At last year’s Operationalize Expanded Access Programs Summit, I explored a question that was on many Sponsors’ minds:
“What is the value of collecting real world data in EAPs, and how can it support commercial goals?”
The discussion sparked strong engagement. Sponsors clearly saw the opportunity to drive value through strategic real world data collection. Yet there was still uncertainty around what data could be collected, how it should be structured, and how it could ultimately be used.
One Year On: A Shift In Mindset
Over the past 12 months, the real world evidence (RWE) landscape in the United States has continued to mature, driven by increasing regulatory alignment and growing Sponsor confidence.
This means that EAPs are no longer being viewed purely as access mechanisms, with RWD capture as a ‘nice to have’ and somewhat ambiguous component. Instead, they are increasingly being seen as clear RWE-generating opportunities and, as a result, as strategic extensions of development and commercialization pathways.
This shift is reflected in the questions Sponsors are now asking:
- Are endpoints defined in a way that supports interpretation?
- Is data capture consistent across sites?
- Can the dataset withstand regulatory scrutiny?
In other words, the conversation has moved from: “Can we collect data?” to “Are we collecting the right data?”
Regulatory Openness – But Not Without Expectations
Regulators, including the US FDA, have continued to signal openness to the use of real world evidence in regulatory decision-making.
However, the use of EAP-derived data in approvals remains relatively limited, and highly dependent on data quality, structure, and context. This reinforces an important point: the value of EAP data is not guaranteed – it is earned through how the program is designed and executed.
The Emerging Gap: Execution
Despite increased awareness and clearer regulatory signals, one challenge remains: execution is still catching up with opportunity. Many programs still face:
- inconsistent data capture across sites
- unclear governance structures
- limited alignment between access and evidence-generation objectives
As a result, many programs with real potential to generate meaningful evidence, end up with datasets that fall short of being interpretable or regulatory-ready.
Why This Matters Now
This brings us to our presentation at this year’s Operationalize Expanded Access Programs Summit:
“The Hidden Cost of Poor EAP Operations: What Regulators Actually Notice.”
The focus is deliberate. As the regulatory and commercial importance of RWE continues to grow, it has become increasingly clear that the gap is no longer conceptual – it is operational. This means the margin for error is narrowing and operational decisions are no longer neutral.
To generate reliable, regulatory-ready evidence from EAPs, three elements consistently stand out:
Clear governance
Defined roles, responsibilities, and oversight structures that ensure consistency and compliance.
Thoughtful data capture
Intentional endpoint selection and standardized collection aligned with downstream evidence goals.
Strong site engagement
Ensuring sites understand not just what to collect, but why it matters – leading to better completeness and data integrity.
This is where many programs succeed or fall short and where we will focus our attention during our panel discussion.
Join The Conversation In Boston
If you will be attending Operationalize Expanded Access Programs Summit 2026, our team will be sharing practical perspectives on how to bridge this gap.
And if you are not attending, but are thinking about how to make data from your EAPs more usable and impactful, we would be happy to continue the conversation.
