13 
transplant of bone marrow cells treated with a biologic and as such falls 
under FDA jurisdiction. 
Mr. Capron asked whether FDA regulates a single investigator treating a single 
patient. Dr. Ackerman said in same instances, FDA regulates the individual 
researcher; human gene therapy may be one such instance. Institutions such as 
the funding agency, the IRB, the medical school, or the pharmacy canpounding 
the substances used in a protocol also may require the investigator to file an 
IND. FDA welcomes voluntary IND filings and will encourage investigators to 
file for human gene therapy protocols. 
Dr. Cook-Deegan asked how FDA would proceed on receipt of an IND on human gene 
therapy. Dr. Ackerman said FDA would first perform a cursory examination of 
the IND for completeness; FDA scientific staff would evaluate the IND with 
regard to safety issues and would also examine the manufacturing process. The 
IND would be routed through the FDA review divisions and the relevant scientific 
divisions. The scientific divisions include virology, blood and blood products, 
vaccines, biochemistry and biophysics, and quality control. 
Cr. Mahoney asked whether the Working Group on Human Gene Therapy and the FDA 
could became involved in a jurisdictional dispute. Dr. welters said the working 
group and RAC have established a process for review of NIH funded protocols. 
Dr. Mahoney asked whether a duplication of effort may occur. Ms. Areen thought 
the working group and FDA would not perform duplicative reviews since the two 
groups will be evaluating proposals from two different perspectives. Cr. Walters 
agreed; he pointed out that FC& will handle submissions in a confidential 
manner. The NIH working group is part of a public process. In this sense, the 
two groups will perform different functions. Do. Gorovitz also agreed that 
different types of issues will be raised by the two groups; the NIH has its 
agenda while the FDA has its own interests. 
Dr. Mahoney asked whether one group might approve of a protocol and the other 
group disapprove. Dr. Ackerman said the FDA frequently reviews clinical trials 
developed by the NIH. No problems in coordinating these reviews has arisen 
in the past. Cr. Bonnie Lee of the FDA said NIH sponsored research involving 
FDA approved substances currently is reviewed by both the NIH and the FDA. 
Such review is complementary. 
Mr. Capron suggested the NIH process could serve to alert investigators to FLA 
requirements and procedures. 
Cr . Anderson said investigators might- wish to submit partial information to 
FDA in a confidential process rather than wait until a complete protocol is 
ready for submission. He asked if an informal cooperative discussion process 
would be useful to the FDA. Or. Ackerman said there are a variety of mechanisms 
for communicating with the FDA short of a formal IND submission; e.g., FDA can 
arrange pre-IND meetings or conferences. He felt these confidential mechanisms 
would be useful to investigators contemplating human gene therapy. 
[579] 
