Agency unveils real-time trial proofs-of-concept and upcoming pilot program
The U.S. Food and Drug Administration today announced two major steps as part of an initiative to advance the implementation of real-time clinical trials (RTCT). First, the agency unveiled the successful initiation of two proof-of-concept clinical trials that will report endpoints and data signals to the agency in real time. Second, the agency released a Request for Information (RFI) regarding a proposed pilot program for RTCT that will launch this summer.
Early-phase clinical trials are a bottleneck in drug development, often characterized by high uncertainty, limited patient populations, and inefficient decision-making processes. Data is typically reported from sites to sponsors, who analyze and subsequently submit data to the FDA. With improvements in AI and data science, sponsors and trial sites have the opportunity to conduct real-time trials in a way that enhances safety monitoring and radically increases efficiency.
“For 60 years, we've been conducting clinical trials in the same way, where key data signals can take years to reach the FDA. The lag time can delay regulatory decisions unnecessarily and slow down the drug development timeline,” said FDA Commissioner Marty Makary, M.D., M.P.H. “We are boldly advancing a modern approach whereby FDA scientists can view safety signals and endpoints in real time as a trial progresses. This will help us accelerate promising therapies, and build toward our ultimate goal of running real-time, continuous trials across all phases of drug development.”
The FDA is announcing the successful initiation of proof-of-concept RTCTs by AstraZeneca and Amgen. AstraZeneca is conducting a Phase 2 multi-site trial, TRAVERSE, in patients with treatment-naïve mantle cell lymphoma, with participation from The University of Texas MD Anderson Cancer Center and University of Pennsylvania. Amgen is conducting a Phase 1b trial, STREAM-SCLC, in patients with limited-stage small cell lung carcinoma and final site selection is in process. For each trial, the FDA met with the sponsor on the establishment of criteria for reporting signals in real time. The agency has since received and validated signals for AstraZeneca’s trial through Paradigm Health, thereby establishing the feasibility of the technical framework required for real-time signal sharing.
The FDA seeks to build on these proofs-of-concept with a broader pilot program. Today’s RFI seeks input on potential pilot program design and implementation, as well as evaluation metrics and success criteria.
“Real-time trials have been talked about for years. We demonstrated that it is not only possible, but also potentially transformative for the clinical trials ecosystem,” said Chief AI Officer Jeremy Walsh. “We have to consider our processes from the standpoint of a patient awaiting a potentially powerful treatment.”
Real-time clinical trials are an important step towards the agency’s goal of facilitating continuous trials. At present, most clinical development occurs in discrete phases. Because each defined phase of clinical development is run according to a protocol and typically as a separate study, there is generally a hiatus in the development program after one phase ends and the next begins. This slows the pace of product development. Because real-time trials allow the FDA to view key insights in real time, this hiatus could be eliminated or reduced to a minimum, enabling “continuous” trials.
The agency will accept comments on the RFI until May 29, 2026. The agency intends to disseminate final selection criteria in July and complete pilot selections in August.
An unofficial translation of this announcement is provided below.
翻译参考：
FDA宣布推进实时临床试验的重大举措
FDA发布实时试验概念验证成果以及即将启动的试点项目
2026年4月28日，美国食品药品管理局宣布，作为推动实时临床试验（RTCT）实施举措的一部分，已采取两项重要措施。首先，FDA公布已成功启动两项概念验证临床试验，这些试验将向监管机构实时报告终点指标和数据信号。其次，FDA发布了一份信息征询公告（RFI），就计划于今年夏季启动的实时临床试验试点项目征求意见。
早期临床试验是药物开发过程中的瓶颈，通常具有高度不确定性、患者规模有限以及决策效率低等特点。传统模式下，数据由试验中心报告给申办方，由申办方分析后再提交给FDA。随着人工智能和数据科学的发展，申办方和试验中心有机会开展实时临床试验，从而提升安全性监测能力并大幅提高效率。
“60年来，我们一直以同样的方式开展临床试验，关键数据往往需要数年时间才能传递到FDA。这种滞后会不必要地延迟监管决策，并减缓药物开发进程，”FDA局长Marty Makary博士表示。“我们正在积极推进一种现代化模式，使FDA科学家能够在试验进行过程中实时查看安全信号和终点指标。这将有助于加速有前景疗法的开发，并朝着在药物开发各阶段开展实时、连续试验的终极目标迈进。”
FDA宣布，阿斯利康和安进公司已成功启动概念验证实时临床试验。阿斯利康正在开展一项名为TRAVERSE的II期多中心试验，研究对象为初治套细胞淋巴瘤患者，参与机构包括德克萨斯大学MD安德森癌症中心和宾夕法尼亚大学。安进公司正在开展一项名为STREAM-SCLC的Ib期试验，研究对象为局限期小细胞肺癌患者，目前试验中心仍在遴选中。对于上述试验，FDA已与申办方就实时信号报告标准进行了沟通。FDA已通过Paradigm Health接收并验证了来自阿斯利康试验的信号，从而证明了实时信号共享技术框架的可行性。
FDA计划在这些概念验证基础上，进一步推进更广泛的试点项目。本次发布的信息征询公告旨在就试点项目的设计与实施方案，以及评估指标和成功标准征求意见。
“实时试验多年来一直停留在讨论层面。我们已经证明，这不仅可行，而且对临床试验生态系统具有潜在的变革性意义，”首席人工智能官Jeremy Walsh表示。“我们需要站在那些正等待潜在有效疗法的患者立场上，来重新审视我们的流程。”
实时临床试验是实现FDA“连续试验”目标的重要一步。目前，临床开发多以相互独立的阶段形式开展。由于各阶段通常按照各自方案分别作为独立研究实施，一个阶段结束到下一个阶段开始之间往往存在间隔，从而减缓开发进程。实时试验使FDA能够实时获取关键信息，有望消除或大幅缩短这一间隔，从而实现“连续”试验。
FDA将于2026年5月29日之前接受对信息征询公告的意见反馈，并计划于7月发布最终遴选标准，8月完成试点项目的遴选。
Sincerely,
Sarah McMullen
Country Director
FDA China Office
Office of Global Operations
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