In a dramatic policy reversal that highlights the power of patient advocacy, the British Columbia government has reinstated coverage for Brineura, a life-saving treatment for children suffering from CLN2 Batten disease. This decision comes after months of desperate appeals from affected families who faced the devastating prospect of watching their children deteriorate without access to the only available treatment.
The announcement marks the end of a tumultuous period that began in April when the province quietly removed the medication from its coverage list, leaving families shocked and scrambling for alternatives. For children like 10-year-old Ellis Hayes and 8-year-old Henry Brown, the decision was potentially life-threatening.
“When they took away the funding, they essentially took away our children’s lives,” said Jennifer Brown, Henry’s mother, speaking from her Vancouver home. “This treatment isn’t optional—it’s the difference between my son being able to walk and talk or losing those abilities forever.”
CLN2 Batten disease is an ultra-rare neurodegenerative disorder that affects approximately one in 200,000 children. Without treatment, affected children typically lose their ability to walk, talk, and see, with most not surviving past their teenage years. Brineura, administered directly to the brain every two weeks, has been shown to dramatically slow this progression.
The province’s initial decision to defund the treatment reportedly stemmed from a Canadian Agency for Drugs and Technologies in Health recommendation citing insufficient evidence of clinical benefit—a position vehemently contested by neurologists treating these children.
Dr. Sylvia Stockler, a metabolic disease specialist at BC Children’s Hospital, provided critical perspective on the treatment’s efficacy. “We have clear evidence that Brineura significantly slows disease progression. Children who would otherwise be unable to walk or communicate are maintaining these abilities years beyond what we would expect without treatment.”
The reversal came after unprecedented public pressure, including media coverage and a Change.org petition that gathered over 23,000 signatures. The affected families also sought legal representation to challenge the decision on constitutional grounds.
Health Minister Adrian Dix acknowledged the policy change in a statement: “After careful reconsideration and consultation with clinical experts, we have determined that continued coverage for Brineura aligns with our commitment to providing necessary medical treatments for British Columbians.”
The medication’s extreme cost—approximately $1 million per patient annually—underscores the difficult calculations provinces must make when determining drug coverage. However, for families like the Browns and Hayes, the financial considerations pale in comparison to their children’s lives.
“This isn’t just about our kids,” said Michelle Hayes, Ellis’s mother. “It’s about establishing that in Canada, we don’t abandon treatment for children with rare diseases simply because they’re expensive or because few people are affected.”
The decision reverberates beyond British Columbia, potentially influencing how other provinces approach coverage for ultra-expensive treatments for rare diseases. Patient advocacy groups are calling for a more transparent and consistent national framework for evaluating and funding such medications.
With treatment now secured, the affected families can return their focus to supporting their children through their ongoing battles with this devastating disease. The reinstatement of coverage means these children will continue receiving their bi-weekly infusions without interruption.
As our healthcare system continues to grapple with limited resources and ever-increasing costs, what responsibility do we have as a society to ensure that patients with rare diseases aren’t left behind simply because their conditions affect too few people to command political attention?
