Explore the Agenda
7:30 am Registration & Morning Coffee
8:25 am Chair’s Opening Remarks
Designing Region-Ready Expanded Access Programs in a Shifting Regulatory Landscape
8:30 am Operationalizing Expanded Access in 2026: From Strategy to Scalable Execution
- Understanding how Expanded Access expectations have evolved following recent FDA guidance – and what this means for program design today
- Navigating the realities of implementing global EAPs across regions with differing regulatory frameworks and infrastructure
- Establishing clear ownership: which functions should lead, support, and govern Expanded Access programs as they scale
9:00 am When Access Creates Evidence: Real-World Data from Expanded Access Programs
- Real-world evidence generated through EAPs that has informed regulatory decisionmaking, contributing to EMA scientific advice discussions and, in specific cases, resulted in FDA labeling indications and marketing authorizations
- This session will focus on the types of approaches and real regulatory results
9:30 am Expanded Access in Latin America: Real‑World Lessons from Rare Disease Programs
- Navigating regulatory and importation bottlenecks in Latin America, including the impact of government changes and evolving approval pathways (with a focus on Mexico, Argentina, and Brazil)
- Managing operational and supply‑chain challenges in rare disease Expanded Access Programs while balancing global standards with country‑specific realities
- Strengthening physician engagement to sustain program momentum and patient access in high‑complexity regions
10:00 am Early Access Community Roundtables: Learn, Share & Solve Together
This interactive session puts you at the centre of the discussion. Join a small group of peers and take part in a structured exchange to share what you are currently working on, where you’re facing challenges, and what you need to move forward.
- Introducing your current programme context, including key challenges around regulation, data, supply, or execution
- Sharing what you are trying to solve right now, from pathway selection to scaling, transitions, or governance
- Collaboratively identifying practical solutions, workarounds, and approaches you can take back and apply immediately
10:30 am Morning Break & Networking
Join our speed networking session, tailored for Expanded Access experts like yourself, to connect with fellow industry peers to facilitate a rapid yet meaningful exchange of insights and expertise. Elevate your networking experience during this session designed for impactful connecting within the space.
11:30 am Building Managed Access Programs That Align with Development, Launch & Evidence Strategy – Where to Start? (US & Europe)
- Deciding when and where to initiate managed access across the US and Europe as assets approach planning for submission
- Structuring early access, individual patient, protocol-based, and post-trial access pathways within a single coherent strategy
- Aligning managed access programs with clinical development plans to avoid trial interference while meeting unmet needs
- Navigating labeling, launch sequencing, and regional differences when operationalizing programs pre-approval
- Embedding managed access into broader investigator-driven research and evidencegeneration capabilities
Post Trial Access: Planning Early, Transitioning Smoothly, & Executing Ethically for Sustainable Patient Access
12:00 pm Designing Post-Trial Access Strategies Before the First Patient Enrolls
- Embedding post-trial access planning into clinical trial design to avoid last-minute ethical and operational dilemmas
- Evaluating when post-trial access, long-term extensions, or hybrid approaches make the most sense by disease, geography, and duration
- Anticipating regulatory constraints that may prevent post-trial access in certain countries and adjusting trial footprints accordingly
- Aligning clinical development, medical affairs, and access teams early to support continuity of care
12:30 pm Transitioning Patients Without Breaking Programs, Budgets, or Trust
- Managing patient transitions as programs move from trial to post-trial access and into commercialization
- Navigating internal handoffs as responsibility shifts from clinical operations to medical affairs or access teams
- Balancing ethical obligations to patients with resource constraints and long-term sustainability
1:00 pm Lunch & Networking
2:00 pm Panel Discussion: Driving Smarter Decisions by Clarifying the Role of RWD in EAPs
- Comparing how FDA, EMA, and national health authorities interpret the use of RWD in expanded and post-trial access programs
- Examining differing company approaches, from limiting programs to treatment-only access to actively incorporating structured evidence generation
- Exploring real-world examples of where regulators accepted or rejected RWD submissions in filings and approvals
- Defining the boundaries of ethical and compliant RWD collection when patient care is the primary purpose
2:30 pm Recent US Regulatory Updates & Future Global Implications for EAPs
- Understanding how EAPs are shifting to integrated access and evidence models generating real-world data
- Accounting for how regulators are formalizing expectations on justification, risk– benefit, and alignment with development strategy
- Learning how external pressures shape expanded access, including MFN/pricing signals, FDA changes, and geopolitics
3:00 pm Network & Learn Break: Access Insights Session
Join us during the afternoon break in the exhibition hall for an interactive Access Insights Session. This informal networking opportunity invites attendees to connect with peers, explore real-world experiences, and exchange practical insights shaping today’s Expanded Access landscape.
Use this dedicated time to discover innovative approaches, discuss operational and regulatory learnings, and engage with colleagues committed to advancing ethical, patient-focused access pathways.
Sustaining Expanded Access for Rare, Pediatric, and High-Cost Therapies
3:45 pm Pediatric Gene Therapy Access: Communicating with Families, Managing Expectations & Supporting Early Expanded Access Requests
- Educating patients and families about the risks and benefits of gene therapy and the expanded access process
- Communicating transparently when programs are early and pediatric data is limited
- Working compliantly with patient advocacy groups, who are monitoring clinical trials and other potential pathways to treatment
- Managing sensitive conversations around eligibility, timelines, and uncertainty in lifelimiting pediatric conditions
4:15 pm Fireside Chat: Delivering Expanded Access for High-Complexity and One-Time Therapies
- How do companies practically operationalize Expanded Access Programs for cell and gene therapies?
- What factors most often determine feasibility when manufacturing constraints, logistics, and cost are extreme?
- How do organizations assess when expanded access is realistic – and when it risks creating false hope for patients and providers?
- How can regulatory incentives and long-term development strategy be thoughtfully integrated into Expanded Access decision-making?