21 
Dr. Davis specifically disagreed with the suggestion that a representative 
of industry be added to the working group; such an addition might give the 
mistaken impression that human gene therapy is or will soon be a large-scale 
industrial activity. 
Mr. Robert Lanman, the NIH legal advisor, argued that the points to consider 
should not request a ccpy of the patient consent form. Dr. Walters said 
questions about the quality of consent forms have recently been raised for 
sane innovative therapies. Mr. Mitchell said the working group was very 
sensitive about this issue and felt in the initial trials the consent form 
should be above reproach. 
Dr. Rapp said huiran gene therapy protocols will be asking for agreements 
different from those requested in most clinical investigations. For example 
in human gene therapy protocols, the subject will be asked to participate 
in long-term followup and to give autopsy permission in advance. For this 
reason, he thought the working group should look at the consent forms for 
the initial gene therapy protocols. 
Dr. Miller said FDA has severed comments on the points to consider document. 
He said the introduction of the document does not accurately represent FDA's 
role in regulating these products. The introduction implies FDA jurisdic- 
tion will begin only vhen somatic-cell gene therapy protocols became more 
widely applied and use manufactured biological materials. Dr. Miller said 
FDA does not regulate gene therapy or technologies per se , but does regulate 
'new drug products as defined in regulations. Dr. Miller said FDA has regula- 
tory jurisdiction vhen a new drug product is used to effect human gene 
therapy, and FDA will indeed be overseeing human gene therapy experiments. 
Dr. Miller proposed the following language be substituted for the last 
sentence of item (5) of the document's Introduction: "For information on 
the jurisdiction of the Food and Drug Admininstration over products employed 
for human gene therapy, please refer to the FDA's Statement of Ftolicy. " 
Dr. Miller said item (8) of the Introduction misrepresents the aim of germ 
line therapy by stating the aim is "changing the set of genes passed on to 
the individual's offspring." He continued: 
"...in general, the object of gene therapy is to the cure the individual 
vho will result from the embryo that is being manipulated. The effects 
on subsequent generations are usually a secondary effect and indeed 
the object is to cure that individual." 
Referring to item (11) of the Introduction, Dr. Miller said “we believe [this 
item] states the question that any degree of teratogenesis is not acceptable." 
He said in conventional medical interventions, clearly that is not the case. 
Oblative radiotherapy before a bone marrow transplant, for example, for 
leukemia is considered acceptable. Oblative radiotherapy produces sterili- 
zation, and the risks to benefits in that situation are considered appropriate. 
Similarly, certain regimens of chemotherapy induce sterility or are clearly 
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