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Reign Johnston, now just shy of 2 years of age, was diagnosed as a newborn with Type 1 spinal muscular atrophy, a rare neurological disease that affects one in 11,000 handout/Courtesy of the family

Reign Johnston is a happy baby who enjoys painting as much as playing with toys. His green eyes light up when his mother plays the clarinet. Even when he is in his stroller – a BiPAP machine hooked to his nose, a gastrostomy feeding tube attached in his stomach – he flashes a warm smile at staring passersby.

As a newborn, Reign was diagnosed with Type 1 spinal muscular atrophy, or SMA – a rare neurological disease that affects one out of 11,000 babies, often leading to devastatingly rapid muscle deterioration and loss of breathing function. When he was five months old, Reign went into cardiac arrest in the middle of a restaurant. He stopped breathing for five minutes before first responders resuscitated him. Reign was confined to the hospital’s intensive care unit for almost seven months after the restaurant episode.

“We were told by doctors and nurses numerous times in the hospital that we can unplug him,” says Reign’s mother, Alex Johnston, a resident of Edmonton. “No one is going to judge you,” assured the hospital staff, she says.

Now, at 22 months, he has endured multiple surgeries and procedures. He is under 24-hour care. But fighting to keep Reign alive and healthy was an easy decision for Ms. Johnston and her fiancé Ryan Fengstad, Reign’s father.

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Reign is injected quarterly with Spiranza, which slows down the progression of the degenerative disease. His injections cost an estimated $450,000 a handout/Handout

But the battle has seemed impossible to win, even as an innovative treatment received federal approval this week. Now that the drug, Zolgensma, is approved for use in Canada, provincial governments will still need to decide whether to cover the staggering $2.8-million price tag for a single dose. They’ll also need to decide who would be eligible for the treatment before SMA patients such as Reign have any hope to access it.

“I just don’t understand how a one-time dose of a drug can cost $3-million,” says Ms. Johnston, who is 19 years old and cares for Reign full-time. “I understand that there is research that goes into it and everything. But, the whole point of doing all that research is to help people, right?”

Without the proper treatment, babies diagnosed with type-1 SMA have a life expectancy of around two years. Currently, Reign is injected quarterly with Spinraza, which slows down the progression of the degenerative disease. His injections cost an estimated $450,000 a year, and as of last March, are funded by provincial drug plans.

Zolgensma is a one-time infusion that replaces the missing or defective survival motor neuron 1 (SMN1) gene, potentially reversing, though not curing, the progression of the disease. The U.S. Food and Drug Administration approved the drug, which is made by Swiss-based pharmaceutical company Novartis, in May, 2019, after more than half of the 36 patients enrolled in the two clinical trials gained the strength to sit on their own.

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Zolgensma, made by Novartis, is a one-time infusion that replaces the missing or defective survival motor neuron 1 (SMN1) gene, potentially reversing, though not curing, the progression of the disease. A single dose is all that is required, but costs $ handout

Zolgensma is believed to be the world’s most expensive drug. In January of this year, the company started a lottery to provide as many as 100 doses of drug free of charge in countries where it is not yet approved.

David Whitrap, a spokesman for the Institute for Clinical and Economic Review (ICER), a non-profit research organization in Boston that evaluates the cost-effectiveness of drugs, said the benefits of the treatment should outweigh the cost.

“Despite Zolgensma’s eye-popping price tag, ICER found it to be cost-effective due to how well it measurably improves and extends the lives of patients,” says Mr. Whitrap. “But a curative treatment for an always fatal disease is precisely the kind of swing-for-the-fences innovation our society should be willing to reward handsomely.”

Mr. Whitrap points out Zolgensma is a one-time treatment, meaning the $2.8-million price tag is the total cost over a patient’s lifetime. Treatments for cystic fibrosis, for example, cost approximately $400,000 a year.

Zolgensma’s application to Health Canada, which evaluates the efficacy, safety and quality of drugs, was granted Priority Review Status in June, which shortened the timeline to 180 days from the standard 300 days.

