Across 3 video-based modules, paired with interactive case studies and activities, learners will gain practical, in-depth guidance on providing ethical oversight of health-related research involving human participants. By the end of the course, learners will be able to explain how a Research Ethics Committee (REC) provides oversight from initial review through study closure; articulate why robust Standard Operating Procedures (SOPs) are essential to REC operations; describe the roles and responsibilities of other stakeholders involved in clinical research oversight; and identify methods to assess and strengthen REC operations.
Presented on: October 22, 2025, at the MRCT Center Annual Symposium
Long-Term Follow-Up (LTFU) studies are critical for understanding the long-term safety and risk/benefit profile of innovative cell and gene therapies. Study participants, patients, treating clinicians, companies, regulators, payers, and society-at-large all derive different value from LTFU studies. However, these studies present unique challenges due to their unprecedented duration. For sponsors, they are challenging and expensive to design, conduct, and operationalize. For patients, LTFU participation can be burdensome in terms of time, expense, and opportunity costs.
In this recorded panel, which was held during the MRCT Center’s Annual Symposium in October 2025, Dr. Carolyn Chapman describes the aims of the Center’s LTFU Working Group, which was launched in September 2024 and benefited from the perspectives of 25 members from outside the Center. Dr. Chapman also previews the release of the LTFU Toolkit, which was collaboratively developed by the Working Group. The Toolkit offers practical guidance to support best practices for LTFU studies for both investigational and approved gene therapies.
In this session, Dr. Chapman leads a discussion with three distinguished industry leaders on a variety of issues addressed in the LTFU Toolkit. These include integrating decentralized elements to reduce burden, refining endpoint selection, harmonizing data for interoperability across LTFU studies, and minimizing loss to follow-up through participant engagement and clear communication. They also highlight the need for flexibility in LTFU study designs to meet evolving patient needs, and the importance of keeping new and current participants informed about protocol changes and any key findings. Looking ahead, the group calls for ongoing dialogue on data harmonization and sharing, results transparency, and innovative approaches to streamline the collection of long-term safety information.
𝗠𝗼𝗱𝗲𝗿𝗮𝘁𝗼𝗿: Carolyn Riley Chapman, PhD, MS – Lead Investigator, Brigham and Women’s Hospital; Member of the Faculty, Harvard Medical School
Panelists:
Pamela Tenaerts, MD, MBA, Chief Medical Officer, Medable
Long-term follow-up (LTFU) studies of gene therapy (GT) recipients are crucial for understanding the overall benefit-risk profile of these innovative products. However, LTFU studies are challenging to design, conduct, and execute, and pose significant burdens on both patients and sponsors.
The MRCT Center CGT Toolkit is comprehensive and provides background information, practical resources, and recommendations to support best practices for LTFU studies for both investigational and approved gene therapies—balancing the generation of critical long-term safety and efficacy data with the need to reduce burdens placed on participants, caregivers, sponsors, and investigators.
Compared to the draft version, the updated Toolkit v2.0 includes two new resources, an Executive Summary and a Patient Resource, which have also been released as standalone versions. Additions included new and/or revised charts, tables, and resources. The Toolkit explores ideas for how LTFU studies could be improved in the future, raising questions that the field should discuss and address.
The Toolkit enables easy navigation to various sections and subsections via multiple clickable, interactive toolbars. The sections are as follows, with the core elements in bold font:
The Toolkit incorporates several interactive features designed to support intuitive navigation and ease of use:
Clickable Table of Contents for rapid access to major sections and subsections.
Right-hand vertical navigation bar on every page, enabling quick movement between tools within the document.
Interactive table of LTFU study types, mirrored by color-coded tabs that remain clickable throughout the associated pages.
Secondary navigation bar at the top of each page within the Considerations & Recommendations section. This feature highlights your location within the nine subsections, allows you to jump directly between subsections by clicking the dots, and includes a grey diamond icon that returns you to the full list of subsections.
Comments provided to: TEHDAS2, coordinated by the Finnish Innovation Fund SITRA
Description:The MRCT Center and Vivli co-host an ongoing, invite-only forum focused on the European Health Data Space (EHDS), bringing together stakeholders to track developments and coordinate responses. The MRCT Center submitted responses to three TEHDAS2 guidance documents offered for public consultation to Health Data Access Bodies:
This meeting was presented to the Bioethics Collaborative. The Bioethics Collaborative is a forum to propose, share, and discuss ethical challenges in multi-national clinical trials. Meetings convene individuals from academia, industry, patient/participant groups, ethics committees, government, and others.
Abstract: Since funding and other resources for clinical research are limited, decisions must be made about which research projects to pursue, which not to pursue, and how to prioritize among the studies that are chosen. The principle of “unmet medical need” is often acknowledged as a guiding consideration in this context, and there have been calls for community input into prioritization and the choice of the study question. Further, addressing unmet medical needs, particularly in the context of the global burden of disease, is important for public health but may not, and likely will not, maximize market opportunity or financial profits – a dynamic that is particularly salient for private industry sponsors. Should prioritization then rest solely or principally with the funder? How should such entities balance economic obligations toward shareholders with the public good?
