Office of Biotechnology Activities; Recombinant DNA Research: Proposed Actions Under the NIH Guidelines

The NIH is proposing changes to the NIH Guidelines to enhance its oversight of human gene transfer research by making modifications to the reporting and analysis of serious adverse events in human gene transfer research studies. The purpose of this Notice is to inform the public about the proposed changes and to seek public comment on them. The proposed changes involve four main issues: (1) The scope and timing of serious adverse event reporting; (2) public access to information about serious adverse events; (3) protection of individually identifiable patient information as it relates to serious adverse event reporting; and (4) a new mechanism for the review and assessment of data on serious adverse events and other relevant safety information. The NIH currently requires all serious adverse events to be reported immediately whether or not they are expected or considered to be associated with the gene transfer product. The first proposed change would require expedited reporting for those serious adverse events that are unexpected and considered possibly associated with the use of the gene transfer product. The proposed change also provides timeframes for expedited reporting and definitions of serious, associated, and unexpected adverse events. Under this proposal, other reportable serious adverse events would be included in annual reports. The second proposed change would clarify that serious adverse event reports submitted to the NIH may not be classified as confidential information and that trade secret or other commercial confidential information should not be included in serious adverse event reports. The third proposed change adds specific language to the NIH Guidelines to prohibit the submission of individually-identifiable patient information in serious adverse event reports. The fourth and final change is the establishment of a working group of the NIH Recombinant DNA Advisory Committee (RAC), to be known as the NIH Gene Transfer Safety Assessment Board, that will be responsible for the review and analysis of serious adverse event reports and other relevant safety information in gene transfer research studies. The working group will report safety information to the RAC and information will, thereby, be disseminated to the scientific and patient communities and the public.