Recombinant DMA Advisory Committee - 5/30-31/91 
discontinued by NIH in light of the current federal budget crisis. 
In summary, she said she supported the concept of a registry but not with a sense of high 
priority or urgency. Further, she said she did not feel the RAC should make it a requirement 
of approving a protocol that investigators in clinical trials participate in a registry. 
Dr. Geiduschek said he and Dr. Leventhal had arrived at similar conclusions by different 
routes. He said the idea of setting up and running a data base to follow gene therapy patients 
is a good one which he thought would receive general approval and would be acceptable to 
the public. However, he felt the RAC should not put a stamp of "exclusive approval" on sucH 
an individual project, and that perhaps a general call for proposals for such a registry should 
be made and allow them to be judged on a competitive basis. He said if the committee 
disagreed with this view and wished to specifically approve this proposal he would make 
additional comments as regards to specific portions of the proposal. However, he commended 
Dr. Ledley for his proposal and thanked him for his work in submitting it. 
Dr. Doi had some specific questions on the proposal but said he felt the proposal, if it worketl 
in the way which was presented would provide useful information to the scientific community, 
the patients, and the public and that the whole concept of having the medical, scientific, and 
ethical answers from this data was a good one. 
Ms. Buc said that the idea of a gene therapy registry seemed to be a useful one, but that she 
felt one thing needed was an ability for the patient to contact the registry, possibly through 
an free WATS line to be able to communicate with the registry. Furthermore, she added that 
with such a long-range endeavor thought should be given to who would be a successor 
principal investigator for such an effort. 
Dr. Krogstad asked if Dr. Ledley had given any thought as to whether ROl funding for this 
project would be the way to go since it is a long-range project and whether there might be 
other funding mechanisms which would be more practical. Further, he asked if Dr. Ledley 
had contemplated a control group for inclusion in the registry so that valid epidemiological 
data could be ascertained. 
Dr. Miller said his own bias was that such a registry might not be appropriate to be funded 
with public monies. Secondly, he said that if such a registry were developed, he wanted to 
know what types of gene therapy were envisioned to be included in the registry. And finally. 
Dr. Miller asked whether the investigators had thought about any mechanism for getting 
companies who are carrying out gene therapy to participate in the registry since it is likely that 
this may likely be the largest group of patients before too long. 
Dr. Mclvor asked for clarification on the term 'Tjiaimually." He suggested that if the proposal 
was to send out a questioimaire every six months that "semi-aimually" was a better term. Dr. 
Ledley said he would note this and ensure it was changed. 
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