2026 Agenda
Monday, September 14:
08:00 AM - Registration Opens
09:00 AM – 12:00 PM | Accelerate RARE Working Group A: Plausible Mechanism Evidence Playbook
Chair: Janet Woodcock, MD, Former Acting Commissioner, FDA, and Former Director, CDER
01:00 PM – 04:00 PM | Accelerate RARE Working Group B: Minimum Readiness Standards for Pediatric Trials
Chair: Ron Bartek, Founder and President, Friedreich’s Ataxia Research Alliance (FARA)
04:00 PM – 05:30 PM | Private Screening of RARE: A Rare Disease Revolution followed by Post-Screening Conversation with Lainey Moseley, Filmmaker and Hulda Björk Svansdóttir, Patient Advocate and Parent, Dancing for Duchenne
05:30 PM – 07:30 PM | Welcome Reception
Tuesday, September 15:
07:30 AM – 08:00 AM | Networking Breakfast
08:00 AM - 08:15 AM | Effie Parks, Main Stage Host, September 15-16,Host, Once Upon a Gene Podcast; Founder, The Effie Parks Foundation
08:15 AM – 08:45 AM | OPENING KEYNOTE - DAY 1
From Breakthrough to Access: How Advocacy and Policy Decide What Reaches Patients
Michele M. Oshman, Chief Patient Advocate, SVP Alliance Development, Biotechnology Innovation Organization
Guests: Joe, Courtney and Maggie Dion, The Dion Family Foundation
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.
Raviv Pryluk, PhD, CEO and Co-Founder, PhaseV
09:45 – 10:30 | Fireside Chat: From Awareness to Activation - Converting Rare Patient Identification into Trial Enrollment. A practical look at rare disease recruitment, including patient engagement and consent.
Dennis Akkaya, MBA, Chief Commercial Officer, myTomorrows
Edward Smith, MD, Medical Director and Principal Investigator, Rare Disease Research
10:45 – 12:00 | Workshop: Mapping the Rare Patient Journey. Teams build an “enrollment funnel” model using real trial data - identifying drop-off points and intervention tactics..
Holly Peay, PhD, Senior Director, Faegre Drinker Consulting
Karin Hoelzer, DVM PhD, Senior Director, Patient Advocacy, Biotechnology Innovation Organization
Danielle Dong, MS, Scientific Advocacy Lead, Global Medical Affairs, Rare Disease, Sanofi
12:00 PM – 01:00 PM | Networking Lunch
Afternoon Set: Global Reach and Retention
01:00 – 01:30 | Presentation: Patient Input and Digital Health Technologies: A Regulatory Perspective Across the Product Lifecycle
Cyndi Grossman, PhD, Division Director, Patient-Centered Development, Office of Strategic Partnerships and Technology Innovation, CDRH, FDA
01:45 – 02:30 | Fireside Chat: Retention by Design - Keeping Families Engaged for Multi-Year Studies. Perspectives on long-term trial participation.
Angi Robinson, Senior Vice President, Specialty Areas, Premier Research
Jennifer McNary, Co-Founder and CEO, Canary Concierge
Hulda Björk Svansdóttir, Patient Advocate and Parent, Dancing for Duchenne
02:45 – 04:15 | Workshop: Bridging the Generational Divide in Rare Disease Clinical Trials: Tailoring Communication to Maximize Recruitment, Retention, and Equity.
Paula Orandash, Director, Patient Engagement, ERGOMED
Kris O’Brien, Director, Program Strategy, Rare Disease, ERGOMED
Track 2 - Operational Efficiency in Rare disease Trials
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.
Craig Lipset, MPH, Co-Chair, Digital Trials & Research Alliance
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.
Amit Gulwadi, MS, VP, Head of Digital and Transformation, Development Regulatory and Safety, Alexion Pharmaceuticals
10:45 – 12:00 | Workshop: Build Your Ideal Decentralized Vendor Ecosystem. Budgeting and compliance for multinational setups.
Lauren Morgenroth, CEO, TRiNDS
Miro Pastrnak, PhD, COO, Gooseberry Research
Gemma Whiteley, MBA, Managing Director, FutureMeds @Home
12:00 PM – 01:00 PM | Networking Lunch
Afternoon Set: Flexible Evidence and Lean Execution
01:00 – 01:30 | Presentation: TBD
01:45 – 02:30 | Fireside Chat: Pediatric Rare Disease Development: Evidence, Flexibility, and Regulatory Pathways. A discussion of pediatric rare disease development, evidence generation, accelerated pathways, and practical considerations.
Jennifer McKenzie, MD FAAP FASN, Senior Clinical Program Leader, Boehringer Ingelheim
Janet Maynard, MD MHS, Director, Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, FDA
02:45 – 04:15 | Workshop: Ultra-Rare, Ultra-Lean: Designing for a Viable ROI Under 1,000 Patients. A practical look at designing and executing ultra-rare trials cost effectively, focusing on what’s essential versus what’s just habit.
Casey McPherson, Founder & CEO, AlphaRose Therapeutics
Brandon Henry, MD FACSc, IFAPP Fellow, Founder & Chief Executive and Medical Officer, Aurelix Bio
Stephanie Manton, PhD, CEO and Co-Founder, WinQure Medicine
04:30 PM - 05:30 PM | CROSS-TRACK STRATEGIC PLENARY
Platform and Basket Trials in Rare Disease: What’s Working, What Isn’t, and What It Takes to Scale. Practical lessons from platform and basket trials in rare disease indications.
