Scaling Expanded Access: What Pharma Leaders Need to Prepare for Next
Expanded Access is changing.
Once viewed primarily as an exception pathway for individual patients who had exhausted other treatment options, Expanded Access Programs (EAPs) are increasingly becoming a strategic and operational consideration for pharmaceutical and biotechnology companies.
Patients are more informed. Physicians have greater visibility into investigational therapies. Patient advocacy organizations are playing a larger role. Programs are becoming more global. And companies are being asked to think about Expanded Access earlier in the development lifecycle.
These shifts were at the center of a recent MedaSystems webinar, “Scaling Expanded Access Programs in a Complex Global Environment,” featuring Gretchen Randlett of Eli Lilly and Sashin Bhuta of Bristol Myers Squibb, moderated by Matt Rosen, Co-Founder and CEO of MedaSystems.
The conversation explored the operational realities behind Expanded Access—from supply planning and global regulations to post-trial access, technology, AI, and the growing need for cross-functional coordination.
Here are several of the most important themes that emerged.
Expanded Access Is Becoming More Visible—and More Strategic
One of the biggest changes in Expanded Access is simply awareness.
Patients today have significantly more access to information about potential therapies. Physicians can learn about emerging clinical data through scientific conferences, publications, online resources, advocacy organizations, and increasingly AI-powered tools.
That means requests may come from physicians and patients who have never participated in clinical research or interacted extensively with a pharmaceutical company.
This is particularly important as Expanded Access moves beyond therapeutic areas traditionally associated with compassionate use. Rare diseases, genetic conditions, personalized medicines, and other serious or life-threatening diseases are expanding the range of physicians and patients engaging with these pathways.
For pharmaceutical companies, the implication is significant: Expanded Access can no longer always be addressed only when the first request arrives.
Start Planning Before the First Patient Request
Perhaps one of the clearest messages from the discussion was the importance of planning early.
Companies should begin asking questions about Expanded Access while an asset is still in development:
Could this therapy eventually generate compassionate use requests?
Under what circumstances would the company consider providing access?
What clinical evidence would be required?
Who would participate in the decision?
Is sufficient supply available?
Which countries might be included?
What happens if demand exceeds expectations?
And importantly: what is the exit strategy?
Having these conversations early allows organizations to establish principles, governance, and operational processes before they are faced with an urgent request involving a patient with limited treatment options.
As Randlett emphasized during the discussion, early planning can help organizations make strategic decisions before they are faced with the emotional pressure of an individual patient request.
Supply Planning Can Make or Break an Expanded Access Strategy
Supply is one of the most complex aspects of Expanded Access—and one of the areas where early planning can have the greatest impact.
A company may want to provide a therapy, but that decision must be supported by the ability to provide treatment not only initially, but potentially over an extended period.
Bhuta described the importance of understanding supply requirements throughout an asset's lifecycle, particularly as companies move closer to commercialization.
Expanded Access demand can sometimes compete with clinical development and commercial launch requirements. That makes collaboration between clinical supply, commercial supply, regulatory, medical, and access teams essential.
Randlett also highlighted several ways companies can create greater flexibility earlier in development, including maintaining appropriate inventory from clinical programs and considering unlabeled or “bright stock” that can potentially be directed based on emerging needs.
The underlying lesson is simple: planning for Expanded Access supply should begin long before demand becomes urgent.
Post-Trial Access Requires an Exit Strategy
Expanded Access also intersects increasingly with another important question: what happens to patients who are benefiting from treatment when a clinical trial ends?
Post-trial access can provide a pathway for continued treatment in certain circumstances, but the requirements vary significantly by country.
The same challenge arises when a therapy receives approval but is not yet commercially available—or when regulatory approval occurs before reimbursement.
Companies therefore need to consider not just how a patient enters an access program, but how and when that patient can transition out of it.
Depending on the market, patients may transition to commercial supply once treatment becomes available. In other situations, companies may have longer-term obligations to continue supplying therapy.
That creates operational, financial, regulatory, and ethical considerations that need to be incorporated into program design from the beginning.
Global Programs Require Local Flexibility
Scaling Expanded Access globally introduces another layer of complexity: there is no single global process.
Requirements can vary considerably by country, including:
Regulatory submissions
Import/export requirements
Labeling
Safety reporting
Data collection
Treatment continuation
Transition to commercial supply
These differences become particularly challenging when companies expand programs into markets where they may have less operational experience.
