Recombinant DNA Advisory Committee- 9/12-13/94 
without waiting for the efficacy data, and the same mistake should not be repeated in 
gene therapy. Only one disaster will damage the entire new field of human gene therapy. 
Dr. Varmus emphasized that redefining the role of the RAC is not totally driven by the 
need for fast track approval of human immunodeficiency virus (HIV) protocols. The 
current dual review system involving both RAC and FDA is cumbersome. Considering 
the rapid increase of gene therapy protocols, it is time to make this process more efficient 
in order to maximize the use of resources of NTH and FDA. He reiterated the need of 
RAC review since new kind of protocols are being proposed to treat diverse diseases. 
Dr. Erickson noted that gene therapy was designed to treat genetic diseases caused by 
single gene defects, and now it has expanded into the areas of AIDS and cancer. False 
hope for children of genetic diseases is detrimental and the concern for terminally ill 
patients with AIDS or cancer is different. He agreed that it is time to redefine the review 
criteria. 
Dr. Chase agreed that streamlining the review process is a good idea that will permit 
RAC to deliberate general issues involved in gene therapy. He pointed out three general 
areas for discussion: (1) quality and value of information to be obtained from gene 
therapy experiments, (2) indemnifying the subjects for research related costs, and (3) 
coordination and information exchange among multiple centers studying the same disease 
by the same approach. 
Mr. Capron did not share the viewpoints expressed by Dr. Miller and Ms. Meyers that the 
system needs no change because of increasing demands of AIDS activists and AIDS 
invetigators, but he agreed with the opinion that most Informed Consent documents are 
inadequate. The recent revision of the Points to Consider concerning the Informed 
Consent states more specifically what is to be included in the document. Mr. Capron 
expressed his disappointment with the IRB system in that it has not met its responsibility 
to amend all the deficiencies found in the Informed Consent documents. The issue of 
compensation for research-injuries is a problem area for most Informed Consent 
documents, and there is no government policy to guide this compensation issue. Mr. 
Capron stated that the RAC should deliberate gene therapy issues of public concern such 
as genetic enhancement and germ line alteration. The time has come for the RAC to 
divest itself from reviewing all protocols, not just because of the AIDS community. The 
RAC needs to articulate the standards for FDA to approve several types of protocols. 
This standard would allow time to deal with the real issues involving intended changes of 
inheritable human characteristics, the concern that led to creation of the RAC in the first 
place. 
Dr. Noguchi stated the FDA’s viewpoint about the role of the RAC. FDA derives its 
regulatory oversight over gene therapy by promulgation of regulations based on the Food, 
Drug and Cosmetic Act (revised) (FD&C Act) and Section 351 of the Public Health 
Service Act (PHS Act). Historically, FDA is mandated by law to do things that are 
codified in the Code of Federal Regulations (CFR). The RAC by contrast is not a 
creation of law and is most appropriate to deal with issues not readily addressed at FDA. 
Recombinant DNA Research, Volume 20 
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