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Evaluation

Workshop/Symposium

Session: Symposia & Workshops: Session 2

Real-world data-based external control for assessing long-term benefit-risk of cell and gene therapies: insights and perspectives

Monday, August 26, 2024
3:30 PM – 5:00 PM CEST
Location: Room IV
Background
Cell and gene therapies (CGTs) are becoming increasingly important to the treatment landscape for rare, chronic, and debilitating diseases. Evaluation of long-term safety and effectiveness of CGTs usually includes extension studies that follow patients for up to 15 years after clinical trial completion, as well as post-authorization safety and effectiveness studies of patients receiving CGTs in routine clinical practice. These long-term follow-up (LTFU) studies are often designed as non-interventional, single cohort studies that complicate comparative analyses to contextualize findings. External controls using real-world data (RWD) may facilitate understanding the long-term benefit-risk of CGTs.

Objectives
The objective of the symposium will be to highlight the opportunities and challenges in the design and conduct of LTFU studies for CGTs, discussing the potential solution of external controls using RWD for comparative research. Presentations will highlight the perspective and/or experience from several stakeholders on practical, operational and methodological specificities of RWD-based external controls applicable to LTFU studies for CGTs.

Description
The workshop is endorsed by the SIG Cell and Gene Therapy. The symposium will begin with an introduction (10min) by the co-moderators followed by presentations from six panelists (10min each). The symposium will wrap up with a Q&A panel session with the audience (20 min).

• Introduction - Benefit-risk and potential long-term clinical consequences of CGTs (Co-moderators: Erwan Muros-Le Rouzic, Seun Osundolire, 10min)
• Patient perspective on LTFU of CGTs: constraints, opportunities for tailored patient-centered approaches (Reid D’Amico, 10 min)
• Regulatory considerations on external controls for comparative LTFU studies for CGTs (Lourens T. Bloem, 10 min)
• Design and conduct of external controls, special challenges in rare disease populations for CGT LTFU studies (Karla Therese (Reese) Sy, 10 min)
• Identifying fit-for-purpose RWD for external controls in CGT LTFU studies (Hannah V. Furby, 10 min)
• Feasibility of assessing the comparative long-term benefit of CGTs using RWD-based external controls (Carla Y. Vossen, 10 min)
• Statistical considerations for the LTFU of gene therapy trial participants: Platform LTFU trials for real-world comparative research of CGTs (Avery McIntosh, 10 min)
• Open discussion (Panelists and audience, 20 min)