2 
Daring the subgroup discussion, it was noted that the document would be 
considered a "working" document; i.e., the language and the requirements of 
the document would be subject to modification as circumstances require. The 
subgroup discussed 'whether RAC would accept proposals involving hunan gene 
therapy for review before the "working" document was complete. Those members 
of the subgroup who are RAC members pointed out that RAC has developed working 
documents and simultaneously reviewed proposals in the past; and in same ways, 
it is simpler to develop a submission guidance document when one actually has 
the experience of reviewing and dealing with a proposal. 
The subgroup agreed that should an investigator wish to submit a proposal to 
RAC for review before the Working Group on Human Gene Therapy has completed its 
guidance document, RAC would review the proposal. The investigator should be 
made aware, however, of the fact that additional time might be required to 
review his or her proposal as it would be part of the process of developing 
guidelines. 
Dr. Anderson noted that while several research groups are almost prepared to 
submit a proposal for review, he did not foresee any proposed being submitted 
before the spring of 1985; this should be sufficient time for the working group ~ 
to develop seme guidance document. He suggested that at that time, several 
research groups might come to RAC with proposals. 
The discussion then focused upon the scope of the proposed document. One 
question addressed was whether RAC and its working group's review would overlap 
with sane LRB functions. It was felt the IRBs would conduct reviews according 
to IRB procedures; if the IRB had questions about the proposals or felt RAC and 
its working group might better address seme of the issues, then the IRBs could 
give a conditional approval and request RAC review. In the review, it might be 
necessary for RAC and its working group to obtain information the investigator 
submitted to the IRB; Mr. Capron suggested that IRBs might be asked to supply 
to RAC a summary of their review. 
During this portion of the discussion, the major question was whether RAC would 
deal with issues an IRB would not address. Dr. Anderson pointed out the IRBs 
do not review and discuss the goals of the procedure; rather, the IRB is pri- 
marily concerned with the rights of the patient. He suggested that as no 
national group currently is discussing the impact of gene therapy procedures on 
society or the long range effects of these procedures; RAC should fill this 
void. Topics might include effects of manipulation on subsequent unborn genera- 
tions, issues of elimination of certain genetic configurations and eugenic 
selection. The subgroup then discussed whether a discussion of these types of 
issues would be scientifically and ethically valid. After sore discussion, the 
subgroup agreed that if the public had concerns, these concerns should be 
addressed. The scientific aspects of proposals should be described and dis- 
cussed in relation to these public concerns. Dr. Gottesman felt that if the 
proposals were fully described, the public could compare their concerns with 
the proposed goals and procedures of the investigation. 
Ms. Areen asked Dr. Miller if the Food and Drug Administration (FDA) would 
review protocols involving human gene therapy. Dr. Miller replied that FDA 
would not necessarily review every case. 
[ 5 ] 
