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Advocacy
We work to advance equitable access to diagnosis, treatment, and lifelong care for individuals affected by PKU and allied disorders across Canada.
CanPKU+ engages in non-partisan public policy dialogue and development activities that support our charitable purposes of relieving sickness, advancing education, and advancing health. We work with families, clinicians, policymakers, and system leaders to improve access, equity, and outcomes. Our advocacy is evidence-informed and independent of sponsor influence.

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Advocacy Articles
Stay informed with advocacy news, drug access updates, consultations, awareness initiatives, policy developments, and patient input opportunities.
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Advocacy
Lived experience strengthens policy solutions.
Focus Areas
Federal
  • Access pathways and reimbursement fairness
  • Regulatory modernization for medical foods and formulas
  • Rare disease strategy implementation
  • Disability Tax Credit equity
Provincial and Territorial
We advocate for equitable access to care and funding across provinces and territories, recognizing that access varies significantly by region.
Newborn Screening
We support timely and comprehensive newborn screening, including expanded screening for conditions such as HCU, where medically appropriate.
Current Priority Areas
Access to Sephience Access to Pegzilarginase Newborn Screening
Advocacy Toolkit
Practical guidance to help individuals communicate effectively with decision-makers. Materials are non-partisan and intended to support informed dialogue.
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Stories Drive Change
Lived experience helps policymakers and system leaders understand the real-world impact of access barriers, delayed diagnosis, and treatment inequities. Sharing your experience contributes to informed, non-partisan public policy dialogue and supports improved health outcomes.
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Upcoming Events
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Below is a historical overview of CanPKU+ advocacy, public engagement, education initiatives, and policy participation efforts in support of individuals and families affected by PKU and allied disorders across Canada.
2026
  • Submitted a patient input submission to Canada's Drug Agency (CDA) on behalf of the Canadian PKU community as part of the review of Sephience™ (sepiapterin). The submission incorporated findings from the national patient and caregiver survey conducted in late 2025, along with one-on-one interviews describing the lived experience of PKU and the impact of current treatment.
  • Submitted a separate patient input submission to Quebec's Institut national d'excellence en santé et en services sociaux (INESSS) as part of its evaluation of Sephience™, ensuring that the experiences of individuals and families affected by PKU were represented within Quebec's review process.
  • Continued the Advocacy in Action program, creating provincial working groups in QC and AB
  • Participated in national discussions related to medicine access, health-system policy, and drug shortages, including stakeholder engagement through the Best Medicines Coalition and related patient advocacy networks.
  • Contributed patient organization perspectives to the development of the pan-Canadian Pharmaceutical Alliance's 2026-2029 Strategic Plan, which outlines future priorities related to people, process, performance, and strategic relationships.
  • Supported broader rare disease advocacy initiatives, including efforts encouraging governments and healthcare decision-makers to recognize the realities faced by Canadians living with rare and inherited metabolic disorders.
  • Continued developing advocacy education resources and website content to help community members better understand Health Canada approval, health technology assessment, reimbursement negotiations, and provincial and territorial drug funding decisions.
2025
2024
  • Supported advocacy capacity-building through completion of formal advocacy training by team members.
  • Launch of the Canadian PKU Registry (May 2, 2024): Read announcement
2023
  • Published Kuvan Fact Sheet (January 2023): Open document
  • Contributed patient perspective to national discussions regarding PMPRB reforms: Read article
2020 - 2022
  • Submitted formal comments to federal consultations, including PMPRB Draft Guidelines (2020), emphasizing the importance of patient access to medicines for rare disorders.
  • Participated in parliamentary and committee-level discussions relating to rare disease medicine access: House of Commons Health Committee record
  • Shared educational resources regarding how Canadian laws and regulations are developed: Justice Canada infographic
2013 - 2019
  • Engaged in national medicine-policy discussions through coalition participation (Best Medicines Coalition), ensuring rare disorder patient perspectives were represented.
  • Supported public funding approvals of Kuvan in multiple provinces beginning in 2013.
  • Advanced awareness of national PKU treatment guidelines and equitable access considerations: Newswire article
  • Supported provincial funding decisions for low-protein medical foods and formulas in multiple jurisdictions (2012–2015).
Foundational Years (2003 - 2012)
  • Grassroots advocacy efforts in multiple provinces contributed to maintaining access to medical formulas and improving standards of care.
  • Patient submissions to Common Drug Review processes regarding Kuvan.
  • CanPKU formally organized in 2008 to provide a national voice for individuals and families affected by PKU.
  • Earlier community-led efforts in 2003 to preserve adult formula access in Ontario helped catalyze later national organization.