FDA is issuing this guidance to clarify how we evaluate real-world data to determine whether it is sufficient for generating the types of real-world evidence that can be used in FDA regulatory decision-making for medical devices. This guidance is applicable to all devices, as that term is defined under section 201(h) of the Federal Food, Drug, and Cosmetic Act (the FD&C Act), including software that meets the definition of a device.
Real-World Data (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 homeuse settings, and data gathered from other sources that can inform on health status, such as mobile devices. RWD sources (e.g., registries, collections of EHRs, and administrative and healthcare claims databases) can be used as data collection and analysis infrastructure to support many types of trial designs, including, but not limited to, randomized trials, such as large simple trials, pragmatic clinical trials, and observational studies (prospective and/or retrospective ).
Real-World Evidence (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. This guidance should not be construed to alter, or change in any way, the existing evidentiary standards applicable to FDA’s regulatory decisionmaking; rather, it describes the circumstances under which RWD may be used to support a
variety of FDA decisions based on the existing evidentiary standards. While FDA encourages the use of relevant and reliable RWD, this guidance neither mandates its use nor restricts other means of providing evidence to support regulatory decision-making. 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.
This document does not address the use of non-clinical data, adverse event reports, secondary use of clinical trial data (e.g., post hoc analyses), or systematic literature reviews. Nor does it address study design/conduct or analytical methodologies. While it does describe the factors that FDA considers when evaluating RWD or RWE, it 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.
This guidance does not affect any federal, state or local laws or regulations, or foreign laws or regulations that may be applicable to the use or collection of RWD, or that provide protections for human subjects (including informed consent requirements) or patient privacy. This guidance should be used to complement, but not supersede, other device-specific and good clinical practice guidance documents.