Recombinant DMA Advisory Committee - 5/30-31/91 
Dr. Ledley then went on to respond to the comments of the reviewers. He said that the 
concept of a gene registry is one which had been discussed in the past and in fact the 
investigators had been prodded by people at the national level to begin to look at a 
mechanism for setting this up. Secondly, he said that Ms. Buc's comments about 
communication with the patients was a key point and he said that a WATS line was 
envisioned to be set up as part of the registry so that the patients can call to ask questions. 
He said they were nervous about this in the sense that ^ey are not the patient's treating 
physician and that all they will be able to tell the patient in many cases is to contact their 
treating physician. Also, he noted the problem of dealing with patients who move from 
pediatric care to adult care is a problem in the cancer registries and that patients are lost to 
follow-up in this process. 
Dr. Ledley explained the process of measuring an adverse reaction and discussed patterns of 
adverse reactions as well as long-term focus on certain groups of patients. He said he was 
aware of the problems surrounding funding. He said he felt that if the registry is established 
the companies involved in gene therapy research would pay for the registry. He stressed that 
he felt it was important that the concept gain the bacldng of the RAC and that a positive 
response by the RAC would impact on the possibility of acquiring funding. 
Dr. Anderson said that for the foreseeable future every physician who treats a patient with 
gene transfer/gene therapy is going to come before the RAC and that if the RAC is going to 
continue lifetime follow-up for patients he felt it inconsistent that it would feel uncomfortable 
in supporting such a registry. 
Dr. Leventhal said there was no requirement for lifetime follow-up, but merely that all the 
data go be maintained in one place. Further, she said no one had said it should be 
disapproved, it was just that the level of enthusiasm was not great. 
Dr. Anderson said what was being sought was support for the concept of a registry. As far 
as who is going to perform the work of starting up and maintaining a registry, he said that it 
would be difficult to send out bids and expect to get someone who would do this. He said an 
ROl was out of the question in light of current budget restraints. He noted that as far as 
funding was concerned NICHD might be open to putting some money into this, as well as 
other organizations, and that it's very possible, as Dr. Ledley said, that some of the biotech 
firms involved in gene therapy would be open to contributing to maintain such a registry. 
Dr. Walters pointed out that the reason the HGTS had not discussed this proposal in detail 
was that it had come in after the agenda had been published for its last meeting and was 
unable to be placed on the agenda at that late date. 
Dr. Mclvor said that since he was involved in gene therapy research and that he felt the data 
generated by such an endeavor may prove valuable in the long-run, that he would make a 
motion. Dr. Mclvor moved that the RAC recognize the potential usefulness of an established 
registry of patients, clinicians and scientists involved in human gene transfer trials, without any 
[662] 
Recombinant DNA Research, Volume 14 