James D. Berry, MD MPH, Chief, Division of Motor Neuron Diseases, MGH Harvard, ALS HEALY Platform Trial
Michelle Mellion, MD, former Chief Medical Officer, EveryONE Medicines, The Master Protocol (EOM-MP1)
Nerissa Kreher, MD MBA, Chief Medical Officer, Alltrna
05:30 PM - 07:30 PM | Exhibitor Reception
Wednesday, September 16:
07:30 AM – 08:15 AM | Networking Breakfast
08:15 AM – 08:45 AM | OPENING KEYNOTE - DAY 2
Theme: TBD
Track 3 - Regulatory and Endpoint Strategy
Morning Set: Designing for Approval
09:00 – 09:30 | Fireside Chat: Inside the FDA Mindset - Negotiating Endpoints and Surrogates. With perspective on EMA, PMDA, and NMPA divergence.
Wendy Erler, MBA, Senior Vice President, Patient Affairs, Sarepta Therapeutics
Sandeep Menon, PhD MPH, Chief Development and Research Officer, BioCryst Pharmaceuticals
09:45 – 10:30 | Panel Discussion: Patient Organizations as Evidence Partners: Shaping the Science Behind Rare Disease Approval. How patient organizations help shape the evidence behind rare disease approvals.
Patricia Furlong, Founder and President, Parent Project Muscular Dystrophy
Philip Yeske, PhD, Science & Alliance Officer, United Mitochondrial Disease Foundation
Terri L. Klein, CEO, National MPS Society
Andrea Faris, Director, The Acid Maltase Deficiency Association - Pompe
10:45 – 12:00 | Workshop: Write Your Own Endpoint Justification. Teams incorporate AI-derived biomarkers and global acceptance criteria.
Caiyan Zhang, PhD, Associate Director - Patient-Centered Outcomes, Takeda
Dominique Pichard, MD MS, Chief Scientific and Medical Officer, International Rett Syndrome Foundation
12:00 PM - 01:00 PM | Networking Lunch
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.
Julia Vitarello, Co-Founder, N=1 Collaborative, Founder and CEO, Mila’s Miracle Foundation
Barrett Tenbarge, JD, Partner, FDA and Life Sciences Regulatory, Faegre Drinker
Jennifer Panagoulias, Chief Operating Officer, Foundation for Angelman Syndrome Therapeutics (FAST) and AS2Bio
Winston Yan, MD PhD, Director, Center for Genetic Surgery, The Broad Institute of MIT and Harvard
Afternoon Set: Evidence and Acceleration
02:00 – 02:30 | Presentation: Leveraging Natural History and RWE for Rare Approvals. Using observational data without tripping over bias and data provenance.
Michael Binks, MD, Chief Medical Officer, Capricor Therapeutics
02:45 – 03:30 | Fireside Chat: Accelerated Pathways and Global Harmonization. How companies align submissions across three continents.
Rachel Smith, Vice President, Rare and Genetic Diseases, Parexel
Chiemeka Ike, MPH, PharmD, MS, Head, HEOR, Celcuity
03:45 – 04:45 | Workshop: Mock Regulatory Advisory Meeting. Sponsor–regulator role-play across multiple regulatory cultures.
Elizabeth Duke, MD, Senior Vice President, Canal Row Advisors, former FDA reviewer
Kim Quaintance-Lunn, VP, Head of Regulatory Science and Execution, Alexion Pharmaceuticals
Souad Messahel, PhD, COO, Aurelix Bio
Track 4 - Commercial and Partnership Value Realization
Morning Set: The Rare Disease Value Case
09:00 – 09:30 | Presentation: Practical Application of Bayesian Networks in Rare Disease Trials.
J. Jaime Caro, MD, Chief Scientist, Evidera, now Thermo Fisher Scientific and Adjunct Professor Medicine, Epidemiology Biostatistics, McGill University
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.
Chris Garabedian, CEO, Xontogeny and Portfolio Manager, Perceptive Xontogeny Venture Fund
Tim Hunt, JD, CEO, Alliance for Regenerative Medicine
Schiffon Wong, MPH, Principal, Schiffon Wong Strategic Advisory
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.
Molly Painter, MBA, Head of Immunology Commercial and National Accounts, Takeda
Jaya Khushalani, MD PhD MBA, National Lead HEOR Epilepsy and Rare Syndromes, UCB
Jake Caines, Chief Revenue Officer, Curant Rare
12:00 PM – 01:00 PM | Networking Lunch
01:00 PM – 01:45 PM | CROSS-TRACK STRATEGIC PLENARY (see the Track 3 above)
Afternoon Set: Ecosystem and Long-Term Value
02:00 – 02:30 | Presentation: From Parent Advocacy to IND: Building a Treatment Pathway for an Ultra-Rare Disease. How the Rare Trait Hope Fund helped move an AGU therapy from diagnosis-driven urgency to translational development, FDA engagement, and clinical trial.
Julia Taravella, MEng, Founder and Executive Director, Rare Trait Hope Fund
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.
Marc O'Connor, MBA, Principal and Chief Business Officer, Curant Rare
Raymond A. Huml, MS, DVM, RAC