2026 Agenda
8:00am - 12:00pm
IAMRARE® Registry Meeting
*By invitation only
Advanced registration required. Please contact [email protected] if you are interested in attending.
10:00am - 5:00pm
Rare Disease Advisory Council Meeting
*By invitation only
Advanced registration required. Please contact [email protected] if you are interested in attending.
12:00pm - 5:30pm
Annual Membership Meeting
*By invitation only
Advanced registration required. Please contact [email protected] if you are interested in attending.
2:00pm - 8:00pm
Registration
Save time Monday morning by picking up your badge early and joining us for a welcome reception.
2:00pm - 5:00pm
Student Leaders Meeting
*By invitation only
Advanced registration required. Please contact [email protected] if you are interested in attending.
6:00pm - 7:30pm
Welcome Reception
7:30pm
Rare Cancer Coalition® Cocktail Reception
7:30am - 8:15am
Conference Registration and Continental Breakfast
8:30am - 8:35am
NORD’s Welcome & Summit Preview
Pamela Gavin, Chief Executive Officer, NORD
8:40am - 8:45am
Rare Impact Award® Presentation: Abbey S. Meyers Leadership Award
8:50am - 8:55am
Opening Patient/Caregiver Keynote
9:00am - 9:10am
FDA Commissioner Keynote Address
Kyle Diamantas, J.D., Acting Commissioner of Food and Drugs, U.S. Food and Drug Administration (FDA)
9:20am - 9:35am
NIH Director Remarks
Jayanta “Jay” Bhattacharya, MD, PhD, Director, National Institutes of Health (NIH)
9:40am - 10:25am
From Patient Voice to Patient Partnership
Move beyond patient input to meaningful partnership. This session explores how patients, advocates, clinicians, and industry can work together to accelerate research, therapeutic development, and better care.
Moderator: Pamela Gavin, Chief Executive Officer, NORD
Panelists:
- Tracy Hart, CEO, Osteogenesis Imperfecta Foundation
- Uthra Sundaram, MBA, Senior Vice President, Business Unit Head, USBU Foundational Therapeutics Platform (FTP); Takeda Pharmaceuticals U.S.A., Inc.
- V. Reid Sutton, MD, Professor of Molecular & Human Genetics, Baylor College of Medicine & Texas Children’s Hospital, a NORD Rare Disease Center of Excellence
10:30am - 10:55am
Networking Break
11:00am - 11:55am
The Next Decade of Rare Disease: A CEO Leadership Dialogue
Hear from chief executives whose organizations have helped shape the rare disease landscape through scientific innovation, sustained investment, and commitment to patients. This discussion will examine the strategic decisions, partnerships, and leadership needed to accelerate progress for patients and families living with rare diseases over the coming decade.
Panelists:
- Renee Gala, President and CEO, Jazz Pharmaceuticals
- Alexander Hardy, MBA, President and CEO, BioMarin
- Tarek Rabah, President and CEO, Otsuka
12:00pm - 12:25pm
Lightning Round Poster Presentations
12:30pm - 1:45pm
Networking Lunch
1:50pm - 1:55pm
Rare Impact Award® Presentation: Youth Champion Award
2:00pm - 2:45pm
Beyond One Disease at a Time: The Future of Individualized Genetic Therapies
Customized genetic therapies are now a scientific reality, allowing the rare disease field to shift toward a scalable, personalized approach to treatment. This session will examine the convergence of platform technologies and individualized therapies, highlighting how academic institutions, nonprofit organizations, industry, and patient communities are collaborating to accelerate development and expand access to transformative treatments.
Panelists:
- Steven Gray, PhD, Professor, UT Southwestern Medical Center, a NORD Rare Disease Center of Excellence
- Janet Woodcock, MD, Former Director, Center for Drug Evaluation and Research, FDA
2:50pm - 2:55pm
Rare Impact Award® Presentation: Lifetime Achievement Award
3:00pm - 3:55pm
Beyond Approval: Getting Breakthroughs to Patients
Approval is a milestone — not the finish line. Explore how stakeholders can work together to overcome coverage, affordability, and access barriers so innovation reaches the patients who need it.
Panelists:
- Elizabeth Ames, MD, PhD, Clinical Associate Professor of Pediatrics, University of Michigan Health, a NORD Rare Disease Center of Excellence
4:00pm - 4:10pm
Networking Break
4:15pm - 5:00pm
Unlocking Investment in Rare Disease Innovation
This session will highlight how patient advocacy, diagnostic innovation, research advancements, regulatory pathways, policy frameworks, and reimbursement strategies collectively shape the investment landscape and capital formation for rare disease treatment development.
Moderator: David Scheer, President, Scheer & Company, Inc.
