Charleigh's case: Disease experts ‘strongly oppose' move to cut rare drug funding
The letter is signed by the Clinical Directors from the U.S. Batten Disease Clinical Centers of Excellence and Batten Disease Clinical Research Consortium and asks the B.C. government to review the current discontinuation criteria.
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.
However, the decision was not reversed.
2:00
Review of Charleigh Pollock's case finished
The doctors and researchers who wrote the letter say their international colleagues and collaborators Angela Schulz MD and Miriam Nickel MD in Germany were instrumental in developing the clinical outcome measures used to track disease progression in CLN2 disease, which is a type of Batten disease.
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'Drawing from our collective and unparalleled expertise in CLN2 disease, we strongly oppose the decision to withdraw funding for life-sustaining ERT from Charleigh Pollock and call for a review of the current discontinuation criteria,' the letter reads.
Prior to 2017, the late-infantile form of CLN2 disease was universally fatal, however, the introduction of intracerebroventricular ERT, which is the medication Pollock was given, 'fundamentally altered the disease trajectory.'
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The experts said the treatment 'has demonstrably prolonged survival, slowed progression, improved seizure control, and enhanced quality of life.'
The B.C. government has always maintained that the decision to stop funding Charliegh's medication was never about money but a panel of experts decided the drug was not helping Charleigh anymore.
'This is a really awful case,' B.C. Premier David Eby said on Thursday. 'This is a beautiful child and a family facing a horrific diagnosis. Medical experts are disagreeing about the best course of action.
'We received a letter this morning from slightly more than a dozen experts in Batten disease from the United States who wrote to the (health) minister and I expressing that their perspective on this medication is that it would continue to provide benefit for patients, including for Charleigh, that are at the stage that she's at.'
Eby said Health Minister Josie Osborne will likely have more to say on the matter on Friday.
2:15
Langford mom devastated after province pulls funding for daughter's expensive drug
The letter from experts said they are concerned by the clinical and scientific justification used by B.C.'s ministry of health to terminate treatment for Charleigh.
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Andrew McFadyen, executive director of the Issac Foundation in Toronto, which is a patient advocacy organization dedicated to supporting individuals living with rare diseases, was one of the experts who signed the letter to Eby and Osborne.
He said they provided the same information to Osborne when they met with her a few weeks ago and he was appalled the government stopped funding the drug in the first place.
'So while this may not be overly new information, I think the idea that every single Batten disease expert in the world has signed on to say, we're unified, we agree, we're imploring you to change this decision, but we're also imploring you to look at developing new criteria so that this situation doesn't have to take place for other children across the country, but also around the world, like it did here now,' McFayden added.
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'We have to figure out a way that works for the doctors and experts advising on whether or not a particular patient should receive a particular treatment, and we need to make sure that it works in a way that it's the experts that are making those decisions, and that that is taking place in a way that's transparent and understandable to the public.' Experts on the 58-member expensive drugs for rare diseases committee had recommended the government cut off funding for Brineura, the drug prescribed to Charleigh Pollock, a 10 year-old Vancouver Island girl who is the only person in the province diagnosed with the rare degenerative brain condition known as Batten disease. In their recommendation, the subcommittee dealing with Pollock's case pointed to the review of Brineura by Canada's Drug Agency, which found evidence that the drug is effective in extending the lives of patients and in reducing symptoms such as seizures was inconclusive. This advertisement has not loaded yet, but your article continues below. It also argued that Pollock was in an advanced stage of Batten disease and Brineura would no longer help her. An advisory board made up of all the chairs of the subcommittees, as well as outside specialists in health ethics, health economics and other fields, backed up the subcommittee's recommendation and the government officially announced it was revoking coverage last month. Jori Fales, Pollock's mother, disputed the finding, stating that she had seen with her own eyes how Brineura helped improve her daughter's quality of life and that, without it, she was worried Charleigh's condition would rapidly decline. After a public outcry, advocacy from Fales and a letter from 13 American experts, Health Minister Josie Osborne reversed the decision and restored funding of the drug for Pollock on July 17. This advertisement has not loaded yet, but your article continues below. The Ministry of Health confirmed 10 members of the expensive drugs for rare diseases committee have now resigned. That's a doubling since last week when the ministry stated there had been five resignations. Dr. Sandra Sirrs, who resigned as chair of the subcommittee that made the decision in Pollock's case, told Postmedia last week that the committee process had been undermined by the NDP's political calculations and that she isn't sure if it will survive the resignations. 'To see this process dragged through the mud, I don't even know whether the process will survive this,' she said. Sirrs would not comment on Eby's latest comments. In a statement, Osborne said she has asked the ministry to review the expensive drugs for rare diseases process to 'identify opportunities for improvement.' 'We also recognize the need to increase public understanding of how the … process works. Greater transparency can help build trust and ensure that patients and families feel heard and informed as decisions are made,' said the minister. alazenby@ Read More Vancouver Canucks Vancouver Whitecaps Golf Sports News
