New active substances approved by EMA or FDA initially in 2014-2016, with approval date tracked until 2017

Objective: To compare review outcome alignment between European Medicines
Agency (EMA) and US Food and Drug Administration (FDA) for medicines
approved by both agencies in the time period 2014 to 2016. Design: Using
publicly available information from FDA and EMA websites, new active
substances (NASs) approved by each agency from 2014-2016 were identified
and their characteristics assessed. Divergences in regulatory outcomes for
simultaneous (within 91 days) submissions to both agencies were identified
and then examined for use of facilitated regulatory pathways and orphan
designations; submitted versus approved indications; and approval times.
Results: In 2014-2016, 115 NASs were approved by EMA or FDA or both;
74/115 were new chemical entities and 41 new biological/biotechnology
entities; 82/115 were approved by both agencies, 24 only by FDA and 9 only
by EMA. Simultaneous submission occurred for 52/115; 13/52 received
expedited review by both agencies and 18 only by FDA; 8/52 received
conditional approval from both agencies, 2/52 only from FDA and 1/52 only
from EMA; 17/52 were designated as orphans by both agencies and 10/52 by
FDA only; 31/52 indications were approved as submitted and 21 changed by
EMA and 29/46 were approved as submitted (6 not assessed) and 17/46
changed by FDA. Median FDA review timelines were 319 days compared with
409 days for EMA. Conclusions: There was general agreement in EMA/ FDA
conditional approvals. FDA used expedited pathways and orphan designation
more often than EMA, suggesting stricter EMA criteria or definitions for
these designations or less flexible processes. Despite consistency in
submitted indications, there was lack of concordance in approved
indications, which should be further investigated. FDA review times are
faster because of a wider range of expedited pathways and the two-step EMA
process; this may change with recent revisions to EMA accelerated
assessment guidelines and the launch of PRIME.