Use of real‐world evidence in regulatory decisions for rare diseases in the United States—Current status and future directions

Following the release of the framework for the Real‐World Evidence (RWE) Program, the US Food and Drug Administration (FDA) is actively evaluating and exploring ways to optimize the utility of real‐world data (RWD) and RWE to support regulatory decision making. For rare conditions, conducting traditional randomized clinical trials may not always be feasible, and RWD and RWE have played and will continue to play an important role. We use three...