RareKids-CAN is committed to transforming Canada’s regulatory and clinical research environment to ensure equitable access to safe and effective therapies for children with rare diseases. Driven by our partnership with the Sick Kids Child Health Policy Accelerator (CHPA) – we are working to modernize policies and streamline processes that directly impact pediatric patients and their families.
Authors
Lori Anderson
Gwendolyn Moncrieff-Gould
Charlotte Moore Hepburn
Page last reviewed: 27-Aug-2026
Key takeaway
- Children are still missing important evidence about the medicines they receive, with many pediatric medications being used “off-label” because they were not specifically studied or approved for children
- Canada’s current clinical trial rules can make pediatric research unnecessarily difficult and expensive, even when a medication is already routinely used and considered safe in children
- Regulatory burden can prevent research from happening as some trials are delayed, become too costly, or are abandoned altogether, limiting the evidence available to improve pediatric care
- Other countries use more risk-based approaches. For example, the U.S., Australia, and U.K. have pathways that reduce or eliminate regulatory requirements for low-risk studies of commonly used medicines while maintaining safety and ethics oversight
- The paper calls for a Canadian exemption pathway for low-risk pediatric trials, allowing researchers to study routinely used medicines with strong existing evidence while continuing to protect children through ethics boards, informed consent, and institutional safety oversight
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