Recombinant DNA Advisory Committee - 03/1-2/93 
the National Cancer Institute who were of the opinion that efficacy data is unnecessary. 
Dr. Miller said that the RAC does not necessarily agree about the requirement for 
efficacy data. Dr. Royston stated that if the RAC had not approved this protocol then 
he would have been forced to challenge the review system. 
Dr. Leventhal stated that the protocol approved by the NIH Director and the FDA is 
not what would normally be considered an emergency protocol. Dr. Leventhal stated 
that if a protocol is planned for only one patient, then it should be designed to answer a 
particular question. This protocol has no endpoint. Dr. Royston said that the Procedures 
for Expedited Review , just approved by the RAC, do not include long-term plans. Dr. 
Leventhal reminded Dr. Royston that this document does include the statement that the 
single patient protocols must be consistent with the Points to Consider which encompasses 
long-term planning. 
Dr. Straus expressed concern that the protocol followed by the investigators is different 
than the protocol submitted to the FDA. Dr. Sobol said that if the RAC perceives that 
there was a deviation from the protocol, the error is probably that the statements were 
not worded clearly enough. Dr. Sobol said that in his opinion the treatments 
administered to the patient were articulated in the protocol. 
Dr. Chase said that the key issue is that the RAC does not have enough information to 
determine whether it would have voted to recommend approval of this protocol on 
scientific grounds at the time that the NIH Director approved the request. One of the 
important aspects of the RAC is the degree of confidence that the public has in the 
decisions made by this committee. The public deserves to know that the RAC has made 
an informed decision about the value of an experiment. A decision made by the RAC 
about a research proposal may be different from a clinical decision made by a physician. 
As a physician. Dr. Healy granted approval of this protocol based on compassion for the 
patient. However, from a public standpoint, it is important that this protocol be 
evaluated for scientific merit by a panel of experts. 
Dr. Royston stated that the public is primarily concerned about safety. Dr. Chase agreed 
that the public is concerned about issues of safety; however, there are other concerns. 
Dr. Royston said that the FDA looks at safety as well as the scientific merit of a 
protocol. However, the criteria used by the FDA may be different from those of the 
RAC. Dr. Chase said that one of the issues that the RAC is concerned about is the 
possibility Drs. Sobol and Royston might return to the NIH Director with other single 
patient expedited review requests, and that the data generated would not be 
interpretable by the scientific community. Dr. Chase stressed the importance that all 
investigators complete the Points to Consider. Dr. Chase stated that it is highly probable 
that if Drs. Sobol and Royston had completed the Cover Sheet for Expedited Review , the 
protocol would not have qualified for expedited review. 
Recombinant DNA Research, Volume 17 
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