Recombinant DNA Advisory Committee - 01/14/93 
the NIH have a basis upon which we would disagree with FDA's conclusion? Upon 
consideration of these questions, NIH could find no basis for determining that the FDA 
had made a mistake in granting this approval. However, it would be a mistake for the 
NIH to state that it would defer all compassionate use requests for gene therapy to the 
FDA just because NIH agreed with the FDA's decision in this case. It would be tragic 
for the RAC to state that because of one exemption, the committee is no longer needed. 
The RAC is needed now more than ever as expanded human therapies are being 
explored. 
Dr. Leventhal said that she would like to discuss the use of the word compassionate as it 
implies to the patient's need for this treatment. The FDA term single patient use is a 
more preferable description of this procedure. The RAC should protect patients from 
feeling that they need a particular therapy that has never been proven to provide clinical 
benefit. 
Dr. Krogstad said that he has trouble separating the development of compassionate use 
procedures from the specific case at hand. It is important to clarify that the 
circumstances surrounding Dr. Royston's request were created when the original protocol 
was rejected by the RAC as being inadequate on both scientific and medical grounds. 
This situation should not erupt again. 
Dr. Parkman asked Dr. Chabner if it is appropriate for the first person to receive a novel 
therapy to be on a compassionate use basis, i.e., there is no supporting data regarding 
the safety of that therapy in human? 
Dr. Parkman reiterated his view that there is no mechanism outside of the current 3 
month meeting cycle that allows the RAC to have a formal advisory role. The rules can 
be changed to allow for expedited review, but that would make the review process 
private instead of public. The RAC must balance the right of the individual to receive 
expedited review with the right of the public to be informed about these proposals. Dr. 
Wivel stated that Dr. Parkman has addressed the essential point; specifically, the NIH 
Guidelines require that the RAC is under obligation to conduct its discussions in public. 
Notice of meeting has to be given in consideration of the Federal Advisory Committee 
Act. The public must be allowed to comment on the agenda, and the meeting materials 
are made public. In the event that the RAC chooses to modify the procedures as they 
currently exist, they would have to decide if the public should be notified in precisely the 
same manner as in the past or decide whether gene therapy has reached the level of 
acceptance that it is no longer viewed as a threat to the safety of the public. 
Dr. Chabner responded to Dr. Parkman's question. Dr. Chabner explained that there is 
always a first patient to receive every therapy, and this usually occurs within the context 
of an experimental protocol. However, there are thousands of patients who receive 
Recombinant DNA Research, Volume 17 
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