1. Describe how historical policies and institutional risk aversion have contributed to the current "data desert" in maternal health
2. Explain how excluding pregnant populations can shift unstudied risks onto patients and their families
3. Identify approaches to adapting clinical trial designs to include pregnant populations based on risk-benefit considerations and patient-centered considerations
4. Discuss potential approaches to addressing systemic barriers to trial participation, including clear recruitment pathways and improved access to clinical trials

1. Examine practical strategies, benefits, and challenges of conducting trials in pregnant populations
2. Describe strategies for meaningfully integrating patient partners with lived experience with pregnancy and lactation into clinical trials
3. Identify what needs to change for pregnant people to be included more often in clinical trials

Welcome & Housekeeping (Kelly Sandhu)

RareKids-CAN Progress and Priorities: Network Achievements and the Revised Strategic Plan (Breanne Stewart)

Session objectives

• Introduce the revised strategic priorities and explain why they matter to researchers, study teams, and patient and family partners.
• Highlight key achievements, milestones, and contributions from across the network.

Session objectives 

• Share concise updates on active RareKids-CAN initiatives and resources.
• Make connections across trial methods, outcomes, case finding, trial matching, and regulatory pathways.
• Prompt follow-up discussion during the poster session.

Presentations

• Development of a Searchable Inventory of Pediatric Rare Disease Clinical Trial Protocols and Reports - Priscilla Lopes
• Co-Designing Relevant Pediatric Rare Disease Clinical Trial Outcomes: A Template for Patient and Family Partners - Veronica Lai
• Uncovering Rare Disease Cases in Pediatric Hospital Data - Josée Vandal
• Pediatric Rare Disease Clinical Trial Matching in Canada: Current Landscape and Future Directions - Emma Iverson
• Regulatory Pathways, Timelines, and Use of Evidence for Therapies: A Cross-Jurisdictional Comparison for SMA and DMD - Lili Meng

Session objectives

• Describe decentralized trial models and the factors that determine which activities can safely and effectively occur closer to home.
• Examine regulatory, operational, research-team, and patient and family perspectives on implementation.
• Identify practical ways to reduce travel, geographic inequity, and family burden through remote services, monitoring, and satellite follow-up.
• Explore AI tools and the role of patient-reported outcomes.
• Introduce the Patient and Family Travel Fund for Pediatric Rare Disease Clinical Trials.

Presentations

• Welcome, objectives, and speaker introductions - Patient partner session chair – Sara Pot
• East, West, Home is Best: Decentralized Clinical Trials Overview - Marshall Summar
• Health Canada’s Regulatory Guidance on Decentralized Elements- Philippe Mineau
• Lived Experience - Trish Guimond
• CRAFT Framework and Its Application in Pediatric Oncology Clinical Trials - Aiman Siddiqi
• Researcher Perspective - Practical Experience - Tamanna Roshan Lal
• Moderated Panel and Audience Questions - Patient partner moderator – Sara Pot
• Bringing the Trial Home: AI-Enabled Tools and Platforms for Decentralized Pediatric Trials - Lawrence Richer
• RAREKIDS First Mile Fund– Sara Ethier

Session objectives 

• Engage directly with poster presenters and explore methods, findings, and opportunities for collaboration.
• Connect patient and family partners, researchers, study teams, network members, government, and industry.
• Identify shared interests that may lead to future partnerships or projects.

Urgency into Action: A Family's Journey to Transform Access to Life-Changing Therapies - Terry Pirovolakis

Session objectives TBD

• Presentations and activities
• 20 mins Introduction, welcome to the day and presentation | Anisa-Mia Elder
• 5 mins Recorded video 
  

Session objectives

• Excellence in Patient and Family Engagement
• Strongest Alignment with RareKids-CAN Strategic Priorities
• Potential Impact on the Rare Disease Community

Participant instructions: Check your assigned group and move to the listed room for Rotation One. Remain in that room for both rotations. During the break, presenters, facilitators, and scribes rotate rooms; participants do not change rooms.

Session objectives

• Explore the unique challenges and opportunities associated with delivering pediatric advanced therapy medicinal product (ATMP) clinical trials in Canada.
• Compare perspectives from investigators, industry, and patients/families.
• Identify key gaps in institutional and national readiness, particularly for investigator-initiated trials.
• Highlight RareKids-CAN’s work to address these gaps and strengthen ATMP trial readiness.
• Engage attendees in defining a shared vision and priority actions for Canada’s pediatric ATMP clinical trial ecosystem over the next 3–5 years.

