Recombinant DNA Advisory Committee - 12/1-2/94 
Dr. Walters noted that a couple of years ago there was a protocol submitted by a company to the FDA and 
discussed at a public FDA advisory committee meeting. Dr. Noguchi explained that the protocol described by 
Dr. Walters was the first HIV gene therapy protocol submitted by Viagene, Inc., San Diego, California. That 
study did not involve any NIH funding. Dr. Noguchi noted that in the future, FDA will bring such protocols to 
the RAC for public review as a part of the NIH /FDA consolidated review process. Dr. Walters said that up 
to now, the public has been informed of every gene therapy study that has been conducted in the United States 
by the FDA or the NIH. 
Mr. Capron asked whether the FDA would reject Dr. Goldhammer’s request for optional RAC review. Dr. 
Noguchi responded that Dr. Goldhammer’s letter was not addressed to the FDA. The FDA does not have 
legal authority to accept or reject that particular concept under its current regulations. Dr. Noguchi noted that 
the pertinent issue is uncertainty regarding the definition of a trade secret or confidential information. It is 
unclear what information can be disclosed to the public by the FDA. Public review of the overall concept and 
the ethical questions poses less of a problem for FDA. If a safety concern is related to vectors, it is unclear 
under the current regulations whether FDA can make that information public without the company’s 
permission. Mr. Capron asked if the information can be shared with FDA’s own advisory committees. Dr. 
Noguchi responded that confidential information can be discussed in closed session. Mr. Capron asked if 
RAC members will be co-appointed by both the NIH and the FDA and whether the RAC will become 
advisory to both agencies. Under such a scenario, would the RAC have access to FDA information? 
Information reviewed in a closed RAC session could be summarized for public disclosure after the session. 
Mr. Capron said it is legal for a company to proceed with an experiment without RACs approval if reviewed 
on a voluntary basis. However, if an adverse event occurred when a sponsoring company was informed by the 
RAC that the research was inadequate and would raise unreasonable risks, the company would not have a 
defensible action. 
Dr. Straus asked if an investigator who develops a drug or a biologic in his own laboratory is permitted to use 
that agent in any therapeutic, diagnostic or prophylactic setting without FDA approval? Dr. Noguchi 
responded that any such agent (with exception of diagnostics) is subject to IND regulation based on the 
October 14, 1993, Federal Register announcement. Any intrastate or interstate drug or biologic is subject to 
IND regulation. Dr. Straus asked whether regulation is intended for investigational use and commercial 
licensure? Dr. Noguchi responded that the regulation covers all uses. The FDA exercises discretion over 
regulation, e.g., bone marrow purging with monoclonal antibodies is not regulated. The more cellular 
manipulation is involved, the greater the likelihood that the experimental reagents will be subject to FDA 
regulation. Any reagent developed in an investigator’s laboratory that has the potential to produce extremely 
potent effects may require other government oversight in addition to local IRB approval (if intended for 
human use). Dr. Straus inquired whether an investigator would be permitted to use a retrovirus vector 
encoding the interleukin (IL)-2 gene for the treatment of diseases other than those for which the vector was 
originally approved? Dr. Noguchi answered that the FDA allows latitude for other uses. Dr. Straus asked if 
FDA regulation would permit use of an approved gene therapy product for novel uses, Le., enhancement or 
even germ line modification? Dr. Noguchi responded that although such a scenario is legally possible, there 
are no gene therapy products approved for commercial purposes. All gene therapy experiments are currently 
in the Phase I clini cal trial stage. Dr. Straus was concerned that such a scenario could occur if there is no 
other mechanism for oversight. 
Dr. Par km an agreed with Mr. Capron’s suggestion that the RAC should be advisory to the FDA in order to 
facilitate the flow of information. In response to Ms. Meyers concern about confidential proprietary 
information. Dr. Par km an said the NIH Guidelines allow the RAC to review confidential information in closed 
session. A summary of the deliberations can be made available to the public that does not include any 
proprietary information. 
Dr. Chase agreed with Dr. Goldhammer’s comments that the RAC should focus on major societal issues and 
not individual review of routine protocols and that novel uses of approved gene therapy reagents should be 
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