Recombinant DNA Advisory Committee - 12/3-4/92 
research will become limited rather than expanded to large numbers of subjects. There 
will always be a degree of uncertainty about research. The purpose of the informed 
consent document is to inform patients of the definable and undefinable risks of that 
research. 
Dr. Post said that he supports the concept that gene therapy is no more predictable than 
any other type of drug research. In fact, gene therapy is probably more predictable 
because the mechanism is known and assays such as PCR are available to monitor 
trafficking and expression. 
Dr. Walters suggested that the draft letter submitted by Dr. Zallen and Ms. Meyers 
should be revised to state that in the course of reviewing gene therapy and gene transfer 
protocols, the RAC has ascertained that there is significant variation among institutions 
regarding the question of research-related non-negligent injury. The RAC recommends 
the formation of a panel to establish policy regarding this issue. Mr. Barton noted that 
the proposed panel would generate declaratory policy rather than real policy. Mr. 
Barton said that since there is a possibility that gene therapy will result in untoward 
effects, any costs associated with such effects would more appropriately fall on the 
Federal government rather than on the individual or the institution. However, these 
liabilities should be dealt with in a way that will not deter research. This issue should be 
considered within the context of health care policy that will be developed by the 
incoming Administration. Proposed policy that will be incorporated into the new health 
care system should not discriminate against research in any unintended way. 
Dr. Schaechter stated that he supports the draft letter because it is important that people 
of all socio-economic segments of society have access to new therapies. Dr. Geiduschek 
said that he is in support of the letter. It is inadequate for the RAC to deny that gene 
therapy has no special status. Dr. Geiduschek proposed that the following 
recommendation should be included in the draft letter to the NIH Director, at a 
minimum, research sponsors or their institutions should be responsible for such costs. 
Dr. Murray said that while patients are often screened to ensure that they can pay for 
the clinical therapies such as bone marrow transplant, there is no financial screening to 
guarantee that they would be able to pay for any complication resulting from untoward 
effects. 
Ms. Meyers said that gene therapy patients are potentially accepting debt for the rest of 
their lives if there are research-related injuries, because no insurance company will cover 
a person who has undergone gene therapy. Compensation is not the issue here. The 
issue is guaranteed medical care for life if no insurance company will cover these 
patients. 
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Recombinant DNA Research, Volume 16 
