Policy Briefs and White Paper

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Policy Advocacy and Regulatory Reform

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.

Our collaborative initiatives aim to remove barriers, promote innovation, and accelerate access to life-saving treatments. We are working on the following policies:

  • PRACTICE: Simplifying and reducing unnecessary regulations for clinical research involving treatments already proven safe and effective, supporting faster innovation and improved access.

  • RARE-SAP: Reforming the Special Access Program (SAP) and Single Patient Study (SPS) systems to improve timely access to life-saving treatments for critically ill children.

  • Precision Regulation: Fast-tracking market approval for drugs listed on the National Priority List of Pediatric Drugs, ensuring children are not left behind in therapeutic innovation.

  • Pediatric Data Equity: Ending Canada’s longstanding reliance on “off-label” prescribing by making the Pediatric Rule permanent and ensuring equitable, evidence-based care for children.

  • RARE-IMD: Advancing Regulatory Advocacy for Reform and Enhancement of Investigational Medical Devices to streamline the approval and oversight of pediatric devices.

Together, these initiatives represent RareKids-CAN’s ongoing commitment to evidence-driven policy reform—ensuring that every child in Canada has timely access to the therapies they need.

Read our policy briefs and white papers below:

PRACTICE Trials: Regulatory Reform to Advance Evidence-Based Pediatric Care