This webinar focused on how to responsibly integrate AI into the design, conduct, and oversight of clinical research, introducing two rapidly evolving applications:
Digital twins – simulated models of individual patients designed to predict disease trajectories and treatment responses, with the potential to enhance statistical power, optimize design, and reduce the number of participants assigned to control arms.
Synthetic data – artificially generated datasets that mirror the statistical properties of verified clinical data, with potential use in trial design, conduct, analysis, and regulatory submissions.
Long-term follow-up (LTFU) studies are essential to assess the benefit–risk profile of gene therapies. Yet they are difficult to design and carry out, placing burdens on both participants and sponsors.
In September 2024, the MRCT Center convened a working group on LTFU studies for gene therapies. The group includes patients and representatives from pharmaceutical companies, clinical research organizations, academic medical centers, institutional review board oversight, and patient advocacy organizations.
This webinar will introduce a Toolkit for Supporting the Design and Conduct of Long-Term Follow-Up Studies for Gene Therapies, which is being released as a draft for public comment. Members of the working group will share their perspectives on the Toolkit’s development and discuss next steps. The session will conclude with audience questions and discussion.
Key Topics:
An overview of the draft Toolkit for Long-Term Follow-Up Studies in Gene Therapies
Comments provided to: U.S. Food and Drug Administration; FDA-2025-D-1797
Description: The Multi-Regional Clinical Trials Center of Brigham and Women’s Hospital and Harvard (MRCT Center) submitted public comments on the draft ICH E21 guidance regarding the inclusion of pregnant and breastfeeding women in clinical trials. The MRCT Center strongly supports the ethical inclusion of these historically understudied populations and emphasized the need for ethical and rigorous, well-designed research guided by participant and individual protections.
This webinar explores the MRCT Center’s work addressing the challenges of post-trial, continued access to investigational products. Post-trial access is a shared responsibility that should be planned before a trial begins. Even with careful planning, complex situations may arise, requiring sponsors to balance competing priorities and the needs of diverse stakeholders.
The MRCT Center’s Post-Trial Continued Access Task Force has developed principles, frameworks, and recommendations to guide equitable and fair decision-making about the continued provision of investigational medicines or the maintenance of significant-risk investigational devices after a trial ends.
The session includes a case study examining a scenario in which a trial’s primary endpoint is not met, but a subpopulation benefits, illustrating the difficult decisions sponsors and investigators must navigate in determining whether—and how—to provide post-trial, continued access.
Speakers:
Karla G. Childers, BA, MSJ, MSBE – Vice President, Bioethics, Policy & Partnerships, Johnson & Johnson
Brandy Ellis – Lived Experience Advocate
Ben Rotz, RPh – Associate Vice President, Global Medical Policy, Strategy, and Operations, Eli Lilly & Company
Moderated by: Sarah White, MPH, Executive Director, MRCT Center
In a Blood Advances article titled “The roadmap to integrate diversity, equity, and inclusion in hematology clinical trials: an American Society of Hematology initiative,” Barbara E. Bierer and co-authors outline a comprehensive strategy to address barriers to diversity, equity, and inclusion (DEI) in clinical research for classical hematologic diseases. These trials face unique challenges, including small sample sizes, methodological limitations, and underrepresentation of diverse populations. Through surveys and five international focus groups with patients, advocates, academia, regulators, industry, and research organizations, the team identified eight priority areas: (1) harmonizing demographic terminology, (2) engaging lived experience experts, (3) mitigating implicit bias, (4) strengthening the role of ethics committees and institutional review boards in upholding justice, (5) broadening eligibility criteria, (6) leveraging decentralized trial designs, (7) improving access to trial information, and (8) increasing engagement of community physicians. Implementing solutions in these areas will foster accessible, inclusive trials and generate representative data to improve research quality, regulatory decision-making, and patient care.
Alice Kuaban, Alysha K. Croker, Jeffrey Keefer, Leonard A. Valentino, Barbara E. Bierer, Stephen Boateng, Donna DiMichele, Patrick Fogarty, C. Michael Gibson, Anna M. Hood, Lloryn Hubbard, Antonella Isgrò, Karin Knobe, Leslie Lake, Iman Martin, Michel Reid, Jonathan C. Roberts, Wendy Tomlinson, Lanre Tunji-Ajayi, Harriette G.C. Van Spall, Caroline Voltz-Girolt, Allison P. Wheeler, Alan E. Mast, Stephanie Seremetis; The roadmap to integrate diversity, equity, and inclusion in hematology clinical trials: an American Society of Hematology initiative. Blood Adv 2025; 9 (4): 687–695. doi: https://doi.org/10.1182/bloodadvances.2024013945
In an Accountability in Research article titled “Disparate data retention standards in biomedical research”, Barbara Bierer and Carolyn Lye, Minal Caron, Lauren Walsh, and Mark Barnes discuss disparate data retention standards for biomedical research. The article summarizes the divergent data retention standards set forth by federal agencies, grant programs, and research institutions, as well as other applicable requirements under law, contract, and policy. The authors also discuss the importance of data retention in the context of research professionalism, data sharing efforts, intellectual property issues, and research integrity challenges, and provide recommendations for both institutions and applicable federal agencies to streamline and clarify data retention standards.
