B.C. Minister of Health Josie Osborne says the ministry is sticking by their decision to stop funding an expensive drug called Brineura after meeting with the family of 10-year-old Charleigh Pollock, a young girl living with a rare neurological disorder.
Pollock is the only child in B.C. living with Batten disease and on June 19, she received her last enzyme replacement infusion. She had received funding for the drug from the province for nearly six years, costing around $1 million a year.
The decision to cancel the funding came after recommendations from provincial Expensive Drugs for Rare Diseases committees (EDRD) and the Canadian Drug Agency's (CDA) clinical criteria for when a treatment should be discontinued.
In a news release from Osborne's office, she said she met with the Pollock family on July 4, along with three Batten disease patient advocates who presented information to the minister and raised concerns that two unpublished poster presentations had not been considered by BCâs Expensive Drugs for Rare Diseases EDRDs or the CDA.
"I committed to urgently provide this information the Ministry of Health and its committees of physicians and clinical experts to confirm whether it was previously considered and is relevant for treatment in Charleighâs case," noted Osborne's release.
She says the ministry confirmed that the evidence provided by the advocates was considered by the EDRD and the CDA back in February when Pollock's physician appealed the decision to discontinue her treatment.
During the meeting, she said, concerns were also raised that two key international experts were not engaged by the CDA.
Osborne says she confirmed that the CDA reviewed and evaluated eight separate publications by the two experts, and confirmed that Biomarin Pharmaceutical, the manufacturer of Brineura, also submitted detailed information to the CDA, including unpublished studies, clinician statements, and materials from these same experts.
"The EDRD expert committees have not revised their recommendation to discontinue coverage given that there is no clinical evidence it would provide further benefits for Charleigh," noted the release.
Pollock's mother has repeatedly claimed Brineura has given Pollock a life free from daily seizures, though the CDA continues to say that they have found âinsufficient evidenceâ to make definitive conclusions about the potential benefits of the drug on quality of life, seizure control, and mortality outcomes.
"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.
"We must all continue to hold hope that ongoing advancements in rare disease research will one day prevent other children and families from experiencing the same suffering, and offer a better future through knowledge, treatment, and ultimately, cures."
She does note that the EDRD regularly denies or discontinues treatment for other patients with rare diseases because they do not meet CDA clinical criteria for continued treatment.
More to come