Lessons From the Field: What Expanded Access Teaches Us About Patients, Data, and Drug Development
Expanded access programs—sometimes called compassionate use—often enter a company’s world unexpectedly. A single request from a physician or patient can quickly become a defining moment that tests a company’s preparedness, values, and systems.
In a recent MedaSystems webinar, Lessons From the Field: A Conversation on Expanded Access, Matt Rosen, Co-Founder and EVP of MedaSystems, sat down with Dr. John Langlands, MedaSystems advisor and veteran drug development leader, to reflect on almost a decade of real-world experience running expanded access programs in oncology.
What emerged from the conversation was more than a retrospective. It was a set of hard-earned lessons about planning ahead, supporting patients and physicians, and capturing insights that can meaningfully influence clinical strategy.
From Discovery to Expanded Access: A Career Built on Experience
Dr. Langlands brings over 30 years of experience in inflammatory diseases, psychiatry, and oncology. His career has spanned academia, small biopharma startups, consulting, and global drug development leadership.
Dr. Langland’s first hands-on experience with expanded access came in 2017 while working with a small, publicly traded biotech focused on glioblastoma (GBM). The company was conducting clinical trials in the U.S. and China when an unexpected request arrived—from a Canadian physician seeking access for a patient who was ineligible for any trial.
Like many companies at the time, they understood the concept of expanded access in theory, but not in practice.
“It was there, but we didn’t think it was going to be part of our life—until suddenly it was.”
The First Request Is Always a Fire Drill
The initial expanded access request forced the team to quickly answer difficult questions:
How will this affect ongoing clinical trials?
How will safety data be interpreted?
Do we have sufficient drug supply?
Can we support this patient without compromising development timelines?
Despite the urgency, the process proved more manageable than expected. Regulatory pathways were clearly outlined, agency responsiveness was strong, and collaboration with Health Canada moved quickly.
Still, those early requests were disruptive.
“The first request was a fire drill. The second was still a fire drill—but less so.”
The turning point came when the team realized expanded access wasn’t a one-off event. Requests would continue, and processes needed to evolve accordingly.
Planning for Supply, Safety, and Scale
One of the most immediate challenges was drug supply. While the company had sufficient inventory, it was not unlimited, and treatment plans often required up to 12 cycles per patient.
The solution was a resupply-based model:
Ship the drug in smaller increments
Resupply only after confirming patient progress
Track cycles closely to avoid waste
This approach not only preserved supply but also created visibility into patient outcomes—without overburdening physicians or sites.
Importantly, regulators made clear that safety data from expanded access is evaluated differently than clinical trial data, easing early concerns about “tainting” trial results.
Who Requests Expanded Access—and When
Over the course of six years, requests followed no strict pattern. There could be weeks of silence, followed by multiple inquiries in a single day.
However, some trends emerged:
Increased outreach after scientific conferences or data releases
Seasonal spikes around major holidays, when families research options together
Requests not only from physicians, but also directly from patients and caregivers
Many patients demonstrated a deep understanding of both their disease and the underlying science.
“I was really quite astounded at how savvy patients are. They know how the drug works, not just its name.”
This reinforced the importance of having clear, empathetic, and consistent processes in place—before the first request ever arrives.
Expanded Access as a Source of Scientific Insight
One of the most powerful themes from the webinar was how expanded access can generate insights that extend far beyond treatment alone.
Over time, the company was able to analyze outcomes from patients who would never have qualified for clinical trials, including:
Patients with multiple recurrences
Patients with multifocal disease
Patients with other high-grade gliomas, such as anaplastic astrocytoma, ependymoma, and diffuse midline glioma
These insights were robust enough to support multiple scientific posters presented at major conferences, including:
Society for Neuro-Oncology (SNO)
AACR Annual Meeting
In several cases, expanded access data revealed safety signals, feasibility of combination therapy, and disease-specific responses that were not observable in controlled trials.
A Single Case That Changed Clinical Direction
Perhaps the most striking example was a 21-year-old patient with diffuse midline glioma—an aggressive disease with limited treatment options.
Through expanded access, the patient received 18 cycles of therapy, including combination treatment and radiation, well beyond what had been observed in trials. The safety profile and durability of response were unexpected and compelling.
That single case, combined with supporting preclinical data, ultimately helped justify a new clinical development program in a new indication.
“It would not have happened without the expanded access program—or it would have been far harder to justify.”
Why Systems Matter
A recurring lesson was the importance of how data is captured, even when treatment—not data generation—is the primary goal.
Initially, information was conveyed through free-text emails and informal updates. Over time, structured data collection—modeled after clinical trial principles—made a measurable difference:
Consistent fields instead of free text
Simple checkboxes and numeric entries
Clear tracking of safety, dosing, and treatment cycles
Using a validated, compliant system ensured that information was:
Timely
Reliable
Suitable for regulatory discussion and scientific dissemination
“The quality and usability of the data changed completely once the system was in place.”
The Takeaway: Plan Early, Learn Continuously
Expanded access is not something companies can opt out of simply by ignoring it. Requests will come—often at the most inconvenient time.
The lessons from the field are clear:
Plan early, even if you think you won’t need it
Treat patients and caregivers with empathy and consistency
Protect supply and operational focus with clear processes
Capture data thoughtfully—it may shape your future pipeline
At its best, expanded access serves patients in urgent need while advancing understanding in areas where traditional trials cannot reach.
And when supported by the right systems, it can become not just a responsibility—but a strategic asset.
