9 
Dr. Miller said the FDA may not review human gene therapy protocols. Ms. Areen 
felt in such a situation FDA concerns beccme part of the charge of the Working 
Group on Human Gene Therapy. She felt the working group should construct a 
document which will elicit the information necessary for a complete re/ie^ even 
in the absence of FDA review. 
Dr. Gottesman did not feel the working group would rqolace FDA functions. She 
thought the IRB would, however, consider same FDA concerns. 
Mr. Capron said Drs. Mot ul sky and Anderson objected at the October 12, 1984, 
meeting to including the FDA points in the working group document (Attachment 
II) ; many of the FDA points to consider apply to manufacturing procedures vhile 
the working group's points to consider apply to research. In addition, the FDA 
procedures were developed to apply to products such as vaccines which would be 
distributed to large numbers of healthy school children; this population would 
not be candidates for human gene therapy. Mr. Capron said at the October 12, 
1984, meeting the working group had agreed with this point of view. He said 
the working group should new focus on the germaine points and suggested 
Dr. Miller might privately inform Dr. Anderson of his concerns. 
Dr. Walters said the primary focus of the working group document is to evaluate 
the safety and efficacy of human gene therapy protocols. The subgroup of 
scientists had concluded this document is sufficient frcm that point of view. 
Dr. Walters suggested Dr. Miller might forward his comments to Dr. Anderson 
who, in consultation with other scientific experts in this area, had composed 
this portion of the document. 
Dr. Gottesman suggested Dr. Miller communicate his concerns to Dr. Anderson; 
Dr. Anderson would decide whether those comments were valid and whether addi- 
tional detail should be incorporated into this portion of the working group 
document. If Drs. Miller and Anderson disagree on whether certain language 
should be included in the document, the working group might resolve the issue 
at the next meeting. The working group will then have an opportunity to review 
the document (Attachment II) when it reviews public comments. Dr. Gottesman 
suggested the working group proceed with review of the document and retain as 
written that portion composed by Dr. Anderson. Dr. Grobstein and Mr. Capron 
felt Dr. Miller should comment during the public comment period. 
Mr. Capron supported the suggestion to accept as part of the working greup 
document the language composed by Dr. Anderson. 
Dr. Grobstein said the working group and the FDA have the same objective; that 
the materials used in human gene therapy should be safe. The differences 
between the working group and the FDA arise in the method chosen to arrive at 
that goal. 
Dr. Harmison said all involved parties would attempt to ensure the quality of 
the science. No good scientist would ignore the considerations detailed in 
the FDA documents; insteal they would plan experiments vhich would be safe and 
efficacious and produce accurate results. Dr. Grobstein agreed; he pointed 
out that the section of the working group document entitled Research Methods 
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