Improving Expanded Access in the United States: The Role of the Institutional Review Board.

Abstract

The FDA allows patients with a serious or immediately life-threatening illness to use investigational medical products outside of clinical trials through its "expanded access" program. In response to criticism that the process to apply for expanded access is too onerous, numerous changes have been made over the last few years. These have been largely focused on the FDA and the pharmaceutical industry, while institutional review boards (IRBs)-which must approve expanded access protocols, except in emergencies when there is not time to do so-have remained relatively unstudied. We conducted a pilot study to review a sample of publicly available IRB policies from the United States to investigate how these entities handle expanded access. We performed an online search to find publicly available policies for IRBs operating in the United States, utilizing a convenience sampling strategy, selecting the first 100 eligible policies we identified. Of the 95 policies reviewed, the majority (92.6%, n = 88) contained language referencing nonemergency expanded access and/or expanded access for emergency requests for a single patient. Of these 88 policies, 11.4% (n = 19) did not explicitly specify detailed procedures for handling nonemergency single-patient expanded access requests. Of the 88 policies that mentioned expanded access in nonemergency situations, 11.5% did not explicitly specify whether full IRB review was required, as was the rule at that time. There was considerable variation in other aspects of these policies, including charging patients for use of investigational products and the use of data from expanded access. Based on the findings of our pilot, IRB policies on expanded access vary considerably. It is often difficult to find, interpret, and understand IRB policies on expanded access. Further research is needed to determine if and to what extent this negatively impacts patient access to investigational products outside of clinical trials.