Clinical Pharmacology, Pharmacy, and Environmental Medicine, Department of Public Health, University of Southern Denmark, Odense, Denmark.
Department of Regional Health Research, University of Southern Denmark, Odense, Denmark.
Clin Transl Sci. 2022 Aug;15(8):1856-1866. doi: 10.1111/cts.13305. Epub 2022 Jun 1.
Pharmacokinetics is the cornerstone of understanding drug absorption, distribution, metabolism, and elimination. It is also the key to describing variability in drug response caused by drug-drug interactions (DDIs), pharmacogenetics, impaired kidney and liver function, etc. This tutorial aims to provide a guideline and step-by-step tutorial on essential considerations when designing clinical pharmacokinetic studies and reporting results. This includes a comprehensive guide on how to conduct the statistical analysis and a complete code for the statistical software R. As an example, we created a mock dataset simulating a clinical pharmacokinetic DDI study with 12 subjects who were administered 2 mg oral midazolam with and without an inducer of cytochrome P450 3A. We provide a step-by-step guide to the statistical analysis of this clinical pharmacokinetic study, including sample size/power calculation, descriptive statistics, noncompartmental analyses, and hypothesis testing. The different analyses and parameters are described in detail, and we provide a complete R code ready to use in supplementary files. Finally, we discuss important considerations when designing and reporting clinical pharmacokinetic studies. The scope of this tutorial is not limited to DDI studies, and with minor adjustments, it applies to all types of clinical pharmacokinetic studies. This work was done by early career researchers for early career researchers. We hope this tutorial may help early career researchers when getting started on their own pharmacokinetic studies. We encourage you to use this as an inspiration and starting point and continuously evolve your statistical skills.
药代动力学是理解药物吸收、分布、代谢和消除的基础。它也是描述药物相互作用(DDI)、药物遗传学、肾功能和肝功能受损等引起的药物反应变异性的关键。本教程旨在为设计临床药代动力学研究和报告结果时的基本考虑因素提供指导和分步教程。这包括如何进行统计分析的综合指南和统计软件 R 的完整代码。作为一个示例,我们创建了一个模拟临床药代动力学 DDI 研究的模拟数据集,该研究涉及 12 名受试者,他们分别服用 2mg 口服咪达唑仑和不服用细胞色素 P450 3A 诱导剂。我们提供了对该临床药代动力学研究进行统计分析的分步指南,包括样本量/功效计算、描述性统计、非房室分析和假设检验。不同的分析和参数都有详细描述,并在补充文件中提供了一个完整的 R 代码,可直接使用。最后,我们讨论了设计和报告临床药代动力学研究时的重要考虑因素。本教程的范围不仅限于 DDI 研究,只需进行微小调整,即可适用于所有类型的临床药代动力学研究。这项工作是由早期职业研究人员为早期职业研究人员完成的。我们希望本教程可以帮助早期职业研究人员在开始自己的药代动力学研究时提供帮助。我们鼓励您将其用作灵感和起点,并不断发展您的统计技能。