Clinical Trials in the 21st Century — Promising Avenues for Better Studies

The Covid-19 pandemic has reminded us of the critical role that well-designed and well-conducted randomized controlled trials (RCTs) play in the generation of clinical knowledge. The pandemic has further confirmed the efficiencies of master protocols that simultaneously evaluate multiple therapies. Yet it also identified numerous limitations to the current landscape of clinical trials, including racial disproportionality of study populations, an inadequate number of larger, well-powered trials, an inability to obtain results fast enough, and controversies about meaningful outcomes and effect sizes. While the pandemic has exposed these limitations, they are neither new nor limited to the study of Covid-19 treatments and vaccines. Indeed, the mantra about the need for better, faster, and cheaper RCTs has been repeated for years, yet limited progress has been achieved to meet these goals. In the following sections, we explore a handful of these issues. The larger series that this article introduces will address them in more detail, explaining the consequences of these problems and proposing some solutions.