Results from expanded access programs: a review of academic literature 137 7♦ DISCUSSION In this paper, we have mapped the landscape of expanded access publications from January 1st 2000 to January 1st 2022. To the best of our knowledge, this is the first literature review of expanded access publications to assess drugs, diseases, patient numbers, and research methods. We have identified 1,632 original investigations of expanded access, of which 1,231 focus on pharmacological therapeutics, and the number of publications increases significantly over time. The increase in publications reflects a general increase in attention for expanded access, as reported by regulators, industry, and through other scholarship. Our work provides the first annotated data set that yields insights into how many patients contributed to the peer-reviewed scientific literature through expanded access programs, across diseases, across geographies, and across drugs. The geographic distribution of expanded access publications highlights the disparity of availability of investigational medicine. High-income countries produce more publications compared with low-income countries, which may be partly explained by excluding non-English literature, but may also be attributed to manufacturer and scientific willingness to provide expanded access and facilitate subsequent research. Our findings reflect the limited access to medicine in developing countries in general, but to investigational medicine in particular – an issue worth exploring in future research. The differences between countries within the European Union may be due to differences in regulatory preferences. Italy, with the highest number of publications per capita, is more liberal in allowing data collection compared with countries such as Sweden and Finland.65 The variance in allowing expanded access programs to generate evidence among European regulators has created a maze of national pathways for manufacturers to navigate.66 Such complexity may provoke reluctance from drug manufacturers to provide expanded access in the first place, which may impede rather than facilitate equity in patient access. The largest share of expanded access research is devoted to oncology and (malignant) hematology, accounting for 53.5% publications.This is driven in part by the large unmet medical need of cancer patients, as well as the abundance of trials in these areas. Furthermore, regulators offer specific guidance for expanded access to oncology (for example, through the FDA’s Project Facilitate),82 educating oncologists and expediting access to anti-cancer drugs. Our findings seem to support the position that, indeed, expanded access programs can be used to collect data that can further the knowledge of an investigational medicine. The stance of some regulators (e.g., Sweden, Finland, Canada) that data collection within an expanded access
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