In general, the Draft Guidance is well written and provides very useful clarification of the implementation of FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (IND). However, we note that the guidance document does not offer adequate information on the appropriate design of an expanded access protocol. In fact, there are two statements in the guidance document (Line 56 and Lines 66-68) that present seemingly contradictory views about the collection of safety and effectiveness information under expanded access uses. Therefore, it would be very helpful to Sponsors for FDA to provide the basic design parameters for an acceptable expanded access use protocol that enables the appropriate collection of data, yet still qualifies as an expanded access protocol rather than a standard protocol. This is especially important as expanded access use of an investigational drug is sometimes the first time the drug is being used in a high-risk population, and data collection can yield new and vital information.
In addition to design parameters, BIO requests guidance on the execution of the expanded access IND or protocol, including responsibilities regarding safety reporting of serious adverse events and adverse events (for both Sponsors and Sponsor-Investigators) and requirements for submission of the patient outcome (Individual Patient IND or protocol) or the final clinical study report (Intermediate-size or access IND or protocol).
It is also unclear whether data generated from an expanded access IND should be included in a package insert, and if so, what information would need to be included.