Conference Day Two | Thursday 9th October
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 Fireside 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
8:00 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
8:30 am Session Reserved for BAP Pharma
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 Designing & Delivering Early Access for Gene Therapies: Lessons from Lenadogene Nolparvovec in Leber Hereditary Optic Neuropathy
Synopsis
• Meeting urgent unmet need in Leber Hereditary Optic Neuropathy through early access
• Exploring unique considerations for gene therapy in rare disease populations
• Overcoming operational, regulatory, and logistical hurdles throughout the EAP journey
• Actionable insights and takeaways for future gene therapy early access programmes
11:00 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
11:30 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
12:00 pm Session Reserved for Inceptua
12:30 pm Lunch & Networking
Streamlining Real-World Data Collection & Use in Early Access Programmes
1:30 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 programs to broader early access frameworks
2:00 pm Session Reserved for Almac
2:30 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
3:00 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
3:30 pm Afternoon Break & Networking
4:00 pm Session Reserved for WEP
4:30 pm Understanding QP Certification in Early Access: Legal Triggers, Country Variation & QP Responsibilities
Synopsis
• Differentiating QP requirements across early access types, including compassionate use, named patient programmes, and cohort-based schemes
• Navigating fragmented EU and UK expectations for QP release in non-commercial product settings
• Clarifying QP responsibilities for oversight, documentation, and compliance amid diverse regulatory interpretations
5:00 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
5:30 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