arXiv:2512.00586cs.LGcs.CL2025-12被引 1

用NLP预测神经科临床试验成败,提升研发决策效率。

Statistical NLP for Optimization of Clinical Trial Success Prediction in Pharmaceutical R&D

  • 基于统计NLP提取临床试验文本特征,构建概率分类器。
  • 生物BERT模型实现0.74的ROC-AUC,比行业基准误差小40%。
  • 适合医药研发机构优化资源分配与降低投资风险。

本研究开发并评估了一种基于NLP的概率分类器,用于估算神经科学领域临床试验的技术与监管成功概率(pTRS)。针对制药研发中高淘汰率与高昂成本问题,尤其在神经科学领域成功率低于10%的情况下,及时识别有前景的研发项目可优化资源配置、降低财务风险。利用ClinicalTrials.gov数据库和新发布的临床试验结果数据集,通过统计NLP技术提取文本特征,并将其输入逻辑回归、梯度提升和随机森林等非大模型框架,生成校准概率评分。在覆盖1976–2024年共101,145项已完成试验的回溯数据集上,整体ROC-AUC达0.64。随后构建基于BioBERT的LLM预测模型,整体ROC-AUC达0.74,Brier Score为0.185,表明其预测平均平方误差比行业基准低40%;在70%的案例中,其预测优于基准值。该模型有望通过融合NLP洞察提升药物研发战略规划与投资决策效率。

原文摘要 · Abstract (English)

This work presents the development and evaluation of an NLP-enabled probabilistic classifier designed to estimate the probability of technical and regulatory success (pTRS) for clinical trials in the field of neuroscience. While pharmaceutical R&D is plagued by high attrition rates and enormous costs, particularly within neuroscience, where success rates are below 10%, timely identification of promising programs can streamline resource allocation and reduce financial risk. Leveraging data from the ClinicalTrials.gov database and success labels from the recently developed Clinical Trial Outcome dataset, the classifier extracts text-based clinical trial features using statistical NLP techniques. These features were integrated into several non-LLM frameworks (logistic regression, gradient boosting, and random forest) to generate calibrated probability scores. Model performance was assessed on a retrospective dataset of 101,145 completed clinical trials spanning 1976-2024, achieving an overall ROC-AUC of 0.64. An LLM-based predictive model was then built using BioBERT, a domain-specific language representation encoder. The BioBERT-based model achieved an overall ROC-AUC of 0.74 and a Brier Score of 0.185, indicating its predictions had, on average, 40% less squared error than would be observed using industry benchmarks. The BioBERT-based model also made trial outcome predictions that were superior to benchmark values 70% of the time overall. By integrating NLP-driven insights into drug development decision-making, this work aims to enhance strategic planning and optimize investment allocation in neuroscience programs.

NLP临床试验药物研发概率预测

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