United States: FDA Issues Draft Guidance On Use Of "Real-World Evidence" In Medical Device Decision-Making

Michael Gaba is a Partner and Jennifer Nowak is an Associate in Holland & Knight's Washington D.C. office


  • The U.S. Food and Drug Administration (FDA) issued draft guidance addressing how it may use data and evidence collected outside of the traditional clinical trial context to support medical device regulatory decision-making.
  • The draft guidance acknowledges that certain "real-world data" (RWD) and associated "real-world evidence" (RWE) could constitute valid scientific evidence that could be used in some FDA regulatory contexts.
  • While the guidance allows the medical device industry to harness additional sources of data, it is important to note that there may also be an increase in government surveillance in the marketplace.

The U.S. Food and Drug Administration (FDA) issued on July 27, 2016, a draft guidance document addressing how it may use data and evidence collected outside of the traditional clinical trial context to support medical device regulatory decision-making. The draft guidance acknowledges that certain "real-world data" (RWD) and associated "real-world evidence" (RWE) could constitute valid scientific evidence that could be used in some FDA regulatory contexts, such as support for the expansion of indications of an approved/cleared device and post-market surveillance.

While the draft guidance presents an opportunity for the medical device industry to harness additional sources of data to demonstrate safety and effectiveness and to conduct post-approval studies, it is important to note the potential for an increase in government surveillance in the marketplace. This is especially significant when considering the new unique device identification (UDI) requirements that mandate all medical devices marketed in the U.S. (with some exceptions)1 bear a unique identifier on the device or its labeling and packaging by 2020,2 allowing for more facile tracing and tracking of specific devices.3

What Kinds of RWD and RWE Does the FDA Contemplate Using?

The FDA defines RWD as data collected from sources outside of traditional clinical trials, which could include the following:

  • large simple trials
  • pragmatic clinical trials
  • prospective observational or registry studies
  • retrospective database studies
  • case reports
  • administrative and healthcare claims
  • electronic health records
  • data obtained as part of a public health investigation or routine public health surveillance
  • registries (e.g., device, procedural or disease registries)

The source of RWD may be paper or electronic inpatient and outpatient medical records and case histories, diagnostic laboratory and imaging data, patient-reported outcome measures or medical device performance data existing within a device, such as self-diagnostics, error codes and patient diagnoses/treatments delivered, including its UDI.

The FDA defines RWE as the evidence derived from the aggregation and analysis of RWD elements.

The FDA stresses that it is not changing the evidentiary standards applicable to its regulatory decision-making. Rather, the FDA believes that there are circumstances under which RWD may be used to meet existing evidentiary standards, and this draft guidance is meant to outline the criteria for determining whether particular RWD is appropriate for generating valid scientific evidence. The FDA recognizes the wealth of data collected during patient care and management, especially with the rapidly growing use of electronic data in most healthcare settings, and is working on ways to use the captured data in fulfilling its mission. The FDA has been working for several years on its plans for a national evaluation system, the goal of which is to leverage RWD to inform its decision-making.

Further, RWE may only be used when the FDA finds sufficient underlying relevance and reliability, including collection and analysis of RWD that takes into consideration the need to limit bias and is designed to assess causal relationships. In many cases, this may require a prospective analysis plan in order to ensure the proper controls are in place. The FDA states that it will consider the use of RWE to support regulatory decision-making for medical devices when it concludes that "the clinical data contained within RWD source(s) used to generate the RWE are of sufficient quality to provide confidence in the analyses necessary to inform or support the regulatory decision throughout the total product life cycle."

The draft guidance provides detail on how the FDA will assess the regulatory relevance of RWD (e.g., by assessing the representativeness of the device use in a real-world population as captured in the data source and the generalizability of the data to the relevant population being evaluated). It also provides information on how the FDA will assess the reliability of RWD, taking into consideration the methods of data accrual, the adequacy of the data to inform the question at hand, and the data quality and integrity.

How Might Valid RWE Be Used By the FDA?

The FDA lists several examples of how RWE might be used at different points in the total product life cycle to inform FDA understanding of medical device performance, including:

  • as evidence in support of expanding a device's labeling to include new indications or to update labeling with new safety and/or effectiveness information
  • for public health surveillance efforts
  • to conduct post-approval studies required as a condition of device approval

The draft guidance provides opportunities for the industry to use nontraditional forms of data to inform decisions in cases in which traditional clinical trials may be impractical or challenging, as well as cases in which data can be gathered without adding additional costs to the development of a device. However, it also provides the FDA with another means of market surveillance. Further, industry members will need to be aware of other concerns, such as patient privacy and cybersecurity, as they seek to collect and share medical device data for regulatory compliance purposes.

Comments on the draft guidance must be submitted by Oct. 25, 2016.

Before submitting comments, interested parties should seek out qualified counsel for guidance on the potential advantages and disadvantages presented by the draft guidance, including potential ancillary concerns, such as data/patient privacy and cybersecurity considerations.


1 The UDI rules allow for certain exceptions. For example, all Class I devices that are exempt from current Good Manufacturing Practices are also exempt from UDI requirements.

2 The rule is being phased in through 2020. Currently, most Class III devices are required to display a UDI on labeling and packaging, and most life-supporting or life-sustaining devices intended to be used more than once and reprocessed before each use are required to bear a permanent marking on the device itself. Compliance dates for other devices can be found here.

3 The FDA also issued a draft guidance on the form and content of the UDI.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

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