Recombinant DNA Advisory Committee- 9/12-13/94 
patent protection before the RAC meeting. Dr. Anderson said that the closed session 
should be discontinued for the RAC meetings except for exceptional circumstances (e.g., 
patient confidentiality). Dr. Miller remarked that Dr. Lyerly’s company has yet to file for 
patent application, but he conceded that there was nothing discussed in yesterday’s closed 
session that could not be reviewed in a public meeting. Dr. Walters remarked that this 
was the first occasion in recent history of RAC meetings that proprietary information was 
reviewed in a closed session since the last instances in the early era of recombinant DNA 
research of 1980 and 1981. The other occasion of a closed session was to protect patient 
confidentiality in the discussion of a single patient expedited review in 1993. 
XIII. CONTINUATION OF THE DISCUSSION REGARDING PROPOSED AMENDMENTS 
TO SECTIONS I, III, IV, V AND APPENDIX M OF THE NIH GUIDELINES 
REGARDING NIH AND FDA CONSOLIDATED REVIEW OF HUMAN GENE 
TRANSFER PROTOCOLS/DRS. WIVEL AND NOGUCHI 
Dr. Walters noted that there was no one from the audience who had submitted written 
comments on the consolidated review and wanted to make a comment. He introduced a 
revised proposal submitted by Dr. Noguchi following the previous discussion on the 
NTH/FDA consolidated review system. The FDA proposal reads as follows: 
o Appendix M, Points to Consider , will not be deleted from the NIH 
Guidelines. The NIH Guidelines will be modified to require Appendix M, 
Points to Consider, to be submitted directly to FDA before the IND. FDA 
will update their guidance documents in a similar manner. When necessary, 
the RAC will continue to be responsible for modifying Appendix M, Points 
to Consider. 
° FDA/ORDA/RAC will decide on the necessity for full RAC review. The 
submitted Appendix M, Points to Consider, will be publicly available for all 
human gene transfer submissions even if RAC review is not required. 
° RAC/FDA will broaden their scope of review in gene transfer to jointly and 
prospectively address global issues on a regular basis, e.g., ethical 
considerations in the implementation of a gene therapy patient registry, 
access for "orphan' 1 genetic disease patients to therapies, criteria for prenatal 
gene therapy, and transgenic technology for xenotransplantation. 
° FDA/ORDA/RAC will establish a working group to enhance data 
monitoring efforts that will be maintained by ORDA. 
° An FDA/ORDA/RAC working group will be established to consider long- 
term consolidation. The working group will have input from public, 
academic and corporate sources. 
Dr. Walters called on Dr. Noguchi to present the FDA proposal. Dr. Noguchi used a 
slide to illustrate the different logistical backgrounds in the creation of the RAC and FDA 
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