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EMERGENCY USE OF EXPERIMENTAL DRUGS OR DEVICES

The use of an experimental drug biologic or device for the treatment of a single patient in a life-threatening situation for which there is no acceptable treatment available, may be undertaken without prospectively obtaining IRB approval provided an emergency situation exists. The following conditions should exist for a situation to be considered an emergency:

  1. The patient is suffering from a life-threatening condition
  2. No acceptable alternative treatment available.
  3. Because of the immediacy of the need to use the drug or device, there is not sufficient time to use existing procedures to obtain IRB approval.

Life-threatening includes the scope of both life-threatening and severely debilitating, as defined below.

Life-threatening means diseases or conditions where the likelihood of death is high unless the course of the disease is interrupted and diseases or conditions with potentially fatal outcomes. The criteria for life-threatening do not require the condition to be immediately life-threatening or to immediately result in death. Rather, the subjects must be in a life-threatening situation requiring intervention before review at a convened meeting of the IRB is feasible.

Severely debilitating means diseases or conditions that cause major irreversible morbidity. Examples of severely debilitating conditions include blindness, loss of arm, leg, hand or foot, loss of hearing, paralysis or stroke.

FDA regulations do not provide for expedited IRB approval in emergency situations. Therefore, the terms "interim", "compassionate", "temporary", or any other terms implying an expedited approval process are not authorized and do not apply for emergency use of test articles.

What to do. If the physician believes it may be necessary to use a test article for emergency treatment, s/he should take the following steps:

1. Assess the patient's eligibility. (a) The patient must be in a life-threatening or severely debilitating situation, according to the definitions given above. (b) The patient must not meet the criteria of any existing study protocol. (c) There is no appropriate IRB-approved study protocol. (d) There is not sufficient time to obtain IRB approval at a convened meeting of the board.

2. Obtain independent assessment. Obtain and document an independent assessment by a physician uninvolved with the patient's medical or surgical care.

3. Contact the producer of the test article. Determine whether the patient may receive treatment under an existing IND or IDE authorization.

4. Contact the Food and Drug Administration (FDA). Obtain an emergency IND or IDE if an IND or IDE is unavailable from the producer of the test article.

5. Notify local officials. Notify appropriate officials in the (a) Pharmacy and (b) administration of the facility (hospital or clinic) where the patient will receive the emergency treatment.

6. Obtain informed consent. Obtain and document informed consent from the patient or the patient's legally authorized representative. See below for details.

7. Inform the IRB: Either prior to or within 24 hours after, inform the IRB of the need to use a test article for emergency treatment, by sending an e-mail to the IRB-1 chairperson, copying the IRB Director and the Institutional Coordinator, Research Review. Contact the IRB office for the current e-mail addressed for the Chair, Director and Coordinator.

Then within five working days after the use of the test article, provide a written report including:

1. The patient's initials and age (if the age is 89 or above, indicate "> 89 yrs" rather than specifying the age)

2. Physical condition and the justification for use of the experimental drug or device (e.g., documentation that no available alternative therapy exists)

3. The date of this report, the date the IRB was initially notified (the e-mail notification), and the date the patient was treated with the test article.

4. Therapeutic plan (e.g., dose, mode of administration, duration of planned therapy)

5. IND/IDE and the name of the sponsor that provided the drug or device

6. The name of a physician not involved in the patient's care who made an independent assessment of the patient and agreed that the drug or device is needed for a life-threatening situation

7. The name of the hospital in which the patient was treated, and

8. A copy of the consent document, or the written certification prepared to document that it was not possible to obtain consent. See procedures described below.

Informed consent.

Even for emergency use, the physician is required to obtain informed consent of the patient or the patient's legally authorized representative, unless the circumstances do not permit it. The consent should be prepared using the template found on the IRB Forms page and should NOT imply that the emergency treatment with a test article is research. Informed consent is not required if the treating physician and another physician who is not otherwise involved in the treatment of the patient, certify in writing that all of the following conditions exist:

  1. The patient is confronted by a life-threatening condition that requires immediate treatment.
  2. Informed consent can not be obtained because of inability to communicate with, or obtain legally effective consent from the patient, or time is not sufficient to obtain consent from the patient's legally authorized representative.
  3. No alternative method of approved or generally recognized therapy is available to provide equal or greater likelihood of saving the patient's life.

The physician should follow the specific guidance for either the emergency use of unapproved drugs or biologics or for the emergency use of unapproved devices, which follow. In all cases, the physician using the test article must notify the IRB-1 Chairperson or designee that an Emergency Use of a test article will occur, or if it has already occurred. Notification will be within 24 hours, by sending an e-mail to the IRB-1 Chairperson, copying both the IRB Director and the Institutional Coordinator, Research Review. Please contact the IRB office for their e-mail addresses to ensure the e-mail is directed to the correct people. This notification will initiate tracking by the IRB office to ensure the treating physician files the required report with the IRB after the emergency treatment with a test article has taken place, as describe below:

The emergency treatment using a test article will be reported to IRB-1 at its next scheduled meeting.

