Recombinant DNA Advisory Committee - 01/14/93 
Dr. Williams urged the RAC to exercise compassion in every case they examine. The 
committee should not adopt guidelines that will slow progress or block options. The 
RAC should not close any doors. 
COMMITTEES DISCUSSION 
Dr. Walters called on Ms. Buc to propose a motion in an attempt to move the RAC 
towards the adoption of a policy statement regarding expedited review of time-sensitive 
gene therapy protocols. Ms. Buc said that many comments have been made throughout 
the day by RAC members and others that can possibly be made into a common thread. 
The RAC is in agreement that it will consider single patient protocols; this issue is 
undisputed. She said that she would not attempt to make a distinction at this stage of 
gene therapy between research and therapy. Information can be obtained from these 
single patient protocols, even if there is no inherent statistical information resulting from 
these protocols. 
Ms. Buc stated that the RAC is in agreement that the criteria are the same for single 
patient and multiple patient protocols, namely, the concern about safety. These criteria 
for safety and efficacy are flexible. If the therapy is apparently safe and the plight of the 
patient is desperate, individuals are more willing to grant approval. However, if safety is 
a concern and the patient is less desperate, the review may be more stringent. 
Ms. Buc suggested that the NIH should publicly state that it has a very strong preference 
for the submission of protocols to the RAC; however, in emergency situations, NIH will 
review requests and apply the same criteria as the RAC. Obviously, such a public 
statement by the NIH will create a multitude of requests. NIH will have to make these 
determinations independently, applying the aforementioned criteria. The NIH should 
present a report at the next RAC meeting explaining what decisions have been made and 
the basis for those decisions. This report will allow for public participation and review. 
If the NIH's decisions are not a rough proxy of the decisions that would have been made 
by the RAC, then there will be divergence. If divergence occurs, public opinion will act 
to reduce that divergence in one way or another. In a sense, the RAC will act as peer 
review for NIH's decisions. In this way where the situation is critical, there is a safety 
valve. 
Ms. Buc explained that as time passes, the RAC may decide to review fewer of these 
single patient protocols under some other more specific set of criteria, i.e., derivative of 
some other protocol. The NIH will handle compassionate plea requests; however, the 
RAC may choose to examine these decisions. The RAC should request that NIH agree 
in advance to provide a report following approval of these requests. 
Committee Motion 
Recombinant DNA Research, Volume 17 
[33] 
