Recombinant DNA Advisory Committee- 9/12-13/94 
patients to gene therapy. Dr. Noguchi suggested that the RAC can influence public policy 
about this concern. Other areas the RAC can address through public discussion are a 
gene therapy patient registry, criteria for prenatal gene therapy, gene therapy for 
enhancement, and transgenic technology for xenotransplantation, in which transgenic 
baboons and pigs will be used as organ donors for human transplantation. 
Dr. Noguchi addressed concern about the logistics and merits of the consolidated review 
system. In order to maintain the public nature of gene therapy protocols, the FDA will 
adopt the current Appendix M, Points to Consider, and the investigators will be required to 
submit this document to FDA/ORDA before submission of an IND. Dr. Noguchi said 
that once an IND is submitted, FDA reviewers are assigned; and it will be given a 
response within 30 days under a statutory mandate. FDA/ORDA/RAC will decide on 
necessity for full RAC review. The RAC review will proceed in the pre-IND period. 
Whether reviewed by the RAC or not, the Points to Consider submitted by the 
investigators will continue to be publicly available. FDA has resources to enhance data 
monitoring efforts, and these data will be made available to the public through the 
ORDA FDA/ORDA/RAC will establish a working group to implement a long-term 
consolidation with input from public, academic, and corporate sources. 
Dr. Parkman expressed the feeling of the RAC about the consolidated review as being 
ambivalent. The political reality is that both Drs. Kessler and Varmus have committed to 
the idea of a one-stop "shopping" mechanism. He was delighted that Dr. Noguchi 
responded to the RAC concern by adopting the Points to Consider and assured the role of 
the RAC in the continued evolution of the document by keeping it as Appendix M of the 
NIH Guidelines. Dr. Parkman said that the list of all protocols, including those deemed 
not to require RAC review, should be reported to the RAC at its next quarterly meeting, 
and that the RAC should retain its ability to recall any of those protocols for full RAC 
review, if necessary. Dr. Parkman expressed the desire to wait until the next meeting to 
vote on this issue when the detailed procedures of the review process are worked out. Mr. 
Capron said the revised FDA proposal is an evolution in the right direction, but he still 
favored deferral of any formal action at present. Ms. Meyers said Dr. Noguchi has 
responded to her two major concerns, i.e., public access to the information submitted by 
investigators in response to the Points to Consider, and the RAC’s role in its continuous 
evolution. Dr. Chase said that it is fruitless to resist the change of the review process, and 
he lauded the administration’s efforts to refocus the RAC’s role to deal with the global 
issues of gene therapy in a public forum. 
Mr. Capron recalled that the creation of the RAC effectively made congressional 
legislation to regulate a nascent scientific field unnecessary. The RAC was created in 
response to the issue of potential dangers of recombinant DNA research expressed in the 
AsUomar Conference of 1975, and to the recommendation by the President’s Commission 
report. Splicing Life, regarding human gene therapy. At those times, Congress held several 
hearings and was considering legislation to regulate these areas of concern. To ease these 
concerns, the RAC was formed consisting of scientific and public members; later a Human 
Gene Therapy Subcommittee provided public oversight in these areas. The industry 
demonstrated its voluntary compliance to the NIH Guidelines. The present reform of the 
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