Recombinant DNA Advisory Committee - 01/14/93 
never resubmitted the protocol. The RAC never heard from Dr. Royston until he 
returned to NIH with a request for a compassionate plea exemption. The RAC has still 
never received a complete submission. The information submitted to the NIH is 
completely unsatisfactory. 
Ms. Meyers stated that the informed consent document that Dr. Royston's patient signed 
was untruthful. Dr. Royston has continually stated that, this is the patient's last chance , 
even though every piece of information submitted to the RAC indicates that this 
protocol will provide no therapeutic benefit. Dr. Royston's patient will not benefit from 
this treatment. The approval of this one compassionate plea request will open the door 
to thousands of similar requests, all asking to receive gene therapy that has not shown 
any effectiveness at all. 
Dr. Chase said that there are aspects of this particular case that make consideration of 
the general problem difficult. These aspects are as follows: (1) there is the issue of 
equity of access; (2) there is a lack of public scrutiny regarding the ultimate decision 
process; (3) public involvement has been eliminated, which is an important principle 
underlying the function of the RAC; (3) there is a lack of scientific evidence supporting 
efficacy of the treatment; (4) there is the possibility that these extraordinary steps were 
taken in order to evade a more stringent review; and (5) there has been a derangement 
of normal Federal government processes. 
VII. STATEMENT-DR. ROYSTON 
Dr. Walters called on Dr. Royston to respond to the RAC members' questions and 
comments. Dr. Walters encouraged Dr. Royston to provide a productive and 
constructive interchange. Specifically, was there any information that was submitted to 
the NIH, the FDA, or both agencies, after the December 1992 RAC meeting that 
contributed to the decision-making process? 
Dr. Royston stated that he was appalled by the comments that have been made by the 
members of the RAC. He said that he is trying to comply with every Federal regulation 
and the NIH Guidelines', there are no base motives. He said that he worked an entire 
weekend on the Points to Consider , only to be informed that approval had been granted 
by the NIH Director. 
As a point of clarification, Dr. Royston said that the protocol submitted in November 
1991 is different from this protocol. He stated that it is his intention to submit the 
revised gene therapy protocol to the RAC in 1993, as soon as the vectors are certified by 
the FDA. Dr. Royston stated that he does not want to rush to bring in a protocol, he 
wants to be methodical and careful. 
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Recombinant DNA Research, Volume 17 
