Recombinant DNA Advisory Committee - 01/14/93 
Ms. Buc said that she had several suggestions for separating the RACs role from NIH's 
role on this issue. During the course of this discussion, the RAC must consider the role 
of an advisory committee versus the government. Advisory committees advise and 
governments govern. The RAC serves an enormously important purpose of exploring, 
discussing, and providing a wide variety of expertise. But as its name states, the RAC is 
simply an advisory committee to the NIH Director. The NIH Director has the authority 
and responsibility to decide on each protocol that is presented to the RAC. 
Ms. Buc stated that Dr. Healy had ample legal authority to make this decision. Perhaps 
more important, she attempted to include the RAC and other individuals in the process. 
Dr. Healy should exercise her authority to grant compassionate use exemptions for gene 
therapy, consult the RAC if possible, and notify the public by an appropriate mechanism. 
Ms. Buc suggested that discussion should focus towards reducing the RACs formal 
exposure to compassionate use requests; however, the committee should assist NIH in 
establishing guidelines for the consideration of such requests. 
Dr. Chase stated that he respected the process by which Dr. Healy made her decision 
regarding Dr. Royston's compassionate plea request. Dr. Chase said that in his opinion, 
the December 1992 RAC meeting would have been more productive if Dr. Healy had 
been present. The RAC would have attempted to support Dr. Healy's original position 
that Dr. Royston's request should not be granted because of both inadequate safety 
review and the lack of demonstrable evidence that the treatment would be efficacious. 
Dr. Healy said that when Dr. Royston's patient was originally brought to NIH's attention 
in October 1992, no safety data had been obtained. This safety documentation was 
submitted in December 1992, around the time of the RAC meeting. Dr. Healy stated 
that her decision to grant this request was based on recent safety data and the advice of 
NIH staff. Safety was the overriding issue that governed the FDA's deliberations. This 
informed judgement was based on new information submitted to the NIH and the FDA. 
Dr. Walters instructed the RAC members and other NIH officials to examine the 3 -page 
proposal that was developed by the working group on compassionate plea exemptions. 
Dr. Walters outlined the working group's proposal. The introductory statement of this 
document explains that the ideal submission and review for compassionate use requests, 
including single patient protocols, is eight weeks in advance of a scheduled RAC 
meeting. However, in the event that this time-frame cannot be maintained, the working 
group proposed the following options: (1) Expedited RAC review, either special 
meetings between regularly scheduled RAC meetings, or some similar mechanism for 
reviewing compassionate plea requests that arise between meetings where emergency 
action is needed. (2) Referral to previously approved protocols; however, the RAC 
must consider the situation where a protocol already has a long waiting list of patients 
who meet the appropriate inclusion/exclusion criteria. (3) Allow new investigators to 
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Recombinant DNA Research, Volume 17 
