arXiv:2511.08986cs.LGstat.AP2025-11

通过复用旧试验数据,让医疗AI模型的临床验证更省钱更快捷。

Data reuse enables cost-efficient randomized trials of medical AI models

  • 利用新旧AI模型预测一致时复用受试者数据,减少新试验招募人数。
  • 乳腺癌筛查模拟显示可减少46.6%招募量,节省超280万美元。
  • 适合关注医疗AI快速落地的研究者与临床试验设计者。

随机对照试验(RCT)是评估医疗人工智能(AI)工具临床价值的金标准,但其高昂成本和漫长周期制约了新模型的及时验证。本文提出BRIDGE数据复用型RCT设计,适用于基于AI的风险预测模型,支持筛查、治疗选择和临床预警等干预。当新旧模型对个体预测一致时,可复用已完成试验的受试者数据,从而降低后续试验的招募需求。我们提供实用检查清单,帮助研究者判断是否可有效进行因果推断并保持Ⅰ类错误率。在乳腺癌、心血管疾病和败血症的真实数据集上,我们发现连续迭代的AI模型在前5%高危人群中有高达64.8%的重叠。模拟乳腺癌筛查试验表明,该设计可将所需招募人数减少46.6%,节省超280万美元,同时保持80%检验效能。该设计将试验转化为可适应、模块化研究,使每轮模型迭代都能生成Ⅰ级证据,加速医疗AI向常规诊疗的低成本转化。

原文摘要 · Abstract (English)

Randomized controlled trials (RCTs) are indispensable for establishing the clinical value of medical artificial-intelligence (AI) tools, yet their high cost and long timelines hinder timely validation as new models emerge rapidly. Here, we propose BRIDGE, a data-reuse RCT design for AI-based risk models. AI risk models support a broad range of interventions, including screening, treatment selection, and clinical alerts. BRIDGE trials recycle participant-level data from completed trials of AI models when legacy and updated models make concordant predictions, thereby reducing the enrollment requirement for subsequent trials. We provide a practical checklist for investigators to assess whether reusing data from previous trials allows for valid causal inference and preserves type I error. Using real-world datasets across breast cancer, cardiovascular disease, and sepsis, we demonstrate concordance between successive AI models, with up to 64.8% overlap in top 5% high-risk cohorts. We then simulate a series of breast cancer screening studies, where our design reduced required enrollment by 46.6%--saving over US$2.8 million--while maintaining 80% power. By transforming trials into adaptive, modular studies, our proposed design makes Level I evidence generation feasible for every model iteration, thereby accelerating cost-effective translation of AI into routine care.

医疗AI随机试验数据复用成本优化

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