Workshop on Long-term Follow-up of Participants in Human Gene Transfer Research

Abstract

On June 1-2, 2004, staff from government agencies, principal investigators (PIs), and industry representatives gathered in Minneapolis just prior to the 7th Annual Meeting of the American Society of Gene Therapy (ASGT) for a workshop to discuss issues pertaining to the long-term follow-up (LTFU) of participants in human gene transfer research. The ASGT co-sponsored this workshop, along with the FDA's Center for Biologics Evaluation and Research (FDA/CBER), the NIH's Office of Biotechnology Activities (NIH/OBA), the Biotechnology Industry Organization (BIO), and the Pharmaceutical Research and Manufacturers of America (PhRMA). Workshop goals comprised (a) identifying the scientific, clinical, ethical, and social challenges associated with long-term follow-up of individuals enrolled in clinical studis of human gene transfer and (b) gathering input from workshop attendees regarding practical options for the collection of LTFU data that will be used to improve the safety of clinical gene transfer procedures. This article summarizes the presentations and overall outcomes of breakout sessions focusing on clinical, scientific, animal model, and legal/social/ethical issues.