Recombinant DNA Advisoiy Committee - 01/14/93 
temporal lobe removed. While Dr. Royston was attending the December 4, 1992, RAC 
meeting, the patient was informed that her tumor had increased in size by 30% in 2 
weeks. 
The patient's husband explained that he provided this background information regarding 
the course of his wife's disease to stress the importance of considering the time table 
involved in these crucial cases. 
XII. STATEMENT-DR. ROBERT SOBOL 
Dr. Robert Sobol of the San Diego Regional Cancer Center introduced himself as the 
co-principal investigator on this single patient gene therapy IND. He stated that he is 
here to share his perspectives regarding compassionate plea approvals in the hope that 
his views will be useful in formulating more effective RAC policies for gene therapy in 
the future, 
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Dr. Sobol said that he and Dr. Royston had hoped to follow the standard RAC and FDA 
review process; however, expeditious action was prompted by the deterioration of his 
patient's clinical condition. This patient has failed all conventional and experimental 
therapies. Dr. Sobol commended the FDA and Dr. Healy for their rapid responses to 
the compassionate plea request. 
Dr. Sobol explained that the only reason that he did not obtain approval from the RAC 
is that the committee does not meet often enough. Currently, the portal of entry for 
Federally supported gene therapy protocols to the RAC is four times per year. These 
meetings are too infrequent. If the members of the RAC believe it is necessary to 
review all gene therapy protocols, then they must be prepared to meet more frequently 
to accommodate these patients. The RAC should also develop guidelines for the review 
of protocols that reflect modifications of previously approved studies. 
XIII. COMMITTEE'S DISCUSSION 
Dr. D. Miller asked Dr. Sobol why the RAC never received the Points to Consider 
responses or the materials that were submitted to the FDA Dr. Sobol said that it was 
the investigators' understanding that this information was not germane to NIH's decision 
regarding the protocol. However, Dr. Sobol stated that the Points to Consider and the 
FDA submission material will be provided and discussed at the March 1993 RAC 
meeting. 
Dr. Royston reminded Dr. D. Miller that it had never occurred to him that the RAC 
would entertain a single patient protocol. Dr. Royston explained that the Points to 
Consider responses were prepared in advance of receiving approval from the NIH 
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