Recombinant DNA Advisory Committee - 01/14/93 
REMARKS OF THE DIRECTOR OF NIH--DR, BERNADINE HEALY 
Dr. Healy explained that she requested this special session of the RAC to discuss the 
NIH approval of Dr. Royston's compassionate plea exemption request and to address the 
urgent need to establish guidelines for handling such requests in the future. 
Dr. Healy presented a chronology of the events that led to her decision to approve Dr. 
Royston's request. In early October, Senator Tom Harkin asked that the NIH give timely 
consideration to individual compassionate plea requests for approval of gene therapy 
procedures for terminally ill patients. Such a step would be a temporary solution to this 
problem until an appropriate , permanent, legislative solution setting forth the clear authority 
and mechanism for handling these cases can be achieved next year. This request was made 
on behalf of a 51-year-old former constituent suffering from a life-threatening Stage IV 
glioblastoma that has been unresponsive to conventional treatments. 
At the time NIH received Senator Harkin's letter, there was insufficient information to 
make a decision regarding this particular patient or the proposed treatment. This 
request was the first compassionate plea for gene therapy received at NIH, and the NIH 
has no experience or mechanism for addressing such pleas. It was imperative that the 
RAC consider this issue at the December 4, 1992, meeting. Accordingly, Dr. Royston 
was invited to attend the December 1992 meeting to provide further information. Dr. 
Healy stated that although the RAC had a lengthy discussion on this issue, a formal 
recommendation concerning either the specific patient or the generic issue of 
compassionate plea exemptions was not forthcoming. Subsequently, Dr. Royston has 
submitted a formal written request for compassionate plea exemption to the NIH, 
including a copy of his Food and Drug Administration (FDA) single patient 
Investigational New Drug (IND) application and protocol, which were already pending at 
the FDA and his Institutional Review Board (IRB). 
Dr. Lance Liotta, Deputy Director for Intramural Research, NIH, reviewed Dr. 
Royston's materials with the assistance of several oncologists at the National Cancer 
Institute (NCI), the staff of the Office of Human Subjects Research, and Dr. Wivel, 
Director of the Office of Recombinant DNA Activities (ORDA). 
Consistent with the RACs discussion on December 4, 1992, a letter was sent to Dr. 
Royston on December 21, 1992, recommending that his patient should consider entry 
into Dr. Edward Oldfield's glioblastoma protocol. At this time, Dr. Liotta requested 
additional information regarding local approvals and the status of the patient. Within 24 
hours of this recommendation, NIH was advised that the patient and her family declined 
to be considered for Dr. Oldfield's protocol, in part because of the requirement for 
additional surgery. Moreover, this patient has already undergone several craniotomies 
that have probably caused her to be ineligible for Dr. Oldfield's protocol. In contrast, 
Recombinant DNA Rr«r arch, Volume 17 
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