Agenda
26-28 October 2026
RAI Convention Center, Amsterdam
Amsterdam, 26 - 28 October 2026
Schedule
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ERDERA Workshop
Developing effective therapies for rare and ultra-rare diseases (U)RD is hindered by inherent challenges such as limited patient numbers, disease heterogeneity, and complexities in defining clinically meaningful endpoints. Accelerating progress requires novel methodological approaches across the entire R&D spectrum. European initiatives like ERDERA, INVENTS, and RealiseD are tackling these challenges head-on through collaborative, multi-stakeholder efforts. This session will showcase how these projects are developing and implementing cutting-edge methodologies to optimise clinical trial design, leverage diverse data sources including Real World Data (RWD), streamline evidence generation, and ensure alignment with regulatory and HTA requirements, all centered on patient needs.
Implementing HTA recommendations, Evidence assessment, economic modelling, and how legislation drives HTA change
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Opening remarks
Keynote panel: EU Pharmaceutical Legislation Revision – what’s new for 2026?
- How will the revision affect rare diseases?
- Navigating Europe’s regulatory incentives and the future of pharmaceutical R&D innovation
- Who do we ensure that changes can facilitate Europe’s access, innovation and sustainability?
- The European Biotech Act and its impact on rare diseases
Keynote Panel: How are patients involved in every step of the orphan drug development and global access?
- Patient led solutions to orphan drug development and access bottlenecks
- How to solve the issue of small patient populations through statistical innovation, regulatory flexibility and patient-centricity
- Utilising Real-World Data and natural history studies
- Creating meaningful endpoints that improves patients’ quality of life
Keynote Panel: The next frontier in rare disease, where will the breakthroughs come from?
-What’s in the pipeline and what rare diseases have been targeted?
-How have global launch strategies been impacted by MFN?
-What are the emerging platforms (gene, cell, RNA etc) and novel trial designs for small patient populations?
-How will regulatory pathways accelerate approval, orphan designation and it’s measure overall risk/benefit
-What impacts are new technologies such as AI, Digital Health and medical devices having on the rare disease ecosystem and early diagnosis?
-Have we developed sustainable reimbursement models to fully embrace new these therapies?
Interactive Working Groups
Working Group 1:
Track 1
From Insight to Action: Leveraging Patient Experience Data Across the Rare Disease Product Lifecyle
Working Group 2:
Track 2
Every Newborn Counts: The Quest for Equitable Newborn Screening in Europe
Working Group 3:
Track 3
Balancing data privacy requirements with the need to collect important demographic data for RD research
Working Group 4:
Track 4
Paediatric studies for Rare Diseases
Working Group 5:
Track 5
Competition of regulatory ecosystems in approving medicines: policy implications in the case of Europe
Working Group 6:
Track 6
The Future of Cell & Gene Therapy for Rare Diseases
A new direction: creating a new Directorate for Health Innovation in France
Beyond Recruitment: The Value of Patient and Care Network Education in Rare Disease Clinical Research
Senior Representative, Ergomed
Clinical Trials in small populations form the EMA
Co-creation with Patients: 2026 classification
Panel: ATMP Centres of Excellence: How will this transform Europe?
- Why ATMPs are a unique opportunity to modernize HTA and drive the EUs competitiveness
- How do we get there?
- Post report patient perspectives
Panel: MFN: A regime change for Europe
R&D to commercialisation
The Role of AI in HEOR: Using AI to generate the Health Economics and Outcomes Research (HEOR) data needed to convince sceptical national payers
Using the new FDA/EMA-approved ´Pocock Win Ratio´ to increase success in rare disease external control trials for regulators and HTAs
Novel Platform for Oral Delivery targeting Duodenum
A new definition of Unmet Medical Need in EU pharma legislation; impact on innovation and access for patients
AI use in policy making – prediction models of reimbursement decisions
Payer strategy: towards sophisticated budget prioritization
-Outcomes-based vs multi-stakeholder approaches
-Innovative funding/contracting models
Unique challenges and unmet need for the treatment of Cushing Syndrome; Phase 2 clinical trial in Europe
Giving back what's theirs: empowering patients through individual data return
Keynote panel: Pushing Boundaries for the future – What’s next for Rare?
- Assessing a policy roadmap towards an EU and Global Action Plan for Rare Diseases (GAPRD)
- How will the WHO UN Resolution impact rare diseases in 2026 and what progress has been made?
- How can regions come together to harmonise, collaborate and put patients’ needs at the forefront of decision making?
CRISPR base-editing therapy: A rare genetic condition (CPS1)
Genome Editing: hemoglobinopathies as pioneers in research
Advancing Care Pathways for Rare and Undiagnosed Diseases: From Concept to Implementation
Measuring the burden and complexity of clinical trials from the patient perspective using a trial friction framework
Reserved for Alexion – AstraZeneca Rare Disease
Panel: Early Detection and Newborn Screening (NBS) in Rare diseases
Panel: Investor and Multistakeholder insights: What makes a Rare Trial De-Risked Globally?
