Pharmaceuticals Inc., a Connecticut-based company that specializes in drugs for rare diseases, bought the enzyme-replacement therapy.

The company brought Strensiq to market a few years later at a price so astronomical that Canada's public drug programs initially balked at covering it.

Talks between the pCPA and Alexion broke off for a time last year, but the parties ultimately came back to the table and signed a letter of intent last month. The company still has to ink formal agreements with each jurisdiction. Alexion has already reached a deal with one province, which it would not name.

"We are pleased that discussions with the pan-Canadian Pharmaceutical Alliance resumed and we were able to reach a sustainable agreement that is reflective of our collective responsibility to balance the need of patients, the control of health-care budgets, and recognition of the value that our therapy provides," said Emily Vlasak, a spokeswoman for Alexion, via e-mail.

The price the two sides settled on will be kept secret, a common practice when the pCPA makes deals with brand-name drug makers.

At least six Canadian adults with HPP, including a pair of Toronto-area sisters whose stories were featured in The Globe and Mail last year, will continue to have access to free Strensiq. The six took part in years of clinical trials that contributed to the approval of Strensiq, but lost access to the drug when the trials ended.

Alexion agreed to pay for their Strensiq after The Globe published its story.

Theresa and Rosanne Luckevich both began receiving free shipments of Strensiq late last summer, after going without their regular injections for about 15 months.

Both are feeling better, but neither is in perfect health.

"I've been on the medicine and I feel much better," said Theresa, a former nurse who lives in Brampton, Ont., north of Toronto. "I'm moving better. It'll never be 100 per cent, but I do feel a lot better."

In a separate case last year, Canada's federal drug-pricing regulator ruled that Alexion charged an "excessive" price in this country for another of its rare-disease drugs, Soliris. The Patented Medicine Prices Review Board ordered Alexion to lower the price of Soliris in Canada and pay millions in revenue it deemed excessive to the federal government.

Alexion denied wrongdoing and vowed to seek a judicial review of the ruling.

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