Comments provided to: TEDHAS2, 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?
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.
The EC/IRB Guide for Understanding Post-Trial Continued Access aims to assist Ethics Committees (EC) and Institutional Review Boards (IRB) in interpreting their role under Paragraph 34 of the Declaration of Helsinki. Paragraph 34 requires sponsors and researchers to arrange post-trial provisions for “participants who still need an intervention identified as beneficial and reasonably safe.” Exceptions to this must be approved by an EC or IRB, necessitating an understanding of when post-trial, continued access is applicable.
This resource outlines principles and criteria for evaluating when continued access is appropriate. It provides tools and questions to guide equitable decisions, ensuring ethical and transparent approaches to post-trial, continued access decisions. Additional resources and frameworks are available through the MRCT Center’s Post-Trial Responsibilities: Continued Access project page to support ethics committees.
Long-term follow-up (LTFU) studies of gene therapy 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.
In September 2024, the MRCT Center launched an LTFU Working Group. The committee comprises patients, as well as representatives from patient advocacy organizations, industry sponsors, academic medical centers, clinical research organizations, and human oversight protection organizations, each bringing diverse perspectives and complementary scientific, medical, regulatory, and ethical expertise.
On November 4, 2025, the MRCT Center released the Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up Studies as a draft for public comment. The Toolkit provides practical guidance regarding best practices for LTFU studies for both investigational and approved gene therapies. It aims to balance the generation of critical long-term safety and efficacy data with the need to reduce burdens placed on participants, caregivers, sponsors, and investigators.
This webinar introduced the Toolkit’s 𝘀𝘁𝗿𝘂𝗰𝘁𝘂𝗿𝗲 𝗮𝗻𝗱 𝗰𝗼𝗻𝘁𝗲𝗻𝘁𝘀, including: 🔹 𝗚𝘂𝗶𝗱𝗶𝗻𝗴 𝗣𝗿𝗶𝗻𝗰𝗶𝗽𝗹𝗲𝘀 🔹 𝗖𝗼𝗻𝘀𝗶𝗱𝗲𝗿𝗮𝘁𝗶𝗼𝗻𝘀 𝗮𝗻𝗱 𝗥𝗲𝗰𝗼𝗺𝗺𝗲𝗻𝗱𝗮𝘁𝗶𝗼𝗻𝘀 🔹 𝗟𝗼𝗼𝗸𝗶𝗻𝗴 𝗙𝗼𝗿𝘄𝗮𝗿𝗱
It also highlighted additional practical resources: 🔹 𝗞𝗲𝘆 𝗱𝗲𝘀𝗶𝗴𝗻 𝗲𝗹𝗲𝗺𝗲𝗻𝘁𝘀 of LTFU studies for FDA-approved gene therapies 🔹 𝗜𝗻𝘁𝗲𝗿𝗻𝗮𝘁𝗶𝗼𝗻𝗮𝗹 𝗿𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗴𝘂𝗶𝗱𝗮𝗻𝗰𝗲 🔹 𝗚𝗹𝗼𝘀𝘀𝗮𝗿𝗶𝗲𝘀 𝗮𝗻𝗱 𝗯𝗮𝗰𝗸𝗴𝗿𝗼𝘂𝗻𝗱 𝗶𝗻𝗳𝗼𝗿𝗺𝗮𝘁𝗶𝗼𝗻 on types of LTFU studies
𝗠𝗼𝗱𝗲𝗿𝗮𝘁𝗼𝗿: Carolyn Riley Chapman, PhD MS – Lead Investigator, Brigham and Women’s Hospital; Member of the Faculty, Harvard Medical School
Long-term follow-up (LTFU) studies of gene therapy 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.
In September 2024, the MRCT Center launched an LTFU Working Group. The committee comprises patients, as well as representatives from patient advocacy organizations, industry sponsors, academic medical centers, clinical research organizations, and human oversight protection organizations, each bringing diverse perspectives and complementary scientific, medical, regulatory, and ethical expertise.
On November 4, 2025, the MRCT Center released the Toolkit for Supporting the Design, Conduct, and Reporting of Long-Term Follow-Up Studies, as a draft for public comment. The Toolkit provides practical guidance regarding best practices for LTFU studies for both investigational and approved gene therapies. It aims to balance the generation of critical long-term safety and efficacy data with the need to reduce burdens placed on participants, caregivers, sponsors, and investigators.
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:
Introduction and Background
Types of LTFU studies for GTs
Flowcharts
Guiding Principles
Considerations and Recommendations for the Design, Conduct, and Reporting of LTFU Studies for GTs
Looking Forward
Key Design Elements of LTFU Studies for FDA-approved GTs
Regulatory Guidance Relating to LTFU of GTs
Compiled Glossary of Scientific LTFU-Related Terminology
Easy-to-Understand (Accessible) LTFU-Related Definitions from the MRCT Center’s Clinical Research Glossary
Appendices
List of Acronyms and Abbreviations Used
References Cited
We welcome your suggestions and feedback on this Toolkit, which has been released as a draft for public comment. Please email us at mrct@bwh.harvard.edu with your comments and/or questions.
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.
