8 
Dr. Rich felt the rationale should ask why particular patient groups had been 
selected. Dr. Walters asked if this issue might be addressed in the clinical 
and public health considerations portion of the document. Mr. Capron felt 
language dealing with this consideration should be in the portion of the draft 
entitled Selection of Subjects (Attachment II). 
Dr. Miller felt Section l-(b) of Objectives and Rationale was redundant and was 
subsumed by Section l-(a). Dr. Grobstein felt Section l-(b) asked the particular 
reasons for choosing to treat this disease at this time. Dr. Walters said 
Section l-(a) requests the rationale for the therapeutic approach; Section l-(b) 
asks why the investigator chose to treat the particular disease. 
Dr. Gorovitz suggested the language of Section l-(b) be modified to specifically 
ask why the disease is an appropriate candidate for therapy at this time. The 
working group does not wish to receive historical or biographical explanations 
but information on disease characteristics. 
Mr. Capron suggested the phrase "possible to assess results" be substituted for 
the word "unambiguous" in Section l-(c). 
Dr. Gorovitz suggested the word "effective" in Section l-(d) be defined. 
He thought a more descriptive phrase would be "vhat are their relative 
disadvantages . " 
Dr. Walters called the attention of the working group to the portion of the 
document entitled Research Methods ' (Attachment II). 
Dr. Miller said he had numerous specific conments on this portion of the docu- 
ment; these comments deal with issues such as stability and purity of product. 
Dr. Childress asked if Dr. Miller's points were substantive or whether they 
were simply a question of adding more detail to the working group document. 
Dr. Miller said he washed to see more detail in the document. 
Dr. Gorovitz asked whether the working group could integrate Dr. Miller's com- 
ments into this portion of the document in Dr. Anderson's absence. He said 
the most efficient way to proceed was for Dr. Miller to c am unicate his con- 
cerns directly to Dr. Anderson. 
Dr. Gottesman said this portion of the document had been widely circulated 
among scientific experts, and the question of how specific the document should 
be in detailing data requirements had been discussed. Dr. Gottesman did not 
feel the level of detail Dr. Miller wished to include in this portion of the 
document was necessary. She thought the current document addresses all the 
relevant issues. She noted that the working group will be polishing this 
language in the future, and if greater detail appears to be necessary, the 
working group could modify the document at that time. Dr. Gottesman did not 
think the working group document should specifically attempt to address issues 
in FDA's regulatory jurisdiction. 
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