Conference Day Two | Thursday 9th October
7:15 am Registration & Morning Coffee
7:50 am Chair’s Opening Remarks
Strategic Access Planning: Managing Exit Strategies & Reimbursement from Start to Finish
8:00 am Panel Discussion: Navigating the Complexities of Market Access & Transitioning to Commercial Launch in the EU
Synopsis
• 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
8:30 am Bridging the Gap Between Regulatory Approval & Commercial Launch: Access Strategies
Synopsis
• 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
9:00 am Panel Discussion: Equity Beyond Borders – How Can We Improve Access in Low- & Middle-Income Countries
Synopsis
- 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 can take to make early access programs more inclusive, sustainable, and impactful in underserved region
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 Imperative
Synopsis
• 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
Synopsis
• 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
Synopsis
• 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 to Operationalise a Patient Centric Early Access Programme – A Multi Stakeholder Perspective
Synopsis
- Exploring operational strategies for embedding patient voices into Early Access Programme design and delivery
- Discussing ethical considerations for Early Access Programmes
- Case studies of patient advocacy and industry collaboration
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
Synopsis
• 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 programmes to broader early access frameworks
1:45 pm Real-World Data in Expanded Access: An Integral Part of Evidence Generation
Synopsis
- 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
2:15 pm Early Access Programmes: An Opportunity to Generate Early RWE & Share Experience
Synopsis
• Navigating compliance boundaries while collecting data within MAPs, learning from successes and overcoming key challenges
• Enhancing understanding of product safety by fulfilling mandatory safety reporting requirements
• Generating early real-world insights, sharing drug experience, and building advocacy through safety data analysis in EAPs
Optimising Quality, Safety & Supply Operations for Seamless Early Access Delivery
2:45 pm Roundtable: The Value of Scenario Planning in High-Risk, High Stakes Programmes
Synopsis
Managing an Early Access Programme is rarely straightforward – but what happens when logistics is the critical bottleneck standing between a patient and life saving treatment? This immersive, discussion-driven session puts participants in the role of a logistics lead navigating the urgent, high-stakes world of EAP delivery.
Participants will learn:
- The hidden logistical challenges unique to EAPs
- Why early planning and global coordination are critical
- How to balance urgency, compliance and cost
- The value of scenario planning in patient access programmes
3:15 pm Afternoon Break & Networking
3:45 pm Streamlining QP Release for Early Access Programmes for Seamless Operation
Synopsis
• Investigating the role of QP release in safeguarding patient safety and how it ties into broader regulatory and ethical considerations
• Exploring the most efficient approaches companies use to ensure quick QP release for early access programmes
• Examining how companies integrate QP release into their overall early access programme strategy for seamless operation
• Maintaining GMP compliance while ensuring timely product release for urgent patient needs in non-standard access settings
4:15 pm Ultimate Strategies in Setting Up EAP Sites for Success
Synopsis
- This session will focus on insights and case studies looking at strategies to plan for and manage site expectations within EAP and PTA Programmes. Often the misalignment between HCP expectations and the sponsor’s premise of an EAP can cause friction, even in the best planned programmes
4:45 pm Early Access Programme Operations in Small Biotech: Driving Operational Excellence from Strategy to Execution
Synopsis
• Streamlining the implementation of EAPs through effective operational planning and resource management in small biotech environments
• Leveraging practical lessons learned to drive efficiency in delivering MAPs from strategy to execution
5:15 pm Distribution Strategies for Early Access in Europe: Forecasting, Hubs & Supply Chain Optimization
Synopsis
• Evaluating distribution models: When and how to establish EU and/or UK hubs to streamline storage, labeling, and cross-border delivery
• Forecasting demand in early access programmes with limited visibility using trial data, advocacy input, and HCP insights
• Managing regulatory and logistical challenges, including country-specific labeling, customs clearance, cold chain integrity, and shipment tracking