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Long-Term Gene Therapy Follow-Up: Patient Considerations


Momentum is increasing in the field of gene therapy, with over 20 products approved and more than 2,000 clinical trials underway worldwide (1). By the year 2025, it is expected that the FDA will be approving 10 to 20 gene therapies per year (2). While gene therapy offers the potential for a cure, it also affects long-acting or permanent changes in the human body. This prolonged period of exposure can increase the risk of delayed adverse events. To understand and mitigate this risk, sponsors may be required to monitor participants of gene therapy trials for a long-term follow-up (LTFU) period of up to 15 years. During this extended period, sponsors will be challenged with not only navigating regulatory requirements and covering the cost of ongoing monitoring, but also keeping patients engaged with their studies.

Understanding the Regulatory Framework

The FDA released updated guidance on Long Term Follow-up After Administration of Human Gene Therapy Products in January 2020. This document provides recommendations on designing LTFU studies to collect data on long-term safety and efficacy and delayed adverse events after gene therapy administration. It also offers guidance on the elements and duration of LTFU observations and the requirements for data collection and reporting. For gene therapy products that integrate vectors and genome-editing products, additional monitoring activities may be necessary.

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Hanna Wide, MS, is Executive Director, Gene and Cell Therapy, Rare Disease & Pediatrics. She leads Premier Researchísgene and cell therapy specialty area, providing strategic leadership and promoting high-quality clinical trials that draw on Premier's significant experience in this space. Prior to this role, Hanna worked as Premierís Director, Program Delivery, where she provided operational oversight for clinical trials in rare diseases with a focus on gene and cell therapy. She has more than 12 years of project management experience in the clinical research industry, most of which has been dedicated to rare disease trials.

Dr Anne-Marie Nagy, PhD, is Executive Director, Program Strategy, Neuroscience & Rare Disease. She is a highly experienced executive director in programme strategy within Premier Researchís neuroscience and rare disease therapeutic areas. She provides strategic planning, coordination, knowledge, and expertise for projects and standalone consultative services. Anne-Marie has more than 20 years of global experience within the CRO and pharmaceutical industries.
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