Figure 1

Schematic overview of potential and authorised treatment populations. The horizontal pipeline indicates the orphan drug designations (ODDs) for either adults only (upper line) or with a potential paediatric indication (lower line) over time (2000 – 2012) and the arrows represent those that obtained MA. The thick vertical line represents the year 2007. Arrows to the right of the thick line are all ODDs that obtained MA after 2007 (middle section: designation date before 2007, rightmost: designation date after 2007). Arrows with broken outline represent ODs that are undergoing further research in the paediatric population (i.e. with an agreed PIP after having received MA, while solid arrows are not undergoing further research in children).