Characteristics of single pivotal trials supporting regulatory approvals of novel non‐orphan, non‐oncology drugs in EU and US from 2012 to 2016

Abstract

For regulatory approval of a new medicine, the gold standard for demonstration of efficacy has traditionally been a minimum of two positive, adequate, and well‐controlled clinical trials. Nevertheless, drugs to treat cancer and rare diseases are usually approved based on a single and often uncontrolled pivotal trial. In contrast, little is known about single pivotal trial approvals for non‐orphan, non‐oncology drugs. Between 2012 and 2016, 23 novel therapeutic drugs were approved by the FDA and/or the EMA for 27 non‐orphan, non‐oncology indications each based on a single pivotal trial. While there was considerable variation in the nature and strength of the efficacy evidence supporting these drug approvals, the majority (85%) of the pivotal trials were randomized and controlled. For all superiority trials, the primary outcome was met with a statistical significance of p ≤ 0.005. Most approvals were supported by additional efficacy data from non‐pivotal studies.

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