B.C. cuts drug treatment for 9-year-old girl with rare disease
Posted June 18, 2025 12:07 pm.
Last Updated June 18, 2025 5:52 pm.
The Ministry of Health says it is discontinuing the coverage of a drug treatment for a nine-year-old B.C. girl who has a rare disease.
It comes after months of reviews by the ministry, advisory committees, expert recommendations, and advocacy from the child’s parents and treating physicians.
CLICK HERE TO LISTEN TO 1130 NEWSRADIO VANCOUVER LIVE!The drug, Brinuera, is one of the only treatment methods for Ceroid Lipofuscinosis Type 2 (CLN2), otherwise known as Batten disease. While it is not a life-saving drug, it aims to slow the progression of the disease.
The Ministry explained on Wednesday that Brineura has clear criteria for initiation, continuation, and discontinuation, and the child has deteriorated to the point where there is no clinical evidence that continuing treatment would have further benefits. The cost of the drug is not a factor when considering whether to cover treatments, the Ministry asserted.
The child’s mother, Jori Fales, has been advocating for her daughter’s treatment for years after Charleigh was diagnosed with Batten disease in 2019. The nine-year-old is the only person in the province diagnosed with the condition, with around a dozen reported cases in the entire country.
Batten disease usually presents between the ages of two and four, and by about six years old, patients lose their vision, require the use of wheelchairs, and are fed via gastrostomy tubes.
In a social media post just five days ago, Fales explained that the report, which contributed to the Ministry’s final decision, had been released by the Canadian Drug Expert Committee at Canada’s Drug Agency.
Fales explained that the CDA review “highlighted that there isn’t any solid evidence to inform when Brineura should be stopped, specifically that there is ‘insufficient data to establish discontinuation criteria.’ Without appropriate data to help inform stopping the drug, discontinuation should truly be a clinical decision made with the health care team and the families impacted by this disease.”
“The report also says that ’emerging evidence that suggests a potential benefit of cerliponase alfa (Brineura) on quality of life, seizure control, and mortality outcomes.'”
Fales says that in her daughter’s case, she “knows it to be true.”
“We’ve gone from almost a hundred seizures a day down to none. We see the improved quality of life, we see how much this drug has slowed her disease progression.”
Fales explained that the report noted that “Clinical experts identified improved quality of life, the mitigation of disease progression, and patient safety as key outcomes in treating people with CLN2 disease. Caregivers shared these priorities and emphasized additional outcomes, including patients’ comfort and happiness.”
“We are seeing all of these outcomes for Charleigh, and our medical team agrees that continuing with therapy is in Charleigh’s best interest. Anything less is simply cruel and wrong,” Fales explained on Instagram.
But the Ministry shared on Wednesday that the CDA found insufficient evidence to make definitive conclusions about the potential benefits to patients’ quality of life, seizure control, and mortality outcomes. The CDA works with science and the best clinical advice from clinicians, the Ministry stated.
The Ministry added that the review found the recommendations from its 2019 review of the drug remain consistent, despite Fales’ call for an update to the eligibility criteria.
Minister Josie Osborne says Charleigh’s file is one of the most challenging her ministry has faced.
She says, given the extraordinary circumstances of the case, the ministry ensured “no stone was left unturned” in determining whether continuing treatment could still have benefits but says the evidence did not support continuing.
“As Minister, my job is to make sure that BC’s drug review processes are rigorous, independent and based on evidence. Notably, all other jurisdictions that provide coverage for Brineura have the same clinical criteria that BC uses for when to discontinue treatment – beyond which there is no evidence of benefit. No matter how challenging and difficult it can be, we must follow the evidence and the recommendations of medical experts, who have clinical experience with rare diseases like this,” Osborne explained.
“I know this is not what Charleigh’s family wanted to hear. It is not what any single one of us wanted to hear. Our hearts go out to Charleigh, her family – and all patient and families who are suffering from and impacted by the devastating toll of a rare and incurable disease.”
Since 2007, she says the Ministry has never provided ongoing coverage for a drug against the recommendations of clinical experts and the criteria established by Canada’s Drug Agency.
Osborne says politicians shouldn’t be making decisions about an individual patient’s care.
“This is ethical and pharmaceutical experts who apply their knowledge using the criteria that have been established, making recommendations to the Ministry of Health around the coverage of a drug like this. Again, no parents, no family, ever wants to be in this situation, and yet we must apply these frameworks fairly across patients and families in British Columbia that are experiencing situations like this,” she said.
“I want her to know that British Columbia is thinking about her and that we are all wishing the very best and the very best care that she’ll look at for the rest of her life.”
Describing the decision as “heartbreaking,” Fales confirmed in a statement to 1130 NewsRadio that Charleigh’s last publicly-funded treatment is on June 19.
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With files from Srushti Gangdev.
