Chapter 7 140 programs will result in publications, and the number of publications is potentially only a proxy for the total number of expanded access programs. Further research could focus on the bias (quality) of expanded access publications or could further explore differences between trial and expanded access publications. This concerns both patient demographics, i.e., are ‘expanded access patients’ really more ‘real-world’ than trial patients?, as well as clinical outcomes, i.e., are expanded access patients potentially worse off than trial patients? Conclusion The increasing interest in access to investigational medicine is reflected by a rise in the number of publications of expanded access programs from 2000-2019 and amplified by the COVID-19 pandemic through 2020 and 2021. The 1,231 publications identified in this review shed a novel light on the characteristics of patients, diseases, and research methods of expanded access programs. Harmonization of research legislation and guidance on the value of expanded access data within real-world data frameworks should ensure that patients in expanded access programs globally contribute efficiently to scientific evidence.
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