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Reign Johnston.

family handout/Courtesy of the family

Alberta has rejected an application to fund treatment for a toddler with a rare disease, leaving a young family hoping an angel donor will foot the bill for an injection that costs millions of dollars.

Alex Johnston, 20, the mother of two-year-old Reign Johnston, said two Alberta officials called her last week to inform her that the province would not pay for her son to receive Zolgensma, a gene therapy designed to slow the progression of spinal muscular atrophy, or SMA. Alberta, at the end of January, said it would review funding for Zolgensma on a case-by-case basis.

SMA is a rare childhood disease that can lead to rapid muscle deterioration and loss of breathing function. Alberta received two applications requesting coverage for Zolgensma – one child received funding and treatment before Ms. Johnston heard back from government officials regarding Reign’s status. Government officials, according to Ms. Johnston, said Reign was denied funding because he needs mechanical assistance to breathe and uses a feeding tube.

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“The ‘no’ isn’t shocking anymore. It is just defeating,” Ms. Johnston said. “It is just so heartbreaking.”

Novartis, which makes Zolgensma, said it is neither privy to the discussions nor the rationale for provincial funding decisions. Asked whether Zolgensma is dangerous or ineffective for children needing mechanical assistance to breathe and feed, Novartis spokeswoman Julie Schneiderman pointed to the drug’s monograph. It notes SMA results in progressive and irreversible damage to motor neurons, and early treatment translates into greater potential benefit.

Further, the “benefits and risks have not been established in patients with profound muscle weakness and respiratory failure, patients on permanent ventilation, and patients unable to swallow,” the monograph states.

Alberta intends to fund Zolgensma for every patient for whom it is “clinically appropriate,” according to spokeswoman Karin Campbell.

“The evaluation includes assessing the latest scientific evidence, current recommendations for the treatment’s use and data that shows whether a patient will benefit from this particular treatment,” she said in a statement.

“As an example – and this applies to many treatments, not just Zogensma – sometimes data will show that when a disease has reached an advanced stage, a particular treatment does not provide any additional clinical improvement over current therapies. In addition, the evidence on the benefits and risks of a new therapy may only be available for a specific group of patients.”

Canada approved Zolgensma late last year and Alberta and Ontario said they would consider funding treatment case by case while negotiating with the drug maker on long-term costs.

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The Canadian Agency for Drugs and Technologies in Health (CADTH), which makes non-binding recommendations regarding whether provinces and territories should pay for certain treatments, released Zolgensma guidelines in a document dated March 26. It recommended funding Zolgensma treatments if a number of conditions are met, including that the prospective patient be 180 days old or younger and “not currently requiring permanent feeding or ventilatory support.”

Reign would not meet these non-binding criteria. But neither would Max Sych, who has Type 2 SMA. He turned 2 in January and Alberta approved funding for his Zolgensma treatment shortly afterward. He received the infusion March 16, according to an Instagram account documenting his life.

CADTH noted its recommendations were based on studies of children younger than six months old when they started treatment, and therefore effectiveness of Zolgensma in patients older than six months is “unknown based on the currently available evidence.”

Moreover, CADTH recommended covering costs only if Novartis drops the price tag. CADTH noted Zolgensma treatment kits cost $2.91-million. This one-time cost would have to drop by more than 90 per cent in order for Zolgensma to be considered cost-effective compared to best supportive care, according to CADTH.

Novartis’s Ms. Schneiderman noted best supportive care, in this case, equates to no treatment. She argued Zolgensma costs less than the 10-year cost of current SMA therapy.

“As a one-time gene therapy for a rare disease, Zolgensma is priced according to the value it provides to patients, caregivers, the health care system and to society as a whole,” she said in a statement.

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Reign was diagnosed with Type 1 SMA when he was five months old. Alberta officials, Ms. Johnston said, told her Reign could still receive the drug – but that the government wouldn’t pay for it. She said they are now hoping an angel donor (she said the donor family does not want to be identified) will cover the cost of Zolgensma for Reign. They are also considering other ways to fundraise.

“We are going to try until we can’t,” Ms. Johnston said.

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