A practical guide to simulation for an adaptive trial design with a single interim analysis.

Area of Science:

• Clinical Trial Design
• Biostatistics
• Pharmaceutical Research

Background:

• Adaptive trial designs offer flexibility and efficiency over traditional fixed designs.
• Planned modifications based on accumulating data are key features of adaptive trials.
• Simulation is essential for adaptive trial design due to the inability of analytical formulae to account for data-driven adaptations.

Purpose of the Study:

• To demonstrate simulating data from a simple adaptive trial with a single interim analysis.
• To provide a foundational framework for making adaptive trial simulation more accessible.
• To aid statisticians and clinicians in designing adaptive trials.

Main Methods:

• Simulation of data from an adaptive trial with one interim analysis.
• Summarizing simulation results to balance type I error and power.
• Utilizing R and Stata programming languages with modular code for comprehensibility.

Main Results:

• The simulation results inform study design by balancing type I error and power.
• Expected sample size can be determined through simulation.
• The provided code facilitates the generation of various adaptive designs.

Conclusions:

• Simulation must be tailored to the specific design requirements of each adaptive trial.
• This tutorial offers a framework to enhance the accessibility of adaptive trial simulation.
• The goal is to empower both statisticians and clinicians in utilizing adaptive trial designs.