Expanded Access (Compassionate Use) for Drugs and Devices - The Multi-Regional Clinical Trials Center of Brigham and Women's Hospital and Harvard

Topic: Expanded Access (Compassionate Use) for Drugs and Devices

Abstract:

The MRCT Center of Brigham and Women’s Hospital and Harvard and Ropes & Gray, LLP are pleased to announce the first meeting of the Research, Development, & Regulatory Roundtable (R3) in 2024. This session will focus on issues surrounding regulatory pathways for Expanded Access and/or Compassionate Use of drugs and devices.

While clinical research continues to yield medical advancements, there remain no approved therapies for a multitude of serious and life-threatening disorders. In some instances, however, investigational treatments show promise while being evaluated in clinical studies, prompting both patients and physicians to seek access to those investigational treatments, even though such treatments have yet to clear the relevant regulatory checkpoints to be brought to market. Several major markets around the globe, however, provide pathways for patients to receive these as-yet-unapproved investigational treatments outside the context of a clinical trial. Depending on the specific market in question, these regulatory pathways go by different names and necessitate different requirements for patients and their treating clinicians both before and after gaining access to investigational treatments.

In this session, the speakers will deliver prepared remarks to deconvolute these pathways – the key terminology, the relevant statutory and regulatory language, and the key differences between one program and the next. While the primary foci of the R3 will remain the U.S. and European markets at this session, references to programs in non-U.S. and non-European markets will also be included. As always, this session will occur under Chatham House Rule and attendees are thus encouraged to participate freely in discussions of specific, real-world challenges with our invited experts.

Research, Development, and Regulatory Roundtable (R3) meetings are open to sponsors. To learn more about R3 and how to become a sponsor, click here.