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sharing a room, health-care workers, or 
family members)? 
d. What measures will be undertaken 
to mitigate the risks, if any, to public 
health? 
5. Qualifications of Investigators, 
Adequacy of Laboratory and Clinical 
Facilities 
Indicate the relevant training and 
experience of the personnel who will be 
involved in the preclinical studies and 
clinical administration of gene therapy. 
In addition, please describe the 
laboratory and clinical facilities where 
the proposed study will be performed. 
a. What professional personnel 
(medical and nonmedical) will be 
involved in the proposed study? What 
are their specific qualifications and 
experience with respect to the disease to 
be treated and with respect to the 
techniques employed in molecular 
biology? Please provide curricula vitae 
(see Section III— DJ. 
b. At what hospital or clinic will the 
treatment be given? Which facilities of 
the hospital or clinic will be especially 
important for the proposed study? Will 
patients occupy regular hospital beds or 
clinical research center beds? Where 
will patients reside during the follow-up 
period? 
C. Selection of Patients 
Estimate the number of patients to be 
involved in the proposed study of gene 
therapy. Describe recruitment 
procedures and patient eligibility 
requirements, paying particular 
attention to whether these procedures 
and requirements are fair and equitable. 
1. How many patients do you plan to 
involve in the proposed study? 
2. How many eligible patients do you 
anticipate being able to identify each 
year? 
3. What recruitment procedures do 
you plan to use? 
4. What selection criteria do you plan 
to employ? What are the exclusion and 
inclusion criteria for the study? 
5. How will patients be selected if it is 
not possible to include all who desire to 
participate? 
D. Informed Consent 
Indicate how patients will be 
informed about the proposed study and 
how their consent will be solicited. The 
consent procedure should adhere to the 
requirements of DHHS regulations for 
the protection of human subjects (45 
CFR Part 46). If the study Involves 
pediatric or mentally handicapped 
patients, describe procedures for 
seeking the permission of parents or 
guardians and, where applicable, the 
assent of each patient. Areas of special 
concern highlighted below include 
potential adverse effects, financial costs, 
privacy, and long-term follow-up. 
1. How will the major points covered 
in Sections I-A through I-C of this 
document be disclosed to potential 
participants in this study and/or parents 
or guardians in language that is 
understandable to them? 
2. How will the innovative character 
and the theoretically-possible adverse 
effects of gene therapy be discussed 
with patients and/or parents or 
guaradians? How will the potential 
adverse effects be compared with the 
consequences of the disease? What will 
be said to convey that some of these 
adverse effects, if they occur, could be 
irreversible? 
3. What explanation of the financial 
costs of gene therapy and any available 
alternative therapies will be provided to 
patients and/or parents or guardians? 
4. Will patients and/or their parents 
or guardians be informed that the 
innovative character of gene therapy 
may lead to great interest by the media 
in the research and in treated patients? 
What special procedures, if any, will be 
followed to protect the privacy of 
patients and their families? 
5. Will patients and/or their parents 
or guardians be informed: 
a. That some of the procedures 
performed in the study will be 
irreversible. 
b. That following the performance of 
such procedures it would not be 
medically advisable for patients to 
withdraw from the study? 
a That a willingness to cooperate in 
long-term follow-up (for at least three to 
five years) will be a precondition for 
participation in the study? 
d. That a willingness to permit an 
autopsy to be performed in the event of 
a patient’s death following treatment is 
also a precondition for a patient's 
participation in the study? (This 
stipulation is included because an 
accurate determination of the precise 
cause of a patient's death would be of 
vital importance to all future gene 
thei-apy patients.) 
E. Privacy and Confidentiality. 
Indicate what measures will be taken 
to protect the privacy of gene therapy 
patients and their families as well as to 
maintain the confidentiality of research 
data. 
1. 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? 
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? 
II. Special Issues 
Although the following issues are 
beyond the normal purview of local 
IRBs, the RAC and its working group 
request that investigators respond to 
questions A and B below. 
A. What steps will be taken to ensure 
that accurate information is made 
available to the public with respect to 
such public concerns as may arise from 
the proposed study? 
B. Do you or your 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? 
III. 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 by your 
local IRB and IBC. The consent form, 
which must have IRB approval, should 
be submitted to the NIH only on request. 
B. Local IRB and IBC minutes and 
recommendations that pertain to your 
protocol. 
C. A one-page scientific abstract of 
the gene therapy protocol. 
D. A one-page description of the 
proposed experiment in nontechnical 
language. 
E. Curricula vitae for professional 
personnel. 
F. An indication of othfer federal 
agencies to which the protocol is being 
submitted for review. 
G. Any other material which you 
believe will aid in theTeview. 
IV. Reporting Requirements 
A. Serious adverse effects of 
treatment should be reported 
immediately to both your local IRB and 
the NIH Office for Protection from 
Research Risks, and a written report 
should be filed with both groups. A copy 
of the report should also be forwarded 
to the NIH Office of Recombinant DNA 
Activities (ORDA). 
B. Reports regarding the general 
progress of patients should be filed at 
six-month intervals with both your local 
IRB and ORDA. These twice-yearly 
reports should continue for a sufficient 
period of time to allow observation of 
major effects (at least three to five 
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