In 2016, the FDA passed the 21st Century Cures Act, which endorses the collection of real world data (RWD), defined as any non-randomized controlled 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.
Agenda
- Define RWD, and discuss why it is collected and when IRBs may encounter proposals to collect it
- Define EA (for both single patients and groups), and discuss how Expanded Access protocols differ from protocols for observational studies or clinical trials
- Discuss opportunities and limitations for collecting RWD from EA, as well as potential benefits and harms of RWD collection from EA from patient, industry, and biostatistics perspectives
- Discuss ways to ensure that protocols collecting RWE from EA meet ethical and research regulatory standards and best practices for IRBs reviewing EA protocols that call for the collection of RWD
By the end of this program, you should be able to:
- Define real world data (RWD), including why it is collected and when proposals for its collection are likely to come before an IRB
- Define Expanded Access (EA), including how EA protocols differ from protocols for observational studies or clinical trials
- Identify opportunities and limitations for collecting RWD from EA, as well as potential benefits and harms of RWD collection from EA from the patient, industry, and biostatistics perspectives
- Outline best practices for ensuring that protocols collecting RWD from EA meet ethical and research regulatory standards
This program is geared toward IRB members, HRPP/IRB staff, institutional oversight personnel, and researchers who want to understand more about EA, real world data (RWD) generated from EA, and how to best evaluate EA protocols that aim to collect RWD from patients for regulatory purposes.
Continuing Education
Webinar participants holding the Certified IRB Professional (CIP®) credential may apply 1.25 continuing education credits towards CIP recertification. Learn More
90-Day Access Interval
Access to live content will be available for 90 days after the recording is made available. After 90 days, access to the content will be removed, but users will retain access to any certificates of attendance earned. To earn a certificate, one must complete all required sections and complete the certificate section within the 90-day access period. After access has expired, users can purchase an additional 90 days of access.