Important: Because the emergency use was initiated prior to approval of a protocol by the convened IRB, the emergency care may not be claimed as research, nor may the outcome of the emergency use be included in any report of a research activity.

  • Emergency use of an investigational drug or biologic.
    The use of an unapproved investigational drug or biologic requires an IND. If the intended patient does not meet the inclusion criteria of an existing clinical study, or if an approved clinical study does not exist, the usual procedure is to contact the manufacturer and determine if the drug or biologic can be made available for emergency use under the company's IND.

  • The emergency use exemption may be used only one time. FDA regulations require that any subsequent use of the investigational drug or biologic at an institution must be done under a protocol with prior review and approval of the IRB.

  • Emergency use of an investigational device.
    An unapproved medical device is defined as a device that is used for a purpose or condition for which the device requires, but does not have, an approved application for premarket approval under section 515 of the Federal Food, Drug, and Cosmetic Act [21 U.S.C. 360(e)]. While an unapproved device may be used in human subjects only if it is approved for clinical testing under an approved application for an Investigational Device Exemption (IDE), the FDA has provided for an emergency exception to that rule: emergencies where an unapproved device may offer the only possible life-saving alternative, but an IDE for the device does not exist, or the proposed use is not approved under an existing IDE, or the physician or institution is not approved under the IDE. The FDA permits a physician to use an unapproved device in such an emergency, provided that the physician later justifies to FDA that an emergency actually existed.

    • Requirements for Emergency Use

    Each of the following conditions must exist to justify emergency use:

    1. the patient is in a life-threatening condition that needs immediate treatment;
    2. no generally acceptable alternative for treating the patient is available; and
    3. because of the immediate need to use the device, there is no time to use existing procedures to get FDA approval for the use.

    The physician must determine whether these criteria have been met, must assess the potential for benefits from the unapproved use of the device, and must have substantial reason to believe that benefits will exist. The physician may not conclude that an "emergency" exists in advance of the time when treatment may be needed based solely on the expectation that IDE approval procedures may require more time than is available. Physicians should be aware that FDA expects them to exercise reasonable foresight with respect to potential emergencies and to make appropriate arrangements under the IDE procedures far enough in advance to avoid creating a situation in which such arrangements are impracticable.

    In the event that a device is to be used in circumstances meeting the criteria listed above, the device developer should notify the Center for Devices and Radiological Health (CDRH), Program Operation Staff by telephone (301-594-1190) immediately after shipment is made. [Note: an unapproved device may not be shipped in anticipation of an emergency.] Nights and weekends, contact the FDA Office of Emergency Operations (HFA-615) 301-443-1240.

The physician should do the following prior to use:

    1. obtain an independent assessment by an uninvolved physician;
    2. obtain informed consent from the patient or a legally authorized representative;
    3. notify officials of the institution where the patient will be treated, as specified by their institutional policies;
    4. notify the Institutional Review Board (IRB); and
    5. obtain authorization from the IDE holder, if an approved IDE for the device exists.

    The physician should do the following after use:

    1. evaluate the likelihood of a similar need for the device occurring again, and if future use is likely, immediately initiate efforts to obtain IRB approval and an approved IDE for the device's subsequent use.
    2. notify the sponsor of the emergency use if an IDE for the use does exist, or if an IDE does not exist, notify the FDA of the emergency use (CDRH Program Operation Staff 301-594-1190) and provide FDA with a written summary of the conditions constituting the emergency, subject protection measures undertaken, and results of treatment.

    •IMPORTANT: Subsequent emergency use of the device may not occur unless the physician or another person obtains approval of an IDE for the device and its use. If an IDE application for subsequent use has been filed with FDA and FDA disapproves the IDE application, the device may not be used even if the circumstances constituting an emergency exist. Developers of devices that could be used in emergencies should anticipate the likelihood of emergency use and should obtain an approved IDE for such uses.

    Exception from Informed Consent for Planned Emergency Research

    The conduct of planned research in life-threatening emergent situations where obtaining prospective informed consent is waived, is provided by 21 CFR 50.24. The research plan must be approved in advance by FDA and the IRB, and publicly disclosed to the community in which the research will be conducted. Such studies are usually not eligible for the emergency approvals described above. The information sheet "Exception from Informed Consent for Studies Conducted in Emergency Settings: Regulatory Language and Excerpts from Preamble," is a compilation of the wording of 21 CFR 50.24 and pertinent portions of the preamble from the October 2, 1996 Federal Register. It is found at the following web address: http://www.fda.gov/oc/ohrt/irbs/except.html Investigators contemplating this type of research are urged to contact the IRB office early in the planning stages.