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
Luis Rojas, PhD, CEO and President, InCSD
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, Harvard Medical School - Massachusetts General Hospital
Sabrina Paganoni, MD, PhD, Associate Professor PM&R, Harvard Medical School - Massachusetts General Hospital
11:15 AM – 12:00 PM | Interactive Workshop: Solutions for Trial Design Challenges in Rare Disease Trials
Sam Hopkins, PhD, Senior Vice President, Therapeutics, AskBio/ Bayer AG
Laura Pisani, MD, MBA FACMG, Vice President Clinical Development, Taysha Gene Therapies
Michael Binks, MD, Chief Medical Officer, Capricor
Sandeep Menon, MD PhD, Chief Development Officer, Head of Clinical Development and Safety, Alnylam Pharmaceuticals
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
Steve Rodems, PhD, Vice President, Research and Nonclinical Development, Travere Therapeutics
Amy Ponte, PhD MPH, Director, Global Scientific Affairs and Diagnostics, Sanofi
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
Megan Parisi, MBA, CCRP, Patient Solutions Lead, Syneos Health
Amy Apostoleris, MBA, CEO, DFnet Research
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
Chelley Casey, MPH, Vice President, Patient Advocacy, Wave Life Sciences
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
Jeffrey Smith, MS, MBA, Vice President, Head, Patient Advocacy, Ionis Pharmaceuticals
Kelly Franchetti, RN, CEO, The Patient View
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
Han C. Phan, MD, Founder and CEO, Rare Disease Research and Gooseberry Research
02:00 PM – 02:30 PM | Fireside Chat: Decentralized by Design: Operationalizing Hybrid Rare Disease Trials
Miro Pastrnak, PhD, Chief Operating Officer, Gooseberry Research
Kristi Clark, MBA, Senior Vice President, Clinical Operations and Data Management, Avidity Biosciences
Rebeca Gonzalez, MA, Director, Decentralized Client Solutions, FutureMeds
Edward Smith, MD, Medical Director and Principal Investigator, Rare Disease Research
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
Casey McPherson, Founder of AlphaRose Therapeutics
Ethan Perlstein, PhD, CEO of Perlara
03:30 PM – 04:15 PM | Panel Discussion: Leveraging Patient Advocacy To Scale Rare Disease Trials
Moderator: Andrea Schelhaas, MS, Genetic Counselor, Genetic Support Foundation
Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy
Sonal Patel, MD, Metachromatic Leukodystrophy Parent
Jen Farmer, MS, CEO , Friedreich’s Ataxia Research Alliance (FARA)
Ryan Fischer, Chief Operating Officer, Foundation for Angelman Syndrome Therapeutics (FAST)
Sharon King, COO, National MPS Society
Luisa Leal, Founder and CEO of The Akari Foundation
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
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
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, Lilly Gene Therapy, Eli Lilly & Company
Lauren Lee, MA, Executive Director, Head of Patient Advocacy, Travere Therapeutics
Lauren Wilcoxen, PhD, RAC, Senior Vice President, Product Development and Regulatory Affairs, Checkpoint Therapeutics/ Sun Pharma
11:15 AM — 12:00 PM | Interactive Workshop: Rare Isn’t Small: Incorporating Operational Excellence into Global, Multisite Rare Disease Trials
Donna Mackey, Vice President, Clinical Operations, Lilly Gene Therapy, Eli Lilly & Company
Rachel Smith, Vice President, Global Head of Rare Disease, Parexel
Leila Cupersmith, CEO, Global Oncology and Rare Disease Clinical Trial Operations Partner, Choice ClinOps
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
Nicole Stansbury, Senior Vice President, Global Clinical Operations, Premier Research
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
Jeremy Tanner, MD, Neurologist, UT Health San Antonio & Channeling Hope Foundation
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
Jennifer Hunt, MS, Health Value Translation Lead, Sanofi
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
Rob Goldstein-Mahon, MS, Director, Patient Advocacy, Ionis Pharmaceuticals
11:15 AM - 12:00 PM | Interactive Workshop: Real-World Evidence That Matters: A Hands-On Workshop in Building Credible Value Stories
Karin Hoelzer, DVM PhD, Senior Director, Patient Advocacy, Biotechnology Innovation Organization (BIO)
Mandi B. Walters, MS, Senior Vice President Business Development, Rare Disease, Sciensus
Wendy Erler, MBA, Senior Vice President, Patient Affairs, Sarepta Therapeutics
12:00 PM – 01:00 PM | Networking Lunch (Exhibit Hall)
01:15 PM – 01:45 PM | Presentation: TBD
Ashish Dugar, PhD MBA, Chief Development Officer, Pretzel Therapeutics
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
Amy Grover, Executive Director of Patient Advocacy, Catalyst Pharmaceuticals
Quinn Dinh, MD, Vice President, Head of Medical Affairs, Wave Life Sciences
02:45 PM – 03:30 PM | Interactive Workshop: Case Studies in Rare Disease Commercialization: Lessons Learned
Susan Sparks, MD, PhD, Vice President, Clinical Development, Tessera Therapeutics
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