Conference Program

Download a short version of the agenda in PDF format HERE

Select Date: -- Select a date -- April 10 April 11 Pre-Clinical Pipeline Workshop

April 10th Agenda

Time	Event	Description
12:00 - 1:00 PM	Welcome Lunch	Location: Tulip Restaurant
1:00 - 1:10 PM	Welcome and Land Acknowledgement [Thierry Lacaze]
1:10 - 1:20 PM	Canadian Rare Disease Network (CRDN) [Svenja Espenhahn]	The Canadian Rare Disease Network (CRDN) is a pan-Canadian, cross-disciplinary initiative uniting leading clinicians, researchers, and patient advocates to address the unique challenges faced by the millions of Canadians affected by rare diseases. This session will explore CRDN's mission to advance care, research, and innovation through its interconnected pillars, which focus on improving diagnosis, treatment access, and patient support.
1:20 - 1:50 PM	RareKids-CAN Year in Review [Breanne Stewart]	High-level summary of RareKids-CAN's activities over the past year, highlighting key milestones and achievements in advancing pediatric rare disease clinical trials and treatments in Canada.
1:50 - 2:00 PM	Speed Networking – Meet & Greet [Breanne Stewart]	Attendees will engage in a fast-paced networking session, introducing themselves to two individuals they have not yet met. This "speed dating" style activity encourages new connections and collaboration within the rare disease research community.
2:00 - 2:15 PM	Showcase Initiative - Strengthening Rare Disease Research and Facilitating Collaboration: Insights from Clinical Trial Navigators [Kelly Sandhu]	This session will showcase the impactful work of Clinical Trial Navigators (CTNs) within the RareKids-CAN network over the past year. Key initiatives include the collection of local research institution metrics, the development of an expert database, and the identification of sites and investigators to support pediatric rare disease clinical trials. Additionally, the session will highlight efforts to strengthen relationships with clinicians and healthcare professionals in the rare disease space, fostering collaboration and enhancing research capacity across Canada.
2:15 - 2:30 PM	Showcase Initiative - Pediatric Rare Disease Clinical Trials Inventory: A Living Scoping Review [Beth Potter / Emma Iverson]	This session will highlight a comprehensive scoping review project that unites several RareKids-CAN sub-platforms in a collaborative effort to maximize efficiency and impact. The project aims to develop a living inventory of pediatric rare disease clinical trials, providing a crucial summary of the current landscape in this field.
2:30 - 2:45 PM	Showcase Initiative - Partnership in Action: Updates on RareKids-CAN and Family Engagement in Research [Sara Pot / Alicia Hilderley]	This session will explore the collaborative efforts within RareKids-CAN, highlighting how partnerships with patients and families drive progress in pediatric rare disease clinical trials. It will also provide updates on the Family Engagement in Research course, emphasizing its role in fostering meaningful research collaborations between families and researchers.
2:45 - 2:55 PM	Ceremonial Welcome [Elder Vincent Kicknosway]
2:55 - 3:15 PM	Break
3:15 - 3:35 PM	Update on Standard of Care Clinical Trial Application Exemption: Advocacy and White Paper [Charlotte Moore Hepburn]	Dr. Charlotte Moore Hepburn, HC Relations Sub-Platform Lead, together with Gwendolyn Moncrieff-Gould (Policy Lead, SickKids Child Health Policy Accelerator) and Lori Anderson (Regulatory Consultant) will present an overview of the current RareKidsCAN advocacy initiatives aimed at reducing administrative burden for pediatric clinical trials studying off-label therapies used as standard of care. This update will illustrate the federal regulatory advocacy process and highlight proposed regulatory changes and their potential impact on pediatric research.
3:35 - 3:45 PM	Pt/Family Experience with the Special Access Program [Jason Arsenault]
3:45 - 3:55 PM	Challenges in Accessing Treatment: A Clinician’s Perspective [Nicola Wright]
3:55 - 5:00 PM	Health Canada Relations & Advocacy Sub-Platform: Special Access Program & Single Patient Studies [Charlotte Moore Hepburn]	Access Pathways Review and Brainstorming: - We will review definitions and current status of the federal Special Access Program (SAP), Open Label Individual Patient (OLIP) trials, and Single Patient Studies (SPS). - Attendees will participate in breakout sessions to discuss the challenges, benefits, and gaps in these existing programs and brainstorm legislative and regulatory solutions to address unmet needs, including exploring potential new policy pathways for accessing highly investigational therapies.
5:00 - 5:15 PM	End of Day Wrap-Up [Breanne Stewart]
6:00 - 9:00 PM	Dinner	Location: Monsella Room
7:00 PM	Keynote Session-Innovative Frontiers: Gene Therapy and Potential Sustainable Access Models for Rare Diseases	This keynote session will feature two distinguished speakers addressing critical challenges and solutions in rare disease therapies: - Dr. Donald Kohn will share his groundbreaking work on Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency (ADA-SCID). Drawing from his extensive experience, Dr. Kohn will provide an overview of this life-saving clinical trial, highlighting its successes, challenges, and the pathway toward FDA approval to make the therapy widely accessible. - Paul Ayoub, CEO of Rarity PBC, will introduce the concept of Public Benefit Corporations as a transformative approach to addressing the high costs of therapies for rare diseases. He will discuss how prioritizing public good over profit can create sustainable models for developing and delivering innovative treatments to patients in need. This session will offer valuable insights into cutting-edge advancements and innovative frameworks aimed at improving access to rare disease therapies globally.

