FDA Evaluating Measures to Modernize Clinical Trials
FDA is moving to modernize how clinical trials are conducted, unveiling a pair of proof-of-concept studies and outlining plans for a broader pilot program aimed at enabling real-time data sharing between sponsors and regulators.
In a 4/28 announcement Tuesday, the agency says two early-stage “real-time clinical trials” (RTCTs) are underway to test new concepts. They are designed to transmit safety signals and efficacy endpoints to reviewers as they emerge, rather than through the traditional, delayed reporting process, according to the agency. At the same time, FDA has issued a Request for Information (RFI) seeking industry input on a pilot program slated to begin this summer.
The initiative targets a longstanding bottleneck in drug development: early-phase trials, which are often marked by small patient populations, fragmented data flows and slow decision-making, according to the agency. Historically, trial data has moved from clinical sites to sponsors for analysis before being submitted to FDA, a process that can take months or years.
“With advances in artificial intelligence and data science, that lag is no longer necessary,” says FDA commissioner Marty Makary, noting that real-time access to trial data could accelerate regulatory decisions and shorten development timelines. He described the effort as part of a broader push toward “continuous” clinical trials spanning multiple phases.
The proof-of-concept studies involve two major drugmakers. AstraZeneca has initiated TRAVERSE, a Phase 2 multi-site study evaluating treatment-naïve mantle cell lymphoma patients, with participation from MD Anderson Cancer Center and the University of Pennsylvania. Meanwhile, Amgen is conducting a Phase 1b trial, STREAM-SCLC, in limited-stage small cell lung cancer, with site selection ongoing.
For both trials, FDA worked with sponsors to establish criteria for real-time signal reporting. The agency says it has already received and validated live data signals from AstraZeneca’s study via Paradigm Health, demonstrating the technical feasibility of continuous data exchange.
FDA chief artificial intelligence officer Jeremy Walsh says the early results show that real-time trials could be “transformative” for the clinical research ecosystem, particularly for patients waiting on new therapies.
The agency’s longer-term goal is to enable continuous clinical development, reducing or eliminating the pauses that typically occur between trial phases. By allowing regulators to monitor data in real time, the agency believes transitions between phases could be streamlined, potentially speeding up product approvals.
The RFI seeks feedback on how to design and evaluate the planned pilot program, including metrics for success. The pilot is intended to assess how AI-enabled technologies can improve efficiency, speed, and quality of decision-making in early-phase clinical trials, according to a Federal Register notice. It will also explore how advances in AI and data science can enhance safety monitoring, facilitate dose selection decisions, and enable more informed early go/no-go decisions (e.g., a regulatory decision as to whether a Phase 1 study may proceed). The pilot will be guided by principles aligned with the National Institute of Standards and Technology (NIST) AI Risk Management Framework, the agency says.
Public comments will be accepted through 5/29, with final selection criteria expected in July and pilot participants chosen by August.