Attachment IV - Page 22 
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17. We suggest the addition of a section at the end of the document such as 
the following, "For information on the jurisdiction of the Food and Drug 
Administration f FDA 1 over products employed for human gene therapy, please 
refer to the FDA's "Statement of Policy for Regiil at 1 ' ng Biotechnology 
Products", Federal Register PS?, 50878, 1984. 
18. We are concerned that the interrogatory style of the document may he 
interpreted as strident or even adversary. Moreover, this style does not 
provide the rationale for manv of the questions although arguably the 
rationales could he invaluable for the formulation of responses. We 
believe that both the tone of the document and the quality of 
communication between NIH and investigators would be improved by a more 
explicative, collaborative approach. As an example of this, Attachment A 
is the FDA's draft "Points to Consider in the Production and Testing of 
New Drugs and Biologicals Produced hv Recombinant DNA Technology." 
19. We have one additional concern. There exists virtual unanimity among 
those conversant with human gene therapy that somatic-cell gene therapy is 
analogous to other conventional medical interventions, such as 
vaccination, exogenous replacement of insulin in diabetics, etc. The 
conclusion of the recent OTA Report, "Human Gene Therapy-Packground Paper" 
is instructive: 
Civic, religious, scientific, and medical groups have all accepted, 
in principle, the appropri ateness of gene therapy of somatic cells in 
human for specific genetic diseases. Somatic cell gene therapy -is 
seen as an extension of present methods of therapy that might be 
preferable to other technologies. 
The FDA has also endorsed this view in principle and has made known its 
intention to subject appropriate products employed for human gene therapy 
to an administrative review similar to that for analogous biological drug 
products. Since as implied above, most of the critical decisions on 
individual investigators' submissions are likely to be scientific and 
medical ones, we believe that it is imperative to reconstitute the 
membership of the RAF Working Group on Human Gene Therapy r which will 
conduct the initial administrative review of proposals! which currently 
includes a large proportion of attorneys, ethicists and public policy 
specialists. The Working Group should be supplemented with more members 
with expertise in molecular bioiogv, pharmacology, medicinal chemistry, 
and medicine. Moreover, we believe that the chair should be a scientist, 
preferably a physician, with expertise in a field closely allied to 
molecular biology, internal medicine, or genetics. 
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