Guidance

Humanitarian Use Device Requirements for U-M Physicians & Investigators

IRBMED
Nov 17, 2023 11:00 am

The Food and Drug Administration (FDA) defines a Humanitarian Use Device (HUD) as “a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year.” Because the research has shown a HUD to have only “probable benefit,” rather than “a reasonable assurance of effectiveness,” every HUD requires IRB approval and oversight, whether for use in clinical care or in research.

A Humanitarian Device Exemption (HDE) is an application that is similar to a premarket approval (PMA) application but is exempt from the effectiveness requirements of the Food, Drug and Cosmetic Act (FD&C Act). FDA approval of an HDE authorizes an applicant to market a HUD subject to certain profit and use restrictions.

  • Basic Instructions

    1. Before submitting an IRBMED application, determine which of the following applies:

    • A HUD will be used for clinical care (HUD Use);
    • Safety and effectiveness data will be collected for the approved use; or
    • The HUD will be studied for a new indication.

    The first two uses are covered under an FDA-issued Humanitarian Device Exemption (HDE). In most instances, the device’s manufacturer holds the HDE. Occasionally, the UM or other physician is the HDE holder.

    The third use requires an Investigational Device Exemption (IDE). An IDE may be issued either by the IRBMED or by the FDA, depending on the risk level associated with the use. The sponsor (whether physician/investigator, manufacturer, or other party) holds an IDE to conduct clinical research designed to determine the appropriateness of the device for a new indication.

    2. Obtain the HDE number or, if the FDA has granted an IDE, the IDE number.

    3. Follow the steps corresponding to the type of HUD use that you are planning:

    • On-label use without collection of safety and effectiveness data (HUD Use)
    • On-label use with collection of safety and effectiveness (HUD Clinical Investigation)
    • Emergency or off-label use of a HUD approved for use at UM
    • Emergency use of a HUD for which no UM IRB-approved project exist
    • Use of a HUD in a clinical investigation designed to determine a device’s suitability for a new indication

    See Types of HUD Use section (below) for steps corresponding to each of the above uses.

    4. See the Required Reporting in HUD Clinical Care section (below) for instructions on reporting adverse events and ORIOs (HUD under an HDE, not an IDE).

  • Types of HUD Use

    1. On-label use of a HUD in clinical care, without collection of safety and effectiveness data

    • On the eResearch section 01-1, select Humanitarian Use Device (HUD) under an HDE.
    • eResearch will route you to the required sections, starting with 01-5. Include the following in your responses:
      • A summary of your proposed use of the device (question 1.8)
      • The identity of the HDE holder (If you hold the HDE, state this in box 1.8; otherwise, include it within the documentation uploaded in 1-5.12.)
      • Any patient follow-up visits, tests, or other procedures (box 1.8; upload further documentation in 1-5.12.)
      • Any screening procedures (questions 1.8, 1-5.3 and 1-5.7)
      • A description of the HUD procedure itself (boxes 1.8, 1-5.5, and 1-5.6)
      • A copy of the HDE approval order (Upload this in 1-5.12.)
      • The product labeling (1-5.3 and 1-5.5, as applicable; if available electronically, explain in text fields and upload documentation in 1-5.12.)
      • The patient information packet that may accompany the HUD (Upload this in 1-5.12.)
      • The IRBMED-recommended HUD informed consent template (Upload this in 1-5.13.)
    • Submit the eResearch application and await IRBMED approval.
    • After receiving notification of IRBMED approval, you may administer the HUD, according to its approved labeling, without obtaining IRBMED approval for each use. When the device is used off-label or used in an emergency, report the use to the IRB within 5 calendar days.
    • Report to the manufacturer and/or FDA, as required.
    • Submit to the IRBMED all required reports (see below).

    2. On-label use of a HUD in clinical care with collection of safety and effectiveness data

    If safety and effectiveness data will support a Premarket Approval (PMA) application, the FDA requires you to follow some standard research regulations, with the exception that no IDE is needed. Complete a standard eResearch application (application type in section 01-1 "Human Subjects research involving interaction or intervention"), using the standard informed consent template for research studies.

