Recombinant DNA Advisory Committee - 01/14/93 
Committee Act; however, Ms. Buc stated that this act may include provisions for 
emergency exceptions. 
Ms. Buc said that the RAC must consider a number of questions. What constitutes an 
emergency exception or a single use exception? What are the criteria for these 
exceptions? What are the criteria that NIH should use in acting on these situations? 
What will the RAC's role be in this process? Should the number of RAC meetings 
increased? How should the NIH respond to a patient that does not fit into the criteria 
that will be established? 
Ms. Buc stated that she was concerned about some of the statements that were made 
regarding the qualifications of the FDA and the RAC. The RAC is comprised of 
members who possess significant expertise. However, she stated that she has 
considerable confidence that the FDA can handle these compassionate requests on a 
single patient basis. It is unfair to say that only the RAC has the scientific competence 
to review a vector or a protocol. 
Dr. Leventhal urged the RAC to defer any further discussion of the individual protocol; 
the remainder of the meeting should be dedicated to developing general policies on the 
issue of compassionate requests. 
Dr. Carmen asked Dr. Royston how he would respond to the multitude of patients 
suffering from glioblastoma if they requested the same therapy that this woman received. 
Dr. Royston responded that he has already received approximately 100 requests, and that 
he referred these patients to Dr. Oldfield's protocol. Dr. Royston said that the NIH will 
now have to deal with compassionate requests for Dr. Oldfield's protocol. 
Dr. Zallen noted that this compassionate request resulted from the rapid progression of 
this patient's disease. The RAC was informed that the 3 month period between 
meetings was too long for the patient to wait for treatment. However, the informed 
consent document is dated August 10, 1992. If Dr. Royston had requested RAC review 
of the protocol on this date, there would have been enough time to proceed through the 
standard RAC review process, i.e., at the December 1992 RAC meeting. Dr. Royston 
said that the reason for the delay was because of his misunderstanding regarding the 
availability of single patient protocol review by the RAC. Dr. Royston stated that a lot 
of things are easier in hindsight. In retrospect, the proposal probably should have been 
submitted as a single patient protocol before the deadline. Dr. Zallen explained that it 
is useful for the RAC to consider that had Dr. Royston been aware that there was a 
mechanism for the review of a single patient protocol, he could have met the submission 
criteria without the need for an alternative mechanism. Dr. Royston responded that if 
the committee had rejected the protocol on the basis of insufficient data and FDA 
Recombinant DNA Research, Volume 17 
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