Randomized controlled trials (RCTs) are the standard for providing confirmatory evidence on the efficacy of a new treatment. However, in a relevant proportion of marketing authorization applications the pivotal clinical data stems from single-arm trials (SATs). This is observed across different therapeutic areas, including for rare diseases.
The purpose of this reflection paper is to outline the current thinking about SATs that are submitted as pivotal evidence for establishing efficacy in marketing authorization applications.
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