2025 Agenda
2025 Agenda
7:30am - 8:00am
Conference Registration and Continental Breakfast
8:00am - 8:10am
NORD’s Welcome & Summit Preview
Pamela Gavin, CEO, NORD
8:10am - 8:40am
Young Voices Keynote Panel
Advances in rare disease diagnosis and treatment have particular relevance for young people living with rare diseases. Young advocates will share their stories, challenges and hopes for the future.
8:40am - 9:00am
FDA Address
9:00am - 9:20am
NIH Address
9:25am - 10:25am
CEO Perspectives
CEOs working in the rare space will share their thoughts on the current landscape for advancing science, addressing cost concerns and ensuring that patients and patient needs are the fundamental driving factor.
Moderator: Patrick Collins, VP of Community & Corporate Affairs, NORD
- Pamela Gavin – CEO, NORD
- Catherine Owen Adams, BSc. – CEO, Acadia
- Paula Ragan, PhD – CEO and President, X4 Pharmaceuticals
- Miguel Fernandez Alcalde – President, EMD Serono
10:30am - 11:00am
Networking Break
11:00am - 11:55am
A Fireside Chat with FDA Leadership (invited)
FDA senior officials will sit down with NORD’s CEO for a conversation about their hopes and priorities and, in particular, how they view the future of rare disease product development and review.
12:00pm - 12:30pm
Lightning Rounds Poster Presentations
Authors of the top selected poster abstracts will share their key findings.
12:30pm - 1:55pm
Lunch
2:00pm - 3:55pm
Breakout Sessions
ROOM 1
2:00 – 2:10 pm: Community Voices
2:10 – 3:05 pm: A New Era for Rare Disease Clinical Trials
This session will explore how technology and innovative trial designs address issues that historically have made clinical trials for rare diseases particularly challenging.
3:10 – 4:05 pm: A Moral Imperative: Addressing the Challenges of Pediatric R & D
Even though about two-thirds of Americans with rare diseases are children, pediatric R & D lags far behind adult studies. What are the special challenges of pediatric research and how do we address them?
Room 2
2:00 – 2:10 pm: Community Voices
2:10 – 3:05 pm: Earlier Diagnosis to Promote Better Outcomes
It’s well established that, for many rare diseases, earlier diagnosis can greatly improve long-term outcomes. What are the current challenges related to newborn screening, genetic testing and genomic sequencing, and how do we address them?
3:10 – 4:05 pm: Rare Disease Communities of Care: Integrating Mental Health Support into Treatment
Rare disease patients and their families face unique mental health challenges that are often overlooked. This session will explore how mental health support can be integrated into the holistic care model for rare disease patients.
4:05pm - 4:30pm
Networking Break
4:30pm - 5:30pm
Curative Therapies: Opportunities and Challenges
This panel of investors and entrepreneurs will share their perspectives on the outlook for rare disease investment in 2026 and beyond.
5:30pm
Exhibit Hall and ‘Sip and Science’ Poster Hall Reception
7:30am - 8:15am
Continental Breakfast
8:15am - 8:45am
Day 2 Community Voice Keynote
8:50am - 9:45am
Making Your Voice Heard: Rare Disease Advocacy and the 119th Congress
Representatives of the Rare Disease Congressional Caucus will share their thoughts on current and upcoming policy priorities, and how patient advocates can have an impact.
9:50am - 10:45am
Case Studies in Collaboration
When patient organizations and companies work together in ways that are ethical, practical and creative, patients and families benefit. This session will look at case studies of collaboration and the best practices they illustrate.
10:45am - 11:15am
Networking Break
11:15am - 12:10pm
How FDA Review Teams Incorporate Patient Data
Panel composed entirely of FDA reviewers sharing their thoughts and first-hand experiences.
12:15pm - 1:25pm
Networking Lunch
1:30pm - 1:40pm
Community Voices: Patient/Caregiver Story
1:40pm - 2:35pm
What’s Next for Gene and Cell Therapies?
This panel will discuss the current state-of-the-art, advances on the horizon and policy and economic challenges.
2:40pm - 3:35pm
The Role of AI in Drug Development
How can we harness the power of AI to expedite rare disease drug development, while still ensuring safety and efficacy? This panel of experts will explore the many ways AI is being utilized to accelerate and transform drug discovery.
3:40pm - 4:35pm
Closing Session: Next Steps for Optimal Impact
NORD’s senior leadership and community thought leaders will share perspectives on current priorities, next steps and how to have optimal impact together in science, public policy and patient care.
4:35pm
Closing Remarks
Pamela Gavin, CEO, NORD
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