Human Gene Therapy Subcommittee - 11/21-22/91 
Dr. Leventhal summarized the draft reporting requirements form. The first section 
requires the investigator to state the dates that the various approvals were obtained. In 
this manner, the HGTS will be able to monitor trouble areas. For example, if an 
investigator applies for FDA and RAC approval simultaneously, useful information will 
be obtained about where the delays occur in the protocol approval process. If a protocol 
is changed at the last phase of the approval process, i.e., the FDA, it is necessary that 
the investigator report any changes that have been made in the original protocol to the 
RAC. Mr. Capron noted that the reporting form as it reads currently would be useful to 
distribute to investigators one year after initial approval; however, it is not entirely clear 
that it would be appropriate for the following years. He suggested that the current form 
should be separated into 2 forms: 1 to be completed initially, and the other to be 
completed annually. Dr. Leventhal agreed that the 2 forms would be an acceptable 
alternative. 
Dr. Parkman said that he had a number of concerns regarding the draft reporting form. 
This form is complicated. In general, the simpler the form is, the higher the compliance 
rate. The investigators have already received approval for their protocols; if the form is 
too complex, most will choose not to complete it, knowing that there will be no course of 
action that the RAC can adopt to deal with noncompliance. He inquired how the RAC 
will analyze the data if the information is submitted by investigators. 
Dr. Parkman explained that it would not be useful for the RAC to receive numerous 
copies of documents because there would not be enough time for the members to review 
the data. An alternative is for the investigator to respond in the form of a cover letter. 
This letter would specifically describe the changes between the original protocol and the 
final approved protocol. Dr. Anderson said that these changes would be an important 
issue for investigators to address. To date, almost every protocol approved by the HGTS 
has changed significantly by the time it has proceeded through the entire review process. 
These changes need to be specifically stated. 
Dr. Leventhal discussed the section of the reporting form that addresses the measure of 
gene transfer success in vitro. Have transduction rates proven to be as expected? Are 
the transduction procedures reproducible? Dr. Parkman suggested that a more effective 
approach to obtaining information is to be more direct. Ask specific questions rather 
than leaving the opportunity for an open-ended response by the investigator. Dr. 
Erickson stated that the form should address what material the investigators are 
administering via what route and whether the protocol is different from what was 
originally proposed. Dr. Parkman said that the goal of the form should be to obtain 
information about whether the outcome of the protocol is the same as predicted; if 
different, how is the outcome different? 
Dr. Leventhal said she was concerned that merely receiving the final version of the 
clinical protocol was not going to provide full information about the procedures that 
were in place. Although many investigators adhere strictly to every detail of a protocol, 
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