The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.
This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.
The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.
This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.
The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.
This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.
Topic 1:
Trump Administration Changes to Notice and Comment Rulemaking at the U.S. Department of Health and Human Services
The notice-and-comment rulemaking process is a cornerstone of U.S. administrative law under the Administrative Procedure Act (APA). The APA, however, exempts from notice-and-comment rulemaking certain regulations that pertain to “agency management or personnel or to public property, loans, grants, benefits, or contracts.” The APA also contains a “good cause” exception that permits rulemaking to occur absent notice and comment in certain situations. In 1970, the U.S. Department of Health and Human Services adopted a policy through memorandum referred to as the “Richardson Waiver” that required HHS as a matter of policy to follow voluntarily the notice-and-comment requirements of the APA even in cases in which the subject matter of the rule could fall within an APA exception. HHS Secretary Robert F. Kennedy rescinded the Richardson Waiver in March 2025.
This session will explore the history of the Richardson Waiver and what its rescission means for future rulemaking by HHS, including a discussion of which types of rules may fall within one of the APA exceptions.
Topic 2:
Certificates of Confidentiality: Background and Practical Implications
Certificates of Confidentiality are a tool created by Congress that prevent the disclosure of research records and biospecimens containing identifiable, sensitive information except when certain conditions set forth in statute are satisfied. Notably, CoCs prevent the disclosure of such research records and biospecimens in any Federal, State, or local civil, criminal, administrative, legislative, or other proceeding. CoCs are issued automatically for all National Institutes of Health-supported research and must be issued by the U.S. Department of Health and Human Services (HHS) for other federal government-funded research. CoCs can also be applied for by persons conducting privately-funded research and in such cases can be issued by HHS on a discretionary basis.
In this session we will explore the history of CoCs, the contours of the protections offered by CoCs, and certain unanswered questions that remain regarding the extent to which law enforcement and courts will respect the protections offered by CoCs.
The Research, Development, and Regulatory Roundtable (R3) is a forum to discuss pre-competitive issues in drug and device development, regulatory oversight of clinical trials, and human subjects research. Meetings convene policymakers, legal counsel, academicians, industry representatives, and global regulators. The R3 is a cooperative endeavor coordinated by the MRCT Center and Ropes & Gray LLP.
This hybrid meeting is open to sponsors of the Research, Development, and Regulatory Roundtable.
Abstract:Congressional Investigations into Research Activities
In recent years, congressional investigations of biopharma companies, research institutions, and universities have frequently involved issues related to clinical research. We anticipate this trend to continue or even accelerate under the new Congress. During this session, we will begin with an overview of congressional investigations, including: (1) Congress’s authority to investigate; (2) the types of information requests that Congress sends private parties; and (3) the mechanics of congressional hearings. We will then provide key takeaways from recent congressional investigations involving clinical research and predict areas of focus in the coming years, taking into account recent actions of the Trump Administration. We will end with a discussion of the various risks of such investigations and provide best practices to mitigate such risk and effectively respond to investigations. Amish Shah, counsel at Ropes & Gray and former Senior Associate White House Counsel and congressional investigator, will lead the session.
Update on DOJ’s Restrictions on Sharing Bulk Sensitive Personal Data with China and Other Countries of Concern
On January 8, 2025, the U.S. Department of Justice published in the Federal Register a Final Rule entitled “Preventing Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons.” Most provisions of the Final Rule will take effect on April 8, 2025, unless delayed by the administration of President Trump. The Final Rule imposes sweeping restrictions on providing access to health data, biometric data, human ‘omic data and biospecimens from which such data may be derived, as well as other categories of sensitive personal data, to the People’s Republic of China, certain other “countries of concern,” and “covered persons” that have ties to those countries. Notably, the Final Rule does not exempt de-identified, pseudonymized or anonymized data except in limited circumstances. During this session we will provide an overview of the Final Rule’s likely effect on research activities, including a discussion of the Rule’s research exemptions.
