Tobias Polak

Chapter 8 172 EPILOGUE In this part, we have established that the utilization of data procured from expanded access is not purely anecdotal. Regulators, reimbursement agencies, and healthcare researchers routinely employ the data from expanded access programs. To see how the results of our first paper would have developed over time, we updated our paper on the regulatory the use of efficacy data by FDA/EMA on the 1st of May 2023, updating results throughout 2022 with the help of Jasmin Schelhaas. This can be seen in Figure 11. It is imperative to note that these figures have yet to undergo peer-review and have been presented solely in academic conferences. Nevertheless, we would like to show the updated statistics to the reader. Regulatory websites may not always be up to date, and hence information on 2022 may only come available in 2023. This delay could explain slight inconsistencies compared with the earlier figure, and also requires caution interpreting the numbers from 2022. Figure 11: Updated Bar Chart analysis of EMA and FDA approvals that (partly) rely on data from expanded access programs to establish the profile of efficacy. Our observations demonstrate that the previous trends have persisted, and regulators have increasingly been using expanded access data in decision-making. Despite the overwhelming enthusiasm surrounding this development, we caution against the mere collection of data without a clearly defined purpose, and a reliable tool to incorporate expanded access data into decision making. In the forthcoming chapter, we will develop such a statistical instrument specifically designed to accommodate the incorporation of expanded access data with data from randomized clinical trials.

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