Figure legends
Figure 1: Overview of selection process of scientific advices. All advice reports from the EMA from 2013 -2018 were collected and the advice reports on products containing human cells for which non-clinical data information was present in the reports were selected. ATMP: advanced therapy medicinal products.
Figure 2: Type of studies performed for the various product classes. In total 234 in vivostudies were performed for 86 products. The purpose of the studies is shown for the products that are categorized based on cell origin and genetic modification status that is depicted by the different colours.
Figure 3: Percentage of dedicated and combination studies. The number of in vivo animal studies is related to the total amount of products (86) addressing pharmacology (75), safety (64), biodistribution (49) and tumourigenicity (46).
Figure 4: Migration analysis in biodistribution studies. The migration towards target tissue only, or both to target and non-target tissues was recorded for the biodistribution studies and specified for product categories based on their cell origin and genetically modification status.
Figure 5: Analysis of the persistence of the cells in the biodistribution studies. The persistence of the cells in the animal was either evaluated (Yes), not evaluated (No) or planned (Planned) to be evaluated. For some products persistence of cells was not mentioned in the advice reports (Not mentioned).
Figure 6: Approach for estimation of the tumorigenic risk. The approach for evaluation of the tumourigenic risk by in vivo and/or in vitro studies or none of these (‘In vivo’, ‘In vitro’ , or both ‘in vivo & in vitro’, or ‘none’). For some products, the tumourigenic risk is not addressed in the scientific advice (‘Not mentioned’).
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