A Pathway to Improved Prospective Observational Post-Authorization Safety Studies

Randomized controlled trials (RCTs) are the gold standard for assessing the efficacy of drugs but not necessarily so for drug safety where inadequate power to detect either multiple or rare adverse events is a major handicap. Furthermore, the conditions under which drugs are approved for market use are often different from the settings in actual use. Indeed, with their control mechanisms, trials are by design largely inadequate for the identification of potential safety signals, especially of the rare type, hence the value of post-marketing surveillance and risk management plan-based activities.