Recombinant DNA Adviaory Committee - 09/14-15/92 
Dr. Parkman suggested an additional agenda item should be included for the next RAC 
meeting; namely, the issue of separation of the therapeutic and gene marking informed 
consent documents. 
Ms. Buc requested that the issue of data reporting and how to enforce compliance 
should be added to the next RAC agenda. Mr. Capron asked if the concerns that 
investigators have regarding the release of data and possible threat to publication in a 
peer reviewed journal is a legitimate concern. Dr. Anderson said that this issue is not 
bogus. Although in principle Science and the New England Journal of Medicine have 
agreed, that the presentation of data at an open forum such as the RAC will not 
interfere with subsequent publication of data in these journals, the reality is that 
reviewers evaluate data and assign priority based on the importance of the paper. In 
certain instances, a journal could put a hold on the publication of this information. Dr. 
Krogstad said that this situation is one of those unusual circumstances in which the RAC 
has a monopoly. If the RAC decides that it requires pertinent data to make intelligent 
decisions regarding the approval of future protocols, then the committee members must 
maintain their position regarding the procurement of data. 
Dr. Murray read a letter, dated June 11, 1992, that was forwarded to all investigators 
who have received approval to initiate human gene transfer/therapy trials. The letter 
requested that meeting abstracts, IRB annual reports, reports of adverse effects, FDA 
annual reports, and published scientific papers should be forwarded to ORDA. The 
response has been minimal. Dr. Secundy asked if this request could become a 
requirement. Ms. Buc noted that the list of requirements was compiled with much 
forethought; and that every listed document is one that has to be created for other 
purposes, not solely for the RAC. The committee has tried to make compliance as easy 
as possible for investigators. 
Ms. Buc said that data reporting is critical because it will lead the RAC in two 
directions. First, it helps to enforce the requirements that the RAC has already 
established. Second, the RAC may begin to categorize certain types of experiments 
based on proven standards of safety. Eventually, experiments labelled as safe could 
qualify for a streamlined review process. The RAC cannot make these decisions without 
solid data. 
Dr. Anderson stated that there are basically two types of data: safety and efficacy. 
Safety data is immediately available to everyone. Fortunately, there have been no side 
effects from any gene therapy protocol in the world. With regard to side effects, the 
Points to Consider require that any side effects must be reported immediately to the 
RAC. Efficacy is the difficult issue. Investigators are fearful that submission of efficacy 
data will be made available to the press. The standard for judging a successful protocol 
for scientists is a published manuscript. These investigators will publish as quickly as 
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Recombinant DNA Research, Volume 16 
