Postlude 252 To this day, legislation surrounding expanded access and its research remains inconsistent among lawmakers. This creates confusion for patients, physicians, regulators, and industry, potentially impeding access rather than expediting it. We provide examples of inconsistent views across regulators, particularly in Europe, and we propose to implement a unified expanded access pathway to enhance regulatory harmonization in the European Union. Furthermore, we advocate for clear guidance on expanded access research by regulators such as the FDA and EMA, including reporting requirements to prevent publication bias, minimal data standards to ensure high data quality, and the inclusion of expanded access data within real-world evidence frameworks, to help clarify when it is appropriate to use these data and when it is not. Such improvements should ensure equitable patient access is tied with robust evidence generation, paving the way to expanded access for patients in need of treatment now, and for future patients to come.
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