Life Science Compliance Update

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December 13, 2017

FDA Issues Guidance on Real-World Evidence for Medical Devices


Earlier this year, the United States Food and Drug Administration (FDA) finalized guidance on the use of real world evidence to support regulatory decision-making for medical devices. The final guidance follows a July 2016 draft guidance on the topic in which the FDA discussed potential uses of real world data and the various factors evaluated to determine whether that data can support a regulatory decision. The FDA believes that this guidance is a “cornerstone” of its strategic priority to build a national evaluation system for health technology.

The final guidance includes clarity as to what it means for companies moving forward, “While FDA encourages the use of relevant and reliable real-world data, this guidance neither mandates its use nor restricts other means of providing evidence to support regulatory decision-making.”

In the final guidance, FDA recognizes that “traditional” clinical trials may sometimes be impractical or overly challenging, and appropriate real world data can sometimes provide comparable information. While clinical trials are controlled and require monitoring and data auditing, they may be narrow in scope. Real world data may be able to generate information on a broader patient population, but studies must be carefully designed, regardless of whether the real world data has already been collected or will be collected in the future. Protocols and analysis plans for real world data “should address the same elements that a traditional clinical trial protocol and statistical analysis plan would cover.”

The final guidance also notes that due to the “rapidly advancing methodology for generating and interpreting real world data, this guidance will not elaborate on the methodological approaches that can be used.”

The FDA believes that real world data drawn from clinical practice settings can provide greater insight into the benefits and risks of medical devices, including how they’re used by health care providers and patients, and how they perform under the conditions of routine medical practice. The guidance does not change the evidentiary standards that are required to make those decisions.

Given the expansion in the availability and use of health data derived from registries, billing claims, and electronic health records, the FDA is taking these steps in hopes of clearly describing the appropriate characteristics of real world evidence that meet the agency's standards of evidence to support regulatory decisions.

To assess reliability of data, the FDA is primarily concerned with how the data were collected (data accrual), and whether there were people and processes in place to provide “adequate assurance” that the data quality and integrity are sufficient. The real world data source should have an operational manual (or similar document) in place specifying the methods and extent of data collection and aggregation. FDA provides a list of factors that it will consider the when assessing data accrual, many of which are also considerations for any clinical trial.

Several weeks after the release of the guidance, FDA Commissioner Scott Gottlieb stated that the agency will need to work with the healthcare system to change the way clinical information is collected.

"We need to close the evidence gap between the information we use to make FDA's decisions and the evidence increasingly used by the medical community, by payers, and by others charged with making healthcare decisions," Gottlieb told attendees of the National Academy of Sciences' workshop on the impact of real world evidence on medical product development.

While noting the current uncertainty among sponsors on the role real world evidence plays in regulatory decision making, Gottlieb said FDA needs to think of itself as an information curator, rather than an arbiter of information, "where a single truth standard is secured to a fixed orthodoxy."

Rachel Sherman, principal deputy commissioner at FDA, added later in the day, "We want a better evidence base to make product approval decisions" and she noted that more guidance will be forthcoming on real world evidence and real world data. "The goal is not to define real world evidence and real world data but to get better information and in a more sensible way," she added.

For more information, we encourage you to read the final guidance and access the FDA webinar on the topic here.

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