Experts are cautiously excited about ‘real-time’ FDA clinical trials

The FDA wants to give clinical trials a makeover.

On April 28, recently departed FDA commissioner Marty Makary announced “real-time” clinical trials that would send prespecified safety signals and clinical endpoint data to the FDA, via its cloud, as a trial is happening.

The current system has significant “dead time” between the start of Phase 1 and the final submission of an approval application to the FDA, Makary said during a press conference, adding that during that period, “there is no ongoing clinical trial. Investigators and staff are doing paperwork and other tasks, many of which are tedious.”

The agency plans to deploy AI to help identify potential signals that could lead a study to be fast tracked or scrapped, Jeremy Walsh, the FDA’s chief AI officer, said during the conference. “What we’re interested in is: Can we make a decision off of less information?” he said. 

Experts told Healthcare Brew they’re excited, but revamping the clinical trial process requires more than just cranking up the speed.

‘Dirty data.’ Major drugmakers AstraZeneca and Amgen are on board and signed on for proof-of-concept tests of this idea for respective Phase 2 and Phase 1b clinical trials involving cancer treatments. The agency has already reviewed signals from AstraZeneca’s trial through a collaboration with clinical trial AI startup Paradigm Health.

But a potential problem with this approach is that data collection and entry is imperfect, Richard Graham, co-founder of real-time clinical trial oversight company TruTechnologies, told Healthcare Brew. Graham has spent nearly 30 years working in drug discovery and development and has seen “countless mistakes” in clinical trials during that time, like mislabeled, lost, or unusable biological samples.

The old approach gives trial sponsors time to organize and clean data before presenting it to the FDA. Real-time data could send false signals, he said.

It’s “great” that the FDA is doing this, he said, but it should be accompanied by improvements in data collection, too, or it’s possible “the FDA is going to have earlier insight into dirty data.”

Paul Burton, chief medical officer of Amgen, said during the April 28 FDA press conference that safety and efficacy standards for real-time clinical trials will have the same “rigor” as all clinical trials do.

“The fundamentals of good science do not change,” he said. “Protocol development, protocol oversight, informed consent, monitoring, strong governance, FDA review—all of that remains absolutely standard and absolutely essential.”

Amgen is still in the process of selecting a final site for its trial, per the FDA’s press release.

Early concerns. Thomas Marron, director of the early-phase trials unit at New York-based Mount Sinai Tisch Cancer Center, told us he doubts this approach would speed up early-phase clinical trials. Delays there are “typically on the sides of the [sponsor] companies who are setting their priorities and what they’re willing to invest in.”

But, he added, there’s a real potential for this idea to help late-stage trials, which decide whether the FDA will approve a drug for market.

As it goes right now, companies finish a trial and put out a press release with their positive results. But even with positive public data, there’s still a long evaluation process at the FDA that causes a gap between the trial’s finish and approval, he said.

“We still end up waiting, I don’t know, four, six, 12 months before we might see an approval. It might even be years down the road,” he said. “If the FDA were analyzing efficacy and toxicity data in real time for registrational studies, we might actually see drugs get FDA-approved sooner.”