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: Minimum Evidence Standards for the Plausible Mechanism Pathway in Rare Disease
01:00 PM – 04:00 PM | Accelerate RARE Working Group B:Minimum Readiness Standards for Infant and Young-Child Rare Disease Trials
04:00 PM – 05:30 PM | Private Screening of RARE: A Rare Disease Revolution ( —> website)
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
From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients. How the commercial and Federal reimbursement environments shape whether scientific progress reaches patients.
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: Myth-Busting Hybrid & Home-Based Trials: What’s Real, What’s Not and What Scales. An evidence-based look at decentralized trial performance in the real world.
09:45 – 10:30 | Fireside Chat: How to De-CRO Rare Disease ClinOps Without Breaking the Machine. How rare disease biotechs can selectively pull clinical operations back in-house, where sponsor control adds the most value, and where CRO support still earns its keep.
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
Platform Trials in Rare Disease: What’s Actually Working and What It Takes to Make Them Real. This plenary brings leaders from ALS, MND, scleroderma, PSP, and Duchenne together to share what is working in rare-disease platform trials, what blocks sponsor participation, and what it takes to build durable, patient-efficient trial infrastructure.
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)
01:00 PM – 01:45 PM | CROSS-TRACK STRATEGIC PLENARY
Theme: The Plausible Mechanism Pathway: A New Evidence Playbook for Rare Disease Development. This plenary examines the plausible mechanism pathway as an emerging evidence framework in rare disease development, including where it may apply and key considerations for sponsors, patient groups, and other stakeholders.
Afternoon Set: Evidence and Acceleration
02:00 – 02:30 | Presentation: Leveraging Natural History and RWE for Rare Approvals. Bias mitigation; evidence provenance; cross-region submission strategies.
02:45 – 03:30 | Fireside Chat: Accelerated Pathways and Global Harmonization. How companies align submissions across three continents.
03:45 – 05:15 | 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: Who Really Decides Which Rare Disease Therapies Survive: FDA, Investors, or Payers?. BD, VC, and licensing executives square off.
10:45 – 12:00 | Workshop: Modeling Value Across the Rare Disease Patient Journey. Teams examine how earlier diagnosis, smoother access, faster therapy start, stronger adherence, and longer persistence influence launch performance and long-term brand value.
12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)
01:00 PM – 01:45 PM | CROSS-TRACK STRATEGIC PLENARY
Theme: The Plausible Mechanism Pathway: A New Evidence Playbook for Rare Disease Development. This plenary examines the plausible mechanism pathway as an emerging evidence framework in rare disease development, including where it may apply and key considerations for sponsors, patient groups, and other stakeholders.
Afternoon Set: Ecosystem and Long-Term Value
02:00 – 02:30 | Presentation: Global Site and Home-Health Partnerships - Operational Scale as Strategic Asset. Where international alliances create speed and retention.
02:45 – 03:30 | Fireside Chat: Commercial Success in Rare Disease Takes an Ecosystem: Aligning Manufacturers, Specialty Pharmacy, Providers, and Patient Support. How rare disease companies align access, care delivery, and patient support to drive durable uptake and long-term value.
03:45 – 05:15 | Workshop: Ultra-Rare, Ultra-Lean: Designing for a Viable ROI Under 1,000 Patients. A practical look at how sponsors design and execute ultra-rare trials with constrained budgets, focusing on what’s essential versus what’s just habit.