The heart-wrenching battle for a British Columbia family seeking life-changing medication for their daughter has taken another disappointing turn. Health Minister Adrian Dix confirmed Monday that provincial authorities have once again denied coverage for Brineura, a medication needed to treat a rare and fatal neurological condition affecting seven-year-old Lily Sanchez.
“I understand the devastating impact this decision has on the family,” Minister Dix told reporters at the provincial legislature. “But our drug review process must evaluate both clinical effectiveness and cost considerations across all rare diseases affecting British Columbians.”
Lily suffers from CLN2 Batten disease, an extremely rare genetic disorder that causes rapid neurological deterioration, including loss of speech, mobility, and vision, typically leading to death by early adolescence. Brineura, which costs approximately $750,000 per year, has shown promising results in slowing disease progression but remains beyond financial reach for most families without public coverage.
The denial marks the third time BC’s Drug Benefit Council has rejected funding for Lily’s treatment, despite mounting pressure from advocacy groups and medical professionals who point to successful coverage models in other provinces including Ontario and Saskatchewan.
Dr. Sylvia Davidson, a pediatric neurologist not involved in Lily’s case, explains the complex situation facing rare disease patients. “When we’re dealing with ultra-rare conditions, traditional cost-effectiveness models often fail these families. There simply aren’t enough patients to generate the large-scale clinical data typically required for drug approvals.”
Lily’s mother, Elena Sanchez, has been advocating tirelessly since her daughter’s diagnosis at age three. “Every month without treatment means irreversible progression,” she said in a tearful statement. “We’re watching precious skills disappear while knowing there’s a treatment that could help.”
The family has raised nearly $235,000 through community fundraising efforts, but this covers less than four months of treatment. Meanwhile, Canadian health policy experts continue to debate how best to address the growing number of ultra-expensive rare disease treatments becoming available.
According to the Canadian Organization for Rare Disorders, approximately one in twelve Canadians lives with a rare disease, though individual conditions like Batten disease may affect just a handful of children nationwide. This fragmentation complicates both research efforts and advocacy for coverage.
BC’s Ministry of Health spokesperson confirmed the province is participating in federal discussions on a national rare disease strategy, which could potentially create a more standardized approach to funding decisions across Canada. However, such frameworks remain years away from implementation.
For families like the Sanchezes, policy discussions provide little comfort against the relentless progression of disease. “We don’t have the luxury of time,” Elena emphasized. “Every day without treatment is another day Lily loses something she’ll never get back.”
As provincial health authorities face increasingly difficult decisions balancing individual needs against system-wide sustainability, one question remains urgently relevant: In a healthcare system founded on principles of universal access, how do we ensure that patients with the rarest conditions aren’t left behind simply because of statistical misfortune?
