P214: Global Trends in Expanded Access: Real-World Operational Insights (2022–2025)
Poster Presenter
Melissa Bechtel
Director, Expanded Access Strategy and Process Standards
Pfizer United States
Objectives
To characterize global Expanded Access (EA) request patterns and assess operational processing timelines across therapeutic areas and countries using real-world data from 2022–2025.
Method
A comprehensive review of 1,326 initial and 4,924 resupply Expanded Access requests (2022–2025) systematically evaluated operational steps, therapeutic areas, annual volumes, and country-specific trends to identify workflow challenges and optimization strategies.
Results
EA requests were primarily concentrated in Oncology (~68% of initial; 72% of resupply requests) and Rare Disease, which together accounted for approximately 79% of initial and 92% of resupply volume. These areas demonstrated the highest resupply-to-initial ratios—6.84-fold in Rare Disease and 3.93-fold in Oncology—reflecting potentially continued treatment needs and sustained demand for ongoing access.
Geographically, activity showed moderate concentration. Turkey and France led initial request volumes, while Korea, Turkey, and Poland accounted for most resupplies. Although the top five countries represented 45–49% of total volume, a substantial long tail of lower-volume countries highlighted the need for unmet need across many countries and need for country-specific logistical and documentation processes.
Operational timelines found differences in initial and resupply timelines. Mean end-to-end processing time for initial requests is 45.6 days, while for resupply it is 18.3. For initial requests, the longest stage was time period awaiting regulatory approval (21.6 days; ~47% of total). For resupply, the longest time period obtaining all required documents to re-supply shipment (6.88 days; ~38%). The largest improvement was observed in regulatory approval (-17.78 days; -82%), while Shipment ? final Delivery showed a slight increase (+0.45 days; +13%).
Therapeutic-area-specific improvements were consistent when comparing initial versus resupply requests: end-to-end timelines decreased from 37.68 ? 16.26 days in Oncology (-57%), 53.28 ? 22.19 days in Rare Disease (-58%), and 64.76 ? 20.21 days in Inflammation & Immunology (-69%). More efficient resupply Expanded Access countries included France (5.12 days), Sweden (5.28), Poland (5.50), the United Kingdom (7.14), and Spain (7.55), whereas longer timelines were observed in India, Iraq, Malaysia, Nepal, and Turkey.
Conclusion
EA request volumes are predominantly concentrated in Oncology and Rare Disease, indicating sustained patient demand and recurring shipments. This concentration calls for robust release planning, supply readiness, and effective shipment coordination, especially in high-volume markets. However, the wide geographic dispersion and the significant number of lower-volume countries emphasize the need for globally consistent processes, comprehensive documentation, and adaptable, country-specific logistics solutions to ensure timely and reliable access.
The regulatory approval interval is the main driver of end-to-end timelines for initial EA requests, whereas obtaining necessary documents to resupply shipments is most critical for resupply requests. Resupply processing is notably faster, with an overall reduction of 27.35 days, largely attributed to an 82% decrease in the regulatory approval interval, thanks to the reuse or updating of previously approved documentation.
Opportunities to reduce initial timelines hinge on a clear understanding of regulatory requirements and responsibilities, often outside the company’s control. In contrast, resupply improvements can focus on company-controlled areas like release planning, batch readiness, and logistics coordination. Country-level variability suggests that high-performing markets benefit from higher volumes and familiarity with EA requirements, while slower markets may need clearer regulatory guidance or operational enhancements.
These real-world insights reveal meaningful opportunities to streamline EA operations and enhance global consistency, ultimately facilitating faster and more reliable access to critical therapies for patients with unmet needs worldwide.
