Clinical Gene Transfer: Adverse Event Reporting Fully Harmonized
Authors: Savio L.C. Woo
Journal: Molecular Therapy (2002)

On June 4 and 5, 2002, the American Society of Gene Therapy (ASGT) offered a Clinical Gene Transfer Comprehensive Review Course to approximately 200 registrants immediately preceding its 5th Annual Society Meeting in Boston, MA. The course was organized into five sections: 1) planning for a clinical trial; 2) preclinical development of gene therapy vectors—from petri dish to patients; 3) clinical trial design, approval process, and trial conduct; 4) clinical trial compliance, monitoring, and oversight; and 5) bioethics, research integrity, and conflicts of interest. The Food and Drug Administration (FDA), the Office of Human Research Protection (OHRP), and the National Institutes of Health Office of Biotechnology Activities (OBA) cosponsored the course, and the OBA helped to finance it. The ASGT will issue completion certificates to those who completed the course and will also keep a registry of these individuals. At the review course, staff members from OBA rolled out their Genetic Modification Clinical Research Information System (GeMCRIS), database software, code-veloped with the FDA, that facilitates the evaluation and analysis of human gene transfer studies. This system can be used to register clinical trial protocols and to facilitate the reporting of adverse events online, as all background information of the registered trials will already be in the system. More importantly, because the adverse event reporting requirements of the OBA and the FDA have recently been completely harmonized, the same information submitted to the OBA on GeMCRIS can be simultaneously submitted to the FDA. This process will eliminate confusion about the requirements of adverse event reporting to the two federal agencies and will minimize inadvertent noncompliance. The ASGT has been a strong advocate for complete harmonization of adverse event reporting in clinical gene transfer studies, and the respective federal agencies are commended for making it a reality. The OBA also indicated that it is working on electronic dissemination of adverse event reports to all relevant Institutes and Centers within the NIH, which will further streamline the process. Although investigators who are also the Investigational New Drug (IND) sponsors can readily adopt the reporting procedure, some uncertainties remain for its application in trials sponsored by alternative parties, such as for-profit corporations. It is the responsibility of the IND sponsors and investigators to report adverse events to the FDA and OBA, respectively. In many instances, including most multi-center trials, investigators need to submit adverse event reports to the IND sponsors who then transmit the information to the FDA. Although the role of reporting to the OBA can be delegated to the IND sponsors, the responsibility of sending the reports to the OBA in a timely fashion still lies with the investigators. It would be a great help if GeMCRIS were further modified to allow the investigators to be certain that adverse events reported to the IND sponsors will be transmitted simultaneously to the FDA and OBA.