The FDA’s real-time clinical trial mechanism allows drug sponsors to transmit data immediately to the regulator through the cloud—a system that could “compress drug development timelines,” Jefferies analysts said.
AstraZeneca and Amgen will pilot the FDA’s newest initiative aimed at streamlining the drug development and review processes via real-time clinical trials.
“What exactly is a real-time clinical trial?” Commissioner Marty Makary posed to reporters at a press event on Tuesday. “It’s when FDA reviewers can see safety signals and clinical endpoints in the cloud, in real-time, as a clinical trial is happening.” Here, Makary is referring to internet-based servers that can host data repositories for drug sponsors.
“It’s a great way to interact with the actual trial on a data-driven basis,” he added.
Real-time clinical trials represent “another step towards reducing structural inefficiencies and accelerating timelines,” according to a Tuesday note to investors from Jefferies. “Receiving data flow in real-time enables the FDA to make decisions faster—all while preserving the foundational requirements of safety, monitoring, governance, and data integrity.”
Two pharma companies have agreed to lend a program to test the FDA’s new mechanism. AstraZeneca will use this cloud-based real-time reporting system for its Phase 2 TRAVERSE study in treatment-naïve mantle cell lymphoma, while Amgen will deploy the system for the Phase 1b STREAM-SCLC study in limited-stage small cell lung cancer.
The FDA has already successfully received and validated data from AstraZeneca’s program, demonstrating that its current technical framework for real-time data sharing indeed works, according to a news release from the agency.
Alongside the first test cases, the FDA on Tuesday also opened its real-time clinical trial framework for public feedback, seeking input regarding the design and implementation of pilot programs, as well as what metrics could be used to evaluate the system and gauge success. The agency expects to complete final selection of pilot participants in August.
“The initiative leverages AI and modern data infrastructure to address inefficiencies in early-phase trials, where data are typically delayed due to sequential/manual reporting and data analyses,” analysts at Jefferies explained. The firm expects that this new mechanism will help “compress drug development timelines” as well as improve productivity across biopharma.
In turn, real-time clinical trials could allow companies to maximize peak sales potential, the analysts continued.
The FDA has in recent months implemented a series of policy changes in an attempt to make the drug development process smoother and faster for companies.
In February, for instance, the agency announced that just one well-controlled trial would be needed for drug applications. Instead of a second trial, which had been the agency’s standard for years, sponsors can file other “confirmative evidence” to support their drug packages, including mechanistic data and findings from related indications.
The FDA has also been initiating new policies to boost therapeutic areas that are relatively underserved and where there is a lot of medical need. Earlier this month, acting on orders from President Donald Trump himself, the regulator awarded Commissioner’s National Priority Review Vouches to three psychedelic drug developers, giving them tickets that could shave the review period down to 1–2 months from the normal 10–12 months.
Last year, the agency also unveiled the “plausible mechanism” pathway for personalized therapies addressing rare diseases, allowing approvals based largely on evidence that a medicine could address the underlying biology of a particular illness.
Despite these initiatives, however, the industry has struggled over the past year with a lack of clarity from the agency and multiple decisions that appeared to be reversals of course for the FDA, blindsiding companies and derailing entire drug programs.
