Recombinant DNA Advisory Committee - 01/14/93 
would be required, i.e., IRB and IBC review? Dr. Healy responded that the FDA 
includes the standard review procedures as part of their compassionate use IND process. 
Dr. Healy reminded Mr. Capron that the NIH did not deliberate with the FDA The 
FDA conducted an independent review using the standard procedures for compassionate 
use single patient INDs. NIH did not make a decision regarding this issue until the FDA 
had granted approval, because the FDA has regulatory authority and statutory authority. 
The NIH used the FDA's approval to help guide its decision. 
Mr. Capron said that he understands FDA has a process, the question is what is the basis 
for this process? If the RAC develops a compassionate use process as has been 
suggested, would the FDA become the only real review mechanism? If an investigator is 
faced with a lengthy 8-week review mechanism with public scrutiny or the closed-session 
quick review provided by the FDA, what course would investigator's choose? Mr. 
Capron stated that he could not imagine that there would be many investigators that 
would choose the long review process. Maybe the committee should face the judgement 
that the RAC approval process is in the evolution of the field , as Dr. Healy has stated; 
and it is no longer necessary. The RAC will no longer be relied upon. At the very least, 
it would be relied upon in those cases where the research is most justified; where the 
design of the research and the preclinical work has been fully carried out and is most 
defensible. In the least defensible cases where those protocols could not pass the RACs 
scrutiny, the alternative approach with less public process will be chosen. Mr. Capron 
said that it is his understanding that Dr. Royston's IBC never granted approval for this 
compassionate plea protocol. The RAC has not seen the data that was presented to the 
FDA 
Dr. Healy reminded Mr. Capron that Dr. Royston's request was not viewed as a research 
proposal. NIH did not grant approval for research to be performed that was not 
believed to have been adequately reviewed as a research protocol by the RAC. Dr. 
Healy explained that the phrase, evolution of the field , does not imply that the field has 
become so advanced that the RAC is no longer needed. This phrase refers to the 
evolution of human gene therapy, expectations about therapies, and evolution to the 
patient. Dr. Healy said that she is deeply concerned about the notion that the FDA 
should be the sole decider of compassionate use exemptions that are derived from 
research that would otherwise come under the purview of the RAC. There might be 
circumstances in which the FDA would approve a compassionate use IND, and NIH 
would deny approval because of concerns about the vector or other safety aspect of the 
procedure. 
Dr. Healy explained that the NIH spent a great deal of time reviewing the safety issues 
surrounding this particular vector. Drs. Liotta, Chabner, Broder, and others, reviewed 
Dr. Royston's request repeatedly and the questions were: Are there safety issues? Is 
there something unique? Is this protocol similar to Dr. Gansbacher's protocol? Does 
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Recombinant DNA Research, Volume 17 
