FDA Launches Real-Time Clinical Trials, Seeks AI Pilot Input
FDA launched two proof-of-concept real-time clinical trials on April 28, 2026, and issued an RFI for an AI-focused pilot program to optimize early-phase trials. The agency aims to reduce gaps between trial phases and modernize drug development.
On April 28, 2026, the Food and Drug Administration (FDA) unveiled two major announcements as part of its initiative to "advance the implementation of real-time clinical trials." In conjunction with a request for information (RFI) for a proposed pilot program, the agency announced the successful initiation of two proof-of-concept real-time clinical trials (RTCTs). The agency's stated goal is to enable continuous trials and reduce the "hiatuses" between the phases of clinical development, ultimately improving the drug development process.
The RTCTs will report endpoints and data signals to FDA in real-time. One of the two proof-of-concept RTCTs is a Phase 2 multi-site trial for patients with treatment-naïve mantle cell lymphoma. The other is a Phase 1b trial for patients with limited-stage small cell lung carcinoma, with final site selection still in process.
FDA is seeking to build on these proofs-of-concept with a broader pilot program and is requesting comment through an RFI published in the Federal Register on April 29, 2026. The proposed pilot program focuses on the use of artificial intelligence (AI) and AI-enabled technologies to optimize early phase clinical trials and assess how AI-enabled technologies can improve efficiency, speed, and quality of decision-making in early phase clinical trials. The proposed pilot seeks to evaluate whether AI can improve trial efficiency, enhance safety monitoring, support dose selection decisions, and enable earlier and more informed go/no-go decisions. The agency is soliciting feedback on multiple aspects of the pilot, including program design and implementation and evaluation metrics and success criteria.
Undertaking an RTCT will have numerous regulatory and compliance implications, including adoption of real-time reporting and impacts on human subject protections, such as the potential need for extended patient monitoring given the shrinking of the traditional gaps between trial phases. Companies often plan their clinical trials to support multiple regulatory authorizations and would need to factor that into their strategies.
The deadline for commenting on the RFI is May 29, 2026, and the agency intends to disseminate final selection criteria in July and complete pilot selections in August. The agency's efforts to modernize drug development with specific focus on optimizing early-phase clinical trials are of broad interest and will likely be an area of focus in the Prescription Drug User Fee Act (PDUFA) reauthorization.