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:
12:00 PM - Registration Opens
02:00 PM – 5:00 PM | Accelerate Rare: PDUFA VIII Working Group (link to a separate agenda)
05:30 PM – 07:30 PM | Welcome Reception (Exhibit Hall)
Tuesday, September 9:
07:30 AM – 8:30 AM | Networking Breakfast (Exhibit Hall)
8:30 AM – 9:30 AM | Opening Keynote: The Evolving Regulatory Environment for Rare Disease Trials (Grand Liberty Ballroom)
Janet Maynard, MD MHS, Director, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, FDA
Amy Rick, JD, Director of Strategic Coalitions, Rare Disease Innovation Hub, FDA
Track 1 — Leveraging Innovative Trial Design, Patient-Centered Measures and AI to Accelerate Trials (Grand Liberty Ballroom)
09:45 AM – 10:15 AM | Presentation: Accelerating Rare Disease Therapies: Strategies for Efficient Clinical Development
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, both Harvard Medical School - Massachusetts General Hospital
11:15 AM – 12:00 PM | Interactive Workshop: Solutions for Trial Design Challenges in Rare Disease Trials
Douglas Warner, MD, Chief Medical Officer, Tyra Biosciences
Sam Hopkins, PhD, Senior Vice President, Therapeutics, AskBio, part of Bayer AG
Laura Pisani, MD, MBA FACMG, Vice President Clinical Development, Taysha Gene Therapies
Michael Binks, MD, Chief Medical Officer, Capricor, ex-Pfizer, GSK
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
Christopher Rudolf, EMBA, Founder and CEO, Volv Global
02:00 PM – 02:30 PM | Fireside Chat: AI-Driven Patient Identification and Recruitment
David Vulcano, MBA, Vice President, Research Compliance & Integrity, HCA Healthcare
Dennis Akkaya, MSc, Chief Commercial Officer, MyTomorrows
02:45 PM – 03:15 PM | Fireside Chat: Operationalizing Precision Medicine in Rare Disease Trials
Thomas Defay, PhD, Deputy Head of Diagnostics, Alexion AstraZeneca
Mandi B. Walters, MS, Executive Vice President, Sciensus
03:30 PM – 04:15 PM | Interactive Workshop: Innovating Clinical Outcome Assessments in Rare Disease Trials: Integrating Patient-Centered Measures and Digital Health Technologies
Ebony Dashiell-Aje, PhD, Executive Director and Head, Patient Centered Outcomes Science, BioMarin Pharmaceutical
Christina Theodore-Oklota, PhD, Vice President, Clinical Outcomes Research, Mahzi Therapeutics
Megan Parisi, MBA, CCRP, Patient Solutions Lead, Syneos Health
Track 2 — Aligning Trials with Patient-Lived Experiences to Scale Trials and Reduce Patient Burden (Independence Ballroom)
09:45 AM – 10:15 AM | Presentation: Reducing Patient Burden in Rare Disease Trials
10:30 AM – 11:00 AM | Fireside Chat: Ethics and Strategies for Returning Individual Patient Data Collected in Clinical Research
Mindy Cameron, President, AdvocacyWorks
Suzanne Gaglianone, PatientCare Field Representative, ReveraGen BioPharma
Holly Peay, PhD, Senior Director, Faegre Drinker Consulting
11:15 AM – 12:00 PM | Interactive Workshop: Aligning Trials with Patient-Lived Experiences
Seth Rotberg, Senior Manager, Patient Advocacy, Solid Biosciences
Julie Lin MD MPH FASN, Senior Vice President, Clinical Development and Clinical Pharmacology, Travere Therapeutics
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
Kristi Clark, MBA, Senior Vice President, Clinical Operations and Data Management, Avidity Biosciences
Iwona Tongbhoyai, MSc, Chief Client Solutions Officer at FutureMeds
02:45 PM – 03:15 PM | Fireside Chat: Beyond Biotech: The Rise of Nontraditional Sponsors in Rare Disease Trials
Craig Martin, CEO, Orphan Therapeutics Accelerator
Ilan Ganot, MBA, CEO, Alesta Therapeutics and Founder, Solid Biosciences
Craig Lipset, Advisor, Buffalo Initiative
