Recombinant DNA Advisory Committee - 2/10-11/92 
be requested by the FDA in terms of efficacy of the gene transfer. 
Other comments 
Dr. Anderson inquired what additional written information the RAC would require other 
than what is in the FDA report and in published papers? If verbal progress reports are 
requested by the RAC, they will become public record. A progress report of this nature 
would be equivalent to publishing results in an unacceptably preliminary form. Dr. R. 
Murray noted that the reporting procedure currently used to promote gene therapy 
protocols has disadvantages; one of those disadvantages is the public nature of the studies. 
Dr. Geiduschek asked if there was a way for the RAC to be able to evaluate protocols 
without having them released to the press, perhaps by taking shelter with the FDA. There 
is a question whether anyone had been truly affected by preliminary release of data. 
Dr. Anderson reminded the RAC of an instance where the press reported in an article 
headline that he was suppressing data when it was presented verbally and not in writing at 
a RAC meeting. The FDA restricts information on the basis of it being proprietary. Dr. 
B. Murray responded that the RAC has the same option if information is proprietary. Dr. 
Kelley stated that it is clear that the RAC is committed to public meetings. The RAC 
must allow the usual scientific critique, review, and publication to be the major 
mechanisms by which the gene therapy field develops. There is a need for a process that 
allows public review, but does not compromise the ability to publish. Dr. Leventhal 
stressed that what was most important to the RAC were the results of the studies, not 
additional ideas developed by the investigators as the studies proceeded. 
Dr. Walters suggested that the RAC should treat safety data and efficacy data differently. 
The RAC's primary purpose is to ensure that patients are not harmed while participating 
in these studies. The RAC should wait until studies are published to determine patterns of 
efficacy. 
More discussion ensued concerning the manner of follow-up reporting, whether it should 
be oral or written and the extent to which it is needed. Ms. Buc recommended that the 
investigators should determine their own manner of follow-up reports, i.e., written, verbal, 
or submission of the FDA report. A subcommittee should be formed to determine which 
format is most appropriate for follow-up reports. 
Dr. Leventhal stated that eventually the protocols would not be coming to the RAC but 
reviewed at the institution level. Ms. Buc said that when the RAC and the public feel that 
review is no longer necessary, the FDA will be responsible for the review. Ms. Buc 
advocated having a special meeting to review this issue. 
Recombinant DNA Research, Volume 15 
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