FDA Provides Guidance on Expanded Access Program
(Thursday, November 3, 2022)
There are two ways patients can get access to experimental drugs outside of participating in a clinical trial: The Right-To-Try (RTT) access or the Expanded Access. FDA prefers patients to use the expanded access program over RTT since that way FDA can better monitor the non-clinical trial use of experimental treatments. FDA’s latest guidance on this topic presents various scenarios in a Q&A format to help sponsors to provide their experimental drugs under expanded access programs. The biggest benefit of participating in a clinical trial for a patient is getting access to a new experimental treatment not available otherwise. This access may be important for patients with serious diseases who have few or no FDA-approved treatment options. However, not all such patients will meet the criteria for enrollment in an ongoing clinical trial. In such cases, he/she may get access to the experimental drug via the expanded access program, also called the Compassionate Use or Emergency Use treatment. There are several misperceptions about how such expanded access programs must be run in compliance with FDA requirements. The biggest confusion is about the scope of the expanded access program. Traditionally, expanded access was designed to help individual patients or small groups of patients with similar situation, for a short-term only. Also, such programs must be designed to assure patient safety and accountability by involving oversight from independent review boards (IRBs) and FDA. The new FDA guidance documents describes the requirements and limitations of various scenarios to emphasize the above tenets. For example, the expanded access program must assure that the ongoing clinical trials with the same treatment being provided to patients outside of the clinical trial, does not adversely affect the conduct of the ongoing clinical trial. Also, it is preferred that the manufacturer would have an active IND prior to offering expanded access and all expanded access treatments would at least have IRB approval and in most cases FDA submissions about such use where FDA gets about 30 calendar days to object to such an expanded access. The guidance document provides several suggestions and templates for the manufacturers to answer requests for the expanded access. Unlike the RTT program, where FDA has practically no oversight, FDA plays an integral role in the expanded access program, which is why FDA prefers the latter over the former. Manufacturers cannot be forced to provide their IND drug to the patients or physicians, and most would prefer to stay compliant with FDA expectations. This guidance provides manufacturers with a formal FDA document to help create expanded access program and a reference to share with the requesters of their drugs.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC