The Challenge of Funding Expensive Drugs for Rare Diseases
The decision to fund expensive drugs for patients with rare diseases often involves complex ethical, financial, and medical considerations. Former British Columbia (B.C.) health minister George Abbott recently shared his perspective on this issue, suggesting that he would have made the same choice as the current New Democratic Party (NDP) government in supporting a 10-year-old girl with a rare condition, despite recommendations from an advisory group.
Abbott served as B.C.’s health minister in 2007 under the Liberal government when an expert advisory group was created to provide guidance on approving costly treatments for rare diseases. At the time, the government faced difficult decisions as new, expensive drugs entered the market. “It’s one of those difficult situations where you can never really get enough good advice on whether to fund or not,” Abbott said, highlighting the challenges of balancing patient needs with limited healthcare resources.
He emphasized that while the advisory group is essential, there are cases where the government may choose not to follow their recommendations after carefully reviewing evidence about a drug’s benefits and considering the patient’s suffering. This dilemma is exemplified by the case of Charleigh Pollock, a 10-year-old girl with neurodegenerative Batten disease who requires Brineura, a drug costing over $800,000 annually.
Balancing Patient Needs and System Constraints
Paul Ramsey, another former B.C. health minister, echoed similar sentiments, stating that while such decisions are inherently difficult, the government must retain the ability to say no to treatment, even if it leads to public backlash. He pointed out that medical decisions are made daily by physicians and institutions, and not all outcomes are positive. “The idea that every medical decision should be public is just counter to the way the system works,” Ramsey said.
The controversy surrounding the funding decision has prompted B.C. Premier David Eby to call for an overhaul of the province’s process for determining coverage of expensive drugs for rare diseases. Ten members of the advisory group resigned in protest after the government chose not to follow their recommendation to stop treatment for Pollock. Eby acknowledged the need for changes to improve transparency and better serve the public.
Initially, the government supported the advisory group’s decision to halt coverage, citing the importance of avoiding political interference. However, public pressure led to a reversal, with the government restoring access to the drug. The advisory group had recommended stopping treatment because the discontinuation criteria were met, indicating that the benefits of the drug were no longer favorable compared to its risks. Pollock’s parents, her doctors, and U.S. researchers treating Batten disease disagreed with this assessment.
Privacy Concerns and Public Accountability
Transparency in these decisions remains a contentious issue. Ramsey noted that introducing greater openness could be challenging due to patient confidentiality concerns. Advisory group members may also be reluctant to have their names made public, fearing an influx of letters and threats. “If they were to provide good advice, they don’t want to be part of a public dialogue or debate,” he said.
This tension between transparency and privacy was highlighted during Ramsey’s tenure as health minister, when pharmaceutical companies sued the B.C. government and him personally over a reference-based pricing system. The system aimed to limit pharmacare coverage by designating a reference drug within each class, requiring patients to pay the difference if they chose a more expensive option. The Therapeutics Initiative, composed of practicing physicians and pharmacists, was established to help select these reference drugs.
Ramsey also pointed out that the profit-driven nature of pharmaceutical companies and their relationships with patient advocacy groups complicate decision-making around drug coverage. These dynamics make it difficult for governments to balance cost, efficacy, and patient needs.
The Growing Financial Burden
Abbott noted that the challenge of funding expensive rare disease drugs is becoming more pronounced as more such treatments emerge. He recalled a previous case where the government funded Naglazime, a drug costing over $1 million annually for a young patient with a progressive condition. While the decision was described as “heart-wrenching,” it did not become public knowledge.
The financial strain on the provincial health budget is growing. Postmedia reported that spending on rare disease treatments reached nearly $200 million last year for 600 people, up from $22 million eight years ago. This trend underscores the increasing complexity of funding decisions and the need for a more transparent and responsive system.
Moving Forward
In response to questions about improving transparency, Health Minister Josie Osborne stated that it is too early to determine what specific changes will be implemented. She emphasized the goal of creating a system that supports patients and families while ensuring committee members have the necessary tools and resources.
Dr. Sandra Sirrs, a member of the advisory group who resigned, acknowledged the importance of transparency but stressed that individual patient decisions cannot be made public due to privacy concerns. However, she suggested that more transparency could be achieved in the process of selecting which drugs to cover and determining eligibility.
As B.C. continues to navigate these challenges, the balance between patient care, financial responsibility, and system integrity remains a central concern.