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130. Emergency, Compassionate, Treatment, and Continued Uses of Investigational Devices

FDA and the University IRB recognize there may be circumstances for a health care provider to use an unapproved or investigational device to save the life of a patient or to help a patient suffering from a serious disease or condition for which there no other alternative therapy exists (per (§812.36). FDA offers five categories of expanded access uses of unapproved/investigational devices:

  • emergency use,
  • planned emergency research,
  • compassionate use (for single patient or small groups),
  • treatment use, and
  • continued access.

Expanded access uses require the treating physician or investigator to follow as many subject protection procedures as possible, including

  • obtaining clearance from her/his institution/business for the use;
  • obtaining prior FDA approval (required for all but emergency use);
  • obtaining authorization from the IDE sponsor;
  • obtaining prior IRB approval for the use (unless an emergency use exemption from requirement for prior IRB approval applies); and
  • obtaining informed consent from the patient or her/his legal representative (unless emergency use requires an exception from general consent requirements).

The requirements and physician responsibilities for each of the five expanded uses are itemized below (obtained from FDA guidance: IDE Early/Expanded Access).

Emergency Use of Unapproved Device

Emergency situations may arise in which there is a need to use an unapproved device in a manner inconsistent with the approved investigational plan or by a physician who is not part of the clinical investigation of the device. Emergency use of an unapproved device may occur before an IDE is approved or when an IDE does not exist.

Emergency use of an unapproved device is not research. Patients receiving an investigational device for emergency use are not research participants and outcome data from emergency use may not be included in reports of research activities subject to DHHS regulations.

Emergency use does not require prior FDA approval.

Criteria for Emergency Use of Unapproved Device

For a physician to use an unapproved device to treat a patient, the following conditions must be true:

  • The patient is faced with a life-threatening disease or serious condition (see online Policy Manual Definitions) requiring immediate use.
  • No generally accepted alternative exists for treating the disease/condition.
  • There is no time to use standard procedures to obtain FDA approval of an IDE.
  • Prior IRB approval* is obtained unless time is insufficient for IRB review.

*If prior IRB approval is not obtained, the physician must notify the IRB within five days of the emergency use.

Physician Responsibilities for Emergency Use of Investigational Device

As noted previously, the treating physician must obtain informed consent for the use of the investigational device from the patient or her/his legally authorized representative unless informed consent cannot be obtained prior to the emergency use. In the latter case, the treating physician must ensure the use complies with regulatory requirements for an emergency use exception from general consent requirements and provide the IRB with a written statement from an Independent Physician confirming the necessity of using the device without informed consent (per FDA regulations at §50.23 and IRB policy for FDA regulations for human research protection). The Independent Physician must confirm that

  • the patient is confronted by a life-threatening situation necessitating the use of the device;
  • the patient is unable to provide legally effective informed consent because of an inability to communicate;
  • time is not sufficient to obtain consent from the patient's legal representative; and
  • no alternative method of approved or generally recognized therapy is available that provides an equal or greater likelihood of saving the life of the patient.

Prior to (if time allows) or within five days of the emergency use, the treating physician must notify the IRB. Obtaining prior approval for or notifying the IRB of emergency use is done through the University's online submission process via IRBNet. The physician should download and complete the form for single patient, emergency use. The certification of the independent physician can be documented on the same form or as a separate document.

After the emergency use, the physician must comply with requirements for reporting problems associated with the device use and patient outcomes to the IDE sponsor as specified in the investigator agreement. The treating physician must report problems associated with the device use to the IRB.

NOTE: Emergency use is specific to a single patients or small group of patients. The physician must obtain an IDE from FDA and prior IRB approval for subsequent uses of the device.

Planned Emergency Research Involving an Investigational Device

FDA regulations at §812.47 codify requirements for planned emergency research involving investigational devices in which human participants are in a life-threatening situation and it is not feasible to obtain informed consent prior to participant involvement in the research.

At this time, neither the University nor its Affiliate sites actively conduct or manage planned emergency research; therefore, the University does not have policies in place for such research. Investigators interested in conducting planned emergency research must contact the Research Integrity Office (RIO) to discuss the possibility of engaging in this type of research. As warranted, the IRB will develop and publish policy for planned emergency research.

Compassionate Use Provision for Investigational Device (for Single Patient/Small Group Access)

FDA and the University IRB recognize that there are circumstances in which an investigational device is the only option available for a patient faced with a serious, albeit not life-threatening, disease or condition. An investigational device is typically approved under the FDA's compassionate use provision for use by an individual patient but may be approved for use by a small group.

Compassionate use may not commence until the FDA approves the use, generally via sponsor submission to FDA of an IDE supplement requesting approval for a compassionate use under section §812.35, item (a).

Criteria for Compassionate Use of Investigational Device

  • Patient is faced with a serious disease or condition (see online Policy Manual Definitions).
  • No generally accepted alternative exists for treating the disease/condition.
  • Patient does not meet the requirements for inclusion in an active IDE Study but the treating physician believes the device may provide a benefit in treating/diagnosing the patient's disease/condition.

Physician Responsibilities for Compassionate Use of Investigational Device

The licensed physician must obtain prior approval from FDA or the device sponsor and the IRB for compassionate use of an investigational device, and must obtain clearance for the device use from her/his institution.

Obtaining prior IRB approval for compassionate use of an investigational device is done through the University's online submission process via IRBNet. The physician should download and complete the form for single patient, expanded uses of drugs/devices.

