Recombinant DNA Advisory Committee - 12/3-4/92 
Presentation-Ms. Meyers 
Ms. Meyers noted that the short-term and long-term implications of gene therapy are 
unknown. Patients who enroll in these protocols should receive special consideration 
and should be monitored for life. The letter that is presented for discussion specifically 
addresses gene therapy because of the uniqueness and uncertainty of the technology. 
The broader issue is that there are many American citizens who are being denied the 
opportunity to participate in research protocols because they are required to pay for 
numerous procedures. If these patients suffer untoward consequences as a result of the 
research, they are currently responsible for these additional costs associated with the 
treatment of these injuries. 
Discussion 
Ms. Buc suggested that the draft letter should be expanded to discuss the issue of 
payment for non-negligent research injury in the broader sense. If the NIH Director is 
advised to convene a panel to propose policy, this issue should not be limited to gene 
therapy. The issues are generalizable to all other areas of research. Gene therapy 
should not be the specific target. 
Dr. Zallen agreed that the proposed issue is not necessarily unique to gene therapy; 
however, the RAC has a responsibility to bring this issue to the NIH Director's attention. 
Gene therapy is separate from other areas of research because it is a novel technology. 
The potential short-term and long-term side effects cannot be anticipated as with other 
therapies, e.g., drug therapy. Dr. Secundy spoke in support of Dr. Zallen's assertion that 
gene therapy is significantly unique. 
Dr. Leventhal stated that gene therapy is probably more accessible to those patients who 
have access to major procedures that are normally used as vehicles for gene therapy, e.g., 
bone marrow transplant. Gene therapy protocols are often submitted as an addition to 
pre-existing clinical protocols. For example, if patients can afford bone marrow 
transplants, they will have access to the gene therapy at no cost. In terms of risk, gene 
therapy probably does not present any more risk than any other Phase I study. 
Dr. Parkman said that the mandate of the RAC is to review gene therapy; however, the 
committee is obliged to consider any aspect of medical research within this context. He 
explained that as a pediatrician, the same questions arise regarding possible long-term 
untoward effects resulting from the chemotherapeutic reagents given to children with 
cancer. To mandate that the long-term effects of these treatments would be 
reimbursable by the government may lead to numerous problems. If the Federal 
government starts to indemnify particular groups of potential research subjects, clinical 
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