Tobias Polak

Chapter 5 88 Figure 2: Flowchart of review process. We manually reviewed and deduplicated 187 approvals related to the candidate documents. All approvals that used expanded access (n=39) to support clinical efficacy were analyzed. For 13 approvals, the evidence from expanded access was the pivotal source of evidence. Finally, we further inspected the group of approvals that included RWD from expanded access programs in terms of disease areas, orphan designation, timing of marketing approval and the number of patients in the expanded access programs relative (nEA) to the total number of patients (N) in the trials. We used descriptive statistics to describe our data set. To compare our subset of approvals to regular approvals with respect to the number of orphan designations, we used chisquare tests with a two-sided significance level of 0.05. To detect a trend over time in the yearly numbers of approvals, we used a Spearman rank correlation test with a two-sided significance level of 0.05.

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