Califf Calls Again for Use of RWE, Acceleration of Confirmatory Studies
Five months into his second go-round as FDA commissioner, Robert Califf once more stressed the importance of developing and employing a real-world evidence (RWE) system to support clinical research, especially for confirmatory trials of accelerated approvals.
Speaking at a virtual event hosted by the Alliance for a Stronger FDA, Califf contended that an RWE-gathering system could be used to ensure confirmatory studies for therapies granted accelerated approval are conducted in a much timelier manner. During his Senate confirmation hearings, the agency chief advocated for such a system that would tap into valuable electronic health record (EHR) data.
“I think we do a really good job of developing medical products up through the early phases, and that system works well,” Califf said. But when it comes to determining the right doses of drugs and how to combine and compare medical products, “I’d say we’re doing a lousy job of generating that evidence.”
“And yet,” he continued, “everyone in the United States has an EHR packed full of data that could be used to do studies very inexpensively.” But data privacy concerns over use of such data have exceeded those of public health interests, he acknowledged.
Califf also addressed issues with the FDA’s accelerated approval pathway, which has come under heavy scrutiny following the controversial clearance of Biogen’s Alzheimer’s drug Aduhelm (aducanumab) in June 2021. More and more of the agency’s approvals are accelerated, he pointed out, and with only early data to evaluate, it’s reasonably likely that a drug’s benefits will appear to outweigh the risks because it hasn’t yet been proven by postmarket confirmatory studies. And the agency’s approach to conducting confirmatory studies is “simply inadequate.”
“I think it was a signal to the very broad community when the initial statement was that it would take nine years to do the follow-up study on Aduhelm,” he said. “People said, ‘wait a minute, that doesn’t sound like a timeframe that’s reasonable.’”