Chapter 5 86 are granted by the EMA via the centralized procedure. Therefore, to obtain an overview of whether data from expanded access programs were used for submissions, we downloaded the Drugs@ FDA database and the EMA medicines overview on May 1st 2019. 17,18 For the FDA database, we downloaded the application documents (labels and reviews) associated with all approvals available in the database. Next to that, we retrieved documents from the drug approval packages sites. For the EMA, for each approved drug, we saved the scientific discussion, label and/or public assessment report that are listed in the database. Figure 1 gives a schematic overview of our method. Figure 1: Flowchart of automated candidate search. We searched through all FDA and EMA documentation for expanded-access related terms2. When these terms appear, the document is considered a candidate. For scanned files, optical character recognition (OCR) was used. 2 ‘compassionate use’, ‘expanded access’, ‘early access’, ‘pre-approval access’, ‘named-patient’ and ‘managed access’
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