Rare events meta-analyses of randomized controlled trials (RCTs) are often underpowered because the outcomes are infrequent. Real-world evidence (RWE) from non-randomized studies may provide valuable complementary evidence about the effects of rare events, and there is growing interest in including such evidence in the decision-making process.…

This article discusses the nonuse, misuse, and proper use of pilot studies in experimental evaluation research. The authors first show that there is little theoretical, practical, or empirical guidance available to researchers who seek to incorporate pilot studies into experimental evaluation research designs. The authors then discuss how pilot…