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.