Looking ahead to the 5th Operationalise: Early Access Programmes Summit Europe

Ahead of the 5th Operationalise: Early Access Programmes Summit Europe in London next week, Joanne Walker of The Evidence Base previews selected sessions and what attendees can expect from discussions on patient needs, evidence generation and the practical delivery of early access programs.

Early access programs (EAPs) are an essential route for patients with serious or life-threatening conditions and limited treatment options to receive medicines that are not yet authorized or commercially available in their country. Establishing these programs requires careful consideration of patient eligibility, regulatory requirements and continuity of treatment, alongside opportunities to collect real-world data (RWD) without placing undue burdens on patients or clinicians.

The 5th Operationalise: Early Access Programmes Summit Europe, taking place on October 19–21, 2026, in London, UK, will examine how organizations address these challenges across different healthcare systems. The Evidence Base will participate for the first time as a media partner, with Joanne Walker attending to share speaker insights and discussions with our audience. Ahead of the meeting, our preview explores five areas where program teams must balance competing demands.

Responding to urgency while acknowledging uncertainty

For patients and families seeking treatment, waiting has consequences that need to be understood alongside the uncertainties associated with an investigational medicine. How patients participate in these decisions, and whose assessment of acceptable risk informs the outcome, are important questions for EAPs.

In “Challenging the Status Quo in Early Access – Addressing the Barriers,” Robert Mitchell-Thain (The PBC Foundation) will examine obstacles to access and patient involvement in decisions about risk. His session will consider whether commonly cited barriers can be challenged and how stakeholders can discuss access more openly.

Meryl Chambers (Soft Bones UK Hypophosphatasia Foundation) will share her family’s experience in “How Early Access to an Investigational Medicine Saved My Son’s Life: A Parent’s Perspective.” Chambers will discuss navigating access during a family crisis, the longer-term impact of treatment and how that experience informed the creation of a patient advocacy organization.

The main question will be how patient perspectives influence program decisions when patients, clinicians and organizations have different views about treatment risks and the implications of waiting, and how those differences can be addressed in practice.

Generating evidence while keeping treatment at the center

EAPs may offer opportunities to learn about a medicine’s use, but deciding what data to collect requires careful consideration of its purpose and the demands placed on patients and healthcare professionals. For The Evidence Base’s audience, this raises questions about where these programs can contribute useful evidence and how data collection can fit within routine program delivery.

The panel “Driving Smarter Decisions by Clarifying the Role of RWD in Early Access Programmes” will bring together Romina Oxborough (Maprium), Flavie Faist (Sobi), Nora Pöntynen (LEO Pharma) and Corinde Staring (Genmab) to examine different approaches to RWD, from programs focused solely on treatment access to those incorporating structured evidence generation. The discussion will consider how authorities interpret these data and the ethical and regulatory boundaries of collection when patient care is the primary purpose.

Practical experience will feature in “Real World Data Collection through Early Access Programmes: Turning Theory into Practice,” presented by Rakesh Davda and Tom Watson (Bionical Emas) at the end of Day 1. Drawing on a local case study, including its methodology and publications, the session will share lessons and recommendations for collecting RWD through EAPs.

For attendees planning evidence generation, these sessions offer an opportunity to consider which research questions are appropriate to the setting, how data collection can fit into clinical practice and what the findings can reasonably contribute to subsequent decisions.

Seeking consistency while responding to local realities

Organizations running programs across countries need clear responsibilities and processes while accommodating differences in national pathways and healthcare systems. The challenge is to establish consistent standards without restricting the flexibility needed to deliver access locally.

In “Early Access in Europe: A Working Theory for Greater Alignment Across Systems,” Peter Takacs (Miltenyi Biomedicine) will explore fragmentation across European pathways and opportunities for greater alignment. The session will consider implications for industry, payers and HTA decision-making, including parallels with emerging European frameworks such as joint clinical assessment.

Ayse Ozlem Yilmaz (Eisai) will address implementation across different settings in “Executing Early Access Programmes Across Emerging Markets: Navigating Complexity, Access Pathways & Sustainability.” Her presentation will examine locally adapted approaches across Central and Eastern Europe, the Middle East and North Africa, and other developing markets, considering patient needs, government priorities and program sustainability.

These discussions should offer useful perspectives on which elements of EAP design can be shared across countries, and which require local adaptation. Of particular interest will be the implications for access and evidence collection when an approach developed in one healthcare system is introduced elsewhere.

Improving awareness while respecting communication boundaries

An access pathway can only help patients if their clinicians know it exists and understand how to use it. This creates a challenge for program teams seeking to provide useful information while respecting the boundaries around communication about unlicensed medicines.

“The Communication Challenge: Navigating awareness & HCP support in EAPs” will feature Ramona Reichenbach (Novartis), Walter Colsanate (The Medicine Access Channel), and Katie Bowen and Debora de Paula (BAP Pharma). The session will consider whether limited awareness of access routes can favor well-connected centers, established clinical networks or more vocal patient communities, and how factual, appropriately governed information can support fairer access.

Gianluca Parziale (Alnylam Pharmaceuticals) will address the responsibilities involved in “Strengthening Physician & Site Engagement Without Creating Compliance or Delivery Risk.” The session will examine how sponsors can support sites without crossing promotional, regulatory or ethical boundaries, including how differences in site experience and uncertainty around documentation and reporting can delay access.

Beyond awareness, effective communication needs to give clinicians a clear understanding of their responsibilities and the support available to them. Attendees can expect practical discussion of how to provide that clarity while preserving clinical independence and avoiding inappropriate expectations.

Enabling access while planning for continued treatment

Decisions made before treatment begins can have lasting implications for patients, particularly when clinical trials end or responsibility for a program changes. Planning for these transitions requires clarity about commitments, supply and the arrangements needed to avoid interruptions.

On Day 2, Olga Andreeva (Novo Nordisk) and Ann Watkins (Jazz Pharmaceuticals) will discuss practical experiences in “Executing Post Trial Access When Clinical Trial Systems Switch Off.” Their case studies will explore the transition from trial supply to post-trial access, including changes to site responsibilities, ordering processes and reporting requirements.

Later that day, Antoine Robert (Sanofi) will address these issues in “Planning Post Trial Access Early: Choosing the Right Pathway Before Trials Close.” The session will examine how post-trial access obligations can be considered during trial design, with budgets, supply and country-level feasibility addressed before trials finish.

For program teams, the practical test is whether plans made before a trial closes translate into reliable treatment access afterward, with clear responsibilities, workable supply arrangements and sufficient support for clinicians and sites during the transition.