“I’m feeling lost as to what to expect in the next few days — whether I should expect a reversal or nothing,” said Charleigh Pollock’s mother.
The parents of a 10-year-old Langford girl who lost coverage of a drug to slow the progression of her terminal disease are hoping for a reprieve, after meeting with the province’s health minister on Friday.
“I’m feeling lost as to what to expect in the next few days — whether I should expect a reversal or nothing,” Jori Fales, mother of Charleigh Pollock, said Monday. “I’m in no man’s land right now.”
For six years, Charleigh has taken the drug Brineura, which costs almost $1 million a year, to slow the progression of Batten disease (CLN2), a fatal neurodegenerative disorder.
But on June 18, Health Minister Josie Osborne announced the province would uphold a recommendation from Canada’s Drug Agency to discontinue coverage of the drug, saying the case had met the discontinuation criteria created in 2019.
Coverage decisions under B.C.’s Expensive Drugs for Rare Disease program are based on the advice of a committee of independent experts using clinical criteria established by Canada’s Drug Agency.
On Friday evening, Charleigh’s parents met with Osborne, along with neuroscientist and Batten disease expert Dr. Ineka Whiteman, Canadian Batten Disease Support and Research Association president Lori Brown and Andrew McFadyen of the Isaac Foundation, who helped the government bring Brineura to B.C.
It was Osborne’s first meeting with the family since she called for a review of the province’s original decision in February to discontinue coverage.
Whiteman, who is based in New Zealand but was in Victoria for the meeting, said Monday that the team presented Osborne with new clinical information and the names of at least a dozen global experts on Brineura and Batten disease who had not been contacted by the rare drugs committee.
She said it also presented clinical evidence of the benefits of Brineura for children with Batten disease that go well beyond the motor skills — walking and talking — on which Charleigh was assessed. Those benefits include significant improvement in seizure control, lifespan and quality-of-life measures, including cognitive and emotional behaviours, she said.
“[The] very team who developed the motor and language score have said it’s not an appropriate tool,” said Whiteman, adding a new tool to measure the added benefits has since been developed.
Whiteman said it’s “really perplexing” that the government didn’t reach out to the experts who led the development of Brineura and the clinical rating scale.
She said Charleigh should continue to receive her infusions until the rare-drug committee has done a thorough review of the new evidence and consulted with the experts, “who were waiting to have meetings with them.”
Whiteman said the health minister appeared to be receptive to the additional information and said she would present it to the advisory committee.
Osborne confirmed on Monday that she has passed on the information to the committee of experts and physicians to determine whether it was previously considered and is relevant.
“If evidence exists that continuing treatment would still provide benefits for cases like these, I encourage the drug manufacturer [BioMarin] to immediately submit it to Canada’s Drug Agency and to request that the discontinuation criteria — which all provinces that provide coverage for Brineura have adopted — be revised,” she said.
Asked about the case at a news conference Monday, Premier David Eby said: “If there’s additional information that was not considered, it should be considered.
“The door is always open.”
Charleigh had her last infusion of Brineura at Victoria General Hospital on June 19, but missed her next one July 3 due to the Health Ministry decision.
“She has now missed her first infusion since the decision from the government, and she’s without therapeutic levels of life-sustaining enzymes in her brain,” said Whiteman, adding Charleigh’s mother is watching the child closely for changes.
“We don’t know how quickly those new symptoms will come on — she may start having seizures for the first time in years, she may start to have these painful movement disorders and start to go downhill.
It could be rapid, or she might have a sort of slower progression,” said Whiteman, adding that the next steps are urgent.
“We just don’t have time, and we don’t want Charleigh to be an example at this point of what happens now that she’s off the drug.”
Whiteman noted that Charleigh is the only patient on the drug in B.C., and one of fewer than 20 in Canada and a few hundred worldwide. She said she’s not aware of any other case where a child was taken off the drug without the family and health providers’ consent, adding it sets a “really dangerous precedent.”
“This case is not just about Charleigh,” said Whiteman.
“We have more than 15 children across all of Canada who may be coming to face the same the same scrutiny in the near future if this case isn’t overturned for Charleigh.”
Hundreds of children on Brineura around the world will also “come under the microscope” if the drug-coverage decision isn’t moved into the hands of the treating clinicians and the child’s family, she said.
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