Agenda 2025

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 8: Registration Opens

06:00 - 08:00 PM | Welcome Reception (Exhibit Hall)

Tuesday, September 9:

08:00 AM – 9:00 AM | Networking Breakfast (Exhibit Hall)

9:00 AM – 9:30 AM | Opening Keynote: The Evolving Regulatory Environment for Rare Disease Trials (Grand Liberty Ballroom)

Track 1 — Leveraging Innovative Trial Design, Digital Health and AI to Accelerate Trials (Grand Liberty Ballroom)

09:45 AM – 10:15 AM | Presentation: Accelerating Rare Disease Therapies: Strategies for Efficient Clinical Development - David Jacoby, MD, PhD, Senior Vice President, Head of Global Clinical Development, Ultragenyx

10:30 AM – 11:00 AM | Fireside Chat: Adaptive & Precision-Driven Trials: Platform and MATCH Models for Rare Disease Breakthroughs - Suma Babu, MPH, MBBS, Associate Professor of Neurology, and Sabrina Paganoni, MD, PhD, Associate Professor PM&R, Harvard Medical School - Massachusetts General Hospital

11:15 AM – 11:45 AM | Interactive Workshop: Solutions for Trial Design Challenges in Rare Disease Trials

12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)

01:15 PM – 01:45 PM | Presentation: Optimizing Rare Disease Clinical Trials with Real World Data Using NLP & AI

02:00 PM – 02:30 PM | Fireside Chat: AI-Driven Patient Identification and Recruitment - David Vulcano, MBA, Vice President, Research Compliance & Integrity, HCA Healthcare

02:45 PM – 03:15 PM | Fireside Chat: Operationalizing Precision Medicine in Rare Disease Trials - Douglas Warner, MD, Chief Medical Officer, Tyra Biosciences

03:30 PM – 04:00 PM | Interactive Workshop: Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies

04:15 PM - 04:45 PM | Closing Panel Discussion: Glimpse of the Future

Track 2 — DCT and Focus on the Patient Experience (Independence Ballroom)

09:45 AM – 10:15 AM | Presentation: Reducing Patient Burden in Rare Disease Trials - Cynthia Cassandro, Vice President, Patient Advocacy, Vigil Neuroscience

10:30 AM – 11:00 AM | Fireside Chat: Ethics and Strategies for Returning Individual Patient Data Collected in Clinical Research - Mindy Cameron, President, AdvocacyWorks

11:15 AM – 12:00 PM | Interactive Workshop: Designing Trials Aligned with Patient-Lived Experiences - Seth Rotberg, Senior Manager, Patient Advocacy, Solid Biosciences

12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)

01:15 PM – 01:45 PM | Presentation: How to Accelerate the Adoption of Decentralized Trials for Rare Disease Indications - Craig Lipset, Co-Chair, Decentralized Trials and Research Alliance

02:00 PM – 02:30 PM | Fireside Chat: Decentralized by Design: Operationalizing Hybrid Rare Disease Trials - Alpa Khushalani, Vice President of Client Success & Delivery, Gooseberry Research

02:45 PM – 03:15 PM | Fireside Chat: Beyond Biotech: The Rise of Nontraditional Sponsors in Rare Disease Trials

03:30 PM – 04:15 PM | Interactive Workshop: Home Health & Remote Best Practices

04:30 PM – 05:00 PM | Closing Panel Discussion: The Patient's and Caregiver’s Perspectives - Sonal Patel, MD, Field Medical Director, Rare Disease Cardiology, Pfizer, Metachromatic Leukodystrophy Parent

02:00 PM – 5:00 PM | CMO Council (link to separate agenda page)

05:00 - 07:00 PM | Exhibitor Reception (Exhibit Hall)

Wednesday, September 10:

08:00 AM – 9:00 AM | Networking Breakfast (Exhibit Hall)

9:00 AM – 9:30 AM | Opening Keynote: The Future of Rare Disease Trials - Ray Huml, DVM, Vice President of Rare Disease Strategy, Sciensus (Grand Liberty Ballroom)

Track 1 — Driving Clinical Operations Excellence in Rare Trials

09:45 AM — 10:15 AM | Presentation: How To Prevent Failure in Rare Disease Trials? - Pannie Trifillis, PhD, Senior Vice President, Medical Affairs & Scientific Communications, Rapid Commercialization Partners, ex-PTC Therapeutics

10:30 AM — 11:00 AM | Fireside Chat : Patient Recruitment and Engagement in in Global, Multisite Rare Disease Trials - Richie Kahn, Co-Founder & COO, Canary Advisors

11:15 AM — 12:00 PM | Interactive Workshop: Rare Isn’t Small: Incorporating Operational Excellence into Global, Multisite Rare Disease Trials - Leila Cupersmith, CEO, Global Oncology and Rare Disease Clinical Trial Operations Partner, Choice ClinOps and Donna Mackey, Vice President, Clinical Operations, Eli Lilly

12:00 PM — 01:00 PM | Networking Lunch (Exhibit Hall)

01:15 PM — 01:45 PM | Presentation: Optimizing CRO and Vendor Selection - Examples from the Field

02:00 PM — 02:30 PM | Fireside Chat: Beyond the Feasibility Survey: Strategic Site Selection to Accelerate Trials and Optimize Resources - Emilio S. Neto, MBA, Global Head of Country and Site Operations, Executive Director, Biogen

2:45 PM — 03:30 PM | Interactive Workshop: More Than Metrics: Using KPIs and Cross-Functional Dashboards to Drive Rare Disease Trial Success

03:30 PM – 04:00 PM | Closing Panel Discussion: TBD

Track 2 — Commercial and Market Access Strategies

09:45 AM – 10:15 AM | Presentation: Navigating the Path to Market: Strategic Planning for Rare Disease Product Launches

10:30 AM – 11:00 AM | Fireside Chat: Pricing and Reimbursement: Balancing Innovation and Accessibility in Rare Diseases

11:15 AM - 12:00 PM | Interactive Workshop: Global Market Access: Strategies for Expanding Reach in Rare Diseases

12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)

01:15 PM – 01:45 PM | Presentation: Innovative Funding Models: Financing Rare Disease Therapies

02:00 PM – 02:30 PM | Fireside Chat: Leveraging Real-World Evidence and Digital Biomarkers to Accelerate Rare Disease Market Access

02:45 PM – 03:30 PM | Interactive Workshop: Case Studies in Rare Disease Commercialization: Lessons Learned - Susan Sparks, MD, PhD, Rare Disease Clinical Development Consultant, ex-Sanofi Genzyme and Mary Hames, PhD, MBA, CEO, BioLogic Pharma Solutions

03:30 PM – 04:00 PM | Closing Panel Discussion: TBD