14 
gene therapy, and there will be greater than average scrutiny. He thought the 
working group in such an environnent would wish to request such information 
even if the final review decision is not influenced by this consideration. 
Mr. Capron questioned whether this portion of the document should address equal 
access considerations; he thought the document might paraphrase the relevant 
IPB regulations and ask how patients had been selected. Mr. Capron said the 
request for information on patient selection would simply be a request to know 
for which there is no right or wrong answer. Dr. McCarthy said several other 
governmental canmittees have struggled with the question of equity of access; 
he said it is very difficult to develcp such criteria. 
Dr. Walters asked the working group to evaluate Section I-C, Informed Consent , 
of the draft document (Attachment II) . 
Ms. Areen said the specific HHS regulations on securing the assent of a child 
should be cited in this portion of the document. The language might include 
a paraphrasing of the HHS regulations. Dr. Mahoney said the preamble to this 
section should state that children will most likely be the first patients for 
human gene therapy. 
Dr. Murray asked whether this portion of the document should address competency 
issues as mental retardation is frequently part of the natural history of these 
diseases. Dr. Anderson did not think competency issues in human gene therapy 
would be different from competency, issues with other therapies. 
Dr. Grobstein noted that follow- up of the patient will also cost money and the 
patient should know who will bear the cost of therapy. Item I-C- (4) should 
request this information. Dr. Gorcvitz questioned whether the cost of alternative 
therapies might be mentioned in this portion of the document. 
Dr. Grobstein suggested language indicating the "innovative character" of the 
therapy be added to Item I-C-(l) which requires that gene therapy protocols 
explain designs and procedures in a clear coherent manner. 
Dr. Gottesman said the patient and the family should be aware that the public 
will be very interested in this novel therapy. She suggested publicity might be i| 
the greatest danger the patient would face as part of the therapy. Dr. Anderson 
thought most families wishing to have members treated will be well-informed 
about the interest this therapy will generate. 
Dr. Mahoney asked if Section I-C of the working group document should point cut 
lew probability risks associated with the therapy such as the possibility of 
activating oncogenic sequences or modifying the germ line. He said the IRB 
usually does not require the investigator to point out low probability risks on 
the consent form. Mr. Capron supported the idea of disclosing lew probability 
risks in light of the innovative nature of the therapy. 
Dr. Motulsky questioned the fairness of burdening the patient and the family 
with knowledge of low probability risks; he wondered whether this burden would 
harm the family. Mr. Capron said the families of these patients might be 
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