2944 
Fedefal Register / Vol. 50, No. 14 / luesday, january 22, 1985 / Notices 
a. What potential benefits or hazards 
are postulated? 
b. Is there any expectation that the 
recombinant DNA will spread from the 
patient to others or to the environment? 
c. What precautions will be taken, if 
any, to protect others (e.g., patients 
sharing a room, health-care workers, or 
family members) from such potential 
hazards? 
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 nomedical) 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. 
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? 
C. Selection of subjects. Estimate the 
number of patients to be involved in the 
proposed study of gene therapy. 
Describe recruitment procedures and 
patient eligibility requirements. Indicate 
how equity consideration in the 
selection of subjects will be handled. 
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? 
It. What equity issues, if any. are 
likely to arise in the selection of 
patients? How will these issues be 
addressed? 
D. Informed consent. Indicate how 
patients will be informed about the 
proposed study and how their consent 
will be solicited. If the study involves 
pediatric or metally handicapped 
patients, describe procedures for 
seeking the permission of parents or 
guardians and, where applicable, the 
assent of each patient. Areas of special 
concern include potential adverse 
effects, financial costs, privacy, and the 
right to withdraw from further 
participation in the study. 
1. Will the major points covered in 
IA-IC of this document be disclosed to 
potential participants in this study and/ 
or parents or guardians in language that 
is understandable to them? (Include a 
copy of the patient consent form as part 
of the documentation requested in Part 
II below). 
2. Will the innovative character and 
the theoretically-possible adverse 
effects of gene therapy be discussed 
with patients and/or parents or 
guardians? Will the potential adverse 
effects be compared with the 
consequences of the disease? What will 
be said to convey that some these 
adverse effects, if they occur, could be 
irreversible? 
3. Will the financial costs of gene 
therapy and any available alternative 
therapies be explained to patients and/ 
or parents or guardians? 
4. Will patients and/or 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 of their right to 
withdraw at any time from the proposed 
study and of the consequences of 
withdrawal at the various stages of the 
experiment? State the extent to which 
subjects will be specifically advised of 
the reversibility or irreversibility of 
procedures that are performed during 
the course of the experiment. 
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. Social Issues 
The following issues are beyond the 
normal purview of local IRBs. However, 
since these issues have arisen in public 
debates about human gene therapy and 
the potential future applications of 
genetic techniques, the RAC and its 
working group request that investigators 
respond to questions A and B below and 
discuss, at their discretion, the general 
issues enumerated in point C. 
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? 
C. The following issues will also be 
considered by the RAC and its working 
group in reviewing each gene therapy 
proposal: 
1. How strong is the evidence that the 
proposed somatic-cell gene therapy will 
not affect the reproductive cells of 
patients? 
2. Is the proposed somatic-cell gene 
therapy and extension of existing 
methods of health care, or does it 
represent a distinct departure from 
present treatments of disease? 
3. Is it likely that somatic-cell therapy 
for human genetic disease will lead to: 
(a) germ-line gene therapy, (b) the 
enhancement of human capabilities 
through genetic means, or (c) eugenic 
programs encouraged or even mandated 
by governments? 
III. Requested Documentation 
In additiorrto responses to the 
questions raised in these ‘‘Points to 
Consider,” please submit the following 
materials: 
A. Your protocol (including consent 
form) as approved by your local IRB and 
IBC. 
B. Local IRB and IBC minutes and 
recommendations that pertain to your 
protocol. 
C. A one-page abstract of the gene 
therapy protocol. 
D. Curricula vitae for professional 
personnel. 
E. An indication of other federal 
agencies to which the protocol is being 
submitted for review. 
F. Any other material which you 
believe will aid in the review. 
G. Other pertinent materials. 
Dated: January 11, 1985. 
Note. — OMB'8 “Mandatory Information 
Requirements for Federal Assistance Program 
Announcements" (45 FR 39592) requires a 
statement concerning the official government 
programs contained in the Catalog of Federal 
Domestic Assistance. Normally N1H lists in 
its announcements the number and title of 
affected individual programs for the guidance 
of the public. Because the guidance in this 
notice covers not only virtually every NIH 
program but also essentially every federal 
research program in which DNA recombinant 
molecule techniques could be used, it has 
been determined to be not cost effective or in 
the public interest to attempt to list these 
[ 294 ] 
