Charleigh Pollock, a nine-year-old girl from Langford, British Columbia, who is fighting neuronal ceroid lipofuscinosis type 2 (CLN2), or Batten disease. Sadly, she’s met with yet another blow in her battle against this awful disease. This rare, terminal genetic disorder severely impacts her day-to-day life, contributing to several seizures a day and resulting in progressive brain damage. Despite the B.C. government’s previous assurances that they would provide regular financial support for her expensive medication, Brineura, that funding has unexpectedly been cut off.
In 2019, the B.C. government took action by announcing it would pay the $1 million per year cost of Brineura itself. This medicine is given by an intravenous infusion directly into the brain to halt the progression of CLN2. The B.C. rare diseases expert committee has just finished reviewing Brineura. They decided that the treatment has lost its therapeutic advantages once a patient develops important declines in their motor capabilities and verbal capabilities.
The committee reviewed eight individual studies on Brineura and requested advice from worldwide authorities on Batten disease. Notably, Charleigh Pollock’s family had identified two international specialists whose opinions were not included in the committee’s assessment. The administration’s decision to stop funding draws the curtain back on a major national problem. It is critical to weigh the need for effective treatment against the scant evidence surrounding Brineura’s long-term effectiveness.
Josie Osborne, B.C.’s Health Minister recognized the emotional impact such decisions have on families who encounter unique and rare diseases. “This has been one of the most challenging issues that I have worked on since becoming health minister,” she stated. She highlighted her efforts to keep B.C.’s drug evaluation processes independent, evidence-based, and equitable.
“Going forward, the ministry will do everything we can to support them and support the team, particularly as Charleigh’s family moves forward with their decision and whether they want to access treatment that they will pay for privately,” Osborne added. She admitted to the committee her awareness of the difficult place they’re put in having to make those painful choices each day.
Osborne emphasized that only about 650 people in B.C. are on treatment for different rare diseases at this point. The committee follows a thorough and multi-step process to make sure every case receives full and equitable review. Charleigh’s funding for her medication has been denied, just as it has been in a similar case from the United Kingdom just last week. There, they will no longer reimburse Brineura for any new Batten disease patients going forward, as there is insufficient evidence of long-term efficacy.
Members of the local community have gathered in support of Charleigh and her family following this unfortunate announcement. Grassroots community support has always been the bedrock foundation for families afflicted with rare diseases. Josie Osborne remarked on the importance of coming together during such challenging times, stating, “As a community, this is what we do. We come together when somebody is in a situation. We come together and provide the support.”
The Pollock family is now in the unfortunate position of having to choose as they consider their options to provide Charleigh with the treatment she needs. In the final analysis, they should consider pursuing private investment for Brineura. The emotional and financial impact of this decision weighs heavily on them.
