FDA Regulation of Off-Label Promotion: An Answer

Abstract

The FDA-approved labeling for prescription drugs contains only the information that FDA has found essential to uses for which the manufacturer has provided 'substantial evidence' of efficacy in accordance with strict regulatory standards. As a result, the labeling, while authoritative, is incomplete. Changes in the environment for prescribing decisions, including the increasing importance of comparative effective research (CER) information, make it timely to consider reforms to the current regulatory framework for drug labeling. This paper sets forth a proposal for legislative adjustments to the Federal Food, Drug, and Cosmetic Act to make the approved labeling for drugs more pertinent to clinical decisions by providing for the inclusion of a more heterogeneous mix of information from CER sources and clinical trials not meeting the traditional 'substantial evidence' standard - without abandoning a central role for FDA review. These reforms would not only clarify the rules governing manufacturer distribution of off-label use information but also enhance patient care by improving prescribing decisions.

Keywords: off-label promotion, substantial evidence, FDA regulation, food & drug