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U.S. Department of Health and Human Services

Training and Continuing Education

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Presentation: Institutional Review Board: Humanitarian Use Devices (HUDs)

(This presentation is also available in video format with captioning and PDF printer-friendly format.)

Presented by Fabienne Santel, MD

Division of Bioresearch Monitoring
Office of Compliance
Center for Devices and Radiological Health
Food and Drug Administration

Learning objectives

  • Define Humanitarian Use Device
  • Describe FDA’s approval
  • Identify the HDE-holder responsibilities
  • Identify the physician responsibilities
  • Describe the IRB responsibilities

Topics

  • Humanitarian Use Device (HUD)
  • Humanitarian Device Exemption (HDE)
  • FDA review
  • HDE-holder responsibilities
  • Physician responsibilities
  • IRB responsibilities
  • FDA concerns

Humanitarian Use Device (HUD)

  • HUD definition
    • Title 21 CFR 814.3(n)
    • A device intended to benefit patients in the treatment or diagnosis of a disease or condition affecting fewer than 4,000 individuals in the US per year
  • Office of Orphan Products Development designates a device as a Humanitarian Use Device (HUD)
    • Verifies that the device is designed to treat or diagnose a disease or condition following the parameters in the definition
    • Reviews a description of the device
    • Reviews a description of the rare disease or condition

Humanitarian Device Exemption (HDE)

  • HDE Definition
    • Title 21 CFR 814.2
    • A premarket approval application submitted to FDA seeking a Humanitarian Device Exemption from the effectiveness requirements of sections 514 and 515 of the Food, Drug, Cosmetic Act.

Humanitarian Device Exemption Application to FDA

  • FDA approval of HDE application
    • HUD does not pose unreasonable risk of injury to patients
    • That the probable benefit outweighs risk of injury from its use
  • FDA approval of the HDE
    HDE label states
    • The device is a Humanitarian Use Device
    • The device, to treat or diagnose a specific disease or condition, is authorized by federal law
    • The effectiveness of this device for this use has not been demonstrated
  • FDA approval of a HDE application allows the HUD to be marketed

HDE-Holder

  • The HDE-holder must
    • Have both HUD designation and approved HDE from FDA before device is shipped to institutions with Institutional Review Board (IRB) oversight
  • The HDE-holder is responsible for ensuring initial and continuing IRB review
  • The HDE-holder may wish to enforce this by not shipping HUDs to institutions until after receiving documentation of IRB approval
  • The HDE-holder is responsible for ensuring
    • the HUD is not administered to or implanted in a patient prior to obtaining IRB approval at the health care facility
  • The HDE-holder must
    • Maintain IRB correspondence
    • Report clinical experience, including safety information, to FDA in annual reports

Physician Responsibilities

  • Obtain IRB approval and continuing approval
  • Follow IRB requirements
  • Give patients HUD information packet
  • Report serious adverse events and deaths using the Medical Device Reporting system at 21 CFR 803.

IRB initial review of HUDs

  • Use approval criteria at 21 CFR 56.111
    • Consideration of the patient’s need for the HUD
    • Likelihood that device is appropriate for the patient’s condition or disease state

IRB continuing review of HUDs

  • Follow written procedures for continuing review
    • Convened meeting, or
    • Expedited review is acceptable because it is an approved device

IRB review of HUDs

  • An IRB may approve the use of the HUD
    • In general
    • For groups of HUD patients that meet certain criteria
    • Under a HUD treatment protocol, or
    • On a case-by-case HUD basis

IRB limitations on HUD use based on

  • One or more measures of disease progression
  • Prior use and failure of alternate treatment modalities
  • Reporting requirements to IRB or IRB Chair
  • Appropriate follow-up precautions and evaluations
  • Any other criteria it determines appropriate

IRB withdrawal of approval

  •  IRBs must be constituted and act in accordance with the agency’s regulations and withdraw approval for:
    • – Failure to follow IRB or FDA requirements
    • – Unexpected serious harm or death
  •  Questions to ask at continuing review
    • – Reporting serious adverse events or deaths
    • – Following IRB conditions of approval or limitations

Medical Device Reporting (MDR) and Humanitarian Use Devices

  • Applies to all FDA approved devices
  • Serious adverse events and deaths must be reported to FDA and the IRB using the Medical Device Reporting system at 21 CFR 803
  • HDE-holders and IRBs should ensure that physicians know about this requirement

FDA Concerns

  • Off label use of an HUD
    • IRB should ensure that physicians are made aware of any restrictions or limitations of off-label use at the time of initial review.
    • FDA recommends informed consent and reasonable patient protections measures
      • Monitoring and considering the specific needs of the patient and limited information about risks and effectiveness of the HUD
    • Summary report to IRB and HDE-holder following the use
  • Research for HDE-approved indication
    • No Investigational Device Exemption (IDE) required
    • IRB review and informed consent recommeded
  • Research outside approved indication
    • Requires an (IDE) 21 CFR 812
    • IRB review and informed consent required

References

Summary

  • Humanitarian Use Device and Humanitarian Device Exemption
  • FDA’s approval criteria
  • HDE-holder responsibilities
  • Physician responsibilities
  • IRB responsibilities