This guidance is intended to assist sponsors in deciding whether a study of marketed drugs or biological products for treating cancer falls within the exemption under § 312.2(b)(1) (21 CFR312.2(b)(1)) from the general requirement to submit an investigational new drug application (IND).  The guidance discusses the Agency's current thinking on when studies of marketed cancer products are exempt from IND regulation based on a risk assessment.  The Agency hopes that clarifying its policy will help sponsors identify which studies are exempt, thus saving them from submitting unnecessary IND applications. 

This guidance revises the guidance of the same title published in September 2003.  In the September 2003 version, the Agency's final statement was that it believed that most randomized studies of a size that could support a labeling supplement would likely not be exempt from IND regulation under § 312.2(b)(1)(i), (ii).  This is because they would be intended to support approval of a new indication, a significant change in the product labeling, or a significant change in advertising.  Experience has shown that this interpretation was formulated too broadly and inappropriately referred to size alone.  The Agency has decided to revise this guidance by removing that statement (the last sentence in section V.B).  Whether a study could support a change in labeling is a complex determination, based on study design, size, and other factors.