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Pragmatic trials aim toassess intervention efficacy in real-world settings, contrasting with explanatory trials conducted in controlled conditions. In aging research, pragmatic trials are important designs for the obtention of real-world evidence in elderly populations, often underrepresented in trials. In this review, we discuss statistical considerations from a frequentist approach to the design and analysis of pragmatic trials. Cluster randomization necessitates careful consideration of sample size calculation and analysis methods, especially regarding missing data and outcome variables. Mixed effects models and Generalized Estimating Equations (GEE) are both recommended for analysis, with tools available for sample size estimation. Multi-arm studies pose challenges in sample size calculation, requiring adjustment for design effects and consideration of multiple comparison correction methods. Secondary analyses are common but require caution due to reduced statistical power. Safety data collection methods should balance pragmatism and data quality. Overall, understanding statistical considerations is crucial for designing rigorous pragmatic trials evaluating interventions in elderly populations under real-world conditions.
Kammar-García et al. (Tue,) studied this question.