B.C.’s health minister confirmed Friday that a decision to cut off coverage of a life-prolonging drug for a 10-year-old Langford girl with a rare terminal illness will stand.
For six years, Charleigh Pollock has taken the drug Brineura, which costs almost $1 million a year, to slow the progression of Batten disease (CLN2), a fatal neurodegenerative disorder. Coverage was discontinued on June 19 and her last infusion was July 3.
Josie Osborne said Friday she’s aware the outcome “is not the one anyone was hoping for, most particularly Charleigh’s family.
“Watching a child face daily challenges that most will never know is heartbreaking beyond words, and I believe that every British Columbian wishes for a world where no child should have to endure such suffering, and no parent should have to navigate such uncertainty,” she said.
Jori Fales, Charleigh’s mother, who had hoped the decision would be reversed, said Friday she was numb upon hearing the news. “I’m just devastated by the disregard for my child’s life.”
Osborne said Island Health is in discussions with Charleigh’s family around the possibility of the family using private funds to pay for the drug. At least two fundraising drives are underway, including one online fundraiser.
The drug would be available to the family if they come up with the funds to pay for it, and the health authority would provide its facilities for it to be administered, Osborne said. “We will do everything we can to support them.”
Fales said paying for infusions privately is not a long-term solution.
“It’s a very sad day for Charleigh,” Fales said through tears. “But we’re extremely grateful for what has come forward in the hopes of having some more infusions and Charleigh being able to enjoy her summer.”
Neuroscientist Dr. Ineka Whiteman, a Batten disease expert, called the decision to discontinue coverage “appalling” and poorly informed.
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.
The decision early this year to stop coverage was based on the committee’s recommendation and was upheld on June 18.
Osborne said the Health Ministry had already asked Canada’s Drug Agency to undertake a review of all of the latest published research in the case, “which they did,” adding their review included engagement with leading clinical experts and considered people with lived experience with the disease and Brineura.
On July 4, Charleigh’s parents met with Osborne, along with 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.
Osborne said Friday that all the “relevant evidence” provided by the patient advocates “was already considered” by the rare disease program expert committees in February when Charleigh’s physician appealed the decision to discontinue treatment and presented evidence.
She also said Canada’s Drug Agency reviewed eight publications by two experts cited during her meeting with Charleigh’s parents — Dr. Angela Schulz, senior physician at the department of pediatrics and adolescent medicine at the University Medical Center Hamburg-Eppendorf, and Dr. Emily de los Reyes, attending pediatric neurologist at Nationwide Children’s Batten Disease Center of Excellence and professor of clinical pediatrics and neurology at Ohio State University College of Medicine.
She added Biomarin Pharmaceutical, the manufacturer of Brineura, also submitted detailed information to Canada’s Drug Agency, including unpublished studies and clinician statements.
The rare disease program experts have not revised their recommendation to discontinue coverage “given that there is no clinical evidence it would provide further benefits for Charleigh,” said Osborne.
Neither Schulz nor los Reyes were contacted directly by the province. Schulz developed the clinical measurement scale — the “motor-and-language” scale used by Canada’s Drug Agency — which she now says is no longer an appropriate tool for assessing disease progression in the mid-to-later stages of CLN2 Batten disease. She and her team are developing a more sensitive measurement scale.
Whiteman said Batten disease experts wanted to share more evidence as well as their experience with hundreds of children worldwide who are taking Brineura.
“It’s deeply disturbing that the minister has stood her ground and used the CDA review as a scapegoat to not go and consider the world experts who maintain that this is an unethical decision,” said Whiteman, who lives in New Zealand but was attending a conference with Batten disease experts in Nebraska on Friday.
Whiteman said she’s unaware of another child having been taken off the drug against the wishes of the family and treating physician. Charleigh is now in unchartered territory in terms of how quickly her symptoms return and how painful it might be, she said.
The Langford girl is the only patient on Brineura in B.C., and one of fewer than 20 in Canada and a few hundred worldwide with Batten disease.
