Work

Analyzing safety data for two-stage randomization designs

Year: 2025

Type: article

Abstract: 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 two-stage 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.

Source: Frontiers in Applied Mathematics and Statistics

DOI: 10.3389/fams.2025.1519056

Authors: Sifiso Vilakati, Giuliana Cortese

Institutions: University of the Free State, University of Padua

Language: English

Topic: Statistical Methods in Clinical Trials

Subfield: Statistics and Probability

Field: Mathematics

Domain: Physical Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $1,418