Recent Developments in FDA Guidance on Real World Evidence
Subject-Directed Recruitment Materials and Testimonials
Abstract: Real World Evidence and Emerging Trends in Clinical Trial Subject Recruitment
Real World Evidence
In recent years, real-world evidence (“RWE”) has increasingly been incorporated into product development programs, spurred by greater access to RWE, legislative changes, and significant policy development at FDA. The use of RWE raises novel opportunities, as well as new challenges for regulators, clinical trial sponsors, and the life sciences and healthcare industry to set common standards for RWE to ensure it can be relied upon with confidence in regulatory decision-making.
During 2023, FDA released two draft and three final guidance documents concerning RWE. These included the following:
Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products (Draft, February 2023)
Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products (Final, August 2023)
Use of Real-World Evidence to Support Regulatory Decision-Marking for Medical Devices (Draft, December 2023)
Data Standards for Drug and Biological Product Submissions Containing Real World Data (Final, December 2023)
Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products (Final, December 2023)
This session will provide an overview of key concepts from these guidance documents, focusing in particular on how they intersect with FDA’s existing regulations on clinical investigations and how they may change the regulatory landscape. In addition to focusing on these recent FDA guidance documents, the speakers will address the application of other U.S. regulatory regimes to RWE activities, including the Common Rule, the Information Blocking Rule, and HIPAA and other data privacy laws.
Clinical Trial Subject Recruitment Trends
Clinical trial sponsors are increasingly exploring different techniques to attract potential subjects to clinical trials, particularly in the context of rare disease studies for which subjects may be difficult to find. These techniques have included the following:
Use of testimonials featuring active clinical trial subjects in clinical trial advertisements
Inclusion of detailed scientific information gathered in pre-clinical or Phase 1 studies in subject-directed recruitment materials
Partnerships with electronic health record vendors to identify potential trial sites or subjects
Each of these techniques raise novel regulatory questions, including concerns about pre-approval promotion, data bias, need for institutional review board review and approval, and data privacy.
This session will analyze these trends and the attendant regulatory considerations for each.
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 “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
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.
Topics: 1) Update on the GDPR, including discussion of the EU/US data privacy framework; 2) Considerations for ensuring accurate clinical trials data
OPEN TO THE PUBLIC
GDPR: This session will involve a panel discussion exploring developments occurring in the past year related to the effect of the European Union’s privacy law, the General Data Protection Regulation (“GDPR”), on clinical research. GDPR was the topic of a one-day meeting held in conjunction with the MRCT Center annual meeting in December 2022, and the intent of this panel is to explore what has changed in the intervening 12-month period. Topics to be addressed will include the Trans-Atlantic Data Privacy Framework, the UK-US Data Bridge, ongoing litigation in the EU regarding the definition of anonymization, and the latest relevant European Data Protection Board guidance. The panel will include representatives from leading life sciences companies who advise on data privacy issues as well as a representative of the U.S. Department of Commerce who worked on the Trans-Atlantic Data Privacy Framework.
Ensuring Integrity of Clinical Trial Data:Clinical trials are most often conducted at sites contracted to perform clinical trials. Historically, trials have most often been sited at organized clinics or hospitals, which have reliable systems of ensuring quality of care and creating and maintaining accurate medical records. Yet in recent years, clinical trials have been sited at stand-alone, for-profit research sites that lack the quality assurance infrastructure of clinics or even physician group practices; and in the wake of COVID, clinical trials have often become “remote” or “decentralized.” This session will explore the challenges in this new clinical trial environment to ensuring accurate recording and retention of data, and whether and how monitoring processes should be revised and enhanced.
This meeting is part of the MRCT Center’s 2023 Annual Meeting in Boston on December 13-14 and is open to all registrants at no cost. Guests are welcome to attend the entire program or individual sessions. While in-person attendance is encouraged, virtual participation is welcome.
