10 
Dr. Walters explained that in an attenpt to perform gene therapy in 1980, 
the investigator proposed treating the recombinant DNA with restriction 
nuclease and administering the resulting ENA fragments to the patients. 
The working group wishes to make clear that this type of procedure is new 
covered by Section III-A-4 of the NIH Guidelines and by the points to con- 
sider document. 
Dr. Davis suggested the language in the first footnote might be improved. 
Several RAC members agreed. 
Dr. Wens ink asked whether the working group might be inappropriately "sec- 
ond guessing" IRBs by dealing with issues such as selection of patients, 
informed consent, privacy, and confidentiality vhich generally fell within 
IRB purview. 
Dr. Walters said the working group evaluated the general regulations on 
research involving human subjects and cited those sections which have 
particular applicability to human gene therapy. The working group does 
not request specific answers but simply asks IRBs and investigators to 
think about a series of questions. The working group is not trying to 
second guess the IRBs; it is only attempting to have the same general 
questions considered by all groups. Dr. Davis asked whether language 
should be inserted in the points to consider document stating that the 
working group is not atterpting to supplant IRB functions. 
Dr. Gottesman said Drs. Davis and Wensirik had raised important questions 
concerning the relationship of the working group to IRBs. The working 
group has discussed this relationship at length. The working group does 
not wish to second guess IRBs but wishes to be certain every issue is 
properly considered particularly in the initial protocols. 
Dr. Walters said it is difficult to predict hew local IRBs will approach 
gene therapy issues. However, the Introduction of the points to consider 
document indicates flexibility by stating that not every point in the 
document will require attention in every proposal. 
Dr. Johnson said RAC and the working group are not regulatory bodies and 
the points to consider are not regulations. These points are sinply a 
listing of considerations relevant to human gene therapy. 
Mr. Daloz said RAC and its working group will review proposals on a case- 
by-case basis. RAC and the working group will then evaluate hew well the 
points to consider work in an actual review situation. 
Dr. Landy asked why the section on "Applicability" was added to the document 
he especially questioned the inclusion of the word "only." Dr. Gartland 
said this language was added at FDA's request to emphasize that NIH is not 
attempting to usurp FDA responsibilities. 
[392] 
