Conference Day One | Wednesday 8th October

7:00 am Registration & Morning Coffee

7:50 am Chair’s Opening Remarks

Driving Faster Access: Shaping Policy & Navigating Regulations to Accelerate Early Access in Europe

8:00 am Fireside Discussion: A Complex Regulatory Environment: Navigating Early Access Challenges in the EU for Better, More Timely Patient Support

  • Chiara Troncatti Head of Business Operations, EMEA, Avidity Biosciences
  • Nina Heiss Director Global Regulatory Affairs and Scientific Policy, PhD, Merck KGaA

Synopsis

• Navigating the complexities of the EU’s regulatory framework for early access: Key challenges and opportunities

• Identifying regulatory bottlenecks and how they affect the speed of patient access in the EU

• Practical strategies for aligning early access programmes with regional and national regulations while maintaining compliance across borders

• Discussing how the evolving regulatory landscape in the EU influences future early access strategies and industry collaboration

8:30 am Rapid Fire Solutions: Tackling Real-World EAP Challenges

  • Annmarie Galli , Head, Global Medical Affairs Research & Compassionate Use / Managed Access Programs, GSK
  • Rachel Harrison Director, Pre-Approval, Post-Trial Access Programmes, Medical Affairs & Evidence Generation, Argenx

Synopsis

From patient burden to last-minute formulation changes, this fast-paced session delivers practical, on-the-ground solutions to common EAP challenges. Featuring a series of concise, ten-minute spotlights from leading biopharma, each presentation highlights a single innovation or tactic that helped unlock access, streamline processes, or support patient continuity. Designed to fuel actionable discussion, this session goes beyond the theory – focusing on what’s actually working, and where we go next.

9:00 am Navigating the Landscape of Patient Access & Support Pathways

Synopsis

• Establishing clear criteria and governance for diverse patient access pathways, including pre-approval, post-trial, and commercial import requests

• Lessons learned from creating and refining policies to close gaps and support patients

• Strategies for ensuring the longevity and scalability of managed access programmes while adapting to internal and external challenges

9:30 am Session Reserved for Early Access Care

10:00 am Speed Networking

Synopsis

Join our speed networking session, tailored for Early 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.

10:30 am Morning Break

Navigating Post-Trial Access in Europe: Ethical Imperatives & Practical Barriers

11:00 am Regulatory Feasibility of Post-Trial Access: Meeting Legal & Ethical Obligations

  • Ben Rotz Associate Vice President - Global Medical Policy, Strategy, & Operations, Eli Lilly & Co.

Synopsis

• Clarifying EU-specific regulatory expectations and legal requirements for post-trial access across member states

• Evaluating feasibility and compliance challenges under GDPR, national regulations, and EMA guidance

• Addressing ethical responsibilities and patient care obligations in the absence of harmonised EU-wide post-trial access policies

11:30 am Defining Overall strategic Roadmap for Post-Trial Access implementation & Evolution

Synopsis

• Collaborating with internal stakeholders at early stages of programme development

• Developing PTA strategies considering disease severity and country-specific regulatory pathways and launch readiness

• Set-up, conduct PTA programmes following internal processes and adapt programmes according to unplanned events

12:00 pm Session Reserved for Bionical Emas

12:30 pm Post-Trial Access: Making it Work Through EAP Mechanisms

Synopsis

• EAP vs. OLE: Choosing the Optimal Pathway to Ensure Patient Continuity and Minimise Operational Burden

• Adapting PTA Operations to Evolving Global Regulations, Internal Readiness & Real-World Complexity

1:00 pm Lunch & Networking

2:00 pm Session Reserved for Marken

Enhancing Early Access Program Success by Bridging Patient, HCP & Advocacy Group Priorities

2:10 pm Building Internal Readiness: Defining Roles & Responsibilities Without a Dedicated EAP Team

  • Maja Gallic Head of Global Medical Affairs Operations & Excellence, Teva Pharmaceuticals

Synopsis

• Navigating early access planning in the absence of a centralised or dedicated EAP function

• Exploring lessons learned from Teva’s evolving operating model and Centre of Excellence approach

• Creating a cross-functional matrix that works: aligning stakeholders across R&D

2:40 pm Session Reserved for myTomorrows

3:10 pm Achieving Stakeholder Alignment in Early Access Programmes: Strategies for Clarity, Consent & Collaboration

Synopsis

• Mapping key stakeholder roles and identifying potential conflicts of interest or misalignment early in programme development

• Practical steps to ensure clear, transparent communication with patients, HCPs, and advocacy groups to manage expectations and ethical concerns

• Case examples of how stakeholder alignment improved operational efficiency, patient enrolment, and long-term treatment outcomes in rare and non-rare diseases

3:40 pm Afternoon Break & Networking

Driving Early Access Success in Diverse Global Markets

4:15 pm Session Reserved for Uniphar

4:45 pm Providing Sustainable Access to Medicines in Low- & Middle-Income Countries

Synopsis

• Exploring the challenges and solutions for providing sustainable access to medicines in LMICs, with a focus on rare diseases

• Sharing insights from the Africa Roadmap project and its impact on connecting countries to early access programmes

• Discussing how innovative approaches, including data sharing and registries, can improve access and support rare disease treatments in resource-constrained regions

5:15 pm The Future is Home: Innovations in Chronic Therapies for Rare Disease Patients

  • Dorian Readnour Executive Director - global patient access, Sarepta Therapeutics
  • Suzanne Webster Director - International Patient Services & Global Access Post Clinical Trial, Sarepta Therapeutics

Synopsis

• Outlining key factors in establishing home health programmes

• Overcoming obstacles encountered by hospitals and patients

• Unveiling the advantages of creating and implementing a home health programme

5:45 pm Chair’s Closing Remarks

5:50 pm End of Conference Day One