How the Landscape of Expanded Access is Evolving: Insights from Industry Leaders
The evolving landscape of expanded access programs—often referred to as compassionate use—is reshaping how life sciences organizations approach patient engagement and clinical development. In our latest webinar, MedaSystems co-founder Matt Rosen sat down with Stephanie Petrone, Principal at Navigate Clinical, to explore the operational, ethical, and strategic realities of running effective expanded access programs.
A Growing Imperative for Proactive Planning
Stephanie began by sharing her first experience with compassionate use—a moment that arrived unexpectedly, as many do. “One afternoon, I got a phone call that said, We need to provide compassionate use for a patient. Go get it done.” That reactive “fire drill” scenario, she explained, remains common in the industry.
Today, however, leading organizations are taking a more proactive approach, integrating expanded access planning into broader clinical and regulatory strategies. “It’s not just a regulatory question,” Matt added. “It’s also a moral and ethical one.” Stephanie emphasized the importance of thoughtful program design, noting that companies must consider whether they have the supply, infrastructure, and medical rationale to responsibly support access.
The Changing Regulatory Environment
One key takeaway: regulations governing compassionate use are not static. While clinical research frameworks tend to be consistent globally, compassionate use varies significantly by country—and changes frequently. France and Brazil, for instance, have recently updated their guidelines, highlighting the need for ongoing regulatory intelligence.
Stephanie’s advice? “Stay up to date. You can’t assume what was true a year ago still applies today.”
Expanding Access Across the Globe
Beyond Europe and North America, there’s growing momentum to extend compassionate use programs to low and middle-income countries. Stephanie cited examples of companies that go to extraordinary lengths—sometimes delivering treatments to war-torn regions like Ukraine—to ensure access for patients in need.
Still, such efforts raise logistical and ethical challenges. Companies must weigh the availability of drug supply, medical oversight capabilities, and fair access practices when expanding globally. “It’s about equity,” Stephanie explained. “Every patient deserves a chance—but companies have to be realistic about what they can support.”
Three Pillars of a Successful Program
Stephanie outlined three foundational pillars for establishing and managing an expanded access program:
Strategy – Define your patient population, geographic scope, and exit plan. Align the program with your clinical development strategy and ensure leadership consensus.
Operations – Treat it like a clinical program. Forecast patient demand, manage documentation, and use technology—not spreadsheets—to ensure traceability and compliance.
Drug Supply – Plan for scenarios where demand may exceed availability. Ethical decision-making in such moments must be guided by clear internal policy.
She emphasized that internal alignment is critical: “The best programs are those where everyone—from the CEO to the clinical team—is on the same page.”
The Human Side of Expanded Access
What keeps professionals like Stephanie committed to this work? “It’s the patients,” she said. “You’re closer to them here than in almost any other role in pharma.” The expanded access community, she added, is highly collaborative—more collegial than competitive—because everyone shares the same mission: helping patients who have no other options.
Technology as a Catalyst for Change
Technology plays a growing role in managing complexity. Stephanie and Matt highlighted that platforms like MedaSystems’ solution help organizations streamline documentation, track patient outcomes, and stay compliant across regions—all critical as programs scale globally.
As Stephanie put it, “It takes a village—and the right systems—to do this well.”
Final Takeaways
Plan early: Don’t wait for the first patient request to start designing your program.
Stay current: Regulatory landscapes evolve—track changes continuously.
Invest in infrastructure: Robust technology ensures data integrity and scalability.
Keep the mission front and center: Every request represents a patient in need.
Expanded access isn’t just a regulatory formality—it’s a bridge of hope between innovation and those waiting for it.
