Recombinant DNA Advisory Committee - 01/14/93 
the scope of the RAC mandate. Second, should the NIH Guidelines be amended such 
that non-research exemptions fall within the purview of the RAC? The letter of the NIH 
Guidelines might support the exclusion of such cases, but the spirit of the RAC mandate, 
to protect the public and provide a process for public comment on recombinant DNA 
research, might argue otherwise. The NIH Guidelines states that, The NIH Guidelines 
cannot anticipate every possible situation. The Guidelines will never be complete or final, 
since all conceivable experiments involving recombinant DNA cannot be foreseen. 
Therefore, it is the responsibility of the institution and those associated with it to adhere to 
the intent of the Guidelines as well as to their specifics. This statement suggests that the 
NIH's broader mandate is to clarify the NIH Guidelines as science evolves. Third, absent 
any mechanism and faced with urgent requests for the use of gene therapy in imminently 
dying patients, an NIH interim policy must be in place while this issue is thoroughly 
reviewed and long-term options are developed. Proposed policy regarding this issue 
should be developed based on input from the members of the RAC, the scientific 
research community, in coordination with FDA procedures and statutory authorities, and 
full public dialogue. 
Dr. Healy stated that this meeting serves two purposes: (1) to inform the RAC of the 
interim approach that has been put into place to handle Dr. Royston's request 
expeditiously; and (2) to develop formal procedures for addressing future compassionate 
plea requests. 
Dr. Healy outlined the interim approach that will be taken by the NIH absent a formal 
procedure: (1) If an experimental recombinant DNA product proposed for 
compassionate therapeutic use is derived from research that is subject to the NIH 
Guidelines, NIH has a responsibility to respond to that request. (2) This response must 
be made in a timely manner consistent with existing practices for compassionate use 
exemptions for other drugs and biologies. To the extent consistent with the need for 
rapid action, public notification will occur. In any case, the decision and the basis for the 
decision will be made public. (3) NIH will gather appropriate information on patient 
and public safety, including all documents submitted to other review bodies. NIH will 
seek advice of the appropriate Institute, Center, and Division representatives; other 
experts; the RAC Chair; and other individuals deemed necessary by the Chair. (4) NIH 
approval of a compassionate plea request will be contingent on FDA, IRB, and 
Institutional Biosafety Committee (IBC) approvals. 
Historically, the strength of the RAC is that it has always been responsible to the 
changing nature of science and public concerns. The submission of a compassionate plea 
request was inevitable. The promise of gene therapy is too great; the needs of the 
patient community are too desperate. 
Recombinant DNA Research, Volume 17 
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