Emergency Use Process
Emergency uses of investigational (unapproved) drugs, biologics and devices are done for the purpose of providing clinical treatment to a patient, and are not considered to be research under federal regulations. This means that any qualified physician at Seattle Children's can utilize the emergency use process to treat a patient; research experience is not necessary. If you have any questions about these materials, contact the IRB.
Emergency Use Documents
IRB Policy for Emergency Use
This Emergency Use guidance, which can also be found in Appendix A-9 of HRP-103-Investigation Manual (in Click Library) outlines the responsibilities of the physician/investigator when an emergency requires that a patient be treated with an investigational Test Article such that there is not sufficient time to obtain IRB review and approval at a convened meeting. At Seattle Children’s, emergency use procedures are used to provide access to investigational products under state and federal “Right to Try” provisions.
Emergency Use Information:
- An Emergency Use is an exception to the general rule that investigational Test Articles may only be used in human subjects who are participating in a clinical investigation/research.
- Emergency Uses are exempt from the requirement to obtain prior IRB review and approval, provided that the use is reported to the IRB within 5 working days after initiation of the Emergency Use. 21 CFR 56.104(c).
- Whenever possible, informed consent should be obtained before the investigational Test Article is used. Please see HRP-506 Template Consent Document: Emergency Use.
- The physician should check with the Investigational Drug Service about the cost of drugs as they can be charged for in certain circumstances.
- The physician should complete as many of the patient protection measures detailed in this guidance as possible before using the investigational article.
- If a physician anticipates using an investigational Test Article more than one time at Seattle Children’s, the physician should contact [email protected] about the need to submit an IRB application for prospective IRB review and approval of a research study.
Emergency Use Procedure:
Step 1: Determine whether an Emergency Use is appropriate.
Criteria for Emergency Use of a Test Article
Drugs/Biologics |
Devices |
Treating physician determines that all of the following are met: (1) Life-threatening or serious* disease or condition that needs immediate treatment; (2) No generally acceptable alternative for treating the patient is available; and (3) Due to the immediate need to use the test article, there is no time to follow existing procedures to obtain IRB approval prior to the use. Additional determinations: (4) FDA also expects the physician to make the determination that the probable risk to the person from the investigational test article is not greater than the probable risk from the disease or condition. 21 CFR 312.310(a)(1). *Relevant definitions in the Drug/Biologic context:
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Treating physician determines that all of the following are met: (1) The patient has a life-threatening* condition that needs immediate treatment; (2) No generally acceptable alternative treatment for the condition exists; and (3) Because of the immediate need to use the device, there is no time to use existing procedures to get IRB and FDA approval for the use. Additional determinations: (4) FDA also expects the physician to assess the potential for benefit from the use of the unapproved device, and to have substantial reason to believe that benefits will exist. See FDA Device Guidance. *Relevant definitions in the Device context:
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FDA must determine (based on information physician provides) that:
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An uninvolved physician (not participating in the emergency use) determines that all of the above emergency use criteria are met.
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Step 2: Take steps to obtain the investigational test article.
To Use Drugs/Biologics --> IND |
To Use Devices --> IDE |
Identify the sponsor: There are two scenarios, depending on whether the manufacturer previously obtained an IND for the drug/biologic:
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Identify the sponsor: Generally there will be a manufacturer who has applied for an IDE and will serve as the “sponsor” of the emergency use. |
Contact the manufacturer/sponsor to determine whether they are willing to provide the investigational drug/biologic. |
Contact the sponsor to determine whether they are willing to provide the investigational device. |
Contact the FDA for an Emergency IND:
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Not Required to Contact the FDA in Advance for an Emergency IDE:
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The FDA usually provides a new IND number for the specific emergency use. |
If no IDE exists, the physician should follow the above procedures and report the emergency use to CDRH as directed in Step 6. |
Type of Test Article (Branch) |
FDA Office/Division to Contact |
Drugs |
Division of Drug Information |
Biologics |
Office of Communication, Outreach and Development, |
Devices |
Office of Device Evaluation, Program Operations Staff, |
All Products, after normal working hours (8am – 4:30pm ET) |
FDA Emergency Call Center |
Step 3: Follow as many patient protection measures as possible.
Patient care should not be compromised in the event there is insufficient time to complete all of these measures:
- Concurrence of the Institutional Review Board Chair or designee.
- Please e-mail the Emergency Use Report Form (HRP-2141) to the IRB representative before calling, if possible, so the IRB representative can reference the form during the call. This form can be found in the Click Document Library.
- Please contact the IRB representatives in the following order:
- IRB Chair, Douglas Diekema, MD, MPH, [email protected].
- IRB Vice-Chair, Danielle Lewis, MD, MPH, [email protected] or at 206-884-1427.
- Investigational Drug Service, Ada Kong, PharmD, [email protected]. Note that IDS serves as an IRB representative for emergency uses of drugs/biologics and devices.
- Senior Vice-President, Chief Medical Officer, Jeff Ojemann, MD, [email protected]. Vice President, Associate Chief Medical Officer, Ruth McDonald, MD, [email protected].
- Once you have e-mailed the Emergency Use Report Form to an IRB representative, please call the paging operator at 206-987-2131 or call the designee at the number listed above to consult with the IRB representative (in the order listed above).