Presentations and Activities

• Welcome, Context, and Objectives -Berge Minassian
• From Discovery to Delivery Building the Infrastructure from an Investigator-Initiated Trial Perspective for Pediatric ATMP Trials in Canada- Ana Stosic
• Industry perspective on site selection and readiness - TBD
• Lived Experience - Trish Guimond
• A Family’s Journey: Navigating Pediatric Gene Therapy Clinical Trials - Rachel Rock O’Hagan
• From Challenges to Action: Building National Readiness for Pediatric ATMP Clinical Trials - Catherine Strandt
• Building the Future: A Shared Vision and Priorities for Pediatric ATMP Trials in Canada Engagement Exercise/ Group
• Report-back of key themes and summary – Facilitators/Catherine Strandt

Session objectives

• Appreciate the importance of selecting meaningful trial outcomes.
• Identify available resources that support trial outcome design.
• Understand the importance of using external controls in rare disease studies.
• Identify important characteristics to support data integration.
• Understand the purpose of a single-patient study.
• Appreciate the methodological challenges of undertaking a single-patient study.
  

Presentations and Activities

• Introduction and agenda - Emma Iverson
• Overview of pediatric rare disease clinical trials - Beth Potter
• Externally controlled trials: presentation, table discussion, and Slido - Christopher Gravel
• Outcome selection: presentation, table discussion, and Slido - Martin Offringa and Veronica Lai
• N-of-1 designs: presentation, table discussion, and Slido - Anna Heath
• Synthesis, buffer, and closing - Emma Iverson

Participants remain in their assigned rooms

Session objectives

• Appreciate the importance of selecting meaningful trial outcomes.
• Identify available resources that support trial outcome design.
• Understand the importance of using external controls in rare disease studies.
• Identify important characteristics to support data integration.
• Understand the purpose of a single-patient study.
• Appreciate the methodological challenges of undertaking a single-patient study.
  

Presentations and Activities

• Introduction and agenda - Emma Iverson
• Overview of pediatric rare disease clinical trials - Beth Potter
• Externally controlled trials: presentation, table discussion, and Slido - Christopher Gravel
• Outcome selection: presentation, table discussion, and Slido - Martin Offringa and Veronica Lai
• N-of-1 designs: presentation, table discussion, and Slido - Anna Heath
• Synthesis, buffer, and closing - Emma Iverson

Session objectives

• Explore the unique challenges and opportunities associated with delivering pediatric advanced therapy medicinal product (ATMP) clinical trials in Canada.
• Compare perspectives from investigators, industry, and patients/families.
• Identify key gaps in institutional and national readiness, particularly for investigator-initiated trials.
• Highlight RareKids-CAN’s work to address these gaps and strengthen ATMP trial readiness.
• Engage attendees in defining a shared vision and priority actions for Canada’s pediatric ATMP clinical trial ecosystem over the next 3–5 years.

Presentations and Activities

• Welcome, Context, and Objectives -Berge Minassian
• From Discovery to Delivery Building the Infrastructure from an Investigator-Initiated Trial Perspective for Pediatric ATMP Trials in Canada- Ana Stosic
• Industry perspective on site selection and readiness - TBD
• Lived Experience - Trish Guimond
• A Family’s Journey: Navigating Pediatric Gene Therapy Clinical Trials - Rachel Rock O’Hagan
• From Challenges to Action: Building National Readiness for Pediatric ATMP Clinical Trials - Catherine Strandt
• Building the Future: A Shared Vision and Priorities for Pediatric ATMP Trials in Canada Engagement Exercise/ Group
• Report-back of key themes and summary – Facilitators/Catherine Strandt

Session objectives

• Provide an update on RareKids-CAN's Health Canada relationships and advocacy priorities.
• Explain the standard-of-care clinical trial exemption announcement and its relevance.
• Introduce the Therapies Unfit for Commercialization and HEAL-CAN pathway.
• Use the virus-specific T-cell demonstration project to illustrate a practical evidence-and-access model.
• Examine reimbursement challenges and identify opportunities for the network community to contribute.

Presentations and Activities

• Health Canada Relations, Advocacy and Regulatory Reform Update - Charlotte Moore-Hepburn
• Health Canada Pediatric Standard of Care Clinical Trials Pilot and Q&A- Natasha Rastogi and Eden Story
• Therapies Unfit for Commercialization- Breanne Stewart
• Harnessing Evidence for Access and Learning Canada (HEAL-CAN) Overview and Virus-Specific T-cell demonstration project - Lori Anderson
• Reimbursement for Therapies Unfit for Commercialization – Gwendolyn Moncrieff-Gould