In 2016, the FDA passed the 21st Century Cures Act, which endorses the collection of real world data (RWD), defined as any non-randomized control trial (RTC) data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. These sources include expanded access (EA) protocols. At this time, there is little regulatory guidance on the collection of RWD in this new and rapidly evolving area; however, data show that EA is becoming more common, as is the collection of RWD from EA.
Expanded access refers to the use of an investigational medical product for a seriously or terminally ill patient without either approved or research options (also known as “compassionate use”). EA is intended as treatment, not research, yet IRBs are involved in reviewing such proposals to ensure appropriate informed consent for use of an unproven intervention. The treatment/research distinction that is already blurry in EA becomes even more tenuous when RWD are being collected, especially when these are intended for use in a regulatory submission. It can be difficult to determine how much and what data should be collected from individuals who are patients, not research subjects; how long follow-up should be, and what is reasonable to expect of clinicians.
In this webinar, we will discuss key ethical and regulatory considerations and best practices for IRBs when reviewing EA protocols, both for single patients and groups, that aim to collect RWD for regulatory use. We’ll share some cautionary tales that illustrate these challenges and considerations, and provide useful guidelines and tips to use at your institutions. Time will be provided for a brief Q&A with the speakers at the end of the session.