Recombinant DNA Advisory Committee - 01/14/93 
Dr. Healy stated that in the context of this pressing matter, the RAC is charged with 
developing recommendations that will provide a long-term procedure for handling 
compassionate plea requests for gene therapy. In executing this charge, the RAC needs 
to consider the following questions: (1) What should the scope of the RAC's role be in 
reviewing non-research compassionate plea requests for gene therapy? (2) How will the 
circumstances under which a non-research compassionate plea request for NIH approval 
be defined? (3) Does the RAC, as currently constituted, have adequate expertise to 
review individual patient requests? (4) How can a time-sensitive mechanism be 
developed for handling these requests that involves the public? This response must 
balance the need for public information with the needs of the patient for privacy and 
timely review. (5) How should the RAC mandate be expanded or enhanced to 
accommodate this new responsibility? 
Dr. Healy concluded her remarks noting that the evolution of the NIH Guidelines must 
reflect the NIH's mission to apply scientific knowledge in order to extend the health of 
human lives and to reduce the burden resulting from their disease and disability. It is in 
this spirit that the RAC is asked to undertake this task. 
COMMITTEE'S DISCUSSION 
Dr. Walters asked if the RAC members' had any questions and comments for Dr. Healy. 
The committee should focus their discussion on the five questions outlined by Dr. Healy. 
Dr. Walters recognized the RAC members who were joining the discussion via 
conference call, namely, Mr. Capron, Dr. Geiduschek, and Dr. Schaechter. Dr. Walters 
noted the order in which speakers would be recognized. The RAC members will have 
the first opportunity to respond, followed by liaison representatives, other NIH 
employees, members of the public who have submitted a formal request to address the 
committee, followed by the public at large. 
Dr. Parkman said that many of the RAC members have had extensive experience with 
the expedited approval or compassionate use process. He inquired whether 
compassionate use implies the probability of efficacy. Specifically, is a Phase I study a 
potential topic for compassionate use? There is a potential ethical and societal 
difference between therapy that has the probability of efficacy and the rights of all 
people to have access to that therapy, regardless if the intent is purely experimental. 
Dr. Parkman explained that the process for determining the answers to these Phase I 
compassionate use questions is already in place at the level of the IRB. Many IRBs 
have a ruling that a compassionate use protocol that presents significant risk has to be 
reviewed by the full body, whereas a protocol that provides minimal risk can be 
administratively approved by the IRB Chair using any assistance necessary. It is 
important that the RAC adopt the same criteria for the assessment of compassionate use 
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