Recombinant DNA Advisory Committee - 03/1-2/93 
Dr. Leventhal said that the RAC should not refer to expedited approval as 
compassionate plea exemptions. The term compassionate indicates that there is some 
inherent value to the procedure. Expedited approvals may have no experimental value; 
therefore, they should not be considered compassionate. She cautioned the committee to 
consider the thousands of terminal cancer patients who will hear about this expedited 
approval process and have their hopes raised that they may also qualify for such an 
exemption. Dr. Parkman said that the Procedures for Expedited Review includes the 
provision that expedited approval will be granted only for a single patient; this provision 
should obviate any concerns about raising false hope for terminal patients. Dr. Walters 
concurred with the statement made by Dr. Parkman and emphasized that the onus is on 
the investigator to provide a valid argument for why the patient cannot wait until the 
next scheduled RAC meeting. The expedited review process is not viewed as a 
mechanism for accruing multiple patients onto a single protocol. 
Dr. Leventhal reminded that committee that Dr. Wilson is an example of an investigator 
who identified several patients who did not fit the inclusion criteria for RAC approval. 
However, Dr. Wilson had sufficient time to request a minor modification to his approved 
protocol and to present the unique set of circumstances to the RAC at its regularly 
scheduled meeting. Dr. Miller reminded the committee that Dr. Wilson's request was 
not for expedited review, but was submitted as a minor modification to a previously 
approved protocol. 
Dr. Parkman said that the objective of the Procedures for Expedited Review is to ensure 
that the scientific quality of the protocol is as good, if not better, than the quality of 
those protocols approved through the regular review process. 
Cover Sheet for Expedited Review 
Dr. Parkman presented a draft checklist of critical information that should be completed 
at the time that an initial request is made by an investigator for expedited review of a 
single patient protocol. This Cover Sheet for Expedited Review will be used by the Office 
of Recombinant DNA Activities (ORDA) to determine whether the investigator has 
minimal requisite preclinical data which would warrant expedited review. If the 
requesting individual cannot complete the entire Cover Sheet for Expedited Review than 
their request will not be forwarded based on the fact that the requisite minimal data to 
evaluate the protocol does not exist. If the investigators do not have the vector 
sequence, gene transduction or expression data, or safety data, then it is not appropriate 
to evaluate the protocol. 
Dr. Post expressed concern that this 1 page document may send a signal to investigators 
that they can submit less information for an expedited review protocol than for a 
protocol reviewed through the normal process. Investigators should be required to 
[106] 
Recombinant DNA Research, Volume 17 
