Human Gene Therapy Subcommittee - 11/21-22/91 
responsible for collecting this information? Dr. Leventhal suggested that the HGTS 
returned to a discussion of the draft reporting form. 
Dr. Leventhal suggested that the section regarding in vivo gene transfer success could be 
reworded to say, "Has there been any evidence of activity of the transferred gene? How 
does this compare with the anticipated outcome?" Dr. Parkman explained that there are 
two categories of outcome, positive effects and negative effects. This section should 
specify that information is required about the positive results as well as any adverse 
consequences. Dr. Erickson agreed that he would like to separate this section into 
positive and negative effects. In addition, investigators must provide an expanded 
explanation of their answer, not just a yes or no answer. 
Dr. Parkman said that the HGTS can argue whether any or all of this information is 
pertinent. However, the point is: Will investigators actually comply with this request? 
Dr. Erickson noted that the Points to Consider states that reports should be submitted 
every six months and suggested that the draft reporting form be developed as a set of 
guidelines that investigators can follow in their semi-annual reports. Dr. Leventhal 
stated that perhaps the question relating to effects on other genes should be omitted 
since this question is already addressed as part of the Points to Consider. Dr. Parkman 
noted that such an effect would be considered a deleterious effect of the therapy and 
should be specifically addressed. Dr. Neiman stated that this question should yield an 
informative response and should remain as part of the reporting form. Dr. Mclvor asked 
for clarification of the question, "Is there evidence of deterioration of disease state in 
relation to therapy?" Does this address the scenario that the patient's condition has 
improved? Dr. Leventhal replied that this question only addressed the patient's status if 
there is disease progression, not improvement. Dr. Mclvor suggested that a specific 
question be incorporated regarding improvement in the patient's status as a result of the 
therapy. 
Dr. Leventhal discussed the patient accrual section. Have the investigators been able to 
treat the number of patients that they originally proposed? Have patients been rejected? 
On what basis? Are patient accrual goals being met in a timely fashion? If not, explain 
why. Dr. Leventhal concluded, noting that the reporting form requires that the 
investigators submit publications that have resulted from their studies. Dr. Walters 
thanked Dr. Leventhal's working group for creating the draft form as a starting point for 
discussions on data management. 
Dr. Leventhal asked the member# of the HGTS if the form should be approved and 
forwarded to the RAC. By what mechanisms can this form be distributed and enforced? 
Dr. Wivel stated that the RAC should review the form and vote on whether it should be 
recommended for approval to the Director of NIH. Dr. Leventhal suggested that the 
reporting form should be on the agenda for the February 1992 RAC meeting. Dr. Wivel 
agreed to add the revised version of the Working Group on Data Management's report 
onto the next RAC agenda. 
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