Post-approval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review


3 May 2017 - Between 2005 and 2012 the FDA approved 117 novel drugs for 123 indications on the basis of a single pivotal trial, pivotal trials that used surrogate markers of disease, or both (single surrogate trials).

Most post-approval studies used active comparators—67 of 77 (87.0%) indications approved on the basis of single pivotal trials, 365 of 554 (65.9%) approvals based on surrogate marker trials, and 100 of 127 (78.7%) approvals based on single surrogate trials—and examined surrogate markers of efficacy as primary endpoints—51 of 77 (66.2%), 512 of 554 (92.4%), and 110 of 127 (86.6%), respectively. 

Overall, no post-approval studies were identified for 43 of the 123 (35.0%) approved indications.

Read BMJ research article

Michael Wonder

Posted by:

Michael Wonder