Clinical trials are vital to advancing medical knowledge and care, yet participation can impose significant financial burdens on participants and their familiesโfrom 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.ย Courses are free for all registrants.
Currently available is:
Module 1: Introduction and Foundational Concepts, published in October 2025
Carolyn Chapman, Menaย 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 riskโbenefit 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 โparticipants who still need an intervention identified as beneficial and reasonably safe.โ 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 Centerโs 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 theย 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 Toolkitโs ๐๐๐ฟ๐๐ฐ๐๐๐ฟ๐ฒ ๐ฎ๐ป๐ฑ ๐ฐ๐ผ๐ป๐๐ฒ๐ป๐๐, including: ๐น ๐๐๐ถ๐ฑ๐ถ๐ป๐ด ๐ฃ๐ฟ๐ถ๐ป๐ฐ๐ถ๐ฝ๐น๐ฒ๐ ๐น ๐๐ผ๐ป๐๐ถ๐ฑ๐ฒ๐ฟ๐ฎ๐๐ถ๐ผ๐ป๐ ๐ฎ๐ป๐ฑ ๐ฅ๐ฒ๐ฐ๐ผ๐บ๐บ๐ฒ๐ป๐ฑ๐ฎ๐๐ถ๐ผ๐ป๐ ๐น ๐๐ผ๐ผ๐ธ๐ถ๐ป๐ด ๐๐ผ๐ฟ๐๐ฎ๐ฟ๐ฑ
It also highlighted additional practical resources: ๐น ๐๐ฒ๐ ๐ฑ๐ฒ๐๐ถ๐ด๐ป ๐ฒ๐น๐ฒ๐บ๐ฒ๐ป๐๐ of LTFU studies for FDA-approved gene therapies ๐น ๐๐ป๐๐ฒ๐ฟ๐ป๐ฎ๐๐ถ๐ผ๐ป๐ฎ๐น ๐ฟ๐ฒ๐ด๐๐น๐ฎ๐๐ผ๐ฟ๐ ๐ด๐๐ถ๐ฑ๐ฎ๐ป๐ฐ๐ฒ ๐น ๐๐น๐ผ๐๐๐ฎ๐ฟ๐ถ๐ฒ๐ ๐ฎ๐ป๐ฑ ๐ฏ๐ฎ๐ฐ๐ธ๐ด๐ฟ๐ผ๐๐ป๐ฑ ๐ถ๐ป๐ณ๐ผ๐ฟ๐บ๐ฎ๐๐ถ๐ผ๐ป on types of LTFU studies
๐ ๐ผ๐ฑ๐ฒ๐ฟ๐ฎ๐๐ผ๐ฟ: Carolyn Riley Chapman, PhD MS โ Lead Investigator, Brigham and Womenโs Hospital; Member of the Faculty, Harvard Medical School
๐ฃ๐ฎ๐ป๐ฒ๐น๐ถ๐๐๐: Durhane Wong-Rieger, PhD – President and CEO, Canadian Organization for Rare Disorders Barbara Isquith Arone, MS โ Vice President, Medical Affairs Category Lead, IQVIA Patrick Cullinan, PhD โ Head of Medical Writing and Transparency, Adverum Biotechnologies
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 Centerโs 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.
On October 30, the MRCT Center hosted a webinar on Applied Health Literacy: Using Teach-Back in Conversations About Clinical Research. The session focused on how the Teach-Back method can improve participant understanding, support safe and equitable clinical research, and strengthen communication throughout the research process. The recording, slides, and a list of resources discussed during the session are provided below.
Overview
The webinar reviewed the essential elements of the Teach-Back method and discussed how study teams can simplify complex information, confirm understanding, and foster dialogue with participants and caregivers during recruitment, informed consent, and ongoing study engagement. Presenters emphasized the role of Teach-Back in enhancing clarity, reducing burden, and supporting participant-centered, high-quality research.
Speakers
Mary Ann Abrams, MD, MPH โ Physician and Health Literacy Expert, Nationwide Childrenโs Hospital; Assistant Professor of Pediatrics, The Ohio State University College of Medicine
Published in:ย 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.ย International Journal of Technology Assessment in Health Care. Published online 2025:1-34. doi:10.1017/S0266462325103188
October 22:ย In the articleย โCharacteristics of long-term follow-up studies for gene therapies registered on ClinicalTrials.gov,โ published inย Gene Therapy,ย co-authorsย Carolyn Chapman, Emina Berbic, Ava Glazier, and Barbara Biererย report a descriptive study of key characteristics of LTFU gene therapy study protocols registered inย ClinicalTrials.gov. The analysis enabled a better understanding of how registered LTFUย 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/orย harmonization of data and outcome reporting may increase the scientific value of these studies.
Chapman, C.R., Glazier, A., Berbiฤ, 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
