7 
Dr. Anderson thought the public would be reassured if the first gene therapy 
proposals are reviewed by a public body such as the RAC. He thought a statement 
indicating RAC's responsibilities should be included in the preamble to the 
document. 
Dr. Gottesman thought the preamble to the document should explain that review 
will be based on available scientific information. She said RAC's role is not 
one of enforcement but rather one of evaluation. 
Dr. Walters called the attention of the working group to Section I of the 
document which listed quotations excerpted fron DHHS regulations dealing with 
informed consent and IRB guidelines. 
Dr. McCarthy said inclusion of selected excerpts from the EHHS regulations in 
the working group document implies not all the EHHS human subject regulations 
will apply to human gene therapy protocols. He thought the working group 
document should simply state that the EHHS regulations regarding the use of 
human subjects apply in toto. 
Ms. Areen agreed with CP. McCarthy. She said her concern with the portion of 
the subgroup draft document (Attachment II) referring to the use of children in 
research protocols stems frcm a similar observation. 
Dr. Walters then drew the attention of the working group to Section I-A-l, The 
Disease and Alternative Therapies , of the document. Dr. Childress suggested 
this section might request a cost ccmparison between alternative therapies and 
human gene therapy. 
Dr. Mot ul sky said minor successes and the parameters used to follcw the patient 
should also be requested in this section of the document. He explained that in 
some diseases the natural history of the disease varies, and it is difficult to 
gauge what is happening. 
Dr. Murray said range of expression is part of the natural history of the 
disease, and the working group document should address differences in the range 
of expression. He suggested the investigator who chooses to attempt therapy of 
a disease difficult to treat might have a simpler task of explaining why he has 
chosen to attempt gene therapy with that particular disease. 
Mr. Capron asked if the working group would insist that protocols reviewed at 
this time only address a disease that is not treatable by other therapies. 
Dr. Walters said he would substitute the word "amelioration" for the word 
"success" in item I-A-l-b of the working group document (Attachment II) . 
Dr. Anderson said the document should ask the investigator why this particular 
disease is appropriately approached in this manner at this time. The document 
should clearly indicate what information the working group is seeking. In this 
way, the public wall see exactly what information is being evaluated. 
[ 95 ] 
