Postlude 256 Future research Several areas remain that warrant further investigation. Our research primarily centered on examining the benefits of expanded access in terms of current clinical merit and future knowledge generation, but we did not explore patient preferences. It would be valuable to understand how patients perceive expanded access,22 the extent to which they are willing to trade-off current and future benefits, and their attitudes towards participating in expanded access research.23 The majority of our research has been conducted through quantitative methods, driven by our aim to provide objective and reproducible research to avoid any potential conflict of interest. For several studies, we therefore sought data that was labelled as ‘pivotal’, ‘supportive’ or ‘safety’, ‘efficacy’, or ‘cost’.15,23 Of note, these labels cannot adequately capture all nuances. By reducing the complex underlying data to these labels, we ourselves are guilty of reducing information dimensions. Although we have sometimes provided qualitative examples to accompany our quantitative findings, future systematic, independent qualitative research could provide useful insights into narrative interpretation of expanded access data. Lastly, our statistical research aimed provide a first idea of developing statistical methods to particularly accommodate for the inclusion of expanded access data.There are ample opportunities for adjustments and comparisons to be made.24 As these methods aim to address differences in patient characteristics among trial and expanded access patients, it would be useful to quantify to what extent patients in expanded access differ from trial patients. Although these patients are arguably more ‘real-world’ than trial patients, this topic has never been adequately researched. One potential approach could be to compare the patient descriptive summaries (e.g.,‘Table One’) in publications of expanded access programs with the descriptive summaries from clinical trials. Public-private partnerships: mutual benefits or concern for scientific integrity? As part of this thesis, the Erasmus MC, the Erasmus University Rotterdam, and myTomorrows engaged in a public-private partnership aimed at facilitating an exchange between private assets such as resources, expertise, and market knowledge of expanded access practices, and academic considerations, including public interest, societal needs, and research integrity.25 Conducting this thesis part-time enabled me to thoroughly investigate everyday issues that I encountered in my work on an academic level. Although such public-private collaborations are increasingly stimulated,26 it is worth mentioning that the scientific validity of such public-private collaborations has faced growing skepticism.27,28 In this context, I delve into whether our research indeed raises similar concerns.
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