Recombinant DNA Advisory Committee - 01/14/93 
advance of the meeting. 
Dr. Parkman said that he disagrees with Dr. Healy's concept of a non-research protocol. 
Any patient that receives gene therapy will be treated at an institution that has an 
ongoing gene therapy research program. The issue is that there is a patient who 
intersects with those research interests and the time frame for instituting therapy does 
not fit within the time constraints of the RAC. Therefore, the term single patient 
protocol is appropriate to describe these situations. Although statistical evaluations will 
not be possible, statements can certainly be made regarding efficacy. 
IX. STATEMENT-MS. KATHERINE MATTHEWS 
Dr. Walters called on Ms. Katherine Matthews, staff attorney for biotechnology, of the 
Foundation on Economic Trends, to present her statement to the RAC. Ms. Matthews 
distributed a proposed amendment to the NIH Guidelines regarding the issue of 
compassionate plea requests. The Foundation on Economic Trends is concerned that as 
progress is being made in gene therapy, public review and scrutiny, is decreasing. The 
Foundation on Economic Trends represents the public interest. The particular case that 
has brought the issue of compassionate use before the RAC is outrageous; the worst 
possible case scenario. Undue political influence is very clearly present in this case. 
Ms. Matthews stressed the importance for adopting an amendment to the NIH Guidelines 
that will prevent this problem from occurring in the future. The RAC needs to address 
the question of how the committee is going to deal with the reality that the NIH 
Director is a political appointee. 
The proposed amendment to the NIH Guidelines that was distributed by Ms. Matthews 
could not be voted on at this meeting because it had not been published for public 
comment in the Federal Register. However, Ms. Matthews suggested that the RAC might 
choose to consider the proposed amendment at the March 1993 RAC meeting. Gene 
therapy is still in its infancy and does not have public understanding. The need to 
reassure the public that the process is being scrutinized by all sectors of society is critical. 
Ms. Matthews stated that there would be grave concerns on the part of the Foundation 
on Economic Trends if gene therapy deliberations were to occur in a manner different 
from what is prescribed in the NIH Guidelines , namely, providing the opportunity for 
public comment. 
Ms. Matthews outlined the following proposed amendments to the NIH Guidelines : (1) 
Section I-A reads, ..administration of gene therapy to human subjects... will be amended to 
read, ...administration of gene therapy either to a group of human subjects or to a single 
human subject..., (2) A new paragraph will be added to Section I-A to read, Any request 
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Recombinant DNA Research, Volume 17 
