Recombinant DNA Advisory Committee - 03/1-2/93 
submit the Points to Consider. Dr. Parkman said that the Cover Sheet for Expedited 
Review is not intended to outline all of the documentation that is required for 
submission; it is intended as a screening process. 
Dr. Haselkorn stated his objection to the use of the word "therapy" in item number 4 of 
the Cover Sheet for Expedited Review. Dr. Wivel suggested that the word "transfer" be 
substituted for the word "therapy". Dr. Haselkorn agreed to this substitute wording. Dr. 
Haselkorn suggested that expedited review should only be considered for post-Phase I 
trials. Phase I studies are inappropriate for expedited review since there is no 
demonstrated therapeutic value. 
Dr. Zallen suggested that the RAC should consider the public's perception and whether 
they will view these investigators as trying to take advantage of desperate individuals who 
can afford to pay for medical interventions. If the public develops this perception, the 
integrity of gene therapy research may be called into question. She suggested that a 
statement be included in the Procedures for Expedited Review requiring that all costs 
associated with the experimental protocol will not be borne by the patient or the 
patient's family. Inclusion of this criterion would maintain public confidence. Ms. 
Meyers agreed that the provision outlined by Dr. Zallen should be included in the final 
document. Dr. Leventhal reminded the RAC that this requirement would not preclude 
patients or their families from making monetary donations to an institution. 
Dr. Chase stated that the RAC should remain confident that making this document "air- 
tight" will develop into long-term implications. One important factor that will be the 
peer pressure of responsible scientists in the area of gene therapy. Eventually, there may 
be an attempt to discredit those investigators who repeatedly make these types of 
requests. The other factor that will weigh heavily is the parallel recommendation by the 
RAC following NIH Director's determination. Eventually, the media and other 
individuals will influence public opinion. The public will realize that some of the 
experiments will have no medical or scientific value. It may take several years for the 
public to actually realize the implications of these expedited approvals. Dr. Krogstad 
recommended that item number 6 of the Procedures for Expedited Review include 
extramural scientists in the review of these protocols. In light of previous discussions 
about the possibility of political pressure being imposed on the review process, the 
inclusion of extramural scientists' review would avoid the appearance of such pressure 
being imposed. 
Dr. Leventhal suggested that a question should be included in the Cover Sheet which 
requires that the investigators define the endpoint of the study, i.e., what will be 
measured? A clear example of the importance of endpoints is the patient who received 
expedited approval in December 1992. The RAC was told that the patient required 
emergency treatment because the tumor was growing rapidly. However, the RAC was 
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