Recombinant DNA Advisory Committee - 01/14/93 
protocols for human gene therapy. In addition, the use of the compassionate use 
mechanism should be granted solely on a one-time basis. 
Dr. Krogstad stated that in terms of public perception, the words compassionate use 
suggests a reasonable expectation of efficacy. During the course of this discussion, the 
RAC should consider the public's perception. It is unfortunate that Dr. Royston's 
protocol has brought the issue of compassionate use of gene therapy to the RAC, 
because this protocol has serious scientific and medical shortcomings. However, Dr. 
Krogstad supported Dr. Healy's effort to develop a mechanism for the approval of 
compassionate plea requests. 
Dr. Leventhal said that the RAC should discuss whether any patient who is treated 
under a compassionate use exemption should be considered a research subject. The 
Office for Protection from Research Risk maintains that such patients cannot be 
considered research subjects. An investigator who receives a single patient 
compassionate use exemption cannot include the results of that patient data in any 
further reports of their research. Considering the public nature of this committee and 
the great interest in gene therapy, it is going to be extremely difficult not to interpret 
results. 
Dr. Zallen noted the NIH has been particularly concerned about the accessibility of 
women and minorities to research protocols. She asked Dr. Healy if these same 
concerns will be applied to compassionate use policies. Dr. Healy stated that 
compassionate use exemptions are granted on a case-by-case basis. These exemptions 
are non-research. Although all U.S. citizens should have equal and fair access to 
research protocols, compassionate use cannot be considered research. Data regarding 
compassionate use patients may not be part of data that is presented as a generalizable 
body of knowledge, which is explicitly stated in existing NIH regulations. 
Dr. Walters recognized the NIH staff who were present at the table: Mr. Robert 
Lanman, NIH Legal Counsel; Ms. Sandy Chamblee, Senior Policy Advisor and Counselor 
to the Director; Dr. John Diggs, Deputy Director for Extramural Research; Dr. John 
Mahoney, Associate Director for Administration; Dr. Lance Liotta, Deputy Director for 
Intramural Research; and Dr. Bruce Chabner, Director of the Division of Cancer 
Treatment, NCI. 
Dr. Chabner responded to the issue of efficacy of compassionate use therapy. Safety 
should be the important consideration, not efficacy. NCI's Division of Cancer Treatment 
handles numerous compassionate use requests; in many instances, there is very little, if 
any, information to suggest that the therapy will be effective. 
Recombinant DNA Research, Volume 17 
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