Both speakers emphasized the value of direct engagement with local regulators when requirements are unclear. Rather than relying solely on assumptions or interpretations, companies may need to work directly with health authorities to understand what is required for a specific patient or program.
Operational processes must therefore balance global consistency with country-level flexibility.
Expanded Access Is Treatment—But Data Is Becoming More Important
Another important theme was the evolving role of data.
Expanded Access is fundamentally intended to provide treatment rather than conduct research. That distinction should remain central to program design.
At the same time, interest is growing in understanding whether information generated through Expanded Access can contribute to real-world evidence.
This may be especially relevant in rare diseases and highly specialized patient populations, where traditional clinical trial populations can be small.
Some countries are also beginning to encourage or require certain types of data collection.
The challenge for companies is determining what information is truly valuable while avoiding unnecessary burden on physicians and patients.
As Randlett noted, organizations should be thoughtful about why they are collecting information and what they intend to do with it. If data is collected, there should be a clear purpose—whether supporting safety monitoring, informing clinical development, contributing to regulatory submissions, or improving understanding of patient experience.
Technology Can Help Manage Growing Complexity
As Expanded Access programs grow, spreadsheets, email, documents, and disconnected workflows can become increasingly difficult to manage.
A single request may involve patient information, physician documentation, regulatory submissions, legal agreements, safety reporting, supply coordination, approvals, and ongoing resupply.
And in urgent cases, these activities may need to happen extremely quickly.
Technology can help create a centralized operational layer across these processes.
Digital workflows can help teams track requirements, identify missing information, coordinate stakeholders, manage documentation, monitor patient and supply activity, and maintain visibility across countries and programs.
The goal isn't simply automation.
It's helping organizations move quickly without sacrificing consistency, oversight, or compliance.
As the discussion highlighted, technology can function almost like a project manager for Expanded Access—helping ensure that required steps are completed before treatment is shipped while reducing the risk that urgency leads to missed documentation or unnecessary rework.
AI Could Further Accelerate Expanded Access Operations
Artificial intelligence may take this transformation even further.
During the webinar, the panel discussed several potential and emerging applications for AI, including:
Reviewing intake forms for completeness
Supporting preliminary workflow decisions
Helping interpret complex requirements
Forecasting potential patient demand
Managing documentation
Supporting program coordination
Redacting sensitive patient information
The opportunity is particularly compelling because speed matters in Expanded Access.
When a patient has exhausted available treatment options, reducing unnecessary administrative delays can have real consequences.
At MedaSystems, we see significant potential for AI and automation to support these workflows while keeping appropriate human oversight at the center of important decisions.
The objective isn't to remove people from the process. It is to give teams better tools to manage complexity, reduce administrative burden, and respond more efficiently.
Collaboration Is Becoming Essential
Perhaps the most important takeaway from the conversation was that successful Expanded Access programs cannot operate in isolation.
They increasingly require coordination among:
Medical Affairs
Regulatory Affairs
Clinical Operations
Clinical Supply
Commercial Supply
Quality
Legal
Safety
Market Access
Patient Advocacy
External partners
And collaboration increasingly extends beyond individual companies.
The panel highlighted industry organizations and forums where professionals share experiences, regulatory knowledge, and operational lessons.
As Randlett observed, Expanded Access is unusual in that companies often have a strong incentive to share what they have learned. When organizations discover a better way to navigate a regulatory or operational challenge, sharing that knowledge can ultimately help another patient receive treatment.
Preparing for the Next Generation of Expanded Access
The therapies themselves are also becoming more complex.
Expanded Access is moving beyond relatively straightforward oral and injectable products toward personalized medicines, cell and gene therapies, nuclear medicine, and other highly specialized treatments.
That evolution raises the stakes for logistics, supply planning, regulatory coordination, data management, and operational execution.
The organizations best prepared for this environment will be those that begin planning early, establish clear governance, connect stakeholders across functions, understand country-level requirements, and build infrastructure capable of adapting as programs grow.
Technology will increasingly be part of that infrastructure.
MedaSystems provides a centralized platform designed to help life sciences organizations manage Expanded Access, Post-Trial Access, and other complex medical programs—from intake and review through approvals, supply, resupply, communications, documentation, and reporting.
As Expanded Access becomes more global, more visible, and more complex, the opportunity is to build systems and processes that make programs easier to operate for pharmaceutical teams and easier to navigate for healthcare providers.
Ultimately, that supports the goal at the center of Expanded Access: helping appropriate patients access potentially important therapies when other treatment options are no longer available.
