Statistical Issues in Biologics Submissions to the FDA

Abstract

A major activity of the Biostatistics Branch in the Food and Drug Administration's (FDA's) Center for Biologics Evaluation and Research (CBER) is the review of investigational new drug (IND) proposals and product license applications (PLAs) that are central to the drug/biologics approval and licensing process. This paper summarizes the recent experience of members of the branch in the statistical review of INDs and PLAs, with focus on design and analysis issues that have either expedited or delayed the review of these documents. The paper places emphasis on Phase III clinical trials, and includes a list of design and analysis items that a statistician may wish to review in preparing statistical documentation for submission to CBER.