Califf and 14 FDA Officials Look to Temper Expectations for Real-World Evidence

Fifteen FDA officials, including Commissioner Robert Califf, called for broader uses of research methodologies, such as intervention and randomization, in real-world settings outside the typical venues of clinical trials.

But they cautioned against expectations for “quick wins” in saving time and money in development, and asked for appropriate expectations of what the data can illustrate.

In an opinion piece in the New England Journal of Medicine, they said that in order to gain confidence in the use of real-world studies, their distinguishing characteristic must be defined as the context in which the evidence is gathered, such as under clinical care or in home or community settings. The distinction should not be based on interventions or randomizations of participants, because those methods are entirely compatible in the real-world setting, they said.

Limitations in source data could lead to flawed conclusions, they wrote. However, the authors were optimistic that the data can add important perspectives to medical device assessments: generate hypotheses for prospective trials, help generalize findings from controlled trials and measure quality in health care delivery.

Prospective registries or single-group trials with external controls have been accepted for regulatory purposes in evaluating medical devices, but because refinements are made over the course of a product’s life cycle, substantial knowledge is available tracking treatment effects and confounding factors.

The authors said they plan to hold public workshops and meetings on real-world evidence, and ultimately publish draft guidances. Indeed, such steps will soon be required of the agency, following the enactment of the 21st Century Cures Act passed by Congress.

The FDA and others are also currently working to develop a system that would combine real-world data with patient registries to monitor safety and effectiveness, they wrote, and the agency plans to develop policy for both drugs and devices under the proposed reauthorization of PDUFA VI next year, as well as in the fourth iteration of the Medical Device User Fee Amendments.

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