Ad hoc subcommittees

VIII.03

 

 

Emergency Use of an Investigational Drug, Biologic, or Unapproved Medical Device

 

 

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Objective

This procedure describes the process of acknowledgement of receipt of notification by a physician of the intended use of an investigational drug or unapproved device in an emergency situation. The procedure also describes the physician’s follow-up after the treatment.

 

Policy/Procedure

I.        If a situation arises necessitating the use of an investigational drug, agent or biologic, in an emergency situation (“Event”) the treating physician must:

 

A.     if the intended subject does not meet the criteria of an existing study protocol, or if an approved study protocol does not exist,  contact the manufacturer of the investigational drug, agent, or biologic to determine if the test article can be made available for the emergency use under the manufacturer’s IND; or

 

B.     if, due to the emergency situation, there is not time for submission of the IND, the treating physician may make a direct request to the FDA to authorize shipment of the test article in advance of the IND submission.

 

C.     Contact the CPHS Chairman of the Executive Committee (Executive Chairman), or designee, who must be an MD, and notify him/her of the Event and the following:

1.      An explanation of the conditions supporting the determination that the patient is in an immediate serious or life-threatening condition that needs immediate treatment;

2.      There is no generally acceptable alternative treatment; and

3.      An informed consent document for review. The physician is required to obtain informed consent of the patient or the patient’s legally authorized representative. The Executive Chairman will determine that the physician will be obtaining informed consent or, if the subject or the legally authorized representative cannot give consent, the Executive Chairman will review the situation to evaluate whether it meets the FDA regulatory requirements for waiver of informed consent with emergency use of a drug or biologic. If the latter is the case, the treating physician and a physician who is not otherwise participating in the treatment of the subject shall certify in writing the following:

a)      The patient is confronted by a life-threatening and/ or severely debilitating situation necessitating the use of the investigational drug, agent, or biologic;

b)      Informed consent cannot be obtained because of an inability to communicate with, or obtain legally effective consent from, the patient;

c)      Time is not sufficient to obtain consent from the patient’s legal representative; and

d)      No alternative method of approved or generally recognized therapy is available that provides an equal or greater likelihood of saving the patient’s life.

4.      If, in the treating physician’s opinion, immediate use of the test article is required to preserve the patient’s life, and if time is not sufficient to obtain an independent physician’s determination that the four conditions listed above apply, the treating physician should make the determination and, within five working days after the use, have the determination reviewed and evaluated in writing by a physician who is not participating in the Event.

5.      The Executive Chairman will review the use to evaluate whether the situation meets the FDA regulatory requirements that allow exemption from CPHS review of the Event. The following may be requested to facilitate the evaluation:

a)      An authorization from the sponsor or manufacturer to allow the use by the treating physician for the test article;

b)      An approved IND or a letter explaining exemption from the FDA;

c)      An adequate description of the situation regarding the use of the test article with an independent physician’s certification, if applicable; and

d)      Any other information that may aid in the evaluation.

6.      The Executive Chairman will notify the Manager of the Office of Research Support Committees of the use within 24 hours of receiving the request and granting permission.

 

D.     After emergency use /treatment of investigational drugs, agents, or biologics

1.      The treating physician is required to submit a written follow-up report to the CPHS within five (5) working days of the emergency use of an investigational drug, agent or biologic. This report should include:

a)      Name of the investigational drug, agent, or biologic;

b)      Conditions under which the investigational drug, agent, or biologic  was administered;

c)      Date and time administered;

d)      Any adverse events or unanticipated problems to recipient or others; and

e)      Outcomes, if known.

2.      Any subsequent use of the test article is subject to CPHS review. FDA acknowledges, however, that it would be inappropriate to deny emergency treatment to a second individual if the only obstacle is that the CPHS has not had sufficient time to convene a meeting to review the issue.

3.      The treating physician is to evaluate the likelihood of a similar need for the drug, agent, or biologic and if future use is likely, immediately initiate efforts to obtain CPHS approval and an FDA approved IND for the drug, agent or biologic’s subsequent use.

