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Hauptverfasser: Wang, Shixuan, Zhang, Jing, Kang, Emily L., Zhang, Bin
Format: Preprint
Veröffentlicht: 2025
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Online-Zugang:https://arxiv.org/abs/2509.25688
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author Wang, Shixuan
Zhang, Jing
Kang, Emily L.
Zhang, Bin
author_facet Wang, Shixuan
Zhang, Jing
Kang, Emily L.
Zhang, Bin
contents Incorporating historical or real-world data into analyses of treatment effects for rare diseases has become increasingly popular. A major challenge, however, lies in determining the appropriate degree of congruence between historical and current data. In this study, we devote ourselves to the capacity of historical data in replicating the current data, and propose a new congruence measure/estimand $p_{CM}$. $p_{CM}$ quantifies the heterogeneity between two datasets following the idea of the marginal posterior predictive $p$-value, and its asymptotic properties were derived. Building upon $p_{CM}$, we develop the pointwise predictive density calibrated-power prior (PPD-CPP) to dynamically leverage historical information. PPD-CPP achieves the borrowing consistency and allows modeling the power parameter either as a fixed scalar or case-specific quantity informed by covariates. Simulation studies were conducted to demonstrate the performance of these methods and the methodology was illustrated using the Mother's Gift study and \textit{Ceriodaphnia dubia} toxicity test.
format Preprint
id arxiv_https___arxiv_org_abs_2509_25688
institution arXiv
publishDate 2025
record_format arxiv
spellingShingle PPD-CPP: Pointwise predictive density calibrated-power prior in dynamically borrowing historical information
Wang, Shixuan
Zhang, Jing
Kang, Emily L.
Zhang, Bin
Methodology
Applications
Incorporating historical or real-world data into analyses of treatment effects for rare diseases has become increasingly popular. A major challenge, however, lies in determining the appropriate degree of congruence between historical and current data. In this study, we devote ourselves to the capacity of historical data in replicating the current data, and propose a new congruence measure/estimand $p_{CM}$. $p_{CM}$ quantifies the heterogeneity between two datasets following the idea of the marginal posterior predictive $p$-value, and its asymptotic properties were derived. Building upon $p_{CM}$, we develop the pointwise predictive density calibrated-power prior (PPD-CPP) to dynamically leverage historical information. PPD-CPP achieves the borrowing consistency and allows modeling the power parameter either as a fixed scalar or case-specific quantity informed by covariates. Simulation studies were conducted to demonstrate the performance of these methods and the methodology was illustrated using the Mother's Gift study and \textit{Ceriodaphnia dubia} toxicity test.
title PPD-CPP: Pointwise predictive density calibrated-power prior in dynamically borrowing historical information
topic Methodology
Applications
url https://arxiv.org/abs/2509.25688