Press Release: RareKids-CAN supports Bill C-265 to improve access to treatments for children with rare diseases
RareKids-CAN, Canada’s pediatric rare disease clinical trials and treatment network funded by the Canadian Institutes for Health Research, supports Bill C-265 which improves access pathways for medical therapies not commercially available in Canada. Introduced by Dr. Marcus Powlowski, Member of Parliament for Thunder Bay – Rainy River, Bill C-265 moves to second reading in Parliament this week.
Bill C-265 aims to streamline Health Canada’s Special Access Program (SAP) which enables healthcare practitioners to request access to medications that are not approved or commercially available in Canada for patients with serious or life-threatening conditions. Authorized under Canada’s Food and Drug Regulations, the program provides an essential pathway for clinicians seeking urgent access to therapies outside the Canadian market. However, as currently structured, the program also introduces unnecessary red tape that delays patient care.
Each year, physicians and pharmacists submit more than 12,000 individual SAP applications to obtain medications unavailable in Canada. These medications range from World Health Organization-recommended antibiotics for infectious diseases, to specially formulated vitamins for cystic fibrosis, to immunotherapies that support cancer treatments. Navigating this process is time-consuming and frustrating for care providers, patients and families alike, especially when requesting access to therapies they have successfully used before, or that are authorized for sale and established in clinical practice in other trusted international jurisdictions and considered the standard of care.
Bill C-265 will modernize the SAP by creating a ‘Pre-Approved Special Access List’ of safe and effective therapies that physicians will be able to prescribe and import without seeking prior approval from Health Canada. This reform will eliminate thousands of unnecessary applications each year, saving time for physicians, pharmacists and bureaucrats alike and accelerating patient access to treatment and care.
The bill will also introduce flexible, risk-based access pathways that enable patients who have exhausted all other options to access newer, more innovative treatments. This is particularly impactful for the 1 in 12 Canadians affected by a rare disease, as less than 10% of rare diseases have an effective, disease-modifying treatment and only 60% of those treatments are available in Canada. “Clinicians rely on the Special Access Program when no other on-market treatments are available,” said Lori Anderson, Regulatory Affairs Advisor, RareKids-CAN. “Bill C-265 proposes practical changes intended to support more efficient access while maintaining appropriate regulatory oversight.”
RareKids-CAN worked alongside the Child Health Policy Accelerator at SickKids to help inform development of the legislation, drawing on input from clinicians, pharmacists, patients, and families across Canada.
“Proposed reforms through MP Powlowski’s Private Member’s Bill are a critical step toward improving timely access to therapies, ensuring that children, adolescents, and young adults in Canada can receive the effective and innovative treatments they need without unnecessary delay” said Breanne Stewart, Network Director, RareKids-CAN. “This legislation reflects perspectives shared by clinicians, patients and families across pediatric research networks nationally.”
RareKids-CAN encourages parliamentarians to support Bill C-265 and continued efforts to create more efficient, patient-centered pathways for accessing therapies not currently available in Canada.
RareKids-CAN in partnership with the SickKids Child Health Policy Accelerator has initiated a letter writing campaign to gain parliamentary support and will host a national webinar on May 14th featuring MP Marcus Powlowski.