Lack of robust post-approval studies under FDA system

Review of drugs approved on the basis of limited evidence concludes that stricter rules are needed for post-approval studies.

US Food and Drug Administration entrance

The US Food and Drug Administration (FDA) has increasingly adopted the approach of approving novel drugs on the basis of limited evidence, enabling them to come to the market quicker, on the understanding that post-approval assessments will be carried out.

Researchers systematically reviewed the evidence on all 117 drugs approved for 123 indications between 2005 and 2012 by the FDA on the basis of a single pivotal trial and/or using surrogate disease markers as primary endpoints.

They found that, after a median of 5.5 years post-approval, fewer than one-third of those approved based on a single trial had a published randomised clinical trial showing superior efficacy versus a comparator. And less than 1 in 10 approved using surrogate endpoints had a subsequent trial demonstrating efficacy on the basis of clinical outcomes.

Reporting in The BMJ
(online, 3 May 2017), the researchers say that stricter rules for post-approval studies should be enforced to make sure high-quality evidence is available to inform clinical practice.


[1] Pease A, Krumholz H, Hines H et al. Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review. BMJ 2017;357:j1680. doi: 10.1136/bmj.j1680

Last updated
Clinical Pharmacist, CP June 2017 online;9(6):DOI:10.1211/PJ.2017.20203062

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