11 
public health; he suggested, however, that this reporting requirement might be 
1 included in the revised section of the document dealing with research methods 
and public health concerns. 
I 
Dr. Milewski said Dr. Temin had suggested the document ask how rearrangement 
or recombination will be monitored and address the issue of monitoring 
sensitivity. Dr. Gottesman agreed this type of information should be requested 
for both the animal tests and for tests on the patient's cells in culture. 
Dr. Rich preposed that item 3 of the section entitled Clinical and Public 
Health Considerations (Attachment II) which deals with the number of patients 
J involved in human gene therapy protocols be moved to the section of the document 
j dealing with selection of subjects. 
Dr. Miller suggested the language of item 6 of Clinical and Public Health 
Considerations which asks about the procedures for follow-up of patients and 
autopsies be rqolaced with the words "Describe intended follow-up." Dr. Grobstein 
pointed out that follow-up of negative results may be just as important as 
follcw-up of positive results and suggested the phrase "Describe method and 
duration of patient followings" be substituted for item 6. 
j 
Dr. Walters called the attention of the working group to item 4 of Clinical 
; and Public Health Considerations vhich reads as follows: 
"What are the clinical endpoints of the study? How will patients be 
monitored to assess specific effects of treatment on the disease. 
Describe clinical and laboratory follow-up studies. How frequently 
will such studies be done? 
"N.B. Note that federal rules require periodic reporting to the local IRB. 
How often will this be done? In addition, it is requested that the 
investigators report to the working groups of the Recombinant ENA Mvisory 
Carmittee regarding follow-up of the gene transfer experiment at six and 
twelve month intervals following gene therapy. " 
Dr. Miller said monitoring requirements should also specify that the patient be 
observed for side effects. Mr. Capron suggested the working group document 
request monitoring procedures be described. The working group agreed monitoring 
for side effects should be part of the informational request under item 4. 
Mr. Capron said the note in item 4 was an important informational request and 
should be a separate item. He also suggested a statement be added to item 4 
regarding routine monitoring and emergency reporting to the Office of Protection 
from Research Risks. 
The working group agreed the first sentence of item 7 dealing with risk to 
individuals other than the patient should be modified to ask what precautions 
are being taken, if any, to protect others. The working group also agreed to 
compose an informational request asking whether the investigator suspects 
that recombinant DNA may spread from the patient to others. The working greup 
agreed to delete the portion of the second sentence of item 7 vhich suggested 
retroviral infections might present a risk. 
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