Contribution of Real-World Evidence in European Medicines Agency's Regulatory Decision Making.

For ( activated ) eptacog alfa , RWD were used to provide efficacy data and natural history data derived from the Glanzmann's Thrombasthenia Registry, whereby the added value of the registry was acknowledged as follows: “An advantage of this registry design is that data can be obtained over several years from a quite significant number of patients with a rare disease who are treated according to practice which is current at the treatment centres.” 24 However, it was recognized that the observational nature of the study is not without limitations related to the data source itself, e.g., risk of bias such as underreporting of negative experience (reporting bias) and physician's choice of treatment leading to individual differences, and to the study design, e.g., no comparable subgroups were predefined ( Table 5 ).

also in 5,705 other papers