A General Approach for Sample Size Calculation With Nonproportional Hazards and Cure Rates

With the ongoing advancements in cancer drug development, a subset of patients can live quite long, or are even considered cured in certain cancer types. Additionally, nonproportional hazards, such as delayed treatment effects and crossing hazards, are commonly observed in cancer clinical trials with immunotherapy. To address these challenges, various cure models have been proposed to integrate the cure rate into trial designs and accommodate delayed treatment effects. In this article, we introduce a unified approach for calculating sample sizes, taking into account different cure rate models and nonproportional hazards. Our approach supports both the traditional weighted logrank test and the Maxcombo test, which demonstrates robust performance under nonproportional hazards. Furthermore, we assess the accuracy of our sample size estimation through Monte Carlo simulations across various scenarios and compare our method with existing approaches. Several illustrative examples are provided to demonstrate the proposed method.

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Publication Details

Journal
Pharmaceutical Statistics
Published
2025-07-01
DOI
https://doi.org/10.1002/pst.70024
Citations
1
Primary Topic
Statistical Methods in Clinical Trials
Type
article
Field-Weighted Citation Impact
2.63
Controls
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article

A General Approach for Sample Size Calculation With Nonproportional Hazards and Cure Rates

Huan Cheng, Jianghua He, Xiaoyun Li
1 citations
Pharmaceutical Statistics
Statistical Methods in Clinical Trials
2.63
article

A General Approach for Sample Size Calculation With Nonproportional Hazards and Cure Rates

Huan Cheng, Jianghua He, Xiaoyun Li
article en
1 citations

Abstract

With the ongoing advancements in cancer drug development, a subset of patients can live quite long, or are even considered cured in certain cancer types. Additionally, nonproportional hazards, such as delayed treatment effects and crossing hazards, are commonly observed in cancer clinical trials with immunotherapy. To address these challenges, various cure models have been proposed to integrate the cure rate into trial designs and accommodate delayed treatment effects. In this article, we introduce a unified approach for calculating sample sizes, taking into account different cure rate models and nonproportional hazards. Our approach supports both the traditional weighted logrank test and the Maxcombo test, which demonstrates robust performance under nonproportional hazards. Furthermore, we assess the accuracy of our sample size estimation through Monte Carlo simulations across various scenarios and compare our method with existing approaches. Several illustrative examples are provided to demonstrate the proposed method.

Pharmaceutical StatisticsVol. 24(4)
HealthONE (US), University of Kansas Medical Center (US)
Good health and well-being
Openalex Percentile: Top 10%
Statistical Methods in Clinical Trials
2.63
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A General Approach for Sample Size Calculation With Nonproportional Hazards and Cure Rates — Huan Cheng, Jianghua He, et al. · Pharmaceutical Statistics (2025) | TGRS Research Map | TGRS