FDA Issues New Guidance on Using Real-World Evidence in Medical Device Decision-Making

On Thursday, August 31, the Food and Drug Administration (FDA) issued guidance to clarify how the agency evaluates real-world data (RWD) to determine whether it is sufficient for generating the types of real-world evidence (RWE) that can be used in FDA regulatory decision-making for medical devices. The new guidance is applicable to all devices, including software that meets the definition of a device.  Overall, the guidance describes the circumstances under which RWD may be used to support a variety of FDA decisions based on the existing evidentiary standards.


Moreover, this guidance highlights some of the potential uses of RWD, and describes the factors that FDA considers when evaluating whether specific RWD is of sufficient quality to inform or support a regulatory decision. It also clarifies when an Investigational Device Exemption (IDE) may be needed to prospectively collect and use RWD for purposes of determining the safety and effectiveness of a device.


It is important to note that FDA's guidance documents do not establish legally enforceable responsibilities. Guidance documents only describe the Agency’s current thinking on a topic and should be viewed only as recommendations. The use of the word “should” in Agency guidance means that something is suggested or recommended, but not required.


The guidance defines two important terms for the agency’s evaluation purposes.  To FDA, RWD are data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.  Examples of RWD include data derived from electronic health records (EHRs), 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.  RWE is the clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of RWD.


Under the right conditions, data derived from real-world sources can be used to support regulatory decisions. RWD and associated RWE may constitute valid scientific evidence depending on the characteristics of the data. 


While it does describe the factors that FDA considers when evaluating RWD or RWE, the guidance does not provide a specific set of pass/fail criteria or other scoring tools for making a determination about the suitability of RWD or RWE for a particular regulatory decision.