Now that it’s approved, the Common Drug Review board of the Canadian Agency for Drugs and Technologies in Health (CADTH), a non-profit organization which advises provincial governments, will issue its recommendation as early as January, leaving it to the provinces to make decisions about funding – a final step that can take six to seven months.

That is time that Reign and many other babies do not have. FDA regulations do not allow Zolgensma to be administered for babies over two years of age, and a similar deadline is expected in Canada. Reign turns two on March 7 of next year.

The Government of Canada is working with provincial and territorial governments as part of the pan-Canadian Pharmaceutical Alliance (pCPA), according to Geoffroy Legault-Thivierge, a spokesperson at Health Canada. The alliance leverages the governments’ buying power to negotiate lower prices on brand name drugs while setting the price point for many generic drugs.

Alberta Health Minister Tyler Shandro wasn’t available for an interview. In a statement, he said the province is pushing to have Zolgensma funded early next year. The statement said the province has been working to get an interim funding agreement in place through the pCPA for children who turn two, even before the drug is fully approved and funded.

“If the provinces agree, then they can collectively get together and bargain with the company over what the exact price is going to be,” said Dr. Joel Lexchin, a board member of Canadian Doctors for Medicare and the professor emeritus of health policy and management at York University.

Kerry Diotte is one of several MPs and Health Ministers that Ms. Johnston and her family have reached out to in hopes of expediting the process through a Clinical Trial or Health Canada’s Special Access Program.

“Common sense seems to say if this treatment is available elsewhere, why are we, as Canadians, behind the times here?” says Mr. Diotte, a Conservative MP for Edmonton-Griesbach. Mr. Diotte, along with other local MPs such as Matt Jeneroux and Heather McPherson, have written letters on behalf of Reign to health ministers and critics in Alberta.

“It’s such a scary situation. The clock is literally ticking on that child’s life,” he said.

In British Columbia, Zolgensma is being considered for possible coverage under the province’s PharmaCare program. For now, the province is not assisting with funding. However, in the case of Lucy Van Doormaal, a Vancouver-based newborn with SMA, the B.C. government is allowing the drug to be imported and administered at the B.C. Children’s Hospital, a first in the province.

Lucy’s family raised nearly $2.5-million over the summer through an aggressive and clever social media campaign, which included a professionally done video, plus global press including CNN. But the child won Novartis’s lottery to receive the treatment for free. Her family plans to use the money raised to support other SMA patients – including Reign eventually – as well as continuing care for Lucy.

The Novartis lottery, formally called a “managed access program,” selects patients at random as long as the child meets the criteria. Ms. Johnston said that because Reign wore a BiPAP for more than 16 hours a day, his application was rejected as ineligible.

Novartis spokeswoman Julie Schneiderman declined to comment on the family’s case, citing patient privacy.

Shortly after Reign was diagnosed with SMA, the family moved to Edmonton from Leduc in order to be close to his doctors. Their south Edmonton apartment is a 15-minute drive to Stollery Children’s Hospital, and directly beside a fire station – just in case they need to call 911. Both Mr. Fengstad and Ms. Johnston’s parents are within 20 minutes to provide help.

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Shortly after Reign was diagnosed with SMA, the family relocated to Edmonton from Leduc, Alta., in order to be close to his doctorsfamily handout

Every morning at 8, Mr. Fengstad leaves for his job as a door-to-door salesman for Sealand Quality Foods and returns home late. As the primary caretaker, Ms. Johnston balances Reign’s needs with raising awareness through social media and the news media.

Ms. Johnston and her family have conducted walkathons, online auctions, sold Reign’s art, and started an Instagram page for Reign. As of December 17, Reign’s GoFundMe page has raised just $131,123, far short of their multi-million dollar goal and just over two months to go.

“I feel like I need to fight hard now or else I’d blame myself in the end,” said Ms. Johnston.

“I would pay it off for the rest of my life if that was an option,” she said. “We simply don’t have the savings to cover a $2.8-million drug. I mean, who does?”

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