When Health Minister Josie Osborne announced in June the province would no longer fund Langford girl Charleigh Pollock’s medication, she said the ministry had ensured “no stone was left unturned” when reaching the decision.
But a month later, it appears a few stones may have been overlooked.
After standing firm on the decision for weeks, Osborne called Pollock’s family July 17 to tell them the ministry is making an about-face, reinstating coverage for the drug Brineura “for as long as the treating physician and the family deem it appropriate.”
For the 10-year-old's mom, Jori Fales, it has been a journey of “hope and despair” since the family was first told in February the province was cutting funding.
But now, she says, “the nightmare is over.”
“There was screaming, happy screams for sure,” said Fales about the moments after her phone call with the health minister. “Just relief this is over … we were never going to give up.
“Charleigh wins at the end of the day.”
Since the age of three, Pollock has been receiving bi-weekly enzyme replacement infusions of the drug Brineura, funded by the province at a cost of around $800,000 a year.
The drug slows the progression of the rare neurological disorder, Batten disease.
But the province believed Pollock’s motor and verbal skills had declined to a point that indicated she was no longer benefiting from the treatment.
Something the girl's family and medical team have long disagreed with. For months they have repeatedly explained the 10-year-old is still benefitting from the infusions, which they said have given her a life free from daily seizures.
"Charleigh has so much life to live, and when someone else makes that decision to cut her life short, I would think any parent would fight to the bitter end," said Fales.
But despite repeated appeals from the family and Batten experts around the world, Osborne and the ministry refused to budge.
Then came a letter July 16 from over a dozen U.S. Batten disease clinicians and researchers, raising concerns about the “clinical and scientific justification” used by the province.
They said the motor-language scoring tool used by the province in Pollock’s case was "never intended" to be used for decision-making regarding treatment discontinuation.
And having cared for the majority of U.S. Batten disease patients treated with Brineura, the experts explained the drug has "demonstrably prolonged survival, slowed progression, improved seizure control and enhanced quality of life."
Osborne said the letter was “the tipping point” for her.
"What has become clear is that there is significant disagreement around the criteria that guide the use and funding of Brineura,” she said. “And Charleigh and her family cannot be caught in the middle of that disagreement.”
According to Fales, the content of the experts’ letter was the same information shared at a private meeting July 4 with Osborne, the mom and three Batten disease experts.
Following that meeting, Osborne again said that Pollock’s funding would not be restored due to a lack of clinical evidence.
“It is very frustrating,” said Fales. “I don't have a MD or PhD, but I feel very much a Batten expert – I live it every single day and I know my daughter better than anyone.
“I do wish my voice had been heard a bit more and the voices that were sitting beside me at that table. But, the outcome is exactly what we needed it to be.”
But there are lessons to be learned for the province, says Fales, who feels her daughter's case was consistently "mishandled."
"I think It was forgotten that ... a 10 year old's life was hanging in the balance," she said.
Alongside her decision to reinstate Pollock's funding, Osborne has asked the Therapeutics Initiative – an independent body based at UBC – to conduct a thorough, evidence-based assessment of Brineura, that will help guide both clinical practice and public policy.
She has also urged the pharmaceutical company that manufactures Brineura, BioMarin Pharmaceuticals, to request a formal review by Canada's Drug Agency of the current discontinuation criteria.
The minister also wants the federal government to "urgently expedite" a national strategy for drugs for rare diseases, to ensure a consistent approach and sustainable funding of rare disease treatments across all provinces and territories.
"I absolutely regret how this has played out," Osborne said speaking to reporters. "And that's why we are taking action to ensure that no family with Batten disease goes through this again."
Fales hopes so too. "It's been hell and I would never wish this on another family."
The news about the funding reversal came on the same day Pollock received her 158th infusion at Victoria General Hospital; but her first paid for privately.
In a matter of days, over $70,000 was raised by the community to help the family purchase Brineura – at a cost of $33,000 per dose – for as long as possible.
“I’m just beyond grateful that they picked us up when we were feeling very down,” said Fales. “I will never forget all that love and support – I’m getting so many messages that just make me cry all the time.”
Osborne has confirmed the province has now paid for the July 17 infusion.
“Charleigh woke up today, just like rebounded,” Fales said. “So we know the medication is in her system – it’s doing exactly what it's meant to do.”
After months of advocacy and fundraising, Fales is looking forward to putting all her energy back into enjoying time with her daughter.
Plus, also allowing time for her and the family to heal.
“A lot of healing needs to happen,” she said. “There’s a lot of trauma that happened in the last six months.
“We go back to our lives … giving Charleigh exactly what she needs … just loving every single day that we have with her.”
