FDA sets out plans for real-time clinical trials, aiming to streamline evidence generation

As part of broader efforts to modernize clinical development, the FDA has introduced real-time clinical trial pilots and proposed a new program for continuous data monitoring. The initiative seeks to address delays in evidence generation and streamline regulatory review processes.

The US Food and Drug Administration (FDA) has announced a new initiative aimed at advancing real-time clinical trials (RTCT), marking a shift in how clinical data may be generated, monitored, and reviewed. The agency confirmed the launch of two proof-of-concept trials alongside a Request for Information (RFI) to inform a broader pilot program expected to begin later this year.

Clinical development has traditionally followed a lengthy sequential, phase-based model, with data collected at trial sites, analyzed by sponsors, and submitted to regulators at defined intervals. According to FDA Commissioner Marty Makary, this model has remained largely unchanged for decades. “For 60 years, we’ve been conducting clinical trials in the same way, where key data signals can take years to reach the FDA,” he said. “The lag time can delay regulatory decisions unnecessarily and slow down the drug development timeline.”

The RTCT initiative seeks to address these constraints by enabling regulators to access safety signals and clinical endpoints as they emerge. Under this model, trial data are transmitted and visualized in near real time, allowing for ongoing oversight rather than retrospective review. Makary described the effort as part of a broader rethinking of the drug development process, stating that “every part of this process is being re-engineered to ask ourselves what workflow can we make more efficient without cutting any corners on safety.”

The FDA also highlighted inefficiencies beyond data submission. Drug development timelines, often estimated at 10–12 years, include substantial periods of inactivity. “Very few people know that 45% of the drug development time is dead time,” Makary said, referring to gaps between trial phases and administrative processes such as data reconciliation and submission preparation. Real-time approaches could reduce these delays by supporting more continuous trial designs and minimizing pauses between phases.

Two industry-sponsored trials are currently serving as test cases. AstraZeneca has initiated a Phase 2 study in mantle cell lymphoma, while Amgen is conducting a Phase 1b trial in small cell lung cancer. In both cases, the FDA worked with sponsors to define criteria for real-time data sharing and reporting. The agency reported that it has already received and validated live data signals from the AstraZeneca study, demonstrating the technical feasibility of the approach.

The technical infrastructure supporting these pilots is being delivered through a collaboration with health-tech company Paradigm Health. Its Study Conduct platform enables real-time data capture, analysis, and reporting by extracting information directly from electronic health records and other structured and unstructured sources. These data are algorithmically assessed against predefined regulatory criteria, with only relevant safety signals and efficacy endpoints transmitted to sponsors and the FDA. The approach is intended to reduce the burden associated with manual data entry and monitoring while streamlining the reporting of critical signals for regulatory review.

Alongside these developments, the FDA has issued an RFI seeking stakeholder input on the design, implementation, and evaluation of a pilot program. The agency plans to finalize selection criteria by July 2026, with pilot participants confirmed in August.

While the announcement has been broadly welcomed as a step toward faster and more responsive evidence generation, stakeholders have raised operational and methodological considerations. Comments from industry observers have highlighted that real-time trials will require robust infrastructure, including site readiness, streamlined data flows, and clear governance across sponsors and contract research organizations. Continuous data availability may also challenge existing regulatory frameworks, particularly around what constitutes sufficient evidence for approval. Some observers note that while reducing “dead time” could improve efficiency, particularly for smaller biotechs, this depends on their ability to operate in continuous data environments. As one commented, “the technology makes the data continuous; the decision frameworks haven’t caught up.”