Postlude 248 SUMMARY Expanded access allows patients and physicians to access unapproved medical treatments. Established in 1987 during the AIDS crisis, this regulated pathway is only an option for patients who are unable to benefit from approved drugs or from trial participation. Over the years, the prominence of expanded access programs has grown, in part due to the increasing availability of online information on the development of new treatments, and, more recently, due to the search for treatments for COVID-19. In my work, I participated in designing and implementing expanded access programs, with a particular emphasis on the efficient collection of data and the subsequent conversion into medical evidence. The motivation for the research in this thesis stems in part from the practical issues that I encountered in my day-to-day work. At first, I aspired to find a comprehensive overview on how to successfully implement expanded access programs with data collection based on research done by others – in the vain hope of repeating previous mistakes myself. Soon, I discovered that there was no existing research on this topic. Although anecdotal examples were described in the literature, the use of expanded access to generate data and the various purposes for which these data could be used, such as regulatory decisions, publications, or reimbursement appraisals, were unclear. Moreover, expanded access programs were initially designed to provide treatment rather than conduct research. This potential shift in the nature of these programs could lead to unexplored ethical concerns, such as whether one should use these programs to generate data, under what circumstances, and when. Practical concerns also arose, such as the legality of collecting these data and the value of such data. My colleagues at work and mentors at Erasmus University and Erasmus Medical Center encouraged me to conduct this research independently. As the current work is the resulting collaboration, I will transition from using ‘I’ to ‘we’ from this point onward. As befits writing a dissertation, our goal was to create a theoretical body of objective, reproducible research. But as theoretical findings have practical implications, we emphasized these throughout the thesis and discussed in depth two real-world examples of published expanded access programs to illustrate their practical applications in the opening pages of this book.
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