The fight to restore provincial funding for Langford girl Charleigh Pollock’s “life-sustaining” medication is over.
Health Minister Josie Osborne has confirmed the ministry is standing by its decision to discontinue coverage for the drug Brineura, “given that there is no clinical evidence it would provide further benefits” for the 10-year-old.
For six years, Pollock has been receiving bi-weekly infusions of the drug, which slows the progression of the rare neurological disorder she lives with: Batten disease.
Her family, medical team and Batten disease advocates have long argued that Pollock is still benefiting from the drug – listing relief from daily seizures as one of its many benefits.
Pollock’s last infusion was June 19.
“Complete devastation,” said mom Jori Fales speaking to Goldstream Gazette about the minister’s announcement July 11. “I don't know if there's another word to express how we're feeling today – it’s a very, very sad day.
“We're just profoundly sorry to Charleigh that this is happening.”
Fales says she was told the news by email, minutes before Osborne held a virtual meeting with media representatives.
“I was receiving texts as I was still reading the email,” she said. “I am utterly disgusted.”
With the support of three Batten disease experts, Fales had met with the minister July 4 to present information they said had not been considered by the Expensive Drugs for Rare Diseases committee (EDRD) or included in the Canada’s Drug Agency (CDA) review this year, which was used to support the decision to discontinue Pollock’s funding.
They also raised concerns that two key international experts were not engaged by the CDA as part of the review. Including Dr. Angela Schulz, who developed the clinical scoring criteria used to end Pollock’s funding.
Schulz has said the criteria she developed is outdated and is creating new clinical scoring criteria, which is more sensitive to the long-term benefits of Brineura.
A statement from Osborne confirms that “all the relevant evidence” provided at the meeting “was already considered” by the EDRD expert committees in February, when Pollock’s physician appealed the original decision to discontinue treatment.
The health minister did not confirm what information, if any, provided at the July 4 meeting was considered irrelevant.
Osborne also said the CDA had reviewed and evaluated eight separate publications by the two experts, including Schulz.
It was also confirmed by the health minister that the manufacturer of Brineura submitted additional unpublished research for review as part of the process.
"This has been one of the most challenging issues that I have worked on since becoming health minister, and I take seriously my role to ensure that B.C. has drug review processes that are independent, evidence-based and treat people equally," said Osborne in a statement.
Fales says she struggles to believe the ministry took the time to properly consider what she and the Batten disease experts presented at the meeting.
She also says the ministry has not contacted Schulz.
“I feel this decision was made months ago, and our family was dragged through hell just to get the exact same outcome,” said Fales.
“Why did they take another seven days to give us the exact same answer, knowing that my daughter can sustain irreversible brain damage.”
Pollock is now eight days past what would have been her next infusion treatment.
Without “therapeutic levels of this life-sustaining enzyme,” Dr. Ineka Whiteman, head of research and medical affairs for the Batten Disease Support Research and Advocacy Foundation, has warned that “time is neurons” for the Langford girl.
“Any brain cells that Charleigh loses from today won’t be regenerated, so every day is damaging for her right now,” she said, speaking to Goldstream Gazette last week.
Fales says she has yet to notice any change in her daughter’s condition.
“But we know it is happening, we just haven't seen a visual cue yet,” she said.
Money raised by the community will allow the family to pay privately for two more infusions, said Fales. Osborne also confirmed the family is working with Island Health to administer the treatment.
“But we know that this isn't a long term solution, so at some point we need to accept that Charleigh will no longer be getting her infusions,” said Fales.
“We're hoping to get those two in her so she can enjoy her summer, which there's a good chance now, is her last summer.
“I'm so grateful to our community for all the love and support, and I appreciate that they can see my daughter's life has a value.”
