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
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
“Defamation Claims Arising from Research Misconduct Cases: Best Practices for Institutions,” published in the Journal of Law, Medicine & Ethics, co-authored by Barbara Bierer and Mark Barnes, as well as Nathaniel Jaffe, Minal Caron, and Lauren Walsh, analyzes nine instances in which defamation suits were employed to obstruct misconduct investigations. It summarizes the legal bases of such claims and key defenses (e.g., Strategic Lawsuit Against Public Participation, truth, opinion), then distills institutional best practices—from calibrated public disclosures to stringent confidentiality protocols—to protect both scientific integrity and institutional resilience.
Jaffe N, Caron M, Walsh L, Bierer B, Barnes M. Defamation Claims Arising from Research Misconduct Cases: Best Practices for Institutions. Journal of Law, Medicine & Ethics. 2025;53(1):47-54. doi:10.1017/jme.2025.37
In the article “Managing Multi-Institutional Jurisdiction in Cases of Research Misconduct,” published in the Journal of Law, Medicine & Ethics, co-authors Mark Barnes and Barbara Bierer examine the persistent challenges of jurisdictional ambiguity when research misconduct spans multiple institutions. Together with Leslie Thornton and Devin Cohen, they outline key gaps in current regulatory guidance and propose a framework for collaborative resolution that safeguards fairness and research integrity. The article calls for clearer delineation of institutional responsibilities and improved mechanisms for inter-institutional coordination in misconduct investigations.
Thornton L, Cohen D, Barnes M, Bierer BE. Managing Multi-Institutional Jurisdiction in Cases of Research Misconduct. Journal of Law, Medicine & Ethics. 2025;53(1):41-46. doi:10.1017/jme.2025.36
In the article “Benefits that Offset Research Risks and Burdens are Qualitatively Different,” published in The American Journal of Bioethics, Barbara Bierer joins co-authors Luke Gelinas and Benjamin C. Silverman to examine how indirect benefits offered to research participants (e.g., payments, access to medicines) differ fundamentally from direct benefits that justify risk. The authors argue that these categories of benefit must be conceptually and ethically distinguished in research design and review. Their commentary challenges current interpretations of benefit-risk assessment and calls for greater nuance in ethical review frameworks.
Gelinas, L., Silverman, B. C., & Bierer, B. E. (2025). Benefits that Offset Research Risks and Burdens are Qualitatively Different. The American Journal of Bioethics, 25(5), 78–80. https://doi.org/10.1080/15265161.2025.2488293
In the Journal of Law, Medicine and Ethics, Barbara Bierer and co-authors Daniel Albert-Rozenberg, David Peloquin, Joseph Liss, and Erika Hanson published “Assessing Federal Policies to Reduce Economic Barriers to Clinical Trial Enrollment.” The article examines how the risk of losing access to means-tested programs—such as Medicaid, Supplemental Security Income (SSI), Supplemental Nutrition Assistance Program (SNAP), and Temporary Assistance for Needy Families (TANF)—can deter low-income Americans from participating in clinical trials. This barrier disproportionately impacts racial and ethnic minority groups, people with disabilities, elderly individuals, and rural populations, hindering efforts to achieve representative enrollment. The authors recommend that Congress exclude clinical trial payments from gross income and expand the participant compensation exclusions for means-tested programs established under the Ensuring Access to Clinical Trials Act of 2015.
Albert-Rozenberg D, Peloquin D, Liss J, Hanson E, Bierer BE. Assessing Federal Policies to Reduce Economic Barriers to Clinical Trial Enrollment. Journal of Law, Medicine & Ethics. Published online 2025:1-10. doi:10.1017/jme.2025.61
The authors press for follow‑up models that reduce patient and caregiver burden, improve retention, and leverage decentralized methods and real‑world evidence. Achieving this vision will require sustained collaboration across disciplines and meaningful engagement with patient communities.
Chapman et al., Patient-centered long-term follow-up for gene therapies aligns with ethics and science, Molecular Therapy (2025), https://doi.org/10.1016/j.ymthe.2025.04.040
Barbara Bierer and Willyanne DeCormier Plosky co-authored an article, led by David Gerber (UT Southwestern), in the Journal of Clinical Oncology titled, “Financial Toxicity in Cancer Clinical Trials: An Issue in Need of Clarity and Solutions.” This work was spearheaded by the Equitable Access to Clinical Trials (EACT) Project, hosted by Lungevity, of which the MRCT Center is one participating organization. The article illustrates some of the non-medical costs that cancer trial participants often pay out-of-pocket, such as an average of $600 US for travel, which are an additional stressor during a difficult time for participants and their families. The authors then detail financial support approaches to address these costs, and special considerations for sponsors, contract research organizations, and sites. Finally, the article highlights recently introduced federal legislation that could significantly ameliorate some of the barriers for sponsors trying to implement financial support approaches and for participants trying to access them.
