用深度学习预测临床试验招募人数并给出不确定性范围。
Deep Learning-based Prediction of Clinical Trial Enrollment with Uncertainty Estimates
- 结合语言模型与表格特征,通过注意力机制融合文本与结构化数据。
- 基于伽马分布设计概率层,可输出招募人数的预测区间。
- 在真实数据上优于传统模型,适合临床研究规划者使用。
临床试验是系统评估新药或疗法安全性和有效性的关键环节,通常需要大量资金投入和精细规划,因此准确预测试验结果至关重要。患者招募情况是决定试验成败的核心因素之一,也是规划阶段的主要挑战。本文提出一种基于深度学习的新方法,采用神经网络模型,利用预训练语言模型(PLMs)提取临床文档中的复杂语义信息,生成高表达性表征,并通过注意力机制与编码后的表格特征融合。为量化招募预测的不确定性,模型引入基于伽马分布的随机层,实现对招募人数的范围估计。假设各研究中心的招募服从泊松-伽马过程,模型用于预测特定临床试验在多个中心的患者招募数量。在真实世界临床试验数据上进行大量实验,结果表明该方法能有效预测患者招募量,性能优于现有基准模型。
原文摘要 · Abstract (English)
Clinical trials are a systematic endeavor to assess the safety and efficacy of new drugs or treatments. Conducting such trials typically demands significant financial investment and meticulous planning, highlighting the need for accurate predictions of trial outcomes. Accurately predicting patient enrollment, a key factor in trial success, is one of the primary challenges during the planning phase. In this work, we propose a novel deep learning-based method to address this critical challenge. Our method, implemented as a neural network model, leverages pre-trained language models (PLMs) to capture the complexities and nuances of clinical documents, transforming them into expressive representations. These representations are then combined with encoded tabular features via an attention mechanism. To account for uncertainties in enrollment prediction, we enhance the model with a probabilistic layer based on the Gamma distribution, which enables range estimation. We apply the proposed model to predict clinical trial duration, assuming site-level enrollment follows a Poisson-Gamma process. We carry out extensive experiments on real-world clinical trial data, and show that the proposed method can effectively predict the number of patients enrolled at a number of sites for a given clinical trial, outperforming established baseline models.
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