Recombinant DNA Advisory Committee - 01/14/93 
use vectors that have already been reviewed and approved by the RAC for human gene 
therapy. (4) Consider compassionate use requests outside of the research system and 
require that data from such treatments cannot be used in research publications. In 
conclusion, the proposal states that the working group does not recommend any of these 
alternative mechanisms; because there are serious reservations about each point. The 
working group has outlined these items so that the entire RAC and Dr. Healy could 
refine a particular option or propose an entirely new alternative. 
Dr. Post said that he agreed with the statement made by Ms. Buc regarding the authority 
of the NIH Director to make these decisions. Since the RAC does not readily fit into 
the compassionate plea request process, perhaps a final alternative should be that the 
RAC should not entertain these requests. 
Ms. Buc stated that she was puzzled by point number 4. There seems to be some kind 
of punishment intended for investigators using compassionate plea requests; namely, they 
do not have the opportunity to cite their results. Denying publication to these 
investigators undercuts an important aspect of our effort; namely, deriving important 
information from these early experiments. The FDA recognizes that compassionate use 
requests are not full-blown research; however, their regulations require that investigators 
must report any information obtained as a result of the procedure. Therefore, the NIH 
and the RAC should not allow investigators to relinquish their obligation to file a report. 
Dr. Healy reminded Ms. Buc that investigators always have the option of publishing a 
case report. This mechanism allows the investigator to relay new information, but it 
does not require the statistical validity of a research publication. Dr. Healy stated that 
compassionate use treatments can be certainly considered valid as case reports. 
Dr. Healy reminded the RAC that her comments regarding the non-research component 
of compassionate use requests were not intended to get the RAC off-the-hook. The 
RAC should have a role if an experimental therapy is a direct derivative of research 
conducted and supported by the NIH. The NIH needs the advice of the RAC. How can 
a system be created in which the RAC can provide advice and balance the needs of a 
patient that is in imminent danger? In turn, if a patient is not in imminent danger, then 
the proposal can wait until the next scheduled RAC meeting. Dr. Healy said she has 
learned during this process that the RAC cannot convene without the appropriate public 
notice. These are uncharted waters. The flexibility of the RAC is important. If NIH 
cannot depend on the advice of the RAC then it will have to seek the advice of some 
other similar body. Dr. Healy stated that advice regarding gene therapy should come 
from the RAC. 
Mr. Capron inquired how the NIH understands the basis for compassionate use 
exemptions granted by the FDA Is there any expectation that other review processes 
Recombinant DNA Research, Volume 17 
[15] 
