Explore the Agenda
6:45 am Registration & Morning Coffee
7:20 am Chair’s Opening Remarks
Strategic Access Planning: Managing Exit Strategies & Reimbursement from Start to Finish
7:30 am Panel Discussion: Navigating the Complexities of Market Access & Transitioning to Commercial Launch in the EU
- Exploring the challenges of transitioning from early access to full reimbursement within the EU’s complex regulatory and reimbursement landscape
- Strategies for managing country-specific reimbursement systems and understanding the interplay between different EU member states’ policies to ensure smooth market access
- Ensuring continuous patient access during the transition from early access programs to commercial launch, focusing on cost-effective solutions and compliance
8:00 am Bridging the Gap Between Regulatory Approval & Commercial Launch: Access Strategies
- Leveraging flexible access pathways between regulatory approval and commercial launch to provide patients with access while minimising costs
- Exploring case studies of successful access strategies in Europe, highlighting how companies have navigated multiple access pathways and overcome challenges
- Identifying cost-saving opportunities in distribution, including localised distribution strategies and reducing reliance on clinical product shipments, without compromising patient access or safety
8:30 am Panel Discussion: Equity Beyond Borders – How Can We Improve Access in Low- & Middle-Income Countries
- Exploring regulatory, logistical, and financial challenges that limit patient access to medicines in LMICs
- Highlighting the role of partnerships with local stakeholders, advocacy groups, and regulators to bridge gaps in access
- Practical steps the industry
9:29 am Breaking Barriers to Early Access in Rare Diseases: Real-World Insights & Best Practices
9:30 am Fireside Discussion: Expanding Compassionate Use for Rare & Small Patient Populations: Emerging Models & Ethical Imperatives
- Adapting compassionate use frameworks for ultra rare conditions as regulatory and ethical models evolve to support access without robust trial data
- Enabling cross-stakeholder coordination through pilot initiatives like the UK’s Rare Therapies Launchpad to build infrastructure for small population access
- Balancing safety, speed, and evidence generation for regulators, clinicians, and sponsors supporting compassionate use in paediatric rare disease settings
10:00 am Morning Break & Networking
10:30 am Running an Early Access Programme for Rare & Ultra-Rare Diseases: Real- World Lessons
- Case studies on successfully setting up and managing rare disease access programmes
- Understanding key regulatory, ethical, and logistical challenges unique to rare diseases
- Highlighting lessons learned: What worked, what didn’t, and what needs to change
11:00 am Navigating Named Patient Requests: Optimising Access for Rare Disease Patients Across Europe
- Tackling real-world challenges in securing early access for rare disease patients through named patient programmes across diverse European healthcare systems
- Exploring country-specific regulatory nuances and their impact on rare disease access pathways
- Gaining practical insights and strategies for coordinating named patient programmes, overcoming cross-jurisdictional hurdles, and ensuring patient access
11:30 am Panel: How How to Operationalise a Patient Centric Early Access Programme? – A Multi Stakeholder Perspective
12:15 pm Lunch & Networking
Streamlining Real-World Data Collection & Use in Early Access Programmes
1:15 pm Real-World Data in EU Early Access Programmes: GSK Case Study
- Defining the minimal dataset that aligns with both regulatory and payor requirements while maintaining feasibility in early access settings
- Addressing variability in RWD collection across EU countries and navigating differing expectations from regulators and health technology assessment (HTA) bodies
- Overcoming data collection limitations in different EAP models, from named-patient programs to broader early access frameworks
1:45 pm Real-World Data in Expanded Access: An Integral Part of Evidence Generation
- Gaining new insights into the practical application of RWD from EAPs through continuation of PhD research in collaboration with University Medical Center Utrecht (2021–2025)
- Learning how to embed EAP-derived RWD into integrated evidence generation that supports both clinical development and post-approval commitments
- Discovering how proactive workflows and high-quality standards maximize data utility for regulatory and payor submissions
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