Recombinant DNA Advisory Committee - 01/14/93 
approval had been obtained, he would have urged the NIH Director to approve the 
request in any case. 
VIII. COMMITTEE'S DISCUSSION 
Dr. Krogstad stated that it is impossible to remove cells from a patient, grow them up, 
transduce them, and demonstrate their safety within 24 hours. Therefore, there should 
never be a need for the RAC to turn such a request around in 24 hours. If the 
investigator initiates the RAC review process at the time the cells are removed with the 
intent of transducing them, there always will be sufficient time to provide thorough 
review. 
Dr. Parkman explained that RAC review of human gene therapy protocols generally 
evolves around three areas: the vector(s), the cells that will be transduced, and the 
laboratory in which the transduction will occur. To say that the same vector and the 
same cell type can be used in another investigator's laboratory is unacceptable. The 
RAC has never defined a protocol solely in terms of the subject(s). Although the RAC 
may choose to entertain single patient versus multiple patient protocols differently, the 
criteria should remain the same. If the RAC chooses to provide expedited review, then 
speed becomes synonymous with non-public review. The NIH Guidelines can be 
amended to accommodate non-public review; however, the RAC will become vulnerable 
to criticism from those individuals that believe strongly in public review of gene therapy. 
He stated that he is uncertain how the RAC should balance speed and efficiency against 
the public's rights to access. 
Dr. Post commented that if the public component becomes non-existent, then should the 
RAC exist? Dr. Parkman stated that even if the RAC chooses to review single patient 
protocols differently from multiple patient protocols, the criteria for such review should 
remain constant even though the process for reviewing these proposals may differ. 
Mr. Capron agreed with Dr. Parkman's description of the objectives; namely, to maintain 
competent standards. The RAC is a deliberative body which incorporates external 
review. This type of review is not easily adapted to the kind of process that Dr. Royston 
has suggested is necessary. Mr. Capron responded to the questions that were posed by 
Dr. Healy during her statement to the RAC: (1) What is the scope of the RACs role in 
reviewing non-research compassionate pleas for gene therapy? Mr. Capron said that the 
expedited review process will have to rely on a different form of advice, which does not 
ensure public scrutiny. The design of the research that the RAC has traditionally been 
concerned about is irrelevant in a non-research situation. (2) What are the 
circumstances under which non-research compassionate pleas ought to require NIH 
approval? The NIH will have to determine the criteria for answering this question, not 
the RAC. (3) Does the RAC, as currently constituted, have adequate expertise to review 
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