Data extraction
The resulting list of the included articles/trials was discussed by JBM and LH to ensure the accuracy of the final decision. XWH performed the manual assessment, and, in case of doubt, the assessment was discussed with JBM. A Microsoft Excel (Microsoft Corporation, Redmond, WA, USA) database was used to collect the relevant information on the included trials. We collected all available trial characteristics (Table 1), incorporating the type of file, the status of authorization, the year of authorization, the name of medicine, the orphan designation, the pivotal status, the accelerated assessment status, AM status, CMA, whether it required PASS, whether it required PAES, NAS status, the types of adaptive designs, the therapeutic area, the system organ class (SOC), the antitumor status, the study progression, the study duration, and the sample size from the respective approval. The keyword served as a pivotal cue to locating targeting approvals to distinguish between studies scheduling adaptive designs in confirmatory trials from eligible approvals.
Table1. Definitons of trial characteristics