Recombinant DNA Advisory Committee- 9/12-13/94 
that he was uncomfortable with the current proposal since the RAC already had adopted 
an Accelerated Review procedure to address the overload problem. Procedurally, the new 
system will limit the RAC’s ability to amend its own guidelines. Dr. Erickson made the 
observation as someone who had served on the RAC before, that the present proposal 
might not greatly simplify the review process. Dr. Doi indicated his inclination for 
deferral until the FDA finalizes its guidelines for IND submission. Dr. Noguchi said that 
consolidated NIH/FDA review system is a radical idea that requires joint effort from both 
agencies and the public in order to finalize its plan. Dr. Anderson agreed on the principle 
of the simplified review system but expressed his concern that the public will lose track of 
all gene therapy protocols including minor modifications if all submissions are routed 
through the confidential FDA process. He stressed that the data management function 
should remain in the public domain within the ORDA Dr. Noguchi said that data 
monitoring will be enhanced with its pilot project to create a comprehensive computer 
data base for its IND process, and that these data will be made available to the public. 
Dr. Anderson said that FDA data base is confidential and not all information can be 
made public. The final agreement has to assure that the RAC is able to make the 
information available even though the submission is to FDA Dr. Noguchi said that public 
access is a crucial point of the proposal. 
Dr. Walters called on Dr. Noguchi to present his prepared remarks. Dr. Noguchi 
acknowledged that the joint agreement for the NIH/FDA consolidated review was drafted 
in a very short order during the AIDS Task Force meeting. Dr. Noguchi said that from 
the FDA’s viewpoint, the public nature of the oversight process has allowed the field of 
human gene therapy to progress very rapidly in the past. The public dissemination of the 
information regarding the adverse effects of Dr. Crystal’s protocol on the use of an 
adenovirus vector to study cystic fibrosis resulted in a timely readjustment of the dosing 
schedule for several other similar clinical trials. FDA has a congressionally mandated 
regulatory authority over certain areas, but it cannot act in areas where there is no legal 
authority. The RAC is not a creation of law and, therefore, can complement the 
restrictions imposed on the FDA FDA gets its authorization when there is a disaster and 
the RAC has prevented disasters. Dr. Noguchi said that the joint review is a real 
opportunity for both parties. 
Dr. Secundy favored deferral for the present and suggested a small group of RAC 
members to work with FDA for the final plan. Ms. Meyers expressed her concern about 
the fact that FDA needs to keep trade secrets confidential. Confidentiality will impede 
public accountability of gene therapy studies. She questioned whether FDA has enough 
resources to perform the additional responsibility relative to human gene therapy, and 
whether FDA has appropriate staff to evaluate the ethical issues and the Informed 
Consent documents. She mentioned and Mr. Capron recalled a lawsuit against the RAC 
in 1989 for failing to duly announce a RAC meeting in the Federal Register. Dr. Walters 
said that a lawsuit was brought by the Foundation on Economic Trends and was settled 
out of court at the time of RAC approval of the first gene marking protocol. Ms. Meyers 
asked if the proposed arrangements will be a problem for the confidential FDA process. 
She considered that public oversight is still needed at the present stage of development of 
human gene therapy since the long-term possibly untoward effects are not clearly 
Recombinant DNA Research, Volume 20 
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