Enhancing Statistical Validity and Power in Hybrid Controlled Trials: A Randomization Inference Approach with Conformal Selective Borrowing
Ke Zhu, Shu Yang, Xiaofei Wang
摘要
External controls from historical trials or observational data can augment randomized controlled trials when large-scale randomization is impractical or unethical, such as in drug evaluation for rare diseases. However, non-randomized external controls can introduce biases, and existing Bayesian and frequentist methods may inflate the type I error rate, particularly in small-sample trials where external data borrowing is most critical. To address these challenges, we propose a randomization inference framework that ensures finite-sample exact and model-free type I error rate control, adhering to the "analyze as you randomize" principle to safeguard against hidden biases. Recognizing that biased external controls reduce the power of randomization tests, we leverage conformal inference to develop an individualized test-then-pool procedure that selectively borrows comparable external controls to improve power. Our approach incorporates selection uncertainty into randomization tests, providing valid post-selection inference. Additionally, we propose an adaptive procedure to optimize the selection threshold by minimizing the mean squared error across a class of estimators encompassing both no-borrowing and full-borrowing approaches. The proposed methods are supported by non-asymptotic theoretical analysis, validated through simulations, and applied to a randomized lung cancer trial that integrates external controls from the National Cancer Database.
问问这篇 Paper
智能体会读完全文。
Lune 把这篇 Paper 索引到了每一个公式,引用它的顶会 Paper 也一样。你提问,回答直接引用原文。
引用它的顶会 Paper2
- Adaptive Data-Borrowing for Improving Treatment Effect Estimation using External ControlsQinwei Yang, Jingyi Li, Peng WuNeurIPS 2025 · 被引用 4 次
- RSA-CP: Efficient Conformal Prediction in Small-Sample Regimes via Random Score AlignmentPankaj Bhagwat, Zhixian Yang, yihao wang, Bei Jiang 等ICML 2026
相关 Paper
- Conformal Classification with Equalized Coverage for Adaptively Selected GroupsYanfei Zhou, Matteo SesiaNeurIPS 2024 · 被引用 14 次
- Conformalized Multiple Testing after Data-dependent SelectionXiaoning Wang, Yuyang Huo, Liuhua Peng, Changliang ZouNeurIPS 2024 · 被引用 5 次
- Robust Conformal Outlier Detection under Contaminated Reference DataMeshi Bashari, Matteo Sesia, Yaniv RomanoICML 2025
- Multivariate Conformal SelectionTian Bai, Yue Zhao, Xiang Yu, Archer Y. YangICML 2025
- General Synthetic-Powered InferenceMeshi Bashari, Yonghoon Lee, Roy Lotan, Edgar Dobriban 等ICML 2026 · 被引用 5 次
