Yes, FDA recognizes that there are circumstances in which an investigational device is the only
option available for a patient faced with a serious or life-threatening condition (hereinafter
referred to as “compassionate use”). Unlike emergency use of an unapproved device discussed
above, prior FDA approval is needed before compassionate use occurs. Section 561(b) of the act
and 21 CFR 812.35. In order to obtain agency approval, the sponsor should submit an IDE
supplement requesting approval for a protocol deviation under section 812.35(a) in order to treat
the patient. The IDE supplement should include:
• A description of the patient’s condition and the circumstances necessitating treatment;
• A discussion of why alternatives therapies are unsatisfactory and why the probable
risk of using the investigational device is no greater than the probable risk from the
disease or condition;
• An identification of any deviations in the approved clinical protocol that may be
needed in order to treat the patient; and
• The patient protection measures listed above that will be followed.
The patient identified in the supplement should not be treated with the device until FDA
approves its use under the proposed circumstances. In reviewing this type of request, FDA will
consider the above information as well as whether the preliminary evidence of safety and
effectiveness justifies such use and whether such use would interfere with the conduct of a
clinical trial to support marketing approval.
If the request is approved, the attending physician should devise an appropriate schedule for
monitoring the patient, taking into consideration the investigational nature of the device and the
specific needs of the patient. The patient should be monitored to detect any possible problems
arising from the use of the device. Following the compassionate use of the device, a follow-up
report should be submitted to FDA in which summary information regarding patient outcome is
presented. If any problems occurred as a result of device use, they should be discussed in the
supplement and reported to the reviewing IRB as soon as possible.
Additional information on the procedures physicians and IRBs should follow in compassionate
use situations may be found in Chapter III Expanded Access to Unapproved Devices of the
guidance entitled, “IDE Policies and Procedures”.
