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

Fifteen FDA officials, including Commissioner Robert Califf, called for broader uses of research methodologies in real-world settings, but cautioned against expectations for “quick wins” in cost-savings and efficiency for device approvals.

In the New England Journal of Medicine, they said that gaining confidence in the use of real-world studies requires distinguished characteristics that must be defined in the context that the evidence is gathered in, such as under clinical care or in home or community settings. The distinction should not be based on interventions or randomizations of participants.

Limitations in source data could lead to flawed conclusions, they wrote, but that data can likely add important perspectives to medical device assessments, generate hypotheses, 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, they wrote.