Focus Area: Clinical Trials & Research

Webinar

Presented on: November 4, 2025

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

𝗣𝗮𝗻𝗲𝗹𝗶𝘀𝘁𝘀:
Durhane Wong-Rieger, PhD – President and CEO, Canadian Organization for Rare Disorders
Barbara Isquith Arone, MS – Vice President, Medical Affairs Category Lead, IQVIA
Patrick Cullinan, PhD – Head of Medical Writing and Transparency, Adverum Biotechnologies

---

---

Related Resources

Cell and Gene Therapies

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/postapproval-methods-capture-safety-and-efficacy-data-cell-and-gene-therapy-products

Patient-Centered Long-Term Follow-Up for Gene Therapies

Innovative Approaches for Gene Therapy Long-Term Follow-Up

Webinar

Presented on: October 30, 2025

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 – Physician and Health Literacy Expert, Nationwide Children’s Hospital; Assistant Professor of Pediatrics, The Ohio State University College of Medicine
• Stephanie Shepherd, MBOE, BSN, RN – Clinical Educator, Nationwide Children’s Hospital
• Marian Ryan, PhD, MA, MPH – Chief Policy & Research Officer, Institute for Healthcare Advancement (IHA)

Moderator

• Sylvia Baedorf Kassis, MPH – Program Director, MRCT Center

What the Session Covered

• How Teach-Back supports participant comprehension, autonomy, and safety
• Approaches to explaining complex study information in accessible, plain language
• Strategies for confirming understanding in an ethical, respectful, and culturally responsive manner
• Practical examples of how to integrate Teach-Back across recruitment, informed consent, and study follow-up processes

Additional Resources

Return of Aggregate Results to Participants Guidance Document Version 3.1

Return of Aggregate Results to Participants Toolkit Version 3.1

MRCT Center Health Literacy Website

Clinical Research Glossary

Health Literacy Resources for IRBs
Health Literacy Best Practices
https://health.gov/our-work/national-health-initiatives/healthy-people/healthy-people-2030/health-literacy-healthy-people-2030
https://nces.ed.gov/naal/health.asp
https://www.ahrq.gov/sites/default/files/wysiwyg/health-literacy/dhhs-2008-issue-brief.pdf
http://justplainclear.com/en
http://www.plainlanguage.gov/whatisPL/definitions/eagleson.cfm
https://Teachbacktraining.org
https://www.Teachbacktraining.org/OrganizationalHealthLiteracyGuidebook2
https://www.hhs.gov/ohrp/regulations-and-policy/belmont-report/read-the-belmont-report/index.html

WMA Declaration of Helsinki – Ethical Principles for Medical Research Involving Human Participants

---

Webinar

Presented on: September 30, 2025

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.

Panelist: Daniele Bertolini, Machine Learning Scientist at Unlearn.ai.

Moderator: Barbara Bierer, Faculty Director at the MRCT Center

---

---

Related Resources

Framework for Review of Clinical Research Involving AI

A Framework for AI Adoption and Oversight in Clinical Research