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Hollistic Approaches to Rare Disease Drug Development: Integrating ESG, Patient Engagement, and Commercial Strategy for Improved EU Access
Lysosomal Storage Disorders (LSDs) present significant challenges, including delayed diagnoses, fragmented care pathways, and limited access to specialist care. Digital health technologies offer a transformative opportunity to address unmet needs by enabling earlier intervention, enhancing care coordination, and empowering patients and healthcare providers with innovative tools.
Utilizing interactive tools and featuring multi-stakeholder perspectives from patients to industry and policymakers, this discussion will explore the integration of digital health innovations ranging from AI to telemedicine, digital medical devices, and other connected care technologies into care pathways for rare diseases across Europe
Rare diseases pose unique challenges to healthcare systems worldwide due to their low prevalence and complex treatment requirements. Traditional financing mechanisms often fail to adequately address the needs of patients with rare diseases, leading to disparities in access to treatment and care. Last June, RDI (Rare Diseases International) launched a campaign for a World Health Assembly (WHA) Resolution on Rare Diseases in 2025. RDI is launching a campaign for a World Health Assembly (WHA) Resolution on Rare Diseases in 2025.
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Understanding patient experiences, preferences, and behaviours is critical to developing treatments that truly meet patient needs. Yet in rare diseases, capturing and applying these insights remains difficult due to small, diverse populations, delayed diagnoses, a lack of standardized methods, and limited regulatory guidance.
This panel will explore how both qualitative, and semi-quantitative patient data can inform more effective, patient-centred decisions across the product lifecycle in the rare disease space. Experts will address key barriers, such as data integration, resource constraints, and methodological gaps, and share practical strategies and real-world examples to overcome them.
Join the panel and gain a clear view of what meaningful patient experience data look like and how they can drive clinical development strategies, as well as pre-launch and market success
Reserved for Clinigen
Reserved for Decisive Consulting
To invite:
Constanz Blume, Global Regulatory Affairs, BionTech
Kent Rogers, CEO, Everyone Medicines
Senior EMA Representative
Senior Representative, UCB
Senior Representative, EUCOPE
More speakers to be confirmed..
More speakers to be confirmed..
Senior Representative, UCB
-Hepcludex reimbursement hurdles (a.o.sudden recall FE exemption, conditional approval; European guideline vs NL HCP position statement)
-Yescarta in 2L DLBCL (a.o. DRG estimated costs vs RW-costs; long waiting time input profession)
Invited:
Francis Pang, SVP, Global Market Access & International Geographic Expansion, Orchard Therapeutics
Liz Fowler, Former Director, CMS Innovation Center; Former VP, Global Health Policy, J&J
Rosana Sovani, Partner, Head of Legal Public Affairs, LS CUBE Studio Legale
Invited:
Senior HTA Representative
Senior Clinician
Senior Geneticist
Invited speakers:
Prof. Peter Mol, Professor, Drug Regulatory Science, University Medical Center Groningen, CHMP Member, CBG-MEB
Senior ERN Representative
Senior Patient Representative
Senior Representative, EVIDERA
Invited:
GBA, H-AS, NICE
Jakub Dvořáček,Deputy Minister,Ministry of Health of Czech Republic – tbc
Adrian Goretzki, President, Healthcare Education Institute – tbc
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Sources of uncertainty in rare disease endpoints and strategies to address them.
Standardizing endpoint definitions and assessment criteria.
Use of blinded independent adjudication committees.
Practical steps for implementing effective adjudication processes.
Invited:
Frauke Naumann-Winter, Committee for Orphan Medicinal Products, Institute for Drugs and Medical Devices – TBC
Invited speakers:
Senior Representative, Miltenyi Biotech
Senior Representative, European Commission
More to be confirmed..
Invited:
Senior Representative, HollandBio
Senior Representative, CSL
Senior ERN Representative
This panel will explore thepractical implications that industry and patient organizations need to consider when engaging given the evolving landscape of HTA in Europe
Invited:
Sofie Alverlind,Coordinator and Project Leader,TLV
We are pleased to confirm the proposed session topics for our Drug Repositioning segment. These include:
Assets (Shelved Assets)– exploring how previously shelved drug candidates can be repurposed for rare diseases.
Success Story (Gomekli)– highlighting the case of Gomekli (mirdametinib) as a successful drug repositioning story.
Accelerator (European Drug Discovery Engine by CTF Europe)– introducing CTF Europe’s new orphan drug discovery engine accelerator initiative.
Investors– discussing how public and private entities can support drug repositioning, and the business rationale behind their involvement.
Invited speaker:
Dr. Bart Van de Warrenburg, Professor of Neurology & Medical Lead, Center for Rare and Genetic movement disorders, Radboudumc
Senior Representative, C-Path
Senior Representative, Miltenyi Biotec
Invited:
Senior Representative, Novo Nordisk
European Hemophilia Consortium
ERN Blood
Senior Representative, FH Europe
More speakers to be confirmed..
Invited: Dr Mohamed Hassany, Assistant Minister for Public Health Initiative, Ministry of Health Egypt