The drug, which costs about $1 million a year, is intended to slow the decline in patients’ ability to walk and talk until they reach the end stage of the disease.
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, noting the decision was based on expert advice.
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.
The ministry said Wednesday that it is satisfied that there is no clinical evidence that continuing treatment would have further benefits.
It says it’s acting on input from the Canadian Drug Expert Committee, which is made up of drug experts as well as lay members appointed by Canada’s Drug Agency to make reimbursement recommendations to participating federal, provincial and territorial publicly funded drug plans.
The ministry says continuing to provide coverage for a drug when a patient does not meet CDA conditions for treatment would undermine the integrity of the drugs for rare diseases program.
Reached by theTimes Coloniston Wednesday, mother Jori Fales was emotional and said she was 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,” Fales said Wednesday.
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.
Despite being unable to walk, Charleigh is “an extremely happy child” with more good days than bad, Fales has said.
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 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, Health Minister Josie Osborne directed ministry staff to “urgently review all of the facts” and determine whether the treatment had quality-of-life benefits for Charleigh.
Charleigh’s physician team had recommended Charleigh continue to receive transfusions of Brineura.
B.C. has about 30 drugs under the rare diseases program. The cost of an individual drug is not a factor considered by the expert committees in recommending coverage — the decision is based on specific patients who meet the clinical criteria, and committee and subcommittee recommendations that go directly to the Health Ministry for approval.
Fales has said any decision to end the drug treatment should be made by her daughter’s medical team and parents.
Courtenay-Comox MLA Brennan Day, the BC 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.
[email protected]