Impact of Expanded Access on FDA Regulatory Action and Product Labeling.

Abstract

The purpose of this study is to address concerns that expanded access may negatively impact the ultimate regulatory action and product labeling for new drugs. We performed queries of FDA's Center for Drug Evaluation and Research (CDER) document tracking system to determine the effect of expanded access on FDA's regulatory decision making from 2010 through 2016. We also examined product labeling to determine whether safety events occurring under expanded access had an adverse effect on the approved product labeling. There were 321 regulatory decisions made by FDA, with 28% of the drugs having prior expanded access. The approval rate for drugs with expanded access (84%) was higher than those that did not (76%). None of the negative regulatory marketing decisions were based on the adverse experiences reported under expanded access. The vast majority of deaths and serious adverse events that occurred under expanded access were not interpreted by FDA to be due to the investigational drug and did not affect product labeling. There was only 1 instance, a drug-drug interaction, for which safety events occurring during expanded access alone lead to potentially adverse product labeling. There was no instance in which expanded access lead to a negative regulatory decision regarding a drug application, and there was only 1 instance that safety events under expanded access had a potentially negative effect on product labeling. Concern that expanded access will have a negative impact on drug development and review is not based on the evidence and is unwarranted.