 

II.     The FDA recognizes that emergencies arise where an unapproved medical 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. Using its enforcement discretion, FDA may choose to approve the use of an unapproved medical device in such an emergency, provided the treating physician justifies to the FDA that an emergency actually existed.

 

A.     To be considered an emergency each of the following must exist:

1.      The patient is in a life-threatening condition that needs immediate treatment; and

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.

 

B.     The FDA expects the treating physician to determine the following:

1.      Whether the criteria for emergency use have been met;

2.      Potential benefits from the unapproved use of the device with substantial reason to believe that benefits will exist;

3.      Assurance the decision that an “emergency” exists is not based solely on the expectation that IDE approval procedures may require more time than is available.

 

C.     The treating physician is expected to follow as many subject protection procedures as possible. These include:

1.      Obtaining an independent assessment by an uninvolved physician;

2.      Obtaining informed consent from the patient or a legal representative;

3.      Notifying institutional officials as specified by institutional policies; and

4.      Notifying the CPHS; and

5.      Obtaining authorization from the IDE holder, if an approved IDE for the device exists.

 

D.           The treating physician shall contact the CPHS Executive Chairman, or designee, (must be an MD),  and notify him/her of the Event and include in writing the items in I.C above.

 

E.            The investigator is required to obtain informed consent of the subject or the subject's legally authorized representative unless both the investigator and a physician who is not otherwise participating in the Event certify in writing all of the following:

1.      The subject is confronted by a life-threatening situation necessitating the use of the unapproved medical device

2.      Informed consent cannot be obtained because of an inability to communicate with, or obtain legally effective consent from, the patient

3.      Time is not sufficient to obtain consent from the patient's legal representative.

4.      No alternative method of approved or generally recognized therapy is available that provides an equal or greater likelihood of saving the patient's life.

 

F.      If, in the treating physician’s opinion, immediate use of the test article is required to preserve the patient's life, and if time is not sufficient to obtain an independent physician's determination that the four conditions above apply, the treating physician should make the determination and, within 5 working days after the use of the unapproved medical device, have the determination reviewed and evaluated in writing by a physician who is not participating in the treatment. 

 

G.     The treating physician is required to submit a written follow-up report to the Executive Chairman, or his/her designee within five (5) working days of the emergency use of an unapproved device. This report should include:

1.      Name of the unapproved device;

2.      Conditions under which the unapproved device was administered;

3.      Date and time administered;

4.      Name, IDE number and source of the device;

5.      Any adverse events or unanticipated problems to recipient or others; and

6.      Outcomes, if known.

 

H.     If an IDE for the use does exist, the treating physician is to notify the sponsor of the emergency use, of if an IDE does not exist, he/she is to notify the FDA of the emergency use and provide the FDA with a written summary of conditions constituting the emergency, patient protection measures, and results.

 

I.        The treating physician is to evaluate the likelihood of a similar need for the unapproved device and if future use is likely, immediately initiate efforts to obtain CPHS approval and an FDA approved IDE for the unapproved device’s subsequent use.

 

 

Applicable Regulations and Guidelines

21CFR 50 (http://www.access.gpo.gov/nara/cfr/waisidx_04/21cfr50_04.html )

21CFR 50(http://www.access.gpo.gov/nara/cfr/waisidx_04/21cfr56_04.html )

38CFR 16 (http://www.access.gpo.gov/nara/cfr/waisidx_04/38cfr16_04.html )

45CFR 46 http://www.access.gpo.gov/nara/cfr/waisidx_04/45cfr46_04.html )

ICH E6 (http://www.ich.org/MediaServer.jser?@_ID=482&@_MODE=GLB )

AAHRPP Evaluation Instrument For Site Visitors (http://www.aahrpp.org/Documents/D000043.PDF )FDA Information Sheets (http://www.fda.gov/oc/ohrt/irbs/drugsbiologics.html#emergency ) & (http://www.fda.gov/oc/ohrt/irbs/devices.html#emergency

 

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Updated 5/2007, Report broken links