Focus Area: Clinical Trials & Research

Description: The FDA has recently issued several guidance documents as part of the Agency’s ongoing efforts to modernize its policies for the conduct of clinical trials.  These include:

• A final guidance on “Conducting Clinical Trials With Decentralized Elements” (the “DCT Final Guidance”);
• A draft guidance on “Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice” (the “Routine Clinical Practice Trials Draft Guidance”);
• A draft guidance on “Considerations for Generating Clinical Evidence from Oncology Multiregional Clinical Development Programs” (the “Oncology Multiregional Clinical Development Programs Guidance”); and
• A draft guidance on “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies” (the “DAP Draft Guidance”).

During this R3 session, we will provide an overview of each of these guidance documents, discuss thematic intersections between them, and address practical implications for study sponsors, investigators and sites.  With respect to the DCT Final Guidance, we will also look at how the final guidance addressed the MRCT Center’s extensive comments on the draft guidance.

This meeting is open to sponsors of the Research, Development, & Regulatory Roundtable. To learn about how to become a sponsor, please contact us at mrct@bwh.harvard.edu

Abstract:

Laboratory Developed Tests

The scope of FDA’s authority over laboratory-developed tests (“LDTs”) has long been a source of controversy and debate among government and industry stakeholders. In May 2024, FDA issued a final rule that affirms FDA’s position that LDTs are in vitro diagnostic products (“IVDs”) regulated as medical devices under the Federal Food, Drug, and Cosmetic Act (“FDCA”). In conjunction with this rule, FDA intends to phase out the enforcement discretion policy it has historically applied to most LDTs. This session will summarize the FDA’s final rule and phaseout policy, analyze the impact on clinical research (including the use of LDTs in pharmaceutical trials), and assess the prospects for ongoing litigation challenging FDA’s rule and potential LDT-related legislation from Congress.

Limitations on Sharing Data with China and Other Countries of Concern

Over the past several months there have been significant U.S. executive and congressional actions related to restricting transactions, data transfers, and certain types of contracts with China, other countries of concern (e.g., Russia), and individuals and businesses connected with these countries. Principally, this includes the enactment in April 2024 of the Protecting Americans’ Data from Foreign Adversaries Act, President Biden’s Executive Order on bulk sensitive personal data transfers and the accompanying U.S. Department of Justice rulemaking activities, and the continued consideration by Congress of the BIOSECURE Act. In this session, we will walk through these different pieces of legislation and proposed regulations, focusing on the implications they each may hold for the clinical research enterprise.

This is a meeting of The Research, Development, and Regulatory Roundtable (R3) and is open to sponsors. Click here to learn more about the Research, Development, and Regulatory Roundtable (R3) and how to become a sponsor.

Topic: (1) Laboratory Developed Tests; (2) Limitations on Sharing Data with China and Other Countries of Concern

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.