2026 Draft Agenda
How do we speed up the development of life-changing therapies for rare diseases while keeping trials accessible, patient-centered, and scientifically sound?
Monday, September 14:
08:00 AM - Registration Opens
09:00 AM – 12:00 PM | Accelerate RARE Working Group A: Evidence Standards, AI Integration, and Global Acceptance of Novel Data Sources.
01:00 PM – 04:00 PM | Accelerate RARE Working Group B: Platform Trials, Expedited Pathways, and US-EU-China Regulatory Coordination.
04:00 PM – 05:30 PM | Movie Screening (TBD)
05:30 PM – 07:30 PM | Welcome Reception (Exhibit Hall)
Tuesday, September 15:
07:30 AM – 08:15 AM | Networking Breakfast (Exhibit Hall)
08:15 AM – 08:45 AM | OPENING KEYNOTE - DAY 1
Theme: The Future of Rare Disease Regulation in a Fragmented Global Landscape. How US, EU, and China regulatory environments are diverging or converging; the role of AI in evidence generation; global pathways; and the next decade of rare-disease approvals.
Track 1 - Patient Identification and Enrollment
Morning Set: Precision Starts with Finding Them
09:00 – 09:30 | Presentation: Beyond the Registry: Integrating Genomics, Real-World Data, and Advocacy Networks. How AI-driven matching, cross-border registries and privacy frameworks enable identification at scale.
09:45 – 10:30 | Fireside Chat: From Awareness to Activation - Turning 1000 Genotypes into 10 Enrolled Patients. Real cases spanning US-EU-China recruitment pipelines; cultural, regulatory, and consent variations.
10:45 – 12:00 | Workshop: Mapping the Rare Patient Journey. Teams identify global drop-off points, data-access barriers, and cross-region consent pitfalls.
12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)
Afternoon Set: Global Reach and Retention
01:00 – 01:30 | Presentation: Global Patient Access: Navigating Cross-Border Enrollment, Consent, and Data Sovereignty. What new national data-protection and AI laws mean for multi-region trials.
01:45 – 02:30 | Fireside Chat: Retention by Design - Keeping Families Engaged for Multi-Year Studies. Global caregiver perspectives; remote monitoring; differing regulatory requirements for home-health.
02:45 – 04:15 | Workshop: Building a Patient-First Recruitment Playbook. Teams incorporate global registries, AI triage, and region-specific barriers.
Track 2 - Operational Efficiency through Decentralization and Technology
Morning Set: From Concept to Compliance
09:00 – 09:30 | Presentation: Hybrid & Home-Based Trials: What Works at Global Scale. Real-world performance metrics for decentralized models.
09:45 – 10:30 | Fireside Chat: Tech Stack Showdown: CTMS, eSource, eCOA, and AI Workflows. Interoperability, data security, and global evidence portability.
10:45 – 12:00 | Workshop: Trial Virtualization Simulation. Teams stress-test workflows for global data integrity and sovereign-cloud constraints.
12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)
Afternoon Set: Data Flow and Vendor Synergy
01:00 – 01:30 | Presentation: Data Liquidity in Rare Trials: Secure, Compliant, and Real-Time. Managing HIPAA, GDPR, and PIPL inside one trial.
01:45 – 02:30 | Fireside Chat: When Tech Meets Humans - Global Training, Oversight, and Adoption. Lessons from multi-region DCT deployments.
02:45 – 04:15 | Workshop: Build Your Ideal Decentralized Vendor Ecosystem. Budgeting and compliance for multinational setups.
04:30 PM - 05:15 PM | CROSS-TRACK STRATEGIC PLENARY
Theme: Rare Disease in a Multipolar World: Regulatory Harmonization and Divergence Across the US, EU, and China. A unified session for all attendees tying together the Day 1 keynote and WG A outputs.
05:30 PM - 07:30 PM | Exhibitor Reception (Exhibit Hall)
Wednesday, September 16:
07:30 AM – 08:15 AM | Networking Breakfast (Exhibit Hall)
08:15 AM – 08:45 AM | OPENING KEYNOTE - DAY 2
Theme: Innovation Leadership and Global Competition in Rare Disease. How innovation ecosystems evolve; US/EU/China innovation dynamics; global rare disease data platforms in the next decade.
Track 3 - Regulatory and Endpoint Strategy
Morning Set: Designing for Approval
09:00 – 09:30 | Presentation: Adaptive and Platform Trials in Rare Disease. Convincing global regulators they aren’t statistical alchemy.
09:45 – 10:30 | Fireside Chat: Inside the FDA Mindset - Negotiating Endpoints and Surrogates. With perspective on EMA, PMDA, and NMPA divergence.
10:45 – 12:00 | Workshop: Write Your Own Endpoint Justification. Teams incorporate AI-derived biomarkers and global acceptance criteria.
12:00 PM — 01:00 PM | Networking Lunch (Exhibit Hall)
Afternoon Set: Evidence and Acceleration
01:00 – 01:30 | Presentation: Leveraging Natural History and RWE for Rare Approvals. Bias mitigation; evidence provenance; cross-region submission strategies.
01:45 – 02:30 | Fireside Chat: Accelerated Pathways and Global Harmonization. How companies align submissions across three continents.
02:45 – 04:15 | Workshop: Mock Regulatory Advisory Meeting. Sponsor–regulator role-play across multiple regulatory cultures.
Track 4 - Commercial and Partnership Value Realization
Morning Set: From Clinical Data to Market Value
09:00 – 09:30 | Presentation: Designing Trials That Tell a Global Payer Story. Embedding HEOR and EQoL data that different regions accept.
09:45 – 10:30 | Fireside Chat: Investor and Partner Perspectives - What Makes a Rare Trial “De-Risked” Globally. BD, VC, and licensing executives from the US, EU, and Asia.
10:45 – 12:00 | Workshop: Trial ROI Calculator. Teams model financial impact of improved recruitment, retention, and cycle-time reductions.
12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)
Afternoon Set: Ecosystem and Long-Term Value
01:00 – 01:30 | Presentation: Global Site and Home-Health Partnerships - Operational Scale as Strategic Asset. Where international alliances create speed and retention.
01:45 – 02:30 | Fireside Chat: The Next Frontier: Platform Trials, Shared Data, and Value-Based Global Partnerships. Pre-competitive collaboration, multi-sponsor alliances, shared registries.
02:45 – 04:15 | Workshop: Design Your Own Rare Trial Partnership Model. Teams develop a multinational sponsor–site–home-health framework.
04:30 PM – 05:15 PM | CROSS-TRACK STRATEGIC PLENARY
Theme: AI, Data Sovereignty, and the Future of Global Rare Trial Evidence. A unified closing session tying together Day 2 Keynote and Working Group B outputs.