The events of recent months emphasize the need for all members of the drug development community to be acutely aware of the need for public education. A continuous, proactive, and reasoned discussion in public forums will be much more useful than reaction after the fact to the sound-bite journalism that characterizes much of the popular press.Industry can and must help this public education on drug development. In presentations at the December meeting of the NIH Recombinant Advisory Committee (RAC) and in testimony at the Senate Public Health Subcommittee hearings in early February, the Biotechnology Industry Organization (BIO) clearly stated its readiness to be involved in the process of public reporting and discussion of gene therapy trials.The FDA has a regulatory authority that is clearly defined but not always well understood by either the public or academic scientists who are not directly involved in drug development. The FDA needs to facilitate further public education and should continue its practice of participating in public meetings such as the RAC and at society meetings such as ASGT.The NIH is now redefining the role of the RAC, which has proven to be a very useful mechanism for public discussion of gene therapy. Initially, this was performed mostly by review of individual protocols. However, the RAC will not be able to continue in this way with the increasing numbers of early gene therapy trials or for large Phase III trials. Several years ago, the Verma committee emphasized the public education role of the RAC to conduct discussion of general topics, examine novel therapies, and assemble a database of information as well as holding public policy meetings. This role was exemplified last year when the RAC conducted meetings on in utero gene therapy, lentiviral vectors, and, at the behest of the FDA, biodistribution of vectors to gonadal tissue. Most recently, in December 1999, RAC addressed the events at the University of Pennsylvania and the general use of adenovirus vectors. In the December meeting, a working group of the RAC listed a number of points to consider for adenovirus gene therapy trials (1xGene therapy on the RAC. Steele, F.R. Mol. Ther. 2000; 1: 1–2Abstract | Full Text | Full Text PDF | Scopus (5)See all References1). These points included standards to quantitate the vector, end-point measurements of vector activity, vector quality control, preclinical evaluation, routes of administration and biodistribution, patient evaluation and monitoring, and the need for control arms in clinical studies. All of these reiterate basic principles for all drug development of which gene therapy is a subset. However, we have yet to accumulate a large database on knowledge of these types of drugs.On its part, the ASGT must examine its role and must understand that gene therapy cannot be viewed simply as an academic scientific discipline but must be a multidisciplinary approach by many rigorous scientific disciplines involved in applying the principles of drug development to gene delivery. Thus, the end result is to move from discovery research in a predominantly academic setting to drug development in an industrial setting. ASGT has many members from academia, industry, and government and is thus in a position to bring together diverse groups for public education. This is a major challenge for our fledgling society that we must accept and implement.The current focus on and discussion of clinical trials and gene therapy serve as an important, helpful, and timely reminder that development and testing of new therapeutic drugs, including gene therapy, are difficult and not without risk. It is incumbent on all of us to exercise constant and maximum due diligence in the design, conduct, analysis, and reporting of clinical trials.