Lye, C. T., Caron, M. M., Walsh, L., Bierer, B. E., & Barnes, M. (2025). Disparate data retention standards in biomedical research. Accountability in Research, 1–28. https://doi.org/10.1080/08989621.2025.2543884
October is Health Literacy Month! Please join us on Thursday, October 30, from 12 – 1 PM ET for a webinar to explore teach-back (TB) within the clinical research context.
We begin with teach-back—its essential elements, how to use it, and why it is important to safe, high-quality, equitable health care and research. We then explore approaches for incorporating teach-back into different types of clinical research conversations.
The session will center on the importance of strong verbal communication skills and the ways teach-back can be used by study teams to foster clear, impactful, and empowering dialogue with patients, participants, and caregivers.
We will explore how study teams can simplify complex verbal information through plain language, assess and confirm understanding via teach-back throughout the recruiting and informed consent process, and support participant understanding throughout their clinical research experience. Registrants will receive tips and resources to support incorporating this best practice into their everyday clinical research processes.
Objectives:
Explain what teach-back is, its importance, and why to use it;
Describe approaches that support incorporating teach-back into clinical research conversations across the clinical trial life cycle;
Identify and access resources that explain how to use teach-back.
Please join us on Tuesday, September 30, 2025, from 11:00 am–12:30 pm ET for a webinar on the applications of AI digital twins and synthetic data to clinical trials. Digital twins refer to simulated models of individual patients designed to predict disease trajectories and/or treatment responses, offering the potential to enhance statistical power, optimize trial design, and limit the number of participants assigned to a control arm. Synthetic data are artificially generated datasets that mirror the statistical properties of verified clinical data. Both technologies are or are being developed for use in the design, conduct, and analysis of clinical trials, and with the intention of use for regulatory submissions. This webinar will serve as an introduction to these emerging technologies and a starting point for examining their implications, opportunities, and challenges in clinical research.
Key Topics:
Introduction to digital twins and synthetic data, including their core functions and how they differ from traditional statistical modeling approaches.
Exploration of ethical, statistical, and regulatory considerations for adopting digital twins and synthetic data in the design and conduct of clinical trials.
A panel discussion featuring expert perspectives on the evolving role of AI digital twins and synthetic data in clinical research.
Reproductive Health Considerations in Clinical Research
The Supreme Court’s June 2022 decision in Dobbs v. Jackson Women’s Health Organization introduced new challenges for the conduct of clinical trials involving people of reproductive potential, for the study of pregnancy-related conditions, and for participants in clinical research and research staff. The changing landscape of reproductive rights in the U.S. requires further consideration by clinical research stakeholders. Among other concerns, reproductive health intersects with clinical research with respect to the privacy of clinical trial participants.
The MRCT Center has started initial work to better understand whether and how reproductive privacy risks are addressed in study protocols, and particularly in informed consent documents. We are also exploring, through interviews with IRB chairs and research leaders across the country, existing approaches to reproductive health privacy protections and participant communication in single and multi-site trials.
Through this process, we are also working to identify recommendations for improved practices, which we plan to develop into a practical toolkit. The toolkit will include guidance documents on key privacy considerations, recommendations for notifying participants of privacy risks and communicating the importance of pregnancy reporting, best practices for addressing incidental pregnancies in research, and guidelines on medical record documentation. It will also explore the protections afforded by and the limits of Certificates of Confidentiality upon which the research community has relied.
OBJECTIVES
Evaluate current practices regarding reproductive privacy risks in clinical research informed consent forms and develop standardized guidance for addressing these concerns.
Develop resources to help research institutions mitigate participant privacy risks in the pregnancy testing and reporting processes, and to support research teams in communicating the risks to staff and participants.
Create standardized guidance for multi-site trial investigators to effectively manage the complexities of conducting research across jurisdictions.
KeY MILESTONES
January 2025: IRB protocol, “Pregnancy-Related Privacy Risks in Clinical Trials: Understanding Current Risks and Approaches,” approved
Dr. Carla Saenz, Regional Advisor for Bioethics at the Pan American Health Organization (PAHO), speaks with Willyanne DeCormier Plosky, MRCT Center Program Director, to unpack the clinical trial landscape in Latin America.
They discuss:
How demographics, healthcare infrastructure, and insurance models shape who can—and does—participate
Ethical and regulatory strategies to broaden inclusion
The influence of CIOMS and other international guidance on local trial ethics
Designing global studies that honor regional contexts without sacrificing scientific rigor
This episode highlights the critical need for equitable access and culturally responsive research in today’s global trial environment.
For more about the MRCT Center’s work on Global Representation in Clinical Trials, click here.
