2025 MRCT Center Annual Symposium | Panel 03: Cell and Gene Therapies Long-Term Follow-Up (LTFU) скачать в хорошем качестве

2025 MRCT Center Annual Symposium | Panel 03: Cell and Gene Therapies Long-Term Follow-Up (LTFU)

On October 22, 2025, the MRCT Center convened stakeholders from across the clinical research ecosystem at Ropes & Gray in Boston for a day-long symposium addressing critical challenges and innovations shaping the future of clinical trials. This on-demand collection features keynote remarks and panel discussions from leading voices in industry, academia, regulatory affairs, patient advocacy, and technology. Panel 03—Cell and Gene Therapies Long-Term Follow-Up (LTFU): Key Deliverables and the Path Forward 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, held during the MRCT Center’s Annual Symposium in October 2025, Dr. Carolyn Chapman explains the goals of the Center’s LTFU Working Group, which was launched in September 2024 and included the insights of 25 members from outside the Center. Dr. Chapman also previews the upcoming release of the LTFU Toolkit, developed collaboratively by the Working Group. The Toolkit provides practical guidance to support best practices for LTFU studies in 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 Daniel Kavanagh, PhD, RAC - Senior Scientific Advisor, Gene Therapy, Vaccines, & Biologics, WCG Lara Gehl, Executive Director, Global Trial Management - Bristol Myers Squibb Learn more about the 2025 MRCT Center Annual Symposium through our website: https://mrctcenter.org/mrct-center-20...