Recombinant DNA Advisory Committee - 12/1-2/94 
addressed. He said that the RAC should preserve the option to review confidential information in closed 
session, but the general recommendations should become public. 
Dr. Zallen disagreed with Dr. Goldhammer’s suggestion that the requirement for IRB approval prior to RAC 
submission should be eliminated. The RAC should not be in the position of providing a pre-review for IRBs. 
Dr. Zallen agreed with Dr. Goldhammer’s suggestion that the FDA should incorporate the RAC’s 
recommendations on Informed Consent into the FDA’s IND regulations. Dr. Zallen noted a similar petition 
to the Office for Protection from Research Risks (OPRR) of NIH. Dr. Noguchi said that a joint meeting 
between the IRBs, FDA, and OPRR is planned to discuss Informed Consent issues. Dr. Noguchi agreed to 
present Dr. Zallen’s suggestions at this meeting. 
Dr. Glorioso questioned if Pis could use vectors that have been approved for a specific disease to treat other 
types of diseases. Dr. Noguchi said if a gene therapy vector is approved for a specific indication, it would 
require a label to state that it has not been shown to be effective in some other types of diseases; however, if a 
physician chose to use that product for other indications and an adverse event resulted, the physician could be 
sued. Dr. Parkman stated that local IRBs are required to review protocols involving the application of FDA- 
approved drugs or biologies for nonapproved uses. 
Dr. Walters said that an NIH and FDA staff meeting (including legal counsel) is scheduled to address 
implementation of the consolidated review process. 
Dr. Wivel stated that a response will be sent to Dr. Goldhammer on behalf of the RAC. Dr. Wivel stated that 
the outcome of the NIH /FDA meeting will be transmitted to the members of the RAC. 
Dr. Walters emphasized that the RAC’s major concern about the consolidated review process is that the 
review process will be as open to the public as possible. Public accountability is extremely important. Ms. 
Meyers added that the RAC exists to provide a public forum to debate the use of this technology and its 
implication for the future of humanity, not to regulate gene therapy products. Mr. Capron stated that although 
he is assured by the FDA’s commitment that the consolidated review process will remain as open as possible, 
there is concern about such a process within the limits of FDA regulations. Mr. Capron said IRB-approved 
Informed Consent documents submitted to the RAC are quite inadequate; however, it is unreasonable for Dr. 
Goldhammer to request RAC to be the first committee to review these documents. IRBs are legally required 
to review Informed Consent documents. The requirement for prior IRB approval serves as a filtering 
mechanism for Informed Consent documents before RAC review. Dr. Walters noted that this requirement 
avoids the review of documents that are not in final form. 
Dr. Haselkom asked whether the possibility exists under the NIH /FDA consolidated review process that the 
FDA would approve a protocol that the RAC did not approve. Dr. Noguchi responded that the RAC serves 
in an advisory capacity, and that the circumstances would be rare that the FDA would not accept the 
recommendations of an advisory committee. 
Dr. Noguchi said that the FDA does not require IRB approval before starting its deliberation; however, IRB 
approval is required before a clinical trial may be initiated. The RAC should consider elimination of this 
requirement. Dr. Straus strongly disagreed with Dr. Noguchi’s suggestion since FDA does not review 
Informed Consent documents as rigorously as the RAC and IRBs are charged to review these documents by 
law. 
Dr. Fumimaro Takaku 
Dr. Walters recognized Dr. Fumimaro Takaku, President of the International Medical Center of Japan in 
Tokyo, Japan. He is chair of the committee responsible for human gene therapy oversight. Dr. Takaku stated 
that this committee plans to hold open meetings similar to the RAC in the near future. 
V-A. DECEMBER 1-2, 1994, DATA MANAGEMENT REPORT/DR. SMITH 
[438] 
Recombinant DNA Research, Volume 20 
