Recombinant DNA Advisory Committee- 9/12-13/94 
understood. Ms. Meyers indicated she could not vote for the proposal at present. 
Dr. Miller asked what portions of the IND submission will be made available to the RAC. 
Dr. Noguchi responded they will include clinical protocol, Points to Consider , Informed 
Consent document, and enhanced data reporting. All communication with the 
investigators will be handled by FDA Dr. Miller was unsure that FDA without the RAC 
input, will be able to adequately follow-up the stipulations attached to RAC approval of 
each protocol. He was concerned about the closed discussions inherent in the FDA 
review. Dr. Miller reiterated his view that the current system functions well. FDA and 
the RAC already have fruitful interactions in the development of criteria for evaluating 
retroviral vectors. 
Responding to a question by Ms. Meyers, Dr. Noguchi said that FDA has adequate 
resources to deal with gene therapy protocols. His Division of Cellular and Gene 
Therapies has 14 Ph.Ds, 10 M.Ds (or M.D.-Ph.D.) research scientists, and an additional 6 
to 8 Ph.D. review scientists. There are 4 to 5 physicians with subspecialty certification 
from the Clinical Trial Designs Group, and 4 Ph.D. scientists from the Development 
Group. There is a total of about 70 full-time equivalent personnel available to review the 
human gene therapy protocols. Dr. Noguchi admitted that FDA does not have ethicists 
on its staff, and the contribution of ethicists and public members on the RAC will 
complement FDA shortcomings in dealing with issues related to Informed Consent 
documents. 
Dr. Parkman said that one point that is unique to the RAC is its ability to evolve its 
review criteria and process as each protocol is reviewed. He was concerned about the 
rigidity of the review process that will be codified in FDA regulation. The present 
categories of Accelerated Review can serve as a dividing line to have those protocols 
reviewed by FDA The RAC should be notified at its meeting of the accelerated 
protocols reviewed by FDA and be able to question its appropriateness. Dr. Parkman 
indicated that he would be more comfortable in approving the consolidated proposal if the 
detailed plan of implementation had been presented. 
Mr. Capron commented that the factors which may contribute to the need for RAC review 
are described in a very elastic language. It is important to have FDA commitment that all 
accelerated protocols and its follow-up on data reporting and minor modifications will be 
reported back to the RAC, and the information will be made publicly available. Dr. 
Wivel clarified a question by Dr. Anderson that data reporting will include all protocols 
that are submitted to FDA and not limited to those proposals reviewed by the RAC. Mr. 
Capron was concerned that if Points to Consider is deleted from the NIH Guidelines, the 
RAC would lose its ability to require certain information from the investigators and to 
effect the continuing evolution of this document. He asked if there is any provision for 
investigators to demand public RAC review of their protocols to minimize their risk of any 
future untoward adverse effects. Mr. Capron asked is there any protocol that has been 
approved by FDA while still pending RAC review. Dr. Noguchi replied that FDA waits 
for final RAC/NIH approval before making its own approval. Dr. Anderson noted a 
single exception of the expedited review of a single patient protocol submitted by Drs. 
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Recombinant DNA Research, Volume 20 
