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Queen’s Park is expanding public coverage for one of the world’s most expensive medications, a move that has buoyed patients in Ontario but devastated those outside the province who cannot get the drug because of where they live.

The Ontario government’s decision to pay for Spinraza for additional children and teens with a neuromuscular disorder that affects fewer than 500 Canadians came as a federal advisory council unveiled a blueprint for a national, single-payer public pharmacare program on Wednesday.

Among the council’s 60 recommendations was the creation of a strategy for expensive drugs for rare diseases that would ensure coverage does not differ wildly from place to place – which is exactly what has happened in the case of Spinraza.

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“It’s akin to being told that my child’s life is not worth it,” said Anne Bélanger, whose 13-year-old son, Miles, is ineligible for public coverage of Spinraza in British Columbia.

Through tears, Ms. Bélanger described how heartbreaking it was to know that if Miles lived in Saskatchewan, Quebec or – now – Ontario, he could have access to the only drug approved to treat his degenerative disease, spinal muscular atrophy (SMA).

“I think it’s unethical and immoral,” she said. “It’s based on postal code. That’s it. I just live in the wrong postal code.”

The Spinraza case highlights the unique challenges posed by rare-disease drugs, which now account for nearly half of all newly launched medications, according to the Patented Medicine Prices Review Board, Canada’s drug-pricing regulator.

Spinraza’s average list price of $708,000 for the first year of treatment and $354,000 for every year thereafter is so high that public and private insurers initially balked at paying it. (The real price has dropped because of confidential discount deals, but no party will reveal by how much.)

A drug’s day of decision: Patients with a rare disorder will learn soon whether funding is coming for a pricey but promising remedy

An expert panel at the Canadian Agency for Drugs and Technologies in Health (CADTH), the organization that advises the provinces and territories on covering new drugs, recommended in March that Spinraza be covered only for SMA patients who were 12 years old or younger who had never walked. The panel concluded the clinical-trial evidence was strongest for that group.

CADTH’s advice is not binding, so Saskatchewan and Ontario decided to broaden their eligibility criteria after patients and their parents lobbied intensely for wider access to Spinraza.

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In Ontario’s case, the government raised the eligibility age to 18 for patients with SMA who had never walked, and said that other patients, including adults, could apply for individual approvals with the support of their doctors.

Eric Hoskins, the former Ontario health minister who chaired the advisory council on pharmacare, said on Wednesday that uneven coverage of expensive drugs for rare diseases is a major flaw of Canada’s existing patchwork of private and public drug plans.

His report recommended that a new Canadian drug agency establish a separate pathway for making reimbursement decisions on rare-disease drugs, including gathering “structured real-world evidence” that could help inform those decisions.

Conducting traditional randomized, placebo-controlled clinical trials on drugs for rare diseases can be difficult because of the small number of patients.

Given that, the advisory council also suggested negotiating pay-for-performance deals on rare-disease drugs for which the scientific evidence is uncertain. If the drug doesn’t work well, its maker would be paid less.

Patients, meanwhile, would be told upfront that if an expensive new drug does not demonstrably improve their health, they would be taken off it.

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“I think this idea of structuring pay-for-performance contracts and evaluating with greater rigour the real-world evidence that Canada could collect is prudent. It’s a good idea," said Steve Morgan, a health economist at the University of British Columbia and a long-time supporter of national pharmacare.

For Jan Genge of Guelph, Ont., Ontario’s decision to broaden coverage for Spinraza means that her son, Burton, 15, will likely get the drug soon. It’s an immense relief after months of fighting for access and years of caring for Burton without hope of a treatment.

As Ms. Genge helped Burton into bed on Wednesday night, he was playing a song out loud on his phone, something he doesn’t usually do.

“He was playing, by Queen, We are the Champions,” Ms. Genge said, laughing. “Isn’t that wonderful?”

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