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ORIGINAL RESEARCH article
Front. Appl. Math. Stat.
Sec. Statistics and Probability
Volume 11 - 2025 | doi: 10.3389/fams.2025.1519056
ANALYZING SAFETY DATA FOR TWO-STAGE RANDOMIZATION DESIGNS
Provisionally accepted- 1 University of the Free State, Bloemfontein, South Africa
- 2 University of Padua, Padua, Veneto, Italy
Ensuring patient safety is paramount in the development of new pharmaceutical products, which, while beneficial, can also pose potential risks. Traditional methods for analyzing safety data have been limited to single-stage randomization clinical trials. However, no existing methodology addresses the complexities of two-stage randomization designs with survival endpoints. This paper introduces a novel methodology utilizing inverse probability weights to analyze safety data in twostage randomization designs. Our approach is applied to data from a leukemia clinical trial, the use of the weighted Aalen-Johansen estimator is recommended while the use of the weighted Kaplan-Meier is discouraged. This advancement provides a crucial tool for enhancing patient safety in complex clinical trial designs.
Keywords: adverse events, Aalen-Johansen estimator, Inverse probability weights, Toxicity, Two-stage randomization designs
Received: 29 Oct 2024; Accepted: 03 Mar 2025.
Copyright: © 2025 Vilakati and Cortese. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Giuliana Cortese, University of Padua, Padua, 35122, Veneto, Italy
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