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
01:00 PM – 04:00 PM | Accelerate RARE Working Group B
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
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 to connect siloed datasets and patient communities without triggering a privacy nightmare.
09:45 – 10:30 | Fireside Chat: From Awareness to Activation - Turning 1000 Genotypes into 10 Enrolled Patients. Sponsor, advocacy, and digital-recruitment leaders dissect real enrollment case studies and missteps.
10:45 – 12:00 | Workshop: Mapping the Rare Patient Journey. Participants build an “enrollment funnel” model using real trial data - identifying drop-off points and intervention tactics.
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 and Consent. Legal, cultural, and logistical obstacles in multi-region rare-disease trials.
01:45 – 02:30 | Fireside Chat: Retention by Design - Keeping Families Engaged for Multi-Year Studies. Site, sponsor, and caregiver perspectives on long-term participation.
02:45 – 04:15 | Workshop: Building a Patient-First Recruitment Playbook. Teams draft recruitment and retention blueprints tailored to specific therapeutic areas.
Track 2 - Operational Efficiency through Decentralization and Technology
Morning Set: From Concept to Compliance
09:00 – 09:30 | Presentation: Hybrid and Home-Based Trials: What Works, What Fails, What’s Next. Real-world performance metrics for decentralized models.
09:45 – 10:30 | Fireside Chat: Tech Stack Showdown - Integrating CTMS, eSource, and eCOA Without Losing Your Mind. CTOs and ops heads discuss interoperability pain points and hidden costs.
10:45 – 12:00 | Workshop: Trial Virtualization Simulation. Attendees design a decentralized workflow and stress-test it for data integrity, logistics, and compliance gaps.
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 Part 11/HIPAA/GDPR data flows across home-health, site, and sponsor.
01:45 – 02:30 | Fireside Chat: When Tech Meets Humans - Training, Oversight, and Adoption. Lessons from DCT vendors and site networks on avoiding “pilot purgatory.”
02:45 – 04:15 | Workshop: Build Your Ideal Decentralized Vendor Ecosystem. Participants evaluate mock vendor options and budget trade-offs to design an integrated solution.
04:30 PM - 05:15 PM | Closing Panel: TBD
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
Track 3 - Regulatory and Endpoint Strategy
Morning Set: Designing for Approval
09:00 – 09:30 | Presentation: Adaptive and Platform Trial Designs in Rare Disease. How to convince regulators and statisticians they’re not black magic.
09:45 – 10:30 | Fireside Chat: Inside the FDA Mindset - Negotiating Endpoints and Surrogates. Regulatory experts and sponsor leads share pre-IND war stories.
10:45 – 12:00 | Workshop: Write Your Own Endpoint Justification. Guided exercise drafting rationale for a novel clinical endpoint or biomarker.
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. Using observational data without tripping over bias and data provenance.
01:45 – 02:30 | Fireside Chat: Accelerated Pathways and Global Harmonization. How companies align U.S., EU, and Asia-Pac submissions for the same molecule.
02:45 – 04:15 | Workshop: Mock Regulatory Advisory Meeting. Participants role-play a sponsor-FDA dialogue on endpoint acceptance and trial design.
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 Payer StoryEmbedding HEOR and quality-of-life measures into the protocol.
09:45 – 10:30 | Fireside Chat: Investor and Partner Perspectives - What Makes a Rare Trial “De-Risked”. BD, VC, and licensing execs dissect the metrics that move valuation.
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. How alliances convert into faster start-ups and better sponsor retention.
01:45 – 02:30 | Fireside Chat: The Next Frontier: Platform Trials, Shared Data, and Value-Based Partnerships. Discussion on pre-competitive collaboration and data-sharing frameworks.
02:45 – 04:15 | Workshop: Design Your Own Rare-Trial Partnership Model. Participants co-create a mock multi-sponsor, multi-stakeholder alliance plan