Rare Diseases & Orphan Drugs Conference

Welcome to the Rare Diseases & Orphan Drugs Conference 2026

From Innovation to Access: Advancing Treatments for Rare Diseases

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.

View The Full Programme

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

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

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Previous Events

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Expert Speakers

Panel Discussions

Focused Driven Day

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.

The Rare Diseases & Orphan Drugs Conference 2025

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.

The Rare Diseases & Orphan Drugs Conference 2025

Get in touch for Group Discounted Tickets

Book An Exhibition Stand + 2 Delegate Passes: Book Now For Just: 4,999 €

Discover how you can be a part of this exciting conference and get your expertise in front of your target audience. Packages Include; Exhibition Area, Main Stage Speaking, One-To-One Meetings.

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Get in touch for Group Discounted Tickets

To register your team now and benefit from huge group savings, call the conference team on +420 776 164 009 (Prague office) || +44 2038 076 227 (London office)

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