Recombinant DNA Advisory Committee - 01/14/93 
of the case. 
Dr. Leventhal said that she had several concerns. First, that every investigator who 
wants to have a clinical gene therapy protocol will be asking for permission on an 
emergency basis. That is the nature of clinical investigators. The proposed mechanism 
will only be adequate if the exceptions are rare. She also expressed concern about the 
additional burden this will create on the staff of the ORDA. Requests for 
compassionate use or single patient exemptions will overwhelm the routine business of 
the RAC. Dr. Wivel responded that it is his understanding that these requests would be 
reviewed by a group of individuals. These decisions will not be confined to ORDA, but 
will be reviewed by Dr. Liotta and other NIH staff. 
Dr. Leventhal stated that there may be factors that lead the NIH Director to grant 
permission for a single patient to be treated that would not bear scrutiny when a full 
protocol is presented. The RAC must prepare themselves for the idea that just because 
the committee grants approval for one patient to be treated in a particular fashion, does 
not necessarily assume that the multiple patient protocol will be approved by the entire 
RAC. 
Dr. DeLeon spoke in favor of the proposed policy recommendations. If the RAC is 
going to review these protocols post facto after NIH approval, then a report on the 
outcome of the trial should be submitted to the RAC. In addition, there should be a 
provision that protocols that have been disapproved by the RAC should not be submitted 
to the NIH Director as a way of circumventing the RAC. 
Dr. Krogstad requested a friendly amendment to the policy statement. An investigator 
will notify ORDA at the time a patient's cells are transduced, which will ultimately be 
used for the purpose of gene therapy. The terms compassionate use or compassionate 
therapy should never be used. Ms. Buc asked if she could rephrase the amendment such 
that when the NIH is deciding whether a protocol is truly an emergency situation, the 
NIH should apprise itself of circumstances (i.e., intention to transduce the patient's cells) 
in order to assess whether there was ample time for the normal RAC review process. 
Dr. Krogstad agreed to Ms. Buc's revision of the amendment. This provision would 
avoid the submission of an emergency request from an investigator who transduced a 
patient's cells as long as 4 months prior to the request. 
Dr. D. Miller stated that he would not vote in favor of the proposed policy statement. 
The NIH Guidelines are entirely suitable and should be strengthened, if anything, to 
include a provision for expedited review. He said that he does not have faith in the 
limited review that would be provided by the NIH or the FDA. Safety criteria might be 
relaxed to such an extent that a replication competent virus will cause 
lymphoproliferative disease in these patients. 
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