8 
Dr. Grobstein questioned whether Section I-A-l of the working group document 
should ask why the investigator wishes to treat a specific disease. He suggested 
a preamble bo Section I might request this information as part of the description 
of the natural history of the disease. He noted that an investigator might 
have criteria which the working group had not envisaged. 
nr. Gottesman said the working group document should require a summary as part 
of the submission. This summary should state why the disease was chosen ard 
what criteria would be used to gauge success. Dr. Gottesman said Section I-B, 
Prior Laboratory Research , of the document should pose detailed questions on 
test parameters. 
Dr. Mot ul sky asked if the working group should address the question of whether 
a patient in the more advanced stages of a disease should be treated first or 
the patient in earlier stages of the illness. 
Dr. Anderson noted that in many of these illnesses the young patient is frequently 
not yet as affected by the disease ard thus might most benefit from therapy. 
Oi the other hand, the older patient might also be helped; and the use of the 
procedure in such patients might be ethically more acceptable. 
Dr. Anderson said an investigator does not wish to discover after the proposal 
has been submitted for review that the working group has a bias or prejudgement 
concerning the type of candidate the working group would prefer as the first 
patient. He said the investigator would like to have some indication of the 
working group bias if a bias exists. Dr. Motulsky felt the working group would 
have to proceed on a case-by-case basis, but thought this issue might be 
discussed in the preamble to the working group document. 
Dr. Miller called the attention of the working group to the FDA manual, IND 
Medical Review Guidelines (Attachment III). He said the FEA manual offers a 
general indication of the information required for review and then details a 
specific comprehensive list of necessary information. He suggested the working 
group incorporate this portion of the FDA manual into the working group document. 
Mr. Capron disagreed with Dr. Miller's proposal; he thought the working group 
draft document more specifically addresses issues in human gene therapy than 
does the FDA manual . Dr. Miller said the working group would have to assure 
the identity and potency of the ENA preparation and would have to develop 
prodecures for stratifying patients; detailed requests for these types of 
information are listed in the FDA manual. Dr. Capron suggested the FDA manual 
might be referenced by the working group document in order to avoid unnecessary 
duplication of information. 
Dr. Miller felt simply referencing the FDA manual would produce an incomplete 
document; the FDA manual is not publically available even though it is in the 
public domain. Ms. Areen supported Dr. Miller's suggestion that the information 
from the FDA manual be incorporated into the working group draft document. 
Dr. Walters suggested most of the relevant information from the FDA manual 
(Attachment III) has already been incorporated into the working group document 
[ 96 ] 
