Abbasi Ali, Weltz Adam, Rivera Donna R, Nagel Steven, Mehta Gautam U, Ashar Binita S, Cunningham Steven Clark
US Food and Drug Administration, Silver Spring, Maryland.
Department of Surgery, University of California, San Francisco.
JAMA Surg. 2025 Oct 1;160(10):1133-1137. doi: 10.1001/jamasurg.2025.3041.
Randomized clinical trials (RCTs), the gold standard of medical evidence, remain relatively rare in surgery. In part, this is the result of challenges that are unique to surgical trials, including surgeon concerns about lack of equipoise, slow recruitment, difficulty standardizing interventions, and a lack of funding and infrastructure for surgical trials. Here, it is argued that increasing the use of pragmatic clinical trials (PCTs) could help overcome some of the challenges of surgical RCTs. The study perspective is informed by work at the US Food and Drug Administration (FDA), which regulates many clinical trials.
While exploratory trials aim to maximize the chance that an intervention demonstrates a hypothesized benefit in a narrow population and indication, PCTs are designed to evaluate an intervention in conditions that resemble routine clinical care. The FDA has been interested in increasing the use of pragmatic design elements in clinical trials because these trials can lower the cost and complexity of evidence generation and result in improved generalizability. In surgical trials, pragmatic design elements could include giving surgeon-investigators flexibility in deciding which patients to enroll and which surgical technique to use, a flexibility that helps to ensure that the resulting evidence is applicable to these decisions in routine practice. To streamline data collection, PCTs could leverage existing surgical registries or use data routinely collected in the electronic health record to measure outcomes.
Increasing the use of PCTs presents an opportunity to overcome the challenges of surgical RCTs. However, there is a need to prioritize questions that matter most to patients and health care professionals and to develop improved training, funding, and institutional support for PCTs in surgery.
随机临床试验（RCT）作为医学证据的金标准，在外科领域仍然相对少见。部分原因在于外科试验所特有的挑战，包括外科医生担心缺乏均衡性、招募缓慢、干预措施难以标准化以及外科试验缺乏资金和基础设施。在此，有人认为增加实用临床试验（PCT）的使用有助于克服外科RCT的一些挑战。该研究观点受到美国食品药品监督管理局（FDA）工作的启发，FDA负责监管许多临床试验。
探索性试验旨在最大化干预措施在狭窄人群和适应症中显示出假设益处的机会，而PCT旨在评估在类似常规临床护理条件下的干预措施。FDA一直对在临床试验中增加实用设计元素的使用感兴趣，因为这些试验可以降低证据生成的成本和复杂性，并提高普遍性。在外科试验中，实用设计元素可能包括给予外科医生 - 研究者在决定纳入哪些患者以及使用哪种手术技术方面的灵活性，这种灵活性有助于确保所产生的证据适用于常规实践中的这些决策。为了简化数据收集，PCT可以利用现有的外科登记系统或使用电子健康记录中常规收集的数据来衡量结果。
增加PCT的使用为克服外科RCT的挑战提供了机会。然而，有必要优先考虑对患者和医疗保健专业人员最重要的问题，并为外科PCT开发更好的培训、资金和机构支持。
