Recombinant DNA Advisory Committee- 9/12-13/94 
Dr. Noguchi said that publicly funded gene therapy studies should be reviewed publicly. 
One imminent issue is gene enhancement therapy. Human fibroblasts can be gene- 
modified to produce human grown hormone. Should this kind of gene therapy be tried in 
children of short stature? There is no easy mechanism to have timely debate of these 
issues at FDA. The public advisory committees of FDA are geared towards approval of 
Phase n/III clinical trials and final approval of drugs. There is no equivalent committee 
functioning like the RAC to deliberate issues of Phase I studies or issues such as prenatal 
gene therapy and enhancement therapy. The RAC is a societal body to consider the 
question of whether the society is ready to permit these types of gene therapy. 
Ms. Meyers inquired if FDA has authority to turn down an experiment with questionable 
ethics such as transferring human growth hormone gene in a short child. Dr. Noguchi said 
that FDA has little designated authority to do that, and no ready mechanism to deliberate 
this issue publicly in a timely fashion. Furthermore, FDA does not have bioethicists 
available to review this kind of ethics question. Ms. Meyers asked if FDA reviews the 
Informed Consent document. Dr. Noguchi said that FDA does have a regulation that 
requires investigators to obtain approval from their IRB, but that RAC can more easily 
recommend useful guidelines for evaluation of informed consent. Dr. Ross said that 
knowledge of the functions of other government agencies is helpful in defining the RAC 
role. 
Dr. Tony Marcel (TMC Development, Paris, France) commented that the RAC has 
international impact. Through the open RAC meetings and distribution of its minutes, 
international audiences are informed about the present concerns relative to gene therapy. 
Mr. Jeff Gustavson from ACT-UP-Goldengate, San Francisco, California, said that the 
AIDS community favors the RAC review in a public forum so that the community’s 
concern can be channeled into the review process. 
Dr. Varmus said that an important task of the ad hoc group to review RAC activities is to 
define the criteria by which a protocol can be reviewed by the one-stop shopping 
mechanism without public review in a RAC meeting. This issue is important since too 
many applications are expected in the future for the RAC to consider in its public 
meeting. Ms. Meyers said that most of these repetitive kinds of studies are very similar, 
except consistency of the quality of Informed Consent documents. Dr. Varmus said that 
efforts should be made to identify elements, either scientific or related to informed 
consent, that require close scrutiny in the Accelerated Review of gene transfer protocols. 
Dr. Noguchi commented that even in repetitive type of studies the collection of data 
regarding adverse effects will be an important RAC activity. 
Dr. Parkman said that recent RAC efforts to define the categories of experiments for 
Accelerated Review are in keeping with what Dr. Varmus has just suggested. Mr. Capron 
asked whether the proposed NIH/FDA consolidated review will be put in place before the 
ad hoc committee review is completed. Dr. Varmus said that he comes to the RAC to 
solicit feedback ideas about this proposal. The implementation of the consolidated review 
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Recombinant DNA Research, Volume 20 
