Explore the Agenda

8:00 am Registration & Morning Coffee

8:50 am Chair’s Opening Remarks

Chief Commercial Officer, myTomorrows

Designing Region-Ready Expanded Access Programs in a Shifting Regulatory Landscape

9:00 am Building Managed Access Programs That Align with Development, Launch & Evidence Strategy – where to start? (US & Europe)

Director - Integrated Evidence Generation, Genmab
  • 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

9:30 am Roundtable – Middle East: Expanding Access in High-Demand, Rapidly Evolving Regulatory Environments

Global Medical Affairs, UltraPrograms, EAPs & ISTs Department Manager, Ultragenyx Pharmaceutical Inc.
  • Supporting access where regulatory frameworks are still evolving
  • Operating without local affiliates or established infrastructure
  • Managing political sensitivity, sanctions, and cross-border care realities

10:00 am Session Reserved for Early Access Care

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10:30 am Morning Break & Speed 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.

Post Trial Access: Planning Early, Transitioning Smoothly, & Executing Ethically for Sustainable Patient Access

11:30 am Designing Post-Trial Access Strategies Before the First Patient Enrolls

Director - Research Grants & Expanded Access, Alkermes plc
  • 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

12:00 pm Transitioning Patients Without Breaking Programs, Budgets, or Trust

Global Medical Affairs, UltraPrograms, EAPs & ISTs Department Manager, Ultragenyx Pharmaceutical Inc.
  • 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

12:30 pm Session Reserved for myTomorrows

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1:00 pm Lunch & Networking

Sustaining Expanded Access for Rare, Pediatric, and High-Cost Therapies

2:00 pm Planning for Long-Duration & Lifelong Treatment Access

Vice President - GU Portfolio & Medical Affairs, Exelixis, Inc.
  • Forecasting demand and cost for chronic, ultra-rare, and lifelong therapies
  • Budgeting for extended treatment durations beyond typical oncology models
  • Aligning ethical responsibility with long-term program sustainability
  • Preparing for patient and physician expectations when benefit continues

2:30 pm Pediatric Gene Therapy Access: Communicating with Families, Managing Expectations & Supporting Early Expanded Access Requests

Executive Director - Patient Advocacy, Tenaya Therapeutics
  • 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

3:00 pm Afternoon Break & Networking

3:45 pm Fireside Chat: Delivering Expanded Access for High-Complexity and One-Time Therapies

Director US Medical Director, Kite Pharma Inc
Senior Associate - Clinical Trial Management, Kite Pharma Inc
  • 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?

Managing Labeling, Forecasting, and Distribution Under Time Pressure

4:15 pm Maintaining Access When Supply Pathways Break Down

Senior Director, Supply Chain, Gilead Sciences
  • Forecasting demand for EAPs when patient numbers, geographies, and treatment duration are unknown
  • Coordinating with distributors and hospitals in regions with limited EAP infrastructure
  • Mitigating access delays in APAC and LMICs caused by importation, customs, and administrative hurdles

4:45 pm Chair’s Closing Remarks

Chief Commercial Officer, myTomorrows

5:00 pm End of Conference Day One