Casey McPherson, Founder of AlphaRose Therapeutics & To Cure a Rose Foundation (StartEngine)
03:30 PM – 04:15 PM | Panel Discussion: Leveraging Patient Advocacy To Scale Rare Disease Trials
Moderator: Paul Kidwell, Story Creator, Senior Public Relations & Patient Advocacy Consultant, Podcaster
Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy
Sonal Patel, MD, Field Medical Director, Rare Disease Cardiology, Pfizer, Metachromatic Leukodystrophy Parent
04:30 PM - 05:15 PM | Closing Panel: Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials (Grand Liberty Ballroom)
Philip J. Brooks, Ph.D., Deputy Director, Division of Rare Diseases Research Innovation, NCATS NIH (tentative)
Mathew Pletcher, PhD, Chief Scientific Officer, Weaver Biosciences, ex-Astellas, Roche, Pfizer
Kiran Musunuru, MD PhD, Professor, University of Pennsylvania, and Scientific Director, Center for Inherited Cardiovascular Disease
Sadik Kassim, PhD, Chief Technology Officer, Genomic Medicines, Danaher Corporation
05:30 PM - 07:30 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 (Grand Liberty Ballroom)
Raymond Huml, DVM, Vice President of Rare Disease Strategy, Sciensus
Track 1 — Driving Clinical Operations Excellence in Rare Trials (Grand Liberty Ballroom)
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
Donna Mackey, Vice President, Clinical Operations, Akouos, part of Eli Lilly
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, ex-ICON
Rachel Smith, Vice President, Global Head of Rare Disease, Parexel
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 Neto, PharmD, MBA, Global Head of Country and Site Operations, Executive Director, Biogen
Jeff Guptill, MD MHS, Disease Area Development Lead - Neuromuscular, Clinical Development, argenx
Lauren Morgenroth, MS, CEO, TRiNDS
2:45 PM — 03:30 PM | Interactive Workshop: Purpose-Driven Metrics: Running Gene Therapy Trials with KPIs That Matter
John Hall, MBA, President, Shore Research & Development and Director of Clinical Operations at REGENXBIO
Brandon Michael Henry, MD, FAPCR, FACSc, BCMAS, Medical Director, Cell and Gene Therapy Center of Excellence, IQVIA
Renata Shih, MD, Principal Investigator, Rare Disease Research
Track 2 — Incorporating Commercial and Market Access Strategies Early to Make the Trials Successful (Independence Ballroom)
09:45 AM – 10:15 AM | Presentation: Designing Trials with the End in Mind: Building Market Access into Rare Disease Clinical Development
Clark Paramore, Head of Global Value Evidence Strategy, Biogen
10:30 AM – 11:00 AM | Fireside Chat: What Matters to Payers: Conversations You Should Start During Phase 2
Marcella Lynch, MBA, Head of Pricing, Forecasting & Contracting - Americas Market Access, Rare Diseases, Chiesi USA
Jennifer Shumsky, BSN, RN, Founder & Market Access & Reimbursement Specialist, JLS Consulting
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: Patient-Centric Commercialization: Integrating Patient Voices into Market Access Strategies
Marc O'Connor, MBA, Chief Business Officer, Curant Rare
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
Tim Mooney, MBA, Senior Director, Program Leader for ALN-APP and ALN-SOD, Alnylam Pharmaceuticals
Jake Caines, Chief Growth Officer, Curant Rare
Mary Hames, PhD, MBA, CEO, BioLogic Pharma Solutions
03:45 PM – 04:30 PM | Closing Panel: From Planning to Performance: Integrating Strategy, Execution, and Accountability Across Rare Disease Trials (Grand Liberty Ballroom)
Kenneth Harvey, MBA, Director Clinical Operations, Epicrispr Biotechnologies
Kelly Kilpatrick, Senior Vice President, Head of US Commercial Ocular Gene Therapy, Spark Therapeutics, part of Roche (tentative)