Chapter 7 126 ABSTRACT Background Although expanded access is an increasingly used pathway for patients to access investigational medicine, little is known on the magnitude and content of published scientific research collected via expanded access. Methods We performed a review of all peer-reviewed expanded access publications between January 1st 2000 and January 1st 2022. We analyzed the publications for drugs, diseases, disease area, patient numbers, time, geographical location, subject, and research methodology (singlecenter/multicenter; international/national; prospective/retrospective). We additionally analyzed endpoints reported in all COVID-19-related expanded access publications. Results We screened 3,810 articles and included 1,231, describing 523 drugs for 354 diseases for 507,481 patients. The number of publications significantly increased over timeppp (p < 0.001). Large geographical disparities existed as Europe and the Americas accounted for 87.4% of all publications, whereas Africa only accounted for 0.6%. Oncology and hematology accounted for 53% of all publications. Twenty-nine percent of all expanded access patients (N=197,187) reported on in 2020 and 2021 were treated in the context of COVID-19. Conclusion By summarizing characteristics of patients, diseases, and research methods described in all scientific literature published on expanded access, we provide a unique data set for future research. We show that published scientific research on expanded access has surged over the past decades, partly due to COVID-19. However, international collaboration and equity in geographic access remain an issue of concern. Lastly, we stress the need for harmonization of research legislation and guidance on the value of expanded access data within real-world data to improve equity in patient access and streamline future expanded access research.
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