9 
(Attachment II). He suggested the working group compare the FDA manual to the 
draft document. 
Dr. McCarthy questioned whether the working group document should address the 
issue of "costs;" costs could include the cost of funding the basic research, 
the cost to the institution, and the cost to the patient. He said currently 
the question of what costs might or should be passed on to the patient is a 
focus of public debate. Dr. McCarthy thought the working group should consider 
the cost issue in greater detail as the IRBs have neither the expertise nor the 
information necessary to evaluate this topic. Mr. Capron said the language of 
the working group document only refers to a comparison of the cost of gene 
therapy to the cost of alternative therapies. Dr. Gottesman felt the working 
group might consider the question of who controls access to human gene therapy 
when the federal government finds the research. 
Dr. Gorovitz said the patient should be informed of the projected cost of the 
treatment in the consent agreement before therapy begins. 
Dr. Motulsky said the working group should consider whether restraints imposed 
on human gene therapy might affect the type of therapy eventually available to 
the public. Such a development might be detrimental to the public; therapies 
which are less successful or "good" might became more widely available because 
they had not been restrained. Dr. Anderson agreed the working group must walk 
a fine line between reviewing the issues and delaying research. 
Dr. Miller and Ms. Areen offered the working group an alternative document to 
the draft (Attachment II) developed by Dr. Walters and the volunteer subgroup. 
The document proposed by Dr. Miller and Ms. Areen would eliminate much of Part 
I of the working group document (Attachment II) and substitute portions of the 
FDA manual (Attachment III). 
Dr. Axel strongly protested that the alternative document preposed by Dr. Miller 
and Ms. Areen did not begin to approach the issues before the working group. 
He pointed cut that the vectors most likely to be used in human gene therapy 
would be modified retroviruses; the FDA manual had been developed to apply to 
pharmaceuticals and should not be applied to recombinant vectors. 
Dr. Gottesman thought the requirements of reviewing protocols involving human 
gene therapy quite different from the requirements specified by the FDA manual 
for reviewing pharmaceuticals. She suggested the working group retain the 
working group document (Attachment II) but reexamine the FDA manual to determine 
if any pertinent points might have been emitted from the working group draft 
document. 
Dr. Motulsky said the alternative document proposed by Dr. Miller and Ms. Areen 
was not relevant and was disrupting the meeting. He suggested the working group 
continue to develop a draft based on the document constructed by Dr. Walters 
and the subgroup of volunteers. Dr. Motulsky pointed out that the working 
group was attempting to develop guidance for the novel use of a biological 
system; such a guidance document should be developed from the ground up. 
Dr . Varmus agreed . 
[ 97 ] 
