13 
(IRB) would first review the protocols. The proposals would then be for- 
warded to the Working Group on Human Gene Therapy for review. The RAC 
will subsequently review the proposals. The NIH Director has final 
authority to approve or reject proposals. (4) Describe a procedure for 
periodic revision of the points to consider document. Gene therapy is a 
very dynamic field, and the working group anticipates enough new information 
will be generated yearly to require revision of the document. 
Dr. Walters said the first section of Part I of the points to consider 
document will refer to the DHHS Regulations for Research Involving Human 
Subjects (45 CER Part 46). These regulations apply to hurran gene therapy 
protocols. In addition, this part of the document will require specific 
types of information be provided by the investigator (s) on: (1) research 
design, risks and benefits; (2) the selection of subjects; (3) the informed 
consent process; and (4) protection of privacy and confidentiality . 
Dr. Walters said the section of the document entitled "Research Design, 
Anticipated Risks and Benefits" will first ask the investigator to describe 
objectives and rationale for the proposed gene therapy. What is the dis- 
ease to be treated? Why was this disease chosen for therapy? Why was this 
approach to therapy chosen? What is the natural history of the disease? 
What alternative treatments are available and have these therapies been 
considered? 
Dr. Walters said the document will then ask for a description of research 
methods including detailed information on the structure of the genetic 
information to be introduced into the patient. The document may request 
information on animal and tissue culture studies including perhaps labora- 
tory cell culture studies performed with the patient's cells. 
Dr. Walters said questions on clinical and public health considerations in 
the treatment of patients will be posed. Hew will therapy be administered 
to the patients? What kind of patient monitoring will be provided? At 
what intervals will the investigators report to the IRB and the Working 
Group on Human Gene Therapy? 
The document will also request information on the investigators' qualifica- 
tions and the adequacy of the facilities. 
Dr. Walters said the section dealing with subject selection is concerned 
primarily with issues of equity and fairness. Human gene therapy will be a 
scarce biomedical resource, at least in the early days; and the working 
group would like to ensure that patients have equal access. 
Dr. Walters said informed consent for human gene therapy may be complicated 
because of the the complexity of the technique, but also because the first 
subjects are likely to be pediatric patients whose parents will be making 
judgments on their behalf. 
Dr. Walters said the section dealing with protection of privacy and con- 
fidentiality will ask the investigator whether measures could be taken to 
[ 145 ] 
