Published in:International Journal of Technology Assessment in Health Care
Abstract: In this scoping review detailing the challenges of assessing new technologies for use in children, authors Nora Hutchinson, Lauren Otterman, Elisa Koppelman, Barbara E. Bierer, and colleagues highlight the substantial difficulties in incorporating children within the population-wide health technology assessment (HTA) system, as well as the uncertainty accompanying pediatric HTAs due to data constraints, lack of guidance and/or variation in guidance, between HTA bodies.
Hutchinson N, Otterman L, Bain PA, Koppelman E, Bierer BE. A Scoping Review of Challenges in Pediatric Health Technology Assessments with a Focus on Pharmaceutical Interventions. International Journal of Technology Assessment in Health Care. Published online 2025:1-34. doi:10.1017/S0266462325103188
October 22: In the article “Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov,” published in Gene Therapy, co-authors Carolyn Chapman, Emina Berbic, Ava Glazier, and Barbara Bierer report a descriptive study of key characteristics of LTFU gene therapy study protocols registered in ClinicalTrials.gov. The analysis enabled a better understanding of how registered LTFU studies are currently designed and stimulated ideas for improvement. Most notably, the results suggest a lack of harmonization in how safety outcomes are monitored and reported across LTFU studies. Standardization and/or harmonization of data and outcome reporting may increase the scientific value of these studies.
Chapman, C.R., Glazier, A., Berbić, E. et al. Characteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov. Gene Ther (2025). https://doi.org/10.1038/s41434-025-00571-4
Please join us on Tuesday, November 18, 2025, from 12:00 pm – 1:00 pm ET for the second webinar in our Digital Twins and Synthetic Data series, focusing on practical use cases in clinical research. This session will explore how digital twins and synthetic data are being used to enhance the efficiency of clinical trials, including reducing the size of control arms, enhancing Bayesian statistical analysis, supporting single-arm trials, and optimizing the design of future trials. Dr. Daniele Bertolini of Unlearn.ai will share examples and reflect on lessons learned from deploying these tools across various therapeutic areas. The session will include a moderated discussion and audience Q&A.
This meeting is open to sponsors of the Bioethics Collaborative. For more information about the Bioethics Collaborative and how to become a sponsor, click here.
This meeting is open to sponsors of the Bioethics Collaborative. For more information about the Bioethics Collaborative and how to become a sponsor, click here.
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
