Coverage of an expensive drug for a nine-year-old Langford girl with a rare fatal condition is being discontinued, B.C.’s Health Ministry says.
Charleigh Pollock, who has a neurodegenerative condition known as Batten disease, will have her last publicly funded infusion of the drug Brineura on Thursday.
The drug, which costs about $1 million a year, is intended to slow — but not stop — the decline in patients’ ability to walk and talk until they reach the end stage of the disease.
Health Minister Jose Osborne said Wednesday that Canada’s Drug Agency has completed its review and there is no new evidence to change its 2019 recommendation to discontinue coverage of the drug, given the advanced stage of Charleigh’s condition.
Osborne said the case has been one of the most difficult for her and her team during her time as minister. “We all share the desire to do everything we can to care for this young girl,” she said. “I empathize deeply with Charleigh’s family and my heart goes out to them.”
Reached by the Times Colonist on Wednesday, mother Jori Fales said she was still processing the news and unable to speak about the decision, which comes in the wake of a previously announced extension of coverage to June 28.
Fales has said that two weeks after discontinuation of the drug, Charleigh likely faces “rapid deterioration, irreversible loss of function and ultimately death.” “I just need to process this for today,” she said.
Charleigh’s parents earlier said their daughter had seen benefits from bi-weekly treatments with Brineura, the only drug approved to treat the form of the disease that Charleigh has, known as ceroid lipofuscinosis type 2 (CLN2).
Charleigh has been taking the drug for almost six years, and her family has said that as a result, she no longer has multiple seizures a day.
Osborne noted that the drug is not an anti-seizure medication, adding there are other drugs to control seizures for patients with CLN2.
Charleigh is the only child in B.C. and one of fewer than 20 in Canada with Batten disease, which began to affect her at the age of three, when she suffered seizures and began to lose her ability to walk and talk.
The family was informed in January that the Canadian Drug Expert Committee had recommended ending the funding, and that Charleigh’s last publicly funded infusion would be Feb. 27.
The committee, made up of drug experts as well as lay members appointed by Canada’s Drug Agency, makes reimbursement recommendations to participating federal, provincial and territorial publicly funded drug plans.
The Health Ministry had said in a statement that while all provinces had adopted the national discontinuation criteria set out by Canada’s Drug Agency, “this disease is so rare that Charleigh is the first Canadian patient to reach the Canada Drug Agency’s criteria for discontinuing Brineura coverage.”
After the family’s story went public, Osborne directed ministry staff to “urgently review all of the facts” and determine whether the treatment had quality-of-life benefits for Charleigh.
Given the extraordinary circumstances, “we wanted to ensure no stone was left unturned,” Osborne said Wednesday.
B.C. covers about 30 drugs under the Expensive Drugs for Rare Disease program. The cost of an individual drug is not a factor considered by the expert committees in recommending coverage, the Health Ministry says.
Since the Expensive Drugs for Rare Diseases review process was established in 2007, the ministry has never provided ongoing coverage for a drug against the recommendations of clinical experts and criteria established by Canada’s Drug Agency, a ministry statement said.
Courtenay-Comox MLA Brennan Day, the B.C. Conservative critic for rural and seniors health, urged the province to reverse the decision on compassionate grounds. “I can’t imagine the emotions the family is feeling,” said Day.
