Recombinant DNA Advisory Committee - 01/14/93 
Dr. D. Miller stated that he sides with the Foundation on Economic Trends with regard 
to the necessity for public review and scrutiny. There has never been a documented cure 
that is attributable to gene therapy. The RAC has reversed itself 180 degrees. Because 
of increasing pressure to approve trials for critically ill patients, investigators will attempt 
to administer anything to a patient in an effort to achieve some sort of an effect. To 
move in this direction is bad scientific policy. Progress will be hampered instead of 
approving good protocols quickly. 
Dr. Woodcock asked to clarify a prevailing misconception about the FDA. The FDA's 
safety review of these protocols, like other investigational biologies, is extremely rigorous 
and includes a very detailed safety assessment of the cell line used to produce these 
products, the potential for adventitious agents contaminating the products, and any other 
safety risks that might occur. The FDA's safety evaluation is the same whether the 
protocol is intended for 1 or 100 patients. 
Dr. Leventhal stated that due to the increasing number of human gene therapy protocols 
that are being submitted for RAC review, the committee might have to increase the 
number of meetings per year. She stated that increasing the number of meetings from 4 
to 6 would decrease the time between meetings from 3 months to 2 months. Increasing 
the number of RAC meetings per year may resolve many of the problems discussed 
today. 
Dr. Walters called on Dr. Wivel to summarize the proposed policy recommendation, 
including the amendments that were offered by the RAC members. Dr. Wivel outlined 
the policy statement as follows: 
1. At the very outset, the RAC will make it clear that it will entertain single patient 
protocols. 
2. No distinction will be made between research and therapy. 
3. Regardless of the method of review, the criteria must be the same for all 
protocols. 
4. NIH will state publicly as part of this policy statement, its preference for the 
traditional method of review. 
5. When time sensitive circumstances prevail, the NIH will perform an internal 
review. 
6. The NIH will take into consideration, among other factors, consanguinity with 
previously approved protocols. 
7. To the extent legally and practically possible, the NIH Director will consult with 
RAC members, NIH experts, and any other experts deemed appropriate to the 
review process. 
8. The NIH will report to the RAC following its internal review. 
Recombinant DNA Research, Volume 17 
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