Panel: Patient Centred Real-World Evidence for Rare Disease Decision-Making
Patient Movement Catalyst
Reimbursement timelines and key access challenges for Cell & Gene therapies in the Netherlands vs other EU countries
Modelling Rare Mitochondrial Diseases Caused by POLG Mutations for Drug Discovery in Children: Opportunities and Challenges
Raya is a mission driven company that is developing 5 different clinical stage NCEs for ALS and other neurodegenerative diseases
Panel: Collaborations between industry and European Reference Networks (ERNs): Unlocking the Potential
Best practices and challenges in industry-ERN partnerships
Reflections of Together4Rare and lessons learned
Panel: The diagnostic odyssey of patients with rare diseases
Panel: Investor and Multistakeholder insights: What makes a Rare Trial De-Risked Globally?
Panel: Optimizing pricing and access to rare disease drugs – Aligning with payers and health policymakers
Innovative treatments for neurological diseases
Reserved for Alexion – AstraZeneca Rare Disease
Orphan Drug Access Protocol
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Keynote panel: Redefining clinical value: Navigating the scientific and strategic era of EU Joint Clinical Assessments and Joint Scientific Consultations
- How can joint clinical assessments reshape patient access across Europe?
- How can joint scientific consultations influence global evidence strategies?
- Strategic implications of EUHTA for global clinical development
- Lessons for orphan medical products (OMPs), practical implications and decision-maker learnings
Keynote Panel: How are patients involved in every step of the orphan drug development and global access?
- Patient led solutions to orphan drug development and access bottlenecks
- How to solve the issue of small patient populations through statistical innovation, regulatory flexibility and patient-centricity
- Utilising Real-World Data and natural history studies
- Creating meaningful endpoints that improves patients’ quality of life
Fireside Chat: Exon-skipping therapy for the treatment of SMA Type 1
Chris Kessler, Father of a Child with a Rare Disease – SMA Type 1
Fondazione Telethon not-for-profit model: from research to distribution of ATMPs for ultra-rare conditions
Fostering shared responsibility in an evolving rare ecosystem
-How are public private partnerships advancing?
-How to create a mutually sustainable healthcare system
-Success factors for rare disease launches
Senior Representative, Norgine
Insilico trials modelling and simulation
Extrapolating data using machine learning/AI to create new learning and data
Panel: EU Biotech Act Phase 1
-How will the new biotech act impact Europe?
-What difference will this make in light of the Critical Medicines Act?
Panel: How do you assess evidence in Rare? The work of HTAi RDIG
Panel: Patient-Led R&D and "Co-Design"
‘N of 1’ clinical trial – How to drive efficiency in the clinical development programme for rare diseases with a single mutation
Engagement of young people in Rare Diseases Research: A roadmap for action
Eyes on the Future: RDH12 Gene Therapy for Inherited Childhood Blindness
Optimizing the Use of Data Sources and Registries
Senior Representative, IRDiRC
Panel: Expanding patient access to multi-indication medicines in Europe through innovative agreements
Case study example of multi-year, multi-indication (MYMI) agreements in Belgium, Italy, Netherlands and UK
Panel: The European Partnership for Personalised Medicine
The impact of the upcoming EU Pharmaceutical legislation on orphan and paediatric medicines
What does this mean for R&D of medicines for small populations?
How will the new mechanism of action provisions impact rare tumours and paediatric diseases with unmet needs?
Panel: What is the Role of Real-World Evidence for Orphan Products with Single-Arm Trials under the European Joint Clinical Assessment Framework?
Senior Representative, EUCOPE
Platform Approaches to Accelerate Gene Therapy Trials
Senior Representative, EUCOPE
The era of Antisense Oligonucleotides (ASOs), from personalised treatments to EU-wide authorisation
Evidence based care in rare diseases
-Patient involvement, policy and care coordination
Senior Representative, EURORDIS
Foster EU leadership in clinical trials for rare diseases through inclusive collaboration between Academy, ERNs, patient groups and industry to accelerate innovation for PLWRD
Panel: Implications of EU HTA Joint Clinical Assessments to Patient Engagement Practices in Rare Disease
Panel: Two Decisions, One Patient Journey: Aligning Regulators, HTA Bodies and Patients for Timely Access
Panel: A global perspective on funding companies developing orphan drugs
-How do investors oversee investment in the rare disease space?
-How does funding in the rare disease space fit into the overall biotech capital investment landscape?
-The types of business models in the orphan and rare disease space attracting funding from corporate investors and private equity firms
Do you need to plan for downstream reimbursement/ market access before investing
Cross Border access for RD patients
Growing emphasis on Ultra-rare conditions: challenge and opportunities and a new research paradigm?
Senior representative, RealiseD
Closing Keynote Panel: Pushing Boundaries for the future – What’s next for Rare?
-Assessing a policy roadmap towards an EU and Global Action Plan for Rare Diseases (GAPRD)
-How will the WHO UN Resolution impact rare diseases in 2026 and what progress has been made?
-How can regions come together to harmonise, collaborate and put patients’ needs at the forefront of decision making?
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