23rd Orphan Drugs and Rare Diseases Global Congress 2025 Europe
Addressing the challenges in drug development, treatment availability, and access.
Copthorne Tara Hotel London Kensington, London, UK, London, United Kingdom
Day 1 - Wednesday 19th March 2025
LANDSCAPE OVERVIEW & CURRENT TRENDS
- Addressing the potential gap in the framework and policy recommendations which support the broad range of people experiencing rare conditions.
- Strategies for more efficient ways of utilising data for earlier detection and diagnosis of rare diseases
- Ensuring equity in access to specialised care and innovative treatments.
- Improving the delivery of clinical research and communication
Moderator:
Panellists:
Stefano Benvenuti, Head of Public Affairs and Market Access, Fondazione Telethon
- Progress update on the actions set out in previous plans
- New actions included in the Rare Disease Action Plan for England from 2024
- Ensuring the plan delivers for people affected by rare conditions
- Reflections on a Rare Disease Action Plan and a coordinated legislative framework
- How a new EU legislative framework defined by the upcoming review of the General Pharmaceutical Legislation, anticipated Biotechnology Act, and life science strategy shape the OMP landscape
- Initial reflections on the EU HTA Regulation and its implications on the OMP and ATMPs will impact the OMP landscape
Alexander Natz, Secretary General, EUCOPE
- Disrupt encourages groundbreaking solutions that fill unmet medical needs.
- Adopt focuses on strategies to ensure market acceptance through regulatory approvals and commercialization.
- Collaborate highlights building partnerships across academia, industry, and regulators.
- Manage emphasizes effective leadership and risk management.
- Adapt stresses flexibility in dynamic regulatory and market environments.
- Resource centers on optimizing financial, human, and technological resources.
Dr James Levine, President, Fondation Ipsen
11:15 am - PLENARY SESSION: Commercialisation of Orphan Drugs: Top issues facing the community today
- Innovative changes that will impact the rare disease community
- Stakeholders collaboration to continue fulfilling unmet needs
- What R&D strategies can be implemented to accelerate the market access for orphan drugs?
- Challenges in bringing orphan drugs to patients after approval
- Practical challenges in conducting orphan drug clinical trials
- Technical aspects of clinical trial design for rare diseases
- Analytical and statistical considerations for clinical trial designs
- Recommendations for improving the clinical trial design of rare diseases
- The idea for this presentation is to explain about data ownership and how Pulse addresses this through our agreements etc.
- We could look at exploring how this has become a hot topic, why, and why industry and patients needs to be aware of the complexity of the subject.
Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.
- Terminology
- Different pathways and their pros and cons
- Specific regulations in several European countries
Dr. Rüdiger Schulze, Vice President and General Manager, DACH, Ultragenyx
RESEARCH & DEVELOPMENT
- Muscular dystrophy as paradigm for orphan drug development
- Most promising gene therapies strategies for the treatment of muscular dystrophy
- Future perspectives; orphan drugs in the pipeline
Alberto Malerba, Gene Medicine Laboratory for Rare Diseases, Royal Holloway, University of London
- VCN-01 oncolytic virus design
- Ph1 clinical data results
- Hurdles encountered during development of a new product in retinoblastoma
Manel Cascalló, General Director EU Subsidiary & Founder, Theriva Biologics
- How do we put a true value on innovation in rare disease medicines development?
Jennifer Schranz, SVP, Global Head of Rare Diseases, Ipsen
- The unmet need in OI
- Rationale for using mesenchymal stem cells
- Challenges of drug development for OI, particularly in very young children
Anthony Hall, Co-Founder, Member of Board & Scientific Advisor, Beacon for Rare Diseases
- How to accelerate drug repositioning?
- Preclinical models and drug testing
- Fast-tracked drug access for patients
Prof Guillaume Canaud, Translational Medicine and Targeted Therapies Unit, Necker Enfants Malades
- Role of RWD/E in orphan disease drug development
- Pitfalls in the use of RWD/E in orphan drug development
- Repurposing and RWD/E
Surabhi Verma, Chief Medical Officer, Leadiant Biosciences
- Remarkable advancements in rare disease research
- Addressing obstacles in development of new treatments and access to trials
- Significant challenges faced by orphan drug developer
- Key initiatives that can guide collaborative approaches and innovative strategies to accelerate progress in the rare-disease field.
- Drug repurposing for rare disease