FDA Explains What is Real World Evidence and How It Can Be Used 
​​[Posted on: Thursday, September 7, 2017]

Real World Evidence (RWE) and Real World Data (RWD) are very important but distinct aspects of the clinical use information available for a drug. The 21st Century Cures Act mandates FDA to use RWE in support of the approval of a product and FDA’s opinion about the utility of such data in support of market approval is evolving rapidly. Last week two publications, one for medical devices and one for drugs highlighted the key elements of how and when RWD can be considered RWE in support of regulatory decisions. RWD is any data related to patients’ health and use of medical products collected from a variety of sources such as electronic health records, claims and billing data, data from product and disease registries, patient-generated data including in home use settings, and data gathered from other sources that can inform on health status, such as mobile devices. RWD is routine generated and collected during routine health delivery. When RWD is used to support the safety and benefits of a product, it becomes RWE. So, in most ways, RWE is a subset of RWD. A treasure trove of RWD is collected routinely and could yield very useful information about a product. FDA had always relied on RWD for monitoring safety of medical products via its adverse event reporting programs. RWD can also be collected in clinical trials, both interventional and observational. However, FDA does not consider most RWD collected in controlled settings such as clinical trials to be truly RWE. FDA defines RWE as data collected in truly real world setting. RWE should be based on holistic review of all available data to reduce bias, and provide a complete clinical use picture without strict inclusion or exclusion criteria for the data collected. The quality of the RWE will define what it can be used for. Establishing safety of a product is the easiest. Expanding indications for approved products is easier. But approval of a new product is only feasible when controlled clinical trials are not feasible such as very rare diseases, and when both the pathophysiology and natural history of the disease were well enough understood to lend confidence in establishing causality. RWE can also be very useful for generating hypotheses, as historical control, and as evidence for the clinical validity for a biomarker. For RWD to be usable as RWE, FDA expects sufficient details to enable addressing the specified question. Additional factors that could influence FDA’s acceptance are aspects such as data from representative real world populations, diversity of data, validated methods to analyze data, data relevant to the question being supported, hypothesis for using the data based on established peer-reviewed evidence, and whether supplemental data sources are available and sufficient to provide any missing information or evidence required for an informed decision. The quality of RWD is defined by how the data were collected and measures to assure that errors were minimized and that data quality and integrity was maintained. RWE is a critical tool for expanding indications or finding new uses for previously approved products. It should reduce the need to clinical trials for uses that can be proved from existing clinical experience.