arXiv:2505.18527cs.LGcs.AI2025-05KDD被引 1

用大模型预训练提升临床试验预测通用性,减少误判。

CLaDMoP: Learning Transferrable Models from Successful Clinical Trials via LLMs

  • 用大语言模型编码入组标准,融合药物分子信息
  • 在相位一、二期试验中比基线提升10.5%的精确率
  • 适合需低样本学习的临床研究场景

现有临床试验结果预测模型多基于特定任务损失函数和分阶段数据优化,虽提升常见疾病与药物的预测性能,却抑制了可迁移表征的学习,导致更多假阳性/假阴性。为此,我们提出CLaDMoP,一种面向临床试验结果预测的新预训练方法,并构建专为此任务设计的“成功临床试验数据集”(SCT)。CLaDMoP利用大语言模型编码试验入组标准,通过新颖的多层级融合技术连接轻量级药物-分子分支。为高效融合多层级长嵌入,引入分组模块,显著降低计算开销。该方法通过“配对匹配”代理任务进行预训练,避免依赖特定任务目标。相较于零样本与少样本基线,其在相位一、二期试验中均显著提升PR-AUC与ROC-AUC。经参数高效微调后,在临床试验结果预测(TOP)基准上,相比先进监督模型MEXA-CTP,CLaDMoP实现最高10.5%的PR-AUC提升与3.6%的ROC-AUC提升,同时保持与之相当的F1分数,展现其在临床试验预测中的潜力。代码与SCT数据集可在https://github.com/murai-lab/CLaDMoP下载。

原文摘要 · Abstract (English)

Many existing models for clinical trial outcome prediction are optimized using task-specific loss functions on trial phase-specific data. While this scheme may boost prediction for common diseases and drugs, it can hinder learning of generalizable representations, leading to more false positives/negatives. To address this limitation, we introduce CLaDMoP, a new pre-training approach for clinical trial outcome prediction, alongside the Successful Clinical Trials dataset(SCT), specifically designed for this task. CLaDMoP leverages a Large Language Model-to encode trials' eligibility criteria-linked to a lightweight Drug-Molecule branch through a novel multi-level fusion technique. To efficiently fuse long embeddings across levels, we incorporate a grouping block, drastically reducing computational overhead. CLaDMoP avoids reliance on task-specific objectives by pre-training on a "pair matching" proxy task. Compared to established zero-shot and few-shot baselines, our method significantly improves both PR-AUC and ROC-AUC, especially for phase I and phase II trials. We further evaluate and perform ablation on CLaDMoP after Parameter-Efficient Fine-Tuning, comparing it to state-of-the-art supervised baselines, including MEXA-CTP, on the Trial Outcome Prediction(TOP) benchmark. CLaDMoP achieves up to 10.5% improvement in PR-AUC and 3.6% in ROC-AUC, while attaining comparable F1 score to MEXA-CTP, highlighting its potential for clinical trial outcome prediction. Code and SCT dataset can be downloaded from https://github.com/murai-lab/CLaDMoP.

临床预测大模型预训练

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