用无监督分组方法在真实医疗数据中评估政策优先级,为有限预算提供可解释的决策支持。
From Unsupervised Subgroups to Hypothetical State-Intervention Policies: An Evaluation of Selected Subgrouping Methods in Observational Health Data

- 基于治疗前特征构建无监督子群,不依赖暴露或结果信息
- 在糖尿病和吸烟数据上,最高收益达0.799,但差异不显著
- 适合预算受限的公共卫生政策制定者参考
传统亚组分析在观察性生物医学数据中常得出不稳定且难以解释的结果,因个体仅观测到单一暴露状态,真实治疗效应不可得,因果结构不确定。本文研究仅基于治疗前特征构建的子群,能否作为预算受限政策优先化的可解释单元。提出融合因果发现引导的协变量选择、发现-评估样本分割、归纳式无监督聚类、不确定性感知子群选择及保留样本双重稳健政策评估的框架。对比了K-means、硬/加权/随机模糊C均值、贝叶斯高斯混合模型,以及监督式因果森林生成的CATE树。在PIMA印度人糖尿病数据集上评估肥胖转非肥胖与高血糖转低血糖的假设干预,在NHANES中评估终身吸烟史对比。最高未约束效用分别为:贝叶斯GMM在BMI政策中0.799,硬/加权模糊C均值在葡萄糖政策中0.735,K-means在吸烟史政策中0.775。所有配对95%置信区间包含零,经霍尔姆校正后均无统计显著性。贝叶斯合并通常保持原分配,而经验伯恩斯坦门控更保守。相似效用政策可能推荐不同人群。结论应视为假设对比下的假设性决策支持,而非干预效益证明。
原文摘要 · Abstract (English)
Conventional subgroup analyses can yield unstable and difficult-to-interpret conclusions, especially in observational biomedical data where each individual is observed under only one exposure state, true individual treatment effects are unavailable, and causal structure is uncertain. We investigate whether subgroups constructed from pretreatment characteristics, without using exposure, outcome, or estimated treatment-effect information, can serve as interpretable units for budget-constrained policy prioritization. We propose a framework combining causal-discovery-informed covariate selection, discovery-evaluation sample splitting, inductive unsupervised clustering, uncertainty-aware subgroup selection, and held-out doubly robust policy evaluation. We compare K-means, hard, membership-weighted, and stochastic Fuzzy C-means, Bayesian Gaussian mixture models, and a supervised causal-forest-derived CATE-tree comparator. Policies are evaluated under a 70% budget for hypothetical obesity-to-non-obesity and elevated-to-lower-glucose state shifts in the PIMA Indians Diabetes dataset and for a lifetime-smoking-history contrast in NHANES. The highest estimated ungated utilities were 0.799 for the BMI policy using Bayesian GMM, 0.735 for the glucose policy using hard or membership-weighted FCM, and 0.775 for the smoking-history policy using K-means. All paired 95% confidence intervals for policy-risk differences included zero, and no comparison remained statistically significant after Holm adjustment. Bayesian pooling generally preserved ungated allocations, whereas Empirical Bernstein gating was more conservative. Policies with similar estimated utility could nevertheless prioritize different individuals. The findings should be interpreted as assumption-dependent decision-support evidence for hypothetical state contrasts rather than proof of intervention benefit.
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