Patient-Focused Strategies For Long-Term Follow-Up In Gene Therapies

By Erin Finot, MS, MBA, Vice President, Immuno-Oncology & Cell and Gene Therapy, IQVIA Biotech

Gene therapy has surged in the clinical research industry, marked by significant growth and interest. In 2023, venture capital funding reached $3.4 billion, with 406 industry-sponsored clinical trials initiated and 76 cell and gene therapies launched globally. Monitoring patients receiving gene therapy over extended periods is essential, with ongoing research needed to better understand delayed adverse events and sustained efficacy.

Regulatory agencies such as the FDA and EMA have established guidelines based on product characteristics for designing long-term follow-up (LTFU) studies. These studies, mandated to span five to 15 years depending on therapy type and patient population, pose considerable challenges for study sponsors, particularly emerging biotech firms.

With biotech companies driving 50% of all gene therapy trial activity and pioneering much of the innovation, understanding their unique challenges is crucial. As the landscape evolves, these sponsors must adopt innovative, patient-centered approaches and technologies to effectively conduct LTFU studies, minimizing the burden on patients and research sites.