What the Industry Must Tackle Next
As demand for timely, equitable access to investigational therapies grows worldwide, Expanded Access Programs (EAPs) are under more pressure than ever. The landscape in 2026 is defined by heightened regulatory expectations, complex operational demands, global disparities in access, and the need to collect meaningful real‑world data, without slowing down treatment delivery. These challenges form the backbone of the 6th Operationalize: Expanded Access Programs Summit, where more than 120 leaders across Access, Medical Affairs, Clinical Operations, and Supply Chain will gather to collaboratively move the field forward.
Below, we explore the key cross‑industry challenges and how they align with the summit’s core content pillars and agenda themes.
1. Navigating Global Regulatory Fragmentation & Evolving Guidance
Regulatory expectations for EAPs have never been more dynamic. New FDA recommendations, changing EMA frameworks, national systems like France’s ATU, and regional variability in emerging markets demand precision and adaptability.
Key industry challenges:
- Interpreting new regulatory guidance and understanding implications for approval timelines and risk‑benefit decisions.
- Aligning global program strategies when each market has distinct rules, ethics requirements, and charging allowances.
- Managing post‑trial access obligations and long‑term treatment continuity.
These issues underpin several summit sessions, including “Expanded Access in 2026: New Guidance, Evolving Expectations” and global regulatory comparison panels, offering attendees clarity and actionable frameworks.
2. Operationalizing EAPs in Challenging Regional Landscapes
EAP delivery is uniquely complex in regions with limited infrastructure, variable healthcare systems, or complex political and payer dynamics. Low‑ and middle‑income countries (LMICs) and regions like Central America and the Caribbean present distinct barriers.
Core pain points include:
- Unpredictable supply chains, import/export challenges, and labeling logistics.
- Gaps in local regulatory clarity and inconsistent payer support.
- Limited digital systems for request management and data collection.
- Difficulties maintaining trust and transparent communication with local stakeholders.
These realities are explored through talks such as “Overcoming Access Barriers in LMICs” and “Operationalizing EAPs in Challenging Regional Landscapes,” providing real‑world strategies for expanding access in underserved markets.
3. Collecting Real‑World Data Without Compromising Access
As regulators increasingly recognize the value of real‑world data (RWD), companies face rising expectations to gather meaningful evidence, yet EAPs are not research studies, and patient care remains the priority.
Key challenges include:
- Determining the minimum viable dataset that preserves ethics and feasibility.
- Leveraging EMRs, retrospective chart reviews, or companion studies without overwhelming physicians.
- Overcoming site engagement barriers where no site contracts exist.
- Ensuring RWD is “fit for purpose” for future filings, safety monitoring, or post‑marketing commitments.
The summit directly addresses these challenges across RWD roundtables, fireside discussions, and case studies from companies like Ferring and Sanofi.
4. Integrating AI & Digital Platforms Without Increasing Risk
Digital transformation is reshaping pre‑approval access. AI‑driven systems and digital portals can streamline request management, forecast supply needs, and offer powerful compliance oversight. But they also introduce new considerations.
Industry concerns include:
- Ensuring AI tools meet regulatory expectations and avoid introducing compliance risk.
- Maintaining the human component of patient and physician interactions.
- Integrating digital systems into established cross‑functional processes.
- Establishing interoperable data flows across Access, Supply, Safety, and Medical Affairs.
Sessions such as “Leveraging AI to Enhance Operational Efficiency in EAPs” explore how the industry can adopt AI responsibly while maintaining patient‑centricity.
5. Supply Chain Complexity & the Challenge of Unpredictable Demand
Few areas bring more operational strain than EAP supply chain management. Unlike clinical trials, EAPs require rapid, individualized distribution, often across multiple countries with differing regulatory & packaging requirements.
Key challenges:
- Forecasting supply with limited visibility into patient numbers.
- Ensuring cold‑chain integrity in remote or LMIC regions.
- Managing multi‑vendor labeling, import/export approvals, and emergency shipments.
- Balancing supply availability with manufacturing constraints and launch readiness.
Insights will be offered by leaders from Takeda, Eli Lilly, and more in sessions dedicated to supply chain risks and operational excellence.
6. Ethical & Patient-Centric Decision-Making Under Resource Constraints
When demand outpaces supply, ethical considerations become acute. Questions arise about patient selection, fairness, transparency, and the role of chargeable EAPs.
The industry continues to grapple with:
- How to design ethically sound paid-for models that protect patient trust.
- Balancing early access with clinical trial integrity.
- Communicating difficult decisions around eligibility, program pauses, or closures.
- Addressing advocacy pressure, especially in rare and ultra‑rare diseases.
The summit’s brand‑new Rapid Fire Solutions: Ethical & Compliant Chargeable Access and rare disease deep‑dive sessions provide much‑needed clarity.
Shaping the Future of Expanded Access Together
In 2026, the pressure to deliver faster, fairer, globally consistent access to investigational therapies is higher than ever. Across every session, workshop, and panel, the 6th Operationalize: Expanded Access Programs Summit provides practical insights and industry‑tested solutions to the challenges that matter most.
For biopharma teams navigating this evolving landscape, the summit remains the premier forum to learn, collaborate, and operationalize programs that place patients firmly at the center.