The drug coverage for a 10-year-old girl on Vancouver Island who has a rare disease will be reinstated, the B.C. Ministry of Health has confirmed.
This is a reversal of the ministry’s announcement last month that it was discontinuing coverage for Charleigh Pollock’s treatment.
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.
“I spoke to Charleigh’s family earlier this evening. I confirmed to them that I have reinstated Charleigh’s Brineura coverage and that coverage will be available to them for as long as the treating physician and the family deem it appropriate,” Health Minister Josie Osborne said in a statement Thursday.
In June, the government announced it was discontinuing its coverage for the drug, saying it 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.
Charleigh’s mother, Jori Fales, says she feels thankful and overwhelmed.
“I am so grateful to everybody that has supported our family,” she said.
“Charleigh, we love you, and we are so happy. I am so happy.”
Fales says she was told her daughter will receive treatment for as long as she needs.
Last month’s announcement had caused a public uproar. Lori Brown, president of Batten Disease Support and Research Association Canada, says public pressure certainly played a role in getting the ministry to reverse it’s decision, “with all of the people in British Columbia and beyond, everyone who was supporting Charleigh and her family and speaking out, sending emails, writing letters, just talking about it.”
She says she is confident there won’t be another interruption in funding.
“I feel like, if they have come out and said this is the decision, I think this is going to have to stick,” Brown said.
Fales had been advocating for her daughter’s treatment for years after Charleigh was diagnosed with Batten disease in 2019. The 10-year-old is the only person in the province diagnosed with the condition, with around a dozen reported cases in the entire country.
Thursday’s reversal comes after more than a dozen U.S. doctors wrote a joint letter to Osborne and Premier David Eby urging them to reconsider the withdrawal of funding.
“I continue to strongly believe that decisions about care should be made by health experts to ensure they are based on the best available evidence,” Osborne said.
“The letter I received today from Batten disease experts confirms there is significant disagreement between health experts on Brineura, and it is not acceptable that Charleigh and her family suffer as a result of that disagreement about the use of Brineura for Batten Disease.”
Brinuera is estimated to cost around $844,000 per year. The ministry says the cost of the drug was not a factor when considering whether to cover treatments.
Minister promises actions to ensure evidence-based progress
Speaking at a media event Friday morning, Osborne apologized to Charleigh’s family for “everything they have endured over the past few months.”
She says the province will reimburse the family for the dose of Brinuera it purchased with help from donations.
In light of the lack of scientific consensus on the criteria to assess the drug’s use, Osborne says the ministry is taking action to help other families across Canada avoid the same experience.
“First, I have asked the Therapeutics Initiative, an independent body based at UBC, to conduct a thorough evidence-based assessment of Brineura findings that will help guide both clinical practice and public policy,” said Osborne.
Other actions include urging the manufacturer to request a formal review by Canada’s Drug Agency of the current reimbursement criteria and urging the federal government to implement a national strategy for drugs for rare diseases.
Osborne says she understands the criticism that many people, political opponents, and experts have shared about the province’s actions.
“I know that British Columbians want decisions about care to be made by health professionals, to be made by medical experts, but I also know that British Columbians never want to see a child like Charleigh suffer— or be put at the middle of that, and that’s why it’s so important to take this course of action now, reinstating the funding, allowing the work that has to be done in order to resolve scientific differences around the use of this drug.”
Osborne said the committee that originally resolved to cut the funding “still stands,” and she will meet with its members to hear their concerns.
“I am incredibly appreciative of the work they do. They are professionals. They provide their expertise and their advice. It is a very valued and needed structure that the Ministry of Health depends on… Again, I think British Columbians want committees comprised of experts and professionals to be providing this advice, to be using evidence in making these recommendations, and that’s the appropriate way forward.”
In an update Friday afternoon, the ministry confirmed that four members of the Expensive Drugs for Rare Diseases Committee had resigned since the morning.
The ministry explained that approximately 50 members comprise the committee, which includes subcommittees.
BC Conservative MLA Brennan Day says the decision to reinstate funding is a “step in the right direction,” but says his party will continue to pressure the governing BC NDP for change.
“They need to follow through and make sure that this is not possible again in B.C.,” said Day.
“We’re really, really hung up on protecting the system and not protecting the patients in British Columbia health care at the moment, and we need a change in the way that we’re handling these sorts of cases.”
—With files from Charlie Carey and Srushti Gangdev.