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U.S. Department of Health and Human Services

Training and Continuing Education

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Presentation: Institutional Review Board: Compassionate and Emergency Use

(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

  • Understand when an emergency need to use an unapproved device may occur
  • Describe the role and responsibilities of the physician who wants to use the device on a patient for emergency or compassionate use
  • Identify the IRB’s responsibilities in the compassionate use of an unapproved device

Emergency use of an unapproved device

  • What is an emergency situation
  • Physician’s role and responsibilities before and after emergency use
  • Informed consent

Compassionate use of an unapproved device

  • Describe what is meant by compassionate use
  • The physician responsibilities with compassionate use
  • The sponsor and the IRB’s role in compassionate use
  • Practice

Emergency use of an unapproved device

Emergency use of an unapproved test article is not research

What is an emergency situation?

  • All conditions must exist
    • Life-threatening disease or serious condition requiring immediate use
    • No generally accepted alternative for treating the condition is available
    • There is no time to use existing procedures to obtain FDA approval of an IDE.

When can a device be used in an emergency situation?

  • There is no Investigational Device Exemption (IDE)
  • Physician wants to use the device in a way not approved under an existing IDE
  • A physician is not part of the IDE study

Physician’s role

  • Determine whether the following conditions are met
    • Patient is in a life threatening situation
    • immediate use of the device is needed
    • no alternative
    • no time for FDA approval of an IDE
  • Assess the potential for benefits from the unapproved use
  • Have substantial reason to believe that benefits will exist

Physician’s responsibilities

  • Physician should follow patient protection measures
    • Institutional clearance per institution policy
    • IRB chairperson concurrence
    • Authorization from the sponsor - if an IDE exists
  • Independent assessment by a physician who is not participating in the investigation
  • Informed consent from patient or legally authorized representative
    • Does not have to follow the informed consent requirements at 21 CFR 50.25
  • What if there is no time to find an uninvolved physician? (21 CFR 50.23)
  • The physician makes the determinations
    • Life threatening disease or condition
    • Immediate need
    • No alternative
    • No time for FDA approval of an IDE
    • Assessment of potential benefit
    • Substantial reason to believe benefit will occur
  • The physician has his/her evaluation reviewed and evaluated in writing by an uninvolved physician
  • Submit that report to IRB within 5 working days after the use
  • What if No informed consent can be obtained? (21 CFR 50.23)
  • The physician and a physician who is not participating in the clinical investigation must certify in writing ALL of the following:
    • Life-threatening situation necessitating the use of the device
    • No alternative therapy

Physician’s responsibilities after emergency use

  • If an IDE exists, notify the sponsor
    • The sponsor must report to FDA
  • If an IDE does not exist, notify FDA of the emergency use and provide FDA
    • a written summary of the emergency use,
    • patient protection measures, and
    • any scientific results
  • Report to the IRB within five days and otherwise comply with IRB provisions
  • Evaluate the likelihood of a similar need for the device
  • If similar need is likely, obtain an IDE from FDA for the subsequent use and IRB approval

Compassionate use of an unapproved test article

  • Single patient or small group use of an unapproved device

Compassionate use of an unapproved device

  • Compassionate use is not research
  • Unapproved device for serious disease or condition
  • No alternative
  • Patient does not meet inclusion criteria

Physician’s responsibilities

  • A physician can use an unapproved device to treat, diagnose, or monitor a patient with a serious disease or condition
  • The probable risk to the patient is not greater than the probable risk from the disease
  • Physician requests authorization from sponsor
  • Sponsor may agree or disagree
  • The physician should not treat the patient until FDA concurs with the use
  • Devise schedule for patient monitoring
  • Address specific needs of the patient
  • Detect possible problems
  • Obtain independent assessment from uninvolved physician
  • Obtain IRB chair’s concurrence
  • Clearance from institution, if appropriate
  • Obtain consent from the patient
  • Report any problems as a result of the device use to the IRB and sponsor
  • Write a summary of the use and give to sponsor

Sponsor responsibilities

  • If sponsor disagrees with the use
    • The physician cannot use the device
  • If sponsor authorizes the use
    • An IDE supplement is submitted to FDA requesting approval for a protocol deviation [21 CFR 812.35(a)].
  • IDE Supplement
    • FDA’s concurrence is based on:
      information submitted; evidence that safety and effectiveness justifies the use; the use would not interfere with the conduct of a clinical trial to support marketing approval
  • Prior FDA concurrence is required before compassionate use occurs

IRB and compassionate use

  • IRB chair concurrence (documented)
  • Ensure FDA concurrence
  • Review consent document
  • Receive reports after the use
  • Receive reports of problems

Let’s put this into practice

  • Interventional Cardiologist completed IDE study of a stent
  • Continued to use the stent in his patients (>10) when he found coronary thrombosis. He declared this emergency use.

What would concern your IRB?

  • On an FDA inspection, we found that a physician used an IDE device for compassionate use. There was no FDA or IRB concurrence.

How can an IRB help prevent this from happening?

  • Approved biliary stent used in the carotid artery for carotid stenosis.
  • Physician said he did this to treat his patient.

Is this emergency use or compassionate use or neither?

References for emergency use

  • Regulation
    • 21 CFR 56.104(c)
    • 21 CFR 50.23
    • 21 CFR 812.35(a)(2)
    • 21 CFR 812.150(a)(4)
  • Guidance
    • Federal Register/vol.50, No. 204/Tuesday, October 22, 1985
    • FDA Information Sheets

References for compassionate use

  • Food, Drug, and Cosmetic Act
    •  Section 561 Expanded Access to Unapproved Therapies and Diagnostics
  • Regulation
    • 21 CFR 812.35(a)
  • Guidance

Key points

  • Physicians can use unapproved devices in emergency situations
  • IRBs must receive reports within five working days
  • Subsequent emergency use-needs IDE
  • Compassionate use requires prior FDA and IRB concurrence
  • In compassionate use, IRB’s should: document their concurrence, ensure FDA concurrence, receive and review reports