Thanks to everyone who pushed the British Columbia government to reverse its decision to cease treatment with Brineura. This high-cost medication is the only drug available to treat Batten disease, a rare neurodegenerative disease. This reinstatement in large part comes after strong public pressure, including public outcry from the community. Brineura was approved by Health Canada in 2018. It’s manufactured by U.S.-based BioMarin Pharmaceuticals and priced at more than $800,000 annually per patient treated. Once an infusion pump has been implanted, the healthcare team can administer the drug through an infusion into the CSF. This process is four hours long and is done biweekly.
It was a subcommittee that first voted to end Charleigh Pollock’s treatment—a 10-year-old girl from Vancouver Island. A broader, citizen-committee advisory group supported this decision. In announcing the decision, Premier David Eby said that it was wrong for politicians to be making medical decisions. Regardless of this rationalization, the public outcry from the rescission allowed the B.C. government to reconsider its position.
Rising Costs of Rare Disease Treatments
B.C. is facing soaring costs for treatments of rare diseases. The cost to the province is approaching $200 million annually on these drugs. Just eight years ago, this spending was a mere $22 million. Now, as the chart above illustrates, the annual number of costly drug approvals and their cumulative costs have skyrocketed since.
Taken together, Brineura is one of the most extreme examples of these high cost treatments. The B.C. government’s first decision to discontinue Charleigh’s treatment raised a lot of alarms. It set off alarms about tightening access to life-saving medications and the morality of making such financial decisions.
Newly published data indicate that Brineura can have a meaningful effect on the quality of life for patients with Batten disease. In addition to improving seizure control, it further improves mortality outcomes. Getting to yes — The Canada Drug Agency published their 50-page Evaluation. At the same time, it underscored the lack of capacity to make strong conclusions about the drug’s relative benefits. This lack of clarity makes it difficult to make clear decisions about funding or improving access.
Public Response and Government Reversal
The original decision to discontinue treatment for Charleigh Pollock sparked national outrage and public outcry. Families impacted by rare diseases mobilized like never before, fighting for access to the treatments that could change their lives. The B.C. government’s eventual about-face underscores the power of grassroots advocacy to shape health policy in public health’s favour.
Dr. Sandra Sirrs voiced her frustration over the situation, stating, “The hypocrisy of it is just astonishing to me. They say no one wants politicians making decisions about your health. Well, that’s exactly what they did.” Her statements highlight the conflict that arises when political choice supersedes medical need.
As public pressure built, the government felt that they had to respond to the arguments posed by families and advocates. The Canadian Organization for Rare Disorders (CORD) emphasized their commitment to advocacy independent of funding sources, stating, “CORD develops policy positions and advocates independently of its funders and always in the interest of Canadians impacted by rare diseases.”
Broader Implications for Healthcare in Canada
Brineura treatment in B.C. foreshadows a costly burden that healthcare systems will soon face all over Canada. This issue is at the heart of access to medications for rare diseases. Joel Lexchin, a prominent healthcare expert, remarked on the complexities involved in drug coverage decisions: “Canada is not unique in terms of figuring out do you pay for all of these drugs? Do you pay for some of them? Do you pay for none of them? Do you pay for everybody who has the disease? Do you just pay for those who have the worst form of the disease?”
His remarks underscore the challenging global conversation between providing equitable access to essential therapies and maintaining the financial viability of our health care systems. Lessons learned from the B.C. government’s experience have led to interesting discussions on how to effectively strike a balance between these conflicting priorities.