    You may, however, follow the "Required Reporting in HUD Clinical Care" guidance below, rather than standard reporting requirements. If so, make note of the study-specific reporting plan in eResearch section 32-1.

    3. Emergency or off-label use of a HUD approved for use at UM

    • Apply consent and patient-protection measures, as required by the FDA.
    • IRBMED does not require the investigator to obtain IRB permission ahead of time to use a HUD off-label. However, FDA recommends that the user submit to the HDE holder follow-up information on the patient’s condition following the use. IRBMED requires that this report also be submitted to IRBMED as an ORIO at the same time it is sent to the HDE holder. Also, the investigator is required to submit any adverse events reports to IRBMED as required by the FDA.
    • Submit to the HDE holder follow-up reports, as required by the HDE holder and recommended by the FDA.

    4. Emergency use of a HUD for which no UM, IRB-approved project exists

    If IRBMED has not yet approved a HUD’s use at UM, follow FDA regulations for emergency use of an unapproved device. You must also follow any UMHS requirements for devices used on patients.

    Within 5 calendar days of use of the device, submit to the IRBMED a new eResearch application for Emergency Use Report.

    • In eResearch application section 01-1, select Single-patient Expanded Access Device.
    • eResearch will route you to the required sections, starting with 01-4
    • Report to the manufacturer and/or FDA, as required.
    • Submit all other reports to the IRBMED, as required (see below).

    4. Use of a HUD in a clinical investigation testing a prospective new indication

    Complete a standard eResearch application (application type in section 01-1 "Human Subjects research involving interaction or intervention"), observing all applicable UM research policies. This type of study falls under the Investigational Device Exemption (IDE) requirement. If the sponsor identifies the proposed use of the device to be non-significant risk, the IRBMED can grant an IDE. If the proposed use of the device is deemed to be significant risk (see definition in 21 CFR 812.3[m]), an IDE from the FDA is required. Generally, all off-label uses of a HUD have been designated as significant risk.

  • Required Reporting in HUD Clinical Care

    (HUD under an HDE, not an IDE)

    See also FDA guidance on the HDE program.

    1. Adverse Events

    Within 14 calendar days, UM and/or the HDE holder must submit to both the FDA and the IRBMED a Medical Device Report (MDR), informing them of one or both of the following events:

    • The HUD appears to have caused or contributed to death or serious injury.
    • The HUD appears to have malfunctioned and is likely to cause or contribute to death or serious injury should the malfunction recur.

    FDA defines a serious injury as one that:

    • is life-threatening;
    • results in permanent impairment of a body function;
    • results in permanent damage to a body structure;
    • necessitates medical or surgical intervention to preclude permanent impairment of a body function; or
    • necessitates medical or surgical intervention to preclude permanent damage to a body structure.

    When reporting the event, use the eResearch UM Serious Adverse Event form. ​As Section 32 of the eResearch application is not triggered for the HUD applications, the above outlined AE reporting plan should be followed.

    2. Other Reportable Information or Occurrences (ORIO)

    ​​​Follow the IRBMED ORIO reporting guidance. ​​ 

  • Review Process

    ​The initial review and approval of an HUD application will be conducted via the ​convened​ board review. Continuing review applications can be reviewed via the expedited process ​(expedited category #9).​​ Amendments will be reviewed per the ​​changes​​​​ proposed in the Amendment submission. ​​ See also IRBMED FAQ, headings ​"Review and Approval Process" and "Ongoing Review." 

Questions?

Contact us at irbmed@umich.edu or 734-763-4768 / (Fax 734-763-1234)
2800 Plymouth Road, Building 520, Room 3214, Ann Arbor, MI 48109-2800

A list of IRBMED staff is available in the Personnel Directory, or view the list of Regulatory Teams.

Edited By: larkspur@umich.edu
Last Updated: November 17, 2023 2:45 PM