Panelists:
- Pamela Gavin, CEO, NORD
- Martin Mackay, PhD, Co-Founder and Chair, RallyBio
- Sean Nolan, CEO, Taysha Gene Therapies
- Camille Samuels, Board Director and Venture Investor
- Stephen Squinto, PhD, Chief Investment Officer, J.P. Morgan Life Science Private Capital
5:30pm
Exhibit Hall and ‘Sip ‘n Science’ Poster Hall Reception
7:30am - 8:00am
Continental Breakfast and Registration
8:15am - 8:25am
Rare Impact Award® Presentation: Policy Changemaker Award
8:30am - 8:40am
Day 2 Opening Patient/Caregiver Keynote
8:45am - 9:45am
FDA Rare Disease Regulatory Roundtable (invited)
Leadership from CDER, CBER, and CDRH will share the latest regulatory perspectives, priorities, and developments shaping rare disease medical product development.
Panelists:
- Michelle E. Tarver, MD, PhD, Director, CDRH
9:50am - 12:15pm
Breakout Sessions
TRACK A
9:50 a.m. Closing the Access Gap in Precision Oncology
Precision oncology is creating new opportunities for people with rare cancers, but access to biomarker testing and the treatments or clinical trials it may identify remains uneven. Using a real-world scenario, this session will examine key barriers and policy solutions to expand equitable access.
10:45 a.m. Networking Break
11:20 a.m. The Next Rare Disease Policy Agenda: Congress, Innovation, and What Comes Next
Bipartisan congressional staff and health policy leaders will discuss the evolving legislative landscape for rare diseases, including the outlook for the remainder of 2026, user fee reauthorization, FDA and NIH modernization and oversight, and U.S. global competitiveness in medical innovation.
TRACK B
9:50 a.m. Finding Answers Faster: Advancing Rare Disease Diagnosis
*More details to follow
10:45 a.m. Networking Break
11:20 a.m. Rare Disease Centers of Excellence in Action: Advancing Diagnosis, Care, and Research
Through real-world examples, NORD Rare Disease Centers of Excellence leaders will share strategies that are improving diagnostic pathways, strengthening multidisciplinary and coordinated care, advancing research, and connecting patients with expertise and opportunities.
Moderator: Marybeth McAfee, MA, GC, Vice President, Community and Medical Affairs, NORD
Panelists:
- Beth Kozel, MD, PhD, Chief of the Division of Genetics and Genomic Medicine and the Director of Constitutional Genomics Translational Research, Nationwide Children’s Hospital, a NORD Rare Disease Center of Excellence
- John A. Morren, MD, MEd, FAAN, FAANEM, Director, Cleveland Clinic Lerner College of Medicine of Case Western Reserve University
- Natasha Shur, MD, Division Chief, Genetics and Metabolism, Medical Geneticist, Children’s National Hospital
12:20pm - 1:25pm
Networking Lunch
1:30pm - 1:40pm
Rare Impact Award® Presentation: Community Champion Awards
1:45pm - 2:40pm
Breakout Sessions
TRACK A
1:45 p.m. States in Transition: What Policy Changes Mean for Rare Disease
This panel will explore the changing health policy landscape across the states, including the changes to Medicaid, and what these shifts could mean for patients, health care systems, and innovation.
TRACK B
1:45 p.m. Knowledge Changes Lives: Building Better Rare Disease Care
Education is a cornerstone of better outcomes in rare disease. From empowering patients and caregivers to equipping clinicians with the knowledge to recognize, diagnose, and manage rare conditions, education drives earlier intervention, stronger partnerships, and more coordinated care.
Moderator: Debra S. Regier, MD, PhD, Director, Ward Winslett Center for Rare Diseases, Atrium Health Levine Children’s Hospital, Advocate Health, a NORD Rare Disease Center of Excellence
Panelists:
- Reena Kartha, MS, PhD, Associate Professor & Associate Director, Center for Orphan Drug Research, Experimental and Clinical Pharmacology, University of Minnesota
- Lindsay Rosen, Vice President, Strategy, Medlive
- Ben Shaberman, MS, MA; Science Communications Advisor, Hope in Focus
- Byeong Yoon, Vice President of Medical, Rare Disease, Amgen
2:45pm - 2:50pm
Quick Break
3:00pm - 3:55pm
Closing Plenary – From AI to Impact: Making AI Work for Rare Disease Patients
Artificial intelligence has the potential to fundamentally reshape the rare disease ecosystem — but realizing its promise requires intentional collaboration. Join leaders from across the community to discuss how AI can strengthen partnerships, accelerate innovation, and improve patient outcomes while ensuring every advancement reflects the priorities and experiences of people living with rare diseases.
Moderator: Pamela Gavin, Chief Executive Officer, NORD
Panelists:
- Elizabeth Hart, MD, Associate Director for Rare Diseases, CDER, FDA
4:00pm
Closing Remarks
Pamela Gavin, Chief Executive Officer, NORD
8:00am - 12:30pm
NORD Corporate Council Meeting
NORD Corporate Council member company representatives are welcome to attend. Please email [email protected] for the registration link.
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