B.C. Review Urges Changes to Rare-Disease Drug System Amid Rising Costs

A review conducted by the British Columbia government four years ago recommended significant changes to the province’s approach to funding and treating rare diseases with expensive drugs. This assessment, which has only recently come to light, emphasizes the urgent need for improved transparency, communication, and decision-making as drug costs are projected to reach an estimated $600 million annually by the end of the decade.

The findings of this review, which were delivered to the government in March 2021, have largely gone unaddressed, even as Premier David Eby and Health Minister Josie Osborne have recently called for reforms. Their push for change follows public outcry over the government’s decision to discontinue an $800,000 annual drug treatment for Charleigh Pollock, a ten-year-old suffering from a degenerative disease.

The leaked report, which was not made public until now, reveals that only two of the over 300 recommendations have been implemented. These include the creation of a website aimed at enhancing transparency and the establishment of an appeal process for drug funding decisions. However, critics argue that these efforts fall far short of what is necessary.

“I think the recommendations are still relevant,” stated Dean Regier, an associate professor at the School of Population and Public Health at the University of British Columbia, who reviewed the recommendations at the request of Postmedia. He highlighted that navigating the complexities of expensive drugs for rare diseases remains a significant challenge for health systems.

In response to inquiries about the implementation of the recommendations, the B.C. Ministry of Health asserted that the “majority” of the report’s suggestions have been addressed or are part of ongoing work. Yet, the Ministry only cited the website and the appeal process as completed initiatives, despite the report emphasizing 39 mentions of transparency involving oversight and public engagement.

The pressure for reform intensified after the government reversed its decision on Charleigh Pollock’s treatment last month, a move that contradicted the advice of its own 58-member advisory committee. Following this reversal, ten members of the committee resigned, expressing dissatisfaction with the decision-making process.

Dr. Sandra Sirrs, one of the advisory committee members who resigned, noted the need for more substantial steps toward transparency. She emphasized that many recommendations from the 2021 report aimed to educate the public about the complexities of funding rare disease treatments. Such initiatives could have better prepared the public for the government’s decisions, which have garnered significant media attention.

The original review stressed the importance of using public feedback to guide funding decisions for these costly treatments. It suggested that a system be established to collect real-world data on the effectiveness of rare disease drugs, a crucial component for determining their continued funding. Last year, British Columbia allocated $200 million on these drugs for just 600 patients.

The report outlines ways to strengthen the decision-making process surrounding the funding of expensive drugs, advocating for increased cooperation with federal health agencies. Many recommendations require collaboration with the federal government, as they address broader issues related to drug approval and funding.

The review included a comprehensive communication and engagement strategy aimed at fostering public trust. This strategy highlighted the need for transparency in decision-making processes regarding drug funding. It called for proactive media strategies to manage public perceptions and to inform the community about the rationale behind decisions that may not always align with public sentiment.

As Premier Eby recently reiterated, the current system must evolve to enhance transparency and ensure that informed experts guide the decision-making process. Health Minister Osborne echoed this sentiment, stating that greater transparency can help build trust, ensuring patients and their families feel heard and understood.

The issues surrounding the funding of rare disease treatments in British Columbia continue to be contentious, as the government faces mounting pressure to address long-standing recommendations. The pace of reform will likely determine how effectively the province can navigate the complex landscape of healthcare funding for rare diseases in the years to come.