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Enhancing Statistical Validity and Power in Hybrid Controlled Trials: A Randomization Inference Approach with Conformal Selective Borrowing

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arxiv 2410.11713 v3 pith:DIGIDZHD submitted 2024-10-15 stat.ME

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keywords externalcontrolsrandomizationinferencetrialserrorpowerapproach
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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.

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