SIG: Cell and Gene Therapy

Title: Gene Therapy Long-term Follow-up Studies: Regulatory, Scientific, and Operational Requirements, Challenges and Opportunities

Date: January 8, 2026

Time: 10:00-11:00 AM EST

Speakers: Lisette Hoogendoorn, Jamie Geier, Nick Honig

Moderator: Emily Bratton

Description: Cell and gene therapies (CGT) often require long-term follow-up (LTFU) studies of safety and effectiveness once they are approved, with up to 15 years of follow-up per patient. While extension studies of clinical trials can also play a role, real-world data (RWD) are especially relevant to enable the required duration of follow-up and the collection of specific data elements. However, the use of RWD still leaves important challenges for the design and conduct of LTFU studies of cell and gene therapies. Such challenges can be related to sample size, data capture, and data quality, among others, potentially more than for other studies involving RWD. This webinar aims to enhance understanding of regulatory, scientific, and operational requirements and considerations for LTFU studies from a global perspective. Examples will be given of challenges with the use of RWD and how novel approaches (such as decentralized studies and the use of tokenization) may support the capture of crucial data for these studies.

Learning Objectives:

• Explain the requirements for LTFU studies to study the safety and effectiveness of cell and gene therapies after their approval.
• Explain the role RWD can play in such LTFU studies and illustrate challenges that can be encountered during the design and conduct of RWD-based LTFU studies.
• Provide perspective on and examples of studies that applied novel data collection approaches to capture crucial data for the study of safety and effectiveness of CGT.

Lisette Hoogendoorn, Epidemiology Director, Global Epidemiology and Database Studies team, IQVIA Real World Solutions

Lisette Hoogendoorn has a long background in epidemiology in different settings and experience in pharmacovigilance, both as a consultant and assessor for the Dutch Medicines Evaluation Board. In her current role, her focus is on scientific leadership, and she is responsible for the design, analysis, and interpretation of observational and minimally interventional studies. Special areas of interest are using real-world data in drug development with a focus on regulatory requirements, cell and gene therapies, and external comparator studies. As a consultant, she is experienced in supporting clients with further defining their research questions and designing studies accordingly. Lisette received her PhD in Epidemiology from the Vrije Universiteit, Amsterdam, the Netherlands.

Jamie Geier, Global Head of Quantitative Safety and Epidemiology (QSE) at Novartis

In Jamie Geier's role, she is responsible for the QSE safety strategies to support clinical development and health authority interactions. She is also the Scientific and Strategic Lead for the Novartis Pregnancy outcomes Intensive Monitoring (PRIM) Initiative as well as a PRIM Steering Committee member. Before joining Novartis in 2020, Jamie was Group Head and Senior Director, Epidemiology Strategist for Inflammation & Immunology at Pfizer, Inc. New York, NY. In this role she led a team of epidemiologists to deliver regulatory safety strategies with a focus on risk characterization. She has 19 years of pharmaceutical industry experience as an Epidemiology Lead and Strategist for a wide range of drug therapies with Neuroscience, Rheumatology, Medical Dermatology & Gastroenterology. Jamie has extensive regulatory and strategic experience, including epidemiological study design strategies and the use of non-interventional data sources for regulatory decision-making. Jamie received her MPH and PhD in Epidemiology from Columbia University in New York, NY USA.

Nick Honig, Executive Director, Highlander Health

Nick Honig oversees the Highlander Health Institute projects and provides support to the Highlander Health Partners portfolio of companies. An attorney by training, Nick was previously Head of Regulatory at Aetion, a company focused on generating high quality real-world evidence. Nick has also worked in Pfizer's Chief of Business Office and was a Summer Associate in the Food and Drug Administration's Office of Chief Counsel.