Join global pioneers as we connect science, policy, and partnerships — from discovery to patient access
Welcome to the
Rare Diseases & Orphan Drugs Conference 2026 - Europe's leading forum uniting researchers, biotech leaders, regulators, and patient advocates to accelerate therapies for rare and ultra-rare conditions. Join global pioneers as we connect science, policy, and partnerships - from discovery to patient access.
6 Key Themes for the Rare Diseases & Orphan Drugs Conference 2025
- Innovative R&D for Rare Diseases: Novel targets, trial design, and translational approaches.
- Regulatory Strategy & Orphan Designation: Incentives, approvals, and cross-border harmonization.
- Market Access & Reimbursement: Value frameworks, payer engagement, and affordability.
- Advanced Therapeutics in Rare Indications: Gene, cell, and RNA therapies for ultra-rare diseases.
- Patient-Centered Approaches: Advocacy partnerships, registries, and digital health integration.
- Global Policy & Collaboration: Building sustainable, equitable systems for rare disease innovation.
Key Themes
Rare Disease Drug Discovery & Development- Leveraging genomics and precision medicine for ultra-rare indications
- Translational research from preclinical to first-in-human studies
- Innovative trial designs for small patient populations
Regulatory Pathways & Orphan Designation- FDA, EMA, and global frameworks for orphan drug approval
- Expedited pathways: PRIME, Breakthrough Therapy, and Adaptive Licensing
- Aligning regulatory incentives with patient needs
Access, Pricing & Reimbursement- Health economics for high-cost therapies
- Innovative payment models and managed access agreements
- Country-level case studies in reimbursement and HTA approval
Advanced Therapeutics & Rare Diseases- Gene therapy, cell therapy, and RNA platforms in orphan indications
- Manufacturing challenges for ATMPs in small populations
- Long-term safety monitoring and registries
Patient-Centric Development- Partnering with patient advocacy groups in R&D
- Patient-reported outcomes and quality-of-life measures
- Building real-world evidence through registries and digital health
Global Roadmap for Rare Disease Innovation- International consortia and cross-border collaborations
- Case studies in access equity across healthcare systems
- Future vision for orphan drug development in a post-genomic era
Why Attend
- Discover the latest breakthroughs in orphan drug discovery and precision medicine.
- Network with biotech leaders, regulators, and patient advocates shaping the field.
- Gain practical insights into trial design, market access, and reimbursement for rare disease therapies.
- Explore real-world case studies in gene therapy, regulatory pathways, and patient partnerships.
Who Will Benefit
Biotech and pharma professionals, translational researchers, regulators, payers, advocacy leaders, and anyone working to bring life-changing therapies to patients with rare diseases.
Who Should Attend
- Rare Disease Researchers & Clinician-Scientists
- Biotech & Pharma R&D Executives
- Drug Development & Clinical Operations Leaders
- Market Access & Health Economics Professionals
- Regulators & Policy Makers
- Patient Advocacy Groups & Foundations
- Healthcare Providers & Payers
Industries Represented
- Biopharmaceutical & Biotech Companies
- Genomics & Advanced Therapy Developers
- Clinical Research Organizations (CROs)
- Orphan Drug Manufacturers
- Market Access & Health Economics Consultancies
- Regulatory & Health Policy Authorities
- Patient Organizations & Advocacy Groups
Speakers
- James D. Chambers, Director, SPEC Database Professor, Tufts University School of Medicine
- Jill Goodrich, Co-Executive Director
- Pedro Franco, Head of Europe Global Regulatory and Scientific Policy, Senior Director.
- Kim Angel, Executive Director International MPS Network
- Dr. Marco Rauland, Vice President Global Head of GVAP Strategy, Pricing & Analytics
- Henriëtte Burghoorn, Associate Director, Market Access| Netherlands
- Patroski J. Lawson, CEO, The KPM Group DC
- Marieangela Economopoulou PhD, MBA Head External Affairs & Patient Value Access
- Arjun Channi, Director, Global Regulatory Lead (Rare Disease)
- Barry Weber, CISO & DPO
- Alan Minsk, Partner at Arnall Golden Gregory LLP
- David Du, Sr. Manager, Market Access Operations, Global Rare Diseases
- Alisha Angdembe, Senior Health Economist
- Adrian Goretzki, President & Founder
- Floortje van Nooten, Head of Market Access EU
- Nathan Chadwick, Senior Director, Therapeutic Strategy Lead – Rare Disease
Please fill in your name and email to receive the Conference Agenda of this event.
Venue
Hotel SB Plaza Europa
Carrer de les Ciències 11, 13, L’Hospitalet de Llobregat, 08908
Barcelona, Spain
