Holding the FDA to Scientific Standards

Pilot studies represent a fundamental phase of the research process. The purpose of conducting a pilot study is to examine the feasibility of an approach that is intended for validity in a larger scale studies. Furthermore, pilot studies are the first step of the entire research protocol and are often a smaller-sized study assisting in planning and modification of the main study.¹

Researchers from Stanford University looked a 117-rejected US Food and Drug Administration (FDA) drug applications from 2013 to 2018 that went through multiple review cycles because of insufficient clinical efficacy.  In only 22 of these applications did the FDA determine an additional review was necessary because of concerns about the end-point, the clinical meaningfulness of the observed effect, and inconsistent results were common bases for the initial FDA rejection. In seven of 22 cases, the approval did not require new evidence but rather new interpretations of the original evidence. Importantly, the FDA decisions never cited reasoning used in previous decisions.

Besides the lack of inconsistency of evaluations, which begs the question of bias and potential conflict of interest, the point of relying on scientific standards for scientific integrity is of the upmost importance. Providing pilot evidence as stated above is the first step in the entire research protocol.  Pilot evidence issued in case law, piloting a jet, and in many aspects of our lives. Yet, the FDA does not have a database using sound scientific applications for prior approval.¹ Are we to believe that when evaluating a new drug application, that every application is so unique that prior decision making has no bearing on the current application.

The FDA is a critical component of the drug approval process throughout the US. Having this prestigious body use sound scientific methods and standards for approvals would teach all of us how science should be conducted.

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Reference:

Lancaster GA, Dodd S, Williamson PR. Design and analysis of pilot studies: recommendations for good practice. J Eval Clin Pract. 2004;10((2)):307–12