Choosing statistical methods for clinical trials

Abstract

Choosing the appropriate statistical methods is vital for ensuring the integrity, validity and efficiency of clinical trials. The selection of the methods to be used depends on trial-specific factors, including the nature of the data collected, hypothesis to be tested, sample size, duration, complexity and nature of the intervention. Once the overall methods are established, power analysis (balancing type I and II error risks, typically targeting 80–90% power to detect true treatment effects) needs to be considered. Variability, allocation ratio, study design and potential attrition also influence the methods selected. Incorporating these methodologies into clinical trial design ensures statistical rigour, resource efficiency and ethical integrity, enabling researchers to generate reliable, impactful evidence to guide clinical practice and advance medical knowledge.