Explore the Agenda
7:30 am Check-In & Morning Coffee
8:20 am Chair’s Opening Remarks
Designing Ethical, Defensible, & Actionable Data Strategies in Expanded Access
8:30 am Regulatory Considerations in Leveraging Data in Rare Disease Development
- Providing expanded and managed access pathways that balance patient access with the collection of data that may be informative for U.S. regulatory purposes
- Designing to leverage data to help address development gaps in rare diseases, such as underrepresented geographies (e.g., U.S. patients)
- Streamlining managed access processes to ensure programs are feasible and accessible for regulators and treating physicians, while maintaining strategic alignment across internal company stakeholders
Who Pays & How? Designing Sustainable Expanded Access Pathways
9:00 am Company‑Sponsored MAPs vs. Individual Access: Operational, Budgetary, & Compliance Trade‑Offs
- Comparing company‑sponsored MAPs versus individual patient access pathways, including when and why each model is used
- Examining budget ownership, FMV considerations, and cost‑of‑care challenges across therapeutic areas such as oncology versus rare disease
- Navigating compliance guardrails, protocol‑mandated activities, and site payment expectations
- Balancing increased company control and operational consistency against added administrative and financial complexity
9:30 am Designing a For-Charge Expanded Access Programme: The Role of Medical & Clinical Teams
In this interactive working session, attendees will explore how Medical and Clinical teams can help shape the design of a for-charge Expanded Access Programme once the decision to charge has been made. Drawing on real-world experience, the session will focus on the practical safeguards, considerations, and cross-functional decisions that ensure programmes remain patient-centric, ethical, and operationally sustainable. Participants will:
- Explore the key design considerations Medical and Clinical teams should influence when supporting a for-charge programme
- Discuss common safeguards, funding considerations, and patient access challenges that can impact programme design
- Assess and prioritise practical programme components through table-based discussion and peer benchmarking
- Leave with a framework of considerations they can take back to internal discussions around for-charge EAP strategy
10:15 am Morning Break & Networking
Building Sustainable Access: Provider, Patient, and System-Level Solutions
11:00 am Identifying Barriers & Solutions to Improve Expanded Access Enrollment
- New insights into knowledge gaps among U.S. oncology health care providers related to Expanded Access Programs
- Key patient enrollment and operational barriers encountered by providers and care teams
- Interactive discussion on how industry can better support oncology health care providers through education, tools, and process improvements
- Practical strategies to help providers more efficiently connect eligible patients to Expanded Access treatment options
11:45 am Patient-Led Models for Scaling Access Across High and Low-Income Countries
- Reaching underserved populations when traditional access mechanisms fail
- Managing chronic treatment access for thousands of patients across diverse health systems
- Coordinating pharma, clinicians, and patient organizations to reduce friction and delay
- Embedding access strategy early to support long-term equity and sustainability
12:15 pm Lunch & Networking
1:15 pm The Future of Early Access: Looking into the Crystal Ball – Scenarios That Could Reshape Patient Access
In this session we will explore what that future could look like for Early Access, and how could it evolve, using the EPISTLE+M methodology.
- Exploring how the role of Early Access may shift in the coming decade
- Examining the external forces quietly reshaping access pathways today
- Challenging current assumptions about what Early Access is, and what it could become
- Opening the discussion on what the future might demand from industry, regulators, and providers
Launching Expanded Access with Limited Teams, Budget & Infrastructure
1:45 pm Building Credible EAPs as a Team of One
- Establishing fit-for-purpose policies and documentation without large pharma systems
- Educating internal stakeholders on how EAPs differ from clinical trials
- Balancing strategic planning with day-to-day operational execution
- Building internal confidence and decision-making credibility without a dedicated team
2:15 pm Roundtable: Securing Buy-In, Funding, & Guardrails in Risk-Averse Organizations
- Navigating leadership concerns around regulatory, reputational, and approval risk
- Making the business and ethical case for EAP investment pre-approval
- Designing guardrails that protect programs without stalling access
Avoiding Patient Disruption as Expanded Access Ends & Commercial Begins
2:45 pm FDA Approval: Now What?
- Defining strategies for EAP patients that are now ready to be transferred to commercial products