Distributional Balancing with Machine Learning for Clinical Trial Augmentation Using Real-World Data

2026-09-20

AI Summary

本文提出DBML方法，通过匹配治疗组与外部数据库控制组的分布来解决临床试验中对照组难以平衡的问题，使用变分自编码器和重加权技术选择合适的对照样本。

Abstract

In clinical trials, randomization of treatment and control groups is typically used to ensure the groups have similar covariate distributions on average, resulting in unbiased causal effect estimation. Such balanced covariate distributions are hard to achieve in practice, however, due to recruitment costs, patient dropouts, and more. One possible solution to this problem is to include control patients from external, real world databases. In this paper, we propose DBML, a method that selects control units from a real world database by matching the distribution between the treatment group and the potential control group, instead of matching units between two groups. DBML has three steps. First, we detect anomalous database units (with respect to the treatment distribution) using a variational autoencoder. Second, we re-weight the remaining database units to match the distribution of the treatments. Finally, we use these weights to sample units for our control group. The proposed method is compared to alternative matching-based algorithms and weighted algorithms, achieving superior performance in covariate balance.

Problem

• clinical trials
• covariate balance
• real-world data

Innovation

• DBML
• distributional balancing
• variational autoencoder
• real-world data