Recombinant DMA Advisoiy Committee - 5/30-31/91 
1. To track the patient through health care providers to maintain a register of the 
names and addresses of the physicians of people who have had gene transfer; 
2. Early and significant assessment of adverse reactions; 
3. To inform the providers of procedures for surveillance, diagnosis or therapy and 
to allow for communication with the health care providers; 
4. To point out to both the research community and the public how many 
procedures have been done and what are the complications that have or have 
not been noted; and, 
5. To allow for a data base to be generated whereby both clinical investigators and 
others who have specific questions will be able to look for a basis for doing 
research in this population. 
He explained that the offices of Mize Information Enterprises would essentially mail a 
questionnaire to health care providers on a twice-annual basis asking for information about 
the patients. All patients would be asked to provide informed consent. Data from either 
health care providers or patients will be reported back to the offices of Mize Information 
Enterprises who will tabulate the data. Whenever a potential adverse reaction is identified 
that information will be reported to Baylor College of Medicine which will immediately 
evaluate the adverse reaction and inform the clinical investigators of the adverse reaction and 
determine whether health care providers and/or the public should be notified of this adverse 
reaction. 
Dr. Ledley noted that this proposal will need to be funded either by the NIH or possibly 
private patient-oriented groups, but that it has the support of the community who have 
thought the most so far about gene therapy. Also, he noted that it could only be successful 
with the support and participation of the investigators performing human gene therapy 
research. He also noted that Dr. Lotze had commented that perhaps the RAC could make 
it a requirement of informed consent forms for this type of research to include a notation that 
such a registry exists and that the patients as well as the investigators should be encouraged 
to participate in the registry. 
Dr. Leventhal said she was not opposed to the proposal, nor was she enthusiastic about it. 
She said there were no valid data to show that the registry would optimize patient care. She 
said that she was familiar with the Bone Marrow Transplant Registry and that it had not been 
particularly useful in determining the efficacy of bone marrow transplantation. She said the 
individual institutional reports of comparative studies had really been what helped to establish 
what is known about the efficacy and toxicity of bone marrow transplantation. She also 
pointed to the Wilm's Tumor Registry as another similar example of a registry which failed 
to identify any further adverse effects of treatment than had already been reported by 
individual clinical investigators. She noted that funding for both these registries had been 
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Recombinant DNA Research, Volume 14 
