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:
- 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
- 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
- The European Parliament is currently reviewing the Pharmaceutical Package, which will potentially be voted on in April – this new text will outline the incentive and regulatory ecosystem for OMP developers. This will be a major milestone in the legislative review.
- The outcomes of these discussions will shape the landscape for OMP developers in the EU for the next 20 years.
- Reflect on the changes taking place outside the scope of the legislative framework, including the role of PPP and ERNs
- 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
- This presentation will focus on the role of RWD in natural history and patient registries.
- How data can be utilized to help inform study design
- The use of RWD as an external control and to find eligible patients for trials
- Using RWD with prior clinical trial data to better understand patient outcomes.
Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.
RESEARCH & DEVELOPMENT
- 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
- Overview of the current trends and recent advancements in NGS technology
- NGS applications in research and diagnostics
- Challenges encountered and future directions of NGS technology
- Different types of nucleotide-based therapeutics?
- What are the advantages of nucleic acid therapies
- Addressing the challenges with the safety and efficacy of these technologies in clinical practice
- Accelerating nucleotide-based therapeutic development through collaborations
- 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
- 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