Researchers, clinicians, policymakers and patients are increasingly
interested in questions about therapeutic interventions that are difficult or costly
to answer with traditional, free-standing, parallel-group randomized controlled
trials (RCTs). Examples include scenarios in which there is a desire to compare
multiple interventions, to generate separate effect estimates across subgroups of
patients with distinct but related conditions or clinical features, or to minimize
downtime between trials. In response, researchers have proposed new RCT designs
such as adaptive platform trials (APTs), which are able to study multiple
interventions in a disease or condition in a perpetual manner, with interventions
entering and leaving the platform on the basis of a predefined decision algorithm.
APTs offer innovations that could reshape clinical trials, and several APTs are now
funded in various disease areas. With the aim of facilitating the use of APTs, here
we review common features and issues that arise with such trials, and offer
recommendations to promote best practices in their design, conduct, oversight
and reporting.
