Use of Real‐World Evidence in Neuroscience‐Related New Drug and Biologics License Applications for Novel Therapeutics

The US Food and Drug Administration (FDA) is evaluating the potential use of real-world evidence (RWE) in regulatory decision making. Some groups have evaluated the use of RWE in regulatory submissions in the United States and abroad, reporting that reliance on RWE to support new product approvals is relatively common. Confusion regarding the use of RWE in drug-approval decisions may arise, however, based on different application of the terms real-world data (RWD) and RWE. We evaluated RWE in new drug applications (NDAs) and biologics license applications (BLAs) from January 2019 to June 2021 for novel drugs and biologics approved by the FDA with indications related to psychiatry, neurology, pain, or sedation (here, termed neuroscience-related). We sought to determine whether the submissions included RWE and to describe the types of data and study designs used. Thirty neuroscience-related NDAs or BLAs were identified for novel drugs and biologics approved during the time-period of interest. Among these approvals, three applications (10%) were adjudicated as containing RWE, one of which included RWE as primary evidence of effectiveness. Our findings highlight how different operational definitions of the terms RWD and RWE can result in demonstrably different reporting of the use of RWE in regulatory decision making for neuroscience-related novel drugs and biologics. A better understanding of this topic, along with awareness of regulatory definitions of RWE, are important factors to promote accurate tracking and reporting of regulatory submissions involving RWE. These factors can also improve awareness among the stakeholder community regarding the role of RWD and RWE in regulatory decision making.