Long-Term Follow-Up For Gene Therapies – Innovative, Patient-Centered Approaches

It is mandatory to monitor patients who receive a gene therapy product over extended periods to assess for delayed adverse events and to potentially evaluate for sustained efficacy. Many regulatory agencies, including the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA), have developed guidelines based on product characteristics regarding the design and key data elements of required long-term follow-up (LTFU) studies. The duration of required gene therapy LTFU studies can range from 5 to 15 years, depending on the nature of the gene therapy. LTFU studies for these therapies present unique challenges, and there are important considerations for different kinds of sponsors, including biotech and emerging biopharma.

Through a tailored, patient-centered approach that may include decentralized solutions, sponsors can successfully conduct LTFU studies of a gene therapy product.

The field of gene therapy is exciting and rapidly growing, requiring innovation in clinical study execution to keep up with the pace of scientific discoveries. Taking a comprehensive and holistic approach, and early on considering the unique requirements for the gene therapy LTFU – clinical issues, data needs, and key study objectives – will allow sponsors to plan a more efficient and cost-effective program and ultimately accelerate clinical development and achieve the goal of getting these important new therapies to patients faster.