One salient principle of distributive justice is “prioritarianism,” the idea that research that stands to benefit the worse-off or those who are already underprivileged should be given priority over research that stands to benefit people in better situations: the well-being of the most disadvantaged is prioritized. Even prior to this, however, questions arise over how to understand the expected goods of research, who the beneficiaries might be, and how the well-being of different possible beneficiary groups should be measured. Further downstream, issues arise over who should engage in prioritization decisions, and in particular, whether the research community should rely solely on high-level, centralized prioritization mechanisms (e.g., industry sponsors, NIH, non-profit funders), or whether individual institutions, local communities, and/or patients and their allies might have some role to play in ensuring that studies are appropriately prioritized at a local level. How should these various voices be heard, should they be represented, and how can—or should—balance be achieved, and if so, what processes should be considered? The March Bioethics Collaborative will seek to address these and other issues in connection with the ethics of research priority-setting.
This meeting was presented to the Bioethics Collaborative. The Bioethics Collaborative is a forum to propose, share, and discuss ethical challenges in multi-national clinical trials. Meetings convene individuals from academia, industry, patient/participant groups, ethics committees, government, and others.
Abstract: The lines between research and care continue to blur. Pragmatic research studies comparing accepted therapies are increasingly embedded seamlessly into clinical practice. More and more, participation in research deemed promising is offered to patients before standard therapies are exhausted, sometimes as a first-line option. While the concept of “therapeutic misconception”—in very broad strokes, the tendency for individuals to misapply attributes of clinical care to research—has been a mainstay of research ethics for over 40 years, these developments provide an occasion, and perhaps even an urgent need, to revisit it and related topics. How exactly should we understand the therapeutic misconception and what it involves, particularly in cases where the line between research and care really is vague and hard to determine? Even more basically, how should we understand the relationship between research and care in the first place? Are concerns over therapeutic misconception still important, or do they perhaps reflect naïve understandings of research and care and the relation between them–particularly in cases where current options are limited?
The Joint Task Force for Clinical Trial Competency (JTF), anchored at the MRCT Center, develops and disseminates standards and practices for the global clinical research workforce. By fostering a cohesive and collaborative approach, the JTF ensures that professionals have the competencies to conduct clinical trials ethically and effectively.
Our international team of investigators, educators, and clinical research professionals has developed and/or utilizes a framework that defines the knowledge, skills, and attitudes necessary for conducting safe, ethical, and high-quality clinical research.
The meeting featured five presentations demonstrating real-world implementation of the JTF Framework:
From Translation to Transformation: Advancing Clinical Research Competencies in Pakistan through the Urdu JTF Framework
Designing Experiential Learning with a Focus on JTF Competencies at North Carolina Central University
Applying JTF Competencies in a Master’s-Level Clinical Data Science Program
Professional Development of the Clinical Research Workforce in New Zealand Public Hospitals
Advancing Professional Standards and Recognition for Australian Clinical Trials Professionals
On-demand Recording on YouTube (with timestamps)
Note: The recording includes timestamps for each of the five presentations.
Clinical trials are vital to advancing medical knowledge and care, yet participation can impose significant financial burdens on participants and their families—from travel and time away from work to uncovered medical and ancillary expenses.
This webinar examined the sources and impact of these costs and highlighted emerging strategies to reduce financial hardship for research volunteers. Presenters also introduced tools, checklists, and other resources developed through the EACT Project, a collaborative forum advancing financial neutrality in clinical research participation.
Good Clinical Practice (GCP) is an international, ethical, scientific, and quality standard for the conduct of trials that involve human participants. The MRCT Center developed and designed this course in collaboration with the ICH E6(R3) Expert Working Group (EWG). This course consists of 5 modules introducing and explaining the key concepts of the E6(R3) guideline. This training is intended for anyone involved in the conduct of an interventional clinical trial. The course links will take the learner to the ICH Training Library website. Courses are free for all registrants.
Currently available is:
Module 1: Introduction and Foundational Concepts, published in October 2025
Carolyn Chapman, Mena Shaikh, Ava Glazier, Andrew Creamer, and Barbara Bierer published Ethical, Legal, and Social Issues (ELSI) in Human Somatic Gene Therapy Clinical Research: A Scoping Review in Human Gene Therapy. Dozens of gene therapies have been approved, and hundreds more are in development, prompting the need to better characterize the ethical, legal, and social implications (ELSI) of this emerging therapeutic class. The authors conducted a scoping review to map these issues across the literature, identifying themes related to risk–benefit assessment, engagement and communication, justice and access, ethical trial design, and the influence of financial and regulatory decision-making. The article also discusses potential approaches to address these ELSI as gene-therapy research expands.
