Drug-funding decision for B.C. girl with rare disease will not be reversed
B.C.'s expensive drugs for rare diseases (EDRD) expert committees and Canada's Drug Agency (CDA) have 'not revised their recommendation to discontinue coverage given that there is no clinical evidence it would provide further benefits,' the Ministry of Health said in a statement on Friday.
Charleigh Pollock has a neurological disorder called Batten disease that leaves her with recurring severe seizures and mobility loss, and will eventually cut her young life short.
On June 18, Charleigh's family was informed that government funding of a drug called Brineura, which her family said has stabilized her condition and radically improved her quality of life since her 2019 diagnosis.
The drug is expensive, carrying an annual bill for the twice-monthly infusions of about $1 million.
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Charleigh's family appealed to the B.C. Ministry of Health to reverse the decision and consider more research and speak to more experts.
B.C. Health Minister Josie Osborne met with Charleigh's family a week ago. The ministry said they were joined by three Batten disease patient advocates who presented information and raised concern that two unpublished poster presentations had not been considered by the EDRD committees or CDA.
Osborne said she provided this information to the Ministry of Health and its committees of physicians and clinical experts.
She also said that during the meeting on Friday, concerns were also raised that the CDA did not engage two key international experts.
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They were informed that all relevant evidence was already considered in February when Charleigh's physician appealed the decision to discontinue treatment.
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In February, the ministry also asked the CDA to complete a review of all the latest research and evidence completed since 2019.
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'The Ministry can confirm that the CDA reviewed and evaluated eight separate publications by these two experts,' the Ministry of Health said in a statement.
'The Ministry also confirms that Biomarin Pharmaceutical, the manufacturer of Brineura, also submitted detailed information to the CDA, including unpublished studies, clinician statements, and materials from these same experts.'
Osborne said cost is not a factor considered in these patient-level decisions.
'I know the outcome of this process is not the one anyone was hoping for, most particularly Charleigh's family,' Osborne said.
'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.
'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.'
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In a previous interview, Charleigh's mother Jori Fales told Global News losing access to the drug would be 'catastrophic' for her daughter.
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'She will have seizures again, her organs will start shutting down, she will require suction to breathe.… It basically means she is going to suffer horrifically and be put to an earlier death,' she said.
'This life is painful enough, having to raise a terminally ill child, and now having to fight to keep her in this world — like, just let us have our life back and let us have our child for the time we have left and let her be pain-free.'
A GoFundMe remains active for anyone who wants to donate to the family and Osborne said the family could use that money to pay for Brineura out of pocket.
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