RWE: The EMA's View

The European Medicines Agency believes that real-world evidence is important for making many decisions about a medicine. It helps support pharmacovigilance activities and the assessment of safety signals, and it can also help measure the impact of any regulatory measures undertaken, says the agency. Such evidence also enables a better understanding of a medicine's risk-benefit balance and its effectiveness as well as informs HTA and health care utilization decisions. These activities apply to a wide range of medicines, including authorized medicines, innovative medicines, orphan medicines and medicines included in Adaptive Pathways. The agency says it sees a wide range of RWE collected from numerous sources, including e-health records, registries, hospital records and insurance data. Meanwhile, data from biobank, genomic and digital phenotyping information are increasingly being integrated into real-world evidence data sets.

Nevertheless, regulators face a number of difficulties that impact their ability to access and analyze real-world data. According to the...