3 
Dr. Miller said the FDA staff manual might be useful in developing portions of 
the guidance document. He offered the example of procedures for testing the 
vector preparation for adventitious contaminants; the FDA staff manual describes 
such procedures. 
Dr. Gottesman suggested the guidance document might consist of two parts, i.e., 
one part deeding with immediate considerations such as scientific protocols and 
patient rights, and a second part deeding with wider social and ethical considera- 
tions. Issues used to develop the document might include: (1) checking for a 
complete and thorough IRB review; (2) addressing whether special concerns exist 
because the experiments involve human gene therapy; (3) addressing scientific 
issues the IRB could not or might not deal with; (4) determining the intent of 
the therapy; and (5) evaluating long-term social considerations. 
Mr. Capron then proposed an outline for the draft document. He suggested that 
this framework might be used to devise Part I of the type of document suggested 
by Dr. Gottesman. Other members of the subgroup then polished the proposed 
outline. The outline reads as follows: 
I. Risk Mangement 
II. Risk Benefit 
A. Disease (description, including alternative therapies) 
B. Research Method 
1. Delivery system (what and how) 
2. Expression 
3. Safety of Research Method (animals and patients) 
C. Statistical Considerations (Is the experiment sufficiently well 
planned? ) 
D. Qualifications of the Team/lnstitution/Hospital 
III. Selection of Patients /Subjects 
IV. Consent 
V. Consent 
VI. Monitoring ( knowledge and contingency) 
VII. Privacy 
The subgroup then categorized the questions (Attachments I and II) sent to the 
working group by Drs. Motulsky and Temin under these rubrics. 
[6] 
