This document is intended to provide guidance to applicants planning to submit new drug applications (NDAs), biologics license applications (BLAs), or supplements to NDAs or BLAs that require the demonstration of substantial evidence of effectiveness.
This guidance revises the draft guidance for industry of the same name issued in December 2019.
When finalized, this guidance will replace the 1998 guidance for industry Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products (May 1998 )
The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a revised draft guidance for industry titled “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products.”
FDA has revised and is reissuing this draft guidance in response to public comments and to changes in drug development by focusing on generating rigorous scientific evidence in the most efficient manner.
Advances in our understanding of biological processes and the increasing availability of high-quality data have transformed the evidentiary landscape for drug development.
Given these advances, the draft guidance discusses the many factors that can impact the strength of evidence of effectiveness for a drug and clarifies how sponsors can rely on one adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the substantial evidence of effectiveness standard.
When final, this guidance will replace the 1998 guidance titled “Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products.