Recombinant DNA Advisory Committee - 01/14/93 
requests for individual patients involved in recombinant DNA intervention? The 
implication is that the RAC does not know how to review protocols that involve patients. 
There is an appropriate combination of expertise on the RAC. Individual patient 
requests differ from multiple patient requests only in that the design of the research and 
its potential to contribute to general knowledge is absent. (4) How can a time-sensitive 
mechanism be developed for handling such requests? Mr. Capron said that expedited 
review will probably have to come from the FDA and the NIH Director. (5) How can 
the RAC mandate be expanded or enhanced? Mr. Capron said that the results of these 
single patient protocols should be reported to the RAC. The RAC should have access to 
any data relating to safety or problems associated with the procedure. 
Dr. Leventhal said that she was uncomfortable with the terminology single patient 
protocol because some of these protocols might be considered research. There may be 
genetic diseases so rare that there is only one person available in whom an important 
experiment can be performed. Therefore, single patient research protocols must be 
distinguishable from single patient non-research protocols. The RAC members have 
indicated that safety needs to be assured in either setting. However, in a non-research 
setting, a single patient may be given material without the strict review and the 
possibility of efficacy required for a research protocol. She stated that she would not 
accept the approval of any experiment in which the results were not reported to the 
RAC. 
Dr. Geiduschek continued, stating that the RAC as a national public committee on 
human gene therapy, has a natural time limit. There will come a point in which the kind 
of review currently undertaken by the RAC will be deemed no longer necessary, and the 
committee will be abandoned. Until that time, the dual system of approval, namely, 
research versus non-research compassionate use, is not appropriate or in the public 
interest. Compassionate use should only be approved in the context of the working 
group's recommendations, i.e., expansion of the inclusion/exclusion criteria of a 
previously approved protocol. If the RAC chooses to grant compassionate use 
exemptions within the context of approved protocols, clear eligibility requirements, 
including the patient and the physician, would have to be established. 
Dr. Geiduschek recommended that the retrospective examination of Dr. Royston's 
protocol will be helpful in bringing the RAC to an agreement on future policy. He 
agreed with the statement made by Mr. Capron; namely, that the qualifications of the 
RAC members are appropriate for the review of both single patient and multiple patient 
protocols. 
Dr. Geiduschek asked for the opportunity to state his views before having to leave the 
telephone conference. He referred the RAC to his written statements and emphasized 
that these statements represented his best judgement at that moment, as they had in 
Recombinant DNA Research, Volume 17 
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