Experimental devices need FDA approval

Informed consent at heart of lawsuit

By J. Mark Waxman, JD.

A report in the March 25 Philadelphia Inquirer regarding the use of experimental treatment on an infant in connection with a heart repair highlights a series of issues related to both the use of devices not approved by the FDA and, in turn, their use on minors, including infants.

According to the story, parents of a 3-year-old born with Down syndrome and a serious heart defect were offered a stent procedure, following two surgeries to correct an underdeveloped left heart pumping chamber, in lieu of a third surgery to reroute the blood flow. This stent procedure, less invasive than the generally accepted third surgery, was allegedly used on 20 children, but its study and use was apparently not considered part of a clinical trial.

The article claims that some doctors were actually testing whether the third surgery could be omitted. It asserts, however, that "records were being compiled for a study," but no IRB approval was either sought or obtained.1

The parents asserted a variety of shortcomings. First, they said that although the manufacturer’s patient consent form clearly stated that the device was not FDA approved and could have a variety of potential complications, the consent form was not provided to them prior to the use of the stent. Instead, they assert the consent form was sent approximately one year later, and they were asked to backdate it. Second, they were never told that the stent procedure was experimental. Finally, they alleged the stent might clog which would result in the third major surgery in any event.

These facts raise myriad concerns about the processes and checks and balances that were in place at the hospitals involved in these practices. In particular: Why was this procedure not the subject of IRB review and oversight?2

Medical devices and their categorization

A general definition of a medical device is "any health care product that does not achieve its primary intended purposes by chemical action or by being metabolized." Clearly stents to reroute blood flow would be medical devices.3

Unless such a medical device is low risk or substantially equivalent [510(k) devices], it must undergo clinical testing and a pre-market approval process. An investigational device is a medical device that is the subject of a clinical study focusing on the effectiveness and safety of a device, and must meet the requirements established by the FDA for an Investigational Device Exemption (IDE).

From the IRB perspective, devices are categorized as either significant risk (SR) or nonsignificant risk (NSR). For SR devices, both an FDA approval resulting in an IDE and IRB study approval is required. For an NSR device, an IDE application is not required (although abbreviated requirements must still be met).4

Sponsors make the initial SR/NSR determination. If a sponsor concludes the device is an NSR device, it provides the basis for that decision to the IRB, including information related to the FDA’s assessment of the risks of use if that assessment has been made. The IRB will review all the information available to it to make its own assessment. The SR/NSR assessment will be made taking into consideration the full context of the trial or study in which the device will be used. For example, a surgery necessary to implant a device would form a part of the SR/NSR determination.

If the IRB determines that the study is SR, then it would notify the sponsor and investigator of its decision. They study would then not be approved until an IDE was obtained.

Policy statement on devices

Any hospital engaged in or allowing the use of new devices, those for which there is not an FDA approval in effect, should have in place a policy guiding their use. An appropriate policy would be along the following lines:

A. Any proposed use of an investigational device (ID) must be reviewed by the hospital for scientific [and financial] merit, as well as compliance with all applicable laws;

B. Any proposed use of an ID shall be reviewed for determination as to coverage prior to their introduction.

J. Mark Waxman, JD is General Counsel for CareGroup Healthcare System in Boston, and Editorial Advisory Board Member of IRB Advisor.


1. In a subsequent article, it was claimed that the informed consent form had been lost, but was found.

2. The issues raised because infants were involved will be discussed in a later article. See also [Kennedy Krieger]

3. See generally 21 CFR Parts 312, 812.

4. 21 CFR § 812.2.