- Written informed consent from the patient or their legally authorized representative. Use the Seattle Children’s template Emergency Use Consent Form (HRP-506) or a form provided by the manufacturer or sponsor. This template can be found in the Click Document Library.
- Since the FDA exempts emergency uses from the usual requirements for prior IRB approval, there is no requirement to obtain IRB approval of the emergency use consent form before using it with the patient.
- OR
- Determine that the criteria are met to waive consent for the emergency use. Before using the test article, determine and, if possible, document (in writing/e-mail) that criteria for waiving emergency use consent (see below) are met, in the opinion of both the:
- Physician treating the patient with the test article; and
- Physician not participating in the emergency use (independent/uninvolved physician)
- If it is not possible to obtain consent, FDA regulations (21 CFR 50.23) allow a waiver of emergency use consent under the following conditions:
- The patient is confronted with a life-threatening situation necessitating the use of the test article;
- Informed consent cannot be obtained from the patient because of an inability to communicate with, or obtain legally effective consent from, the patient;
- Time is not sufficient to obtain consent from the patient’s legally authorized representative; and
- No alternative method of approved or generally recognized therapy is available that provides equal or greater likelihood of saving the patient’s life.
- If immediate use of the test article is needed to preserve the patient’s life, and there is not sufficient time to secure an independent physician’s determination that the four conditions for a waiver of emergency use consent (described above) apply, the treating physician should make the determination that consent cannot be obtained and proceed, but must have his/her written determination reviewed in writing by an independent physician within five working days after the emergency use of the test article. 21 CFR 50.23(b) & (c); 21 CFR 812.150(a)(5).
- DEVICES ONLY (not required for drugs/biologics): As noted above in Step 1, obtain a written assessment from an uninvolved physician that the criteria for emergency use (this is not about waiving consent) are satisfied for the patient.
- DEVICES ONLY: As noted above in Step 2, obtain authorization from the IDE sponsor, if an approved IDE exists for the device.
Step 4: Initiate treatment with the investigational agent.
Initiate Use of Drug/Biologic (IND) |
Initiate Use of Device (IDE) |
Treatment may begin when the emergency use is authorized by the FDA reviewing official. 21 CFR 312.305(d)(2)(i). |
Emergency use of the device may begin when the use is authorized by the IDE sponsor. |
Step 5: Submit a follow-up report to the IRB within 5 working days of initiating treatment with the investigational agent. The report form is located in the Click Document Library.
The report should include the following:
- Completed Emergency Use Report Form (HRP-2141) (found in Click Document Library);
- All relevant attachments requested in the report form.
Step 6: Submit a follow-up report to the FDA or sponsor within 5 working days.
Reports for Drugs/Biologics = IND |
Reports for Devices = IDE |
Physician must submit an expanded access submission to FDA within 15 working days of FDA’s authorization of the emergency use. 21 CFR 312.310(d)(2). See Form FDA 1571 and Instructions, http://www.fda.gov/Drugs/DevelopmentApprovalProcess/ |
Deviations from the investigational plan: An investigator shall notify the IDE sponsor (who will notify the FDA) of any deviation from the investigational plan to protect the life or physical well-being of a subject in an emergency. Such notice shall be given as soon as possible, but in no event later than 5 working days after the emergency use occurred. 21 CFR 812.35(a)(2); 812.150(a)(4). |
Step 7: Convened IRB reviews follow-up report of the emergency use.
- The IRB will review whether the situation satisfied the emergency use criteria, whether the physician followed reasonable patient protection measures under the circumstances, the patient outcome information, and whether future uses of the investigational test article are anticipated such that an application should be submitted to the IRB.
- Subsequent emergency uses of the investigational test article for the same indication should not occur unless the physician or another Seattle Children’s provider obtains FDA and IRB approval for the drug/biologic/device and its use. (FDA acknowledges that it would be inappropriate to deny emergency treatment to a second patient if the only obstacle is that the IRB has not had sufficient time to convene a meeting to review the application.)
- If an IND or IDE application for subsequent use has been filed with FDA and FDA disapproves the application, the drug/biologic/device may not be used even if the circumstances constituting an emergency exist.
Step 8: Physician/investigator receives IRB correspondence.
The IRB outcome letter will either confirm that all regulatory requirements have been satisfied, or will highlight deficiencies and request additional actions.
Emergency Use Report Form
The Emergency Use Report Form (HRP-2141 in the Click Library) is used by the treating physician as follows:
- Form and contact: Complete parts A and B of the form to confirm that an emergency use is appropriate.
- Email the form to the IRB chair or designee (per the Investigator Manual), if possible, so the IRB representative can reference the form.
- Call the paging operator at 206-987-2131 and request the IRB chair or designee to discuss the emergency use.
- Follow-up: Within five working days of initiating the emergency use, complete the remainder of the form and submit it to the IRB for review.
- If you cannot access the document in Click IRB, download the Emergency Use Report Form (DOC).
Emergency Use Consent Form Template
Seattle Children's Emergency Use Consent Form Template (HRP-506 in the Click Library) may be used to obtain consent from the patient (or their legally authorized representative), or the treating physician may use a form supplied by the manufacturer.
The IRB does not need to review or approve an emergency use consent form prior to using it with a patient or their representative.
- If you cannot access the document in Click IRB, download the Emergency Use Consent Form Template (DOCX).