Who Should Attend?
Are you committed to advancing the conversation on rare diseases and bringing more new treatments to market?
Join over 900 rare disease advocates, experts, and stakeholders from around the world to tackle the most pressing issues facing the rare disease community.
Pharma and Biotech
Learn about the latest trends in rare disease and orphan product development directly from top FDA leaders, connect with other industry professionals to share best practices and explore new business ventures, and build relationships with patient organization leaders and advocates.
Government Officials and Regulators
Understand the impact of the current policy and regulatory environment for orphan products and the rare disease community. Hear perspectives from across the rare disease ecosystem about how stakeholders are working together in this exciting era of scientific advancement.
CROS and Solutions Companies
Connect with your current clients and cultivate prospects who require your expertise and services to develop their pipelines, conduct successful trials and studies, and address their commercialization needs.
Patient Advocacy Leaders
Understand the impact of the current policy and regulatory environment for orphan products and the rare disease community. Hear perspectives from across the rare disease ecosystem about how stakeholders are working together in this exciting era of scientific advancement.
Researchers and Scientists
Engage with cutting-edge research and innovative solutions from across the rare disease landscape. Network with peers and explore collaboration opportunities to drive your work forward.
Why Attend?
Relevant and Dynamic Agenda
Sessions offering real-world, actionable information on critical topics such as:
- Newborn Screening
- Balancing Cost, Innovation and Access
- Enhancing Patient Data
- Genetic Testing
- Orphan Product Investment
- The Evolving Policy Landscape
Network with Influencers and Rare Disease Community Stakeholders
Forge meaningful connections and collaborate with influential stakeholders across the rare disease community.
- Take advantage of 1:1 networking meetings — Meetings can be scheduled through the event App. Details will be shared via email when the app opens ahead of the event.
- Roundtable discussion groups
- Join fellow attendees at the Welcome Reception
- Engage with others during the Poster Hall and Exhibit Hall Reception
- Connect face-to-face and build relationships with 65+ sponsors and exhibitors
Opportunities to Learn About Research and Public Health Projects
View 70+ posters from fellow academics, researchers, industry, government agencies, healthcare professionals, patient organizations, and students. The top selected poster abstracts will be presented on the main stage during lightning round presentations.