Keywords: real world evidence, BLA, post-marketing, safety
In December 2018, the FDA introduced a framework for evaluating the potential use of real-world data (RWD), that is, data derived from sources other than traditional clinical trials, in developing real-world evidence (RWE) to help support a new indication or satisfy post approval study requirements for already approved drugs or biologics. This framework may be seen as part of the ongoing work in the use of alternative data sources when traditional trials are deemed infeasible or impractical to implement. In CBER, such work has included the use of historical controls in therapeutic products for rare diseases or as comparison groups in safety event surveillance studies, establishment of safety registries for post marketing requirements, and use of electronic health records and other health insurance claim databases for characterizing long-term effectiveness or better detection of safety signals in a large population. In this talk, I will discuss several examples illustrating the use, including pros and cons, of these alternative data sources in the context of biologic product approvals.