PHARMACEUTICAL 
JOHN JENNINGS, M. D. 
VICE PRESIDENT 
SCIENCE AND TECHNOLOGY 
MOO FIFTEENTH STREET, N. W. 
WASHINGTON, D. C. 20005 
AREA CODE (202) S35-3540 
CABLE-PHARM/WASHINGTON, D. C- 
TWX-7IOQ22 9494-PMA-WSH 
Apri 111, 1985 
Director 
Office of Recombinant DNA Activities 
Building 31, Room 3B10 
National Institutes of Health 
Bethesda, MD 20205 
Dear Sirs: 
Re: "Points to Consider in the Design 
And Submission of Human Somatic- 
Cell Gene Therapy Protocols" 
The Pharmaceutical Manufacturers Association is a nonprofit trade 
association representing 120 manufacturers of prescription phar- 
-maceut ica Is , biologicals, in vivo and in vitro diagnostic 
products. Gene therapy is a therapeutic development which 
evolves, in part, from the knowledge and extensive experience of 
pharmaceutical research and development, and as a subject for 
public comment, it is of concern to the pharmaceutical industry. 
We, therefore, are pleased to offer the following comments on the 
recent NIH Federal Register notice entitled "Points to Consider 
in the Design and Submission of Human Somatic-Cell Gene Therapy 
Protocols. " 
The notice provides a balanced overview of and perspective on the 
short term or practical considerations regarding clinical exper- 
imentation in humans involving somatic-cell therapy, as well as 
the longer term social and ethical implications of research in 
this area. However, the PMA suggests that, in the preamble, a 
clear distinction be made between somatic-cell therapy and 
germ cell therapy. The Congressional Office of Technology As- 
sessment, in its report entitled "Human Gene Therapy -- Back- 
ground Paper", noted that "...because cells that are used in re- 
production are not involved, gene therapy (in somatic cells) is 
quite similar to other kinds of medical therapy, and does not 
pose new kinds of risks. When considering gene therapy that does 
not result in inherited change, the factor that most distinguish- 
es it from other medical technologies is its conspicuousness in 
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