After obtaining FDA and IRB approval for the compassionate use, the physician must monitor the patient for problems arising from use of the device, with consideration for the investigational nature of the device and the specific needs of the patient.

As soon as possible after the compassionate use, the physician must submit a follow-up report to the FDA directly or via the IDE sponsor, and to the IRB. The report should provide summary information about patient outcome and problems that occurred as a result of the device use, and any other information as required by the investigator agreement.

Expanded Use of Investigational Device under a Treatment IDE

During the IDE Study or prior to final action on the marketing application for an investigational device, it may be appropriate to expand use of the device to treat patients not in the trial under the provisions of a treatment investigational device exemption (AKA: Treatment IDE) (per FDA regulations at §812.36).

Criteria for Expanded Use under Treatment IDE

  • The device is intended to treat or diagnose an immediately serious or life-threatening disease/condition (see online Policy Manual Definitions).
  • No comparable or satisfactory alternative device or other therapy exists for treating or diagnosing the disease/condition.
  • The device is under investigation in a controlled clinical trial for the same use under an approved IDE, or such clinical trials have been completed.
  • Data from the IDE Study suggest that the device is effective for the approved use.
  • The sponsor of the investigation is actively pursuing marketing approval of the device.

Physician Responsibilities for Treatment IDE

The licensed physician must obtain approval from FDA or the device sponsor (commonly under a Treatment IDE) and the IRB prior to any non-emergency, treatment uses of an investigational device.

Obtaining prior IRB approval for non-emergency, treatment use of an investigational device is done through the University's online submission process via IRBNet. The physician should download and complete the form for single patient, expanded uses of drugs/devices. NOTE: Treatment uses may not be limited to a single patient.

The licensed physician who receives an investigational device for treatment use under a treatment IDE is an "investigator" under the IDE and is responsible for meeting all applicable investigator responsibilities under the following regulations:

  • §812.100 and §812.110 for investigational device exemptions, including requirements for IRB and FDA approval, compliance with signed investigator agreement, supervising device use, financial disclosure, and disposition of unused devices;
  • §50 for the protection of human subjects including requirements for informed consent; and
  • §56 for IRB review and approval.

A physician using an investigational device under a treatment IDE must report all unanticipated adverse device effects to the IRB within five business days and the IDE sponsor within 10 days of learning of the effect (or as specified in the investigator agreement).

A physician using an investigational device under a treatment IDE must submit progress and final reports to the IRB and the IDE sponsor with sufficient information about the local device use for the sponsor to comply with FDA IDE reporting requirements. Report content must include

  • the physician-investigator's name;
  • device name;
  • number of devices received and used;
  • the number of patients the physician treated with the device under the treatment IDE;
  • brief summary of the study in progress;
  • summary of anticipated and unanticipated adverse device effects;
  • deviations from the approved investigational plan;
  • (final report only), disposition of unused devices, and summary of results and conclusions; and
  • other reporting requirements as specified in the investigator agreement.

Continued Access Policy for Extended Use of Investigational Device

FDA may allow continued enrollment of participants or patients after completion of a controlled clinical trial under an IDE to allow access to an investigational medical device while the marketing application is being prepared by the sponsor or reviewed by FDA.

Such extended investigations permit patients and/or physicians continued access to the devices while allowing the collection of additional safety and effectiveness data to support the marketing application or address new questions regarding the investigational device.

Sponsors often propose a separate Continued Access Study, independent from the pivotal trial but for the same patient population. Sponsor requirements for an extended investigation can be found in the FDA guidance: IDE Early/Expanded Access.

Criteria for Continued Access to Investigational Device

After completion of the clinical trial, FDA may allow extended investigations if there is

  • a public health need for the device; or
  • preliminary evidence indicates the device is likely to be effective and no significant safety concerns have been identified for the proposed indication.

Physician/Investigator Responsibilities for Continued Access to Investigational Device

The licensed physician must obtain prior approval from FDA or the device sponsor and the IRB for any extended investigations involving an investigational device.

Obtaining prior IRB approval for extended investigations involving an investigational device is done through the University's online submission process via IRBNet. The physician should download and complete the form for single patient, expanded uses of drugs/devices. NOTE: Extended investigation uses may not be limited to a single patient.

The licensed physician who uses an investigational device under the Continued Access Policy is an "investigator" under the IDE and is responsible for meeting all applicable investigator responsibilities under the following regulations:

  • §812.100 and §812.110 for investigational device exemptions, including requirements for IRB and FDA approval, compliance with signed investigator agreement, supervising device use, financial disclosure, and disposition of unused devices;
  • §50 for the protection of human subjects including requirements for informed consent; and
  • §56 for IRB review and approval.

A physician using an investigational device under the Continued Access Policy must continue to report all unanticipated adverse device effects to the IRB within five business days and the IDE sponsor within 10 days of learning of the effect (or as specified in the investigator agreement).

A physician using an investigational device under the Continued Access Policy must submit progress and final reports to the IRB and the IDE sponsor with sufficient information about the local device use for the sponsor to comply with FDA IDE reporting requirements. Report content must include

  • the physician-investigator's name;
  • device name;
  • number of devices received and used;
  • the number of patients the physician treated with the device under the Continued Access Policy;
  • brief summary of the continued access study in progress;
  • summary of anticipated and unanticipated adverse device effects;
  • deviations from the approved investigational plan for continued access;
  • (final report only), disposition of unused devices, and summary of results and conclusions; and
  • Other reporting requirements as specified in the investigator agreement.

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