Tutorial: Statistical analysis and reporting of clinical pharmacokinetic studies

Area of Science:

• Pharmacology
• Clinical Research
• Statistical Analysis

Background:

• Pharmacokinetics (PK) is crucial for understanding drug behavior and response variability.
• Factors like drug-drug interactions (DDIs), pharmacogenetics, and organ function impact PK.
• A need exists for accessible guidelines on designing and analyzing clinical PK studies.

Purpose of the Study:

• To provide a comprehensive guideline and tutorial for designing clinical pharmacokinetic studies.
• To offer a step-by-step statistical analysis guide, including R code.
• To assist early career researchers in PK study design and reporting.

Main Methods:

• Development of a mock dataset simulating a DDI study.
• Detailed explanation of statistical analyses: sample size/power calculation, descriptive statistics, noncompartmental analysis, and hypothesis testing.
• Provision of complete R code for statistical analysis.

Main Results:

• A simulated DDI study dataset was created for illustrative purposes.
• Step-by-step statistical analysis procedures for clinical PK studies are detailed.
• A functional R code package is provided for practical application.

Conclusions:

• This tutorial offers a practical framework for designing and reporting clinical pharmacokinetic studies.
• The provided R code and methodology are adaptable for various PK study types beyond DDIs.
• It serves as a valuable resource for early career researchers entering the field of clinical pharmacokinetics.