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Results from Expanded Access Programs: A Review of Academic Literature
Tobias B Polak1,2,3,4, David G J Cucchi5,6, Jasmin Schelhaas7
1Real-World Data Department, myTomorrows, Amsterdam, The Netherlands. t.polak@erasmusmc.nl.
Published scientific research on expanded access has surged, driven partly by COVID-19. However, geographic disparities and a need for harmonized research guidance persist, impacting equitable patient access to investigational medicines.
Area of Science:
- Medical research
- Pharmaceutical sciences
- Clinical research
Background:
- Expanded access is a growing pathway for investigational medicine.
- Limited understanding exists regarding the scope and content of research published via expanded access.
- This study addresses the knowledge gap on expanded access research publications.
Conclusions:
- Published expanded access research has grown substantially, partly due to COVID-19.
- International collaboration and equitable geographic access remain significant concerns.
- Harmonization of research legislation and guidance on real-world data value is crucial for improving equity and streamlining future research.
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