April 11th Agenda

Time	Event	Description
7:00 - 8:00 AM	Breakfast	Location: Foyer | Monsella Room
8:00 - 8:10 AM	Summary from Day 1 [Breanne Stewart]
8:10 - 9:15 AM	Precision Medicine: National Initiatives and Collaborations	- Precision Health Initiative | BC Children's Hospital [Stuart Turvey] - Precision Child Health Partnership | Sick Kids- St. Justine [Tomasz Czarny] - Precision Medicine Initiative | University of Calgary [Nicola Wright] - D2R, DNA to RNA | McGill University [Ma’n Zawati] - Q&A Session
9:15 - 10:00 AM	Guest Speaker: International Initiative for Tackling the High Cost of Pediatric Rare Disease Therapies: Insights from AGORA [Dr. Arjan Lankester]	An international perspective on addressing the economic challenges of accessing gene therapies for rare pediatric diseases. The focus will be on AGORA (Access to Gene Therapies for Rare Disease), a consortium uniting academic groups, regulators, funders, and patient advocates to develop sustainable solutions for equitable access to life-saving treatments. Dr. Lankester will explore the hurdles in commercializing gene therapies, highlight innovative approaches to reducing costs, and discuss potential pathways for ensuring these transformative therapies reach the children who need them most.
10:00 - 10:15 AM	Break
10:15 - 11:50 AM	Shaping the Future of RareKids-CAN: Strategic Planning and Gap Analysis [Breanne Stewart / Thierry Lacaze / TBD]	This session will focus on RareKids-CAN's strategic direction for the coming years. The discussion will encompass: 1. Gap Analysis: We will reflect on the insights gained from our guest speakers and presentations to identify current gaps in the Canadian pediatric rare disease landscape. 2. RKC's Role: We will explore how RareKids-CAN can leverage its unique position to address these identified gaps, considering our network's strengths and capabilities. 3. Future Initiatives: Participants will brainstorm potential new initiatives and areas for resource investment over the next four years. This strategic planning session aims to align RareKids-CAN's future efforts with the evolving needs of the pediatric rare disease community in Canada, ensuring we continue to make meaningful progress in improving health outcomes for children affected by rare diseases
11:50 AM - 12:00 PM	Wrap Up/Event Summary [Breanne Stewart]
12:00 - 1:00 PM	Lunch	Location: Foyer
12:00 - 1:00 PM	RareKids-CAN Executive Committee Lunch Meeting [Invite-Only]	Location: Ballad Room
1:00 - 4:00 PM	Pre-Clinical Pipeline Workshop [Invite-Only]	Location: Monsella Room

Pre-Clinical Pipeline Workshop Agenda

Time	Event	Description
1:00 - 1:20 PM	Welcome & Introductions [Leanne Ward & All]
1:20 - 2:00 PM	Pre-Clinical Drug Development in Canada: Who, What, Why, and Where We Go from Here [Eric Hoffman]	Dr. Eric Hoffman, CEO and Co-founder of AGADA Biosciences and ReveraGen Biopharm, will share insights from his extensive experience in therapy development and regulatory approval. His presentation will explore the key milestones and checkpoints that guide a therapy from discovery to clinical trials, the challenges encountered along the way, and effective strategies to overcome them. Dr. Hoffman will also discuss the vital role of rare disease networks, government, universities, and research institutes in advancing pre-clinical drug development. Finally, he will highlight the importance of strengthening Canada’s pre-clinical drug development pipeline to foster innovation and improve patient outcomes.
2:00 - 2:45 PM	Breakout/Group Discussion: Identifying Challenges and Gaps	Attendees will break into small groups to discuss the key challenges in the pre-clinical therapy development process, and identify existing gaps. Each group will then share their insights with the larger audience to foster collaborative discussion and knowledge exchange.
2:45 - 3:00 PM	Break
3:00 - 3:45 PM	Breakout/Group Discussion: Identifying Strengths and Opportunities	Attendees will break into small groups to explore Canada's existing strengths and resources that support pre-clinical therapy development. Discussions will focus on opportunities to enhance domestic drug development efforts and identify key requirements for advancing therapies more efficiently within Canada.
3:45 - 4:00 PM	Closing & Next Steps [Leanne Ward]

Hilton Garden Inn Floor Plan can be viewed HERE

Please note that all conference events will be held on the ground floor of the hotel. 
The main conference sessions, as well as the dinner, will take place in the Monsella Room. 
Other meals and breaks will be served either in the Tulip Restaurant (if so indicated in the agenda) or in the foyer near the Monsella Room. 
Information on breakout rooms for specific parts of the conference can be found HERE