M.J. Welsh and A.E. Smith, RAC Application 
E. PRIVACY AND CONFIDENTIALITY 
Indicate what measures will be taken to protect the privacy of patients and their 
families as well as to maintain the confidentiality of research data. 
E.l. What provisions will be made to honor the wishes of individual patients (and the 
parents or guardians of pediatric or mentally handicapped patients) as to whether, 
when, or how the identity of patients is publicly disclosed? 
The U.S. Food and Drug Administration (FDA) and Genzyme Corporation may inspect and 
copy medical records relating to this study, and the results of the study will be reported to 
the FDA and perhaps to other regulatory agencies. This information will be treated 
confidentially and, in the event of any publication regarding this study, patient identity will 
not be disclosed. 
E.2. What provision will be made to maintain the confidentiality of research data, at least 
in cases where data could be linked to individual patients? 
A record of patient participation in this research will be maintained. This record will be 
kept confidential. The patient will be assigned a study number and the information will be 
kept under this study number rather than the patient name. 
H. SPECIAL ISSUES 
Although the following issues are beyond the normal purview of local IRBs. the RAC 
and its Subcommittee request that investigators respond to questions A and B below . 
A. What steps will be taken, consistent with point I-E above, to ensure that accurate and 
a ppropriate information is made available to the public with respect to such public 
concerns as may arise from the proposed study? 
We will coordinate a response to inquires from the press with the University of Iowa Health 
Sciences Affairs Office, the Howard Hughes Medical Institute, and Genzyme Corporation. 
B. Do vou or vour funding sources intend to protect under patent or trade secret laws 
either the products or the procedures developed in the proposed study? If so. what 
steps will be taken to permit as full communication as possible among investigators 
and clinicians concerning research methods and results? 
Genzyme Corporation will protect the product and procedure developed in the study. We 
plan to publish all findings from the study. 
HI. REQUESTED DOCUMENTATION 
In addition to responses to the questions raised in these Points to Consider, please 
submit the following materials : 
A. Your protocol as approved bv vour local IRB and IBC . 
Protocol: Appendix 1. Informed Consent: appendix 2 
Recombinant DNA Research, Volume 16 
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