Recombinant DNA Advisory Committee - 12/3-4/92 
Dr. Blaese said that the European committee is not asking the RAC for a commitment, 
only for assurance that they will provide assistance. If the EC can be confident that the 
RAC will be receptive to this request, then they will be able to focus their strategy. 
Dr. Schaechter stated that there is a serious question about the valuable information that 
would be obtained from this exercise. However, the RAC should not consider the 
efficacy of this request. The RAC should take the position that if the EC believes that 
useful information will be derived from the exercise, then the RAC should support them. 
Dr. Murray added that if the request were to come from an appropriate official body, 
this request would be an important consideration. 
j 
Committee Motion 
A motion was made by Dr. D. Miller and seconded by Dr. DeLeon to provide a positive 
response to this request. The motion passed by a vote of 13 in favor, 0 opposed, and 1 
abstentions. 
Dr. Murray noted that in the interest of time the next agenda item regarding the 
separation of the gene marking informed consent document from the therapeutic 
informed consent document will be postponed to a future RAC meeting. 
XV. DISCUSSION REGARDING COMPASSIONATE PLEA EXEMPTIONS TO RAC 
REVIEW/DR WALTERS AND MR BARTON 
Presentation-Dr. Walters 
Dr. Murray called on Dr. Walters to give his primary review on compassionate plea 
exemptions. Dr. Walters explained that a request has been made to the NIH Director 
regarding the treatment of a single brain tumor patient on a human gene therapy 
protocol that has not been reviewed or approved by the RAC. He explained that he is 
sympathetic with the plight of the patient but suggested that the RAC should not become 
involved in advocating the compassionate treatment of individual patients in protocols 
that have not been formally reviewed and recommended for approval by the committee. 
He proposed an alternative to the compassionate use policy for protocols; namely, the 
approval of minor variations to approved protocols, e.g., relaxation of the 
inclusion/exclusion criteria of a particular study. If a patient was presented who was 
otherwise untreatable and failed on 2 of 3 inclusion criteria, there ought to be a 
mechanism to respond to that particular patient without waiting until the next scheduled 
RAC meeting. The RAC should establish a mechanism for approving minor deviations 
to previously approved protocols. 
Recombinant DNA Research, Volume 16 
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